Myeloid neoplasm

disease
On this page

Also known as myeloid malignancymyeloid tumormyeloid tumour

Summary

Myeloid neoplasm (MONDO:0005170) is a cancer with 5 cohort genes (3 GWAS associations across 2 studies; 5 CIViC-evidence somatic drivers; 1 ClinVar predisposition record) and 63 clinical trials. Molecularly, ZMYM2::FGFR1 Fusion OR BCR::FGFR1 Fusion OR CEP43::FGFR1 Fusion OR TRIM24::FGFR1 Fusion OR FGFR1 Translocation confers sensitivity to Pemigatinib in Myeloid Neoplasm (CIViC Level A); 5 further subtype–drug associations are mapped below. Top therapeutic interventions include mitoxantrone, cladribine, and busulfan.

At a glance

  • Classification: Cancer
  • Cohort genes: 5
  • GWAS associations: 3
  • ClinVar variants: 1
  • Clinical trials: 63
  • Precision-medicine evidence (CIViC): 6 subtype–drug associations

Clinical features

No curated clinical features (Orphanet) for this disease.

Identifiers

Disease identifiers

FieldValue
Canonical namemyeloid neoplasm
Mondo IDMONDO:0005170
EFOEFO:0002427
DOIDDOID:0070004
NCITC9290
UMLSC2939461
MedGen445430
GARD0024160
Is cancer (heuristic)yes

Also known as: myeloid malignancy · myeloid neoplasm · myeloid tumor · myeloid tumour

Data availability: 1 ClinVar variant · 3 GWAS associations (2 studies).

Disease family

An umbrella term covering 4 Mondo subtypes.

Classification path: disease › human disease › disease by etiologic mechanism › cancer or benign tumorneoplastic disease or syndromeneoplasmhematopoietic and lymphoid system neoplasmhematopoietic and lymphoid cell neoplasmmyeloid neoplasm

Related subtypes (7): central nervous system hematopoietic neoplasm, refractory hematologic cancer, leukemia, lymphoid neoplasm, histiocytic and dendritic cell neoplasm, myeloid/lymphoid neoplasms associated with eosinophilia and abnormality of PDGFRA, PDGFRB, FGFR1 or JAK2, myelodysplastic syndrome with excess blasts

Subtypes (4): mast cell neoplasm, plasma cell myeloma, CD4+/CD56+ hematodermic neoplasm, myeloproliferative neoplasm

Genetics & variants

GWAS landscape

3 GWAS associations across 2 studies. Top hits map to 0 distinct genes (as reported by GWAS).

Top associations by p-value

rsIDp-valueGeneRisk alleleOdds ratio
chr17:767368776e-37T126.89
chr20:324346381e-18AG25.91
chr9:50737702e-18T22.99

Top studies (by case count)

StudyLead authorYearCasesControlsTitle
GCST90079643Backman JD2021572387,341Exome sequencing and analysis of 454,787 UK Biobank participants.
GCST90083629Backman JD2021572387,341Exome sequencing and analysis of 454,787 UK Biobank participants.

Variant details and genetic-evidence tiers

Tier distribution (top 50 variants)

TierVariants
Tier 1: coding0
Tier 2: splice/UTR0
Tier 3: regulatory0
Tier 4: intronic/intergenic3

MAF distribution

BucketVariants
common (>=0.05)0
low_freq (0.01-0.05)0
rare (<0.01)0
unknown3

Functional consequences

ConsequenceCount
unknown3

Top variants

rsIDChrPosAllelesMAFConsequenceGenep-valueTier
chr17:767368776e-37Tier 4: intronic/intergenic
chr20:324346381e-18Tier 4: intronic/intergenic
chr9:50737702e-18Tier 4: intronic/intergenic

ClinVar germline variants

1 retrieved; paginated sample, class counts are floors:

1 pathogenic

ClinVarVariant (HGVS)GeneClassificationReview
1711135t(13;14)(q12.2;q32.12)TRIP11Pathogenicno assertion criteria provided

