NPM1

Summary

NPM1 (nucleophosmin 1, HGNC:7910) is a protein-coding gene on chromosome 5q35.1, encoding Nucleophosmin (P06748). 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. In precision oncology, NPM1 EXON 11 MUTATION confers sensitivity to Tretinoin in Acute Myeloid Leukemia (CIViC Level B); 8 further curated variant–drug associations are listed below. It is a selective cancer dependency (DepMap: 81.3% of cell lines).

The protein encoded by this gene is involved in several cellular processes, including centrosome duplication, protein chaperoning, and cell proliferation. The encoded phosphoprotein shuttles between the nucleolus, nucleus, and cytoplasm, chaperoning ribosomal proteins and core histones from the nucleus to the cytoplasm. This protein is also known to sequester the tumor suppressor ARF in the nucleolus, protecting it from degradation until it is needed. Mutations in this gene are associated with acute myeloid leukemia. Dozens of pseudogenes of this gene have been identified.

Source: NCBI Gene 4869 — RefSeq curated summary.

At a glance

• Gene–disease (curated): dyskeratosis congenita (Strong, GenCC) — +1 more curated relationship
• GWAS associations: 4
• Clinical variants (ClinVar): 90 total — 8 pathogenic
• Phenotypes (HPO): 99
• Druggable target: yes — 5 molecules with ChEMBL bioactivity
• Precision-oncology evidence (CIViC): 9 curated variant–drug associations
• Cancer driver (intOGen): activating (oncogene-like) across 1 cancer types
• Cancer dependency (DepMap): dependent in 81.3% of screened cell lines
• Transcription factor: yes — 12 downstream targets (CollecTRI)
• MANE Select transcript: NM_002520

Identifiers

Gene identifiers

Field	Value
HGNC ID	HGNC:7910
Approved symbol	NPM1
Name	nucleophosmin 1
Location	5q35.1
Locus type	gene with protein product
Status	Approved
Aliases	B23, NPM
Ensembl gene	ENSG00000181163
Ensembl biotype	protein_coding
OMIM	164040
Entrez	4869

Gene structure

Transcript identifiers

Ensembl transcripts: 64 — 44 protein_coding, 16 retained_intron, 4 nonsense_mediated_decay

ENST00000296930, ENST00000351986, ENST00000393820, ENST00000517671, ENST00000518587, ENST00000519955, ENST00000521260, ENST00000521672, ENST00000521710, ENST00000523339, ENST00000523622, ENST00000524204, ENST00000676504, ENST00000676589, ENST00000676613, ENST00000676625, ENST00000677297, ENST00000677325, ENST00000677357, ENST00000677467, ENST00000677600, ENST00000677672, ENST00000677682, ENST00000677741, ENST00000677904, ENST00000677907, ENST00000678186, ENST00000678267, ENST00000678280, ENST00000678774, ENST00000679006, ENST00000679190, ENST00000679233, ENST00000867397, ENST00000867398, ENST00000923456, ENST00000923457, ENST00000923458, ENST00000923459, ENST00000923460, ENST00000923461, ENST00000923462, ENST00000923463, ENST00000923464, ENST00000923465, ENST00000923466, ENST00000923467, ENST00000923468, ENST00000923469, ENST00000923470, ENST00000923471, ENST00000923472, ENST00000923473, ENST00000923474, ENST00000923475, ENST00000923476, ENST00000923477, ENST00000923478, ENST00000923479, ENST00000923480, ENST00000923481, ENST00000923482, ENST00000923483, ENST00000964720

RefSeq mRNA: 7 — MANE Select: NM_002520 NM_001037738, NM_001355006, NM_001355007, NM_001355009, NM_001355010, NM_002520, NM_199185

CCDS: CCDS43399, CCDS4376, CCDS4377, CCDS93824

Canonical transcript exons

ENST00000296930 — 11 exons

Exon	Start	End
ENSE00001026390	171405302	171405403
ENSE00001026397	171407700	171407774
ENSE00001084445	171400153	171400210
ENSE00001084451	171400839	171400925
ENSE00001084453	171392914	171392978
ENSE00001084454	171392710	171392816
ENSE00003517265	171390051	171390130
ENSE00003536019	171391305	171391424
ENSE00003625205	171391706	171391799
ENSE00003906984	171387849	171388006
ENSE00003909747	171410527	171410900

Expression profiles

Bgee: expression breadth ubiquitous, 276 present calls, max score 99.78.

FANTOM5 (CAGE): breadth ubiquitous, TPM avg 326.0238 / max 6404.1810, expressed in 1828 samples.

FANTOM5 promoters (1 alternative TSS)

Promoter ID	TPM avg	Samples expressed
60178	326.0238	1828

Top tissues by expression

284 total, by Bgee expression score (0-100, higher = more expressed):

Tissue	Anatomy ID	Expression score	Quality
calcaneal tendon	UBERON:0003701	99.78	gold quality
left ovary	UBERON:0002119	99.70	gold quality
ventricular zone	UBERON:0003053	99.67	gold quality
right ovary	UBERON:0002118	99.66	gold quality
endocervix	UBERON:0000458	99.64	gold quality
body of uterus	UBERON:0009853	99.63	gold quality
ganglionic eminence	UBERON:0004023	99.62	gold quality
body of pancreas	UBERON:0001150	99.61	gold quality
right uterine tube	UBERON:0001302	99.56	gold quality
right testis	UBERON:0004534	99.56	gold quality
ectocervix	UBERON:0012249	99.56	gold quality
left testis	UBERON:0004533	99.54	gold quality
cortical plate	UBERON:0005343	99.53	gold quality
adrenal tissue	UBERON:0018303	99.52	gold quality
colonic epithelium	UBERON:0000397	99.51	gold quality
mucosa of stomach	UBERON:0001199	99.50	gold quality
left uterine tube	UBERON:0001303	99.50	gold quality
gall bladder	UBERON:0002110	99.49	gold quality
metanephros cortex	UBERON:0010533	99.49	gold quality
stromal cell of endometrium	CL:0002255	99.48	gold quality
muscle layer of sigmoid colon	UBERON:0035805	99.48	gold quality
body of stomach	UBERON:0001161	99.47	gold quality
popliteal artery	UBERON:0002250	99.47	gold quality
tibial artery	UBERON:0007610	99.47	gold quality
left lobe of thyroid gland	UBERON:0001120	99.46	gold quality
islet of Langerhans	UBERON:0000006	99.45	gold quality
rectum	UBERON:0001052	99.44	gold quality
right lung	UBERON:0002167	99.43	gold quality
adenohypophysis	UBERON:0002196	99.43	gold quality
descending thoracic aorta	UBERON:0002345	99.42	gold quality

Single-cell (SCXA)

Detected in 35 experiment(s), a significant marker in 16.

Experiment	Marker?	Max mean expression
E-MTAB-10042	yes	3577.33
E-CURD-112	yes	3454.76
E-CURD-98	yes	3444.88
E-HCAD-4	yes	2629.11
E-MTAB-9801	yes	2281.16
E-HCAD-1	yes	2238.93
E-CURD-122	yes	1775.80
E-GEOD-125970	yes	1218.02
E-MTAB-9467	yes	69.98
E-MTAB-9221	yes	54.02
E-CURD-88	yes	35.89
E-CURD-46	yes	35.00
E-GEOD-134144	yes	31.27
E-HCAD-13	yes	28.22
E-MTAB-9067	yes	22.16

Regulation

Is transcription factor: yes

Downstream targets (CollecTRI)

12 targets.

Target	Regulation
ATF5	Repression
BRD4	Unknown
CCND1	Unknown
CDKN1A	Activation
DNMT1	Unknown
ELF4	Unknown
FBP1	Repression
HOXA9	Repression
PTPN6	Activation
RPL3	Unknown
TAF1A	Activation
USP7	Unknown

Upstream regulators (CollecTRI, top): AP1, ATF5, CEBPB, CTNNB1, ELF4, GLI1, HIF1A, JUN, JUNB, MYC, RARA, STAT5A, TFAP2A, TP53, TXK, YY1

miRNA regulators (miRDB)

52 targeting NPM1, top 30 by miRDB confidence (max_score; target_count = how many genes the miRNA targets in total — lower means more specific):

miRNA	Max score	Avg score	miRNA target_count
HSA-MIR-200B-3P	100.00	73.31	2693
HSA-MIR-200C-3P	100.00	73.35	2685
HSA-MIR-429	100.00	73.44	2698
HSA-MIR-3646	100.00	73.56	5283
HSA-MIR-4682	100.00	68.89	1258
HSA-MIR-12136	99.98	72.81	5713
HSA-MIR-1229-3P	99.97	66.49	906
HSA-MIR-548AJ-3P	99.96	73.38	5345
HSA-MIR-548X-3P	99.96	73.38	5345
HSA-MIR-548AA	99.96	70.64	3753
HSA-MIR-548AP-3P	99.96	70.64	3753
HSA-MIR-548T-3P	99.96	70.64	3753
HSA-MIR-23A-3P	99.95	74.24	3163
HSA-MIR-23B-3P	99.95	74.24	3163
HSA-MIR-23C	99.95	73.92	3192
HSA-MIR-548J-3P	99.94	72.61	4881
HSA-MIR-548AE-3P	99.93	72.66	4867
HSA-MIR-548AH-3P	99.93	72.54	4872
HSA-MIR-548AM-3P	99.93	72.54	4872
HSA-MIR-548AQ-3P	99.93	72.66	4867
HSA-MIR-205-3P	99.92	69.92	3165
HSA-MIR-5680	99.91	69.83	3421
HSA-MIR-7-1-3P	99.91	71.53	4384
HSA-MIR-7-2-3P	99.91	71.40	4394
HSA-MIR-182-5P	99.87	74.03	2589
HSA-MIR-6739-5P	99.80	67.87	2806
HSA-MIR-6733-5P	99.74	67.94	2759
HSA-MIR-4729	99.69	72.18	4233
HSA-MIR-587	99.64	70.86	2611
HSA-MIR-3153	99.55	67.59	2337

Functional genomics

DepMap (CRISPR cell-line fitness): dependent in 81.3% of screened cell lines.

