PDGFRA

Summary

PDGFRA (platelet derived growth factor receptor alpha, HGNC:8803) is a protein-coding gene on chromosome 4q12, encoding Platelet-derived growth factor receptor alpha (P16234). Tyrosine-protein kinase that acts as a cell-surface receptor for PDGFA, PDGFB and PDGFC and plays an essential role in the regulation of embryonic development, cell proliferation, survival and chemotaxis. In precision oncology, PDGFRA D842V confers sensitivity to Imatinib in Gastrointestinal Stromal Tumor (CIViC Level A); 36 further curated variant–drug associations are listed below.

This gene encodes a cell surface tyrosine kinase receptor for members of the platelet-derived growth factor family. These growth factors are mitogens for cells of mesenchymal origin. The identity of the growth factor bound to a receptor monomer determines whether the functional receptor is a homodimer or a heterodimer, composed of both platelet-derived growth factor receptor alpha and beta polypeptides. Studies suggest that this gene plays a role in organ development, wound healing, and tumor progression. Mutations in this gene have been associated with idiopathic hypereosinophilic syndrome, somatic and familial gastrointestinal stromal tumors, and a variety of other cancers.

Source: NCBI Gene 5156 — RefSeq curated summary.

At a glance

• Gene–disease (curated): gastrointestinal stromal tumor (Definitive, ClinGen) — +4 more curated relationships
• GWAS associations: 34 (top 3% of genes corpus-wide)
• Clinical variants (ClinVar): 3,990 total — 7 pathogenic (top 1% of genes corpus-wide)
• Regional missense constraint (gnomAD): 1 of 5 regions constrained (≈9% of expected missense variation)
• GTEx eGene: eQTL in 9 tissues, sQTL in 15 (q < 0.05)
• Phenotypes (HPO): 55
• Drug target: yes — 6 curated mechanism-of-action drugs, 24 via a complex or family; 77 molecules with ChEMBL bioactivity (top 1% of genes corpus-wide)
• Approved drugs (ChEMBL mechanism of action): 4 — Avapritinib, Olaratumab, Ripretinib, +1 more
• Approved drugs acting on a complex or family including it: 6 — Becaplermin, Midostaurin, Nintedanib Esylate, +3 more (Platelet-derived growth factor receptor)
• Precision-oncology evidence (CIViC): 37 curated variant–drug associations
• Cancer driver (intOGen): activating (oncogene-like) across 7 cancer types (inferred from mutation patterns)
• Dosage sensitivity (ClinGen): haploinsufficiency no evidence, triplosensitivity no evidence
• Constraint (gnomAD): pLI 1.00 (loss-of-function intolerant), LOEUF 0.26 (highly constrained)
• MANE Select transcript: NM_006206
• Interaction hub: 4,474 STRING partners (top 1% of genes corpus-wide)

Identifiers

Gene identifiers

Field	Value
HGNC ID	HGNC:8803
Approved symbol	PDGFRA
Name	platelet derived growth factor receptor alpha
Location	4q12
Locus type	gene with protein product
Status	Approved
Aliases	CD140a, PDGFR2, GAS9
Ensembl gene	ENSG00000134853
Ensembl biotype	protein_coding
OMIM	173490
Entrez	5156

Gene structure

Transcript identifiers

Ensembl transcripts: 30 — 26 protein_coding, 3 retained_intron, 1 nonsense_mediated_decay

MANE Select: ENST00000257290

Transcript support level (Ensembl TSL; 1 = fully mRNA-supported): TSL 1: 5, TSL 3: 3, TSL 4: 2

ENST00000257290, ENST00000461294, ENST00000503856, ENST00000504461, ENST00000507536, ENST00000508170, ENST00000509092, ENST00000509490, ENST00000512143, ENST00000512522, ENST00000870889, ENST00000870890, ENST00000958745, ENST00000958746, ENST00000958747, ENST00000958748, ENST00000958749, ENST00000958750, ENST00000958751, ENST00000958752, ENST00000958753, ENST00000958754, ENST00000958755, ENST00001110484, ENST00001110485, ENST00001113065, ENST00001113066, ENST00001113067, ENST00001123502, ENST00001137597

RefSeq mRNA: 5 — MANE Select: NM_006206 NM_001347827, NM_001347828, NM_001347829, NM_001347830, NM_006206

CCDS: CCDS3495

Canonical transcript exons

ENST00000257290 — 23 exons

Exon	Start	End
ENSE00001226318	54295125	54298245
ENSE00002028061	54229293	54229415
ENSE00003462897	54289009	54289114
ENSE00003464046	54278362	54278515
ENSE00003471835	54290313	54290554
ENSE00003482129	54258757	54258817
ENSE00003483772	54285371	54285486
ENSE00003529069	54277896	54278006
ENSE00003547009	54267552	54267741
ENSE00003548932	54274531	54274625
ENSE00003566972	54267289	54267460
ENSE00003570052	54285841	54285963
ENSE00003571737	54274841	54274973
ENSE00003580939	54264919	54265049
ENSE00003584929	54273537	54273730
ENSE00003586981	54288799	54288898
ENSE00003626526	54272394	54272520
ENSE00003634368	54263667	54263927
ENSE00003647873	54280316	54280482
ENSE00003673604	54277388	54277492
ENSE00003674470	54270633	54270748
ENSE00003681641	54287430	54287541
ENSE00003785223	54261095	54261412

Expression profiles

Bgee: expression breadth ubiquitous, 289 present calls, max score 99.38.

FANTOM5 (CAGE): breadth ubiquitous, TPM avg 34.1851 / max 610.7707, expressed in 1119 samples.

Highest expression (Bgee): tibia, decidua, synovial joint, pericardium, lower lobe of lung.

Genetic regulation across tissues (GTEx eQTL / sQTL)

PDGFRA is an eGene in GTEx v10 — its expression is genetically regulated (eQTL) in 9 tissues and its splicing (sQTL) in 15 (q < 0.05). Strongest associations first; slope = normalized effect of the lead variant’s alternative allele.

showing 20 of 24 significant tissue × QTL associations:

Tissue	QTL	q-value	Slope	Lead variant
Adipose Subcutaneous	sqtl	8.4e-13	-0.61	4:54281602:C:T
Cells Cultured fibroblasts	sqtl	1.4e-12	-0.29	4:54281602:C:T
Ovary	sqtl	1.2e-08	-0.57	4:54281602:C:T
Nerve Tibial	sqtl	3.8e-07	-0.45	4:54281602:C:T
Skin Sun Exposed Lower leg	sqtl	6.5e-07	-0.49	4:54281602:C:T
Artery Aorta	sqtl	6.7e-07	-0.51	4:54281602:C:T
Colon Transverse	sqtl	4.4e-06	-0.49	4:54281602:C:T
Esophagus Muscularis	sqtl	4.4e-06	-0.48	4:54281602:C:T
Artery Tibial	sqtl	2.4e-05	-0.46	4:54292430:A:G
Cells Cultured fibroblasts	eqtl	4.6e-05	+0.10	4:54211207:T:C
Esophagus Mucosa	eqtl	1.2e-04	+0.23	4:53597475:T:A
Colon Sigmoid	sqtl	2.9e-04	-0.52	4:54281602:C:T
Adipose Visceral Omentum	sqtl	2.0e-03	-0.48	4:54281602:C:T
Heart Left Ventricle	eqtl	2.1e-03	+0.17	4:54434753:CG:C
Lung	sqtl	2.6e-03	-0.31	4:54281602:C:T
Esophagus Muscularis	eqtl	3.5e-03	-0.63	4:53560896:C:T
Spleen	sqtl	6.9e-03	+0.62	4:54292430:A:G
Artery Tibial	eqtl	1.6e-02	+0.11	4:54170242:T:C
Nerve Tibial	eqtl	2.4e-02	-0.25	4:53533457:G:A
Spleen	eqtl	2.5e-02	-0.35	4:55161678:T:C

FANTOM5 promoters

showing 10 of 13 alternative TSS:

Promoter ID	TPM avg	Samples expressed
47637	21.0494	875
47636	6.3735	758
47644	2.3539	725
47638	1.1781	491
47635	0.5677	339
47640	0.5548	242
47643	0.5311	320
47646	0.4616	155
47641	0.3160	195
47647	0.2525	109

Top tissues by expression

showing 100 of 296 tissues by Bgee expression score (0-100, higher = more expressed):

Tissue	Anatomy ID	Expression score	Quality
tibia	UBERON:0000979	99.38	gold quality
decidua	UBERON:0002450	99.29	gold quality
synovial joint	UBERON:0002217	99.19	gold quality
pericardium	UBERON:0002407	99.04	gold quality
lower lobe of lung	UBERON:0008949	98.85	gold quality
pylorus	UBERON:0001166	98.75	gold quality
stromal cell of endometrium	CL:0002255	98.74	gold quality
parietal pleura	UBERON:0002400	98.67	gold quality
caput epididymis	UBERON:0004358	98.58	gold quality
left ovary	UBERON:0002119	98.56	gold quality
pleura	UBERON:0000977	98.43	gold quality
cauda epididymis	UBERON:0004360	98.42	gold quality
right ovary	UBERON:0002118	98.29	gold quality
layer of synovial tissue	UBERON:0007616	98.11	gold quality
visceral pleura	UBERON:0002401	98.06	gold quality
germinal epithelium of ovary	UBERON:0001304	97.97	gold quality
urethra	UBERON:0000057	97.96	gold quality
ovary	UBERON:0000992	97.96	gold quality
mucosa of urinary bladder	UBERON:0001259	97.96	gold quality
mammary duct	UBERON:0001765	97.90	gold quality
skin of hip	UBERON:0001554	97.84	gold quality
superficial temporal artery	UBERON:0001614	97.82	gold quality
calcaneal tendon	UBERON:0003701	97.81	gold quality
tendon of biceps brachii	UBERON:0008188	97.78	gold quality
epithelium of mammary gland	UBERON:0003244	97.69	gold quality
heart right ventricle	UBERON:0002080	97.56	gold quality
cardia of stomach	UBERON:0001162	97.54	gold quality
corpus epididymis	UBERON:0004359	97.47	gold quality
jejunal mucosa	UBERON:0000399	97.30	gold quality
tendon	UBERON:0000043	97.28	gold quality
colonic epithelium	UBERON:0000397	97.21	gold quality
mucosa of sigmoid colon	UBERON:0004993	97.18	gold quality
periodontal ligament	UBERON:0008266	96.95	gold quality
vena cava	UBERON:0004087	96.88	gold quality
upper leg skin	UBERON:0004262	96.86	gold quality
jejunum	UBERON:0002115	96.71	gold quality
cardiac muscle of right atrium	UBERON:0003379	96.71	gold quality
thoracic mammary gland	UBERON:0005200	96.68	gold quality
mammary gland	UBERON:0001911	96.67	gold quality
trabecular bone tissue	UBERON:0002483	96.66	gold quality
urinary bladder	UBERON:0001255	96.65	gold quality
endometrium	UBERON:0001295	96.55	gold quality
myometrium	UBERON:0001296	96.54	gold quality
myocardium	UBERON:0002349	96.53	gold quality
right lung	UBERON:0002167	96.52	gold quality
diaphragm	UBERON:0001103	96.51	gold quality
left uterine tube	UBERON:0001303	96.42	gold quality
penis	UBERON:0000989	96.36	gold quality
placenta	UBERON:0001987	96.27	gold quality
olfactory bulb	UBERON:0002264	96.26	gold quality
mucosa of stomach	UBERON:0001199	96.20	gold quality
seminal vesicle	UBERON:0000998	95.92	gold quality
uterus	UBERON:0000995	95.72	gold quality
female reproductive system	UBERON:0000474	95.70	gold quality
colonic mucosa	UBERON:0000317	95.55	gold quality
descending thoracic aorta	UBERON:0002345	95.55	gold quality
caecum	UBERON:0001153	95.32	gold quality
oral cavity	UBERON:0000167	95.26	gold quality
lung	UBERON:0002048	95.22	gold quality
mammalian vulva	UBERON:0000997	95.19	gold quality
smooth muscle tissue	UBERON:0001135	95.15	gold quality
endocervix	UBERON:0000458	95.08	gold quality
cartilage tissue	UBERON:0002418	95.03	gold quality
upper arm skin	UBERON:0004263	95.01	gold quality
mucosa of paranasal sinus	UBERON:0005030	94.97	gold quality
left ventricle myocardium	UBERON:0006566	94.96	gold quality
body of uterus	UBERON:0009853	94.72	gold quality
thoracic aorta	UBERON:0001515	94.62	gold quality
ascending aorta	UBERON:0001496	94.57	gold quality
right coronary artery	UBERON:0001625	94.56	gold quality
connective tissue	UBERON:0002384	94.52	gold quality
vermiform appendix	UBERON:0001154	94.43	gold quality
adipose tissue	UBERON:0001013	94.40	gold quality
gluteal muscle	UBERON:0002000	94.39	gold quality
saphenous vein	UBERON:0007318	94.31	gold quality
subthalamic nucleus	UBERON:0001906	94.13	gold quality
gall bladder	UBERON:0002110	94.09	gold quality
skeletal muscle tissue of biceps brachii	UBERON:0004502	94.07	gold quality
adipose tissue of abdominal region	UBERON:0007808	93.97	gold quality
inferior vagus X ganglion	UBERON:0005363	93.91	gold quality
body of tongue	UBERON:0011876	93.91	gold quality
coronary artery	UBERON:0001621	93.83	gold quality
peritoneum	UBERON:0002358	93.79	gold quality
upper lobe of lung	UBERON:0008948	93.79	gold quality
omental fat pad	UBERON:0010414	93.78	gold quality
sigmoid colon	UBERON:0001159	93.75	gold quality
adult organism	UBERON:0007023	93.68	gold quality
left coronary artery	UBERON:0001626	93.65	gold quality
fundus of stomach	UBERON:0001160	93.45	gold quality
upper lobe of left lung	UBERON:0008952	93.45	gold quality
duodenum	UBERON:0002114	93.41	gold quality
nipple	UBERON:0002030	93.38	gold quality
subcutaneous adipose tissue	UBERON:0002190	93.28	gold quality
vagina	UBERON:0000996	93.09	gold quality
rectum	UBERON:0001052	93.07	gold quality
corpus callosum	UBERON:0002336	93.04	gold quality
muscle layer of sigmoid colon	UBERON:0035805	93.02	gold quality
spleen	UBERON:0002106	92.95	gold quality
dorsal root ganglion	UBERON:0000044	92.91	gold quality
metanephros cortex	UBERON:0010533	92.87	gold quality

Single-cell (SCXA)

Detected in 19 experiment(s), a significant marker in 16.

Experiment	Marker?	Max mean expression
E-MTAB-9435	yes	6405.27
E-MTAB-9906	yes	1346.80
E-MTAB-8410	yes	1153.67
E-GEOD-84465	yes	1100.06
E-HCAD-56	yes	469.12
E-GEOD-109979	yes	294.20
E-MTAB-10287	yes	120.59
E-HCAD-10	yes	66.64
E-GEOD-135922	yes	59.67
E-HCAD-35	yes	55.65
E-GEOD-134144	yes	38.69
E-MTAB-6678	yes	28.01
E-CURD-46	yes	22.15
E-MTAB-9543	yes	20.19
E-MTAB-9388	yes	16.03

HPA expression

RNA tissue specificity: Tissue enhanced

showing 60 of 77 HPA tissues / cells by nTPM:

Tissue / cell	Axis	nTPM	Protein (IHC)
Ovary	tissue	188.3	medium
Urinary bladder	tissue	59.6	not detected
Placenta	tissue	58.9	low
Choroid plexus	tissue	54.4
Smooth muscle	tissue	44.8	not detected
Endometrium 1	tissue	41.0	low
Adipose tissue	tissue	40.8	not detected
Tongue	tissue	38.9
Cervix	tissue	38.3	not detected
Appendix	tissue	38.0	high
Breast	tissue	37.4	medium
Spinal cord	tissue	37.4
Vagina	tissue	36.8	not detected
Hippocampus	tissue	36.8	not detected
Gallbladder	tissue	36.0	not detected
Spleen	tissue	35.5	not detected
Rectum	tissue	33.0	high
Amygdala	tissue	32.6
Fallopian tube	tissue	32.1	not detected
Caudate putamen	tissue	31.9
Nucleus accumbens	tissue	31.3
Thalamus	tissue	29.8
Locus coeruleus	tissue	28.5
Hypothalamus	tissue	27.9
Hippocampal formation	tissue	27.7
Colon	tissue	27.6	low
Adrenal gland	tissue	27.3	not detected
Frontal cortex	tissue	27.2
Stomach 1	tissue	26.9	low
Substantia nigra	tissue	26.8
Midbrain	tissue	26.4
Cerebral cortex	tissue	25.6	low
Insular cortex	tissue	25.6
Septum	tissue	25.5
Corpus callosum	tissue	25.0
Kidney	tissue	24.7	medium
Medulla oblongata	tissue	24.4
Pons	tissue	24.3
Prefrontal cortex	tissue	24.1
Putamen	tissue	24.1
Periaqueductal gray	tissue	24.1
Ventral forebrain	tissue	23.5
Small intestine	tissue	23.3	not detected
Motor cortex	tissue	23.2
Retrosplenial cortex	tissue	22.5
Postcentral gyrus	tissue	22.4
Entorhinal cortex	tissue	22.0
Heart muscle	tissue	21.7	not detected
Basal ganglia	tissue	21.6	not detected
Retrosplenial and cingulate cortex	tissue	21.3
Duodenum	tissue	21.2	not detected
Superior colliculi	tissue	20.9
Esophagus	tissue	20.7	not detected
Epididymis	tissue	20.4	not detected
Temporal cortex	tissue	19.9
Lung	tissue	19.1	not detected
Seminal vesicle	tissue	17.8	not detected
Liver	tissue	14.9	not detected
Occipital cortex	tissue	14.9
Somatosensory cortex	tissue	14.8

Brain sub-regions (HPA Brain Atlas): detected in 191; highest nTPM — Cerebellar white matter (54.5), Nucleus ambiguus (50.5), Lateral funiculus (white matter) (50.1), Nucleus raphe obscurus (49.7), Posteroventral cochlear nucleus (45.7).

