Disease · MONDO_0018881

myelodysplastic syndrome

6 sourcesdiseases-2026-09-18-0.1.0

Cite this record

ConductScience Library. myelodysplastic syndrome. Diseases, record MONDO_0018881. Release diseases-2026-09-18-0.1.0, 18 Sept 2026. https://conductscience.org/library/diseases/MONDO_0018881

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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: myelodysplastic syndrome; Open Targets
Open Targets ID: MONDO_0018881; Open Targets
Other names: MDS, MDS, unclassifiable, MDS-U, Myelodysplastic Syndromes, dysmyelopoietic syndrome, hematopoeitic - myelodysplastic syndrome (MDS), myelodysplasia, myelodysplastic neoplasm, myelodysplastic syndrome (MDS)-like, myelodysplastic syndrome, somatic, myelodysplastic syndrome, unclassifiable, myelodysplastic syndrome/neoplasm, oligoblastic leukaemia, oligoblastic leukemia, preleukemia, smoldering leukemia, smouldering leukaemia; Open Targets
Disease Ontology: DOID:0050908; Disease Ontology
NCI Thesaurus: C3247; NCI Thesaurus
Orphanet: ORPHA:52688; Orphanet
Highest stage of its drugs: Approval, for 10 of its 421 drugs, from ClinicalTrials.gov, DailyMed, EMA Human Drugs, FDA, PMDA and TTD; Open Targets

Drugs421

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
AZACITIDINE	CHEMBL1489	Small molecule	Approval	DailyMed, EMA Human Drugs, PMDA and TTD	299	285	268	Drugs and targets
DECITABINE	CHEMBL1201129	Small molecule	Approval	DailyMed and TTD	162	152	148	Drugs and targets
LENALIDOMIDE	CHEMBL848	Small molecule	Approval	DailyMed and EMA Human Drugs	64	59	59	Drugs and targets
FILGRASTIM	CHEMBL1201567	Protein	Approval	PMDA	52	49	33	Drugs and targets
CLOFARABINE	CHEMBL1750	Small molecule	Approval	TTD	42	41	41	Drugs and targets
LUSPATERCEPT	CHEMBL3039545	Protein	Approval	DailyMed, EMA Human Drugs and PMDA	31	26	26	Drugs and targets
CEDAZURIDINE	CHEMBL3237547	Small molecule	Approval	FDA	27	24	20	Drugs and targets
DEFERASIROX	CHEMBL550348	Small molecule	Approval	DailyMed	23	21	21	Drugs and targets
EPOETIN ALFA	CHEMBL1201565	Protein	Approval	EMA Human Drugs	19	16	13	Drugs and targets
IMETELSTAT	CHEMBL2107856		Approval	ClinicalTrials.gov and EMA Human Drugs	5	3	3	Drugs and targets

1 to 10 of 421

See all 421 drugs

Targets354

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
PDGFRB	Platelet-derived growth factor receptor	12	Platelet-derived growth factor receptor beta inhibitor; Platelet-derived growth factor receptor inhibitor	Phase 3	https://conductscience.org/library/genes/5159	Drugs and targets
TOP2A	DNA topoisomerase 2-alpha	12	DNA topoisomerase II alpha inhibitor; DNA topoisomerase II inhibitor	Phase 3	https://conductscience.org/library/genes/7153	Drugs and targets
FLT3	Receptor-type tyrosine-protein kinase FLT3	11	Tyrosine-protein kinase receptor FLT3 inhibitor	Phase 3	https://conductscience.org/library/genes/2322	Drugs and targets
KIT	Mast/stem cell growth factor receptor Kit	11	Stem cell growth factor receptor inhibitor	Phase 3	https://conductscience.org/library/genes/3815	Drugs and targets
ABL1	Bcr/Abl fusion protein	8	Bcr/Abl fusion protein inhibitor; Tyrosine-protein kinase ABL inhibitor	Phase 2	https://conductscience.org/library/genes/25	Drugs and targets
KDR	Vascular endothelial growth factor receptor	8	Vascular endothelial growth factor receptor 2 inhibitor; Vascular endothelial growth factor receptor inhibitor	Phase 3	https://conductscience.org/library/genes/3791	Drugs and targets
FLT1	Vascular endothelial growth factor receptor	7	Vascular endothelial growth factor receptor inhibitor	Phase 2/3	https://conductscience.org/library/genes/2321	Drugs and targets
FLT4	Vascular endothelial growth factor receptor	7	Vascular endothelial growth factor receptor inhibitor	Phase 2/3	https://conductscience.org/library/genes/2324	Drugs and targets
HDAC1	Histone deacetylase	7	Histone deacetylase 1 inhibitor; Histone deacetylase inhibitor	Phase 3	https://conductscience.org/library/genes/3065	Drugs and targets
HDAC2	Histone deacetylase	7	Histone deacetylase 2 inhibitor; Histone deacetylase inhibitor	Phase 3	https://conductscience.org/library/genes/3066	Drugs and targets