Genes & proteins

Mendelian disease overlap and somatic drivers

GenCC: 0 · Orphanet: 55 · OMIM-shared: 0 · Dual-evidence (GWAS+Mendelian): 0

Somatic driver evidence (intOGen + CIViC, cohort fanout)

GeneintOGen roleCancer typesCIViC
SRSF2ActAMLCIViC #5210
TP53LoFACC,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,WTCIViC #45
FGFR1ActBLCA,GBM,OVT,PANCREAS,PAST,PGNG,WDTCCIViC #1885
FGFR3ActBLADDER,BLCA,HNSC,LUSC,PCM,PLMESO,UTUCCIViC #23
TRIP11LoFBLCA

Orphanet rare-disease linkage (cohort genes)

GeneOrphanet IDRare disease
SRSF2Orphanet:98823Chronic myelomonocytic leukemia
SRSF2Orphanet:98849Systemic mastocytosis with associated hematologic neoplasm
SRSF2Orphanet:98850Aggressive systemic mastocytosis
TP53Orphanet:1333Familial pancreatic carcinoma
TP53Orphanet:145Hereditary breast and/or ovarian cancer syndrome
TP53Orphanet:1501Adrenocortical carcinoma
TP53Orphanet:210159Adult hepatocellular carcinoma
TP53Orphanet:251576Gliosarcoma
TP53Orphanet:251579Giant cell glioblastoma
TP53Orphanet:251899Choroid plexus carcinoma
TP53Orphanet:2807Papilloma of choroid plexus
TP53Orphanet:293199Pleomorphic rhabdomyosarcoma
TP53Orphanet:3318Essential thrombocythemia
TP53Orphanet:524Li-Fraumeni syndrome
TP53Orphanet:52688Myelodysplastic syndrome
TP53Orphanet:585909B-lymphoblastic leukemia/lymphoma with t(9;22)(q34.1;q11.2)
TP53Orphanet:667662Breast implant-associated anaplastic large cell lymphoma
TP53Orphanet:668Osteosarcoma
TP53Orphanet:67038B-cell chronic lymphocytic leukemia
TP53Orphanet:70573Small cell lung cancer
TP53Orphanet:96253Cushing disease
TP53Orphanet:99756Alveolar rhabdomyosarcoma
TP53Orphanet:99757Embryonal rhabdomyosarcoma
FGFR1Orphanet:168953Myeloid/lymphoid neoplasm associated with FGFR1 rearrangement
FGFR1Orphanet:2117Hartsfield syndrome
FGFR1Orphanet:220386Semilobar holoprosencephaly
FGFR1Orphanet:2396Encephalocraniocutaneous lipomatosis
FGFR1Orphanet:251576Gliosarcoma
FGFR1Orphanet:251579Giant cell glioblastoma
FGFR1Orphanet:251615Pilomyxoid astrocytoma
FGFR1Orphanet:2645Osteoglosphonic dysplasia
FGFR1Orphanet:280200Microform holoprosencephaly
FGFR1Orphanet:314950Primary hypereosinophilic syndrome
FGFR1Orphanet:3157Septo-optic dysplasia spectrum
FGFR1Orphanet:3366Non-syndromic metopic craniosynostosis
FGFR1Orphanet:432Normosmic congenital hypogonadotropic hypogonadism
FGFR1Orphanet:478Kallmann syndrome
FGFR1Orphanet:93258Pfeiffer syndrome type 1
FGFR1Orphanet:93924Lobar holoprosencephaly
FGFR1Orphanet:99798Oligodontia
FGFR3Orphanet:15Achondroplasia
FGFR3Orphanet:1860Thanatophoric dysplasia type 1
FGFR3Orphanet:2363Lacrimoauriculodentodigital syndrome
FGFR3Orphanet:251576Gliosarcoma
FGFR3Orphanet:251579Giant cell glioblastoma
FGFR3Orphanet:35099Non-syndromic bicoronal craniosynostosis
FGFR3Orphanet:429Hypochondroplasia
FGFR3Orphanet:53271Muenke syndrome
FGFR3Orphanet:794Saethre-Chotzen syndrome
FGFR3Orphanet:85164Camptodactyly-tall stature-scoliosis-hearing loss syndrome

Cohort genes → proteins

5 cohort genes, 5 distinct canonical proteins.