Regulation

Is transcription factor: no

Upstream regulators (CollecTRI, top): ASCL1, ATF4, CEBPB, CEBPD, CEBPG, CREB1, E2F1, FOS, FOXO1, GATA4, GATA6, GLI1, GLI2, HOXC6, MIXL1, NFKB, PAX1, PAX3, POU5F1, PPARG, SOX10, SP1, SP3, YY1, ZNF148, ZNF354C

miRNA regulators (miRDB)

Targeting PDGFRA by miRDB confidence (max_score; target_count = how many genes the miRNA targets in total — lower means more specific).

showing 30 of 192 miRNAs:

miRNA	Max score	Avg score	miRNA target_count
HSA-MIR-8485	100.00	77.57	4731
HSA-MIR-4262	100.00	73.26	3931
HSA-MIR-3613-3P	100.00	76.36	7965
HSA-MIR-4795-3P	100.00	74.62	4024
HSA-MIR-196A-5P	100.00	68.16	684
HSA-MIR-196B-5P	100.00	68.16	681
HSA-MIR-29A-3P	100.00	73.11	1835
HSA-MIR-29B-3P	100.00	73.18	1833
HSA-MIR-29C-3P	100.00	73.15	1833
HSA-MIR-126-5P	100.00	72.71	3180
HSA-MIR-450A-1-3P	100.00	69.33	1837
HSA-MIR-432-3P	100.00	67.86	705
HSA-MIR-4713-3P	100.00	65.92	505
HSA-MIR-340-5P	100.00	72.50	4437
HSA-MIR-6833-3P	100.00	70.63	3197
HSA-MIR-5011-5P	100.00	83.46	5820
HSA-MIR-4481	100.00	66.42	1669
HSA-MIR-190A-3P	100.00	80.35	5520
HSA-MIR-4768-5P	100.00	69.49	2861
HSA-MIR-33A-5P	99.99	68.62	1055
HSA-MIR-33B-5P	99.99	68.58	1062
HSA-MIR-34A-5P	99.99	71.21	1784
HSA-MIR-449A	99.99	71.05	1776
HSA-MIR-181A-5P	99.99	72.96	2995
HSA-MIR-181B-5P	99.99	72.97	2996
HSA-MIR-181C-5P	99.99	72.95	2996
HSA-MIR-181D-5P	99.99	73.04	2997
HSA-MIR-548C-3P	99.99	74.01	7587
HSA-MIR-3617-3P	99.98	67.86	918
HSA-MIR-5696	99.98	72.36	4487

Functional genomics

gnomAD constraint: pLI 1, LOEUF 0.264, missense Z 3.42 — population loss-of-function intolerance (pLI ≥ 0.9 = LoF-intolerant; lower LOEUF = more constrained). gnomAD

Regional missense constraint (gnomAD)

ENST00000257290 splits into 5 regions with different missense tolerance; 1 is missense-constrained (≈9% of the expected missense variation). Missense variants in a constrained region (low observed/expected) are more likely to be pathogenic. gnomAD

Region (GRCh38)	Missense obs / exp	o/e	Constrained
4:54,258,769–54,274,904	575 / 701.8	0.82	no
4:54,274,905–54,278,378	61 / 125.3	0.49	no
4:54,278,379–54,285,444	128 / 147.6	0.87	no
4:54,285,445–54,288,820	40 / 127.4	0.31	yes (p 9.7e-15)
4:54,288,821–54,295,269	204 / 243.0	0.84	no
ClinGen dosage: haploinsufficiency 0 (no evidence), triplosensitivity 0 (no evidence). ClinGen Gene Dosage Map

Literature-anchored findings (GeneRIF, showing 40)

• high expression associated with long survival in patients with grade 2 astrocytomas and oligoastrocytomas (PMID:12023424)
• PDGFRA is endogenenously activated in medulloblastoma tumor cell lines (PMID:12176024)
• KIT and PDGFRA mutations appear to be alternative and mutually exclusive oncogenic mechanisms in gastrointestinal stromal tumors (PMID:12522257)
• The hypereosinophilic syndrome may result from a novel fusion tyrosine kinase - FIP1L1-PDGFRalpha - that is a consequence of an interstitial chromosomal deletion. (PMID:12660384)
• observations suggest that the FIP1L1-PDGFRA rearrangement occurs in an early hematopoietic progenitor and suggests that the molecular pathogenesis for a subset of SMCD patients is similar to that of HES (PMID:12842979)
• results indicate that the fusion of FIP1L1 to PDGFRA occurs rarely in leukemia cell lines (PMID:14630792)
• Result from our case-control study demonstrated that the frequencies of haplotypes with low transcription activity were significantly higher in NTD mothers than that observed in control mothers (odds ratio=2.2, 95% CI=1.0-4.6). (PMID:14741194)
• results confirm the high frequency of BCR-ABL kinase domain mutations in patients with secondary resistance to imatinib and exclude mutations of the activation loops of KIT, PDGFRA and PDGFRB as causes of resistance in patients without ABL mutations (PMID:14745431)
• PDGFRa expression is regulated by C-EBP (PMID:14766209)
• PDGFRA mutations were found in 7 (25.0%)gastrointestinal stromal tumors (GISTs) , carrying either D842 V mutations or exon 18 deletions. (PMID:15154005)
• FIP1L1-PDGFRA is a relatively infrequent but treatment-relevant mutation in primary eosinophilia that is indicative of an underlying systemic mastocytosis. (PMID:15284118)
• gene expression profiling of 26 gastrointesstinsal stromsal tumors with known KIT and PDGFRA mutational status (PMID:15326474)
• These results suggest that myxoid epithelioid GISTs are a distinct subtype of GISTs that are closely correlated with the PDGFRA gene mutation (PMID:15492989)
• link between single nucleotide polymorphisms (SNPs) within the PDGFRalpha gene promoter and the occurrence of brain tumours (PMID:15635072)
• The Receptor, PDGF alpha (PDGFRA)is implicated in the pathogenesis of AML, and the mechanisms of cytogenetic progression and oncoprotein-driven function may be similar in AML expressing oncogenic forms of PDGFRA. (PMID:15674355)
• High sensitivity of the imatinib-resistant KIT -T670I and KIT -V654A and of PDGFRA -D842V mutants to PKC412 in gastrointestinal stromal tumors. (PMID:15685537)
• study demonstrates that platelet-derived growth factor receptor alpha and beta, and c-kit protein are expressed in a high percentage of epithelial ovarian cancers (PMID:15791568)
• Western blot showed a high amount of PDGFRalpha and beta proteins in primary stromal cells that could not be detected in prostatic cancer cells. (PMID:15862965)
• In human ASMCs, IFNgamma again stimulated a transient STAT1-binding to the PDGF-Ralpha promoter. (PMID:15871904)
• single example of Gastrointestinal Stromal Tumor with activating mutation and immunoreactivity without CD117 positivity (PMID:15894928)
• The point mutations of c-kit and platelet- derived growth factor receptor alpha genes may play a limited role in the tumorigenesis of type 1 neurofibromatosis-associated gastrointestinal stromal tumors. (PMID:15897742)
• PDGFRA mutations are associated with gastrointestinal stromal tumors (PMID:15928335)
• The hypereosinophilic syndrome may result from a novel fusion tyrosine kinase - FIP1L1-PDGFRalpha - that is a consequence of an interstitial chromosomal deletion. (PMID:16116920)
• The current identification of cis-acting elements in the PDGFRA promoter and the transcription factors that bind them, provides a new strategy for the identification of genes that are potentially involved in neural tube defects. (PMID:16126374)
• An intronic insertion IVS17-50insA at exon 18 in all sequenced cases. None of these genetic alterations was correlated with PDGFR-alpha expression in breast cancer. (PMID:16168125)
• Two-basepair deletion in the C terminus of PDGFRA (exon 23) found in glioblastomas. (PMID:16186508)
• PDGFRA P1 promoter region haplotypes do not have a major role in the development of spina bifida meningomyelocele . (PMID:16283668)
• data describe percentage of bone marrow cells expressing receptors for interleukin-1, platelet-derived growth factor, fibroblast growth factor, transforming growth factor-beta, epidermal growth factor and c-Fos and c-Myc in untreated lung & breast cancer (PMID:16305343)
• Specific PDGFR inhibition by siRNA or a neutralizing antibody to the receptor inhibited PDGF-stimulated receptor activation and cell proliferation, suggesting that receptor targeting has a role in ovarian cancer treatment (PMID:16331269)
• describes frequence mutation of PDGFRA in malignant periphaerl nerve sheath tumors often associated with coamplification of KIT (PMID:16357008)
• activating mutations of PDGFR-alpha, c-kit and B-RAF are absent in gliosarcomas (PMID:16373964)
• Demonstrates the utility of screening for PDGFRA kinase domain overexpression in patients with IHES and has identified KIF5B as a third PDGFRA fusion partner in chronic myeloproliferative disorders. (PMID:16498388)
• Fusion protein is detected in hypereosinophilic syndrome and chronic eosinophilic leukemia. (PMID:16502585)
• The most intense expression of PDGFRalpha was observed in fibroblasts in the lung carcinoma outer rim. (PMID:16675559)
• Six of the 20 gastrointestinal stomach tumors lacking KIT mutations had mutations in exon 18 of PDGFRA, and 1 had a mutation in exon 12 in this receptor. (PMID:16685437)
• FIP1L1-PDGFRalpha activation requires disruption of the juxtamembrane domain of PDGFRalpha and is FIP1L1-independent (PMID:16690743)
• Gastrointestinal stromal tumours (GIST) with platelet-derived growth factor receptor mutations often have reduced expression of the KIT protein in immunohistochemistry (IHC), suggesting that IHC may be potentially useful in identification of such GISTs. (PMID:16785193)
• 77% of the examined primary central nervous system lymphoma specimens showed expression of PDGFRalpha. Tumours expressing survivin frequently co-expressed PDGFRalpha. (PMID:16850112)
• PDGFRalpha mutations have been identified as alternative oncogenic mechanism in gastrointestinal stromal tumors. (PMID:16892550)
• In midgut carcinoid tumor stroma PDGFRalpha was expressed in 35%, PDGFRbeta in 94% and EGFR in 9%. (PMID:17047316)

Cross-species orthologs

3 orthologs

Organism	Symbol	Gene ID
danio_rerio	pdgfra	ENSDARG00000070494
mus_musculus	Pdgfra	ENSMUSG00000029231
rattus_norvegicus	Pdgfra	ENSRNOG00000002244

Paralogs (53): INSRR (ENSG00000027644), MUSK (ENSG00000030304), FLT4 (ENSG00000037280), EPHA3 (ENSG00000044524), ROS1 (ENSG00000047936), LTK (ENSG00000062524), ERBB3 (ENSG00000065361), TIE1 (ENSG00000066056), FGFR2 (ENSG00000066468), FGFR3 (ENSG00000068078), EPHA8 (ENSG00000070886), FGFR1 (ENSG00000077782), EPHA6 (ENSG00000080224), TYRO3 (ENSG00000092445), FLT1 (ENSG00000102755), MET (ENSG00000105976), EPHB6 (ENSG00000106123), PDGFRB (ENSG00000113721), EPHA4 (ENSG00000116106), TEK (ENSG00000120156), FLT3 (ENSG00000122025), KDR (ENSG00000128052), EPHB2 (ENSG00000133216), EPHA7 (ENSG00000135333), IGF1R (ENSG00000140443), NTRK3 (ENSG00000140538), ERBB2 (ENSG00000141736), EPHA2 (ENSG00000142627), EPHA5 (ENSG00000145242), EGFR (ENSG00000146648), EPHA1 (ENSG00000146904), NTRK2 (ENSG00000148053), MERTK (ENSG00000153208), EPHB1 (ENSG00000154928), KIT (ENSG00000157404), FGFR4 (ENSG00000160867), DDR2 (ENSG00000162733), RYK (ENSG00000163785), MST1R (ENSG00000164078), LMTK2 (ENSG00000164715), RET (ENSG00000165731), AXL (ENSG00000167601), ROR2 (ENSG00000169071), ALK (ENSG00000171094), INSR (ENSG00000171105), ERBB4 (ENSG00000178568), CSF1R (ENSG00000182578), EPHB3 (ENSG00000182580), EPHA10 (ENSG00000183317), ROR1 (ENSG00000185483), EPHB4 (ENSG00000196411), NTRK1 (ENSG00000198400), DDR1 (ENSG00000204580)

Mouse-model phenotypes

Observed phenotypes of the mouse ortholog (MGI:97530) from MGI (Alliance of Genome Resources) — knockout/mutant phenotypes, ranked by annotation count. 147 distinct phenotype terms.

Mouse phenotype (MP)	Records
midline facial cleft	3
abnormal nasal bone morphology	2
abnormal frontal bone morphology	2
rib fusion	2
abnormal sternum morphology	2
abnormal testis morphology	2
small testis	2
pulmonary hypoplasia	2
failure of somite differentiation	2
edema	2
hemorrhage	2
persistent truncus arteriosus	2
spina bifida	2
ventricular septal defect	2
embryonic lethality during organogenesis, incomplete penetrance	2
lethality throughout fetal growth and development, incomplete penetrance	2
impaired ossification of basisphenoid bone	1
abnormal parietal bone morphology	1
cleft palate	1
abnormal scapula morphology	1
abnormal rib morphology	1
rib bifurcation	1
absent sternum	1
decreased circulating HDL cholesterol level	1
increased cell proliferation	1

+122 more (showing top 25).

Additional cross-species homologs

Sequence homologs in species beyond Ensembl Compara’s model organisms, by Diamond local protein alignment (% sequence identity; best hit per species). Lower-identity rows are more distant homologs. The matched accessions’ gene symbols aren’t in biobtree yet, so each row shows the UniProt accession.

Organism	UniProt	Identity	Method
Pan troglodytes	Q4VSN5	99.5%	Diamond
Bos taurus	P43481	99.4%	Diamond
Capra hircus	Q28317	99.4%	Diamond
Felis catus	Q28889	99.2%	Diamond
Macaca mulatta	Q20CR4	98.9%	Diamond
Canis lupus familiaris	O97799	98.4%	Diamond
Sus scrofa	Q2HWD6	98.1%	Diamond
Callithrix jacchus	Q76II0	94.6%	Diamond
Gallus gallus	P18461	92.7%	Diamond
Notophthalmus viridescens	Q91147	87.9%	Diamond
Pleurodeles waltl	Q91285	87.8%	Diamond
Coturnix coturnix	Q90330	81.0%	Diamond
Xenopus laevis	P22182	80.6%	Diamond
Takifugu rubripes	Q8AXC7	73.2%	Diamond
Caenorhabditis elegans	Q9XTC6	68.9%	Diamond
Ciona intestinalis	Q4H3K6	60.1%	Diamond
Drosophila melanogaster	Q09147	55.0%	Diamond
Dugesia japonica	Q8MY86	51.0%	Diamond
Hydra vulgaris	Q86PM4	50.2%	Diamond
Strongylocentrotus purpuratus	Q26614	46.4%	Diamond
Bombyx mori	D2IYS2	44.0%	Diamond
Feline sarcoma virus (STRAIN HARDY-ZUCKERMAN 4)	P04048	99.2%	Diamond
Feline sarcoma virus (STRAIN MCDONOUGH)	P00545	99.1%	Diamond

Genomic neighbors (locus context)

Genes flanking this one on the chromosome (NCBI/Entrez gene neighborhood). Positional context only — some neighbors share regulatory elements (bidirectional promoters, antisense/overlapping transcripts), but chromosomal adjacency is not itself a functional relationship (see Interactions for that).

