Efficacy of Immediate Allogeneic Hematopoietic Stem Cell Transplantation Versus Bridging Therapy Followed by Transplantation in Higher-Risk Myelodysplastic Syndrome Patients
Efficacy of Immediate Allogeneic Hematopoietic Stem Cell Transplantation Versus Bridging Therapy Followed by Transplantation in Higher-Risk Myelodysplastic Syndrome Patients: A Multicenter, Randomized Controlled, Open-Label, Phase 2 Clinical Trial
Institute of Hematology & Blood Diseases Hospital, China
236 participants
Jul 24, 2025
INTERVENTIONAL
Conditions
Summary
This study aims to evaluate whether immediate allogeneic hematopoietic stem cell transplantation (HSCT) is non-inferior to HSCT following bridging therapy in patients with higher-risk myelodysplastic syndrome (HR-MDS).
Eligibility
Inclusion Criteria7
- Age ≥18 years
- High relapse risk MDS, defined by:
- IPSS-R score ≥3.5.
- IPSS-M stratification as intermediate-high, high, or very high risk.
- Eligible for allogeneic HSCT (including matched or mismatched related/unrelated donor transplantations).
- Karnofsky Performance Status (KPS) ≥60.
- Signed informed consent.
Exclusion Criteria7
- Severe organ dysfunction:
- Left ventricular ejection fraction <50%.
- Oxygen supplementation requirement.
- Serum bilirubin >1.5x upper limit of normal (unless due to Gilbert syndrome) or AST/ALT >5x upper limit of normal.
- Estimated glomerular filtration rate (eGFR) <50 mL/min.
- History of prior allogeneic HSCT.
- Any condition deemed unsuitable by the investigator.
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Interventions
Patients undergo direct allogeneic HSCT.
Patients receive one to two cycles of bridging therapy before undergoing allogeneic HSCT. o Bridging Therapy Regimen: Hypomethylating agents (HMA) alone or HMA-based combination chemotherapy, e.g., azacitidine (AZA) 100 mg/day + venetoclax (VEN) 400 mg/day for 7 days. Targeted therapies (e.g., IDH1 inhibitors) will be used for eligible patients.
Locations(5)
View Full Details on ClinicalTrials.gov
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NCT06918834