Christopher G Kanakry 1 , Hua-Ling Tsai 1 , Javier Bolaños-Meade 1 , B Douglas Smith 1 , Ivana Gojo 1 , Jennifer A Kanakry 1 , Yvette L Kasamon 1 , Douglas E Gladstone 1 , William Matsui 1 , Ivan Borrello 1 , Carol Ann Huff 1 , Lode J Swinnen 1 , Jonathan D Powell 1 , Keith W Pratz 1 , Amy E DeZern 1 , Margaret M Showel 1 , Michael A McDevitt 1 , Robert A Brodsky 1 , Mark J Levis 1 , Richard F Ambinder 1 , Ephraim J Fuchs 1 , Gary L Rosner 1 , Richard J Jones 1 , Leo Luznik 1
Dec 11 2014
High-dose, posttransplantation cyclophosphamide (PTCy) reduces severe graft-versus-host disease (GVHD) after allogeneic blood or marrow transplantation (alloBMT), but the impact of PTCy on long-term, disease-specific outcomes is unclear. We conducted a retrospective study of 209 consecutive adult patients transplanted for acute myeloid leukemia (AML, n = 138), myelodysplastic syndrome (n = 28), or acute lymphoblastic leukemia (ALL, n = 43) using PTCy as sole GVHD prophylaxis after myeloablative conditioning and HLA-matched-related or -unrelated T-cell-replete allografting. At alloBMT, 30% of patients were not in morphologic complete remission. The cumulative incidences of grades II to IV and III to IV acute GVHD at 100 days and chronic GVHD at 2 years were 45%, 11%, and 13%, respectively. Forty-three percent of patients did not require immunosuppression for any reason beyond PTCy. At 3 years, relapse cumulative incidence was 36%, disease-free survival was 46%, survival free of disease and chronic GVHD was 39%, and overall survival was 58%. Lack of remission at alloBMT, adverse cytogenetics, and low allograft nucleated cell dose were associated with inferior survival for AML patients. Minimal residual disease but not t(9;22) was associated with inferior outcomes for ALL patients. The ability to limit posttransplantation immunosuppression makes PTCy a promising transplantation platform for the integration of postgrafting strategies to prevent relapse.