Disease · MONDO_0011908

juvenile myelomonocytic leukemia

6 sourcesdiseases-2026-09-18-0.1.0

Cite this record

ConductScience Library. juvenile myelomonocytic leukemia. Diseases, record MONDO_0011908. Release diseases-2026-09-18-0.1.0, 18 Sept 2026. https://conductscience.org/library/diseases/MONDO_0011908

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Disease

Names and IDs as Open Targets gives them. Its IDs in MeSH, Orphanet, the Disease Ontology and the NCI Thesaurus are those Open Targets states for it.

Name: juvenile myelomonocytic leukemia; Open Targets
Open Targets ID: MONDO_0011908; Open Targets
Other names: JCML, JMML, chronic myelomonocytic leukemia, juvenile chronic myelogenous leukaemia, juvenile chronic myelogenous leukemia, juvenile chronic myeloid leukaemia, juvenile chronic myeloid leukemia, juvenile chronic myelomonocytic leukaemia, juvenile chronic myelomonocytic leukemia, juvenile myelomonocytic leukemia, autosomal dominant, somatic mutation, leukemia, juvenile myelomonocytic, autosomal dominant, somatic mutation, leukemia, juvenile myelomonocytic, somatic; Open Targets
Disease Ontology: DOID:0050458; Disease Ontology
MeSH: D054429; MeSH
NCI Thesaurus: C9233; NCI Thesaurus
Orphanet: ORPHA:86834; Orphanet
Highest stage of its drugs: Approval, for 1 of its 46 drugs, from TTD; Open Targets

Drugs46

The drugs Open Targets' clinical reports study for the disease, the highest stage first, then the most trial reports. The stage is Open Targets', the highest any of its sources reports for the pair, and each row names the sources that report it.

Drug	ChEMBL ID	Type	Open Targets' stage	Stage reported by	Reports	Of them trials	Trials shown here	Source
GLASDEGIB	CHEMBL2043437	Small molecule	Approval	TTD	2	1	1	Drugs and targets
FILGRASTIM	CHEMBL1201567	Protein	Phase 3	ClinicalTrials.gov	1	1	1	Drugs and targets
CYTARABINE	CHEMBL803	Small molecule	Phase 2	ClinicalTrials.gov	7	7	7	Drugs and targets
AZACITIDINE	CHEMBL1489	Small molecule	Phase 2	ClinicalTrials.gov	6	6	6	Drugs and targets
CYCLOPHOSPHAMIDE	CHEMBL1200796	Small molecule	Phase 2	ClinicalTrials.gov	6	6	6	Drugs and targets
BUSULFAN	CHEMBL820	Small molecule	Phase 2	ClinicalTrials.gov	5	5	5	Drugs and targets
FLUDARABINE	CHEMBL1568	Small molecule	Phase 2	ClinicalTrials.gov	5	5	5	Drugs and targets
DECITABINE	CHEMBL1201129	Small molecule	Phase 2	ClinicalTrials.gov	4	4	4	Drugs and targets
MELPHALAN	CHEMBL852	Small molecule	Phase 2	ClinicalTrials.gov	3	3	3	Drugs and targets
DAUNORUBICIN	CHEMBL178	Small molecule	Phase 2	ClinicalTrials.gov	2	2	2	Drugs and targets

1 to 10 of 46

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Targets83

The targets ChEMBL's mechanism records give for the disease's drugs, through Open Targets, the most drugs first. A mechanism record says what a drug acts on, not that the target causes the disease.

Target	Protein	Its drugs that act on it	Mechanisms	Highest stage of those drugs	Gene in Genes	Source
POLA1	DNA polymerase (alpha/delta/epsilon)	4	DNA polymerase (alpha/delta/epsilon) inhibitor	Phase 2	https://conductscience.org/library/genes/5422	Drugs and targets
POLA2	DNA polymerase (alpha/delta/epsilon)	4	DNA polymerase (alpha/delta/epsilon) inhibitor	Phase 2	https://conductscience.org/library/genes/23649	Drugs and targets
POLD1	DNA polymerase (alpha/delta/epsilon)	4	DNA polymerase (alpha/delta/epsilon) inhibitor	Phase 2	https://conductscience.org/library/genes/5424	Drugs and targets
POLD2	DNA polymerase (alpha/delta/epsilon)	4	DNA polymerase (alpha/delta/epsilon) inhibitor	Phase 2	https://conductscience.org/library/genes/5425	Drugs and targets
POLD3	DNA polymerase (alpha/delta/epsilon)	4	DNA polymerase (alpha/delta/epsilon) inhibitor	Phase 2	https://conductscience.org/library/genes/10714	Drugs and targets
POLD4	DNA polymerase (alpha/delta/epsilon)	4	DNA polymerase (alpha/delta/epsilon) inhibitor	Phase 2	https://conductscience.org/library/genes/57804	Drugs and targets
POLE	DNA polymerase (alpha/delta/epsilon)	4	DNA polymerase (alpha/delta/epsilon) inhibitor	Phase 2	https://conductscience.org/library/genes/5426	Drugs and targets
POLE2	DNA polymerase (alpha/delta/epsilon)	4	DNA polymerase (alpha/delta/epsilon) inhibitor	Phase 2	https://conductscience.org/library/genes/5427	Drugs and targets
POLE3	DNA polymerase (alpha/delta/epsilon)	4	DNA polymerase (alpha/delta/epsilon) inhibitor	Phase 2	https://conductscience.org/library/genes/54107	Drugs and targets
PRIM1	DNA polymerase (alpha/delta/epsilon)	4	DNA polymerase (alpha/delta/epsilon) inhibitor	Phase 2	https://conductscience.org/library/genes/5557	Drugs and targets