Literature-anchored findings (GeneRIF, showing 40)

• overexpressed in acute myeloid leukemia while translocations associated with this gene are absent (PMID:12031912)
• marker of proliferation activity of human peripheral lymphocytes (PMID:12057857)
• NPM is a crucial regulator of p53 (PMID:12080348)
• involvement of NPM/B23 in the acute response of mammalian cells to environmental stress, such as UV rays (PMID:12374805)
• overexpression of NPM suppressed PKR activity, enhanced protein synthesis, and inhibited apoptosis. Fanconi anemia lymphoblasts expressed low levels of NPM, which correlated with high ground-state activation of PKR and hypersensitivity to apoptotic cues (PMID:12882984)
• These findings suggest that an NPM/MLF1 fusion is the primary molecular abnormality in t(3;5) MDS and AML with multilineage dysplasia, and that cases with NPM/MLF1 may be clinically distinct from other MDS-associated disease (PMID:14506644)
• unique B23 conformation in hepatoma (PMID:14519847)
• Src-kinases, particularly pp60(c-src), associate with and are activated by NPM-ALK expression in various cells, and in cell lines established from patients with large cell lymphoma (PMID:14563642)
• Results reveal a molecular mechanism of ARF in regulating ribosome biogenesis and cell proliferation via inhibiting B23. (PMID:14636574)
• NPM/ALK-mediated induction of Bcl-XL (PMID:14656879)
• cleavage of B23 by granzyme b in intact vascular smooth muscle cells is dependent upon both cell type and phenotype (PMID:14730621)
• These data suggest that nucleophosmin is an early responder to DNA damage that prevents premature activation of p53. (PMID:15082766)
• nucleophosmin is an NF-kappaB co-activator for the induction of the human SOD2 gene (PMID:15087454)
• BRCA1-BARD1 catalyzes the polyubiquitination of nucleolar phosphoprotein nucleophosmin/B23 (PMID:15184379)
• identification of Ser-4 in B23 as a major physiological substrate of Polo-like kinase 1 (PMID:15190079)
• NPM inhibits hypoxia-induced p53 phosphorylation at Ser-15 and interacts with p53 in hypoxic cells; hypoxia-driven cancer progression may require increased expression of NPM to suppress p53 activation and maintain cell survival (PMID:15310764)
• Aloe-emodin caused increase in the amount of proform and fragment of nucleophosmin in cytoplasm may be one of the important events for aloe-emodin-induced H460 cell apoptosis pf mpm-small cell lung cancer. (PMID:15514966)
• c-Myc-mediated expression of NPM decreases during retinoic acid-induced differentiation of HL-60 cells. (PMID:15589822)
• a novel association of B23 and Gadd45a and implicate B23 as an important regulator in Gadd45a nuclear import. (PMID:15644315)
• Cytoplasmic NPM is a characteristic feature of a large subgroup of patients with AML who have a normal karyotype, NPM gene mutations, and responsiveness to induction chemotherapy. (PMID:15659725)
• CDK2-BRCA1-NPM pathway coordinately functions in cell growth and tumor progression pathways. (PMID:15665273)
• NPM inhibits ARF’s p53-dependent activity by targeting it to nucleoli and impairing ARF-Mdm2 association. (PMID:15684379)
• The molecular signature of cytoplasmic NPM+ AML includes up-regulation of several genes putatively involved in the maintenance of a stem-cell phenotype, suggesting that cytoplasmic NPM+ AML may derive from a multipotent hematopoietic progenitor. (PMID:15831697)
• base sequences are given for NPM1 mutated childhood AML patients; consistent deletion/insertion features are presented (PMID:15870172)
• polyamine depletion increased expression of the NPM gene and enhances NPM nuclear translocation and increased NPM interacts with and stabilizes p53, leading to inhibition of IEC-6 cell proliferation (PMID:15872011)
• NPM inhibits ionizing irradiation-induced p53 transactivation, and interacts with p53 in hematopoietic cells. (PMID:15964625)
• the NPM1 mutation is not necessarily an early event during leukemogenesis; the NPM1 mutation was a favorable factor for achieving complete remission but was associated with a high relapse rate (PMID:15994285)
• Nucleophosmin (NPM) may be a Ran-Crm1 substrate that controls centrosome duplication. (PMID:16041368)
• results indicate that HCV core can recruit B23 and p300 to relieve the repression effect of YY1 on B23 promoter activity (PMID:16170350)
• Enforced expression of an NPM cytoplasmic mutant acutely attenuates p53-dependent and -independent functions of Arf tumor suppressor protein, and also affects endogenous NPM localization. (PMID:16205118)
• These findings indicate the involvement of nucleophosmin in the regulation of pre-mRNA processing, and its activity is controlled by CDK2-mediated phosphorylation on Thr(199). (PMID:16376875)
• FRGY2a and YB1 disassemble nucleoli by sequestering B23, which is associated with pre-ribosomes and other structurally important nucleolar components (PMID:16415342)
• both alterations are crucial for acute myeloid leukemia NPM mutant export from nucleus to cytoplasm; NPM leukemic mutants in turn recruit the wild-type NPM from nucleoli to nucleoplasm and cytoplasm (PMID:16455950)
• NPM1 mutations represent a common genetic abnormality in adult acute myeloid leukemia and appears to identify patients with improved response toward treatment. (PMID:16455956)
• The sensitive RQ-PCR assays for NPM1 mutations can now monitor and quantify MRD in AML patients with normal karyotype and NPM1 gene mutations. (PMID:16541144)
• results suggest that B23 plays an important role in the intracellular localization of the core protein and replication of Japanese encephalitis virus (PMID:16547420)
• NPM1 mutations may be also involved in the pathogenesis of myelodysplastic syndromes. (PMID:16678898)
• the functional integrity of the B23 core motif is required for stability, efficient nucleolar localization as well as ARF binding (PMID:16679321)
• constitutive expression of C/EBPbeta in ALK-positive anaplastic large cell lymphoma and its relationship to NPM-ALK (PMID:16709933)
• new exon-12 NPM mutations in acute myeloid leukemia (PMID:16720834)

Cross-species orthologs

3 orthologs

Organism	Symbol	Gene ID
danio_rerio	npm1a	ENSDARG00000014329
drosophila_melanogaster	Nlp	FBGN0016685
drosophila_melanogaster	Nph	FBGN0039735

Paralogs (2): NPM3 (ENSG00000107833), NPM2 (ENSG00000158806)

Protein

Protein identifiers

Nucleophosmin — P06748 (reviewed: P06748)

Alternative names: Nucleolar phosphoprotein B23, Nucleolar protein NO38, Numatrin

All UniProt accessions (15): A0A0S2Z491, A0A0S2Z4G7, A0A140VJQ2, A0A7I2V2X9, A0A7I2V3G5, P06748, A0A7I2V3U2, A0A7I2V433, A0A7I2V4G8, A0A7I2V579, A0A7I2V5J8, A0A7I2V5S2, A0A7I2YQC0, E5RGW4, E5RI98

UniProt curated annotations — full annotation on UniProt →

Function. 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. Binds ribosome presumably to drive ribosome nuclear export. Associated with nucleolar ribonucleoprotein structures and bind single-stranded nucleic acids. Acts as a chaperonin for the core histones H3, H2B and H4. Stimulates APEX1 endonuclease activity on apurinic/apyrimidinic (AP) double-stranded DNA but inhibits APEX1 endonuclease activity on AP single-stranded RNA. May exert a control of APEX1 endonuclease activity within nucleoli devoted to repair AP on rDNA and the removal of oxidized rRNA molecules. In concert with BRCA2, regulates centrosome duplication. Regulates centriole duplication: phosphorylation by PLK2 is able to trigger centriole replication. Negatively regulates the activation of EIF2AK2/PKR and suppresses apoptosis through inhibition of EIF2AK2/PKR autophosphorylation. Antagonizes the inhibitory effect of ATF5 on cell proliferation and relieves ATF5-induced G2/M blockade. In complex with MYC enhances the transcription of MYC target genes. May act as chaperonin or cotransporter in the nucleolar localization of transcription termination factor TTF1.

Subunit / interactions. Decamer formed by two pentameric rings associated in a head-to-head fashion. Disulfide-linked dimers under certain conditions. The SWAP complex consists of NPM1, NCL, PARP1 and SWAP70. Interacts with NSUN2 and SENP3. Interacts with the methylated form of RPS10. Interacts (via N-terminal domain) with APEX1; the interaction is RNA-dependent and decreases in hydrogen peroxide-damaged cells. Interacts with isoform 1 of NEK2. Interacts with ROCK2 and BRCA2. Interacts with RPGR. Interacts with CENPW. Interacts with EIF2AK2/PKR. Interacts with CEBPA (isoform 4). Interacts with DDX31; this interaction prevents interaction between NPM1 and HDM2. Interacts with MYC; competitive with NOP53. Interacts with NOP53; the interaction is direct and competitive with MYC. Interacts with LRRC34. Interacts with RRP1B. Interacts with NPM3. Interacts with ALKBH2. Interacts with TTF1 (via C-terminal region). Interacts with NOP2. Interacts with ARID3C (via REKLES DOMAIN); the interaction mediates ARID3C nuclear shuttling. (Microbial infection) Interacts with hepatitis delta virus S-HDAg. (Microbial infection) Interacts with HTLV1 Rex protein (via N-terminal nuclear localization signal).

