Incidence, Risk Factors and Outcomes of Therapy-Related Myeloid Malignancies after Hematopoietic Cell Transplantation: Analysis of the Japan Society for Hematopoietic Cell Transplantation Late Complications and QOL Working Group

Background Therapy-related acute myeloid leukemia and myelodysplastic syndrome (t-AML/MDS) occur after hematopoietic cell transplantation (HCT). The incidence, risk factors and outcomes of t-AML/MDS among patients who underwent autologous or allogeneic HCT for a variety of disease have not been examined systemically. To address these questions, we conducted a retrospective cohort study using the Japanese national transplant registry database. Methods The study cohort included 43,790 patients of all ages who underwent autologous (n=24,916) or allogeneic HCT (n=18,874) for lymphoid malignancies, solid cancer and non-malignant diseases between 1977 and 2015 in Japan. Patients who had antecedent myeloid malignancies and those who had inherited disorders were excluded. Results With a median follow-up of 61 months among survivors, 354 patients developed t-AML/MDS, including 306 after autologous HCT and 48 after allogeneic HCT. The 10-year cumulative incidence of t-AML/MDS was 1.8% (95% CI, 1.6-2.0%) after autologous HCT and 0.3% (95% CI, 0.2-0.4%) after allogeneic HCT. The median patient age of t-AML/MDS diagnosis was higher in patients who had autologous HCT than those who had allogeneic HCT (59 vs 42 years, p Conclusions The incidence of t-AML/MDS was remarkably higher in patients who had autologous HCT than those who had allogeneic HCT. Risk factors and prognostic factors identified in this study would provide physicians with important information for counseling and managing patients about t-AML/MDS after HCT.