1 to 10 of 354

See all 354 targets

Trials1,221

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. 32 more are not shown, since the checks do not confirm them.

Trial	Title	Phase	Participants	Results first posted	Conditions	Drugs	Linked by	Source
NCT04313881	Magrolimab + Azacitidine Versus Azacitidine + Placebo in Untreated Participants With Myelodysplastic Syndrome (MDS)	Phase 3	539	2024-03-21	Myelodysplastic Syndromes	AZACITIDINE, MAGROLIMAB	Its record names the disease	ClinicalTrials.gov
NCT04266301	Study of Efficacy and Safety of MBG453 in Combination With Azacitidine in Subjects With Intermediate, High or Very High Risk Myelodysplastic Syndrome (MDS) as Per IPSS-R, or Chronic Myelomonocytic Leukemia-2 (CMML-2)	Phase 3	530	2025-12-12	Myelodysplastic Syndromes, Leukemia, Myelomonocytic, Chronic	AZACITIDINE, SABATOLIMAB	Its record names the disease	ClinicalTrials.gov
NCT01757535	Efficacy of Oral Azacitidine Plus Best Supportive Care as Maintenance Therapy in Subjects With Acute Myeloid Leukemia (AML) in Complete Remission	Phase 3	472	2020-11-06	Leukemia, Myeloid, Acute	AZACITIDINE	Its record names the disease	ClinicalTrials.gov
NCT03268954	Pevonedistat Plus Azacitidine Versus Single-Agent Azacitidine as First-Line Treatment for Participants With Higher-Risk Myelodysplastic Syndromes (HR MDS), Chronic Myelomonocytic Leukemia (CMML), or Low-Blast Acute Myelogenous Leukemia (AML)	Phase 3	454	2022-09-22	Myelodysplastic Syndrome, Leukemia, Myelomonocytic, Chronic, Leukemia, Myeloid, Acute	AZACITIDINE, PEVONEDISTAT	Its record names the disease	ClinicalTrials.gov
NCT00074490	Donor Stem Cell Transplant With No or Low-Intensity Chemotherapy Using Sirolimus and Treated Immune Cells to Treat Blood and Lymph Cancers	Phase 2	442	2018-12-31	Lymphoma, Leukemia, Myeloproliferative Disorders, Multiple Myleoma, Myelodysplastic Syndrome	SIROLIMUS	Its record names the disease	ClinicalTrials.gov
NCT02907359	Guadecitabine (SGI-110) vs Treatment Choice in Adults With MDS or CMML Previously Treated With HMAs	Phase 3	417	2023-05-18	Myelodysplastic Syndromes, Leukemia, Myelomonocytic, Chronic	GUADECITABINE	Its record names the disease	ClinicalTrials.gov
NCT01261312	SGI-110 in Participants With Myelodysplastic Syndromes (MDS) or Acute Myelogenous Leukemia (AML)	Phase 1/2	414	2025-01-23	MDS, CMML, AML	GUADECITABINE	Its record names the disease	ClinicalTrials.gov
NCT03682536	A Study to Compare the Efficacy and Safety of Luspatercept (ACE-536) Versus Epoetin Alfa for the Treatment of Anemia Due to IPSS-R Very Low, Low, or Intermediate Risk Myelodysplastic Syndromes (MDS) Participants Who Require Red Blood Cell Transfusions and Are ESA Naïve	Phase 3	363	2024-06-26	Myelodysplastic Syndromes	EPOETIN ALFA, LUSPATERCEPT	Its record names the disease	ClinicalTrials.gov
NCT00071799	A Survival Study in Patients With High Risk Myelodysplastic Syndromes Comparing Azacitidine Versus Conventional Care	Phase 3	358	2010-07-05	Myelodysplastic Syndromes	AZACITIDINE	Its record names the disease	ClinicalTrials.gov
NCT02719574	Open-label Study of FT-2102 With or Without Azacitidine or Cytarabine in Patients With AML or MDS With an IDH1 Mutation	Phase 1/2	336	2025-06-26	Acute Myeloid Leukemia, Acute Myelogenous Leukemia, Myelodysplastic Syndrome	AZACITIDINE, CYTARABINE, OLUTASIDENIB	Its record names the disease	ClinicalTrials.gov