Subcellular location. Nucleus. Nucleolus. Nucleoplasm. Cytoplasm. Cytoskeleton. Microtubule organizing center. Centrosome.

Post-translational modifications. Acetylated at C-terminal lysine residues, thereby increasing affinity to histones. ADP-ribosylated. Phosphorylated at Ser-4 by PLK1 and PLK2. Phosphorylation at Ser-4 by PLK2 in S phase is required for centriole duplication and is sufficient to trigger centriole replication. Phosphorylation at Ser-4 by PLK1 takes place during mitosis. Phosphorylated by CDK2 at Ser-125 and Thr-199. Phosphorylation at Thr-199 may trigger initiation of centrosome duplication. Phosphorylated by CDK1 at Thr-199, Thr-219, Thr-234 and Thr-237 during cell mitosis. When these four sites are phosphorylated, RNA-binding activity seem to be abolished. May be phosphorylated at Ser-70 by NEK2. The Thr-199 phosphorylated form has higher affinity for ROCK2. CDK6 triggers Thr-199 phosphorylation when complexed to Kaposi’s sarcoma herpesvirus (KSHV) V-cyclin, leading to viral reactivation by reducing viral LANA levels. Sumoylated by ARF. Ubiquitinated. Ubiquitination leads to proteasomal degradation. Deubiquitinated by USP36.

Disease relevance. A chromosomal aberration involving NPM1 is found in a form of non-Hodgkin lymphoma. Translocation t(2;5)(p23;q35) with ALK. The resulting chimeric NPM1-ALK protein homodimerize and the kinase becomes constitutively activated. A chromosomal aberration involving NPM1 is found in a form of acute promyelocytic leukemia. Translocation t(5;17)(q32;q11) with RARA. A chromosomal aberration involving NPM1 is a cause of myelodysplastic syndrome (MDS). Translocation t(3;5)(q25.1;q34) with MLF1. Defects in NPM1 are associated with acute myelogenous leukemia (AML). Mutations in exon 12 affecting the C-terminus of the protein are associated with an aberrant cytoplasmic location.

Similarity. Belongs to the nucleoplasmin family.

Isoforms (3)

UniProt ID	Names	Canonical?
P06748-1	1	yes
P06748-2	2
P06748-3	3

RefSeq proteins (7): NP_001032827, NP_001341935, NP_001341936, NP_001341938, NP_001341939, NP_002511, NP_954654 (=MANE)

Domains & families (InterPro)

ID	Name	Type
IPR004301	Nucleoplasmin	Family
IPR024057	Nucleoplasmin_core_dom	Domain
IPR032569	NPM1_C	Domain
IPR036824	Nucleoplasmin_core_dom_sf	Homologous_superfamily

Pfam: PF03066, PF16276

UniProt features (117 total): modified residue 36, sequence conflict 17, cross-link 17, mutagenesis site 17, strand 8, region of interest 5, compositionally biased region 5, helix 3, site 3, short sequence motif 2, splice variant 2, chain 1, turn 1

Structure

Experimental structures (PDB)

8 structures.

PDB	Method	Resolution (Å)
7OBG	X-RAY DIFFRACTION	1.8
7OBH	X-RAY DIFFRACTION	2
5EHD	X-RAY DIFFRACTION	2.55
2P1B	X-RAY DIFFRACTION	2.75
8AH2	X-RAY DIFFRACTION	2.9
8AS5	ELECTRON MICROSCOPY	3.53
2LLH	SOLUTION NMR
2VXD	SOLUTION NMR

Predicted structure (AlphaFold)

Model	pLDDT	Fraction very-high
AF-P06748-F1	73.42	0.38

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 (3): 55 (interaction between pentamers); 80 (interaction between pentamers); 175–176 (breakpoint for translocation to form npm1-mlf1)

Post-translational modifications (53): 254, 257, 1, 4, 10, 32, 43, 67, 70, 75, 95, 125, 137, 139, 150, 154, 199, 207, 212, 219 …

Mutagenesis-validated functional residues (17):

Position	Phenotype
4	abolishes phosphorylation by plk2 and impairs centriole duplication.
4	mimicks phosphorylation state, inducing accumulation of centrioles.
95	does not affect phosphorylation by plk2.
125	does not affect phosphorylation by plk2.
199	partial loss of phosphorylation. does not affect phosphorylation by plk2.
219	partial loss of phosphorylation.
234	partial loss of phosphorylation; when associated with a-237.
237	partial loss of phosphorylation.
248	partial destabilization of the structure.
250	increase in the stabilization of the structure.
263	increase in the stabilization of the structure and partial delocalization to the nucleoplasm. complete delocalization to
263	no change in the sumoylation level.
267	increase in the stabilization of the structure and complete delocalization to the nucleoplasm. complete delocalization t
268	complete destabilization of the structure and loss of nucleolus localization; when associated with a-276.
276	complete destabilization of the structure and loss of nucleolus localization; when associated with a-268.
288	complete destabilization of the structure; when associated with a-290.
290	partial destabilization of the structure. complete destabilization of the structure; when associated with a-288.

Function

Pathways and Gene Ontology

Reactome pathways

10 pathways

ID	Pathway
R-HSA-180746	Nuclear import of Rev protein
R-HSA-3899300	SUMOylation of transcription cofactors
R-HSA-606279	Deposition of new CENPA-containing nucleosomes at the centromere
R-HSA-6804115	TP53 regulates transcription of additional cell cycle genes whose exact role in the p53 pathway remain uncertain
R-HSA-8869496	TFAP2A acts as a transcriptional repressor during retinoic acid induced cell differentiation
R-HSA-9692914	SARS-CoV-1-host interactions
R-HSA-9700645	ALK mutants bind TKIs
R-HSA-9725370	Signaling by ALK fusions and activated point mutants
R-HSA-9725371	Nuclear events stimulated by ALK signaling in cancer
R-HSA-9833482	PKR-mediated signaling

MSigDB gene sets: 0 (showing top):

GO Biological Process (50): ribosomal large subunit export from nucleus (GO:0000055), ribosomal small subunit export from nucleus (GO:0000056), regulation of cell growth (GO:0001558), DNA repair (GO:0006281), nucleosome assembly (GO:0006334), chromatin remodeling (GO:0006338), protein import into nucleus (GO:0006606), cell volume homeostasis (GO:0006884), intracellular protein transport (GO:0006886), nucleocytoplasmic transport (GO:0006913), centrosome cycle (GO:0007098), signal transduction (GO:0007165), intracellular protein localization (GO:0008104), positive regulation of cell population proliferation (GO:0008284), negative regulation of cell population proliferation (GO:0008285), regulation of centrosome duplication (GO:0010824), positive regulation of centrosome duplication (GO:0010825), negative regulation of centrosome duplication (GO:0010826), macrophage differentiation (GO:0030225), positive regulation of protein ubiquitination (GO:0031398), cellular response to UV (GO:0034644), ribosome assembly (GO:0042255), ribosomal large subunit biogenesis (GO:0042273), ribosomal small subunit biogenesis (GO:0042274), negative regulation of apoptotic process (GO:0043066), regulation of DNA damage response, signal transduction by p53 class mediator (GO:0043516), negative regulation of protein kinase activity by regulation of protein phosphorylation (GO:0044387), positive regulation of translation (GO:0045727), positive regulation of DNA-templated transcription (GO:0045893), positive regulation of transcription by RNA polymerase II (GO:0045944), regulation of centriole replication (GO:0046599), negative regulation of mRNA splicing, via spliceosome (GO:0048025), protein stabilization (GO:0050821), obsolete positive regulation of NF-kappaB transcription factor activity (GO:0051092), regulation of eIF2 alpha phosphorylation by dsRNA (GO:0060735), cellular senescence (GO:0090398), regulation of mRNA stability involved in cellular response to UV (GO:1902629), positive regulation of cell cycle G2/M phase transition (GO:1902751), positive regulation of protein localization to nucleolus (GO:1904751), positive regulation of biosynthetic process (GO:0009891)

GO Molecular Function (19): core promoter sequence-specific DNA binding (GO:0001046), chromatin binding (GO:0003682), transcription coactivator activity (GO:0003713), RNA binding (GO:0003723), protein kinase inhibitor activity (GO:0004860), rRNA binding (GO:0019843), protein kinase binding (GO:0019901), Tat protein binding (GO:0030957), histone binding (GO:0042393), protein homodimerization activity (GO:0042803), ribosomal large subunit binding (GO:0043023), ribosomal small subunit binding (GO:0043024), NF-kappaB binding (GO:0051059), obsolete unfolded protein binding (GO:0051082), molecular adaptor activity (GO:0060090), DNA-binding transcription factor binding (GO:0140297), nucleic acid binding (GO:0003676), protein binding (GO:0005515), identical protein binding (GO:0042802)

GO Cellular Component (17): granular component (GO:0001652), nucleus (GO:0005634), nucleoplasm (GO:0005654), nucleolus (GO:0005730), cytoplasm (GO:0005737), centrosome (GO:0005813), cytosol (GO:0005829), focal adhesion (GO:0005925), large ribosomal subunit (GO:0015934), small ribosomal subunit (GO:0015935), membrane (GO:0016020), nuclear speck (GO:0016607), spindle pole centrosome (GO:0031616), protein-containing complex (GO:0032991), protein-DNA complex (GO:0032993), ribonucleoprotein complex (GO:1990904), cytoskeleton (GO:0005856)

Reactome top-level categories

Rollup of top-9 pathways:

