Methotrexate Versus Mycophenolate Mofetil Prophylaxis in Allogeneic Hematopoietic Cell Transplantation for Chronic Myeloid Malignancies: A Retrospective Analysis on Behalf of the Chronic Malignancies Working Party of the EBMT
American Journal of Hematology, vol. 100, pp. 38–51
Abstract
Prophylaxis strategies for Graft versus host disease (GVHD) in allogeneic hematopoietic cell transplantation (allo-HCT) frequently encompass a combination of a calcineurin inhibitor (CNI) with either methotrexate (MTX) or mycophenolate mofetil (MMF). The aim of this retrospective, EBMT registry-based study was to determine outcome differences for chronic myeloid malignancies and secondary acute myeloid leukemia (sAML) between MTX- and MMF-based prophylaxis regimens while taking potential heterogeneity between subgroups into consideration. Eligible were patients transplanted between 2007 and 2017 who received either MTX- or MMF prophylaxis in combination with a CNI. Endpoints after allo-HCT were overall survival, relapse-free survival (RFS), relapse incidence, non-relapse mortality (NRM), and Grades 2-4 acute GVHD (aGvHD). Overall, 13 699 patients from 321 centers were included. Median follow-up was 42.8 months (IQR 19.8-74.5 months). MTX prophylaxis was associated with reduced overall mortality (HR 0.87, 95% CI 0.81-0.95, p = 0.001) and NRM (HR 0.86, 95% CI 0.78-0.96, p = 0.006) compared with MMF in multivariable Cox regression models in the whole cohort without significant interaction between prophylaxis and subgroups. In contrast, there was no significant association of prophylaxis with risk of relapse (HR 1.03 MTX vs. MMF, 95% CI 0.94-1.14, p = 0.53) or RFS (HR 0.95, 95% CI 0.88-1.01, p = 0.12). There was a reduced risk of Grades 2-4 acute GVHD and reduced mortality after acute GVHD with MTX prophylaxis but no association with outcome in a landmark analysis in patients without aGvHD at 3 months after allo-HCT. In conclusion, MTX-complemented CNI prophylaxis was associated with favorable survival, and with favorable survival after aGVHD compared with MMF.
Authors 25
-
Thomas Luft corresponding
Affiliation as printed
Department of Internal Medicine V, Hematology, Oncology and Rheumatology University of Heidelberg Heidelberg Germany
-
European Society for Blood and Marrow Transplantation
Affiliation as printed
EBMT Leiden Study Unit Leiden The Netherlands
-
European Society for Blood and Marrow Transplantation
Affiliation as printed
EBMT Leiden Study Unit Leiden The Netherlands
-
University Medical Center Hamburg-Eppendorf
Affiliation as printed
Interdisziplinäre Klinik und Poliklinik für Stammzelltransplantation University Hospital Eppendorf Hamburg Germany
-
Affiliation as printed
Department of Hematology Stem Cell Transplantation Essen Germany
-
Affiliation as printed
Medical Clinic and Policinic 1, University Hospital Leipzig Leipzig Germany
-
Technische Universität Dresden
Affiliation as printed
Medizinische Klinik I, University Hospital TU Dresden Dresden Germany
-
Affiliation as printed
Saint‐Louis Hospital BMT Unit Paris France
-
University of Münster · University Hospital Münster
Affiliation as printed
Medical Clinic of Internal Medicine A, University of Muenster Muenster Germany
-
Affiliation as printed
Bone Marrow Transplant Unit L 4043, University of Copenhagen Copenhagen Denmark
-
Medizinische Hochschule Hannover
Affiliation as printed
Clinic for Hematology, Hemostaseology, Oncology and Stem Cell Transplantation Hannover Medical School Hannover Germany
-
Charité - Universitätsmedizin Berlin
Affiliation as printed
BMT Department Charité, University Medicine Berlin Berlin Germany
-
Affiliation as printed
Department of Internal Medicine V, Hematology, Oncology and Rheumatology University of Heidelberg Heidelberg Germany
-
Inserm · Université de Lille · Centre Hospitalier Universitaire de Lille · Institute for Translational Research in Inflammation
Affiliation as printed
CHU de Lille Université de Lille, INSERM U1286 Infinite France
-
Universitair Ziekenhuis Leuven
Affiliation as printed
Department of Haematology University Hospital Gasthuisberg Leuven Belgium
-
Affiliation as printed
Helsinki University Central Hospital, HUCH Comprehensive Cancer Center Helsinki Finland
-
Universitätsklinikum Tübingen · University of Tübingen
Affiliation as printed
Innere Medizin II, Hematology, Oncology, Clinical Immunology and Rheumatology, University Hospital Tuebingen Tuebingen Germany
-
Karolinska University Hospital
Affiliation as printed
Department of Cell Therapy and Allogeneic Stem Cell Transplantation Karolinska University Hospital Stockholm Sweden
-
Heinrich Heine University Düsseldorf
Affiliation as printed
Department of Haematology, Oncology and Clinical Immunology Heinrich Heine Universitaet Düsseldorf Germany
-
Affiliation as printed
Department of Haematology St.Savvas Oncology Hospital Athens Greece
-
Liesbeth C. de Wreede Aachen Department of Biomedical Data Sciences Leiden University Medical Center Leiden The Netherlands
Leiden University Medical Center
Affiliation as printed
Department of Biomedical Data Sciences Leiden University Medical Center Leiden The Netherlands
-
University College London Hospitals NHS Foundation Trust · University College London
Affiliation as printed
Department of Haematology and Stem Cell Transplantation University College London Hospitals NHS Trust London UK
-
Medical University of Warsaw · Central Clinical Hospital
Affiliation as printed
Central Clinical Hospital The Medical University of Warsaw Warsaw Poland
-
University College London Hospitals NHS Foundation Trust · University College London
Affiliation as printed
Department of Haematology and Stem Cell Transplantation University College London Hospitals NHS Trust London UK
-
Affiliation as printed
Saint‐Louis Hospital BMT Unit Paris France
Cited by 3 stored of 3
3 results
No patents citing this paper on Lens.org (checked 2026-10-11).
References 17
17 results