Whether one or two agents added to post-transplant cyclophosphamide (PTCy) are needed in HLA-matched allogeneic hematopoietic stem cell transplantation (allo-HSCT) with peripheral blood stem cells (PBSC) is debated. We retrospectively compared PTCy in association with a calcineurin inhibitor (PTCy+CNI) or with a CNI plus mycophenolate mofetil (PTCy+CNI+MMF) in adult patients transplanted for acute myeloid leukemia in first complete remission and receiving PBSC in the period from 2010 to 2020. Propensity score matching was performed using exact matching for donor type (related or unrelated) and the nearest neighbor for other variables (i.e. age, adverse cytogenetics, Karnofsky performance status, patient and donor cytomegalovirus serology, conditioning intensity). Each group comprised 146 patients, with 63% in total undergoing matched unrelated-allo-HSCT. Median follow up was longer for PTCy+CNI (36 [IQR 31-39] months versus 25 [IQR 19-30] months for PTCy+CNI+MMF, p < 0.01). At 2 years, PTCy+CNI was associated with a higher incidence of extensive chronic GVHD (16% [95% CI 10-22] versus 6% [95% CI 3-12] for PTCy+CNI+MMF, p < 0.03) while no differences were observed for all the other transplant outcomes. Addition of MMF to PTCy and CNI may help to prevent extensive chronic GVHD in HLA-matched allo-HSCT with PBSC.

1st State Pavlov Medical University of St Petersburg Raisa Gorbacheva Memorial Research Institute for Paediatric Oncology Haematology and Transplantation St Petersburg St Petersburg Russia

Chaim Sheba Medical Center Tel Hashomer Ramat Gan Israel

Clatterbridge Cancer Centre Liverpool Royal Liverpool University Hospital Clatterbridge Cancer Centre NHS Foundation Trust Division of Stem Cell Transplantation and Haematology Liverpool Liverpool UK

Department of Clinical Medicine and Surgery Federico 2 University of Naples Naples Italy

Department of Hematology Hospital General Universitario Gregorio Marañon Instituto de Investigacion Sanitaria Gregorio Marañon Departamento de Medicina Universidad Computense de Madrid Madrid Spain

Department of Hematology University Medical Center Groningen University of Groningen Groningen The Netherlands

Department of Internal Medicine Division of Hematology GROW School for Oncology and Developmental Biology Maastricht University Medical Center Maastricht The Netherlands

Dipartimento di Diagnostica per Immagini Radioterapia Oncologica ed Ematologia Fondazione Policlinico A Gemelli IRCCS Sezione di Ematologia Dipartimento di Scienze Radiologiche ed Ematologiche Università Cattolica del Sacro Cuore Roma Italy

EBMT Paris study office Paris France

Erasmus MC Cancer Institute University Medical Center Rotterdam Department of Haematology Rotterdam Rotterdam The Netherlands

Federico 2 University of Naples Hematology Department Naples Italy

Hematology Department Hôpital Saint Antoine Service d'Hématologie et Thérapie Cellulaire Paris France

Hematology Department Hospital Universitari i Politècnic La Fe Departament de Medicina Universitat de Valencia CIBERONC Instituto Carlos 3 Valencia Spain

Hospital Clinic BMT Unit Dept of Haematology Institute of Haematology and Oncology IDIBAPS Institut Josep Carreras Barcelona Barcelona Spain

Institute of Haematology and Blood Transfusion Prague Czech Republic

Medicana International Hospital Istanbul Bone Marrow Transplant Unit Istanbul Istanbul Turkey

Ospedale San Raffaele s r l Haematology and BMT Milano Italy

Sorbonne Universités UPMC Univ Paris 06 INSERM Centre de Recherche Saint Antoine Paris France

VU University Medical Center Department of Haematology Amsterdam Amsterdam The Netherlands

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