acute myeloid leukemia by FAB classification

Summary

acute myeloid leukemia by FAB classification (MONDO:0015667) is a cancer (an umbrella term covering 9 Mondo subtypes) with 18 cohort genes (18 CIViC-evidence somatic drivers) and 1,496 clinical trials. Molecularly, FLT3 ITD confers sensitivity to Gilteritinib in Acute Myeloid Leukemia (CIViC Level A); 169 further subtype–drug associations are mapped below. Top therapeutic interventions include fludarabine phosphate, daunorubicin, and gemtuzumab ozogamicin.

At a glance

• Classification: Cancer
• Prevalence: 1-9 / 1 000 000 (Europe) [Orphanet-validated]
• Umbrella term: 9 Mondo subtypes
• Cohort genes: 18
• Clinical trials: 1,496
• Precision-medicine evidence (CIViC): 170 subtype–drug associations

Clinical features

Epidemiology

Prevalence records

1 prevalence record(s), Orphanet:

Type	Class	Value	Geography	Validation
Annual incidence	1-9 / 1 000 000	0.49	Europe	Validated

Identifiers

Disease identifiers

Field	Value
Canonical name	acute myeloid leukemia by FAB classification
Mondo ID	MONDO:0015667
Orphanet	167714
NCIT	C27753
UMLS	C5679583
MedGen	1842303
GARD	0012760
Is cancer (heuristic)	yes

Also known as: acute myeloid leukaemia · acute myeloid leukaemia NOS · acute myeloid leukaemia not otherwise categorised · acute myeloid leukaemia not otherwise specified · acute myeloid leukemia · acute myeloid leukemia NOS · acute myeloid leukemia not otherwise categorized · acute myeloid leukemia not otherwise specified · acute myeloid leukemia, NOS · AML, NOS · unclassified acute myeloid leukaemia · unclassified acute myeloid leukemia · unclassified AML

Data availability: 153 cell lines · 71 intOGen driver records.

Disease family

An umbrella term covering 9 Mondo subtypes.

Classification path: human disease › disease by etiologic mechanism › cancer or benign tumor › neoplastic disease or syndrome › neoplasm › hematopoietic and lymphoid system neoplasm › hematopoietic and lymphoid cell neoplasm › leukemia › myeloid leukemia › acute myeloid leukemia › acute myeloid leukemia by FAB classification

Related subtypes (77): childhood acute myeloid leukemia, acute monocytic leukemia, acute myeloid leukemia with t(8;21)(q22;q22) translocation, inherited acute myeloid leukemia, acute myeloid leukemia with CEBPA somatic mutations, acute myeloid leukemia with t(8;16)(p11;p13) translocation, acute myeloid leukemia with t(6;9)(p23;q34), acute myeloid leukemia with t(9;11)(p22;q23), acute myeloid leukemia with inv3(p21;q26.2) or t(3;3)(p21;q26.2), megakaryoblastic acute myeloid leukemia with t(1;22)(p13;q13), acute myeloid leukemia with NPM1 somatic mutations, acute myeloid leukemia with multilineage dysplasia, therapy related acute myeloid leukemia and myelodysplastic syndrome, acute leukemia of ambiguous lineage, acute myeloid leukemia with abnormal bone marrow eosinophils inv(16)(p13q22) or t(16;16)(p13;q22), acute myeloid leukemia with 11q23 abnormalities, acute myeloid leukemia with BCR-ABL1, acute myeloid leukemia with mutated NPM1, acute myeloid leukemia, inv(16)(p13.1;q22), acute myeloid leukemia, t(16;16)(p13.1;q22), acute myeloid leukemia, t(15;17)(q24;q21), acute myeloid leukemia, t(9;11)(p21.3;q23.3), acute myeloid leukemia, t(10;11)(p12;q23), acute myeloid leukemia, t(10;11)(p11.2;q23), acute myeloid leukemia, t(1;11)(q21;q23), acute myeloid leukemia, t(4;11)(q21;q23), acute myeloid leukemia, t(6;11)(q27;q23), acute myeloid leukemia, t(6;9)(p23;q34.1), acute myeloid leukemia, t(11;19)(q23;p13), acute myeloid leukemia, t(11;19)(q23;p13.1), acute myeloid leukemia, t(11;19)(q23.3;p13.3), acute myeloid leukemia, t(v;11q23.3), acute myeloid leukemia, Monosomy 7, acute myeloid leukemia, Monosomy 5, acute myeloid leukemia, Trisomy 8, acute myeloid leukemia, der12p, acute myeloid leukemia, t(2;12), acute myeloid leukemia, t(11;17), acute myeloid leukemia, t(8;16), acute myeloid leukemia, t(1;22), acute myeloid leukemia, t(5;11)(q35;p15), acute myeloid leukemia, t(7;12)(q36;p13), acute myeloid leukemia, t(9;22)(q34.1;q11.2), acute myeloid leukemia, inv(3)(q21.3;q26.2), acute myeloid leukemia, t(3;3)(q21.3;q26.2), acute myeloid leukemia, t(3;12)(q23;p12.3), acute myeloid leukemia, del(5q31-q32), acute myeloid leukemia, del(13q14-q21), acute myeloid leukemia, loss of chromosome 17p, acute myeloid leukemia, MLL gene rearrangement, acute myeloid leukemia, Non-KMT2A MLLT10 rearrangement positive, acute myeloid leukemia, inv(16)(p13.3;q24.3), acute myeloid leukemia, t(11;15)(p15;q35), acute myeloid leukemia, t(16;21)(q24;q22), acute myeloid leukemia, t(3;5)(q25;q34), acute myeloid leukemia, t(16;21)(p11;q22), acute myeloid leukemia, monoallelic CEBPA gene mutation, acute myeloid leukemia, biallelic CEBPA gene mutation, acute myeloid leukemia, CEBPA gene mutation, acute myeloid leukemia, FLT3 internal tandem duplication, acute myeloid leukemia, FLT3 tyrosine kinase domain point mutation, acute myeloid leukemia, WT1 gene mutation, acute myeloid leukemia, KIT exon 17 mutation, acute myeloid leukemia, KIT exon 8 mutation, acute myeloid leukemia, KIT gene mutation, acute myeloid leukemia, GATA1 gene mutation, acute myeloid leukemia, RUNX1 gene mutation, acute myeloid leukemia, PTPN11 gene mutation, acute myeloid leukemia, NRAS gene mutation, acute myeloid leukemia, KRAS gene mutation, core binding factor acute myeloid leukemia, acute myeloid leukemia, t(8;21)(q22; q22.1), acute myeloid leukemia, t(1;22)(p13;q13), acute myeloid leukemia with CBFA2T3-GLIS2 fusion, acute myeloid leukemia with FUS-ERG fusion, acute myeloid leukemia with MNX1-ETV6 fusion, acute myeloid leukemia with NPM1-MLF1 fusion

Subtypes (9): acute myeloid leukemia with minimal differentiation, acute myeloblastic leukemia without maturation, myeloid sarcoma, acute erythroid leukemia, acute myelomonocytic leukemia M4, acute megakaryoblastic leukemia, acute panmyelosis with myelofibrosis, acute basophilic leukemia, acute myeloblastic leukemia with maturation

Genetics & variants

GWAS landscape

No GWAS associations recorded — common-variant (GWAS) studies don’t cover this disease (typical for Mendelian / rare diseases). See the curated gene cohort and Mendelian overlap below.

