作者
Christof Scheid,Diderik-Jan Eikema,Michel van Gelder,Urpu Salmenniemi,Johan Maertens,Jakob Passweg,Didier Blaise,Jenny Byrne,Nicolaus Kröger,Katja Sockel,Patrice Chevallier,Jean Henri Bourhis,Jan J. Cornelissen,Henrik Sengeløv,Jürgen Finke,John A. Snowden,Tobias Gedde‐Dahl,Jérôme Cornillon,Urs Schanz,Asha Patel,Linda Köster,Liesbeth C. de Wreede,Patrick Hayden,Kavita Raj,Joanna Drozd‐Sokołowska,Carmelo Gurnari,Francesco Onida,Donal P. McLornan,Marie Robin,Ibrahim Yakoub‐Agha
摘要
In MDS patients higher IPSS-R at transplant is associated with worse transplant outcome. Thus, it may seem beneficial to improve IPSS-R by therapeutic intervention prior to transplantation in order to "down-stage" the disease risk. However, there is no evidence to date to support this approach. A retrospective analysis of the EBMT transplant registry was performed to investigate the role of therapeutic interventions prior to transplantation with regard to changes in IPSS-R and transplant outcomes. A total of 1482 MDS patients with sufficient data to calculate IPSS-R at diagnosis and at time of transplantation were selected and analysed for transplant outcome in a multivariable Cox model including IPSS-R at diagnosis, treatment intervention, change in IPSS-R before transplant and several patient and transplant variables. Transplant outcome was unaffected by IPSS-R change in untreated patients and moderately superior in chemotherapy-treated patients with improved IPSS-R at transplant. Improved IPSS-R after hypomethylating agents (HMA) or other therapies showed no beneficial effect. However, when IPSS-R progressed after chemotherapy, (HMA) 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 down staging or reduction of BM blasts after chemotherapy and no benefit for HMA or other treatments and thus question the role of prior therapy in MDS patients scheduled for transplantation. The model-based survival estimates should help inform decision making for both doctors and patients.