Chronic myelomonocytic leukemia

Summary

Chronic myelomonocytic leukemia (MONDO:0020311) is a cancer with 2 cohort genes (2 CIViC-evidence somatic drivers; 1 ClinVar predisposition record) and 279 clinical trials. Molecularly, ZMIZ1::ABL1 Fusion confers sensitivity to Azacitidine + Dasatinib in Chronic Myelomonocytic Leukemia (CIViC Level C). Top therapeutic interventions include fludarabine phosphate, tacrolimus anhydrous, and decitabine.

At a glance

• Classification: Cancer
• Prevalence: 1-9 / 1 000 000 (Worldwide) [Orphanet-validated]
• Cohort genes: 2
• ClinVar variants: 1
• Clinical trials: 279
• Precision-medicine evidence (CIViC): 1 subtype–drug association

Clinical features

Epidemiology

Prevalence records

1 prevalence record(s), Orphanet:

Type	Class	Value	Geography	Validation
Annual incidence	1-9 / 1 000 000	0.68	Worldwide	Validated

Identifiers

Disease identifiers

Field	Value
Canonical name	chronic myelomonocytic leukemia
Mondo ID	MONDO:0020311
EFO	EFO:1001779
MeSH	D015477
Orphanet	98823
DOID	DOID:0080188
ICD-10-CM	C93.1
ICD-11	2073226578
NCIT	C3178
SNOMED CT	127225006
UMLS	C0023480
MedGen	44125
GARD	0008225
MedDRA	10009018
Is cancer (heuristic)	yes

Also known as: chronic myelomonocytic leukaemia (CMML) · chronic myelomonocytic leukemia · chronic myelomonocytic leukemia (CMML) · CMML

Data availability: 1 ClinVar variant · 1 cell line.

Disease family

An umbrella term covering 1 Mondo subtype.

Classification path: disease › 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 › chronic leukemia › chronic myelomonocytic leukemia

Related subtypes (7): prolymphocytic leukemia, chronic monocytic leukemia, B-cell chronic lymphocytic leukemia, chronic eosinophilic leukemia, chronic neutrophilic leukemia, T-cell large granular lymphocyte leukemia, aggressive NK-cell leukemia

Subtypes (1): juvenile myelomonocytic leukemia

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

ClinVar germline variants

1 retrieved; paginated sample, class counts are floors:

1 likely pathogenic

ClinVar	Variant (HGVS)	Gene	Classification	Review
590266	t(12;13)(p13.2;q12.2)	ETV6	Likely pathogenic	no assertion criteria provided

Genes & proteins

Mendelian disease overlap and somatic drivers

GenCC: 0 · Orphanet: 18 · OMIM-shared: 0 · Dual-evidence (GWAS+Mendelian): 0

Somatic driver evidence (intOGen + CIViC, cohort fanout)

Gene	intOGen role	Cancer types	CIViC
KRAS	Act	ALL,AML,ANSC,BLADDER,BLCA,BRCA,CEAD,CESC,CHOL,CLLSLL,COAD,COADREAD,DLBCLNOS,EGC,ESCA,ESCC,HCC,LUAD,LUSC,MEL,MGCT,MT,NSCLC,OVT,PAAD,PANCREAS,PAST,PCM,PRAD,PRCC,READ,STAD,STOMACH,UCEC,UCS,WDTC	CIViC #30
ETV6	Act	ALL,BLCA,DLBCLNOS	CIViC #1769

Orphanet rare-disease linkage (cohort genes)

Gene	Orphanet ID	Rare disease
KRAS	Orphanet:1333	Familial pancreatic carcinoma
KRAS	Orphanet:1340	Cardiofaciocutaneous syndrome
KRAS	Orphanet:144	Lynch syndrome
KRAS	Orphanet:146	Differentiated thyroid carcinoma
KRAS	Orphanet:2396	Encephalocraniocutaneous lipomatosis
KRAS	Orphanet:251615	Pilomyxoid astrocytoma
KRAS	Orphanet:2612	Linear nevus sebaceus syndrome
KRAS	Orphanet:268114	RAS-associated autoimmune leukoproliferative disease
KRAS	Orphanet:3339	Oculoectodermal syndrome
KRAS	Orphanet:648	Noonan syndrome
KRAS	Orphanet:86834	Juvenile myelomonocytic leukemia
ETV6	Orphanet:146	Differentiated thyroid carcinoma
ETV6	Orphanet:168629	Autosomal thrombocytopenia with normal platelets
ETV6	Orphanet:2030	Fibrosarcoma
ETV6	Orphanet:2665	Congenital mesoblastic nephroma
ETV6	Orphanet:314950	Primary hypereosinophilic syndrome
ETV6	Orphanet:585929	B-lymphoblastic leukemia/lymphoma with t(12;21)(p13.2;q22.1)
ETV6	Orphanet:98823	Chronic myelomonocytic leukemia

Cohort genes → proteins

2 cohort genes, 2 distinct canonical proteins.

