Summary
Cytarabine 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lists 8 registered trials linking Cytarabine to Myelodysplastic Syndromes (MDS): 6 have completed; 2 are terminated, withdrawn, suspended or of unknown status. The most advanced is Phase 3 (NCT00046930). It plans or enrolled 449 participants. Registration activity spans 1994 to 2013. The literature layer holds 11 publications for this pair: 4 meta-analyses, 2 systematic reviews and 5 randomised controlled trial publications. Publication years run from 2011 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 | 73.0 (trials 23.0, literature 30.0, tier 15, approved bonus 5.0) |
| Registered trials | 8 total: 0 recruiting, 0 active / not yet recruiting, 6 completed, 2 other |
| Linked publications | 11 (5 randomised controlled trial publications, 4 meta-analyses, 2 systematic reviews) |
| 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trial scores by status (recruiting / active / enrolling 3, completed 2.5, not yet recruiting 2, unknown 1, terminated / withdrawn / suspended 0.5) plus a phase bonus (phase 3-4 +2, phase 2 +1, phase 1 +0.5).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 |
|---|---|---|---|---|
| NCT00046930 | Daunorubicin & Cytarabine +/- Zosuquidar inTreating Older Patients With Newly Diagnosed Acute Myeloid Leukemia or Refractory Anemia 2002 | completed | Phase 3 | 449 |
| NCT00015951 | Bevacizumab, Cytarabine, and Mitoxantrone on Treating Patients With Hematologic Cancers 2001 | completed | Phase 2 | — |
| NCT00002833 | Peripheral Stem Cell Transplantation Plus Filgrastim in Treating Patients With Acute or Chronic Myelogenous Leukemia 1994 | completed | Phase 2 | 53 |
| NCT00368355 | T Cell Depletion for Recipients of HLA Haploidentical Related Donor Stem Cell Grafts 2000 | completed | Phase 2 | 46 |
| NCT00740181 | Decitabine, Cytarabine, GCSF for Refractory AML/MDS 2008 | terminated | Phase 2 | 9 |
| NCT01302106 | Study of Clofarabine in Combination With Low Dose Cytarabine to Treat Myelodysplastic Syndromes | withdrawn | Phase 2 | — |
| NCT01297543 | Safety Study of Human Myeloid Progenitor Cells (CLT-008) After Chemotherapy for Leukemia 2011 | completed | PHASE1, PHASE2 | 45 |
| NCT01831232 | Idarubicin, Cytarabine, and Pravastatin Sodium in Treating Patients With Acute Myeloid Leukemia or Myelodysplastic Syndromes 2013 | completed | Not applicable | 24 |
Published literature
- Randomized controlled trial Patient-reported outcomes in newly diagnosed patients with FLT3-internal-tandem-duplication-positive acute myeloid leukaemia receiving standard chemotherapy plus quizartinib or placebo (QuANTUM-First): a global, randomised, placebo-controlled, phase 3 trialOlíva EN, Cottone F, Unni S et al. · The Lancet. Haematology · 2026 · PMID 41791832
- Randomized controlled trial Addition of the nuclear export inhibitor selinexor to standard intensive treatment for elderly patients with acute myeloid leukemia and high risk myelodysplastic syndromeJanssen JJWM, Löwenberg B, Manz M et al. · Leukemia · 2022 · PMID 35869267
- Systematic review TKIs combined with chemotherapy followed by allo-HSCT in Philadelphia chromosome-positive myelodysplastic syndrome: A case report and literature reviewQi S, Wang F, Liu Y et al. · Medicine · 2022 · PMID 36401464
- Randomized controlled trial Subsequent malignant neoplasms among children with Hodgkin lymphoma: a report from the Children's Oncology GroupGiulino-Roth L, Pei Q, Buxton A et al. · Blood · 2021 · PMID 33512412
- Meta-analysis [Efficacy and Safety of Decitabine Combined with CAG (Cytarabine, Aclarubicin, G-CSF) for Patients with Intermediate or High Risk Myelodysplastic Syndrome and Acute Myeloid Leukemia: a Meta-Analysis]Zhang JL, Cao YP, Li JG · Zhongguo shi yan xue ye xue za zhi · 2019 · PMID 30998160
- Randomized controlled trial [Clinical Efficacy of Low-Dose Decitabine alone for Treatment of Myelodysplastic Syndrome]Shi R, Guo SQ, Chen YY et al. · Zhongguo shi yan xue ye xue za zhi · 2019 · PMID 31607313
- Randomized controlled trial Glasdegib plus intensive/nonintensive chemotherapy in untreated acute myeloid leukemia: BRIGHT AML 1019 Phase III trialsCortes JE, Dombret H, Merchant A et al. · Future oncology (London, England) · 2019 · PMID 31516032
- Systematic review Myeloid neoplasms with t(12;22)(p13;q12)/MN1-EVT6: a systematic review of 12 casesShao H, Cen J, Chen S et al. · Annals of hematology · 2018 · PMID 29273914
- Meta-analysis HAG (Homoharringtonine, Cytarabine, G-CSF) Regimen for the Treatment of Acute Myeloid Leukemia and Myelodysplastic Syndrome: A Meta-Analysis with 2,314 ParticipantsXie M, Jiang Q, Li L et al. · PloS one · 2016 · PMID 27706258
- Meta-analysis Outcomes after (90) Yttrium-ibritumomab tiuxetan-BEAM in diffuse large B-cell lymphoma: a meta-analysisAuger-Quittet S, Duny Y, Daures JP et al. · Cancer medicine · 2014 · PMID 24740968
- Meta-analysis A meta-analysis of CAG (cytarabine, aclarubicin, G-CSF) regimen for the treatment of 1029 patients with acute myeloid leukemia and myelodysplastic syndromeWei G, Ni W, Chiao JW et al. · Journal of hematology & oncology · 2011 · PMID 22082134
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 Cytarabine approved for Myelodysplastic Syndromes (MDS)?
Cytarabine 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 Cytarabine in clinical trials for Myelodysplastic Syndromes (MDS)?
ClinicalTrials.gov lists 8 registered trials linking Cytarabine to Myelodysplastic Syndromes (MDS): 6 have completed; 2 are terminated, withdrawn, suspended or of unknown status. The most advanced is Phase 3 (NCT00046930). It plans or enrolled 449 participants. Registration activity spans 1994 to 2013.
What does the evidence show for Cytarabine in Myelodysplastic Syndromes (MDS)?
Cytarabine 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.