Variant details and genetic-evidence tiers

No tiered GWAS variants or ClinVar records for this disease.

Genes & proteins

Mendelian disease overlap and somatic drivers

GenCC: 0 · Orphanet: 125 · OMIM-shared: 0 · Dual-evidence (GWAS+Mendelian): 0

Somatic driver evidence (intOGen + CIViC, cohort fanout)

Gene	intOGen role	Cancer types	CIViC
RUNX1	LoF	ACYC,ALL,AML,BRCA,GBM	CIViC #43
SRSF2	Act	AML	CIViC #5210
STAG2	LoF	AML,BLCA,CCRCC,ES,GBM,LUSC,MBL,PAST,PRCC,UCEC,WDTC	CIViC #8553
TP53	LoF	ACC,ALL,AML,ANGS,ANSC,BCC,BL,BLADDER,BLCA,BRCA,CCRCC,CEAD,CESC,CHOL,CHRCC,CLLSLL,COAD,COADREAD,CSCC,DLBCLNOS,EGC,ES,ESCA,ESCC,GB,GBC,GBM,GIST,HCC,HGGNOS,HNSC,LGGNOS,LIPO,LMS,LNM,LUAD,LUSC,MBL,MEL,MLYM,MT,NBL,NETNOS,NHL,NPC,NSCLC,OS,OVT,PAAD,PANCREAS,PAST,PCM,PLMESO,PRAD,PRCC,PROSTATE,RCC,READ,SACA,SARCNOS,SCLC,SIC,SKCM,SKIN,SOFT_TISSUE,STAD,STOMACH,THYM,UCEC,UCS,UTUC,VULVA,WDTC,WT	CIViC #45
U2AF1	Act	AML,CHOL,LUAD,NSCLC,PAAD,PRAD,STAD,UCEC,UCS	CIViC #48
WT1	LoF	AML,MEL,PAAD	CIViC #49
ASXL1	LoF	AML,BLCA,BRCA,CCRCC,CHOL,CLLSLL,COAD,ESCA,HGGNOS,HNSC,MBL,PAST,PRAD,STOMACH	CIViC #68
CEBPA	Act	AML	CIViC #15
CSF3R	LoF	AML,BLADDER,HNSC	CIViC #1239
TET2	LoF	AML,MDS,MLYM,NHL,PCM,RCC,SOFT_TISSUE	CIViC #55
DNMT3A	LoF	AML,BRCA,CCRCC,HCC,LGGNOS,MDS,PCM,PRCC,WDTC	CIViC #18
FGF13			CIViC #1872
FLT3	Act	ALL,AML	CIViC #24
IDH1	Act	AML,CHOL,GB,GBM,HCC,HGGNOS,LGGNOS,MBL,MEL,MT,OS,PAST,PCM,PRAD,SKCM	CIViC #26
IDH2	Act	AML,BLCA,CHOL,LGGNOS,OS	CIViC #27
KIT	Act	AML,GIST,MEL,MGCT	CIViC #29
NPM1	Act	HCC	CIViC #35
NRAS	Act	ALL,AML,ANGS,CHOL,CLLSLL,COAD,COADREAD,GBM,HCC,LGGNOS,LUAD,LUSC,MEL,MGCT,NPC,OVT,PCM,PROSTATE,SKCM,THYM,UCEC,WDTC	CIViC #36

Orphanet rare-disease linkage (cohort genes)

Gene	Orphanet ID	Rare disease
RUNX1	Orphanet:102724	Acute myeloid leukemia with t(8;21)(q22;q22) translocation
RUNX1	Orphanet:521	Chronic myeloid leukemia
RUNX1	Orphanet:71290	Familial platelet disorder with associated myeloid malignancy
RUNX1	Orphanet:98850	Aggressive systemic mastocytosis
SRSF2	Orphanet:98823	Chronic myelomonocytic leukemia
SRSF2	Orphanet:98849	Systemic mastocytosis with associated hematologic neoplasm
SRSF2	Orphanet:98850	Aggressive systemic mastocytosis
STAG2	Orphanet:220386	Semilobar holoprosencephaly
STAG2	Orphanet:521258	Xq25 microduplication syndrome
STAG2	Orphanet:93925	Alobar holoprosencephaly
TP53	Orphanet:1333	Familial pancreatic carcinoma
TP53	Orphanet:145	Hereditary breast and/or ovarian cancer syndrome
TP53	Orphanet:1501	Adrenocortical carcinoma
TP53	Orphanet:210159	Adult hepatocellular carcinoma
TP53	Orphanet:251576	Gliosarcoma
TP53	Orphanet:251579	Giant cell glioblastoma
TP53	Orphanet:251899	Choroid plexus carcinoma
TP53	Orphanet:2807	Papilloma of choroid plexus
TP53	Orphanet:293199	Pleomorphic rhabdomyosarcoma
TP53	Orphanet:3318	Essential thrombocythemia
TP53	Orphanet:524	Li-Fraumeni syndrome
TP53	Orphanet:52688	Myelodysplastic syndrome
TP53	Orphanet:585909	B-lymphoblastic leukemia/lymphoma with t(9;22)(q34.1;q11.2)
TP53	Orphanet:667662	Breast implant-associated anaplastic large cell lymphoma
TP53	Orphanet:668	Osteosarcoma
TP53	Orphanet:67038	B-cell chronic lymphocytic leukemia
TP53	Orphanet:70573	Small cell lung cancer
TP53	Orphanet:96253	Cushing disease
TP53	Orphanet:99756	Alveolar rhabdomyosarcoma
TP53	Orphanet:99757	Embryonal rhabdomyosarcoma
WT1	Orphanet:220	Denys-Drash syndrome
WT1	Orphanet:242	46,XY complete gonadal dysgenesis
WT1	Orphanet:251510	46,XY partial gonadal dysgenesis
WT1	Orphanet:3097	Meacham syndrome
WT1	Orphanet:347	Frasier syndrome
WT1	Orphanet:654	Nephroblastoma
WT1	Orphanet:656	Hereditary steroid-resistant nephrotic syndrome
WT1	Orphanet:83469	Desmoplastic small round cell tumor
WT1	Orphanet:893	WAGR syndrome
ASXL1	Orphanet:86845	Acute myeloid leukaemia with myelodysplasia-related features
ASXL1	Orphanet:97297	Bohring-Opitz syndrome
ASXL1	Orphanet:98823	Chronic myelomonocytic leukemia
ASXL1	Orphanet:98849	Systemic mastocytosis with associated hematologic neoplasm
ASXL1	Orphanet:98850	Aggressive systemic mastocytosis
CEBPA	Orphanet:102724	Acute myeloid leukemia with t(8;21)(q22;q22) translocation
CEBPA	Orphanet:319465	Inherited acute myeloid leukemia
CEBPA	Orphanet:319480	Acute myeloid leukemia with CEBPA somatic mutations
CSF3R	Orphanet:279943	Hereditary neutrophilia
CSF3R	Orphanet:420702	Autosomal recessive severe congenital neutropenia due to CSF3R deficiency
CSF3R	Orphanet:86829	Chronic neutrophilic leukemia

Cohort genes → proteins

18 cohort genes, 18 distinct canonical proteins.