Symbol	Type	Entrez
LINC02260	ncRNA	339978
KIT	protein-coding	3815
LINC02283	ncRNA	105377656
GSX2	protein-coding	170825

Protein

Protein identifiers

Platelet-derived growth factor receptor alpha — P16234 (reviewed: P16234)

Alternative names: Alpha platelet-derived growth factor receptor, Alpha-type platelet-derived growth factor receptor, CD140 antigen-like family member A, CD140a antigen, Platelet-derived growth factor alpha receptor, Platelet-derived growth factor receptor 2

All UniProt accessions (5): D6RDX0, D6RG11, D6RIG5, D6RJH0, P16234

UniProt curated annotations — full annotation on UniProt →

Function. Tyrosine-protein kinase that acts as a cell-surface receptor for PDGFA, PDGFB and PDGFC and plays an essential role in the regulation of embryonic development, cell proliferation, survival and chemotaxis. Depending on the context, promotes or inhibits cell proliferation and cell migration. Plays an important role in the differentiation of bone marrow-derived mesenchymal stem cells. Required for normal skeleton development and cephalic closure during embryonic development. Required for normal development of the mucosa lining the gastrointestinal tract, and for recruitment of mesenchymal cells and normal development of intestinal villi. Plays a role in cell migration and chemotaxis in wound healing. Plays a role in platelet activation, secretion of agonists from platelet granules, and in thrombin-induced platelet aggregation. Binding of its cognate ligands - homodimeric PDGFA, homodimeric PDGFB, heterodimers formed by PDGFA and PDGFB or homodimeric PDGFC -leads to the activation of several signaling cascades; the response depends on the nature of the bound ligand and is modulated by the formation of heterodimers between PDGFRA and PDGFRB. Phosphorylates PIK3R1, PLCG1, and PTPN11. Activation of PLCG1 leads to the production of the cellular signaling molecules diacylglycerol and inositol 1,4,5-trisphosphate, mobilization of cytosolic Ca(2+) and the activation of protein kinase C. Phosphorylates PIK3R1, the regulatory subunit of phosphatidylinositol 3-kinase, and thereby mediates activation of the AKT1 signaling pathway. Mediates activation of HRAS and of the MAP kinases MAPK1/ERK2 and/or MAPK3/ERK1. Promotes activation of STAT family members STAT1, STAT3 and STAT5A and/or STAT5B. Receptor signaling is down-regulated by protein phosphatases that dephosphorylate the receptor and its down-stream effectors, and by rapid internalization of the activated receptor.

Subunit / interactions. Interacts with homodimeric PDGFA, PDGFB and PDGFC, and with heterodimers formed by PDGFA and PDGFB. Monomer in the absence of bound ligand. Interaction with dimeric PDGFA, PDGFB and/or PDGFC leads to receptor dimerization, where both PDGFRA homodimers and heterodimers with PDGFRB are observed. Interacts (tyrosine phosphorylated) with SHB (via SH2 domain). Interacts (tyrosine phosphorylated) with SHF (via SH2 domain). Interacts (tyrosine phosphorylated) with SRC (via SH2 domain). Interacts (tyrosine phosphorylated) with PIK3R1. Interacts (tyrosine phosphorylated) with PLCG1 (via SH2 domain). Interacts (tyrosine phosphorylated) with CRK, GRB2 and GRB7. Interacts with CD248; this interaction promotes PDGF receptor signaling pathway. (Microbial infection) Interacts with human cytomegalovirus/HHV-5 envelope glycoprotein B/gB. Also interacts with the trimeric complex gH-gL-gO. Trimer-PDGFRA interaction has an inhibitory effect on PDGFRA signaling.

Subcellular location. Cell membrane. Cell projection. Cilium. Golgi apparatus.

Tissue specificity. Detected in platelets (at protein level). Widely expressed. Detected in brain, fibroblasts, smooth muscle, heart, and embryo. Expressed in primary and metastatic colon tumors and in normal colon tissue.

Post-translational modifications. N-glycosylated. Ubiquitinated, leading to its internalization and degradation. Autophosphorylated on tyrosine residues upon ligand binding. Autophosphorylation occurs in trans, i.e. one subunit of the dimeric receptor phosphorylates tyrosine residues on the other subunit. Phosphorylation at Tyr-731 and Tyr-742 is important for interaction with PIK3R1. Phosphorylation at Tyr-720 and Tyr-754 is important for interaction with PTPN11. Phosphorylation at Tyr-762 is important for interaction with CRK. Phosphorylation at Tyr-572 and Tyr-574 is important for interaction with SRC and SRC family members. Phosphorylation at Tyr-988 and Tyr-1018 is important for interaction with PLCG1.

Disease relevance. A chromosomal aberration involving PDGFRA is found in some cases of hypereosinophilic syndrome. Interstitial chromosomal deletion del(4)(q12q12) causes the fusion of FIP1L1 and PDGFRA (FIP1L1-PDGFRA). Mutations that cause overexpression and/or constitutive activation of PDGFRA may be a cause of hypereosinophilic syndrome. Gastrointestinal stromal tumor (GIST) [MIM:606764] Common mesenchymal neoplasms arising in the gastrointestinal tract, most often in the stomach. They are histologically, immunohistochemically, and genetically different from typical leiomyomas, leiomyosarcomas, and schwannomas. Most GISTs are composed of a fairly uniform population of spindle-shaped cells. Some tumors are dominated by epithelioid cells or contain a mixture of spindle and epithelioid morphologies. Primary GISTs in the gastrointestinal tract commonly metastasize in the omentum and mesenteries, often as multiple nodules. However, primary tumors may also occur outside of the gastrointestinal tract, in other intra-abdominal locations, especially in the omentum and mesentery. The gene represented in this entry may be involved in disease pathogenesis. Mutations causing PDGFRA constitutive activation have been found in gastrointestinal stromal tumors lacking KIT mutations. GIST-plus syndrome (GISTPS) [MIM:175510] A disorder characterized by multiple mesenchymal tumors of the gastrointestinal tract, including gastrointestinal stromal tumor, inflammatory fibroid polyps, and fibroid tumors. Additional features are coarse facies and skin, broad hands and feet, and premature tooth loss. GISTPS is an autosomal dominant disease with incomplete penetrance. Gastrointestinal stromal tumor and inflammatory fibroid polyps may also occur in isolation. The disease is caused by variants affecting the gene represented in this entry.

Activity regulation. Present in an inactive conformation in the absence of bound ligand. Binding of PDGFA and/or PDGFB leads to dimerization and activation by autophosphorylation on tyrosine residues. Inhibited by imatinib, nilotinib and sorafenib.

Similarity. Belongs to the protein kinase superfamily. Tyr protein kinase family. CSF-1/PDGF receptor subfamily.

Isoforms (3)

UniProt ID	Names	Canonical?
P16234-1	1	yes
P16234-2	2
P16234-3	3

RefSeq proteins (5): NP_001334756, NP_001334757, NP_001334758, NP_001334759, NP_006197* (*=MANE)

Domains & families (InterPro)

ID	Name	Type
IPR000719	Prot_kinase_dom	Domain
IPR001245	Ser-Thr/Tyr_kinase_cat_dom	Domain
IPR001824	Tyr_kinase_rcpt_3_CS	Conserved_site
IPR003598	Ig_sub2	Domain
IPR003599	Ig_sub	Domain
IPR007110	Ig-like_dom	Domain
IPR008266	Tyr_kinase_AS	Active_site
IPR011009	Kinase-like_dom_sf	Homologous_superfamily
IPR013098	Ig_I-set	Domain
IPR013783	Ig-like_fold	Homologous_superfamily
IPR017441	Protein_kinase_ATP_BS	Binding_site
IPR020635	Tyr_kinase_cat_dom	Domain
IPR027290	PDGFRA	Family
IPR036179	Ig-like_dom_sf	Homologous_superfamily
IPR050122	RTK	Family
IPR055238	VEGFR1-3_N_Ig-like	Domain

Pfam: PF07679, PF07714, PF22854, PF25305

Enzyme classification (BRENDA)

• EC 2.7.10.1 — receptor protein-tyrosine kinase (BRENDA: 44 organisms, 214 substrates, 574 inhibitors, 11 Km, 0 kcat entries)

Substrate kinetics (BRENDA)

4 substrates with measured Km, best-characterized 4. Km ranges are aggregated across organisms/conditions.

Substrate	Km (mM)	Measurements
ATP	0.0011–0.129	4
AC-DYFE-6-CHLORO-W-NHME	0.0051	1
AC-DYFGW-NHME	0.07	1
YFEW	0.232	1

Catalyzed reactions (Rhea)

• L-tyrosyl-[protein] + ATP = O-phospho-L-tyrosyl-[protein] + ADP + H(+) (RHEA:10596)

UniProt features (155 total): strand 46, helix 28, sequence variant 26, modified residue 11, glycosylation site 8, domain 6, mutagenesis site 6, turn 4, disulfide bond 4, splice variant 4, compositionally biased region 2, binding site 2, topological domain 2, signal peptide 1, chain 1, region of interest 1, active site 1, site 1, transmembrane region 1

Structure

Experimental structures (PDB)

19 structures by resolution:

PDB	Title	Method	Resolution (Å)
5GRN	Crystal structure of PDGFRA in Complex with WQ-C-159	X-RAY DIFFRACTION	1.77
8PQJ	PDGFRA wild-type kinase domain	X-RAY DIFFRACTION	1.82
6A32	Crystal structure of PDGFRA kinase domain mutant T674I	X-RAY DIFFRACTION	1.87
6JOL	Crystal structure of PDGFRA in complex with imatinib by co-crystallization	X-RAY DIFFRACTION	1.9
8PQK	APO crystal structure of PDGFRA-T674I kinase domain	X-RAY DIFFRACTION	2
9TFB	PDGFRA-G680R mutant kinase domain in complex with a …	X-RAY DIFFRACTION	2
5K5X	Crystal structure of human PDGFRA	X-RAY DIFFRACTION	2.17
9GZH	PDGFRA-T674I in complex with covalent ponatinib derivative 19	X-RAY DIFFRACTION	2.2
9TF9	PDGFRA-G680R mutant kinase domain in complex with avapritinib	X-RAY DIFFRACTION	2.2
9TFA	PDGFRA-G680R mutant kinase domain in complex with a 6,7-dimethoxyquinazoline-based …	X-RAY DIFFRACTION	2.2
1GQ5	Structural Determinants of the NHERF Interaction with beta2-AR and PDGFR	X-RAY DIFFRACTION	2.2
9TFC	PDGFRA-G680R mutant kinase domain in complex with a 7-methoxyquinazolin-6-ol-based …	X-RAY DIFFRACTION	2.3
8PQH	PDGFRA T674I mutant kinase domain in complex with avapritinib	X-RAY DIFFRACTION	2.5
6JOJ	Crystal structure of PDGFRA T674I in complex with crenolanib by soaking	X-RAY DIFFRACTION	2.6
8PQI	PDGFRA T674I mutant kinase domain in complex with avapritinib derivative 9	X-RAY DIFFRACTION	2.6
7LBF	CryoEM structure of the HCMV Trimer gHgLgO in complex with human Platelet-derived growth …	ELECTRON MICROSCOPY	2.8
6JOI	Crystal structure of PDGFRA T674I in complex with crenolanib by co-crystallization	X-RAY DIFFRACTION	3.1
7RAM	Cryo-EM Structure of the HCMV gHgLgO Trimer Derived from AD169 and TR strains in complex …	ELECTRON MICROSCOPY	3.43
6JOK	Crystal structure of PDGFRA in complex with sunitinib by soaking	X-RAY DIFFRACTION	3.8

Predicted structure (AlphaFold)

Model	pLDDT	Fraction very-high
AF-P16234-F1	72.91	0.25

Antibody-complex structures (SAbDab)

1 antibodies co-crystallized with this protein: 7LBF

Human Protein Atlas

Protein class: Enzymes, ENZYME proteins, Transferases, Kinases, Tyr protein kinases, CD markers, Predicted membrane proteins, Predicted intracellular proteins, Plasma proteins, RAS pathway related proteins, Cancer-related genes, Candidate cancer biomarkers, Mutational cancer driver genes, Disease related genes, FDA approved drug targets, Biotech drugs, Small molecule drugs, Human disease related genes

Protein evidence: Evidence at protein level

Subcellular location (HPA, imaging): cell junctions; nucleoplasm; plasma membrane; cytosol; nuclear bodies; primary cilium

Antibody reliability (HPA): IH supported · IF approved

Functional residue map

Curated UniProt residues grouped by drug-discovery relevance — catalytic, ligand-binding, modification, and mutation-validated positions. Source: UniProtKB sequence features.

Catalytic / active sites (2)

Position	Role
818	proton acceptor
578–579	breakpoint for interstitial deletion to form the fip1l1-pdgfra fusion protein

Ligand- & substrate-binding residues (2)

Position	Role
599–607	ATP
627	ATP

Post-translational modifications (11)

Type	Positions
Phosphotyrosine	572, 574, 720, 731, 742, 754, 762, 768, 849, 988, 1018

Disulfide bonds (4)

Type	Positions
Disulfide bond	49–100, 150–189, 235–290, 435–501

Glycosylation sites (8)

Type	Positions
N-linked (glcnac…) asparagine	42, 76, 103, 179, 353, 359, 458, 468

Mutagenesis-validated functional residues (6)

Position	Phenotype
572	abolishes interaction with src-family members and impairs internalization of the activated receptor; when associated with f-574.
574	abolishes interaction with src-family members and impairs internalization of the activated receptor; when associated with f-572.
720	strongly reduced interaction with ptpn11 and grb2.
731	no effect on autophosphorylation and phosphorylation of plcg1. abolishes activation of phosphatidylinositol 3-kinase.
742	no effect on autophosphorylation and phosphorylation of plcg1. abolishes activation of phosphatidylinositol 3-kinase.
762	abolishes interaction with crk.

Function

Pathways and Gene Ontology

Reactome pathways

13 curated (TAS) of 24; 13 disease pathways. Curated-first; evidence = TAS (manually curated) vs IEA (electronic inference).

