The association between graft-versus-host disease (GVHD) occurrence and acute myeloid leukemia (AML) relapse in patients treated with HLA-haploidentical allogeneic hematopoietic stem cell transplantation (Haplo-HCT) with post-transplant cyclophosphamide (PTCy)-based GVHD prophylaxis has remained debated. Here, we addressed this issue in patients with active AML at transplantation. 2-year cumulative incidences of relapse and leukemia-free survival (LFS) were 49% and 32.3%, respectively. There were no associations between acute nor chronic GVHD of any grade and lower relapse incidence. However, grade I acute GVHD was associated with better LFS (HR = 0.71, 95% CI 0.51-0.99, P = 0.04). In contrast, grade III-IV acute (HR = 3.09, 95% CI 1.87-5.12, P < 0.0001) as well as extensive chronic (HR = 3.3, 95% CI 1.81-6.04, P = 0.0001) GVHD correlated with higher nonrelapse mortality leading to lower LFS (HR = 1.36, 95% CI 0.99-1.86, P = 0.056 and HR = 1.97, 95% CI 1.35-2.89, P = 0.0004, respectively). In conclusion, these data suggest a dissociation of graft-versus-leukemia effects from GVHD in patients with active AML treated with PTCy-based Haplo-HCT.

Department of Hematology Saint Antoine Hospital Paris France

Department of Internal Medicine 3 LMU University Hospital of Munich Campus Grosshadern Munich Germany

Dept D`Hematologie CHU Nantes Nantes France

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

Division of Hematology and Bone Marrow Transplantation The Chaim Sheba Medical Center Tel Hashomer Ramat Gan Israel

Division of Hematology Azienda Ospedaliero Universitaria di Udine Udine Italy

EBMT Paris Study Office CEREST TC Paris France

Haematology and BMT Ospedale San Raffaele S R L Milan Italy

INSERM UMR 938 Paris France

Institute of Hematology and Blood Transfusion Prague Czech Republic

IRCCS Ospedale Policlinico San Martino Genoa Italy

Laboratory of Hematology GIGA I3 University of Liege and CHU of Liège Sart Tilman 4000 Liège Belgium

Programme de Transplantation et d'immunothérapie Cellulaire Management Sport Cancer Lab Institut Paoli Calmettes Aix Marseille University Marseille France

Sorbonne University Paris France

University Hospital Dresden Medizinische Klinik und Poliklinik TU Dresden Dresden Germany

University Hospital Schleswig Holstein Kiel Germany

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Post-transplant cyclophosphamide separates graft-versus host disease and graft versus leukemia effects after HLA-matched stem-cell transplantation for acute myeloid leukemia