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Post-transplant cyclophosphamide and sirolimus based graft-versus-host disease prophylaxis after allogeneic stem cell transplantation for acute myeloid leukemia. 2022
Lorenzo Lazzari, and
Aitana Balaguer-Roselló, and
Juan Montoro, and
Raffaella Greco, and
Rafael Hernani, and
Maria Teresa Lupo-Stanghellini, and
Marta Villalba, and
Fabio Giglio, and
Ana Facal, and
Francesca Lorentino, and
Manuel Guerreiro, and
Alessandro Bruno, and
Ariadna Pérez, and
Elisabetta Xue, and
Daniela Clerici, and
Simona Piemontese, and
José Luis Piñana, and
Miguel Ángel Sanz, and
Carlos Solano, and
Javier de la Rubia, and
Fabio Ciceri, and
Jacopo Peccatori, and
Jaime Sanz
Hematology and Bone Marrow Transplantation Unit, IRCCS Ospedale San Raffaele, Milan, Italy. lazzari.lorenzo@hsr.it.
Post-transplant cyclophosphamide (PTCy) has emerged as a promising graft-versus-host disease (GvHD) prophylaxis in allogeneic hematopoietic stem cell transplantation (allo-HSCT). However, no studies have reported the efficacy of a GvHD prophylaxis based on PTCy with sirolimus (Sir-PTCy) in patients with acute myeloid leukemia (AML). In this retrospective study, we analyze the use of sirolimus in combination with PTCy, with or without mycophenolate mofetil (MMF), on 242 consecutive adult patients with AML undergoing a myeloablative first allo-HSCT from different donor types, in three European centers between January 2017 and December 2020. Seventy-seven (32%) patients received allo-HSCT from HLA-matched sibling donor, 101 (42%) from HLA-matched and mismatched unrelated donor, and 64 (26%) from haploidentical donor. Except for neutrophil and platelet engraftment, which was slower in the haploidentical cohort, no significant differences were observed in major transplant outcomes according to donor type in univariate and multivariate analysis. GvHD prophylaxis with Sir-PTCy, with or without MMF, is safe and effective in patients with AML undergoing myeloablative allo-HSCT, resulting in low rates of transplant-related mortality, relapse/progression, and acute and chronic GvHD in all donor settings.
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