Prognostic value of post-transplantation measurable residual disease in patients with myelodysplastic syndrome: A prospective cohort study.

Objective: Allogeneic hematopoietic stem cell transplantation (allo-HSCT) is the only potentially curative method for treating myelodysplastic syndrome (MDS). Post-HSCT measurable residual disease (post-HSCT MRD) is associated with inferior transplant outcomes. In this prospective study, we aimed to investigate the prognostic value of post-HSCT MRD in relapse prediction in MDS.

Methods: A total of 166 patients diagnosed with MDS were prospectively enrolled in this study. The Kaplan-Meier method was used to calculate the survival probabilities. Potential risk factors for outcomes after transplantation were evaluated through univariate and multivariate Cox regression models.

Results: For patients with negative and positive post-HSCT MRD, the cumulative incidence of relapse (CIR) and disease-free survival (DFS) at 3 years were 5.9% and 69.6% (P<0.001) and 82.7% and 26.1% (P<0.001), respectively. In the multivariate analysis, post-HSCT MRD (HR=22.801, P<0.001) and Revised International Prognostic Scoring System (IPSS-R) risk stratification (HR=4.346, P=0.003) were independently correlated with relapse. A scoring system for relapse prediction was built based on post-HSCT MRD and IPSS-R stratification. The cumulative incidence of relapse at 3 years was 1.1%, 15.8%, and 91.7% for patients with scores of 0, 1, and 2, respectively (P<0.001).

Conclusions: Our results demonstrated both post-HSCT MRD and IPSS-R scores were independent prognostic factors for OS, DFS, and relapse for MDS patients after allo-HSCT. The risk score system could better predict transplant outcomes and refine the risk stratification than alone in patients with MDS.