Category	Pathways
Signaling by ALK in cancer	2
Interactions of Rev with host cellular proteins	1
SUMO E3 ligases SUMOylate target proteins	1
Nucleosome assembly	1
TP53 Regulates Transcription of Cell Cycle Genes	1
Transcriptional regulation by the AP-2 (TFAP2) family of transcription factors	1
SARS-CoV-1 Infection	1
Signaling by ALK fusions and activated point mutants	1
Antimicrobial mechanism of IFN-stimulated genes	1

GO top-level categories

Rollup of top GO terms by namespace:

Category	Terms
cellular anatomical structure	5
binding	4
centrosome duplication	3
ribosomal subunit export from nucleus	2
chromatin organization	2
cell population proliferation	2
regulation of cell population proliferation	2
regulation of centrosome duplication	2
RNA polymerase II-specific DNA-binding transcription factor binding	2
protein binding	2
ribosome binding	2
nuclear lumen	2
intracellular membraneless organelle	2
ribosomal subunit	2
protein-containing complex	2
cell growth	1
regulation of growth	1
regulation of cellular component organization	1
DNA metabolic process	1
DNA damage response	1
nucleosome organization	1
protein-DNA complex assembly	1
intracellular protein transport	1
protein localization to nucleus	1
import into nucleus	1
establishment of protein localization to organelle	1
regulation of cell size	1
cellular homeostasis	1
intracellular protein localization	1
protein transport	1
intracellular transport	1
nuclear transport	1
cell cycle process	1
microtubule organizing center organization	1
cell communication	1
cellular process	1
signaling	1
regulation of cellular process	1
cellular response to stimulus	1
macromolecule localization	1

Protein interactions and networks

STRING

4872 interactions, top by confidence (×1000):

Protein A	Protein B	Partner UniProt	Score
NPM1	NUCLEOLIN	P19338	999
NPM1	CDKN2A	P42771	995
NPM1	ALK	Q9UM73	990
NPM1	AGO2	Q9UKV8	989
NPM1	TP53	P04637	967
NPM1	PARP1	P09874	950
NPM1	FLT3	P36888	935
NPM1	RARA	P10276	915
NPM1	CTCF	P49711	914
NPM1	MDM2	Q00987	880
NPM1	FBL	P22087	862
NPM1	SENP3	Q9H4L4	858
NPM1	SURF6	O75683	857
NPM1	ASXL1	Q8IXJ9	853
NPM1	HJURP	Q8NCD3	852

IntAct

566 interactions, top by confidence:

A	B	Type	Score
PINX1	NPM1	psi-mi:“MI:0915”(physical association)	0.810
PINX1	NPM1	psi-mi:“MI:0407”(direct interaction)	0.810
PINX1	NPM1	psi-mi:“MI:0403”(colocalization)	0.810
NPM2	NPM1	psi-mi:“MI:0915”(physical association)	0.740
NPM1	NPM2	psi-mi:“MI:0915”(physical association)	0.740
RRP1B	NPM1	psi-mi:“MI:0914”(association)	0.710
NPM1	USP7	psi-mi:“MI:0915”(physical association)	0.700
USP7	NPM1	psi-mi:“MI:0915”(physical association)	0.700
NPM1	USP7	psi-mi:“MI:2364”(proximity)	0.700
H4C16	HAT1	psi-mi:“MI:0914”(association)	0.700
PINX1	TERT	psi-mi:“MI:0914”(association)	0.680
LARP4	NPM1	psi-mi:“MI:0915”(physical association)	0.670
RPS6	NPM1	psi-mi:“MI:0915”(physical association)	0.650
CENPA	NPM1	psi-mi:“MI:0914”(association)	0.600
CENPA	NPM1	psi-mi:“MI:0403”(colocalization)	0.600
NPM1	KPNA3	psi-mi:“MI:0407”(direct interaction)	0.590
M	NPM1	psi-mi:“MI:0915”(physical association)	0.580
M	NPM1	psi-mi:“MI:0914”(association)	0.580

BioGRID (2151): NPM1 (Affinity Capture-MS), NPM1 (Affinity Capture-MS), NPM1 (Affinity Capture-MS), NPM1 (Two-hybrid), NPM2 (Two-hybrid), NPM1 (Affinity Capture-RNA), NPM1 (Affinity Capture-RNA), NPM1 (Affinity Capture-MS), NPM1 (Affinity Capture-MS), NPM1 (Affinity Capture-MS), NPM1 (Affinity Capture-MS), NPM1 (Affinity Capture-MS), NPM1 (Affinity Capture-MS), NPM1 (Affinity Capture-Western), NPM1 (Reconstituted Complex)

ESM2 similar proteins: A2XU85, A2ZX50, A6H767, B8AEC1, B8AW64, B8B2R4, B8B4K9, B9FU45, F4JEI8, O42584, O59797, P06748, P07222, P13825, P25293, P28656, P51860, P53853, P53997, P54397, P55209, P78920, Q18240, Q1HTZ9, Q28EB4, Q2TA40, Q3T160, Q4U0Y4, Q53WK4, Q55ED1, Q5AAI8, Q5MGA9, Q5R4D4, Q5U2Z3, Q5VND6, Q69JW2, Q70Z16, Q70Z17, Q70Z18, Q70Z19

Diamond homologs: O42584, O75607, P05221, P06748, P07222, P13084, P16039, P91753, Q1HTZ8, Q1HTZ9, Q27415, Q3T160, Q5RC37, Q61937, Q80W85, Q86SE8, Q9CPP0, Q9NLA3

SIGNOR signaling

32 interactions.

A	Effect	B	Mechanism
CDK1	“down-regulates activity”	NPM1	phosphorylation
PLK1	up-regulates	NPM1	phosphorylation
PLK2	up-regulates	NPM1	phosphorylation
CDK1	unknown	NPM1	phosphorylation
CDK2	unknown	NPM1	phosphorylation
CyclinE/CDK2	“down-regulates activity”	NPM1	phosphorylation
CyclinB/CDK1	“down-regulates activity”	NPM1	phosphorylation
CDKN2A	“down-regulates quantity by destabilization”	NPM1	binding
NPM1	“down-regulates activity”	HEXIM1	binding
NPM1	“down-regulates quantity by repression”	HOXA9	“transcriptional regulation”
NPM1	“up-regulates activity”	CENPA	binding
NPM1	“down-regulates quantity by repression”	FBP1	“transcriptional regulation”
RNF144B	“down-regulates quantity by destabilization”	NPM1	ubiquitination
USP36	“up-regulates quantity by stabilization”	NPM1	deubiquitination
AKT1	“down-regulates activity”	NPM1	phosphorylation
DUSP3	“down-regulates activity”	NPM1	dephosphorylation
CHUK	“up-regulates activity”	NPM1	phosphorylation
ATM	“up-regulates activity”	NPM1	phosphorylation
CDK2	“down-regulates activity”	NPM1	phosphorylation
NPM1	“up-regulates activity”	CDKN2A	binding

Enriched among interaction partners

Reactome pathways and GO biological processes over-represented among this gene’s 179 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
HCMV Early Events	10	6.9×	1e-03

GO biological processes:

GO term	Partners	Fold	FDR
mRNA stabilization	6	14.5×	2e-03
intrinsic apoptotic signaling pathway	6	14.2×	2e-03
negative regulation of protein ubiquitination	6	11.3×	5e-03

Disease & clinical

Cancer significance

From CIViC — curated cancer-variant interpretation:

AML with mutated NPM1 is a provisional entity in the WHO classification of AML and is recommended to be tested in patients with cytogenetically normal AML (CN-AML). FLT3 mutations should be evaluated concurrently as they have prognostic consequences. NPM1 mutations are concentrated in exon 12, most frequently W288fs which results in cytoplasmic sequestration of the protein. Exon 12 NPM1 mutations in the absence of FLT3-ITD are associated with good prognostic outcomes. Mice expressing the Npm1-W288fs mutation develop myeloproliferative neoplasms but not overt leukemia, indicating it may require additional mutations to promote leukemic development.

From intOGen — cancer-driver classification: activating (oncogene-like) across 1 cancer types — HCC.

Clinical variants and AI predictions

ClinVar

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

Classification	Count (floor)
Pathogenic	8
Likely pathogenic	0
Uncertain significance	25
Likely benign	9
Benign	16

Top pathogenic / likely-pathogenic (8)

Variant ID	HGVS	Classification
13998	NM_002520.7(NPM1):c.860_863dup (p.Trp288fs)	Pathogenic
13999	NM_002520.7(NPM1):c.863_864insCATG (p.Trp288fs)	Pathogenic
14000	NM_002520.7(NPM1):c.863_864insCGTG (p.Trp288fs)	Pathogenic
14001	NM_002520.7(NPM1):c.863_864insCCTG (p.Trp288fs)	Pathogenic
1701937	NM_002520.7(NPM1):c.885A>G (p.Ter295=)	Pathogenic
1701958	NM_002520.7(NPM1):c.875del (p.Lys292fs)	Pathogenic
632551	NM_002520.7(NPM1):c.869_875delinsCCCTGGCTAGG (p.Trp290fs)	Pathogenic
810668	NM_002520.7(NPM1):c.864_873delinsTTTAAGGATTCGTC (p.Trp288fs)	Pathogenic

SpliceAI

1524 predictions. Top by Δscore:

