Summary
Azacitidine has reached the approved stage (maximum clinical phase 4) for at least one indication, so its use in Myelodysplastic Syndromes (MDS) would be drug repurposing rather than first-in-human development. This does not mean it is approved for Myelodysplastic Syndromes (MDS). ClinicalTrials.gov lists 8 registered trials linking Azacitidine to Myelodysplastic Syndromes (MDS): 1 is active or not yet recruiting; 3 have completed; 4 are terminated, withdrawn, suspended or of unknown status. The most advanced is Phase 2 (NCT03151304). The largest enrolment is 1500 participants (NCT01595295). Registration activity spans 2009 to 2022. The literature layer holds 13 publications for this pair: 5 Cochrane reviews, 3 meta-analyses and 5 clinical trial publications. Publication years run from 2018 to 2026. Because at least one synthesis-level source exists (systematic review, meta-analysis or Cochrane review), this pair has been assessed beyond single studies, although the synthesis may concern a different indication.
The RepurpOS disease-intelligence file for Myelodysplastic Syndrome ranks 50 candidate therapeutics from Open Targets, ChEMBL, DGIdb and PubMed; only a minority carry direct clinical evidence, and this page covers one of those. Spontaneous remission context recorded for the condition: Essentially zero; progressive cytopaenia and transformation to AML in 30-40% of higher-risk MDS over 2-5 years; median survival low-risk MDS ~5 years, high-risk ~1 year without treatment.
Evidence table
| Evidence tier | A · Strong |
|---|---|
| Evidence score | 67.5 (trials 17.5, literature 30.0, tier 15, approved bonus 5.0) |
| Registered trials | 8 total: 0 recruiting, 1 active / not yet recruiting, 3 completed, 4 other |
| Linked publications | 13 (5 Cochrane reviews, 5 clinical trial publications, 3 meta-analyses) |
| Agent type | Drug (Small molecule) |
| Development stage (any indication) | Approved |
| Mechanism / class | Not recorded |
| Data sources | DGIdb |
| Linked via biomarker / target | CDA |
How the evidence score is calculated
- Each registered trial scores by status (recruiting / active / enrolling 3, completed 2.5, not yet recruiting 2, unknown 1, terminated / withdrawn / suspended 0.5) plus a phase bonus (phase 3-4 +2, phase 2 +1, phase 1 +0.5). The trial component is capped at 30.
- Each literature item scores by design (Cochrane review 5, meta-analysis 4, systematic review 3, RCT 3, clinical trial publication 2, curated reference 1.5, other PubMed record 1). The literature component is capped at 30.
- The existing evidence tier adds 15 (A / Strong), 10 (B / Moderate), 5 (C / Preliminary) or 0 (D / Anecdotal).
- Agents approved for any indication (max clinical phase 4) add 5, because an approved agent has an established safety profile that lowers the barrier to repurposing trials.
- The score ranks what has been studied, not what works. It does not read effect sizes or directions of effect.
Registered clinical trials
| NCT ID | Title | Status | Phase | Enrolment |
|---|---|---|---|---|
| NCT01893372 | Eltrombopag With or Without Hypomethylating Agent After Hypomethylating Agent Failure For Patients With Myelodysplastic Syndrome (MDS) 2013 | completed | Phase 2 | 29 |
| NCT03151304 | A Safety and Efficacy Study of Pracinostat and Azacitidine in Patients With High Risk Myelodysplastic Syndromes 2017 | terminated | Phase 2 | 64 |
| NCT03486353 | A Study of FF-10501-01 in Combination with Azacitidine in Patients with Myelodysplastic Syndrome 2019 | withdrawn | Phase 2 | — |
| NCT03613532 | Venetoclax Added to Fludarabine + Busulfan Prior to Transplant and to Maintenance Therapy for AML, MDS, and MDS/MPN 2018 | active, not recruiting | Phase 1 | 102 |
| NCT05829226 | A Phase 1 Study With LYT-200 in Patients With Relapsed/Refractory Acute Myeloid Leukemia (AML), or With Relapsed/Refractory, High-risk Myelodysplastic Syndrome (MDS) 2022 | completed | Phase 1 | 101 |
| NCT04638309 | APR-548 in Combination With Azacitidine for the Treatment of TP53 Myelodysplastic Syndromes (MDS) 2021 | terminated | Phase 1 | 4 |
| NCT01260714 | Azacitidine, Mitoxantrone Hydrochloride, and Etoposide in Treating Older Patients With Poor-Prognosis Acute Myeloid Leukemia 2010 | terminated | Phase 1 | 13 |
| NCT01595295 | Registry on Hypomethylating Agents in Myeloid Neoplasms 2009 | completed | Not applicable | 1,500 |
Published literature
- Clinical trial publication Selinexor in combination with azacitidine or ruxolitinib in myelodysplastic/myeloproliferative neoplasm overlap syndromes: A multicenter prospective studyLin X, Liu Z, Yang C et al. · Cancer · 2026 · PMID 42318939
- Clinical trial publication Checkpoint immunotherapy is associated with preferential activation of tumor antigen-specific CD4(+) T cells in MDSGriffiths EA, Srivastava P, Gomez EC et al. · Blood neoplasia · 2025 · PMID 40809194