Evidence partition

Subset	Genes
civic_only	18

Cohort genes (full)

Symbol	HGNC	Ensembl	UniProt	Name	Evidence
RUNX1	HGNC:10471	ENSG00000159216	Q01196	Runt-related transcription factor 1	civic_evidence
SRSF2	HGNC:10783	ENSG00000161547	Q01130	Serine/arginine-rich splicing factor 2	civic_evidence
STAG2	HGNC:11355	ENSG00000101972	Q8N3U4	Cohesin subunit SA-2	civic_evidence
TP53	HGNC:11998	ENSG00000141510	P04637	Cellular tumor antigen p53	civic_evidence
U2AF1	HGNC:12453	ENSG00000160201	Q01081	Splicing factor U2AF 35 kDa subunit	civic_evidence
WT1	HGNC:12796	ENSG00000184937	P19544	Wilms tumor protein	civic_evidence
ASXL1	HGNC:18318	ENSG00000171456	Q8IXJ9	Polycomb group protein ASXL1	civic_evidence
CEBPA	HGNC:1833	ENSG00000245848	P49715	CCAAT/enhancer-binding protein alpha	civic_evidence
CSF3R	HGNC:2439	ENSG00000119535	Q99062	Granulocyte colony-stimulating factor receptor	civic_evidence
TET2	HGNC:25941	ENSG00000168769	Q6N021	Methylcytosine dioxygenase TET2	civic_evidence
DNMT3A	HGNC:2978	ENSG00000119772	Q9Y6K1	DNA (cytosine-5)-methyltransferase 3A	civic_evidence
FGF13	HGNC:3670	ENSG00000129682	Q92913	Fibroblast growth factor 13	civic_evidence
FLT3	HGNC:3765	ENSG00000122025	P36888	Receptor-type tyrosine-protein kinase FLT3	civic_evidence
IDH1	HGNC:5382	ENSG00000138413	O75874	Isocitrate dehydrogenase [NADP] cytoplasmic	civic_evidence
IDH2	HGNC:5383	ENSG00000182054	P48735	Isocitrate dehydrogenase [NADP], mitochondrial	civic_evidence
KIT	HGNC:6342	ENSG00000157404	P10721	Mast/stem cell growth factor receptor Kit	civic_evidence
NPM1	HGNC:7910	ENSG00000181163	P06748	Nucleophosmin	civic_evidence
NRAS	HGNC:7989	ENSG00000213281	P01111	GTPase NRas	civic_evidence

Cohort function summary

Lead sentence per gene, UniProt-curated.

Symbol	Protein name	Function (lead sentence)
RUNX1	Runt-related transcription factor 1	Forms the heterodimeric complex core-binding factor (CBF) with CBFB.
SRSF2	Serine/arginine-rich splicing factor 2	Necessary for the splicing of pre-mRNA.
STAG2	Cohesin subunit SA-2	Component of cohesin complex, a complex required for the cohesion of sister chromatids after DNA replication.
TP53	Cellular tumor antigen p53	Multifunctional transcription factor that induces cell cycle arrest, DNA repair or apoptosis upon binding to its target DNA sequence.
U2AF1	Splicing factor U2AF 35 kDa subunit	Plays a critical role in both constitutive and enhancer-dependent splicing by mediating protein-protein interactions and protein-RNA interactions required for accurate 3’-splice site selection.
WT1	Wilms tumor protein	Transcription factor that plays an important role in cellular development and cell survival.
ASXL1	Polycomb group protein ASXL1	Probable Polycomb group (PcG) protein involved in transcriptional regulation mediated by ligand-bound nuclear hormone receptors, such as retinoic acid receptors (RARs) and peroxisome proliferator-activated receptor gamma (PPARG).
CEBPA	CCAAT/enhancer-binding protein alpha	Transcription factor that coordinates proliferation arrest and the differentiation of myeloid progenitors, adipocytes, hepatocytes, and cells of the lung and the placenta.
CSF3R	Granulocyte colony-stimulating factor receptor	Receptor for granulocyte colony-stimulating factor (CSF3), essential for granulocytic maturation.
TET2	Methylcytosine dioxygenase TET2	Dioxygenase that catalyzes the conversion of the modified genomic base 5-methylcytosine (5mC) into 5-hydroxymethylcytosine (5hmC) and plays a key role in active DNA demethylation.
DNMT3A	DNA (cytosine-5)-methyltransferase 3A	Required for genome-wide de novo methylation and is essential for the establishment of DNA methylation patterns during development.
FGF13	Fibroblast growth factor 13	Microtubule-binding protein which directly binds tubulin and is involved in both polymerization and stabilization of microtubules.
FLT3	Receptor-type tyrosine-protein kinase FLT3	Tyrosine-protein kinase that acts as a cell-surface receptor for the cytokine FLT3LG and regulates differentiation, proliferation and survival of hematopoietic progenitor cells and of dendritic cells.
IDH1	Isocitrate dehydrogenase [NADP] cytoplasmic	Catalyzes the NADP(+)-dependent oxidative decarboxylation of isocitrate (D-threo-isocitrate) to 2-ketoglutarate (2-oxoglutarate), which is required by other enzymes such as the phytanoyl-CoA dioxygenase.
IDH2	Isocitrate dehydrogenase [NADP], mitochondrial	Plays a role in intermediary metabolism and energy production.
KIT	Mast/stem cell growth factor receptor Kit	Tyrosine-protein kinase that acts as a cell-surface receptor for the cytokine KITLG/SCF and plays an essential role in the regulation of cell survival and proliferation, hematopoiesis, stem cell maintenance, gametogenesis, mast cell develo…
NPM1	Nucleophosmin	Involved in diverse cellular processes such as ribosome biogenesis, centrosome duplication, protein chaperoning, histone assembly, cell proliferation, and regulation of tumor suppressors p53/TP53 and ARF.
NRAS	GTPase NRas	Ras proteins bind GDP/GTP and possess intrinsic GTPase activity.

Protein-family classification

Druggable: 6 · Difficult: 4 · Unknown: 8 · Druggable fraction: 0.33

Family distribution

Cohort families vs a genome-wide background (hypergeometric, BH-FDR; fold = observed/expected). Counts kept; sorted by enrichment, so the catch-all Other/Unknown bucket no longer leads.