Evidence partition

Subset	Genes
civic_only	1
multi_evidence	1

Cohort genes (full)

Symbol	HGNC	Ensembl	UniProt	Name	Evidence
KRAS	HGNC:6407	ENSG00000133703	P01116	GTPase KRas	civic_evidence
ETV6	HGNC:3495	ENSG00000139083	P41212	Transcription factor ETV6	clinvar

Cohort function summary

Lead sentence per gene, UniProt-curated.

Symbol	Protein name	Function (lead sentence)
KRAS	GTPase KRas	Ras proteins bind GDP/GTP and possess intrinsic GTPase activity.
ETV6	Transcription factor ETV6	Transcriptional repressor; binds to the DNA sequence 5’-CCGGAAGT-3'.

Protein-family classification

Druggable: 1 · Difficult: 0 · Unknown: 1 · Druggable fraction: 0.5

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
Enzyme (other)	1	6.0×	0.320
Other/Unknown	1	0.9×	0.805

Per-gene assignment

Symbol	Family	Druggable?	EC	InterPro (top 3)
KRAS	Enzyme (other)	yes	3.6.5.2	Small_GTPase, Small_GTP-bd, Small_GTPase_Ras-type
ETV6	Other/Unknown	no		Ets_dom, Pointed_dom, SAM/pointed_sf

Expression context

Cohort genes with no expression data: 0.

2 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)	2
unknown	0

Top tissues across cohort

Tissue	Cohort genes
nipple	1
pylorus	1
trigeminal ganglion	1
mammary duct	1
mucosa of paranasal sinus	1
parotid gland	1

Per-gene tissue summary (top 30)

Symbol	Bgee breadth	FANTOM5 breadth	SCXA	Top tissues
KRAS	298	ubiquitous	marker	trigeminal ganglion, pylorus, nipple
ETV6	252	ubiquitous	marker	mucosa of paranasal sinus, parotid gland, mammary duct

Protein interactions among cohort

Intra-cohort edges: 0.

Hub genes (top 10 by interactor count)

Symbol	Interactor count
KRAS	14,509
ETV6	2,225

Structural data

PDB: 2 · AlphaFold-only: 0 · No structure: 0

Cohort genes with PDB structures (top 30)

Symbol	UniProt	PDB entries
KRAS	P01116	511
ETV6	P41212	44

Function

Pathway analysis

Distinct Reactome pathways touched by cohort: 72. Enrichment computed across 2 evidence-associated genes (2 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 2 annotated cohort genes). Counts and members are kept as ground-truth; sorted by enrichment.

Pathway	Cohort genes	Fold	FDR	Sample cohort genes
Signaling by FLT3 fusion proteins	2	571.0×	2e-04	ETV6, KRAS
Signaling by RAS GAP mutants	1	1903.3×	0.006	KRAS
Signaling by RAS GTPase mutants	1	1903.3×	0.006	KRAS
Activation of RAS in B cells	1	1142.0×	0.006	KRAS
Signaling by membrane-tethered fusions of PDGFRA or PDGFRB	1	1142.0×	0.006	ETV6
RAS signaling downstream of NF1 loss-of-function variants	1	815.7×	0.006	KRAS
Estrogen-stimulated signaling through PRKCZ	1	815.7×	0.006	KRAS
SOS-mediated signalling	1	713.8×	0.006	KRAS
Activated NTRK3 signals through RAS	1	634.4×	0.006	KRAS
EGFR Transactivation by Gastrin	1	571.0×	0.006	KRAS
SHC-related events triggered by IGF1R	1	571.0×	0.006	KRAS
RUNX3 regulates p14-ARF	1	571.0×	0.006	KRAS
Activated NTRK2 signals through RAS	1	571.0×	0.006	KRAS
MET activates RAS signaling	1	519.1×	0.006	KRAS
Signaling by FGFR4 in disease	1	475.8×	0.006	KRAS
Activated NTRK2 signals through FRS2 and FRS3	1	475.8×	0.006	KRAS
Constitutive Signaling by Overexpressed ERBB2	1	475.8×	0.006	KRAS
p38MAPK events	1	439.2×	0.006	KRAS
Signaling by PDGFRA transmembrane, juxtamembrane and kinase domain mutants	1	439.2×	0.006	KRAS
Signaling by PDGFRA extracellular domain mutants	1	439.2×	0.006	KRAS
PTK6 Regulates RHO GTPases, RAS GTPase and MAP kinases	1	407.9×	0.006	KRAS
GRB2 events in EGFR signaling	1	380.7×	0.006	KRAS
Erythropoietin activates RAS	1	380.7×	0.006	KRAS
Signaling by FLT3 ITD and TKD mutants	1	380.7×	0.006	KRAS
SHC1 events in ERBB4 signaling	1	356.9×	0.006	KRAS
SHC1 events in EGFR signaling	1	356.9×	0.006	KRAS
Constitutive Signaling by EGFRvIII	1	356.9×	0.006	KRAS
Signalling to RAS	1	335.9×	0.006	KRAS
Insulin receptor signalling cascade	1	335.9×	0.006	KRAS
Signaling by ERBB2 ECD mutants	1	335.9×	0.006	KRAS