24 pathways:

ID	Pathway	Evidence
R-HSA-2219530	Constitutive Signaling by Aberrant PI3K in Cancer ⚕	curated
R-HSA-9674396	Imatinib-resistant PDGFR mutants ⚕	curated
R-HSA-9674428	PDGFR mutants bind TKIs ⚕	curated
R-HSA-9674403	Regorafenib-resistant PDGFR mutants ⚕	curated
R-HSA-9673770	Signaling by PDGFRA extracellular domain mutants ⚕	curated
R-HSA-9673767	Signaling by PDGFRA transmembrane, juxtamembrane and kinase domain mutants ⚕	curated
R-HSA-9674404	Sorafenib-resistant PDGFR mutants ⚕	curated
R-HSA-9674401	Sunitinib-resistant PDGFR mutants ⚕	curated
R-HSA-186763	Downstream signal transduction	curated
R-HSA-6811558	PI5P, PP2A and IER3 Regulate PI3K/AKT Signaling	curated
R-HSA-1257604	PIP3 activates AKT signaling	curated
R-HSA-5673001	RAF/MAP kinase cascade	curated
R-HSA-186797	Signaling by PDGF	curated
R-HSA-1643685	Disease ⚕	—
R-HSA-5663202	Diseases of signal transduction by growth factor receptors and second messengers ⚕	—
R-HSA-9674415	Drug resistance of PDGFR mutants ⚕	—
R-HSA-2219528	PI3K/AKT Signaling in Cancer ⚕	—
R-HSA-9671555	Signaling by PDGFR in disease ⚕	—
R-HSA-9006925	Intracellular signaling by second messengers	—
R-HSA-5683057	MAPK family signaling cascades	—
R-HSA-5684996	MAPK1/MAPK3 signaling	—
R-HSA-199418	Negative regulation of the PI3K/AKT network	—
R-HSA-162582	Signal Transduction	—
R-HSA-9006934	Signaling by Receptor Tyrosine Kinases	—

MSigDB gene sets: 747 (showing top): GOBP_PLATELET_DERIVED_GROWTH_FACTOR_RECEPTOR_SIGNALING_PATHWAY, GOBP_REGULATION_OF_CELL_ACTIVATION, GOBP_HEMATOPOIETIC_PROGENITOR_CELL_DIFFERENTIATION, MODULE_52, GOBP_EMBRYO_DEVELOPMENT_ENDING_IN_BIRTH_OR_EGG_HATCHING, ACTACCT_MIR196A_MIR196B, GOBP_BODY_MORPHOGENESIS, GOBP_MUSCLE_TISSUE_DEVELOPMENT, BENPORATH_ES_WITH_H3K27ME3, IIZUKA_LIVER_CANCER_EARLY_RECURRENCE, GOBP_VASCULAR_ENDOTHELIAL_GROWTH_FACTOR_SIGNALING_PATHWAY, GOBP_REGULATION_OF_CALCIUM_MEDIATED_SIGNALING, GOBP_METANEPHROS_DEVELOPMENT, GOBP_REGULATION_OF_WOUND_HEALING, GOBP_CELL_CHEMOTAXIS

GO Biological Process (32): luteinization (GO:0001553), cell activation (GO:0001775), cell surface receptor protein tyrosine kinase signaling pathway (GO:0007169), positive regulation of cell population proliferation (GO:0008284), negative regulation of platelet activation (GO:0010544), cell migration (GO:0016477), peptidyl-tyrosine phosphorylation (GO:0018108), positive regulation of cell migration (GO:0030335), regulation of actin cytoskeleton organization (GO:0032956), cellular response to reactive oxygen species (GO:0034614), platelet-derived growth factor receptor-alpha signaling pathway (GO:0035790), peptidyl-tyrosine autophosphorylation (GO:0038083), positive regulation of cell proliferation by VEGF-activated platelet derived growth factor receptor signaling pathway (GO:0038091), wound healing (GO:0042060), protein autophosphorylation (GO:0046777), platelet-derived growth factor receptor signaling pathway (GO:0048008), positive regulation of fibroblast proliferation (GO:0048146), embryonic digestive tract morphogenesis (GO:0048557), embryonic cranial skeleton morphogenesis (GO:0048701), embryonic skeletal system morphogenesis (GO:0048704), positive regulation of calcium-mediated signaling (GO:0050850), positive regulation of chemotaxis (GO:0050921), positive regulation of phosphatidylinositol 3-kinase/protein kinase B signal transduction (GO:0051897), cardiac myofibril assembly (GO:0055003), cell chemotaxis (GO:0060326), retina vasculature development in camera-type eye (GO:0061298), positive regulation of ERK1 and ERK2 cascade (GO:0070374), platelet aggregation (GO:0070527), metanephric glomerular capillary formation (GO:0072277), regulation of mesenchymal stem cell differentiation (GO:2000739), protein phosphorylation (GO:0006468), chemotaxis (GO:0006935)

GO Molecular Function (16): protein kinase activity (GO:0004672), transmembrane receptor protein tyrosine kinase activity (GO:0004714), platelet-derived growth factor alpha-receptor activity (GO:0005018), vascular endothelial growth factor receptor activity (GO:0005021), platelet-derived growth factor receptor binding (GO:0005161), ATP binding (GO:0005524), vascular endothelial growth factor binding (GO:0038085), protein homodimerization activity (GO:0042803), protein-containing complex binding (GO:0044877), platelet-derived growth factor binding (GO:0048407), phospholipase C activator activity (GO:0160185), nucleotide binding (GO:0000166), protein tyrosine kinase activity (GO:0004713), protein binding (GO:0005515), kinase activity (GO:0016301), transferase activity (GO:0016740)

GO Cellular Component (12): nucleus (GO:0005634), cytoplasm (GO:0005737), endoplasmic reticulum membrane (GO:0005789), Golgi apparatus (GO:0005794), plasma membrane (GO:0005886), microvillus (GO:0005902), cilium (GO:0005929), external side of plasma membrane (GO:0009897), membrane (GO:0016020), protein-containing complex (GO:0032991), signaling receptor complex (GO:0043235), cell surface (GO:0009986)

Reactome top-level categories

13 top-level categories:

Category	Pathways
Drug resistance of PDGFR mutants	4
Signaling by PDGFR in disease	3
Intracellular signaling by second messengers	1
Signaling by PDGF	1
Signaling by Receptor Tyrosine Kinases	1
PI3K/AKT Signaling in Cancer	1
MAPK1/MAPK3 signaling	1
Negative regulation of the PI3K/AKT network	1
PIP3 activates AKT signaling	1
Diseases of signal transduction by growth factor receptors and second messengers	1
Disease	1
Signal Transduction	1
MAPK family signaling cascades	1

GO top-level categories

showing 60 of 85 GO namespaces:

Category	Terms
cellular anatomical structure	3
cell population proliferation	2
protein phosphorylation	2
positive regulation of cell population proliferation	2
embryonic organ morphogenesis	2
growth factor binding	2
binding	2
intracellular membrane-bounded organelle	2
female gonad development	1
ovulation cycle process	1
cellular process	1
multicellular organismal process	1
enzyme-linked receptor protein signaling pathway	1
regulation of cell population proliferation	1
positive regulation of cellular process	1
regulation of platelet activation	1
platelet activation	1
negative regulation of blood coagulation	1
negative regulation of cell activation	1
cell motility	1
cell migration	1
regulation of cell migration	1
positive regulation of cell motility	1
actin cytoskeleton organization	1
regulation of actin filament-based process	1
regulation of cytoskeleton organization	1
response to reactive oxygen species	1
cellular response to oxidative stress	1
cellular response to oxygen-containing compound	1
platelet-derived growth factor receptor signaling pathway	1
peptidyl-tyrosine phosphorylation	1
protein autophosphorylation	1
VEGF-activated platelet-derived growth factor receptor signaling pathway	1
response to wounding	1
tissue regeneration	1
cell surface receptor protein tyrosine kinase signaling pathway	1
fibroblast proliferation	1
regulation of fibroblast proliferation	1
digestive tract morphogenesis	1
embryonic digestive tract development	1
embryonic skeletal system morphogenesis	1
cranial skeletal system development	1
skeletal system morphogenesis	1
embryonic skeletal system development	1
kinase activity	1
phosphotransferase activity, alcohol group as acceptor	1
catalytic activity, acting on a protein	1
protein tyrosine kinase activity	1
transmembrane receptor protein kinase activity	1
platelet-derived growth factor receptor activity	1
platelet-derived growth factor receptor-alpha signaling pathway	1
transmembrane receptor protein tyrosine kinase activity	1
vascular endothelial growth factor signaling pathway	1
vascular endothelial growth factor binding	1
growth factor receptor binding	1
adenyl ribonucleotide binding	1
purine ribonucleoside triphosphate binding	1
anion binding	1
identical protein binding	1
protein dimerization activity	1

Experimentally-supported GO annotations

GO terms backed by direct experimental evidence (ECO experimental codes) — 27 of 60 annotations. The rest are phylogenetic / computational / electronic (IEA).

27 experimentally-supported GO terms:

GO term	NS	Evidence
cellular response to reactive oxygen species	BP	direct assay
negative regulation of platelet activation	BP	direct assay
peptidyl-tyrosine phosphorylation	BP	direct assay
platelet aggregation	BP	mutant phenotype
platelet-derived growth factor receptor signaling pathway	BP	direct assay
platelet-derived growth factor receptor-alpha signaling pathway	BP	mutant phenotype
positive regulation of ERK1 and ERK2 cascade	BP	mutant phenotype
positive regulation of calcium-mediated signaling	BP	mutant phenotype
positive regulation of cell migration	BP	direct assay
positive regulation of cell population proliferation	BP	direct assay
positive regulation of cell proliferation by VEGF-activated platelet derived growth factor receptor signaling pathway	BP	direct assay
positive regulation of chemotaxis	BP	mutant phenotype
positive regulation of fibroblast proliferation	BP	direct assay
protein autophosphorylation	BP	direct assay
regulation of mesenchymal stem cell differentiation	BP	mutant phenotype
membrane	CC	high-throughput assay
plasma membrane	CC	direct assay
protein-containing complex	CC	direct assay
phospholipase C activator activity	MF	mutant phenotype
platelet-derived growth factor alpha-receptor activity	MF	direct assay
platelet-derived growth factor binding	MF	direct assay
platelet-derived growth factor receptor binding	MF	physical interaction
protein homodimerization activity	MF	direct assay
protein kinase activity	MF	direct assay
transmembrane receptor protein tyrosine kinase activity	MF	direct assay
vascular endothelial growth factor binding	MF	physical interaction
vascular endothelial growth factor receptor activity	MF	direct assay

Protein interactions and networks

STRING

showing 15 of 4,474 interactions by confidence (×1000):

Protein A	Protein B	Partner UniProt	Score
PDGFRA	FIP1L1	Q6UN15	950
PDGFRA	PDGFA	P04085	887
PDGFRA	PDGFC	Q9NRA1	864
PDGFRA	PDGFB	P01127	828
PDGFRA	PDGFD	Q9GZP0	813
PDGFRA	NF1	P21359	810
PDGFRA	ANO1	Q5XXA6	805
PDGFRA	OLIG2	Q13516	784
PDGFRA	PTEN	P60484	778
PDGFRA	CSPG4	Q6UVK1	768
PDGFRA	SDHC	Q99643	765
PDGFRA	GFAP	P14136	758
PDGFRA	SOX10	P56693	747
PDGFRA	CD34	P28906	742
PDGFRA	PECAM1	P16284	731

IntAct

Human interaction partners of PDGFRA in IntAct, one row per partner (best MIscore; records = supporting IntAct interaction records).

29 partners by confidence:

Partner	Interaction type	MIscore	Records
PDGFRB	association	0.770	2
PDGFB	physical association	0.730	11
PDGFA	proximity	0.690	3
EGFR	physical association	0.680	4
CRK	proximity	0.670	4
PDGFC	direct interaction	0.660	1
CRKL	proximity	0.570	3
KASH5	physical association	0.560	3
PLCG1	proximity	0.540	2
PKM	phosphorylation reaction	0.440	1
SRPK1	phosphorylation reaction	0.440	1
PIK3R1	proximity	0.410	1
PSG5	physical association	0.400	3
SFN	physical association	0.400	2
YWHAE	physical association	0.400	2
ERBB3	physical association	0.370	1
TGFBR2	physical association	0.370	1
ULBP2	physical association	0.370	1
GXYLT2	association	0.350	2
MYO1B	association	0.350	1
QSOX1	association	0.350	1
RNPS1	association	0.350	1
FBXW7	proximity	0.270	4
SMAD4	proximity	0.270	4
SMARCA4	proximity	0.270	4
PTEN	proximity	0.270	2
FAM171A2	proximity	0.270	1
STAT1	proximity	0.270	1
STAT3	proximity	0.270	1

BioGRID (436 interactions; top human partners by supporting records): PLCG1 (Co-localization; 6 records), PDGFRB (Affinity Capture-MS; 5 records), PIK3R1 (Co-localization; 5 records), CBL (Affinity Capture-Western; 4 records), CRK (Co-localization; 4 records), PDGFA (Co-localization; 4 records), SLC9A3R1 (Far Western; 4 records), CAV1 (Affinity Capture-Western; 3 records), EGFR (Two-hybrid; 3 records), GRB2 (Affinity Capture-Western; 3 records), MAP2K3 (FRET; 3 records), PDGFB (Reconstituted Complex; 3 records), PDGFC (Reconstituted Complex; 3 records), PTPN11 (Affinity Capture-Western; 3 records), CALM3 (Affinity Capture-MS; 2 records)

SIGNOR signaling

showing 26 of 33 signaling interactions:

A	Effect	B	Mechanism
PDGFB	“up-regulates activity”	PDGFRA	binding
PDGFA	up-regulates	PDGFRA	binding
sunitinib	down-regulates	PDGFRA	“chemical inhibition”
PDGFRA	up-regulates	CRK	binding
N-(1,3-benzodioxol-5-ylmethyl)-4-(4-benzofuro[3,2-d]pyrimidinyl)-1-piperazinecarbothioamide	down-regulates	PDGFRA	“chemical inhibition”
Crenolanib	down-regulates	PDGFRA	“chemical inhibition”
linifanib	down-regulates	PDGFRA	“chemical inhibition”
PDGFRA	“up-regulates activity”	SRC	phosphorylation
PDGFRA	up-regulates	AKT1
PDGFRA	up-regulates	ERK1/2
imatinib	“down-regulates activity”	PDGFRA	“chemical inhibition”
regorafenib	“down-regulates activity”	PDGFRA	“chemical inhibition”
PDGFRA	“up-regulates activity”	PTK2	phosphorylation
PDGFRA	up-regulates	PLCG1	phosphorylation
CBL	“down-regulates quantity by destabilization”	PDGFRA	ubiquitination
PDGFRA	“up-regulates activity”	PDGFRA	phosphorylation
PTPRG	“up-regulates activity”	PDGFRA	dephosphorylation
nintedanib	“down-regulates activity”	PDGFRA	“chemical inhibition”
axitinib	“down-regulates activity”	PDGFRA	“chemical inhibition”
“2-Hydroxy-3-[N-[4-[methyl-[2-(4-methylpiperazin-1-yl)acetyl]amino]phenyl]-C-phenylcarbonimidoyl]-1H-indole-6-carboxylic acid”	“down-regulates activity”	PDGFRA	“chemical inhibition”
canertinib	“down-regulates activity”	PDGFRA	“chemical inhibition”
masitinib	“down-regulates activity”	PDGFRA	“chemical inhibition”
nilotinib	“down-regulates activity”	PDGFRA	“chemical inhibition”
tandutinib	“down-regulates activity”	PDGFRA	“chemical inhibition”
“pazopanib hydrochloride”	“down-regulates activity”	PDGFRA	“chemical inhibition”
ponatinib	“down-regulates activity”	PDGFRA	“chemical inhibition”

Protein complexes (CORUM) (11)

Experimentally-characterized complexes this protein is a subunit of (★ = the complex contains a known drug target).

11 complexes by size:

Complex	Subunits	Members
★ CRK-CRKL-PDGFRA-RAPGEF1 complex	4	CRK, CRKL, PDGFRA, RAPGEF1
★ GRB2-PDGFRA-TNK2 complex	3	GRB2, PDGFRA, TNK2
★ ERRFI1-GRB2-PDGFRA complex	3	ERRFI1, GRB2, PDGFRA
★ PDGFRA-PDGFRB complex	2	PDGFRA, PDGFRB
★ PDGFRA-SHF complex	2	PDGFRA, SHF
★ PDGFRA-SHB complex	2	PDGFRA, SHB
★ PDGFRA-STAT1 complex	2	PDGFRA, STAT1
★ PDGFRA-STAT3 complex	2	PDGFRA, STAT3
★ PDGFRA-STAT5A complex	2	PDGFRA, STAT5A
★ PDGFRA-PTPN11 complex	2	PDGFRA, PTPN11
★ PDGFRA homodimer	1	PDGFRA

Ligand–receptor pairs (CellPhoneDB) (4)

Curated ligand–receptor partners (cell-cell communication), distinct from the protein–protein interactions above.

4 ligand–receptor pairs:

Partner	This gene’s role	Signaling class
PDGFRA;PDGFRB		Signaling by Platelet-derived growth factor
PDGFC	receptor	Signaling by Platelet-derived growth factor
PDGFA	receptor	Signaling by Platelet-derived growth factor
PDGFB	receptor	Signaling by Platelet-derived growth factor

Enriched among interaction partners

Reactome pathways and GO biological processes over-represented among this gene’s 29 IntAct physical interaction partners (hypergeometric vs the genome-wide background, BH-FDR, gene-set size 15–500, ranked by fold). A functional readout of the neighbourhood — distinct from this gene’s own memberships above, and biased toward well-studied / hub proteins, so read it as themes rather than proof.