1 to 10 of 83

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Trials29

Trials an Open Targets clinical report links to the disease, posted results first, then the largest. A trial is shown when the release's checks confirm its link to the disease and Drugs and targets shows its link to one of the disease's drugs.

Trial	Title	Phase	Participants	Results first posted	Conditions	Drugs	Linked by	Source
NCT01522976	Azacitidine With or Without Lenalidomide or Vorinostat in Treating Patients With Higher-Risk Myelodysplastic Syndromes or Chronic Myelomonocytic Leukemia	Phase 2	282	2016-03-22	Chronic Myelomonocytic Leukemia, Chronic Myelomonocytic Leukemia-1, Chronic Myelomonocytic Leukemia-2, Myelodysplastic Syndrome, Myelodysplastic Syndrome With Excess Blasts, Myelodysplastic Syndrome With Excess Blasts-1, Myelodysplastic Syndrome/Acute Myeloid Leukemia	AZACITIDINE, LENALIDOMIDE, VORINOSTAT	Its record names the disease	ClinicalTrials.gov
NCT02103478	Pharmacokinetic Guided Dose Escalation and Dose Confirmation With Oral Decitabine and Oral Cytidine Deaminase Inhibitor (CDAi) in Patients With Myelodysplastic Syndromes (MDS)	Phase 1/2	130	2020-10-19	Myelodysplastic Syndrome, MDS	CEDAZURIDINE, DECITABINE	Open Targets' reading of its record	ClinicalTrials.gov
NCT04802161	Comparing the Addition of an Anti-Cancer Drug, Pomalidomide, to the Usual Chemotherapy Treatment (Daunorubicin and Cytarabine Liposome) in Newly Diagnosed Acute Myeloid Leukemia With Myelodysplastic Syndrome-Related Changes	Phase 2	49	2025-12-19	Acute Myeloid Leukemia, Acute Myeloid Leukemia Post Cytotoxic Therapy, Acute Myeloid Leukemia With Multilineage Dysplasia, Chronic Myelomonocytic Leukemia, Myelodysplastic Syndrome, Myeloproliferative Neoplasm	CYTARABINE, DAUNORUBICIN, POMALIDOMIDE	Its record names the disease	ClinicalTrials.gov
NCT00953758	A Study Of PF-04449913 In Select Hematologic Malignancies	Phase 1	47	2024-04-25	Hematologic Malignancies	GLASDEGIB	Open Targets' reading of its record	ClinicalTrials.gov
NCT01824693	Busulfan, Cyclophosphamide, and Melphalan or Busulfan and Fludarabine Phosphate Before Donor Hematopoietic Cell Transplant in Treating Younger Patients With Juvenile Myelomonocytic Leukemia	Phase 2	30	2018-12-05	Juvenile Myelomonocytic Leukemia	BUSULFAN, CYCLOPHOSPHAMIDE, FLUDARABINE PHOSPHATE, MELPHALAN	Its record names the disease	ClinicalTrials.gov
NCT00450450	Donor Bone Marrow Transplant With or Without G-CSF in Treating Young Patients With Hematologic Cancer or Other Diseases	Phase 3	27	2017-05-09	Childhood Acute Lymphoblastic Leukemia in Remission, Childhood Acute Myeloid Leukemia in Remission, Childhood Chronic Myelogenous Leukemia, Childhood Myelodysplastic Syndromes, Chronic Phase Chronic Myelogenous Leukemia, de Novo Myelodysplastic Syndromes, Juvenile Myelomonocytic Leukemia, Previously Treated Myelodysplastic Syndromes, Recurrent Childhood Acute Lymphoblastic Leukemia, Secondary Myelodysplastic Syndromes	FILGRASTIM	Its record names the disease	ClinicalTrials.gov
NCT00167219	Stem Cell Transplant for Juvenile Myelomonocytic Leukemia (JMML)	Phase 1/2	15	2023-11-07	Juvenile Myelomonocytic Leukemia	BUSULFAN, CYCLOPHOSPHAMIDE, MELPHALAN	Its record names the disease	ClinicalTrials.gov
NCT03190915	Trametinib in Treating Patients With Relapsed or Refractory Juvenile Myelomonocytic Leukemia	Phase 2	10	2024-07-16	Juvenile Myelomonocytic Leukemia, Neurofibromatosis Type 1	TRAMETINIB	Its record names the disease	ClinicalTrials.gov
NCT00509600	Etanercept (Enbrel) for Juvenile Myelomonocytic Leukemia	Phase 2	1	2010-12-02	Leukemia	ETANERCEPT	Its record names the disease	ClinicalTrials.gov
NCT00609739	Cytosine Arabinoside and Mitoxantrone for Patients With Juvenile Myelomonocytic Leukemia Receiving Repeat Stem Cell Transplantation	Phase 1/2	1	2012-03-02	Leukemia	CYTARABINE, MITOXANTRONE	Its record names the disease	ClinicalTrials.gov