GO biological processes by enrichment

Over-representation of cohort genes vs the genome-wide background (hypergeometric test, Benjamini-Hochberg FDR; fold = observed/expected over 2 annotated cohort genes). Counts and members are kept as ground-truth; sorted by enrichment.

GO term	Cohort genes	Fold	FDR	Sample cohort genes
response to mineralocorticoid	1	8426.0×	0.005	KRAS
forebrain astrocyte development	1	2808.7×	0.005	KRAS
response to isolation stress	1	2106.5×	0.005	KRAS
vitellogenesis	1	1685.2×	0.005	ETV6
response to gravity	1	1404.3×	0.005	KRAS
type I pneumocyte differentiation	1	766.0×	0.006	KRAS
mesenchymal cell apoptotic process	1	766.0×	0.006	ETV6
myoblast proliferation	1	702.2×	0.006	KRAS
positive regulation of cellular senescence	1	648.1×	0.006	KRAS
negative regulation of epithelial cell differentiation	1	601.9×	0.006	KRAS
regulation of synaptic transmission, GABAergic	1	526.6×	0.006	KRAS
regulation of long-term neuronal synaptic plasticity	1	495.6×	0.006	KRAS
striated muscle cell differentiation	1	495.6×	0.006	KRAS
glial cell proliferation	1	443.5×	0.006	KRAS
epithelial tube branching involved in lung morphogenesis	1	421.3×	0.006	KRAS
positive regulation of glial cell proliferation	1	351.1×	0.007	KRAS
positive regulation of Rac protein signal transduction	1	324.1×	0.007	KRAS
hematopoietic stem cell proliferation	1	324.1×	0.007	ETV6
cardiac muscle cell proliferation	1	290.6×	0.007	KRAS
Rac protein signal transduction	1	280.9×	0.007	KRAS
homeostasis of number of cells within a tissue	1	221.7×	0.008	KRAS
skeletal muscle cell differentiation	1	172.0×	0.010	KRAS
response to glucocorticoid	1	162.0×	0.010	KRAS
visual learning	1	153.2×	0.010	KRAS
liver development	1	110.9×	0.013	KRAS
female pregnancy	1	105.3×	0.013	KRAS
neurogenesis	1	104.0×	0.013	ETV6
Ras protein signal transduction	1	102.8×	0.013	KRAS
neuron apoptotic process	1	92.6×	0.014	KRAS
MAPK cascade	1	76.6×	0.016	KRAS

Therapeutics

Drugs indicated or in trials for this disease

10 approved drugs — disease-direct ChEMBL indications, not inferred from the associated-gene cohort below.

Drug	Status
Azacitidine	Approved (phase 4)
Decitabine	Approved (phase 4)
Asparaginase	Approved (phase 3)
Busulfan	Approved (phase 3)
Cytarabine	Approved (phase 3)
Etoposide	Approved (phase 3)
Fludarabine Phosphate	Approved (phase 3)
Idarubicin	Approved (phase 3)
Methotrexate	Approved (phase 3)
Thioguanine	Approved (phase 3)

16 drugs in clinical trials for this disease (phase 2–3, investigational): efficacy not established — a trial record, not an indication.

