Overview
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).
Description
A total of 236 patients will be randomized in a 1:1 ratio into the immediate transplantation group (n=118) and the disease control group (n=118). The study will continue until at least 124 events occur.
Eligibility
Inclusion Criteria:
- 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 Criteria:
- 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.