Family	Genes	Fold	FDR
Complement	1	14.9×	0.331
Kinase	2	3.1×	0.331
Transcription factor	4	1.8×	0.331
Antibody/Immunoglobulin	1	1.6×	0.559
Enzyme (other)	2	1.3×	0.559
Other/Unknown	8	0.8×	0.886

Per-gene assignment

Symbol	Family	Druggable?	EC	InterPro (top 3)
RUNX1	Transcription factor	no		AML1_Runt, p53-like_TF_DNA-bd_sf, p53/RUNT-type_TF_DNA-bd_sf
SRSF2	Other/Unknown	no		RRM_dom, RRM_euk-type, Nucleotide-bd_a/b_plait_sf
STAG2	Other/Unknown	no		STAG, ARM-type_fold, SCD
TP53	Transcription factor	no		p53_tumour_suppressor, p53-like_TF_DNA-bd_sf, p53_tetrameristn
U2AF1	Transcription factor	no		RRM_dom, Znf_CCCH, RRM_euk-type
WT1	Transcription factor	no		Wilms_tumour_N, Znf_C2H2_type, Znf_C2H2_sf
ASXL1	Other/Unknown	no		Asxl_HARE-HTH, ASX/ASX-like, ASX-like_PHD
CEBPA	Other/Unknown	no		bZIP, C/EBP_chordates, C/EBP
CSF3R	Antibody/Immunoglobulin	yes		Hematopoietin_rcpt_Gp130_CS, FN3_dom, IgC2-like_lig-bd
TET2	Other/Unknown	no		2OGFeDO_JBP1/TET_oxygenase_dom, TET1/2/3, TET_oxygenase
DNMT3A	Complement	yes	2.1.1.37	PWWP_dom, C5_MeTfrase, C5_DNA_meth_AS
FGF13	Other/Unknown	no		Fibroblast_GF_fam, IL1/FGF
FLT3	Kinase	yes	2.7.10.1	Prot_kinase_dom, Ser-Thr/Tyr_kinase_cat_dom, Tyr_kinase_rcpt_3_CS
IDH1	Enzyme (other)	yes	1.1.1.42	Isocitrate_DH_NADP, IsoCit/isopropylmalate_DH_CS, IsoPropMal-DH-like_dom
IDH2	Enzyme (other)	yes	1.1.1.42	Isocitrate_DH_NADP, IsoCit/isopropylmalate_DH_CS, IsoPropMal-DH-like_dom
KIT	Kinase	yes	2.7.10.1	Prot_kinase_dom, Ser-Thr/Tyr_kinase_cat_dom, Tyr_kinase_rcpt_3_CS
NPM1	Other/Unknown	no		Nucleoplasmin, Nucleoplasmin_core_dom, NPM1_C
NRAS	Other/Unknown	no		Small_GTPase, Small_GTP-bd, Small_GTPase_Ras-type

Expression context

Cohort genes with no expression data: 0.

18 cohort genes are a single-cell marker in ≥1 SCXA experiment.

Breadth distribution (Bgee present_calls)

Bucket	Genes
narrow (1-5 tissues)	0
moderate (6-20)	0
broad (>20)	18
unknown	0

Top tissues across cohort

Tissue	Cohort genes
sural nerve	3
ventricular zone	3
mucosa of paranasal sinus	2
tendon of biceps brachii	2
calcaneal tendon	2
ganglionic eminence	2
adrenal tissue	2
epithelium of nasopharynx	2
secondary oocyte	2
epithelium of bronchus	1
olfactory segment of nasal mucosa	1
embryo	1
tibia	1
adenohypophysis	1
bone marrow	1
left uterine tube	1
germinal epithelium of ovary	1
metanephric glomerulus	1
renal glomerulus	1
sperm	1

Per-gene tissue summary (top 30)

Symbol	Bgee breadth	FANTOM5 breadth	SCXA	Top tissues
RUNX1	253	ubiquitous	marker	olfactory segment of nasal mucosa, epithelium of bronchus, mucosa of paranasal sinus
SRSF2	295	ubiquitous	marker	tibia, embryo, tendon of biceps brachii
STAG2	299	ubiquitous	marker	mucosa of paranasal sinus, calcaneal tendon, sural nerve
TP53	223	ubiquitous	marker	ventricular zone, ganglionic eminence, tendon of biceps brachii
U2AF1	134	ubiquitous	marker	adenohypophysis, left uterine tube, bone marrow
WT1	168	broad	marker	germinal epithelium of ovary, renal glomerulus, metanephric glomerulus
ASXL1	294	ubiquitous	marker	sural nerve, sperm, adrenal tissue
CEBPA	258	ubiquitous	marker	nipple, upper arm skin, penis
CSF3R	192	broad	marker	granulocyte, monocyte, blood
TET2	249	ubiquitous	marker	palpebral conjunctiva, amniotic fluid, epithelium of nasopharynx
DNMT3A	223	ubiquitous	marker	sural nerve, ganglionic eminence, ventricular zone
FGF13	268	ubiquitous	marker	endothelial cell, dorsal root ganglion, cortical plate
FLT3	166	broad	marker	male germ line stem cell (sensu Vertebrata) in testis, cerebellar hemisphere, cerebellar cortex
IDH1	294	ubiquitous	marker	corpus epididymis, jejunal mucosa, adrenal tissue
IDH2	292	ubiquitous	marker	apex of heart, gastrocnemius, hindlimb stylopod muscle
KIT	263	broad	marker	lateral nuclear group of thalamus, secondary oocyte, oocyte
NPM1	276	ubiquitous	marker	calcaneal tendon, left ovary, ventricular zone
NRAS	278	ubiquitous	marker	gingival epithelium, epithelium of nasopharynx, secondary oocyte

Protein interactions among cohort

Intra-cohort edges: 36.

Hub genes (top 10 by interactor count)

Symbol	Interactor count
TP53	22,736
NRAS	7,598
NPM1	7,589
KIT	6,087
CEBPA	5,784
IDH1	5,464
RUNX1	4,994
IDH2	4,912
DNMT3A	4,771
WT1	3,938

Intra-cohort edges

A	B	Sources
ASXL1	DNMT3A	string_interaction
ASXL1	FLT3	string_interaction
ASXL1	IDH1	string_interaction
ASXL1	IDH2	string_interaction
ASXL1	NPM1	string_interaction
ASXL1	RUNX1	string_interaction
ASXL1	SRSF2	string_interaction
ASXL1	TET2	string_interaction
ASXL1	U2AF1	string_interaction
CEBPA	CSF3R	string_interaction
CEBPA	FLT3	string_interaction
CEBPA	RUNX1	string_interaction
CEBPA	TP53	string_interaction
CSF3R	KIT	string_interaction
DNMT3A	FLT3	string_interaction
DNMT3A	TET2	string_interaction
DNMT3A	U2AF1	string_interaction
FLT3	IDH1	string_interaction
FLT3	IDH2	string_interaction
FLT3	NPM1	string_interaction
FLT3	RUNX1	string_interaction
FLT3	TET2	string_interaction
IDH1	IDH2	biogrid_interaction
IDH1	TET2	string_interaction
IDH1	TP53	string_interaction
IDH1	U2AF1	string_interaction
IDH2	TET2	string_interaction
KIT	TP53	biogrid_interaction
NPM1	TP53	string_interaction
NRAS	TP53	string_interaction
RUNX1	SRSF2	string_interaction
SRSF2	TET2	string_interaction
SRSF2	U2AF1	intact, string_interaction
TET2	U2AF1	string_interaction
TET2	WT1	intact
TP53	WT1	intact, string_interaction