Reactome pathways:

Pathway	Partners	Fold	FDR
Downstream signal transduction	9	131.8×	2e-15
Signaling by PDGF	6	58.6×	1e-07
Constitutive Signaling by Aberrant PI3K in Cancer	6	29.3×	5e-06
PI5P, PP2A and IER3 Regulate PI3K/AKT Signaling	6	22.3×	2e-05
PIP3 activates AKT signaling	6	15.4×	1e-04
RAF/MAP kinase cascade	6	14.1×	1e-04
SARS-CoV Infections	5	10.7×	1e-03
Viral Infection Pathways	5	5.9×	8e-03

GO biological processes:

GO term	Partners	Fold	FDR
positive regulation of miRNA transcription	5	51.9×	8e-06
phosphatidylinositol 3-kinase/protein kinase B signal transduction	5	37.6×	2e-05
positive regulation of ERK1 and ERK2 cascade	7	21.3×	8e-06
positive regulation of phosphatidylinositol 3-kinase/protein kinase B signal transduction	6	16.8×	1e-04
positive regulation of cell migration	6	13.2×	2e-04
intracellular signal transduction	5	6.8×	8e-03

Disease & clinical

Cancer significance

From CIViC — curated cancer-variant interpretation:

Commonly mutated in GI tract tumors, PDGFR family genes (mutually exclusive to KIT mutations) are a hallmark of gastrointestinal stromal tumors. Gene fusions involving the PDGFRA kinase domain are highly correlated with eosinophilia, and the WHO classifies myeloid and lymphoid neoplasms with these characteristics as a distinct disorder. Mutations in the 842 region of PDGFRA have been often found to confer resistance to the tyrosine kinase inhibitor, imatinib.

From intOGen — cancer-driver classification: activating (oncogene-like) across 7 cancer types — CSCC, GB, GBM, HGGNOS, LGGNOS, LUSC, PAST.

intOGen’s role is a computational inference from cohort mutation patterns and may diverge from the gene’s curated/literature role; read it alongside the evidence above.

Genetic variants

Variant evidence for PDGFRA: 3,990 ClinVar records (7 pathogenic / likely pathogenic); 17 CIViC cancer variants; 34 GWAS associations. Per-variant detail is on Sugi Variant.

ClinVar

3,990 variants total. Per-class counts are floors (≥ shown; pagination cap):

Classification	Count (floor)
Pathogenic	7
Likely pathogenic	0
Uncertain significance	2062
Likely benign	1241
Benign	100

Top pathogenic / likely-pathogenic (7)

Showing all 7 pathogenic / likely-pathogenic ClinVar records, ranked by ClinVar review status (expert panel → multiple submitters → single submitter), then by number of submitting labs.

Variant ID	HGVS	Classification	Review	Submitters
13552	NM_006206.6(PDGFRA):c.1664A>G (p.Tyr555Cys)	Pathogenic	criteria provided, single submitter	2
13545	NM_006206.6(PDGFRA):c.2533_2544del (p.His845_Asn848del)	Pathogenic	no assertion criteria provided	1
13547	NM_006206.6(PDGFRA):c.1681_1682insAGAGGG (p.Arg560_Val561insGluArg)	Pathogenic	no assertion criteria provided	1
13548	NM_006206.6(PDGFRA):c.1679_1693del (p.Arg560_Ser564del)	Pathogenic	no assertion criteria provided	1
13549	NM_006206.6(PDGFRA):c.1696_1713del (p.Ser566_Glu571del)	Pathogenic	no assertion criteria provided	1
635777	NM_006206.6(PDGFRA):c.1957_1958delinsTT (p.Pro653Leu)	Pathogenic	no assertion criteria provided	1
635778	NM_006206.6(PDGFRA):c.2537A>T (p.Asp846Val)	Pathogenic	no assertion criteria provided	1

Per-variant reference — Sugi Variant

Explore PDGFRA’s pathogenic and likely-pathogenic variants individually — each with its ClinVar classification and conditions, applied ACMG criteria (ClinGen expert panel, where curated), the calibrated in-silico predictors (AlphaMissense, REVEL, SaProt, conservation), gnomAD frequency, and the predictor-disagreement QC signal — on Sugi Variant.

SpliceAI

Top 10 splice-impact sites by Δscore (adjacent positions with the same effect merged into one range; 22 predictions of 1,984):

Position(s)	Effect	Δscore	Sites
4:54229416	donor_loss	1.0000	2
4:54261413–54261414	donor_loss	1.0000	4
4:54263884	donor_gain	1.0000
4:54267421	donor_gain	1.0000
4:54272534	donor_gain	1.0000
4:54273764	donor_gain	1.0000
4:54274974–54274975	donor_loss	1.0000	5
4:54280479	donor_gain	1.0000
4:54285487–54285488	donor_loss	1.0000	5
4:54285836	acceptor_gain	1.0000

AlphaMissense

2,704 missense substitutions predicted likely pathogenic (of 7,179 scored), across 621 residues. Top 20 hotspot residues, ranked by the number of likely-pathogenic substitutions (of those reachable by a single-nucleotide change), then by mean score:

Residue	Likely-pathogenic substitutions	Mean am_pathogenicity	Max am_pathogenicity
M862	9	0.998	1.000
M806	9	0.986	1.000
M628	9	0.978	0.996
M915	9	0.978	1.000
M929	9	0.965	0.999
M648	9	0.919	1.000
M734	9	0.888	0.988
M642	9	0.854	0.988
D818	8	1.000	1.000
N823	8	1.000	1.000
D836	8	1.000	1.000
D877	8	1.000	1.000
F604	8	0.999	1.000
H816	8	0.999	1.000
F837	8	0.998	1.000
F905	8	0.998	1.000
F868	8	0.997	1.000
D681	8	0.995	1.000
D846	8	0.993	0.999
H654	8	0.991	0.999

HPA cancer prognostics

RNA cancer specificity: Cancer enhanced (Glioblastoma Multiforme)

Human Protein Atlas survival analysis — favorable = higher expression predicts better survival.

1 prognostic cancer types:

Cancer	Prognostic	p-value
Head and Neck Squamous Cell Carcinoma (TCGA)	favorable	6.22e-05

Disease associations

OMIM: gene MIM:173490 | disease phenotypes: MIM:606764, MIM:175510

GenCC curated gene-disease

5 curated gene-disease records:

Disease	Classification	Inheritance	Records
polyps, multiple and recurrent inflammatory fibroid, gastrointestinal	Definitive	Autosomal dominant	3
gastrointestinal stromal tumor	Definitive	Autosomal dominant	2
coloboma	Strong	Autosomal dominant
congenital heart disease	Limited	Autosomal dominant
isolated cleft palate	Limited	Unknown

ClinGen Gene-Disease Validity

Expert-panel classifications — Definitive > Strong > Moderate > Limited > Disputed > Refuted.

2 validity classifications:

Disease	Classification	Inheritance
gastrointestinal stromal tumor	Definitive	AD
congenital heart disease	Limited	AD

Genomics England panels (PanelApp)

Clinical diagnostic gene panels including this gene (6 panels, 3 green/diagnostic-grade). Confidence: green = diagnostic, amber = moderate.

6 panels:

Panel	Rating
Childhood solid tumours	green
Inherited predisposition to GIST	green
Sarcoma cancer susceptibility	green
COVID-19 research	amber
Childhood solid tumours cancer susceptibility	amber
Structural eye disease	amber

Mondo (5): gastrointestinal stromal tumor (MONDO:0011719), polyps, multiple and recurrent inflammatory fibroid, gastrointestinal (MONDO:0008285), congenital heart disease (MONDO:0005453), isolated cleft palate (MONDO:0007336), coloboma (MONDO:0001476)

Orphanet (1): Gastrointestinal stromal tumor (Orphanet:44890)

HPO phenotypes

55 phenotypes (HPO-id order):

HPO	Term
HP:0000006	Autosomal dominant inheritance
HP:0000175	Cleft palate
HP:0000202	Orofacial cleft
HP:0000220	Velopharyngeal insufficiency
HP:0000327	Hypoplasia of the maxilla
HP:0000403	Recurrent otitis media
HP:0000405	Conductive hearing impairment
HP:0000689	Dental malocclusion
HP:0000707	Abnormality of the nervous system
HP:0000750	Delayed speech and language development
HP:0000988	Skin rash
HP:0000989	Pruritus
HP:0001392	Abnormality of the liver
HP:0001442	Typified by somatic mosaicism
HP:0001611	Hypernasal speech
HP:0001723	Restrictive cardiomyopathy
HP:0001744	Splenomegaly
HP:0001880	Increased total eosinophil count
HP:0001903	Anemia
HP:0002015	Dysphagia
HP:0002017	Nausea and vomiting
HP:0002019	Constipation
HP:0002033	Poor suck
HP:0002113	Pulmonary infiltrates
HP:0002239	Gastrointestinal hemorrhage
HP:0002240	Hepatomegaly
HP:0002576	Intussusception
HP:0003326	Myalgia
HP:0003745	Sporadic
HP:0004395	Malnutrition
HP:0004936	Venous thrombosis
HP:0005214	Intestinal obstruction
HP:0005547	Myeloproliferative disorder
HP:0006292	Abnormality of dental eruption
HP:0006342	Peg-shaped maxillary lateral incisors
HP:0006685	Endocardial fibrosis
HP:0006753	Neoplasm of the stomach
HP:0007378	Neoplasm of the gastrointestinal tract
HP:0007400	Irregular hyperpigmentation
HP:0008872	Feeding difficulties in infancy
HP:0009088	Speech articulation difficulties
HP:0010294	Palate fistula
HP:0011044	Abnormal number of permanent teeth
HP:0012378	Fatigue
HP:0100242	Sarcoma
HP:0100273	Neoplasm of the colon
HP:0100334	Unilateral cleft palate
HP:0100337	Bilateral cleft palate
HP:0100723	Gastrointestinal stroma tumor
HP:0100743	Neoplasm of the rectum
HP:0100751	Esophageal neoplasm
HP:0100833	Neoplasm of the small intestine
HP:0200008	Intestinal polyposis
HP:0200136	Oral-pharyngeal dysphagia
HP:0200153	Agenesis of lateral incisor

GWAS associations

showing 30 of 34 associations:

Study	Trait	p-value
GCST006011_105	Mean corpuscular volume	3e-126
GCST005993_24	Mean corpuscular hemoglobin	6e-109
GCST005996_20	Red blood cell count	1e-93
GCST000585_9	Mean corpuscular volume	2e-29
GCST007269_297	Pulse pressure	3e-29
GCST000587_9	Mean corpuscular hemoglobin	3e-25
GCST002698_1	Serum VEGFR2 concentration	5e-25
GCST005994_20	Hematocrit	2e-20
GCST008413_427	Core binding factor acute myeloid leukemia	3e-18
GCST009158_34	Uterine fibroids	5e-18
GCST000588_1	Red blood cell count	2e-17
GCST005995_8	Hemoglobin	6e-16
GCST009724_61	Vertical cup-disc ratio (multi-trait analysis)	1e-15
GCST009724_62	Vertical cup-disc ratio (multi-trait analysis)	1e-14
GCST001803_1	Corneal curvature	5e-14
GCST009723_52	Vertical cup-disc ratio (adjusted for vertical disc diameter)	4e-12
GCST004332_3	Red blood cell count	4e-10
GCST006976_46	Macular thickness	8e-10
GCST006462_42	Uterine fibroids	9e-10
GCST001101_2	Corneal curvature	1e-9
GCST007267_150	Systolic blood pressure	2e-9
GCST005803_1	Corneal astigmatism	6e-9
GCST005991_10	Platelet count	7e-9
GCST001339_4	Corneal astigmatism	8e-9
GCST002502_4	Corneal curvature	8e-9
GCST008413_428	Core binding factor acute myeloid leukemia	6e-8
GCST006585_344	Blood protein levels	1e-7
GCST000136_2	Height	4e-7
GCST002479_6	Lupus nephritis in systemic lupus erythematosus	5e-7
GCST002936_29	Cadmium levels	1e-6

EFO canonical traits (from GWAS)

11 canonical traits:

EFO ID	Trait name
EFO:0004509	hemoglobin measurement
EFO:0004527	mean corpuscular hemoglobin
EFO:0004305	erythrocyte count
EFO:0004345	corneal topography
EFO:0004713	FEV/FVC ratio
EFO:1002040	Corneal astigmatism
EFO:0004309	platelet count
EFO:0004348	hematocrit
EFO:0006335	systolic blood pressure
EFO:0005763	pulse pressure measurement
EFO:0006939	cup-to-disc ratio measurement

MeSH disease descriptors

4 disease descriptors:

Descriptor	Name	Tree numbers
D003103	Coloboma	C11.250.110; C11.270.147; C16.131.384.282
D046152	Gastrointestinal Stromal Tumors	C04.557.450.565.370; C06.301.371.308; C06.405.249.308
D006330	Heart Defects, Congenital	C14.240.400; C14.280.400; C16.131.240.400
C566774	Polyps, Multiple And Recurrent Inflammatory Fibroid, Gastrointestinal *(supp.)*

Drugs & pharmacology

Drug and pharmacology data

Is drug target: yes

ChEMBL targets (3): CHEMBL2007 (SINGLE PROTEIN), CHEMBL2095189 (PROTEIN COMPLEX), CHEMBL4630733 (PROTEIN-PROTEIN INTERACTION)

Drugs by curated mechanism of action (ChEMBL)

Drugs whose curated ChEMBL mechanism of action targets the PDGFRA protein itself — single-protein targets only (drugs acting on a protein family or complex that merely includes it are excluded). Includes antibodies and oligonucleotides that bioactivity screening does not capture.

6 curated-mechanism drugs:

Drug	Modality	Max phase
AVAPRITINIB	Small molecule	4
OLARATUMAB	Antibody	4
RIPRETINIB	Small molecule	4
SUNITINIB	Small molecule	4
AMUVATINIB	Small molecule	2
TOVETUMAB	Antibody	2

Drugs acting on a complex or family that includes this protein

Curated ChEMBL mechanism-of-action drugs whose target is a protein complex or family of ≤10 members that includes PDGFRA — the drug acts on the assembly or class, not necessarily on this subunit alone.

24 drugs via a complex or family:

Drug	Target (ChEMBL)	Members	Max phase
BECAPLERMIN	Platelet-derived growth factor receptor	2	4
MIDOSTAURIN	Platelet-derived growth factor receptor	2	4
NINTEDANIB ESYLATE	Platelet-derived growth factor receptor	2	4
PAZOPANIB HYDROCHLORIDE	Platelet-derived growth factor receptor	2	4
QUIZARTINIB	Platelet-derived growth factor receptor	2	4
REGORAFENIB	Platelet-derived growth factor receptor	2	4
CEDIRANIB	Platelet-derived growth factor receptor	2	3
CRENOLANIB	Platelet-derived growth factor receptor	2	3
DOVITINIB	Platelet-derived growth factor receptor	2	3
FAMITINIB	Platelet-derived growth factor receptor	2	3
LINIFANIB	Platelet-derived growth factor receptor	2	3
MASITINIB	Platelet-derived growth factor receptor	2	3
MOTESANIB	Platelet-derived growth factor receptor	2	3
ORANTINIB	Platelet-derived growth factor receptor	2	3
SERALUTINIB	Platelet-derived growth factor receptor	2	3
VATALANIB	Platelet-derived growth factor receptor	2	3
ENMD-981693	Platelet-derived growth factor receptor	2	2
FORETINIB	Platelet-derived growth factor receptor	2	2
ILORASERTIB	Platelet-derived growth factor receptor	2	2
SU-014813	Platelet-derived growth factor receptor	2	2
X-82	Platelet-derived growth factor receptor	2	2
XL-820	Platelet-derived growth factor receptor	2	2
XL-999	Platelet-derived growth factor receptor	2	2
TAK-593	Platelet-derived growth factor receptor	2	1

Molecules with ChEMBL bioactivity

Phase ≥1, by development phase (incl. off-target/promiscuous compounds). Patent mentions across the top 20 by phase: 508,031 (via chembl_molecule»patent_compound — counts attach to the compound, not the gene–compound relationship, so off-target/promiscuous molecules can dominate).

showing 60 of 77 molecules by phase:

