Choice of calcineurin inhibitor may impact the outcome of patients undergoing T-cell replete hematopoietic cell transplantation (HCT) with post-transplant cyclophosphamide (PT-Cy) and mycophenolate mofetil (MMF) for prophylaxis of graft-versus-host disease (GVHD). We retrospectively analyzed 2427 patients with acute myeloid leukemia (AML) in first remission transplanted from a haploidentical (n = 1844) or unrelated donor (UD, n = 583) using cyclosporine A (CSA, 63%) or tacrolimus (TAC, 37%) and PT-Cy/MMF. In univariate analysis, CSA and TAC groups did not differ in 2-year leukemia-free or overall survival, cumulative incidence (CI) of relapse or non-relapse mortality. CI of severe grade III-IV acute GVHD was lower with TAC (6.6% vs. 9.1%, p = 0.02), without difference in grade II-IV acute GVHD or grade III-IV acute GVHD/severe chronic GVHD, relapse-free survival (GRFS). In multivariate analysis, TAC was associated with a lower risk of severe grade III-IV acute GVHD solely with haploidentical donors (HR 0.64 [95% CI, 0.42-0.98], p = 0.04), but not UD (HR 0.49 [95% CI, 0.2-1.21], p = 0.12). There was no significant difference for chronic GVHD. In conclusion, PT-Cy/MMF-based GVHD prophylaxis resulted in favorable OS and GRFS, irrespective of the CNI added. In haploidentical HCT, TAC seemed to prevent severe acute GVHD more effectively than CSA without impact on other outcome parameters.

Chaim Sheba Medical Center Tel Hashomer Israel

Department of Hematology Hospital General Universitario Gregorio Marañon Instituto de Investigación Sanitaria Gregorio Marañón Univesidad Complutense de Madrid Madrid Spain

Department of Oncology Hematology IRCCS Humanitas Research Hospital via Manzoni 56 20089 Rozzano Milan Italy

Dipartimento di Diagnostica per Immagini Radioterapia Oncologica ed Ematologia Fondazione Policlinico Universitario A Gemelli IRCCS Università Cattolica Sacro Cuore Rome Italy

Goethe University Frankfurt University Hospital Dept of Medicine 2 Frankfurt am Main Germany

Hospital Universitario de Salamanca Salamanca Spain

Institute of Hematology and Blood Transfusion Prague Czech Republic

IRCCS San Martino Hospital Genova Italy

IRCCS San Raffaele Scientific Institute Milan Italy

Leiden University Medical Center Leiden Netherlands

Maria Sklodowska Curie National Research Institute of Oncology Gliwice Branch Gliwice Poland

Medicana International Hospital Istanbul Turkey

RM Gorbacheva Research Institute Pavlov University St Petersburg Russian Federation

Sorbonne University Department of Hematology Saint Antoine Hospital INSERM UMR 938 Paris France

Stem Cell Transplantation and Cellular Therapies Unit Grande Ospedale Metropolitano Bianchi Melacrino Morelli Reggio Calabria Italy

Transplant and cellular immunotherapy program Department of hematology Institut Paoli Calmettes Management Sport Cancer lab Luminy Aix Marseille University Marseille France

Turku University Hospital Turku Finland

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