- Clinical trial publication Olutasidenib alone or combined with azacitidine in patients with mutant IDH1 myelodysplastic syndromeCortes JE, Yang J, Roboz GJ et al. · Blood advances · 2025 · PMID 40668616
- Clinical trial publication Bexmarilimab plus azacitidine for high-risk myelodysplastic syndrome and relapsed or refractory acute myeloid leukaemia: results from the dose-escalation part of a multicentre, single-arm, phase 1/2 trialKontro M, Stein AS, Pyörälä M et al. · The Lancet. Haematology · 2025 · PMID 40449509
- Clinical trial publication Pivotal results of SELECT-MDS-1 phase 3 study of tamibarotene with azacitidine in newly diagnosed higher-risk MDSDeZern AE, Thepot S, de Botton S et al. · Blood advances · 2025 · PMID 40334070
- Meta-analysis Emerging treatment approaches for VEXAS syndrome: a systematic review and meta-analysisKilic B, Sacin E, Tanin MK et al. · Annals of hematology · 2025 · PMID 40287866
- Cochrane review The efficacy and safety of venetoclax and azacytidine combination treatment in patients with acute myeloid leukemia and myelodysplastic syndrome: systematic review and meta-analysisDu Y, Li C, Yan J · Hematology (Amsterdam, Netherlands) · 2023 · PMID 37036307
- Meta-analysis Efficacy of epigenetic agents for older patients with acute myeloid leukemia and myelodysplastic syndrome in randomized controlled trials: a systematic review and network meta-analysisOh S, Kim E · Clinical and experimental medicine · 2023 · PMID 36964818
- Meta-analysis Azacitidine Monotherapy in Patients With Treatment-Naïve Higher-risk Myelodysplastic Syndrome: A Systematic Literature Review and Meta-analysisHasegawa K, Wei AH, Garcia-Manero G et al. · Clinical lymphoma, myeloma & leukemia · 2023 · PMID 36428152
- Cochrane review Maintenance With Hypomethylating Agents After Allogeneic Stem Cell Transplantation in Acute Myeloid Leukemia and Myelodysplastic Syndrome: A Systematic Review and Meta-AnalysisKungwankiattichai S, Ponvilawan B, Roy C et al. · Frontiers in medicine · 2022 · PMID 35242779
- Cochrane review Comparison Between Decitabine and Azacitidine for Patients With Acute Myeloid Leukemia and Higher-Risk Myelodysplastic Syndrome: A Systematic Review and Network Meta-AnalysisMa J, Ge Z · Frontiers in pharmacology · 2021 · PMID 34483903
- Cochrane review The efficacy and adverse events of venetoclax in combination with hypomethylating agents treatment for patients with acute myeloid leukemia and myelodysplastic syndrome: a systematic review and meta-analysisLiu B, Guo Y, Deng L et al. · Hematology (Amsterdam, Netherlands) · 2020 · PMID 33191860
- Cochrane review A systematic review and network meta-analysis comparing azacitidine and decitabine for the treatment of myelodysplastic syndromeAlmasri J, Alkhateeb HB, Firwana B et al. · Systematic reviews · 2018 · PMID 30227896
Mechanism and notes
No mechanism of action is recorded for this pair in the source databases.
OSMF's existing evidence tier for this pair is A (Strong), derived from the strength of the disease association recorded in DGIdb, in this case via the biomarker or target CDA. The tier describes how well the drug-disease link is documented, not how well the drug works.
Frequently asked questions
Is Azacitidine approved for Myelodysplastic Syndromes (MDS)?
Azacitidine has reached the approved stage (maximum clinical phase 4) for at least one indication, so its use in Myelodysplastic Syndromes (MDS) would be drug repurposing rather than first-in-human development. This does not mean it is approved for Myelodysplastic Syndromes (MDS). Approval status for the specific indication should always be confirmed with the relevant regulator and prescribing information.
Is Azacitidine in clinical trials for Myelodysplastic Syndromes (MDS)?
ClinicalTrials.gov lists 8 registered trials linking Azacitidine to Myelodysplastic Syndromes (MDS): 1 is active or not yet recruiting; 3 have completed; 4 are terminated, withdrawn, suspended or of unknown status. The most advanced is Phase 2 (NCT03151304). The largest enrolment is 1500 participants (NCT01595295). Registration activity spans 2009 to 2022.
What does the evidence show for Azacitidine in Myelodysplastic Syndromes (MDS)?
Azacitidine has both completed registered trials and synthesis-level publications linked to Myelodysplastic Syndromes (MDS). That is the strongest profile in this database, but the summaries here do not extract effect sizes, so read the linked reviews for direction and magnitude of benefit. OSMF's existing evidence tier for this pair is A (Strong), derived from the strength of the disease association recorded in DGIdb, in this case via the biomarker or target CDA. The tier describes how well the drug-disease link is documented, not how well the drug works.
Cite this page
Data: ClinicalTrials.gov, PubMed, Open Targets, ChEMBL, DGIdb and the OSMF therapeutic agent database. Last updated 2026-07-06. Page built 2026-10-07.