Drug	Highest phase
Aldesleukin	Phase 3
Dexamethasone	Phase 3
Filgrastim	Phase 3
Hydrocortisone	Phase 3
Lonafarnib	Phase 3
Clofarabine	Phase 2
Cobimetinib	Phase 2
Epoetin Beta	Phase 2
Fludarabine	Phase 2
Lenalidomide	Phase 2
Melphalan	Phase 2
Mycophenolate Mofetil	Phase 2
Pevonedistat	Phase 2
Rituximab	Phase 2
Tacrolimus Anhydrous	Phase 2
Tipifarnib	Phase 2

Drug target analysis

Approved (phase 4): 2 · Phase ≥3: 2 · Phased (≥1): 2 · Undrugged: 0

Druggability breadth: 2 of 2 evidence-associated genes (100%) 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
KRAS	VEMURAFENIB
ETV6	CERITINIB

Top cohort targets by molecule count

Symbol	Molecules	Max phase
KRAS	11	4
ETV6	4	4

Drugs targeting cohort genes (top 30)

Molecule	Max phase	Targets in cohort
VEMURAFENIB	4	KRAS
DABRAFENIB	4	KRAS
LONAFARNIB	4	KRAS
SOTORASIB	4	KRAS
ADAGRASIB	4	KRAS
CERITINIB	4	ETV6
GILTERITINIB	4	ETV6
ERDAFITINIB	4	ETV6
OPNURASIB	3	KRAS
DIVARASIB	2	KRAS
GLECIRASIB	2	KRAS
BMS-214662	1	KRAS
LY-3009120	1	KRAS
MRTX-1133	1	KRAS
LY-2874455	1	ETV6

Bioactivity and enzyme data

Enzyme cohort genes (≥1 EC): 1.

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

Symbol	Assays	Type breakdown
KRAS	861	Binding:829, Functional:32
ETV6	11	Binding:11

Cohort enzymes (BRENDA EC)

Symbol	EC numbers	Names
KRAS	3.6.5.2	small monomeric GTPase

Cohort genes with high screening signal

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

Symbol	ChEMBL assays
KRAS	861

Pharmacogenomics

Cohort genes with a PharmGKB record: 2; with CPIC/DPWG dosing guidelines: 0.

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

Drug repurposing candidates

15 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)
VEMURAFENIB	4	KRAS
DABRAFENIB	4	KRAS
LONAFARNIB	4	KRAS
SOTORASIB	4	KRAS
ADAGRASIB	4	KRAS
CERITINIB	4	ETV6
GILTERITINIB	4	ETV6
ERDAFITINIB	4	ETV6
OPNURASIB	3	KRAS
DIVARASIB	2	KRAS
GLECIRASIB	2	KRAS
BMS-214662	1	KRAS
LY-3009120	1	KRAS
MRTX-1133	1	KRAS
LY-2874455	1	ETV6

Druggability pyramid

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

Tier	Definition	Genes	Symbols
A	Approved (phase 4 drug)	2	KRAS, ETV6
B	Phased (≥1) drug, not yet approved	0
C	Druggable family + PDB, no drug	0
D	Druggable family + AlphaFold only, no drug	0
E	Difficult family or no structure, no drug	0

Undrugged target profiles

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

Clinical trials & evidence

Clinical trials

Clinical trials: 279.

Phase distribution (across all retrieved trials)

Phase	Trials
PHASE2	101
PHASE1	86
PHASE1/PHASE2	51
Not specified	26
PHASE3	11
PHASE2/PHASE3	2
EARLY_PHASE1	2

