Broers AEC, de Jong CN, Bakunina K, et al. Posttransplant Cyclophosphamide for Prevention of Graft-versus-Host Disease: The Prospective Randomized HOVON-96 Trial. Blood Advances. 2022; (doi: 10.1182/bloodadvances.2021005847).
Posttransplant cyclophosphamide (PT-Cy) following non-myeloablative (NMA)-matched allogeneic hematopoietic stem cell transplantation (HSCT) significantly increased survival without severe graft-versus-host-disease (GVHD), according to new research. The prospective, randomized Phase III trial compared PT-Cy plus a short course of cyclosporine A (CsA) with a combination of CsA and mycophenolic acid (MPA) in patients diagnosed with high-risk hematological malignancy and a matched related or at least eight of eight HLA matched unrelated donor. In all, 52 patients received CsA/MPA and 99 received PT-Cy/CsA. At six months, CsA/MPA recipients saw a 48% cumulative incidence of grade II-IV acute GVHD, compared with 30% for PT-Cy/CsA recipients. The two-year cumulative incidence of chronic extensive GVHD was 48% and 16%, respectively. The one-year GVHD-free, relapse-free survival estimate was 21% compared with 45% in the two groups respectively. Relapse incidence, progression-free, and overall survival were not significantly different between treatment groups at a median follow-up of 56.4 months. PT-Cy combined with CsA improved survival due to significant reduction in severe acute and chronic GVHD.