Evidence partition

SubsetGenes
civic_only4
multi_evidence1

Cohort genes (full)

SymbolHGNCEnsemblUniProtNameEvidence
SRSF2HGNC:10783ENSG00000161547Q01130Serine/arginine-rich splicing factor 2civic_evidence
TP53HGNC:11998ENSG00000141510P04637Cellular tumor antigen p53civic_evidence
FGFR1HGNC:3688ENSG00000077782P11362Fibroblast growth factor receptor 1civic_evidence
FGFR3HGNC:3690ENSG00000068078P22607Fibroblast growth factor receptor 3civic_evidence
TRIP11HGNC:12305ENSG00000100815Q15643Thyroid receptor-interacting protein 11clinvar

Cohort function summary

Lead sentence per gene, UniProt-curated.

SymbolProtein nameFunction (lead sentence)
SRSF2Serine/arginine-rich splicing factor 2Necessary for the splicing of pre-mRNA.
TP53Cellular tumor antigen p53Multifunctional transcription factor that induces cell cycle arrest, DNA repair or apoptosis upon binding to its target DNA sequence.
FGFR1Fibroblast growth factor receptor 1Tyrosine-protein kinase that acts as a cell-surface receptor for fibroblast growth factors and plays an essential role in the regulation of embryonic development, cell proliferation, differentiation and migration.
FGFR3Fibroblast growth factor receptor 3Tyrosine-protein kinase that acts as a cell-surface receptor for fibroblast growth factors and plays an essential role in the regulation of cell proliferation, differentiation and apoptosis.
TRIP11Thyroid receptor-interacting protein 11Is a membrane tether required for vesicle tethering to Golgi.

Protein-family classification

Druggable: 2 · Difficult: 1 · Unknown: 2 · Druggable fraction: 0.4

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.

FamilyGenesFoldFDR
Kinase211.1×0.036
Transcription factor11.6×0.713
Other/Unknown20.7×0.877

Per-gene assignment

SymbolFamilyDruggable?ECInterPro (top 3)
SRSF2Other/UnknownnoRRM_dom, RRM_euk-type, Nucleotide-bd_a/b_plait_sf
TP53Transcription factornop53_tumour_suppressor, p53-like_TF_DNA-bd_sf, p53_tetrameristn
FGFR1Kinaseyes2.7.10.1Prot_kinase_dom, Ser-Thr/Tyr_kinase_cat_dom, Ig_sub2
FGFR3Kinaseyes2.7.10.1Prot_kinase_dom, Ser-Thr/Tyr_kinase_cat_dom, Ig_sub2
TRIP11Other/UnknownnoGRIP_dom

Expression context

Cohort genes with no expression data: 0.

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

Breadth distribution (Bgee present_calls)

BucketGenes
narrow (1-5 tissues)0
moderate (6-20)0
broad (>20)5
unknown0

Top tissues across cohort

TissueCohort genes
tendon of biceps brachii2
calcaneal tendon2
embryo1
tibia1
ganglionic eminence1
ventricular zone1
buccal mucosa cell1
stromal cell of endometrium1
skin of hip1
upper arm skin1
upper leg skin1
adrenal tissue1
male germ line stem cell (sensu Vertebrata) in testis1

Per-gene tissue summary (top 30)

SymbolBgee breadthFANTOM5 breadthSCXATop tissues
SRSF2295ubiquitousmarkertibia, embryo, tendon of biceps brachii
TP53223ubiquitousmarkerventricular zone, ganglionic eminence, tendon of biceps brachii
FGFR1292ubiquitousmarkerbuccal mucosa cell, stromal cell of endometrium, calcaneal tendon
FGFR3262broadmarkerupper leg skin, skin of hip, upper arm skin
TRIP11270ubiquitousmarkercalcaneal tendon, male germ line stem cell (sensu Vertebrata) in testis, adrenal tissue

Protein interactions among cohort

Intra-cohort edges: 0.