Top trials by phase / activity

NCT	Phase	Status	Title
NCT00843882	PHASE3	ACTIVE_NOT_RECRUITING	Lenalidomide With or Without Epoetin Alfa in Treating Patients With Myelodysplastic Syndrome and Anemia
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)
NCT04708054	PHASE2/PHASE3	RECRUITING	Venetoclax to Improve Outcomes of Fractionated Busulfan Regimen in Patients With High-Risk AML and MDS
NCT05153226	PHASE3	ACTIVE_NOT_RECRUITING	GvHD Prophylaxis in Unrelated Donor HCT: Randomized Trial Comparing PTCY Versus ATG
NCT06647862	PHASE3	RECRUITING	IMM01+Azacitidine VS Placebo +Azacitidine in Patients With Newly Diagnosed Chronic Myelomonocytic Leukemia (CMML1-2)
NCT00002798	PHASE3	COMPLETED	Combination Chemotherapy With or Without Bone Marrow Transplantation in Treating Children With Acute Myelogenous Leukemia or Myelodysplastic Syndrome
NCT00799461	PHASE3	COMPLETED	Internet-Based Program With or Without Telephone-Based Problem-Solving Training in Helping Long-Term Survivors of Hematopoietic Stem Cell Transplant Cope With Late Complications
NCT01241500	PHASE3	COMPLETED	Randomized Study of ON 01910.Na in Refractory Myelodysplastic Syndrome Patients With Excess Blasts
NCT01305200	PHASE3	COMPLETED	Supersaturated Calcium Phosphate Rinse in Preventing Oral Mucositis in Young Patients Undergoing Autologous or Donor Stem Cell Transplant
NCT01749111	PHASE3	TERMINATED	Comparison Between Cyclophosphamide and Combination of Methotrexate + Calcineurin Inhibitor for GVHD Prophylaxis
NCT01928537	PHASE3	COMPLETED	Efficacy and Safety of IV Rigosertib in MDS Patients With Excess Blasts Progressing After Azacitidine or Decitabine
NCT03306264	PHASE3	COMPLETED	Study of ASTX727 vs IV Decitabine in Participants With MDS, CMML, and AML
NCT04842604	PHASE3	COMPLETED	Continuation Study of B1371019(NCT03416179) and B1371012(NCT02367456) Evaluating Azacitidine With Or Without Glasdegib In Patients With Previously Untreated AML, MDS or CMML
NCT00392353	PHASE1/PHASE2	ACTIVE_NOT_RECRUITING	Vorinostat and Azacitidine in Treating Patients With Myelodysplastic Syndromes or Acute Myeloid Leukemia
NCT01522976	PHASE2	ACTIVE_NOT_RECRUITING	Azacitidine With or Without Lenalidomide or Vorinostat in Treating Patients With Higher-Risk Myelodysplastic Syndromes or Chronic Myelomonocytic Leukemia
NCT01885689	PHASE2	ACTIVE_NOT_RECRUITING	Clofarabine and Melphalan Before Donor Stem Cell Transplant in Treating Patients With Myelodysplasia, Acute Leukemia in Remission, or Chronic Myelomonocytic Leukemia
NCT02727803	PHASE2	RECRUITING	Personalized NK Cell Therapy in CBT
NCT02935361	PHASE1/PHASE2	ACTIVE_NOT_RECRUITING	Guadecitabine and Atezolizumab in Treating Patients With Advanced Myelodysplastic Syndrome or Chronic Myelomonocytic Leukemia That Is Refractory or Relapsed
NCT03128034	PHASE1/PHASE2	RECRUITING	211^At-BC8-B10 Before Donor Stem Cell Transplant in Treating Patients With High-Risk Acute Myeloid Leukemia, Acute Lymphoblastic Leukemia, Myelodysplastic Syndrome, or Mixed-Phenotype Acute Leukemia
NCT03289910	PHASE2	ACTIVE_NOT_RECRUITING	Topotecan Hydrochloride and Carboplatin With or Without Veliparib in Treating Advanced Myeloproliferative Disorders and Acute Myeloid Leukemia or Chronic Myelomonocytic Leukemia
NCT03383575	PHASE2	RECRUITING	Azacitidine and Enasidenib in Treating Patients With IDH2-Mutant Myelodysplastic Syndrome
NCT03418038	PHASE2	RECRUITING	Ascorbic Acid and Chemotherapy for the Treatment of Relapsed or Refractory Lymphoma, CCUS, and Chronic Myelomonocytic Leukemia
NCT03670966	PHASE1/PHASE2	RECRUITING	211At-BC8-B10 Followed by Donor Stem Cell Transplant in Treating Patients With Relapsed or Refractory High-Risk Acute Leukemia or Myelodysplastic Syndrome
NCT03672539	PHASE2	ACTIVE_NOT_RECRUITING	Liposome-encapsulated Daunorubicin-Cytarabine and Gemtuzumab Ozogamicin in Treating Patients With Relapsed or Refractory Acute Myeloid Leukemia (AML) or High Risk Myelodysplastic Syndrome
NCT03683433	PHASE2	RECRUITING	Enasidenib and Azacitidine in Treating Patients With Recurrent or Refractory Acute Myeloid Leukemia and IDH2 Gene Mutation
NCT03722407	PHASE2	ACTIVE_NOT_RECRUITING	Ruxolitinib for the Treatment of Chronic Myelomonocytic Leukemia (CMML): A Phase 2 Expansion
NCT03850574	PHASE1/PHASE2	RECRUITING	Clinical Trial to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Tuspetinib (HM43239) in Patients With Relapsed or Refractory Acute Myeloid Leukemia
NCT03862157	PHASE1/PHASE2	ACTIVE_NOT_RECRUITING	Azacitidine, Venetoclax, and Pevonedistat in Treating Patients With Newly Diagnosed Acute Myeloid Leukemia
NCT03999723	PHASE2	RECRUITING	Combining Active and Passive DNA Hypomethylation
NCT04093570	PHASE2	ENROLLING_BY_INVITATION	A Study for Participants Who Participated in Prior Clinical Studies of ASTX727 (Standard Dose)
NCT04140487	PHASE1/PHASE2	RECRUITING	Azacitidine, Venetoclax, and Gilteritinib in Treating Patients With Recurrent/Refractory FLT3-Mutated Acute Myeloid Leukemia, Chronic Myelomonocytic Leukemia, or High-Risk Myelodysplastic Syndrome/Myeloproliferative Neoplasm
NCT04195633	PHASE2	RECRUITING	Donor Stem Cell Transplant With Treosulfan, Fludarabine, and Total-Body Irradiation for the Treatment of Hematological Malignancies
NCT04239157	PHASE2	RECRUITING	A Phase II, Open-Label, Study of Subcutaneous Canakinumab, an Anti-IL-1β Human Monoclonal Antibody, for Patients With Low or Int-1 Risk IPSS/IPSS-R Myelodysplastic Syndromes and Chronic Myelomonocytic Leukemia
NCT04409639	PHASE2	ACTIVE_NOT_RECRUITING	Cobimetinib in Newly Diagnosed or HMA-treated CMML Patients With RAS Pathway Mutations
NCT04493138	PHASE1/PHASE2	ACTIVE_NOT_RECRUITING	Azacitidine and Quizartinib for the Treatment of Myelodysplastic Syndrome or Myelodysplastic/Myeloproliferative Neoplasm With FLT3 or CBL Mutations
NCT04550442	PHASE1/PHASE2	ACTIVE_NOT_RECRUITING	Venetoclax and Azacitidine for the Treatment of Relapsed or Refractory High-Risk Myelodysplastic Syndrome or Chronic Myelomonocytic Leukemia
NCT04581512	PHASE1/PHASE2	RECRUITING	Study to Evaluate the Safety and Tolerability of EP0042
NCT04655755	PHASE1/PHASE2	ACTIVE_NOT_RECRUITING	Venetoclax in Combination With ASTX727 for the Treatment of Treatment-Naive High-Risk Myelodysplastic Syndrome or Chronic Myelomonocytic Leukemia
NCT04730258	PHASE1/PHASE2	ACTIVE_NOT_RECRUITING	A Study of CFI-400945 With or Without Azacitidine in Patients With AML, MDS or CMML
NCT04734990	PHASE1/PHASE2	ACTIVE_NOT_RECRUITING	Seclidemstat and Azacitidine for the Treatment of Myelodysplastic Syndrome or Chronic Myelomonocytic Leukemia

