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A myelodysplastic syndrome defined by 10-19% blasts in the bone marrow or 5-19% blasts in the blood and <10% blasts in the bone marrow. Approximately 33% of cases progress to acute leukemia. The prognosis is usually poor. (WHO)
Estimated prevalence: Unknown (Unknown prevalence).
5 clinical trials registered, 1 recruiting. Interventions under study include drug therapy and other interventions. Pipeline includes 2 PHASE3, 3 PHASE1. Research is sponsored by a mix of industry and academic institutions.
NCT ID | Title | Phase | Sponsor | Status |
|---|---|---|---|---|
[NCT03850574](https://clinicaltrials.gov/study/NCT03850574) |
Data assembled from 4 of 12 sources · Last updated Sep 19, 2026, 12:30 AM UTC
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European rare disease database
Genetic and Rare Diseases Info Center
Clinical Trial to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Tuspetinib (HM43239) in Patients With Relapsed or Refractory Acute Myeloid Leukemia
PHASE1 |
Aptose Biosciences Inc. |
RECRUITING |
[NCT04027309](https://clinicaltrials.gov/study/NCT04027309) | A Study of Gilteritinib Versus Midostaurin in Combination With Induction and Consolidation Therapy Followed by One-year Maintenance in Patients With Newly Diagnosed Acute Myeloid Leukemia or Myelodysplastic Syndromes With Excess Blasts-2 With FLT3 Mutations Eligible for Intensive Chemotherapy | PHASE3 | Stichting Hemato-Oncologie voor Volwassenen Nederland | ACTIVE_NOT_RECRUITING |
[NCT03839771](https://clinicaltrials.gov/study/NCT03839771) | A Study of Ivosidenib or Enasidenib in Combination With Induction Therapy and Consolidation Therapy, Followed by Maintenance Therapy in Patients With Newly Diagnosed Acute Myeloid Leukemia or Myedysplastic Syndrome EB2, With an IDH1 or IDH2 Mutation, Respectively, Eligible for Intensive Chemotherapy | PHASE3 | Stichting Hemato-Oncologie voor Volwassenen Nederland | UNKNOWN |
[NCT05703542](https://clinicaltrials.gov/study/NCT05703542) | BXCL701 Phase 1 R/R Acute Myeloid Leukemia or Myelodysplastic Syndrome | PHASE1 | Eric Stephen Winer, MD | UNKNOWN |
[NCT02530034](https://clinicaltrials.gov/study/NCT02530034) | Hu8F4 in Treating Patients With Advanced Hematologic Malignancies | PHASE1 | M.D. Anderson Cancer Center | ACTIVE_NOT_RECRUITING |
5 publications have been identified in PubMed for myelodysplastic syndrome with excess blasts-2. Research spans Case Report / Case Series (40%), Epidemiology / Natural History (40%), and Clinical Trial Publication (20%).
Sicre de Fontbrune F (2026). [PMID: 39159950](https://pubmed.ncbi.nlm.nih.gov/39159950/). *British journal of haematology*. [Clinical Trial Publication]
Conchinha AF (2025). [PMID: 41523364](https://pubmed.ncbi.nlm.nih.gov/41523364/). *Cureus*. [Case Report / Case Series]
Shah MV (2025). [PMID: 40085954](https://pubmed.ncbi.nlm.nih.gov/40085954/). *Blood Adv*. [Epidemiology / Natural History]
Guo W (2024). [PMID: 38969929](https://pubmed.ncbi.nlm.nih.gov/38969929/). *Annals of hematology*. [Epidemiology / Natural History]
Gürbüz AF (2024). [PMID: 38995367](https://pubmed.ncbi.nlm.nih.gov/38995367/). *Strahlentherapie und Onkologie : Organ der Deutschen Rontgengesellschaft ... [et al]*. [Case Report / Case Series]