Structural data

PDB: 18 · AlphaFold-only: 0 · No structure: 0

Cohort genes with PDB structures (top 30)

Symbol	UniProt	PDB entries
TP53	P04637	313
IDH1	O75874	61
KIT	P10721	52
DNMT3A	Q9Y6K1	43
NRAS	P01111	35
WT1	P19544	28
FLT3	P36888	11
IDH2	P48735	11
STAG2	Q8N3U4	9
NPM1	P06748	8
TET2	Q6N021	6
RUNX1	Q01196	5
SRSF2	Q01130	4
ASXL1	Q8IXJ9	4
FGF13	Q92913	3
CEBPA	P49715	2
U2AF1	Q01081	1
CSF3R	Q99062	1

Function

Pathway analysis

Distinct Reactome pathways touched by cohort: 276. Enrichment computed across 18 evidence-associated genes (18 with Reactome annotation).

Pathways by enrichment

Over-representation of cohort genes vs the genome-wide background (hypergeometric test, Benjamini-Hochberg FDR; fold = observed/expected over 18 annotated cohort genes). Counts and members are kept as ground-truth; sorted by enrichment.

Pathway	Cohort genes	Fold	FDR	Sample cohort genes
Abnormal conversion of 2-oxoglutarate to 2-hydroxyglutarate	1	634.4×	0.013	IDH1
Dasatinib-resistant KIT mutants	1	634.4×	0.013	KIT
Imatinib-resistant KIT mutants	1	634.4×	0.013	KIT
KIT mutants bind TKIs	1	634.4×	0.013	KIT
Masitinib-resistant KIT mutants	1	634.4×	0.013	KIT
Nilotinib-resistant KIT mutants	1	634.4×	0.013	KIT
Regorafenib-resistant KIT mutants	1	634.4×	0.013	KIT
Signaling by kinase domain mutants of KIT	1	634.4×	0.013	KIT
Sunitinib-resistant KIT mutants	1	634.4×	0.013	KIT
Signaling by juxtamembrane domain KIT mutants	1	634.4×	0.013	KIT
Sorafenib-resistant KIT mutants	1	634.4×	0.013	KIT
Drug resistance of KIT mutants	1	634.4×	0.013	KIT
Signaling by extracellular domain mutants of KIT	1	634.4×	0.013	KIT
FLT3 mutants bind TKIs	1	634.4×	0.013	FLT3
KW2449-resistant FLT3 mutants	1	634.4×	0.013	FLT3
semaxanib-resistant FLT3 mutants	1	634.4×	0.013	FLT3
crenolanib-resistant FLT3 mutants	1	634.4×	0.013	FLT3
gilteritinib-resistant FLT3 mutants	1	634.4×	0.013	FLT3
lestaurtinib-resistant FLT3 mutants	1	634.4×	0.013	FLT3
midostaurin-resistant FLT3 mutants	1	634.4×	0.013	FLT3
pexidartinib-resistant FLT3 mutants	1	634.4×	0.013	FLT3
ponatinib-resistant FLT3 mutants	1	634.4×	0.013	FLT3
quizartinib-resistant FLT3 mutants	1	634.4×	0.013	FLT3
sorafenib-resistant FLT3 mutants	1	634.4×	0.013	FLT3
sunitinib-resistant FLT3 mutants	1	634.4×	0.013	FLT3
tandutinib-resistant FLT3 mutants	1	634.4×	0.013	FLT3
linifanib-resistant FLT3 mutants	1	634.4×	0.013	FLT3
tamatinib-resistant FLT3 mutants	1	634.4×	0.013	FLT3
Loss of function of TP53 in cancer due to loss of tetramerization ability	1	634.4×	0.013	TP53
Signaling by FLT3 ITD and TKD mutants	2	84.6×	0.013	FLT3, NRAS

GO biological processes by enrichment

Over-representation of cohort genes vs the genome-wide background (hypergeometric test, Benjamini-Hochberg FDR; fold = observed/expected over 18 annotated cohort genes). Counts and members are kept as ground-truth; sorted by enrichment.

GO term	Cohort genes	Fold	FDR	Sample cohort genes
hemopoiesis	4	59.4×	2e-04	RUNX1, ASXL1, FLT3, KIT
glyoxylate cycle	2	936.2×	2e-04	IDH1, IDH2
myeloid cell differentiation	3	108.0×	4e-04	RUNX1, CEBPA, TET2
isocitrate metabolic process	2	374.5×	9e-04	IDH1, IDH2
cytokine-mediated signaling pathway	4	29.0×	9e-04	CEBPA, CSF3R, FLT3, KIT
myeloid progenitor cell differentiation	2	267.5×	0.002	FLT3, KIT
regulation of DNA damage response, signal transduction by p53 class mediator	2	234.1×	0.002	TP53, NPM1
negative regulation of glial cell proliferation	2	187.2×	0.002	TP53, IDH2
NADP+ metabolic process	2	170.2×	0.002	IDH1, IDH2
bone marrow development	2	170.2×	0.002	TP53, ASXL1
positive regulation of transcription by RNA polymerase II	7	5.8×	0.004	RUNX1, TP53, WT1, ASXL1, CEBPA, TET2, NPM1
negative regulation of gene expression via chromosomal CpG island methylation	2	117.0×	0.004	WT1, DNMT3A
2-oxoglutarate metabolic process	2	104.0×	0.005	IDH1, IDH2
positive regulation of tyrosine phosphorylation of STAT protein	2	81.4×	0.008	FLT3, KIT
hematopoietic stem cell proliferation	2	72.0×	0.009	RUNX1, CEBPA
negative regulation of helicase activity	1	936.2×	0.014	TP53
regulation of phospholipid catabolic process	1	936.2×	0.014	IDH1
regulation of eIF2 alpha phosphorylation by dsRNA	1	936.2×	0.014	NPM1
negative regulation of metanephric glomerular mesangial cell proliferation	1	936.2×	0.014	WT1
cellular response to actinomycin D	1	936.2×	0.014	TP53
melanocyte adhesion	1	936.2×	0.014	KIT
positive regulation of pyloric antrum smooth muscle contraction	1	936.2×	0.014	KIT
regulation of intrinsic apoptotic signaling pathway by p53 class mediator	1	936.2×	0.014	TP53
regulation of mRNA stability involved in cellular response to UV	1	936.2×	0.014	NPM1
negative regulation of G1 to G0 transition	1	936.2×	0.014	TP53
positive regulation of colon smooth muscle contraction	1	936.2×	0.014	KIT
regulation of connective tissue replacement	1	936.2×	0.014	RUNX1
tricarboxylic acid cycle	2	56.7×	0.014	IDH1, IDH2
macrophage differentiation	2	52.0×	0.014	CEBPA, NPM1
RNA splicing	3	14.7×	0.014	SRSF2, U2AF1, WT1

Therapeutics

Drug target analysis

Approved (phase 4): 8 · Phase ≥3: 8 · Phased (≥1): 11 · Undrugged: 7

Druggability breadth: 14 of 18 evidence-associated genes (78%) have a ChEMBL target (buckets above are over the deeply-mined display cohort).