Variant	Effect	Δscore
5:171390045:TTACA:T	acceptor_loss	1.0000
5:171390046:TACA:T	acceptor_loss	1.0000
5:171390047:ACAGG:A	acceptor_loss	1.0000
5:171390048:CAGGT:C	acceptor_loss	1.0000
5:171390049:AGG:A	acceptor_loss	1.0000
5:171390050:G:T	acceptor_loss	1.0000
5:171390050:GGTT:G	acceptor_gain	1.0000
5:171390126:GAACG:G	donor_gain	1.0000
5:171390127:AACGG:A	donor_loss	1.0000
5:171390128:ACGG:A	donor_loss	1.0000
5:171390129:CGG:C	donor_loss	1.0000
5:171390131:GTA:G	donor_loss	1.0000
5:171390132:T:A	donor_loss	1.0000
5:171391300:CACA:C	acceptor_loss	1.0000
5:171391302:CA:C	acceptor_loss	1.0000
5:171391304:GGTC:G	acceptor_gain	1.0000
5:171391304:GGTCA:G	acceptor_gain	1.0000
5:171391420:CAACG:C	donor_loss	1.0000
5:171391424:GGT:G	donor_loss	1.0000
5:171391425:G:C	donor_loss	1.0000
5:171391425:G:GG	donor_gain	1.0000
5:171391426:T:C	donor_loss	1.0000
5:171392708:A:AG	acceptor_gain	1.0000
5:171392709:G:GG	acceptor_gain	1.0000
5:171392709:GCT:G	acceptor_gain	1.0000
5:171392763:T:G	donor_gain	1.0000
5:171392763:T:TG	donor_gain	1.0000
5:171392777:A:T	donor_gain	1.0000
5:171392812:CACAG:C	donor_loss	1.0000
5:171392814:CAG:C	donor_loss	1.0000

AlphaMissense

1966 scored. Top likely-pathogenic:

Variant	Protein change	am_pathogenicity
5:171388003:T:C	F19L	1.000
5:171388005:C:A	F19L	1.000
5:171388005:C:G	F19L	1.000
5:171390060:T:C	L23P	1.000
5:171390114:A:C	Q41P	1.000
5:171390117:T:C	L42S	1.000
5:171390123:T:C	L44S	1.000
5:171390126:G:T	R45I	1.000
5:171391306:T:A	V47D	1.000
5:171391345:T:A	V60D	1.000
5:171391396:C:A	A77E	1.000
5:171391402:T:C	L79S	1.000
5:171391402:T:G	L79W	1.000
5:171391707:T:A	V87D	1.000
5:171391713:T:A	L89H	1.000
5:171391716:G:A	G90E	1.000
5:171391718:G:C	G91R	1.000
5:171391721:T:C	F92L	1.000
5:171391722:T:C	F92S	1.000
5:171391723:T:A	F92L	1.000
5:171391723:T:G	F92L	1.000
5:171391728:T:A	I94K	1.000
5:171391728:T:G	I94R	1.000
5:171391734:C:A	P96Q	1.000
5:171391746:T:C	L100S	1.000
5:171391752:T:C	L102S	1.000
5:171391760:G:C	G105R	1.000
5:171391761:G:A	G105D	1.000
5:171391766:G:A	G107R	1.000
5:171391766:G:C	G107R	1.000

dbSNP variants (sampled 300 via entrez): RS1000206771 (5:171397942 C>A,G), RS1000234164 (5:171386338 T>G), RS1000243124 (5:171404905 G>A), RS1000244265 (5:171394454 A>G), RS1000296763 (5:171394628 C>T), RS1000325987 (5:171410889 G>A), RS1000400114 (5:171389228 T>C), RS1000504702 (5:171389539 G>A), RS1000542268 (5:171399010 C>A,T), RS1000776886 (5:171405743 G>A), RS1000854150 (5:171389409 A>G), RS1000980810 (5:171406107 C>T), RS1000983258 (5:171388780 C>A,G,T), RS1001032020 (5:171393999 G>A), RS1001079413 (5:171405661 C>G,T)

Disease associations

OMIM: gene MIM:164040 | disease phenotypes: MIM:601626, MIM:614675, MIM:127550

GenCC curated gene-disease

Disease	Classification	Inheritance
dyskeratosis congenita	Strong	Autosomal dominant

ClinGen Gene-Disease Validity (1)

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

Disease	Classification	Inheritance
bone marrow failure syndrome	Limited	AD

Mondo (4): acute myeloid leukemia (MONDO:0018874), bone marrow failure syndrome (MONDO:0000159), dyskeratosis congenita (MONDO:0015780), acute myeloid leukemia with multilineage dysplasia (MONDO:0019456)

Orphanet (3): Acute myeloid leukemia (Orphanet:519), Dyskeratosis congenita (Orphanet:1775), Acute myeloid leukaemia with myelodysplasia-related features (Orphanet:86845)

HPO phenotypes

99 total (30 of 99 shown, HPO-id order):

HPO	Term
HP:0000006	Autosomal dominant inheritance
HP:0000008	Abnormal morphology of female internal genitalia
HP:0000035	Abnormal testis morphology
HP:0000164	Abnormality of the dentition
HP:0000212	Gingival overgrowth
HP:0000225	Gingival bleeding
HP:0000327	Hypoplasia of the maxilla
HP:0000365	Hearing impairment
HP:0000421	Epistaxis
HP:0000498	Blepharitis
HP:0000499	Abnormal eyelash morphology
HP:0000518	Cataract
HP:0000534	Abnormal eyebrow morphology
HP:0000600	Abnormality of the pharynx
HP:0000668	Hypodontia
HP:0000670	Carious teeth
HP:0000679	Taurodontia
HP:0000704	Periodontitis
HP:0000790	Hematuria
HP:0000819	Diabetes mellitus
HP:0000939	Osteoporosis
HP:0000967	Petechiae
HP:0000975	Hyperhidrosis
HP:0000978	Bruising susceptibility
HP:0000979	Purpura
HP:0000982	Palmoplantar keratoderma
HP:0001034	Hypermelanotic macule
HP:0001053	Hypopigmented skin patches
HP:0001231	Abnormal fingernail morphology
HP:0001263	Global developmental delay

GWAS associations

4 associations (top):

Study	Trait	p-value
GCST008163_209	Height	3.000000e-07
GCST010702_141	Subcortical volume (MOSTest)	9.000000e-14
GCST010703_175	Brain morphology (MOSTest)	4.000000e-11
GCST012334_7	Multisite chronic pain	3.000000e-08

EFO canonical traits (2, from GWAS)

EFO ID	Trait name
EFO:0004346	neuroimaging measurement
EFO:0010100	multisite chronic pain

MeSH disease descriptors (2)

Descriptor	Name	Tree numbers
D019871	Dyskeratosis Congenita	C15.378.190.223.500.750; C16.131.831.150; C16.320.322.108; C16.320.850.235; C17.800.804.150; C17.800.827.235
D015470	Leukemia, Myeloid, Acute	C04.557.337.539.275; C15.378.508.539.275

Drugs & pharmacology

Drug and pharmacology data

Is drug target: yes

ChEMBL targets (4): CHEMBL2111387 (CHIMERIC PROTEIN), CHEMBL4296165 (PROTEIN-PROTEIN INTERACTION), CHEMBL4748220 (PROTEIN-PROTEIN INTERACTION), CHEMBL5178 (SINGLE PROTEIN)

Molecules with ChEMBL bioactivity

5 molecules (phase ≥1), by development phase (incl. off-target/promiscuous compounds). Patent mentions across the top 20 by phase: 37,412 (via chembl_molecule»patent_compound — counts attach to the compound, not the gene–compound relationship, so off-target/promiscuous molecules can dominate).

Molecule	Name	Phase	Patents
CHEMBL2403108	CERITINIB	4	8,551
CHEMBL288441	BOSUTINIB	4	12,255
CHEMBL601719	CRIZOTINIB	4	14,403
CHEMBL1232461	MOLIBRESIB	2	1,538
CHEMBL3545360	ASP-3026	1	665

Clinical evidence (CIViC)

Drug × variant × indication: 9 predictive associations from 10 curated evidence items; also 17 prognostic, 10 diagnostic.

Variant	Therapy	Indication	Effect	Level	CIViC
NPM1 EXON 11 MUTATION	Tretinoin	Acute Myeloid Leukemia	Sensitivity/Response	CIViC B	EID137 +1
NPM1 EXON 11 MUTATION	Daunorubicin	Acute Myeloid Leukemia	Sensitivity/Response	CIViC B	EID147
NPM1 EXON 11 MUTATION	Valproic Acid	Acute Myeloid Leukemia	Sensitivity/Response	CIViC B	EID148
NPM1 MUTATION	Revumenib	Acute Myeloid Leukemia	Sensitivity/Response	CIViC B	EID12743
NPM1 EXON 11 MUTATION	NSC348884 + Tretinoin	Acute Myeloid Leukemia	Sensitivity/Response	CIViC D	EID149
NPM1 W288FS	NSC348884	Acute Myeloid Leukemia	Sensitivity/Response	CIViC D	EID152
NPM1 EXON 11 MUTATION	Anti-CD123 + Anti-CD33	Acute Myeloid Leukemia	Sensitivity/Response	CIViC E	EID150
NPM1 EXON 11 MUTATION	Anti-CD33	Acute Myeloid Leukemia	Sensitivity/Response	CIViC E	EID151
NPM1 W288FS	Daunorubicin + Cytarabine + Etoposide	Acute Myeloid Leukemia	Sensitivity/Response	CIViC E	EID153

PharmGKB: 1 entry (VIP=true, CPIC=false)

ChEMBL bioactivities

82 potent at pChembl≥5 of 87 total, top 50 by pChembl (potency: 10 = 0.1 nM, 6 = 1 µM).