1 to 10 of 1,221

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Comparisons in its trials244

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
Magrolimab + Azacitidine vs Placebo + Azacitidine: deaths from any cause	145 of 268	132 of 271	539	NCT04313881	Trial comparisons
Sabatolimab (MBG453) + Azacitidine vs Placebo + Azacitidine: deaths from any cause	161 of 265	160 of 265	530	NCT04266301	Trial comparisons
Sabatolimab (MBG453) + Azacitidine vs Placebo + Azacitidine: serious adverse events	174 of 263	164 of 265	528	NCT04266301	Trial comparisons
Magrolimab + Azacitidine vs Placebo + Azacitidine: serious adverse events	189 of 263	136 of 264	527	NCT04313881	Trial comparisons
Oral Azacitidine Plus Best Supportive Care vs Placebo Plus Best Supportive Care: deaths from any cause	172 of 238	176 of 234	472	NCT01757535	Trial comparisons
Oral Azacitidine Plus Best Supportive Care vs Placebo Plus Best Supportive Care: serious adverse events	110 of 236	109 of 233	469	NCT01757535	Trial comparisons
Azacitidine 75 mg/m^2 vs Pevonedistat 20 mg/m^2 + Azacitidine 75 mg/m^2: Number of Participants With at Least 1 Inpatient Hospital Admissions Related to HR MDS, CMML or Low-blast AML	3 of 227	9 of 227	454	NCT03268954	Trial comparisons
Azacitidine 75 mg/m^2 vs Pevonedistat 20 mg/m^2 + Azacitidine 75 mg/m^2: Sixty-Day Mortality Reported as Number of Participants Who Died Up to Day 60	15 of 220	14 of 223	443	NCT03268954	Trial comparisons
Azacitidine 75 mg/m^2 vs Pevonedistat 20 mg/m^2 + Azacitidine 75 mg/m^2: Thirty-Day Mortality Reported as Number of Participants Who Died Up to Day 30	6 of 220	5 of 223	443	NCT03268954	Trial comparisons
Azacitidine 75 mg/m^2 vs Pevonedistat 20 mg/m^2 + Azacitidine 75 mg/m^2: deaths from any cause	153 of 220	150 of 223	443	NCT03268954	Trial comparisons

1 to 10 of 244

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Papers from its trials544

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
41385341	RESULT	1	PubMed
40238563	RESULT	1	PubMed
39919267	RESULT	1	PubMed
39844769	RESULT	1	PubMed
36961350	RESULT	1	PubMed
35612815	RESULT	1	PubMed
35154579	RESULT	1	PubMed
35071585	RESULT	1	PubMed
35061876	RESULT	1	PubMed
34804017	RESULT	1	PubMed

1 to 10 of 544

See all 544 papers from its trials

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_0018881

GET https://conductscience.org/api/diseases/v1/MONDO_0018881/drugs?page=2

API reference For AI assistants