Alexandre Bazinet, Alex Bataller, Kelly Chien, Koji Sasaki, Guillermo Montalban-Bravo, Danielle Hammond, Ian Bouligny, Mahesh Swaminathan, Wei Ying Jen, Eitan Kugler, Rashmi Kanagal-Shamanna, Tapan Kadia, Gautam Borthakur, Courtney DiNardo, Nicholas Short, Naveen Pemmaraju, Elias Jabbour, Naval Daver, Farhad Ravandi, Samuel Alejandro Urrutia, Georgina Gener-Ricós, Julie Braish, Robert Briski, Juan José Rodríguez‐Sevilla, Ziyi Li, Richard E. Champlin, Elizabeth J. Shpall, Amin Alousi, Betul Oran, Chitra Hosing, Jeremy Ramdial, Amanda Olson, Sherry Pierce, Julianne Chen, Hagop Kantarjian, Guillermo Garcia-Manero, Uday Popat
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.