Hub genes (top 10 by interactor count)

SymbolInteractor count
TP5322,736
FGFR15,693
FGFR34,510
SRSF23,311
TRIP112,991

Structural data

PDB: 4 · AlphaFold-only: 1 · No structure: 0

Cohort genes with PDB structures (top 30)

SymbolUniProtPDB entries
TP53P04637313
FGFR1P1136283
FGFR3P2260715
SRSF2Q011304

AlphaFold-only cohort genes (top 30 by pLDDT)

SymbolUniProtpLDDT
TRIP11Q1564366.78

Function

Pathway analysis

Distinct Reactome pathways touched by cohort: 95. Enrichment computed across 5 evidence-associated genes (5 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 5 annotated cohort genes). Counts and members are kept as ground-truth; sorted by enrichment.

PathwayCohort genesFoldFDRSample cohort genes
t(4;14) translocations of FGFR312284.0×0.010FGFR3
Signaling by FGFR3 fusions in cancer12284.0×0.010FGFR3
Loss of function of TP53 in cancer due to loss of tetramerization ability12284.0×0.010TP53
PI3K Cascade2108.8×0.010FGFR1, FGFR3
Constitutive Signaling by Aberrant PI3K in Cancer250.8×0.011FGFR1, FGFR3
Signaling by FGFR1 amplification mutants11142.0×0.012FGFR1
Regulation of TP53 Expression11142.0×0.012TP53
PI5P, PP2A and IER3 Regulate PI3K/AKT Signaling238.7×0.012FGFR1, FGFR3
FGFR1c and Klotho ligand binding and activation1571.0×0.015FGFR1
Transcriptional activation of cell cycle inhibitor p211571.0×0.015TP53
Signaling by plasma membrane FGFR1 fusions1571.0×0.015FGFR1
PIP3 activates AKT signaling226.7×0.017FGFR1, FGFR3
Activation of NOXA and translocation to mitochondria1380.7×0.018TP53
RAF/MAP kinase cascade224.4×0.018FGFR1, FGFR3
FGFR3b ligand binding and activation1326.3×0.018FGFR3
RUNX3 regulates CDKN1A transcription1326.3×0.018TP53
Epithelial-Mesenchymal Transition (EMT) during gastrulation1285.5×0.019FGFR1
FGFR1b ligand binding and activation1253.8×0.019FGFR1
PI5P Regulates TP53 Acetylation1253.8×0.019TP53
Activation of PUMA and translocation to mitochondria1228.4×0.019TP53
Signaling by activated point mutants of FGFR11190.3×0.019FGFR1
Signaling by activated point mutants of FGFR31190.3×0.019FGFR3
TP53 Regulates Transcription of Caspase Activators and Caspases1190.3×0.019TP53
TP53 Regulates Transcription of Death Receptors and Ligands1190.3×0.019TP53
FGFR3c ligand binding and activation1175.7×0.019FGFR3
Phospholipase C-mediated cascade; FGFR31175.7×0.019FGFR3
Urea cycle1175.7×0.019TP53
Regulation of TP53 Activity through Association with Co-factors1163.1×0.019TP53
FGFR1c ligand binding and activation1152.3×0.019FGFR1
TP53 regulates transcription of several additional cell death genes whose specific roles in p53-dependent apoptosis remain uncertain1152.3×0.019TP53

GO biological processes by enrichment

Over-representation of cohort genes vs the genome-wide background (hypergeometric test, Benjamini-Hochberg FDR; fold = observed/expected over 5 annotated cohort genes). Counts and members are kept as ground-truth; sorted by enrichment.

