Does IPSS-R downstaging before transplantation improve the prognosis of patients with myelodysplastic neoplasms?

Abstract

AbstractIn patients with myelodysplastic syndrome (MDS) higher revised International Prognostic Scoring System (IPSS-R) scores at transplant are associated with worse transplant outcome. Thus, it may seem beneficial to improve IPSS-R scores by therapeutic intervention before transplantation in order to “downstage” the disease risk. However, there is no evidence, to date, to support this approach. A retrospective analysis of the European Society for Blood and Marrow Transplantation transplant registry was performed to investigate the role of therapeutic interventions before transplantation with regard to changes in IPSS-R and transplant outcomes. A total of 1482 patients with MDS with sufficient data to calculate IPSS-R score at diagnosis and at time of transplantation were selected and analyzed for transplant outcome in a multivariable Cox model including IPSS-R score at diagnosis, treatment intervention, change in IPSS-R score before transplant, and several patient and transplant variables. Transplant outcome was unaffected by IPSS-R score change in untreated patients and moderately superior in patients treated with chemotherapy with, improved IPSS-R score at transplant. Improved IPSS-R score after hypomethylating agents (HMAs) or other therapies showed no beneficial effect. However, when IPSS-R score progressed after chemotherapy, HMAs, or other therapies, transplant outcome was worse than without any prior treatment. Similar results were found when reduction or increase in bone marrow (BM) blasts between diagnosis and transplantation was considered. The results show a limited benefit of IPSS-R score downstaging or reduction of BM blasts after chemotherapy and no benefit for HMAs or other treatments and thus question the role of prior therapy in patients with MDS scheduled for transplantation. The model-based survival estimates should help inform decision-making for both doctors and patients.