Transplantation in patients with lower-risk MDS: a prospective phase 2 trial based on donor availability

Allogeneic hematopoietic stem cell transplantation (HSCT) remains the only potential curative therapy for myelodysplastic syndrome (MDS), recommended in higher risk disease according to the International Prognostic Scoring System International Prognostic Scoring System: A system that turns patient data into a score. The score tells how quickly a myelodysplastic syndrome (MDS) case is progressing and helps predict what may happen with the patient's MDS in the future. Also called IPSS. (IPSS). We conducted a phase 2 multicenter trial (MDS-ALLO-RISK) investigating whether allogeneic HSCT improves overall survival (OS) in patients with lower-risk MDS who exhibit additional high-risk features (intermediate or higher revised-IPSS risk, thrombocytopenia thrombocytopenia: (throm-buh-sie-tuh-PEE-nee-uh) A condition in which there is a shortage of platelets in the bloodstream. This results in a low platelet count. Bleeding and bruising often occur with thrombocytopenia. with <20 × 109/L, neutropenia neutropenia: (noo-truh-PEE-nee-uh) A condition in which there is a shortage of neutrophils in the bloodstream. Neutrophils are a type of white blood cell. This results in a low white blood cell count. <0.5 × 109/L, or failure to 2 lines of therapy). A total of 77 patients (median age, 62.5 years) with low or intermediate-1 IPSS scores were enrolled and stratified based on the presence of a matched HLA HLA: See human leukocyte antigen. donor, 62 patients in the donor arm and 15 without a donor. Despite high remission rates in patients who had received a transplant (67.8% vs 21.4%), the 3-year OS did not significantly differ between arms (57.6% in the donor arm vs 64.3% in the no-donor arm; hazard ratio, 0.75; P = .53). The adjusted analysis using inverse probability of treatment weighting confirmed the lack of survival benefit with HSCT. Transplantation was associated with higher rates of chronic graft-versus-host disease, severe infections, and nonrelapse mortality (24.7%). Although quality of life improved slightly over time in patients who had received a transplant, the difference was not statistically significant. The trial was stopped early due to slow enrollment and futility. The findings highlight the need for improving posttransplant outcomes to justify HSCT in patients with lower-risk MDS with poor prognostic features. This trial was registered at www.clinicaltrials.gov as #NCT02757989.