Molecule	Name	Phase	Patents
CHEMBL4204794	AVAPRITINIB	4	1,416
CHEMBL4216467	RIPRETINIB	4	1,151
CHEMBL535	SUNITINIB	4	80,232
CHEMBL1289926	AXITINIB	4	16,510
CHEMBL288441	BOSUTINIB	4	12,869
CHEMBL2403108	CERITINIB	4	9,002
CHEMBL5416410	DASATINIB	4	674
CHEMBL553	ERLOTINIB	4	109,860
CHEMBL1287853	FEDRATINIB	4	3,770
CHEMBL941	IMATINIB	4	113,105
CHEMBL1642	IMATINIB MESYLATE	4	70,926
CHEMBL1852688	INFIGRATINIB	4	2,298
CHEMBL1834657	INFIGRATINIB PHOSPHATE	4	330
CHEMBL1289601	LENVATINIB	4	9,224
CHEMBL608533	MIDOSTAURIN	4	7,641
CHEMBL255863	NILOTINIB	4	39,362
CHEMBL502835	NINTEDANIB	4	8,978
CHEMBL3039504	NINTEDANIB ESYLATE	4	691
CHEMBL477772	PAZOPANIB	4	16,241
CHEMBL3813873	PEXIDARTINIB	4	3,751
CHEMBL1171837	PONATINIB	4
CHEMBL576982	QUIZARTINIB	4
CHEMBL1946170	REGORAFENIB	4
CHEMBL1336	SORAFENIB	4
CHEMBL1289494	TIVOZANIB	4
CHEMBL24828	VANDETANIB	4
CHEMBL428690	ALVOCIDIB	3
CHEMBL415049	BARASERTIB	3
CHEMBL377300	BRIVANIB	3
CHEMBL31965	CANERTINIB	3
CHEMBL491473	CEDIRANIB	3
CHEMBL2105728	CRENOLANIB	3
CHEMBL522892	DOVITINIB	3
CHEMBL5095033	IBCASERTIB	3
CHEMBL603469	LESTAURTINIB	3
CHEMBL223360	LINIFANIB	3
CHEMBL1908391	MASITINIB	3
CHEMBL572881	MOTESANIB	3
CHEMBL2316582	OLVEREMBATINIB	3
CHEMBL5314535	PIMICOTINIB	3
CHEMBL377559	RETASPIMYCIN HYDROCHLORIDE	3
CHEMBL217092	SARACATINIB	3
CHEMBL276711	SEMAXANIB	3
CHEMBL101253	VATALANIB	3
CHEMBL5095202	VIMSELTINIB	3
CHEMBL2103851	AMUVATINIB	2
CHEMBL495727	AT-9283	2
CHEMBL3809489	BEMCENTINIB	2
CHEMBL460702	BMS-777607	2
CHEMBL1967878	CENISERTIB	2
CHEMBL2010872	CEP-11981	2
CHEMBL215152	DEFOSBARASERTIB	2
CHEMBL103667	DORAMAPIMOD	2
CHEMBL16699	ELLIPTINIUM	2
CHEMBL482968	ENMD-2076	2
CHEMBL1230609	FORETINIB	2
CHEMBL1980297	ILORASERTIB	2
CHEMBL3979920	MIVAVOTINIB	2
CHEMBL119385	NEFLAMAPIMOD	2
CHEMBL253969	OSI-632	2

Clinical evidence (CIViC)

Drug × variant × indication, from 45 curated evidence items; also 1 diagnostic. CIViC evidence level: A (validated) → B (clinical) → C (case study) → D (preclinical) → E (inferential).

showing 30 of 37 predictive associations by evidence level:

Variant	Therapy	Indication	Effect	Level	CIViC
PDGFRA D842V	Imatinib	Gastrointestinal Stromal Tumor	Sensitivity/Response	CIViC A	EID11545
PDGFRA Exon 18 Mutation	Avapritinib	Gastrointestinal Stromal Tumor	Sensitivity/Response	CIViC A	EID7809
PDGFRA Mutation	Ripretinib	Gastrointestinal Stromal Tumor	Sensitivity/Response	CIViC A	EID11333
PDGFRA Mutation	Imatinib	Gastrointestinal Stromal Tumor	Sensitivity/Response	CIViC B	EID11331 +1
PDGFRA D842V	Avapritinib	Gastrointestinal Stromal Tumor	Sensitivity/Response	CIViC B	EID7479
PDGFRA D842V	Imatinib	Gastrointestinal Stromal Tumor	Resistance	CIViC B	EID15 +4
PDGFRA D842V	Sunitinib	Gastrointestinal Stromal Tumor	Resistance	CIViC B	EID4058 +2
PDGFRA Exon 18 Mutation	Imatinib	Gastrointestinal Stromal Tumor	Sensitivity/Response	CIViC C	EID2486
PDGFRA V561D	Imatinib	Gastrointestinal Stromal Tumor	Sensitivity/Response	CIViC C	EID2474
FIP1L1::PDGFRA Fusion AND PDGFRA T674I	Imatinib	Myeloid And Lymphoid Neoplasms With Eosinophilia And Abnormalities Of PDGFRA, PDGFRB, And FGFR1	Resistance	CIViC C	EID1446 +1
FIP1L1::PDGFRA Fusion AND PDGFRA T674I	Ponatinib	Myeloid And Lymphoid Neoplasms With Eosinophilia And Abnormalities Of PDGFRA, PDGFRB, And FGFR1	Sensitivity/Response	CIViC D	EID1779
PDGFRA Amplification	Trametinib	Cancer	Sensitivity/Response	CIViC D	EID12457
PDGFRA Amplification	Sunitinib	Lung Non-small Cell Carcinoma	Sensitivity/Response	CIViC D	EID1771
PDGFRA Amplification	Sunitinib + Imatinib	Lung Squamous Cell Carcinoma	Sensitivity/Response	CIViC D	EID7989
PDGFRA D842I	Crenolanib	Gastrointestinal Stromal Tumor	Sensitivity/Response	CIViC D	EID43
PDGFRA D842V	Dasatinib	Gastrointestinal Stromal Tumor	Sensitivity/Response	CIViC D	EID4057
PDGFRA D842V	Crenolanib	Gastrointestinal Stromal Tumor	Sensitivity/Response	CIViC D	EID44
PDGFRA D842V	Bosutinib	Chronic Myeloid Leukemia	Sensitivity/Response	CIViC D	EID7514
PDGFRA D842Y	Crenolanib	Gastrointestinal Stromal Tumor	Sensitivity/Response	CIViC D	EID45
PDGFRA D842_I843delinsVM	Crenolanib	Gastrointestinal Stromal Tumor	Sensitivity/Response	CIViC D	EID47
PDGFRA G853D	Crenolanib + Imatinib	Melanoma	Sensitivity/Response	CIViC D	EID1977
PDGFRA H845Y	Crenolanib + Imatinib	Melanoma	Sensitivity/Response	CIViC D	EID1976
PDGFRA I843DEL	Imatinib	Gastrointestinal Stromal Tumor	Sensitivity/Response	CIViC D	EID1309
PDGFRA I843DEL	Crenolanib	Gastrointestinal Stromal Tumor	Sensitivity/Response	CIViC D	EID46
PDGFRA Overexpression	Dasatinib	High Grade Glioma	Sensitivity/Response	CIViC D	EID7934
PDGFRA Overexpression	Sunitinib	Rhabdomyosarcoma	Sensitivity/Response	CIViC D	EID7988
PDGFRA P577S	Imatinib + Crenolanib	Melanoma	Sensitivity/Response	CIViC D	EID1974
PDGFRA R841K	Crenolanib + Imatinib	Melanoma	Sensitivity/Response	CIViC D	EID1975
PDGFRA V561A	Imatinib	Gastrointestinal Stromal Tumor	Sensitivity/Response	CIViC D	EID652
FIP1L1::PDGFRA Fusion AND PDGFRA T674I	Dasatinib	Myeloid And Lymphoid Neoplasms With Eosinophilia And Abnormalities Of PDGFRA, PDGFRB, And FGFR1	Resistance	CIViC D	EID4420

CIViC column: a representative evidence item (EID); +N = additional CIViC items supporting the same association.

CIViC curated variants (17)

Named clinical variants curated in CIViC for this gene (the catalogue beneath the predictive evidence above):

D842Y (missense variant), I843DEL (inframe deletion), D842_I843delinsVM (missense variant), V561A (missense variant), Exon 18 Mutation, Overexpression, Mutation, T674I (missense variant), Amplification (transcript amplification), P577S (missense variant), R841K (missense variant), H845Y (missense variant), G853D (missense variant), V561D (missense variant), D842_H845DELDIMH (inframe deletion), D842I (missense variant), D842V (missense variant).

PharmGKB: 1 entry (CPIC guideline: no)

GtoPdb / IUPHAR curated pharmacology

(IUPHAR/BPS Guide to Pharmacology — expert-curated)

Target class: catalytic receptor — Type III RTKs: PDGFR, CSFR, Kit, FLT3 receptor family

Most potent curated ligand interactions (25 total), top 25:

Ligand	Action	Affinity	Parameter
olaratumab	Binding	10.4	pKd
ibcasertib	Inhibition	9.0	pIC50
AC710	Inhibition	8.89	pKd
compound 8h [PMID: 21561767]	Inhibition	8.89	pIC50
PP121	Inhibition	8.7	pIC50
compound 8i [PMID: 22765894]	Inhibition	8.7	pIC50
crenolanib	Inhibition	8.68	pKd
compound 8h [PMID: 22765894]	Inhibition	8.52	pIC50
elenestinib	Inhibition	8.35	pIC50
toceranib	Inhibition	8.3	pKi
CP-673451	Inhibition	8.0	pIC50
quizartinib	Inhibition	7.96	pKd
ilorasertib	Inhibition	7.96	pIC50
lucitanib	Inhibition	7.89	pIC50
CHMFL-KIT-64	Inhibition	7.6	pIC50
sitravatinib	Inhibition	7.52	pIC50
cediranib	Inhibition	7.44	pIC50
velzatinib	Inhibition	7.3	pIC50
BPR1R024	Inhibition	7.28	pIC50
ENMD-2076	Inhibition	7.25	pIC50
nintedanib	Inhibition	7.23	pIC50
dovitinib	Inhibition	6.68	pIC50
masitinib	Inhibition	6.27	pIC50
vimseltinib	Inhibition	6.0	pIC50
PDGF receptor tyrosine kinase inhibitor II	Inhibition	5.96	pIC50

Binding affinities (BindingDB)

1246 measured of 1898 human assays (1905 total across all organisms); most potent 50 below. Values come from heterogeneous assays and are not directly comparable.

Ligand	Measure	Value	Patent
[1-(4-chloro-2,3,5,6-tetradeuteriophenyl)-2,2,2-trideuterioethyl] 4-[6-(1-methylpyrazol-4-yl)pyrazolo[1,5-a]pyridin-3-yl]piperazine-1-carboxylate	IC50	0.016 nM	US-20250129067: PLATELET-DERIVED GROWTH FACTOR RECEPTOR (PDGFR) ALPHA INHIBITORS AND USES THEREOF
CHEMBL3808884	EC50	0.04 nM
3-[2-(Imidazo[1,2-b]pyridazin-3-yl)ethynyl]-4-methyl-N-{4-[(4-methylpiperazin-1-yl)methyl]-3-(trifluoromethyl)phenyl}benzamide	EC50	0.05 nM
2-Chloro-N-[4-(4,5-dichloro-2-fluoro-phenylamino)-quinazolin-6-yl]-acetamide	IC50	0.07 nM
CHEMBL3904768	IC50	0.09 nM
(E)-4-Dimethylamino-but-2-enoic acid [4-(4,5-dichloro-2-fluoro-phenylamino)-quinazolin-6-yl]-amide	IC50	0.11 nM
CHEMBL3979322	IC50	0.18 nM
CHEMBL6150730	IC50	0.18 nM
CHEMBL3940697	IC50	0.19 nM
CHEMBL6162898	IC50	0.22 nM
CHEMBL6114929	IC50	0.28 nM
CHEMBL3913766	IC50	0.32 nM
1-[2-(4-methylphenyl)-5-tert-butyl-pyrazol-3-yl]-3-[4-(2-morpholin-4-ylethoxy)naphthalen-1-yl]urea	KD	0.37 nM
AZD1152, 33	IC50	0.37 nM
N-[4-[(3-chloro-4-fluorophenyl)amino]pyrido[3,4-d]pyrimidin-6-yl]-5-(4-methyl-1-piperazinyl)-2-pentynamide	IC50	0.5 nM
CHEMBL3975580	IC50	0.53 nM
[(1R)-1-(4-chlorophenyl)ethyl] 2,2,3,3,5,5,6,6-octadeuterio-4-[6-(1-methylpyrazol-4-yl)pyrazolo[1,5-a]pyridin-3-yl]piperazine-1-carboxylate	IC50	0.56 nM	US-20250129067: PLATELET-DERIVED GROWTH FACTOR RECEPTOR (PDGFR) ALPHA INHIBITORS AND USES THEREOF
CHEMBL2385195	IC50	0.59 nM
CHEMBL6163448	IC50	0.6 nM
2-[4-(4-aminopyrrolo[2,1-f][1,2,4]triazin-6-yl)pyrazol-1-yl]-2-methylpropan-1-ol	IC50	0.65 nM	US-10829493: Compositions and methods for treating KIT- and PDGFRA-mediated diseases
3-[4-[4-[4-[5-[(1S)-1-amino-1-(4-fluorophenyl)ethyl]pyrimidin-2-yl]piperazin-1-yl]pyrrolo[2,1-f][1,2,4]triazin-6-yl]pyrazol-1-yl]cyclobutan-1-ol	IC50	0.65 nM	US-10829493: Compositions and methods for treating KIT- and PDGFRA-mediated diseases
[1-[4-[4-[4-[5-[(1S)-1-amino-1-(4-fluorophenyl)ethyl]pyrimidin-2-yl]piperazin-1-yl]pyrrolo[2,1-f][1,2,4]triazin-6-yl]pyrazol-1-yl]cyclopropyl]methanol	IC50	0.65 nM	US-10829493: Compositions and methods for treating KIT- and PDGFRA-mediated diseases
(3R,4R)-4-[4-[4-[4-[5-[(1S)-1-amino-1-(4-fluorophenyl)ethyl]pyrimidin-2-yl]piperazin-1-yl]pyrrolo[2,1-f][1,2,4]triazin-6-yl]pyrazol-1-yl]oxolan-3-ol	IC50	0.65 nM	US-10829493: Compositions and methods for treating KIT- and PDGFRA-mediated diseases
trans-(1S,2S)-2-[4-[4-[4-[5-[(1S)-1-amino-1-(4-fluorophenyl)ethyl]pyrimidin-2-yl]piperazin-1-yl]pyrrolo[2,1-f][1,2,4]triazin-6-yl]pyrazol-1-yl]cyclobutan-1-ol	IC50	0.65 nM	US-10829493: Compositions and methods for treating KIT- and PDGFRA-mediated diseases
2-[4-[4-[4-[5-[(1S)-1-amino-1-(4-fluorophenyl)ethyl]pyrimidin-2-yl]piperazin-1-yl]pyrrolo[2,1-f][1,2,4]triazin-6-yl]pyrazol-1-yl]-2-methylpropan-1-ol	KD	0.65 nM	US-12552795
N-[5-[[2-(1-methylpyrazol-4-yl)-4-pyridinyl]oxy]-2-pyridinyl]-5-oxo-6-azaspiro[3.4]octane-6-carboxamide	IC50	0.66 nM	US-11591328: Highly active CSF1R inhibitor compound
CHEMBL3950278	IC50	0.75 nM
CHEMBL3985689	IC50	0.96 nM
Jak Inhibitor 1	EC50	1 nM
CHEMBL2022856	IC50	1 nM
BAY-579352	IC50	1 nM
4-[[(1R)-1-cyclopropylethyl]amino]-7-(4-methylsulfonylphenyl)cinnoline-3-carboxamide	IC50	1 nM	US-9884828: Substituted cinnolines as inhibitors of LRRK2 kinase activity
4-[[2-[[(1R,2R)-2-hydroxycyclohexyl]amino]-1,3-benzothiazol-6-yl]oxy]-N-methylpyridine-2-carboxamide	IC50	1 nM	US-20250360145: CSF-1R INHIBITORS AND USES THEREOF
CHEMBL4763875	IC50	1 nM
CHEMBL4751287	IC50	1 nM
CHEMBL4798876	IC50	1.1 nM
CHEMBL4795462	IC50	1.1 nM
CHEMBL6177746	IC50	1.1 nM
CHEMBL3947262	IC50	1.2 nM
CHEMBL4798601	IC50	1.2 nM
CHEMBL6175201	IC50	1.2 nM
N-[6-methyl-5-[[2-(1-methylpyrazol-4-yl)-4-pyridinyl]oxy]-2-pyridinyl]-5-oxo-6-azaspiro[3.4]octane-6-carboxamide	IC50	1.2 nM	US-11591328: Highly active CSF1R inhibitor compound
CHEMBL3895824	IC50	1.3 nM
CHEMBL3955987	IC50	1.3 nM
CHEMBL4074755	IC50	1.4 nM
CHEMBL2419669	IC50	1.4 nM
CHEMBL4759047	IC50	1.4 nM
N-[5-[[2-(1-methylpyrazol-4-yl)-4-pyridinyl]oxy]-2-pyridinyl]-2-methylsulfonyl-5-oxo-2,6-diazaspiro[3.4]octane-6-carboxamide	IC50	1.4 nM	US-11591328: Highly active CSF1R inhibitor compound
Avapritinib	IC50	1.5 nM	US-10807985: Compositions useful for treating disorders related to kit
CHEMBL3958173	IC50	1.5 nM