GO termCohort genesFoldFDRSample cohort genes
positive regulation of phospholipase activity21348.2×1e-04FGFR1, FGFR3
stem cell proliferation2124.8×0.006TP53, FGFR1
chondrocyte differentiation2120.4×0.006FGFR1, FGFR3
fibroblast growth factor receptor signaling pathway2114.2×0.006FGFR1, FGFR3
negative regulation of developmental growth13370.4×0.006FGFR3
negative regulation of helicase activity13370.4×0.006TP53
cellular response to actinomycin D13370.4×0.006TP53
regulation of intrinsic apoptotic signaling pathway by p53 class mediator13370.4×0.006TP53
negative regulation of G1 to G0 transition13370.4×0.006TP53
positive regulation of mitochondrial membrane permeability11685.2×0.006TP53
vitamin D3 metabolic process11685.2×0.006FGFR1
oligodendrocyte apoptotic process11685.2×0.006TP53
fibroblast growth factor receptor apoptotic signaling pathway11685.2×0.006FGFR3
positive regulation of mitotic cell cycle DNA replication11685.2×0.006FGFR1
negative regulation of glucose catabolic process to lactate via pyruvate11685.2×0.006TP53
negative regulation of pentose-phosphate shunt11685.2×0.006TP53
positive regulation of parathyroid hormone secretion11685.2×0.006FGFR1
regulation of extrinsic apoptotic signaling pathway in absence of ligand11685.2×0.006FGFR1
regulation of phosphate transport11123.5×0.006FGFR1
obsolete homolactic fermentation11123.5×0.006TP53
fibroblast growth factor receptor signaling pathway involved in orbitofrontal cortex development11123.5×0.006FGFR1
regulation of lateral mesodermal cell fate specification11123.5×0.006FGFR1
bone maturation11123.5×0.006FGFR3
signal transduction by p53 class mediator11123.5×0.006TP53
negative regulation of miRNA processing11123.5×0.006TP53
intrinsic apoptotic signaling pathway in response to hypoxia11123.5×0.006TP53
regulation of fibroblast apoptotic process11123.5×0.006TP53
MAPK cascade261.3×0.006FGFR1, FGFR3
skeletal system development250.3×0.006FGFR1, FGFR3
T cell proliferation involved in immune response1842.6×0.007TP53

Therapeutics

Drug target analysis

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

Druggability breadth: 4 of 5 evidence-associated genes (80%) 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.

SymbolExample approved molecule
TP53NITROFURANTOIN
FGFR1PONATINIB
FGFR3PONATINIB

Top cohort targets by molecule count

SymbolMoleculesMax phase
TP531964
FGFR1934
FGFR3644
SRSF212
TRIP1100

Drugs targeting cohort genes (top 30)

MoleculeMax phaseTargets in cohort
NITROFURANTOIN4TP53
DIOSMIN4TP53
VERTEPORFIN4TP53
CANDESARTAN CILEXETIL4TP53
DIENESTROL4TP53
CLOTRIMAZOLE4TP53
COLCHICINE4TP53
NABUMETONE4TP53
SALMETEROL XINAFOATE4TP53
AMIODARONE HYDROCHLORIDE4TP53
FURAZOLIDONE4TP53
AMOXAPINE4TP53
RALOXIFENE HYDROCHLORIDE4TP53
NICARDIPINE HYDROCHLORIDE4TP53
SULCONAZOLE NITRATE4TP53
PYRITHIONE ZINC4TP53
LACTIC ACID4TP53
OXYMETHOLONE4TP53
CHLOROXINE4TP53
PROPIOLACTONE4TP53
CLOMIPRAMINE HYDROCHLORIDE4TP53
PHENYL AMINOSALICYLATE4TP53
THIORIDAZINE HYDROCHLORIDE4TP53
AMITRIPTYLINE HYDROCHLORIDE4TP53
ETHOPROPAZINE HYDROCHLORIDE4TP53
MECHLORETHAMINE HYDROCHLORIDE4TP53
ECONAZOLE NITRATE4TP53
TRIFLUPROMAZINE HYDROCHLORIDE4TP53
PROCHLORPERAZINE EDISYLATE4TP53
DEQUALINIUM CHLORIDE4TP53

Bioactivity and enzyme data

Enzyme cohort genes (≥1 EC): 2.

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

SymbolAssaysType breakdown
FGFR11,465Binding:1428, Functional:24, ADMET:13
FGFR3975Binding:948, Functional:18, ADMET:9
TP53869Binding:775, ADMET:83, Functional:10, Toxicity:1
SRSF28Binding:8

Cohort enzymes (BRENDA EC)

SymbolEC numbersNames
FGFR12.7.10.1receptor protein-tyrosine kinase
FGFR32.7.10.1receptor protein-tyrosine kinase

Cohort genes with high screening signal

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

SymbolChEMBL assays
TP53869
FGFR11,465
FGFR3975

Pharmacogenomics

Cohort genes with a PharmGKB record: 5; 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.