Genes with an approved drug

The molecule shown is one approved compound that hits the gene — not necessarily a drug of choice or one indicated for this disease.

Symbol	Example approved molecule
RUNX1	APOMORPHINE HYDROCHLORIDE
TP53	NITROFURANTOIN
TET2	VADADUSTAT
FLT3	PONATINIB
IDH1	ENASIDENIB
IDH2	ENASIDENIB
KIT	PONATINIB
NPM1	CERITINIB

Top cohort targets by molecule count

Symbol	Molecules	Max phase
TP53	196	4
FLT3	143	4
KIT	99	4
IDH1	10	4
IDH2	7	4
NPM1	5	4
TET2	3	4
RUNX1	2	4
SRSF2	1	2
U2AF1	1	2

Drugs targeting cohort genes (top 30)

Molecule	Max phase	Targets in cohort
APOMORPHINE HYDROCHLORIDE	4	RUNX1
NITROFURANTOIN	4	TP53
DIOSMIN	4	TP53
VERTEPORFIN	4	TP53
CANDESARTAN CILEXETIL	4	TP53
DIENESTROL	4	TP53
CLOTRIMAZOLE	4	TP53
COLCHICINE	4	TP53
NABUMETONE	4	TP53
SALMETEROL XINAFOATE	4	TP53
AMIODARONE HYDROCHLORIDE	4	TP53
FURAZOLIDONE	4	TP53
AMOXAPINE	4	TP53
RALOXIFENE HYDROCHLORIDE	4	TP53
NICARDIPINE HYDROCHLORIDE	4	TP53
SULCONAZOLE NITRATE	4	TP53
PYRITHIONE ZINC	4	TP53
LACTIC ACID	4	TP53
OXYMETHOLONE	4	TP53
CHLOROXINE	4	TP53
PROPIOLACTONE	4	TP53
CLOMIPRAMINE HYDROCHLORIDE	4	TP53
PHENYL AMINOSALICYLATE	4	TP53
THIORIDAZINE HYDROCHLORIDE	4	TP53
AMITRIPTYLINE HYDROCHLORIDE	4	TP53
ETHOPROPAZINE HYDROCHLORIDE	4	TP53
MECHLORETHAMINE HYDROCHLORIDE	4	TP53
ECONAZOLE NITRATE	4	TP53
TRIFLUPROMAZINE HYDROCHLORIDE	4	TP53
PROCHLORPERAZINE EDISYLATE	4	TP53

Bioactivity and enzyme data

Enzyme cohort genes (≥1 EC): 5.

Cohort genes with ChEMBL bioactivity (full, sorted by assay count)

Symbol	Assays	Type breakdown
FLT3	3,132	Binding:3096, Functional:24, ADMET:8, Toxicity:4
KIT	2,305	Binding:2242, ADMET:32, Functional:22, Toxicity:9
TP53	869	Binding:775, ADMET:83, Functional:10, Toxicity:1
IDH1	488	Binding:475, Functional:12, ADMET:1
DNMT3A	120	Binding:118, ADMET:1, Functional:1
NPM1	113	Binding:108, Functional:5
IDH2	84	Binding:84
TET2	24	Binding:24
RUNX1	20	Binding:17, Functional:3
NRAS	18	Binding:18
SRSF2	8	Binding:8
U2AF1	8	Binding:8
CSF3R	3	Binding:3

Cohort enzymes (BRENDA EC)

Symbol	EC numbers	Names
DNMT3A	2.1.1.37	DNA (cytosine-5-)-methyltransferase
FLT3	2.7.10.1	receptor protein-tyrosine kinase
IDH1	1.1.1.42	isocitrate dehydrogenase (NADP+)
IDH2	1.1.1.42	isocitrate dehydrogenase (NADP+)
KIT	2.7.10.1	receptor protein-tyrosine kinase

Cohort genes with high screening signal

≥100 ChEMBL assays — a studied-ness signal; see Therapeutics for approved-drug status.

Symbol	ChEMBL assays
TP53	869
DNMT3A	120
FLT3	3,132
IDH1	488
KIT	2,305
NPM1	113

Pharmacogenomics

Cohort genes with a PharmGKB record: 18; with CPIC/DPWG dosing guidelines: 0.

No cohort gene has a CPIC/DPWG genotype-guided dosing guideline (PharmGKB).

Drug repurposing candidates

30 approved/phased drugs hit cohort targets but don’t yet appear in disease-level clinical trials. Target-inhibition rationale is strongest for cancer driver genes; a bioactivity hit is a screening signal, not a treatment claim.

Compound	Max phase	Cohort target (bioactivity)
APOMORPHINE HYDROCHLORIDE	4	RUNX1
NITROFURANTOIN	4	TP53
DIOSMIN	4	TP53
VERTEPORFIN	4	TP53
CANDESARTAN CILEXETIL	4	TP53
DIENESTROL	4	TP53
CLOTRIMAZOLE	4	TP53
COLCHICINE	4	TP53
NABUMETONE	4	TP53
SALMETEROL XINAFOATE	4	TP53
AMIODARONE HYDROCHLORIDE	4	TP53
FURAZOLIDONE	4	TP53
AMOXAPINE	4	TP53
RALOXIFENE HYDROCHLORIDE	4	TP53
NICARDIPINE HYDROCHLORIDE	4	TP53
SULCONAZOLE NITRATE	4	TP53
PYRITHIONE ZINC	4	TP53
LACTIC ACID	4	TP53
OXYMETHOLONE	4	TP53
CHLOROXINE	4	TP53
PROPIOLACTONE	4	TP53
CLOMIPRAMINE HYDROCHLORIDE	4	TP53
PHENYL AMINOSALICYLATE	4	TP53
THIORIDAZINE HYDROCHLORIDE	4	TP53
AMITRIPTYLINE HYDROCHLORIDE	4	TP53
ETHOPROPAZINE HYDROCHLORIDE	4	TP53
MECHLORETHAMINE HYDROCHLORIDE	4	TP53
ECONAZOLE NITRATE	4	TP53
TRIFLUPROMAZINE HYDROCHLORIDE	4	TP53
PROCHLORPERAZINE EDISYLATE	4	TP53

Druggability pyramid

Cohort genes binned by druggability tier (high → low):

Tier	Definition	Genes	Symbols
A	Approved (phase 4 drug)	8	RUNX1, TP53, TET2, FLT3, IDH1, IDH2, KIT, NPM1
B	Phased (≥1) drug, not yet approved	3	SRSF2, U2AF1, NRAS
C	Druggable family + PDB, no drug	2	CSF3R, DNMT3A
D	Druggable family + AlphaFold only, no drug	0
E	Difficult family or no structure, no drug	5	STAG2, WT1, ASXL1, CEBPA, FGF13

Undrugged target profiles

7 cohort genes are undrugged. Ranked by ‘starting-point quality’ (assay depth + drugged-partner adjacency).

