Impact of allogeneic stem cell transplantation in patients with higher risk myelodysplastic syndromes

Allogeneic stem cell transplantation (SCT) is a curative treatment in myelodysplastic syndromes (MDS). We performed a retrospective single center study of all patients with newly diagnosed higher risk (HR) MDS (IPSS-R > 3.5 points) between 2000 and 2023. We identified 2045 patients with HR-MDS, of which 427 (21%) underwent SCT. The median post-SCT overall survival (OS) was 23.9 months, progression-free survival (PFS) was 14.4 months, 5-year cumulative incidence (CI) of relapse was 37%, and 5-year CI of treatment-related mortality (TRM) was 25%. There were no significant differences in OS, PFS, CI of relapse, or CI of TRM between age categories. Survival improved over the observation period (median 11.8 months in 2000-2010, 28.2 months in 2011-2016, and 40.2 months in 2017-2023; p = 0.01). By multivariate analysis, TP53 status was the most important predictor of post-SCT OS. Patients with TP53-wild type MDS had an OS of 69% at 5 years (HR for death 0.32 with SCT, p < 0.001). Patients with TP53 mutations had poor outcomes, with OS of 9.1 months in monoallelic (HR for death 0.88 with SCT, p = 0.69) and 6.8 months in biallelic (HR for death 0.76 with SCT, p = 0.14). SCT can lead to excellent long-term survival in TP53-wild type HR MDS.