CompoundMax phaseCohort target (bioactivity)
NITROFURANTOIN4TP53
DIOSMIN4TP53
VERTEPORFIN4TP53
CANDESARTAN CILEXETIL4TP53
DIENESTROL4TP53
CLOTRIMAZOLE4TP53
COLCHICINE4TP53
NABUMETONE4TP53
SALMETEROL XINAFOATE4TP53
AMIODARONE HYDROCHLORIDE4TP53
FURAZOLIDONE4TP53
AMOXAPINE4TP53
RALOXIFENE HYDROCHLORIDE4TP53
NICARDIPINE HYDROCHLORIDE4TP53
SULCONAZOLE NITRATE4TP53
PYRITHIONE ZINC4TP53
LACTIC ACID4TP53
OXYMETHOLONE4TP53
CHLOROXINE4TP53
PROPIOLACTONE4TP53
CLOMIPRAMINE HYDROCHLORIDE4TP53
PHENYL AMINOSALICYLATE4TP53
THIORIDAZINE HYDROCHLORIDE4TP53
AMITRIPTYLINE HYDROCHLORIDE4TP53
ETHOPROPAZINE HYDROCHLORIDE4TP53
MECHLORETHAMINE HYDROCHLORIDE4TP53
ECONAZOLE NITRATE4TP53
TRIFLUPROMAZINE HYDROCHLORIDE4TP53
PROCHLORPERAZINE EDISYLATE4TP53
DEQUALINIUM CHLORIDE4TP53

Druggability pyramid

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

TierDefinitionGenesSymbols
AApproved (phase 4 drug)3TP53, FGFR1, FGFR3
BPhased (≥1) drug, not yet approved1SRSF2
CDruggable family + PDB, no drug0
DDruggable family + AlphaFold only, no drug0
EDifficult family or no structure, no drug1TRIP11

Undrugged target profiles

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

SymbolChEMBL assaysDrugged partners (top 3)
TRIP110

Clinical trials & evidence

Clinical trials

Clinical trials: 63.

Phase distribution (across all retrieved trials)

PhaseTrials
PHASE222
PHASE111
PHASE1/PHASE210
Not specified10
PHASE36
PHASE2/PHASE33
PHASE41