pChembl	Type	Value	Unit	Molecule
8.91	IC50	1.24	nM	CHEMBL6162078
8.70	IC50	2	nM	CRIZOTINIB
8.52	IC50	3	nM	CHEMBL4762256
8.22	IC50	6	nM	CHEMBL4762256
8.17	IC50	6.8	nM	ASP-3026
8.15	IC50	7	nM	CHEMBL4762861
8.08	Kd	8.259	nM	CHEMBL3752910
8.08	ED50	8.259	nM	CHEMBL3752910
8.00	IC50	10	nM	MOLIBRESIB
7.89	IC50	13	nM	CHEMBL4798141
7.70	IC50	20	nM	CHEMBL3651854
7.58	IC50	26	nM	CERITINIB
7.58	IC50	26	nM	CHEMBL3651854
7.52	IC50	30	nM	CHEMBL2158531
7.52	IC50	30	nM	CHEMBL3651854
7.48	IC50	33	nM	CHEMBL4791252
7.42	IC50	38	nM	CHEMBL4762861
7.40	IC50	40	nM	CHEMBL2158527
7.33	IC50	47	nM	CHEMBL3651885
7.33	IC50	47	nM	CHEMBL4779222
7.30	IC50	50	nM	CHEMBL1822527
7.29	IC50	51	nM	CRIZOTINIB
7.22	IC50	60	nM	CHEMBL2158525
7.22	IC50	60	nM	CHEMBL1822525
7.19	IC50	64	nM	CHEMBL3734798
7.16	IC50	70	nM	CHEMBL2158519
7.16	IC50	70	nM	CHEMBL2158518
7.16	IC50	70	nM	CHEMBL2158529
7.16	IC50	70	nM	CHEMBL2158526
7.16	IC50	70	nM	CHEMBL1822523
7.10	IC50	80	nM	CHEMBL2158516
7.10	IC50	80	nM	CHEMBL2158530
7.10	IC50	80	nM	CHEMBL1822521
7.10	IC50	80	nM	CHEMBL1822526
7.07	IC50	85	nM	CHEMBL1822522
7.00	IC50	100	nM	CHEMBL1934334
7.00	IC50	100	nM	CHEMBL2158511
7.00	IC50	100	nM	CHEMBL2158528
7.00	IC50	100	nM	CHEMBL1822519
7.00	IC50	100	nM	CHEMBL1822520
6.93	EC50	116.5	nM	CHEMBL4875115
6.82	IC50	150	nM	CHEMBL2158515
6.82	IC50	150	nM	CHEMBL2158586
6.82	IC50	150	nM	BOSUTINIB
6.82	IC50	150	nM	CHEMBL4755518
6.82	IC50	151	nM	CHEMBL3734798
6.70	IC50	200	nM	CHEMBL2158587
6.70	IC50	200	nM	CHEMBL2158521
6.70	IC50	200	nM	BOSUTINIB
6.70	IC50	200	nM	CHEMBL1822524