ChEMBL bioactivities

1422 potent at pChembl≥5 of 1494 total, top 50 by pChembl (potency: 10 = 0.1 nM, 6 = 1 µM).

pChembl	Type	Value	Unit	Molecule
10.10	Ki	0.07943	nM	CHEMBL196363
10.05	IC50	0.09	nM	CHEMBL3904768
9.74	IC50	0.18	nM	CHEMBL3979322
9.74	IC50	0.18	nM	CHEMBL6150730
9.72	IC50	0.19	nM	CHEMBL3940697
9.70	IC50	0.2	nM	CHEMBL3915941
9.66	IC50	0.22	nM	CHEMBL6162898
9.62	IC50	0.24	nM	AVAPRITINIB
9.62	IC50	0.24	nM	CHEMBL1668411
9.55	IC50	0.28	nM	CHEMBL6114929
9.52	IC50	0.3	nM	CHEMBL6173067
9.49	IC50	0.32	nM	CHEMBL3913766
9.39	Kd	0.41	nM	CEDIRANIB
9.38	IC50	0.418	nM	STAUROSPORINE
9.37	IC50	0.43	nM	STAUROSPORINE
9.35	IC50	0.45	nM	STAUROSPORINE
9.33	Kd	0.47	nM	DASATINIB
9.31	Kd	0.49	nM	CHEMBL1908396
9.29	Kd	0.51	nM	AXITINIB
9.28	IC50	0.53	nM	CHEMBL3975580
9.24	IC50	0.58	nM	STAUROSPORINE
9.24	IC50	0.577	nM	STAUROSPORINE
9.22	IC50	0.6	nM	STAUROSPORINE
9.22	IC50	0.6	nM	RIPRETINIB
9.22	IC50	0.6	nM	PONATINIB
9.15	IC50	0.7	nM	RIPRETINIB
9.14	IC50	0.73	nM	CHEMBL3642837
9.12	IC50	0.75	nM	CHEMBL3950278
9.10	Ki	0.7943	nM	ILORASERTIB
9.10	IC50	0.793	nM	STAUROSPORINE
9.10	Kd	0.79	nM	SUNITINIB
9.10	IC50	0.8	nM	CHEMBL6177679
9.09	IC50	0.81	nM	TAS-115
9.02	IC50	0.96	nM	CHEMBL3985689
9.01	IC50	0.98	nM	CHEMBL1173411
9.00	IC50	1	nM	CHEMBL2071201
9.00	IC50	1	nM	CHEMBL4216473
9.00	Kd	1	nM	CHEMBL1079545
8.96	IC50	1.1	nM	PONATINIB
8.96	IC50	1.1	nM	CHEMBL5176837
8.96	IC50	1.1	nM	STAUROSPORINE
8.96	Kd	1.1	nM	SU-014813
8.92	IC50	1.2	nM	CHEMBL2347053
8.92	IC50	1.2	nM	CHEMBL3947262
8.92	IC50	1.2	nM	CHEMBL6177746
8.90	Ki	1.259	nM	CHEMBL1999414
8.89	Kd	1.3	nM	CHEMBL2206278
8.89	IC50	1.3	nM	CHEMBL3955987
8.89	IC50	1.3	nM	CHEMBL3895824
8.89	Kd	1.3	nM	CHEMBL4741283

PubChem BioAssay actives

1032 with measured affinity, of 3411 total; 50 most potent distinct compounds. Largely complementary to BindingDB; screening values are coarse (µM, 4 dp), so sub-nM hits tie at the floor.

Compound	Assay	Type	Value	Unit
N-[2-fluoro-5-[[2-(4-methylpiperazin-1-yl)phenyl]methylcarbamoyl]phenyl]-7-(1H-pyrazol-5-yl)imidazo[1,2-a]pyridine-3-carboxamide	1316152: Inhibition of PDGFRalpha kinase domain (unknown origin) assessed as reduction in probe peptide substrate phosphorylation by capillary electrophoresis	ic50	0.0001	uM
(2S,3R,4R,6R)-3-methoxy-2-methyl-4-(methylamino)-29-oxa-1,7,17-triazaoctacyclo[12.12.2.12,6.07,28.08,13.015,19.020,27.021,26]nonacosa-8,10,12,14,19,21,23,25,27-nonaen-16-one	350286: Inhibition of PDGFRalpha	ic50	0.0001	uM
N-[5-[(3,4-difluorophenyl)methylcarbamoyl]-2-fluorophenyl]-7-(2-methylpyrazol-3-yl)imidazo[1,2-a]pyridine-3-carboxamide	1316152: Inhibition of PDGFRalpha kinase domain (unknown origin) assessed as reduction in probe peptide substrate phosphorylation by capillary electrophoresis	ic50	0.0001	uM
(4S,6Z,9S,10R,12E)-9,10,18-trihydroxy-16-methoxy-4-methyl-3-oxabicyclo[12.4.0]octadeca-1(14),6,12,15,17-pentaene-2,8-dione	568303: Inhibition of human recombinant PDGFRalpha in cell free system after 60 mins	ic50	0.0002	uM
Avapritinib	2141012: Inhibition of PDGFRA (unknown origin)	ic50	0.0002	uM
N-(3-tert-butyl-1,2-oxazol-5-yl)-2-[4-[5-(1-methylpyrazol-4-yl)benzimidazol-1-yl]phenyl]acetamide	2214733: Inhibition of PDGFR-alpha (unknown origin)	ic50	0.0002	uM
N-(3-tert-butyl-1,2-oxazol-5-yl)-2-[4-[5-(1H-pyrazol-4-yl)benzimidazol-1-yl]phenyl]acetamide	2214733: Inhibition of PDGFR-alpha (unknown origin)	ic50	0.0002	uM
N-[2-fluoro-5-[[2-(4-methylpiperazin-1-yl)phenyl]methylcarbamoyl]phenyl]-7-(2-methylpyrazol-3-yl)imidazo[1,2-a]pyridine-3-carboxamide	1316152: Inhibition of PDGFRalpha kinase domain (unknown origin) assessed as reduction in probe peptide substrate phosphorylation by capillary electrophoresis	ic50	0.0002	uM
N-[5-[2-(2,2-dimethylpyrrolidin-1-yl)ethylcarbamoyl]-2-fluorophenyl]-7-(1-methylpyrazol-4-yl)imidazo[1,2-a]pyridine-3-carboxamide	1316152: Inhibition of PDGFRalpha kinase domain (unknown origin) assessed as reduction in probe peptide substrate phosphorylation by capillary electrophoresis	ic50	0.0002	uM
N-[5-[2-(2,2-dimethylpyrrolidin-1-yl)ethylcarbamoyl]-2-methyl-3-pyridinyl]-6-(1-methylpyrazol-4-yl)pyrazolo[1,5-a]pyridine-3-carboxamide	1316152: Inhibition of PDGFRalpha kinase domain (unknown origin) assessed as reduction in probe peptide substrate phosphorylation by capillary electrophoresis	ic50	0.0002	uM
N-(3-tert-butyl-1,2-oxazol-5-yl)-2-[4-(5-pyridin-3-ylbenzimidazol-1-yl)phenyl]acetamide	2214733: Inhibition of PDGFR-alpha (unknown origin)	ic50	0.0003	uM
N-[3-(2-imidazo[1,2-b]pyridazin-3-ylethynyl)-4-methylphenyl]-4-(prop-2-enoylamino)-3-(trifluoromethyl)benzamide	2267785: Inhibition of wild type PDGFRA (unknown origin) extracted from Sf9 cells by HTRF assay	ic50	0.0003	uM
N-[2-fluoro-5-[[2-(4-methylpiperazin-1-yl)phenyl]methylcarbamoyl]phenyl]-7-(1-methylpyrazol-4-yl)imidazo[1,2-a]pyridine-3-carboxamide	1316152: Inhibition of PDGFRalpha kinase domain (unknown origin) assessed as reduction in probe peptide substrate phosphorylation by capillary electrophoresis	ic50	0.0003	uM
4-[(4-fluoro-2-methyl-1H-indol-5-yl)oxy]-6-methoxy-7-(3-pyrrolidin-1-ylpropoxy)quinazoline	625034: Binding constant for PDGFRA kinase domain	kd	0.0004	uM
N-(2-chloro-6-methylphenyl)-2-[[6-[4-(2-hydroxyethyl)piperazin-1-yl]-2-methylpyrimidin-4-yl]amino]-1,3-thiazole-5-carboxamide;hydrate	435827: Binding constant for PDGFRA kinase domain	kd	0.0005	uM
N-[5-[(5,5-dimethyloxolan-2-yl)methylcarbamoyl]-2-fluorophenyl]-7-(2-methylpyrazol-3-yl)imidazo[1,2-a]pyridine-3-carboxamide	1316152: Inhibition of PDGFRalpha kinase domain (unknown origin) assessed as reduction in probe peptide substrate phosphorylation by capillary electrophoresis	ic50	0.0005	uM
Axitinib	625034: Binding constant for PDGFRA kinase domain	kd	0.0005	uM
1-[4-(6,7-dimethoxyquinolin-4-yl)oxy-2-methoxyphenyl]-3-[1-(1,3-thiazol-2-yl)ethyl]urea	625034: Binding constant for PDGFRA kinase domain	kd	0.0005	uM
Ponatinib	2267785: Inhibition of wild type PDGFRA (unknown origin) extracted from Sf9 cells by HTRF assay	ic50	0.0006	uM
Ripretinib	2267785: Inhibition of wild type PDGFRA (unknown origin) extracted from Sf9 cells by HTRF assay	ic50	0.0006	uM
3-(2-imidazo[1,2-b]pyridazin-3-ylethynyl)-4-methyl-N-[4-(prop-2-enoylamino)-3-(trifluoromethyl)phenyl]benzamide	2267785: Inhibition of wild type PDGFRA (unknown origin) extracted from Sf9 cells by HTRF assay	ic50	0.0008	uM
4-[2-fluoro-4-[(2-phenylacetyl)carbamothioylamino]phenoxy]-7-methoxy-N-methylquinoline-6-carboxamide	2095663: Inhibition of wild type human PDGFRalpha transfected with CHO cells by ADP-Glo assay	ic50	0.0008	uM
1-[4-[4-amino-7-[1-(2-hydroxyethyl)pyrazol-4-yl]thieno[3,2-c]pyridin-3-yl]phenyl]-3-(3-fluorophenyl)urea	673707: Inhibition of PDGFRA	ki	0.0008	uM
Sunitinib	435827: Binding constant for PDGFRA kinase domain	kd	0.0008	uM
N-[2-fluoro-5-[[2-(4-methylpiperazin-1-yl)phenyl]methylcarbamoyl]phenyl]-7-pyridin-3-ylimidazo[1,2-a]pyridine-3-carboxamide	1316152: Inhibition of PDGFRalpha kinase domain (unknown origin) assessed as reduction in probe peptide substrate phosphorylation by capillary electrophoresis	ic50	0.0008	uM
N-[4-[(5-tert-butyl-1,2-oxazol-3-yl)carbamoylamino]phenyl]-3-hydroxypyridine-2-carboxamide	469532: Binding affinity to PDGFRalpha	kd	0.0010	uM
5-[(Z)-[6-[[3-(4-methoxyanilino)-3-oxopropanoyl]amino]-2-oxo-1H-indol-3-ylidene]methyl]-2,4-dimethyl-1H-pyrrole-3-carboxylic acid	1184098: Inhibition of PDGFRalpha (unknown origin) using EAIYAAPFAKKK substrate by radioisotope-based P81 filter-binding assay	ic50	0.0010	uM
N’-[(3Z)-3-[[3,5-dimethyl-4-(2-pyrrolidin-1-ylethylcarbamoyl)-1H-pyrrol-2-yl]methylidene]-2-oxo-1H-indol-6-yl]-N-(4-methoxyphenyl)propanediamide	1184098: Inhibition of PDGFRalpha (unknown origin) using EAIYAAPFAKKK substrate by radioisotope-based P81 filter-binding assay	ic50	0.0010	uM
N-[4-[[5-fluoro-7-(2-methoxyethoxy)quinazolin-4-yl]amino]phenyl]-2-(4-propan-2-yltriazol-1-yl)acetamide	1934913: Inhibition of PDGFRalpha (unknown origin)	ic50	0.0010	uM
5-[(Z)-[6-[(4-methoxyphenyl)carbamoylamino]-2-oxo-1H-indol-3-ylidene]methyl]-4-methyl-1H-pyrrole-3-carboxylic acid	491704: Inhibition of PDGFRalpha	ic50	0.0010	uM
(1S,2S,3R,4R)-3-[[5-bromo-2-[(1-methylpyrazol-4-yl)amino]pyrimidin-4-yl]amino]bicyclo[2.2.1]hept-5-ene-2-carboxamide	677881: Inhibition of PDGFRalpha by TR-FRET analysis	ic50	0.0010	uM
N-[2-methyl-5-[[2-(4-methylpiperazin-1-yl)phenyl]methylcarbamoyl]-3-pyridinyl]-6-(2-methylpyrazol-3-yl)pyrazolo[1,5-a]pyridine-3-carboxamide	1316152: Inhibition of PDGFRalpha kinase domain (unknown origin) assessed as reduction in probe peptide substrate phosphorylation by capillary electrophoresis	ic50	0.0010	uM
5-[(Z)-(5-fluoro-2-oxo-1H-indol-3-ylidene)methyl]-N-[(2S)-2-hydroxy-3-morpholin-4-ylpropyl]-2,4-dimethyl-1H-pyrrole-3-carboxamide	435827: Binding constant for PDGFRA kinase domain	kd	0.0011	uM
6-[7-methoxy-6-(1-methylpyrazol-4-yl)imidazo[1,2-a]pyridin-3-yl]-N-pyrrolidin-3-ylpyridin-2-amine	1894591: Inhibition of PDGFR alpha (unknown origin)	ic50	0.0011	uM
3-(2-imidazo[1,2-b]pyridazin-3-ylethynyl)-N-[3-iodo-4-(prop-2-enoylamino)phenyl]-4-methylbenzamide	2267785: Inhibition of wild type PDGFRA (unknown origin) extracted from Sf9 cells by HTRF assay	ic50	0.0012	uM
(3Z)-3-[[5-(2-nitrophenyl)-1H-pyrazol-4-yl]methylidene]-1H-indol-2-one	739576: Inhibition of PDGFRalpha (unknown origin) after 10 mins by mobility shift assay	ic50	0.0012	uM
N-[2-fluoro-5-[[2-(4-methylpiperazin-1-yl)phenyl]methylcarbamoyl]phenyl]-6-(2-methylpyrazol-3-yl)pyrazolo[1,5-a]pyridine-3-carboxamide	1316152: Inhibition of PDGFRalpha kinase domain (unknown origin) assessed as reduction in probe peptide substrate phosphorylation by capillary electrophoresis	ic50	0.0012	uM
N-[5-[2-(2,2-dimethylpyrrolidin-1-yl)ethylcarbamoyl]-2-fluorophenyl]-7-(2-methylpyrazol-3-yl)imidazo[1,2-a]pyridine-3-carboxamide	1316152: Inhibition of PDGFRalpha kinase domain (unknown origin) assessed as reduction in probe peptide substrate phosphorylation by capillary electrophoresis	ic50	0.0013	uM
trans-(1S,2S)-2-[[6-[(2-amino-3-chloro-4-pyridinyl)methoxy]-1,3-benzothiazol-2-yl]amino]cyclohexan-1-ol	1723267: Binding affinity to human wild type PDGFRalpha (V575 to D1002 residues) expressed in mammalian expression system by kinome scan assay	kd	0.0013	uM
5-[2-[5-[[4-[[4-(2-hydroxyethyl)piperazin-1-yl]methyl]-3-(trifluoromethyl)phenyl]carbamoyl]-2-methylphenyl]ethynyl]-N,1-dimethylimidazole-2-carboxamide	601222: Inhibition of human PDGFRalpha using poly[Glu:Tyr] by Hotspot assay	ic50	0.0013	uM
N-[4-[(5-tert-butyl-1,2-oxazol-3-yl)carbamoylamino]phenyl]-5-(1-ethyl-2,2,6,6-tetramethylpiperidin-4-yl)oxypyridine-2-carboxamide	712315: Binding affinity to PDGFRalpha expressed in HEK-293 cells	kd	0.0013	uM
N-[5-[2-[(2R,6S)-2,6-dimethylpiperidin-1-yl]ethylcarbamoyl]-2-fluorophenyl]-7-(2-methylpyrazol-3-yl)imidazo[1,2-a]pyridine-3-carboxamide	1316152: Inhibition of PDGFRalpha kinase domain (unknown origin) assessed as reduction in probe peptide substrate phosphorylation by capillary electrophoresis	ic50	0.0013	uM
N-[3-(2-imidazo[1,2-b]pyridazin-3-ylethynyl)-4-methylphenyl]-1,3-benzothiazole-5-carboxamide	2267785: Inhibition of wild type PDGFRA (unknown origin) extracted from Sf9 cells by HTRF assay	ic50	0.0014	uM
N-[3-(2-imidazo[1,2-b]pyridazin-3-ylethynyl)-4-methylphenyl]-3-(prop-2-enoylamino)-5-(trifluoromethyl)benzamide	2267785: Inhibition of wild type PDGFRA (unknown origin) extracted from Sf9 cells by HTRF assay	ic50	0.0015	uM
1-(2,3-difluorophenyl)-3-[4-(6,7-dimethoxyquinolin-4-yl)oxyphenyl]urea	241088: Inhibition of platelet-derived growth factor receptor alpha	ic50	0.0016	uM
N-[5-[2-[tert-butyl(ethyl)amino]ethylcarbamoyl]-2-fluorophenyl]-7-(2-methylpyrazol-3-yl)imidazo[1,2-a]pyridine-3-carboxamide	1316152: Inhibition of PDGFRalpha kinase domain (unknown origin) assessed as reduction in probe peptide substrate phosphorylation by capillary electrophoresis	ic50	0.0016	uM
3-(2-imidazo[1,2-b]pyridazin-3-ylethynyl)-4-methyl-N-[3-(prop-2-enoylamino)-5-(trifluoromethyl)phenyl]benzamide	2267785: Inhibition of wild type PDGFRA (unknown origin) extracted from Sf9 cells by HTRF assay	ic50	0.0016	uM
N-[4-acetamido-3-(trifluoromethyl)phenyl]-3-(2-imidazo[1,2-b]pyridazin-3-ylethynyl)-4-methylbenzamide	2267785: Inhibition of wild type PDGFRA (unknown origin) extracted from Sf9 cells by HTRF assay	ic50	0.0016	uM
Quizartinib	526666: Binding affinity to PDGFRalpha	kd	0.0016	uM
3-(2-imidazo[1,2-b]pyridazin-3-ylethynyl)-4-methyl-N-[4-(prop-2-enoylamino)naphthalen-1-yl]benzamide	2267785: Inhibition of wild type PDGFRA (unknown origin) extracted from Sf9 cells by HTRF assay	ic50	0.0018	uM