Top trials by phase / activity

NCTPhaseStatusTitle
NCT03608059PHASE4COMPLETEDATG/PTCy in Haplo-PBSCT Randomized Controlled,Multi-center
NCT07052422PHASE2/PHASE3NOT_YET_RECRUITINGVEN+DAC+Bu2Flu4 vs Bu2Flu5 Conditioning Regimen for Elderly Myeloid Malignancies Undergoing Allo-HSCT
NCT07396480PHASE3RECRUITINGFludarabine Plus Melphalan Versus Addition of Venetoclax to Fludarabine/Melphalan Conditioning Regimen for Allogeneic Hematopoietic Stem Cell Transplantation in AML/MDS Patients Aged > 50 Years: a Multicenter, Randomized, Phase 3 Trial
NCT00866918PHASE3COMPLETEDCombination Chemotherapy in Treating Young Patients With Newly Diagnosed Acute Promyelocytic Leukemia
NCT01307579PHASE3COMPLETEDCaspofungin Versus Fluconazole in Preventing Invasive Fungal Infections (IFI) in Patients Undergoing Chemotherapy for Acute Myeloid Leukemia
NCT01366612PHASE3TERMINATEDFludarabine and Busulfan vs. Fludarabine, Busulfan and Total Body Irradiation
NCT01371981PHASE3COMPLETEDBortezomib and Sorafenib Tosylate in Treating Patients With Newly Diagnosed Acute Myeloid Leukemia
NCT01817075PHASE3COMPLETEDChlorhexidine Gluconate Cleansing in Preventing Central Line Associated Bloodstream Infection and Acquisition of Multi-drug Resistant Organisms in Younger Patients With Cancer or Undergoing Donor Stem Cell Transplant
NCT04098653PHASE2/PHASE3UNKNOWNDecitabine + BUCY vs BUCY Conditioning Regimen for Myeloid Tumors Undergoing Allo-HSCT
NCT04123392PHASE2/PHASE3UNKNOWNDecitabine + BUCY vs BUCY Conditioning Regimen for TP53+ Myeloid Tumors Undergoing Allo-HSCT
NCT03850418PHASE2RECRUITINGAzacitidine and Chimerism in MDS or AML Patients After Allogeneic Stem Cell Transplant
NCT03862157PHASE1/PHASE2ACTIVE_NOT_RECRUITINGAzacitidine, Venetoclax, and Pevonedistat in Treating Patients With Newly Diagnosed Acute Myeloid Leukemia
NCT04195945PHASE2ACTIVE_NOT_RECRUITINGCPX-351 or CLAG-M Regimen for the Treatment of Acute Myeloid Leukemia or Other High-Grade Myeloid Neoplasms in Medically Less-Fit Patients
NCT04501120PHASE1/PHASE2RECRUITINGStudy of Lisaftoclax (APG-2575) Single Agent and Combination With Therapy in Patients Relapsed/Refractory AML
NCT04797767PHASE1/PHASE2RECRUITINGVenetoclax and CLAG-M for the Treatment of Acute Myeloid Leukemia and High-Grade Myeloid Neoplasms
NCT05031897PHASE2RECRUITINGTwo Step Haplo With Radiation Conditioning
NCT05656248PHASE2ACTIVE_NOT_RECRUITINGStudy of CPX-351 (VYXEOS) in Individuals < 22 Years With Secondary Myeloid Neoplasms
NCT05796570PHASE2RECRUITINGA Pilot Study to Evaluate the Feasibility of Post-Hematopoietic Stem Cell Transplant Prophylaxis With Decitabine Combined With Filgrastim for Children and Young Adults With AML, MDS and Related Myeloid Malignancies
NCT05841771PHASE2RECRUITINGHypomethylating Agent and Venetoclax After Allo-HSCT in Patients With High-risk Myeloid Malignancies.
NCT06129734PHASE1/PHASE2RECRUITINGDecitabine and Venetoclax Treatment as Maintenance Therapy in Patients Post Allograft Stem Cell Transplant
NCT06383572PHASE1/PHASE2RECRUITINGPhase I/II Study of Engineered T Cell Receptor-Modified NK Cells Targeting PRAME in Conjunction With Lymphodepleting Chemotherapy for the Management of Relapse/Refractory Myeloid Malignancies
NCT06434662PHASE2RECRUITINGMitoxantrone Hydrochloride Liposome Injection, Cytarabine Combined With Venetoclax in the Treatment of R/R AML
NCT06621212PHASE2RECRUITINGMitoxantrone Hydrochloride Liposome, Standard-dose of Cytarabine and Venetoclax in the Treatment of R/R AML
NCT06668584PHASE2RECRUITINGA Phase II Open-label Study of Olutasidenib Post-transplant Maintenance Therapy for Patients With IDH1-mutated Myeloid Malignancies
NCT06917105PHASE1/PHASE2NOT_YET_RECRUITINGExploratory Clinical Study on the Safety and Efficacy of CAR-T Cell Therapy in the Treatment of Relapsed/Refractory Myeloid Malignancies
NCT06930651PHASE1/PHASE2RECRUITINGA Phase I/II Study of CAR.70-Engineered IL15-Transduced Cord Blood-Derived NK Cells With TGF-beta Receptor 2 (TGFBR2) Knock Out in Conjunction With Lymphodepleting Chemotherapy for the Management of Relapsed/Refractory Myeloid Malignancies
NCT07011186PHASE1/PHASE2RECRUITINGA Clinical Trial of TQB3909 Tablets in Combination With Azacitidine for the Treatment of Myeloid Malignancies
NCT01596699PHASE2TERMINATEDPilot Trial of Clofarabine Added to Standard Busulfan and Fludarabine for Conditioning Prior to Allogeneic Hematopoietic Cell Transplantation
NCT01731951PHASE2COMPLETEDImetelstat Sodium in Treating Participants With Primary or Secondary Myelofibrosis
NCT02452697PHASE2UNKNOWNPh2 NK Cell Enriched DCIs w/wo RLR9 Agonist, DUK-CPG-001 From Donors Following Allogeneic SCT
NCT02756572PHASE2COMPLETEDEarly Allogeneic Hematopoietic Cell Transplantation in Treating Patients With Relapsed or Refractory High-Grade Myeloid Neoplasms
NCT02958384PHASE1/PHASE2UNKNOWNA Clinical Research of LeY-Targeted CAR-T in Myeloid Malignancies
NCT02958397PHASE1/PHASE2UNKNOWNA Clinical Research of CD33-Targeted CAR-T in Myeloid Malignancies
NCT03012672PHASE2COMPLETEDHigher or Lower Dose Cladribine, Cytarabine, and Mitoxantrone in Treating Medically Less Fit Patients With Newly Diagnosed Acute Myeloid Leukemia or Myeloid Neoplasm
NCT03270748PHASE2COMPLETEDPost Transplant High-Dose Cy as GvHD Prophylaxis in 1 HLA Mismatched Unrelated HSCT for Myeloid Malignancies
NCT04526288PHASE2TERMINATEDCPX-351 Versus Immediate Stem Cell Transplantation for the Treatment of High-Grade Myeloid Cancers With Measurable Residual Disease
NCT04778410PHASE2TERMINATEDStudy of Magrolimab Combinations in Participants With Myeloid Malignancies
NCT04906031PHASE2UNKNOWNSodium Stibogluconate in the MDS/AML With One of the 65 Defined p53 Mutations
NCT05115630PHASE2COMPLETEDOff-the-shelf NK Cells + SCT for Myeloid Malignancies
NCT05579769PHASE2TERMINATEDPediatric Study of GVHD Ppx w/o Calcineurin Inhibitors After Day60 Post First Allo HSCT for Hematological Malignancies.