Symbol	ChEMBL assays	Drugged partners (top 3)
WT1	0	TP53
ASXL1	0	TET2
CEBPA	0	TP53
DNMT3A	120	—
STAG2	0	—
CSF3R	3	—
FGF13	0	—

Clinical trials & evidence

Clinical trials

Clinical trials: 1,496.

Phase distribution (across all retrieved trials)

Phase	Trials
PHASE2	412
PHASE1	298
PHASE1/PHASE2	211
PHASE3	112
PHASE2/PHASE3	33
PHASE4	17
EARLY_PHASE1	17

Top trials by phase / activity

NCT	Phase	Status	Title
NCT02386800	PHASE4	ACTIVE_NOT_RECRUITING	CINC424A2X01B Rollover Protocol
NCT06370000	PHASE4	RECRUITING	Oral Azacitidine in Transplant-Eligible Patients With Acute Myeloid Leukemia (AML) Suffering From Health-Inequality
NCT06571825	PHASE4	RECRUITING	RIC Allo-HSCT vs. Venetoclax-Based Consolidation in Elderly AML Patients After First CR
NCT07044687	PHASE4	RECRUITING	Study to Assess Adverse Events and Change in Disease Activity of Oral Venetoclax in Combination With Subcutaneous (SC) or Intravenous (IV) Azacitidine in Newly Diagnosed Adult Participants With Acute Myeloid Leukemia (AML) Who Are Ineligible for Standard Induction Therapy in India
NCT07561892	PHASE4	RECRUITING	Study of the Effectiveness and Safety of Daunorubicin /Idarubicin ± Silibinin in Treating Newly Diagnosed AML (Non-M3).
NCT00199147	PHASE4	UNKNOWN	Efficacy of G-CSF-Priming in Elderly AML Patients
NCT00488709	PHASE4	COMPLETED	Fludarabine, Cytarabine, Topotecan in Treating Patients With Relapsed or Refractory Acute Myeloid Leukemia
NCT01347996	PHASE4	COMPLETED	Maintenance Therapy With Ceplene® (Histamine) and IL-2 on Immune Response and MRD in Acute Myeloid Leukemia
NCT01587430	PHASE4	UNKNOWN	3 Anthracyclines, 2 Types of Consolidation With Different ARA-C Doses and Maintenance in Adult Acute Myeloid Leukemia
NCT01819792	PHASE4	COMPLETED	Respiratory Viral Infections During Acute Myeloid Leukemia (AML)Chemotherapy Related Aplasia
NCT02024308	PHASE4	UNKNOWN	AML1-ETO Acute Myeloid Leukemia With Fludarabine and Cytarabine Chemotherapy
NCT02027064	PHASE4	UNKNOWN	Interferon for the Intervention of Molecular Relapse in t (8; 21) AML After Allo-HSCT
NCT02277847	PHASE4	UNKNOWN	Idarubicin at Different Dosages as Induction Therapy for Newly Diagnosed Acute Myeloid Leukaemia
NCT02926586	PHASE4	COMPLETED	Fludarabine and Cytarabine Versus High-dose Cytarabine for CBF-AML
NCT02933333	PHASE4	UNKNOWN	G-CSF Alone or Combination With GM-CSF on Prevention and Treatment of Infection in Children With Malignant Tumor
NCT03026842	PHASE4	UNKNOWN	Decitabine Versus Conventional Chemotherapy for Maintenance Therapy of Acute Myeloid Leukemia With t(8;21)
NCT03150134	PHASE4	UNKNOWN	Early Tapering of Immunosuppressive Agents to Immunomodulation to Improve Survival of AML Patients
NCT02521493	PHASE3	ACTIVE_NOT_RECRUITING	Response-Based Chemotherapy in Treating Newly Diagnosed Acute Myeloid Leukemia or Myelodysplastic Syndrome in Younger Patients With Down Syndrome
NCT02665065	PHASE3	ACTIVE_NOT_RECRUITING	Study of Iomab-B vs. Conventional Care in Older Subjects With Active, Relapsed or Refractory Acute Myeloid Leukemia
NCT02724163	PHASE3	RECRUITING	International Randomised Phase III Clinical Trial in Children With Acute Myeloid Leukaemia
NCT03480360	PHASE3	ACTIVE_NOT_RECRUITING	Haploidentical Allogeneic Peripheral Blood Transplantation: Examining Checkpoint Immune Regulators’ Expression
NCT03507842	PHASE3	ENROLLING_BY_INVITATION	A Prospective Randomized Comparison of HDAC Vs AD in the Induction Chemothrapy for AML.
NCT03701308	PHASE2/PHASE3	ACTIVE_NOT_RECRUITING	Daunorubicin and Cytarabine With or Without Uproleselan in Treating Older Adult Patients With Acute Myeloid Leukemia Receiving Intensive Induction Chemotherapy
NCT03839771	PHASE3	ACTIVE_NOT_RECRUITING	A Study of Ivosidenib or Enasidenib in Combination With Induction Therapy and Consolidation Therapy, Followed by Maintenance Therapy in Patients With Newly Diagnosed Acute Myeloid Leukemia or Myedysplastic Syndrome EB2, With an IDH1 or IDH2 Mutation, Respectively, Eligible for Intensive Chemotherapy
NCT03844048	PHASE3	ACTIVE_NOT_RECRUITING	An Extension Study of Venetoclax for Subjects Who Have Completed a Prior Venetoclax Clinical Trial
NCT03897127	PHASE3	ACTIVE_NOT_RECRUITING	Study of Standard Intensive Chemotherapy Versus Intensive Chemotherapy With CPX-351 in Adult Patients With Newly Diagnosed AML and Intermediate- or Adverse Genetics
NCT04027309	PHASE3	ACTIVE_NOT_RECRUITING	A Study of Gilteritinib Versus Midostaurin in Combination With Induction and Consolidation Therapy Followed by One-year Maintenance in Patients With Newly Diagnosed Acute Myeloid Leukemia or Myelodysplastic Syndromes With Excess Blasts-2 With FLT3 Mutations Eligible for Intensive Chemotherapy
NCT04168502	PHASE3	RECRUITING	Gemtuzumab Chemotherapy MRD Levels; Adult Untreated, de Novo, Fav Interm Risk AML
NCT04173533	PHASE3	ACTIVE_NOT_RECRUITING	Randomised Study of Oral Azacitidine vs Placebo Maintenance in AML or MDS Patients After Allo-SCT
NCT04217278	PHASE2/PHASE3	ACTIVE_NOT_RECRUITING	A Trial of Treatments to Assess the Effects on Outcome of Adults With AML and MDS Undergoing Allogeneic SCT
NCT04229979	PHASE3	ACTIVE_NOT_RECRUITING	Galinpepimut-S Versus Investigator’s Choice of Best Available Therapy for Maintenance in AML CR2/CRp2
NCT04256317	PHASE2/PHASE3	RECRUITING	A Multi-phase Study of ASTX030 (Azacitidine and Cedazuridine) in Myeloid Neoplasm Alone or in Combination With Venetoclax in AML (AZTOUND Study)
NCT04293562	PHASE3	RECRUITING	A Study to Compare Standard Chemotherapy to Therapy With CPX-351 and/or Gilteritinib for Patients With Newly Diagnosed AML With or Without FLT3 Mutations
NCT04628026	PHASE3	RECRUITING	Phase III Study of Induction and Consolidation Chemotherapy With Venetoclax in Patients With Newly Diagnosed AML or MDS-EB-2
NCT04708054	PHASE2/PHASE3	RECRUITING	Venetoclax to Improve Outcomes of Fractionated Busulfan Regimen in Patients With High-Risk AML and MDS
NCT05183035	PHASE3	RECRUITING	Venetoclax in Children With Relapsed Acute Myeloid Leukemia (AML)
NCT05316701	PHASE3	ACTIVE_NOT_RECRUITING	Precision-T: A Randomized Study of Orca-T in Recipients Undergoing Allogeneic Transplantation for Hematologic Malignancies
NCT05356169	PHASE2/PHASE3	ACTIVE_NOT_RECRUITING	Intensive Therapy Combined With Venetoclax for Adult Acute Myeloid Leukemia
NCT05457556	PHASE3	ACTIVE_NOT_RECRUITING	Mismatched Related Donor Versus Matched Unrelated Donor Stem Cell Transplantation for Children, Adolescents, and Young Adults With Acute Leukemia or Myelodysplastic Syndrome
NCT05805098	PHASE2/PHASE3	RECRUITING	Venetoclax Combined With Homoharringtonine and Cytarabine in Induction for AML