CTD chemical–gene interactions

91 total (human), top 30 by PubMed support.

Chemical	Actions (top 5)	PubMed papers
Valproic Acid	affects cotreatment, increases expression, affects expression	7
bisphenol A	increases expression, affects cotreatment, affects methylation	3
trichostatin A	increases expression, affects cotreatment	3
ponatinib	decreases activity, decreases phosphorylation	3
Imatinib Mesylate	increases reaction, affects binding, increases response to substance, decreases expression	3
Tretinoin	increases expression	3
Cadmium Chloride	decreases reaction, decreases expression, increases abundance, increases expression, increases phosphorylation	3
perfluorooctane sulfonic acid	affects expression, affects cotreatment, decreases expression	2
monomethylarsonous acid	increases expression	2
bisphenol S	affects methylation, decreases expression	2
Sunitinib	decreases expression, decreases phosphorylation	2
Fulvestrant	decreases reaction, increases phosphorylation, decreases methylation	2
Vorinostat	affects cotreatment, increases expression	2
Panobinostat	affects cotreatment, increases expression	2
Benzo(a)pyrene	affects methylation, decreases expression, decreases methylation	2
Cadmium	decreases expression, increases abundance, increases phosphorylation, decreases reaction	2
Lead	increases abundance, increases expression, decreases expression	2
Phenylmercuric Acetate	increases expression, affects cotreatment	2
Progesterone	increases secretion, decreases expression, increases reaction, affects binding, increases phosphorylation	2
Aflatoxin B1	decreases expression, decreases methylation	2
bisphenol F	affects cotreatment, increases expression	1
ammonium 2,3,3,3-tetrafluoro-2-(heptafluoropropoxy)-propanoate	affects cotreatment, affects expression	1
TL8-506	affects cotreatment, increases expression	1
methylmercuric chloride	increases expression	1
propionaldehyde	increases expression	1
pirinixic acid	affects binding, decreases expression, increases activity	1
lead acetate	increases expression, increases abundance	1
N(4)-hydroxycytidine	decreases expression	1
2-methyl-4-isothiazolin-3-one	increases expression	1
arsenite	increases methylation	1

ChEMBL screening assays

1172 unique, capped per target: 1160 binding, 8 functional, 4 admet

Representative assays (with source publication via chembl_document):

Assay ID	Type	Description	Source paper
CHEMBL1009914	Binding	Inhibition of PDGFRalpha D842V mutant	Fragment-based discovery of the pyrazol-4-yl urea (AT9283), a multitargeted kinase inhibitor with potent aurora kinase activity. — *J Med Chem*
CHEMBL1963805	Functional	PUBCHEM_BIOASSAY: Navigating the Kinome. (Class of assay: other) Panel member name: PDGFRA	PubChem BioAssay data set
CHEMBL4044106	ADMET	Inhibition of wild-type human partial length PDGFRA (V575 to D1002 residues) expressed in HEK293 cells assessed as residual activity at 1000 nM after 30 mins by Kinomescan method relative to control	2-Oxo-3, 4-dihydropyrimido[4, 5-d]pyrimidinyl derivatives as new irreversible pan fibroblast growth factor receptor (FGFR) inhibitors. — *Eur J Med Chem*

Cellosaurus cell lines

28 cell lines: 20 cancer cell line, 5 induced pluripotent stem cell, 2 factor-dependent cell line, 1 transformed cell line

First 10 cell lines (id-ordered, not curated):

Cellosaurus	Name	Category	Sex
CVCL_0258	EoL-1	Cancer cell line	Male
CVCL_0D47	NZM087	Cancer cell line	Male
CVCL_0D59	NZM099	Cancer cell line	Sex unspecified
CVCL_9973	SUP-T13	Cancer cell line	Female
CVCL_A5MZ	UKAi007-A	Induced pluripotent stem cell	Male
CVCL_A5NA	UKAi007-B	Induced pluripotent stem cell	Male
CVCL_A5NB	UKAi008-A	Induced pluripotent stem cell	Male
CVCL_A5NC	UKAi008-B	Induced pluripotent stem cell	Male
CVCL_A5ND	UKAi008-C	Induced pluripotent stem cell	Male
CVCL_B0YH	Abcam SH-SY5Y PDGFRA KO	Cancer cell line	Female

Clinical trials (associated diseases)

Via MONDO — disease-level, not drug-specific.

showing 60 of 600 trials:

NCT	Phase	Status	Title
NCT00668824	Phase 4	UNKNOWN	Improved Diagnosis of Congenital Heart Disease by Magnetic Resonance Imaging Using Vasovist
NCT01368705	Phase 4	COMPLETED	Nitrogen Balance in Infants After Post Cardiothoracic Surgery
NCT01619982	Phase 4	COMPLETED	Pre-operative Prophylaxis With Vancomycin and Cefazolin in Pediatric Cardiovascular Surgery Patients
NCT02122679	Phase 4	WITHDRAWN	Tranexamic Acid Effect on Platelet Aggregation Following Infant Cardiopulmonary Bypass
NCT02527811	Phase 4	UNKNOWN	Ulinastatin Injection in in Pediatric Patients Undergoing Open Heart Surgery
NCT03014700	Phase 4	COMPLETED	Fibrinogen Concentrate vs Cryoprecipitate
NCT03408340	Phase 4	TERMINATED	Paravertebral Nerve Blocks in Neonates
NCT03630796	Phase 4	UNKNOWN	Effect of Sevoflurane in Postoperative Troponin I Levels in Children Undergoing Congenital Heart Defects Surgery
NCT03667703	Phase 4	COMPLETED	Stress Ulcer Prophylaxis Versus Placebo in Critically Ill Infants With Congenital Heart Disease
NCT04453761	Phase 4	UNKNOWN	Thiamine Influenced on Substrate Energy Effectiveness in Indonesian Children Undergoing Cardiopulmonary Bypass
NCT06668389	Phase 4	RECRUITING	Sodium-Glucose Cotransporter 2 Inhibitors for Repaired Tetralogy of Fallot Patients for Enhancement of Cardio-Pulmonary Status Trial
NCT07499154	Phase 4	NOT_YET_RECRUITING	Perioperative Lidocaine for Lung Protection in Infants Undergoing Cardiac Surgery
NCT00171977	Phase 4	COMPLETED	Post-Marketing Clinical Study of Postoperative Adjuvant Therapy With Imatinib Mesylate in Patients With Gastrointestinal Stromal Tumors (GIST)
NCT00510354	Phase 4	COMPLETED	Treatment of Patients With Everolimus and Imatinib Mesylate Who Have Progressive Gastro Intestinal Stromal Tumors (GIST) and Are Resistant to Imatinib Mesylate
NCT00756509	Phase 4	COMPLETED	Treatment of Patients With Metastatic or Unresectable Gastrointestinal Stromal Tumors in First Line With Nilotinib
NCT00777504	Phase 4	UNKNOWN	Study to the Optimal Duration of Therapy With Oral Angiogenesis Inhibitors
NCT02800330	Phase 4	COMPLETED	The Effects of the Proton Pump Inhibitor Esomeprazole on the Bioavailability of Regorafenib
NCT04825574	Phase 4	COMPLETED	Study for Patients Previously Treated in Avapritinib Clinical Trials
NCT00000470	Phase 3	COMPLETED	Infant Heart Surgery: Central Nervous System Sequelae of Circulatory Arrest
NCT00000494	Phase 3	COMPLETED	Management of Patent Ductus in Premature Infants
NCT01134302	Phase 3	UNKNOWN	Hybrid Versus Norwood Management Strategies in Infants Undergoing Single Ventricle Palliation
NCT01607983	Phase 3	WITHDRAWN	Effects of Pulmonary Vasodilation Upon VA Coupling in Fontan Patients
NCT01662011	Phase 3	UNKNOWN	Application of Neurally Adjusted Ventilatory Assist to Children After Congenital Cardiac Surgery
NCT02320669	Phase 3	COMPLETED	Phase 3 Triiodothyronine Supplementation for Infants After Cardiopulmonary Bypass
NCT02615262	Phase 3	COMPLETED	Intraoperative Dexamethasone in Pediatric Cardiac Surgery
NCT03153137	Phase 3	COMPLETED	Clinical Study Assessing the Efficacy and Safety of Macitentan in Fontan-palliated Subjects
NCT03154476	Phase 3	COMPLETED	Role of Sildenafil for Fontan Associated Liver Disease (SiFALD) Study
NCT04536194	Phase 3	COMPLETED	Dopamine Versus Norepinephrine Under General Anesthesia
NCT04702373	Phase 3	UNKNOWN	Training in Exercise Activities and Motion for Growth (TEAM 4 Growth) RCT
NCT05049590	Phase 3	COMPLETED	Acute Normovolemic Hemodilution in Complex Cardiac Surgery
NCT06406517	Phase 3	UNKNOWN	Comparative Effectiveness of Gadopiclenol for Evaluation of Adult Congenital Heart Anatomy and Hemodynamics
NCT06693674	Phase 3	RECRUITING	Effect of Sacubitril-Valsartan on Cardiac Structure and Function
NCT06955260	Phase 3	RECRUITING	SGLT2 Inhibition With Empagliflozin in Fontan Circulatory Failure
NCT00009906	Phase 3	TERMINATED	Comparison of Two Different Doses of STI571 in Treating Patients With Metastatic or Unresectable Gastrointestinal Stromal Tumor
NCT00041197	Phase 3	COMPLETED	Imatinib Mesylate in Treating Patients With Primary Gastrointestinal Stromal Tumor That Has Been Completely Removed By Surgery
NCT00075218	Phase 3	COMPLETED	A Study To Assess The Safety And Efficacy Of SU11248 In Patients With Gastrointestinal Stromal Tumor(GIST)
NCT00103168	Phase 3	COMPLETED	Imatinib Mesylate or Observation Only in Treating Patients Who Have Undergone Surgery for Localized Gastrointestinal Stromal Tumor
NCT00293124	Phase 3	COMPLETED	Open-label Trial of GlivecWith Unresectable or Metastatic Malignant Gastrointestinal Stromal Tumors
NCT00324987	Phase 3	TERMINATED	Imatinib Mesylate With or Without Bevacizumab in Treating Patients With Metastatic or Unresectable Gastrointestinal Stromal Tumor
NCT00372567	Phase 3	TERMINATED	Safety And Effectiveness Of Daily Dosing With Sunitinib Or Imatinib In Patients With Gastrointestinal Stromal Tumors
NCT00471328	Phase 3	COMPLETED	Efficacy and Safety of Nilotinib (AMN107) Compared With Current Treatment Options in Patients With GIST Who Have Failed Both Imatinib and Sunitinib
NCT00685828	Phase 3	UNKNOWN	Imatinib Mesylate in Treating Patients With Unresectable or Metastatic Gastrointestinal Stromal Tumor
NCT00688766	Phase 3	TERMINATED	Study Evaluating IPI-504 in Patients With Gastrointestinal Stromal Tumors (GIST) Following Failure of at Least Imatinib and Sunitinib
NCT00751036	Phase 3	TERMINATED	Nilotinib 800 Mg And Imatinib 800 Mg For The Treatment Of Patients With Gastrointestinal Stromal Tumors (Gist) Refractory To Imatinib 400 Mg
NCT00785785	Phase 3	COMPLETED	A Study of Nilotinib Versus Imatinib in GIST Patients
NCT00812240	Phase 3	TERMINATED	Masitinib in First Line Treatment of Gastro-Intestinal Stromal Tumor (GIST)
NCT00956072	Phase 3	TERMINATED	Imatinib Mesylate With or Without Surgery in Treating Patients With Metastatic Gastrointestinal Stromal Tumor That is Responding to Imatinib Mesylate
NCT01031628	Phase 3	TERMINATED	Study of Dose Escalation Versus no Dose Escalation of Imatinib in Metastatic Gastrointestinal Stromal Tumors (GIST) Patients
NCT01151852	Phase 3	COMPLETED	Rechallenge of Imatinib in GIST Having no Effective Treatment: RIGHT
NCT01265810	Phase 3	COMPLETED	Caphosol in Oral Mucositis Due to Targeted Therapy
NCT01271712	Phase 3	COMPLETED	Study of Regorafenib as a 3rd-line or Beyond Treatment for Gastrointestinal Stromal Tumors (GIST)
NCT01289028	Phase 3	COMPLETED	Efficacy of Nilotinib in Adult Patients With Gastrointestinal Stromal Tumors Resistant to Imatinib and Sunitinib.
NCT01462994	Phase 3	COMPLETED	Detection of CF-DNA in Patients With Gastrointestinal Stromal Tumors (GIST)
NCT01694277	Phase 3	COMPLETED	Masitinib in Patients With Gastrointestinal Stromal Tumour After Progression With Imatinib
NCT02260505	Phase 3	COMPLETED	Efficiency of Imatinib Treatment Maintenance or Interruption After 3 Years of Adjuvant Treatment in Patients With Gastrointestinal Stromal Tumours (GIST)
NCT02576080	Phase 3	UNKNOWN	Efficacy of Imatinib in Patients With Intermediate-risk Gastrointestinal Stromal Tumor With a High-risk Genomic Grade Index
NCT02866045	Phase 3	UNKNOWN	EUS-FNB vs. Single-incision Needle-knife (SINK) Biopsy for Gastrointestinal SELs
NCT03353753	Phase 3	COMPLETED	Phase 3 Study of DCC-2618 vs Placebo in Advanced GIST Patients Who Have Been Treated With Prior Anticancer Therapies
NCT03426722	Phase 3	COMPLETED	L-carnitine vs Placebo for the Treatment of Muscle Cramps After Imatinib in Gastrointestinal Stromal Tumors
NCT03465722	Phase 3	COMPLETED	(VOYAGER) Study of Avapritinib vs Regorafenib in Patients With Locally Advanced Unresectable or Metastatic GIST

Predicted chemistry — Sugi Predict

The predicted patent-compound landscape for PDGFRA across ~30M SureChEMBL compounds — explore on Sugi Predict.