Drugs tested across these trials (top 30)

MoleculeMax phaseTrials referencing
MITOXANTRONE48
CLADRIBINE45
BUSULFAN43
DECITABINE43
MERCAPTOPURINE ANHYDROUS43
SORAFENIB43
TACROLIMUS ANHYDROUS42
ARSENIC TRIOXIDE41
ASPARAGINASE41
BORTEZOMIB41
CASPOFUNGIN ACETATE41
CLOFARABINE41
DAUNORUBICIN HYDROCHLORIDE41
FLUCONAZOLE41
FLUDARABINE PHOSPHATE41
IDARUBICIN41
MELPHALAN41
OLUTASIDENIB41
SODIUM STIBOGLUCONATE41
TRETINOIN41
VENETOCLAX41
EPRENETAPOPT31
FLUDARABINE31
IMETELSTAT31
MAGROLIMAB31
NAVITOCLAX31
OLVEREMBATINIB31
PEVONEDISTAT31
APG-257521
CHEMBL477688105

Precision-medicine subtype map (CIViC)

Drug × molecular subtype: 6 predictive associations from 6 curated evidence items; also 6 oncogenic, 1 predisposing, 1 prognostic.

Molecular subtypeTherapyEffectLevelCIViC
ZMYM2::FGFR1 Fusion OR BCR::FGFR1 Fusion OR CEP43::FGFR1 Fusion OR TRIM24::FGFR1 Fusion OR FGFR1 TranslocationPemigatinibSensitivity/ResponseCIViC AEID11324
ETV6::ABL1 FusionImatinib + Dasatinib + NilotinibSensitivity/ResponseCIViC BEID11326
PCM1::JAK2 Fusion OR BCR::JAK2 FusionRuxolitinibSensitivity/ResponseCIViC BEID11325
SRSF2 P95HCTX-712Sensitivity/ResponseCIViC DEID11028
SRSF2 P95LCTX-712Sensitivity/ResponseCIViC DEID11027
FGFR3 V555MFexagratinib + AZ12908010 + PD173074ResistanceCIViC DEID6408