Drugs tested across these trials (top 30)

Molecule	Max phase	Trials referencing
FLUDARABINE PHOSPHATE	4	109
DAUNORUBICIN	4	58
GEMTUZUMAB OZOGAMICIN	4	29
MITOXANTRONE	4	22
AZACITIDINE	4	21
CLADRIBINE	4	21
CLOFARABINE	4	21
GILTERITINIB	4	19
MIDOSTAURIN	4	19
VENETOCLAX	4	19
IDARUBICIN	4	18
DECITABINE	4	17
QUIZARTINIB	4	16
CEDAZURIDINE	4	13
BUSULFAN	4	12
ENASIDENIB	4	12
THIOTEPA	4	12
IVOSIDENIB	4	10
PLERIXAFOR	4	10
TREOSULFAN	4	8
TRETINOIN	4	8
REVUMENIB	4	7
GLASDEGIB	4	6
TAGRAXOFUSP	4	6
CYTARABINE	4	5
OLUTASIDENIB	4	5
OMACETAXINE MEPESUCCINATE	4	5
SELINEXOR	4	5
ARSENIC TRIOXIDE	4	4
ASPARAGINASE	4	4

Precision-medicine subtype map (CIViC)

Drug × molecular subtype: 170 predictive associations from 199 curated evidence items; also 130 prognostic, 35 oncogenic, 26 diagnostic, 16 predisposing.

Molecular subtype	Therapy	Effect	Level	CIViC
FLT3 ITD	Gilteritinib	Sensitivity/Response	CIViC A	EID12621 +2
IDH1 R132 AND IDH1 R132L	Ivosidenib	Sensitivity/Response	CIViC A	EID12899 +1
IDH2 Mutation	Enasidenib	Sensitivity/Response	CIViC A	EID5069 +1
BCR::ABL1 Fusion	Imatinib	Sensitivity/Response	CIViC A	EID259
FLT3 D835 OR FLT3 I836	Gilteritinib	Sensitivity/Response	CIViC A	EID12622
FLT3 ITD AND FLT3 D835 AND FLT3 I836	Gilteritinib	Sensitivity/Response	CIViC A	EID11260
FLT3 ITD AND FLT3 D835 AND FLT3 I836	Chemotherapy + Midostaurin	Sensitivity/Response	CIViC A	EID11261
FLT3 ITD OR FLT3 D835 OR FLT3 I836	Gilteritinib	Sensitivity/Response	CIViC A	EID7728
FLT3 Mutation	Midostaurin	Sensitivity/Response	CIViC A	EID5261
IDH1 Mutation	Ivosidenib + Azacitidine	Sensitivity/Response	CIViC A	EID10313
IDH1 Mutation	Ivosidenib	Sensitivity/Response	CIViC A	EID7278
FLT3 ITD	Sorafenib	Sensitivity/Response	CIViC B	EID1040 +2
FLT3 F691L	Pexidartinib	Sensitivity/Response	CIViC B	EID8674 +1
FLT3 ITD	Midostaurin	Sensitivity/Response	CIViC B	EID7061 +1
FLT3 ITD	Sorafenib + Hematopoietic Cell Transplantation	Sensitivity/Response	CIViC B	EID9069 +1
FLT3 Mutation	Nilotinib	Sensitivity/Response	CIViC B	EID5575 +1
NPM1 EXON 11 MUTATION	Tretinoin	Sensitivity/Response	CIViC B	EID137 +1
CEBPA Mutation	Tretinoin	Sensitivity/Response	CIViC B	EID122
DNMT3A Mutation	Decitabine	Sensitivity/Response	CIViC B	EID1587
DNMT3A R882	Idarubicin	Sensitivity/Response	CIViC B	EID18
FLT3 D835 & I836	Lestaurtinib + Quizartinib + Sorafenib + FLT3/ABL/Aurora Kinase Inhibitor KW-2449	Sensitivity/Response	CIViC B	EID8925
FLT3 ITD	Lestaurtinib	Sensitivity/Response	CIViC B	EID297
FLT3 ITD	Pacritinib	Sensitivity/Response	CIViC B	EID9217
FLT3 ITD & TKD MUTATIONS	Midostaurin	Sensitivity/Response	CIViC B	EID8516
FLT3 Mutation	Gilteritinib	Sensitivity/Response	CIViC B	EID7283
FLT3 TKD MUTATION	Midostaurin	Sensitivity/Response	CIViC B	EID1295
IDH1 R132C	Ivosidenib	Sensitivity/Response	CIViC B	EID2331
IDH1 R132S	Ivosidenib	Sensitivity/Response	CIViC B	EID2340
KIT RS3733542	Selumetinib	Sensitivity/Response	CIViC B	EID1136
NPM1 EXON 11 MUTATION	Daunorubicin	Sensitivity/Response	CIViC B	EID147

+140 more predictive associations (showing top 30 by evidence level).