Survival benefits with transplantation in secondary AML evolving from myelodysplastic syndrome with hypomethylating treatment failure

The prognosis for patients with myelodysplastic syndrome with hypomethylating treatment failure (MDS-HTF) has been known to be poor. However, the clinical outcomes and optimal treatment options for secondary AML evolving from MDS-HTF (sAML/MDS-HTF) are not well known. This retrospective analysis was conducted to evaluate the clinical outcomes and influences of treatment options on survival in 46 consecutive patients with sAML/MDS-HTF. The median OS rates were 1.4 months in the best supportive care group ( n =15) and 9.4 months in the active treatment group ( n =31). One-year OS rates were 13.3% and 36.8%, respectively ( P =0.001). Active treatment ( P <0.001), lower BM blast (<33%) at sAML ( P =0.007), non-poor NCCN (National Cancer Comprehensive Network) cytogenetics ( P =0.001) and good performance status (ECOG (Eastern Cooperative Oncology Group) ⩽1) ( P =0.024) were significant predictors affecting favorable OS in a multivariate analysis. Of the active treatment options, allo-SCT with prior chemotherapy (CTx) showed better OS compared with CTx only or SCT without CTx ( P =0.019). Our analyses suggest that active treatment, particularly SCT following CTx, should be considered in patients with sAML/MDS-HTF if the patient is medically fit.