Drugs tested across these trials (top 30)

Molecule	Max phase	Trials referencing
FLUDARABINE PHOSPHATE	4	47
TACROLIMUS ANHYDROUS	4	26
DECITABINE	4	7
CLOFARABINE	4	6
ENASIDENIB	4	6
AZACITIDINE	4	5
CEDAZURIDINE	4	5
ELTROMBOPAG	4	5
PACRITINIB	4	5
CYCLOPHOSPHAMIDE ANHYDROUS	4	3
GLASDEGIB	4	3
PONATINIB	4	3
SUNITINIB MALATE	4	3
TAGRAXOFUSP	4	3
TREOSULFAN	4	3
VENETOCLAX	4	3
ARSENIC TRIOXIDE	4	2
BUSULFAN	4	2
CLADRIBINE	4	2
COBIMETINIB	4	2
IDARUBICIN	4	2
IDELALISIB	4	2
IMATINIB MESYLATE	4	2
MYCOPHENOLATE SODIUM	4	2
QUIZARTINIB	4	2
VORINOSTAT	4	2
ALDESLEUKIN	4	1
ALEMTUZUMAB	4	1
AMSACRINE	4	1
ASPARAGINASE	4	1

Precision-medicine subtype map (CIViC)

Drug × molecular subtype: 1 predictive associations from 1 curated evidence items; also 3 diagnostic, 1 predisposing.

Molecular subtype	Therapy	Effect	Level	CIViC
ZMIZ1::ABL1 Fusion	Azacitidine + Dasatinib	Sensitivity/Response	CIViC C	EID12636