Cyclosporine A (CSA) and methotrexate (MTX) is the standard graft-versus-host disease (GVHD) prophylaxis regimen for matched sibling donor (MSD) allogeneic hematopoietic cell transplantation (allo-HCT). Recently, post-transplantation cyclophosphamide (PTCy) has been shown to be effective in GVHD prevention. In this registry-based study, we compared outcomes of 118 patients treated with PTCy and 1202 patients with CSA/MTX who underwent MSD allo-HCT for acute myelogenous leukemia. In a matched-pair analysis, PTCy was associated with a higher incidence of relapse at 2 years compared with CSA/MTX (41.1% versus 21.3%; P =.039). The incidences of day +180 grade II-IV acute GVHD and 2-year chronic GVHD were comparable in the PTCy and CSA/MTX arms (25.2% versus 25.4% [P =.90] and 42.6% versus 42.6% [P =.84], respectively). Similarly, 2-year leukemia-free survival (LFS; 54.4% versus 74.32%; P =.052), overall survival (OS; 70.6% versus 79.7%; P =.15), and GVHD-free relapse-free survival (GRFS; 38.1% versus 52.5%; P =.49) were not statistically different in the 2 arms. Our data show that GVHD prophylaxis with PTCy is feasible, resulting in similar incidences of GVHD, GRFS, LFS, and OS as seen with conventional CSA/MTX in patients undergoing allo-HCT from an MSD. The higher rate of relapse observed with PTCy needs further evaluation in a prospective study.
Bibliographical noteFunding Information:
The authors thank the EBMT centers and national registries for contributing patient information and data collection. Supporting information is available at the EBMT website ( www.ebmt.org). Reporting institutions included in this study are available in the Online Supporting Information Appendix. There is no relevant funding to report. A.N. M.M. and M.L. contributed to the conception and design of the study; M.L. analyzed the data; B.D. B.N.S. A.N. M.L. M.M. contributed to the writing of the manuscript; all authors critically reviewed the manuscript and approved the final version. A.N. and B.D. contributed equally to this work and should be considered co-first authors. Requests for the raw data used in this study should be addressed to M.L.
© 2021 The American Society for Transplantation and Cellular Therapy