PubChem BioAssay actives

80 with measured affinity, of 235 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
Crizotinib	2161822: Inhibition of human N-terminal GST-fused NPM1-ALK (1 to 680 residues) expressed in baculovirus expression system using Srctide as substrate measured after 1 hr by off-chip mobility shift assay relative to control	ic50	0.0020	uM
2-N-[5-methyl-4-(1-methylpiperidin-4-yl)-2-propan-2-yloxyphenyl]-4-N-(2-propan-2-ylsulfonylphenyl)-7H-pyrrolo[2,3-d]pyrimidine-2,4-diamine	1711502: Inhibition of NMP-ALK (unknown origin) expressed in human KARPAS-299 cells assessed as reduction in cell growth	ic50	0.0030	uM
2-N-[2-methoxy-4-[4-(4-methylpiperazin-1-yl)piperidin-1-yl]phenyl]-4-N-(2-propan-2-ylsulfonylphenyl)-1,3,5-triazine-2,4-diamine	2161822: Inhibition of human N-terminal GST-fused NPM1-ALK (1 to 680 residues) expressed in baculovirus expression system using Srctide as substrate measured after 1 hr by off-chip mobility shift assay relative to control	ic50	0.0068	uM
3-methyl-6-N-(5-methyl-4-piperidin-4-yl-2-propan-2-yloxyphenyl)-4-N-(2-propan-2-ylsulfonylphenyl)-2H-pyrazolo[3,4-d]pyrimidine-4,6-diamine	1711502: Inhibition of NMP-ALK (unknown origin) expressed in human KARPAS-299 cells assessed as reduction in cell growth	ic50	0.0070	uM
4-methyl-3-[(1-methyl-6-pyridin-3-ylpyrazolo[3,4-d]pyrimidin-4-yl)amino]-N-[3-(trifluoromethyl)phenyl]benzamide	2148885: Binding affinity to human NPM1 incubated for 45 mins by Kinobead based pull down assay	kd	0.0083	uM
2-[(4S)-6-(4-chlorophenyl)-8-methoxy-1-methyl-4H-[1,2,4]triazolo[4,3-a][1,4]benzodiazepin-4-yl]-N-ethylacetamide	2179037: Inhibition of NPM1 (unknown origin) incubated for 1 hr by colloidal coomassie staining based LC-MS/MS analysis	ic50	0.0100	uM
6-N-[5-methyl-4-(1-methylpiperidin-4-yl)-2-propan-2-yloxyphenyl]-4-N-(2-propan-2-ylsulfonylphenyl)-1H-pyrazolo[5,4-d]pyrimidine-4,6-diamine	1711446: Inhibition of NMP-ALK (unknown origin) expressed in mouse BaF3 cells assessed as reduction in cell proliferation incubated for 2 to 3 days by Bright-Glo luciferase assay	ic50	0.0130	uM
3-methyl-6-N-[5-methyl-4-(1-methylpiperidin-4-yl)-2-propan-2-yloxyphenyl]-4-N-(2-propan-2-ylsulfonylphenyl)-2H-pyrazolo[3,4-d]pyrimidine-4,6-diamine	1711446: Inhibition of NMP-ALK (unknown origin) expressed in mouse BaF3 cells assessed as reduction in cell proliferation incubated for 2 to 3 days by Bright-Glo luciferase assay	ic50	0.0200	uM
Ceritinib	1711446: Inhibition of NMP-ALK (unknown origin) expressed in mouse BaF3 cells assessed as reduction in cell proliferation incubated for 2 to 3 days by Bright-Glo luciferase assay	ic50	0.0260	uM
(2S)-3-[4-[3-methoxy-4-[[7-(2-methoxyphenyl)pyrrolo[2,1-f][1,2,4]triazin-2-yl]amino]phenyl]piperidin-1-yl]propane-1,2-diol	694696: Inhibition of human NPM-ALK phosphorylation using 4-MeUP substrate by cell based assay	ic50	0.0300	uM
6-N-(5-methyl-4-piperidin-4-yl-2-propan-2-yloxyphenyl)-4-N-(2-propan-2-ylsulfonylphenyl)-1H-pyrazolo[5,4-d]pyrimidine-4,6-diamine	1711446: Inhibition of NMP-ALK (unknown origin) expressed in mouse BaF3 cells assessed as reduction in cell proliferation incubated for 2 to 3 days by Bright-Glo luciferase assay	ic50	0.0330	uM
N-[2-[2-(2-methoxy-4-piperidin-4-ylanilino)pyrrolo[2,1-f][1,2,4]triazin-7-yl]phenyl]-N-methylmethanesulfonamide	694696: Inhibition of human NPM-ALK phosphorylation using 4-MeUP substrate by cell based assay	ic50	0.0400	uM
2-N-(5-methyl-4-piperidin-4-yl-2-propan-2-yloxyphenyl)-4-N-(2-propan-2-ylsulfonylphenyl)-7H-pyrrolo[2,3-d]pyrimidine-2,4-diamine	1711446: Inhibition of NMP-ALK (unknown origin) expressed in mouse BaF3 cells assessed as reduction in cell proliferation incubated for 2 to 3 days by Bright-Glo luciferase assay	ic50	0.0470	uM
5-N-[5-methyl-4-(1-methylpiperidin-4-yl)-2-propan-2-yloxyphenyl]-7-N-(2-propan-2-ylsulfonylphenyl)-2H-triazolo[4,5-d]pyrimidine-5,7-diamine	1711446: Inhibition of NMP-ALK (unknown origin) expressed in mouse BaF3 cells assessed as reduction in cell proliferation incubated for 2 to 3 days by Bright-Glo luciferase assay	ic50	0.0470	uM
N-[2-[2-[2-methoxy-4-(4-morpholin-4-ylpiperidin-1-yl)anilino]pyrrolo[2,1-f][1,2,4]triazin-7-yl]phenyl]-N-methylmethanesulfonamide	617856: Inhibition of NPM/ALK phosphorylation in human KARPAS299 cells by ELISA	ic50	0.0500	uM
N-[2-[2-[4-[4-[(2S)-2-hydroxypropyl]piperazin-1-yl]-2-methoxyanilino]pyrrolo[2,1-f][1,2,4]triazin-7-yl]phenyl]-N-methylmethanesulfonamide	617856: Inhibition of NPM/ALK phosphorylation in human KARPAS299 cells by ELISA	ic50	0.0600	uM
N-[2-[2-[4-[1-(2-hydroxyethyl)piperidin-4-yl]-2-methoxyanilino]pyrrolo[2,1-f][1,2,4]triazin-7-yl]phenyl]-N-methylmethanesulfonamide	694696: Inhibition of human NPM-ALK phosphorylation using 4-MeUP substrate by cell based assay	ic50	0.0600	uM
3-[(1R)-1-(2,6-dichloro-3-fluorophenyl)ethoxy]-5-(1-piperidin-4-ylpyrazol-3-yl)pyridin-2-amine	1711502: Inhibition of NMP-ALK (unknown origin) expressed in human KARPAS-299 cells assessed as reduction in cell growth	ic50	0.0640	uM
N-[2-methoxy-4-[4-(4-methylpiperazin-1-yl)piperazin-1-yl]phenyl]-7-(2-methoxyphenyl)pyrrolo[2,1-f][1,2,4]triazin-2-amine	617856: Inhibition of NPM/ALK phosphorylation in human KARPAS299 cells by ELISA	ic50	0.0700	uM
N-[2-[2-[4-[1-[(2R)-2-hydroxypropyl]piperidin-4-yl]-2-methoxyanilino]pyrrolo[2,1-f][1,2,4]triazin-7-yl]phenyl]-N-methylmethanesulfonamide	694696: Inhibition of human NPM-ALK phosphorylation using 4-MeUP substrate by cell based assay	ic50	0.0700	uM
N-[2-methoxy-4-(1-methylpiperidin-4-yl)phenyl]-7-(2-methoxyphenyl)pyrrolo[2,1-f][1,2,4]triazin-2-amine	694696: Inhibition of human NPM-ALK phosphorylation using 4-MeUP substrate by cell based assay	ic50	0.0700	uM
2-[4-[3-methoxy-4-[[5-methyl-7-[2-[methyl(methylsulfonyl)amino]phenyl]pyrrolo[2,1-f][1,2,4]triazin-2-yl]amino]phenyl]piperidin-1-yl]acetamide	694696: Inhibition of human NPM-ALK phosphorylation using 4-MeUP substrate by cell based assay	ic50	0.0700	uM
2-[4-[4-[[5-chloro-7-[2-[methyl(methylsulfonyl)amino]phenyl]pyrrolo[2,1-f][1,2,4]triazin-2-yl]amino]-3-methoxyphenyl]piperidin-1-yl]acetamide	694696: Inhibition of human NPM-ALK phosphorylation using 4-MeUP substrate by cell based assay	ic50	0.0700	uM
(2R)-1-[4-[3-methoxy-4-[[7-(2-methoxyphenyl)pyrrolo[2,1-f][1,2,4]triazin-2-yl]amino]phenyl]piperazin-1-yl]propan-2-ol	617856: Inhibition of NPM/ALK phosphorylation in human KARPAS299 cells by ELISA	ic50	0.0800	uM
N-[2-[2-[4-[4-[(2R)-2-hydroxypropyl]piperazin-1-yl]-2-methoxyanilino]pyrrolo[2,1-f][1,2,4]triazin-7-yl]phenyl]-N-methylmethanesulfonamide	617856: Inhibition of NPM/ALK phosphorylation in human KARPAS299 cells by ELISA	ic50	0.0800	uM
1-fluoro-3-[4-[3-methoxy-4-[[7-(2-methoxyphenyl)pyrrolo[2,1-f][1,2,4]triazin-2-yl]amino]phenyl]piperidin-1-yl]propan-2-ol	694696: Inhibition of human NPM-ALK phosphorylation using 4-MeUP substrate by cell based assay	ic50	0.0800	uM
2-[4-[3-methoxy-4-[[7-[2-[methyl(methylsulfonyl)amino]phenyl]pyrrolo[2,1-f][1,2,4]triazin-2-yl]amino]phenyl]piperidin-1-yl]acetamide	694696: Inhibition of human NPM-ALK phosphorylation using 4-MeUP substrate by cell based assay	ic50	0.0800	uM
N-[2-methoxy-4-(4-morpholin-4-ylpiperazin-1-yl)phenyl]-7-(2-methoxyphenyl)pyrrolo[2,1-f][1,2,4]triazin-2-amine	617856: Inhibition of NPM/ALK phosphorylation in human KARPAS299 cells by ELISA	ic50	0.0850	uM
N-[2-methoxy-4-(4-methylpiperazin-1-yl)phenyl]-7-(2-methoxyphenyl)pyrrolo[2,1-f][1,2,4]triazin-2-amine	617856: Inhibition of NPM/ALK phosphorylation in human KARPAS299 cells by ELISA	ic50	0.1000	uM
(2S)-1-[4-[3-methoxy-4-[[7-(2-methoxyphenyl)pyrrolo[2,1-f][1,2,4]triazin-2-yl]amino]phenyl]piperazin-1-yl]propan-2-ol	617856: Inhibition of NPM/ALK phosphorylation in human KARPAS299 cells by ELISA	ic50	0.1000	uM
(2R)-1-[4-[3-methoxy-4-[[7-(2-methoxyphenyl)pyrrolo[2,1-f][1,2,4]triazin-2-yl]amino]phenyl]piperidin-1-yl]propan-2-ol	694696: Inhibition of human NPM-ALK phosphorylation using 4-MeUP substrate by cell based assay	ic50	0.1000	uM
2-[4-[3-methoxy-4-[[7-(2-methoxyphenyl)pyrrolo[2,1-f][1,2,4]triazin-2-yl]amino]phenyl]piperidin-1-yl]-N-methylacetamide	694696: Inhibition of human NPM-ALK phosphorylation using 4-MeUP substrate by cell based assay	ic50	0.1000	uM
2-[4-[3-methoxy-4-[[7-(2-methoxyphenyl)pyrrolo[2,1-f][1,2,4]triazin-2-yl]amino]phenyl]piperidin-1-yl]acetamide	694696: Inhibition of human NPM-ALK phosphorylation using 4-MeUP substrate by cell based assay	ic50	0.1000	uM
8-[4-[2-[[2-(2,6-dioxopiperidin-3-yl)-1,3-dioxoisoindol-4-yl]amino]acetyl]piperazin-1-yl]-9-ethyl-6,6-dimethyl-11-oxo-5H-benzo[b]carbazole-3-carbonitrile	1847263: Protac activity at CRBN/NPM-ALK in human KARPAS-299 cells assessed as induction of NPM-ALK fusion protein degradation incubated for 48 hrs by Western blot analysis	ec50	0.1165	uM
6-N-[5-methyl-4-(1-methylpiperidin-4-yl)-2-propan-2-yloxyphenyl]-4-N-(2-propan-2-ylsulfonylphenyl)-2H-pyrazolo[3,4-d]pyrimidine-3,4,6-triamine	1711446: Inhibition of NMP-ALK (unknown origin) expressed in mouse BaF3 cells assessed as reduction in cell proliferation incubated for 2 to 3 days by Bright-Glo luciferase assay	ic50	0.1500	uM
Bosutinib	271009: Inhibition of NPM/ALK L256T mutant kinase activity in BaF3 cells by radioenzymatic assay	ic50	0.1500	uM
2-amino-1-[4-[3-methoxy-4-[[7-(2-methoxyphenyl)pyrrolo[2,1-f][1,2,4]triazin-2-yl]amino]phenyl]piperidin-1-yl]-2-methylpropan-1-one	694696: Inhibition of human NPM-ALK phosphorylation using 4-MeUP substrate by cell based assay	ic50	0.1500	uM
2-[4-[3-methoxy-4-[[7-(3-methoxy-4-pyridinyl)pyrrolo[2,1-f][1,2,4]triazin-2-yl]amino]phenyl]piperidin-1-yl]acetamide	694696: Inhibition of human NPM-ALK phosphorylation using 4-MeUP substrate by cell based assay	ic50	0.1500	uM
azetidin-2-yl-[4-[3-methoxy-4-[[7-(2-methoxyphenyl)pyrrolo[2,1-f][1,2,4]triazin-2-yl]amino]phenyl]piperidin-1-yl]methanone	694696: Inhibition of human NPM-ALK phosphorylation using 4-MeUP substrate by cell based assay	ic50	0.2000	uM
N-[2-[2-[2-methoxy-4-(4-methylpiperazin-1-yl)anilino]pyrrolo[2,1-f][1,2,4]triazin-7-yl]phenyl]-N-methylmethanesulfonamide	617856: Inhibition of NPM/ALK phosphorylation in human KARPAS299 cells by ELISA	ic50	0.2000	uM
N-[2-[2-[5-fluoro-4-[4-[(2S)-2-hydroxypropyl]piperazin-1-yl]-2-methoxyanilino]pyrrolo[2,1-f][1,2,4]triazin-7-yl]phenyl]-N-methylmethanesulfonamide	694696: Inhibition of human NPM-ALK phosphorylation using 4-MeUP substrate by cell based assay	ic50	0.2000	uM
2-[4-[4-[[7-(2-cyanophenyl)pyrrolo[2,1-f][1,2,4]triazin-2-yl]amino]-3-methoxyphenyl]piperidin-1-yl]acetamide	694696: Inhibition of human NPM-ALK phosphorylation using 4-MeUP substrate by cell based assay	ic50	0.2500	uM
6-methyl-2-N-(5-methyl-4-piperidin-4-yl-2-propan-2-yloxyphenyl)-4-N-(2-propan-2-ylsulfonylphenyl)-7H-pyrrolo[2,3-d]pyrimidine-2,4-diamine	1711502: Inhibition of NMP-ALK (unknown origin) expressed in human KARPAS-299 cells assessed as reduction in cell growth	ic50	0.3280	uM
4,7-bis[4-(4-methylpiperazin-1-yl)phenyl]pyrazino[1,2-a]benzimidazole	1070241: Inhibition of NPM/ALK (unknown origin) transfected in mouse BAF3 cells assessed as cell growth inhibition after 72 hrs by [3H]-thymidine incorporation assay	ic50	0.3400	uM
3-[4-amino-7-[3-(hydroxymethyl)cyclobutyl]pyrrolo[2,3-d]pyrimidin-5-yl]phenol	1774882: Inhibition of NPM-ALK (unknown origin) expressed in mouse BaF3 cells assessed as reduction in cell proliferation incubated for 48 hrs by cell proliferation assay	ic50	0.3700	uM
2-[4-[4-[[7-(2,4-dimethoxyphenyl)pyrrolo[2,1-f][1,2,4]triazin-2-yl]amino]-3-methoxyphenyl]piperidin-1-yl]acetamide	694696: Inhibition of human NPM-ALK phosphorylation using 4-MeUP substrate by cell based assay	ic50	0.5000	uM
7-(4-methoxyphenyl)-4-[(E)-2-phenylethenyl]pyrazino[1,2-a]benzimidazole	1070241: Inhibition of NPM/ALK (unknown origin) transfected in mouse BAF3 cells assessed as cell growth inhibition after 72 hrs by [3H]-thymidine incorporation assay	ic50	0.8400	uM
3-(4-amino-7-cyclopentylpyrrolo[2,3-d]pyrimidin-5-yl)phenol	1774882: Inhibition of NPM-ALK (unknown origin) expressed in mouse BaF3 cells assessed as reduction in cell proliferation incubated for 48 hrs by cell proliferation assay	ic50	0.8700	uM
2-N-[5-methyl-4-(1-methylpiperidin-4-yl)-2-propan-2-yloxyphenyl]-4-N-(2-propan-2-ylsulfonylphenyl)quinazoline-2,4-diamine	1711446: Inhibition of NMP-ALK (unknown origin) expressed in mouse BaF3 cells assessed as reduction in cell proliferation incubated for 2 to 3 days by Bright-Glo luciferase assay	ic50	1.0820	uM
7-bromo-4-chloropyrazino[1,2-a]benzimidazole	1070241: Inhibition of NPM/ALK (unknown origin) transfected in mouse BAF3 cells assessed as cell growth inhibition after 72 hrs by [3H]-thymidine incorporation assay	ic50	1.1000	uM