1 to 10 of 29

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Comparisons in its trials3

Comparisons Trial comparisons calculated in the trials shown here, most participants first. A comparison in one of these trials does not always compare a treatment for the disease, so read the two groups.

Comparison	Group 1, with the outcome	Group 2, with the outcome	Participants	Trial	Source
Phase 2 Dose Confirmation vs Phase 2 Fixed-Dose Combination: Number of Participants With Overall Response in Phase 2	29 of 50	19 of 30	80	NCT02103478	Trial comparisons
Experimental G-BM Arm vs Control BM Arm: serious adverse events	0 of 13	1 of 13	26	NCT00450450	Trial comparisons
Arm I (Busulfan, Cyclophosphamide, Melphalan) vs Arm II (Busulfan, Fludarabine Phosphate): Number of Participants Who Experience Treatment-Related Mortality (TRM) by Day 100	0 of 6	1 of 9	15	NCT01824693	Trial comparisons

Papers from its trials18

The papers the trials shown here list on ClinicalTrials.gov as their results (RESULT) or as drawn from them (DERIVED), results first, newest first.

PMID	Listed as	Trials listing it	Source
29528181	RESULT	1	PubMed
29396905	RESULT	1	PubMed
29129488	RESULT	1	PubMed
25933833	RESULT	1	PubMed
25564399	RESULT	1	PubMed
39352751	DERIVED	1	PubMed
38867349	DERIVED	1	PubMed
37944074	DERIVED	1	PubMed
37245522	DERIVED	1	PubMed
35349491	DERIVED	1	PubMed

1 to 10 of 18

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Data dates

The date of each source this release read.

Open Targets: 2026-09-18; Open Targets
Drug links and their checks, from Drugs and targets: 2026-09-18; Drugs and targets
ClinicalTrials.gov, from Trials: 2026-11-12; Trials
Comparisons, from Trial comparisons: 2026-03-23; Trial comparisons
PubMed, for the checks: 2026-11-06; PubMed
MeSH, for the checks: 2026-11-10; MeSH

Sources 6

ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
ChEMBL, through Open TargetsCC BY-SA 3.0
ClinicalTrials.govNo license stated
NLM's MeSHNLM's terms
Open TargetsCC0 1.0
PubMedNLM's terms

Each source's terms

ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
ChEMBL, through Open TargetsCC BY-SA 3.0, for the parts from ChEMBL. Read on 8 Nov 2026 at https://platform-docs.opentargets.org/licence
ClinicalTrials.govnone stated
NLM's MeSHNLM's
Open TargetsCC0 1.0. Open Targets asks that users cite its latest paper: Buniello et al. 2025, Open Targets Platform: facilitating therapeutic hypotheses building in drug discovery, Nucleic Acids Research 53(D1):D1467, PMID 39657122. Read on 8 Nov 2026 at https://platform-docs.opentargets.org/licence
PubMedNLM's terms

ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.

For software

Read this disease from software.

The record and each of its lists are JSON, twenty rows a page. No key.

GET https://conductscience.org/api/diseases/v1/MONDO_0011908

GET https://conductscience.org/api/diseases/v1/MONDO_0011908/drugs?page=2

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