CTD chemical–gene interactions

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

Chemical	Actions (top 5)	PubMed papers
bisphenol A	affects expression, affects localization, decreases expression	4
sodium arsenite	affects binding, increases reaction, decreases expression, increases abundance, increases expression	4
Arsenic Trioxide	decreases reaction, increases response to substance, increases expression, affects response to substance	3
Arsenic	affects methylation, increases expression, decreases expression, increases abundance	3
Tretinoin	affects binding, increases reaction, decreases expression	3
bisphenol S	affects localization, increases expression	2
Cisplatin	increases expression, decreases response to substance	2
Estradiol	decreases phosphorylation, increases expression	2
Quercetin	decreases expression, decreases phosphorylation	2
Valproic Acid	decreases expression	2
aristolochic acid I	decreases expression	1
UF010 compound	increases acetylation	1
FR900359	affects phosphorylation	1
bisphenol F	increases expression	1
dicrotophos	decreases expression	1
deoxynivalenol	increases expression	1
geraniol	decreases expression	1
sodium arsenate	decreases expression	1
titanium dioxide	increases phosphorylation	1
pyrogallol 1,3-dimethyl ether	affects cotreatment, affects localization, increases expression, decreases expression	1
hexamethylene bisacetamide	decreases expression	1
methylparaben	decreases expression, increases expression	1
afimoxifene	decreases expression	1
tetrabromobisphenol A	affects localization	1
manganese chloride	increases abundance, increases expression	1
cerous chloride	affects localization, decreases expression	1
lanthanum chloride	affects localization, decreases expression	1
coumarin	increases phosphorylation	1
triphenyltin	increases expression	1
cupric oxide	increases phosphorylation	1

ChEMBL screening assays

113 unique, capped per target: 108 binding, 5 functional

Representative assays (with source publication via chembl_document):

Assay ID	Type	Description	Source paper
CHEMBL1005525	Binding	Inhibition of NPM-ALK in human Karpas299 cells at 50 nM	Identification of NVP-TAE684, a potent, selective, and efficacious inhibitor of NPM-ALK. — Proc Natl Acad Sci U S A
CHEMBL861020	Functional	Antiproliferative activity against SUDHL1 expressing NPM-ALK by MTT assay	Identification of ellagic acid as potent inhibitor of protein kinase CK2: a successful example of a virtual screening application. — J Med Chem

Cellosaurus cell lines

24 cell lines: 24 cancer cell line

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

Cellosaurus	Name	Category	Sex
CVCL_0538	SU-DHL-1	Cancer cell line	Male
CVCL_1170	DEL	Cancer cell line	Male
CVCL_1176	DMS 273	Cancer cell line	Female
CVCL_1324	Karpas-299	Cancer cell line	Male
CVCL_1711	SR	Cancer cell line	Male
CVCL_1844	OCI-AML-3	Cancer cell line	Male
CVCL_2093	Ki-JK	Cancer cell line	Male
CVCL_2098	L-82	Cancer cell line	Female
CVCL_2209	SUP-M2	Cancer cell line	Female
CVCL_5227	OCI-AML3/HaABCG2 clone 6.2	Cancer cell line	Male

Clinical trials (associated diseases)

318 trials via MONDO — disease-level, not drug-specific.

Trial	Phase	Status	Title
NCT00199147	PHASE4	UNKNOWN	Efficacy of G-CSF-Priming in Elderly AML Patients
NCT00304447	PHASE4	COMPLETED	Study Evaluating the Effect of Corticosteroids on Mylotarg® Infusion-Related Adverse Events in Patients With Leukemia
NCT00464217	PHASE4	COMPLETED	Treatment of the Acute Myeloblastic Leukaemia in Patients Over 65 Years
NCT00487448	PHASE4	COMPLETED	SMD_FLAG-IDA_98: FLAG-IDA in Induction Treatment of High Risk Myelodysplastic Syndromes or Secondary Acute Myeloblastic Leukemia
NCT00488709	PHASE4	COMPLETED	Fludarabine, Cytarabine, Topotecan in Treating Patients With Relapsed or Refractory Acute Myeloid Leukemia
NCT00686543	PHASE4	COMPLETED	Oral Posaconazole in High Risk Patients With Gastrointestinal Dysfunction (Study P05115)
NCT01041040	PHASE4	COMPLETED	LAM07: Study to Analyze the Efficacy of a Risk Adapted Treatment Strategy, Including Gemtuzumab Ozogamicin (GO) During Consolidation, for Patients With Acute Myeloid Leukemia (AML)
NCT01198054	PHASE4	TERMINATED	LENA-LMA-5:Lenalidomide in Acute Myeloid Leukemia (AML)
NCT01200355	PHASE4	COMPLETED	Posaconazole Versus Micafungin for Prophylaxis Against Invasive Fungal Infections During Neutropenia in Patients Undergoing Chemotherapy for Acute Myelogenous Leukemia, Acute Lymphocytic Leukemia or Myelodysplastic Syndrome
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
NCT02386800	PHASE4	ACTIVE_NOT_RECRUITING	CINC424A2X01B Rollover Protocol
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
NCT05144243	PHASE4	ACTIVE_NOT_RECRUITING	Study to Assess Adverse Events and Change in Disease State of Oral Venetoclax in Combination With Subcutaneous (SC) Azacitidine in Newly Diagnosed Adult Participants With Acute Myeloid Leukemia (AML) Who Are Ineligible for Intensive Chemotherapy in China
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
NCT07016165	PHASE4	RECRUITING	Ciprofloxacin vs Ceftazidime for Empirical Treatment of High-Risk Neutropenic Fever in Children With Hematologic Malignancies
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
NCT07486713	PHASE4	RECRUITING	Olutasidenib DDI Study in Patients With IDH1 Mutation Positive Malignancies
NCT07561892	PHASE4	RECRUITING	Study of the Effectiveness and Safety of Daunorubicin /Idarubicin ± Silibinin in Treating Newly Diagnosed AML (Non-M3).
NCT00000589	PHASE3	COMPLETED	Trial to Reduce Alloimmunization to Platelets (TRAP)
NCT00044486	PHASE3	COMPLETED	Prophylaxis Trial of Posaconazole Versus Standard Azole Therapy for Neutropenic Patients (Study P01899)
NCT00093990	PHASE3	COMPLETED	Tipifarnib Versus Best Supportive Care in the Treatment of Newly Diagnosed Acute Myeloid Leukemia (AML)
NCT00125606	PHASE3	TERMINATED	Phase 3 Trial for AML Patients in CR2 Comparing 8Gy TBI /Fludarabine to Conditioning With TBI 12Gy/Cyclophosphamide
NCT00136084	PHASE3	COMPLETED	Treatment of Patients With Newly Diagnosed Acute Myeloid Leukemia or Myelodysplasia
NCT00146120	PHASE3	COMPLETED	Risk-Adapted Therapy of Acute Myeloid Leukemia of Adults (18-60 Years) According to the Cytogenetic Result
NCT00150878	PHASE3	TERMINATED	Standard vs. Reduced-Intensity Conditioning in Patients With Acute Myeloid Leukemia in First Remission
NCT00151255	PHASE3	COMPLETED	All-Trans Retinoic Acid in Combination With Standard Induction and Consolidation Therapy in Older Patients With Newly Diagnosed Acute Myeloid Leukemia
NCT00152139	PHASE3	COMPLETED	Stem Cell Transplantation for Patients With Hematologic Malignancies
NCT00152594	PHASE3	TERMINATED	Voriconazole or Placebo in the Prophylaxis of Lung Infiltrates in Patients Undergoing Induction Chemotherapy for Acute Myelogenous Leukemia
NCT00186966	PHASE3	COMPLETED	Treatment of Children and Adolescents With Refractory or Relapsed Acute Myeloid Leukemia
NCT00226512	PHASE3	WITHDRAWN	To Determine the Role of Adding Campath-1H or ATG Given In-vivo in Addition to Fludarabine and Low Dose Busulfex on Outcome in Patients Treated With Reduced Intensity Conditioning
NCT00260832	PHASE3	COMPLETED	Trial of Decitabine in Patients With Acute Myeloid Leukemia