[en] Acute graft-versus-host disease (aGvHD) remains a significant complication of allogeneic hematopoietic cell transplantation, with 40% of patients failing to respond to high-dose steroids. Ruxolitinib has become the standard treatment for steroid-refractory aGvHD (SR-GvHD), but its failure in approximately one-third of cases highlights the need for alternative therapies. Mesenchymal stromal cells (MSCs), known for their immunomodulatory properties, are suggested as a treatment option, but their role in SR-GvHD remains unclear. To better understand MSC therapy outcomes, the EBMT Cellular Therapy & Immunobiology Working Party conducted a survey of centers treating >20 SR-GvHD patients with MSCs between 2007 and 2020. Data from 313 patients were analyzed, revealing a 44.5% overall response rate at day 28. Responders at day 7 had a higher likelihood of maintaining responses by day 28. Using a landmark analysis, the overall survival at 12 months, conditional on being alive at day 28, was 39.2%. Survival at 12 months was 48.6% for responders, compared to 24.4% for non-responders. Despite manufacturing variabilities, MSCs produced by academic pharma appear effective in SR-GvHD, offering a viable treatment alternative for heavily pretreated patients. These findings support further investigation of MSCs to establish standardized protocols and validate their efficacy as third-line therapy for SR-GvHD.
Disciplines :
Hematology
Author, co-author :
Daenen, L G M ; Department of Hematology, University Medical Centre Utrecht, Utrecht, The Netherlands
van der Wagen, L E; Department of Hematology, University Medical Centre Utrecht, Utrecht, The Netherlands
Bonneville, E F ; EBMT Study Unit, Leiden, The Netherlands ; Department of Biomedical Data Sciences, Leiden University Medical Center, Leiden, The Netherlands
López-Corral, L ; Hematology Department. Hospital Universitario de Salamanca (Spain), IBSAL, CIBERONC. Centro de Investigación del Cáncer-IBMCC (USAL-CSIC), Salamanca, Spain
Bukauskas, A; Vilnius University Hospital Santaros Klinikos, Vilnius, Lithuania
Bornhäuser, M ; University Hospital Dresden, TU Dresden, Dresden, Germany
Beguin, Yves ; Université de Liège - ULiège > Département des sciences cliniques
Itäla-Remes, M; Turku University Hospital, Turku, Finland
Hoogenboom, J D; EBMT Study Unit, Leiden, The Netherlands
de Wreede, L C ; Department of Biomedical Data Sciences, Leiden University Medical Center, Leiden, The Netherlands
Malard, F ; Service d'Hématologie Clinique et Thérapie Cellulaire, Hôpital Saint-Antoine, AP-HP, Sorbonne Université, Centre de Recherche Saint-Antoine (CRSA), INSERM, Paris, France
Chabannon, C ; Institut Paoli-Calmettes, Centre de Lutte Contre le Cancer, Centre d'Investigations Cliniques en Biothérapies, Université d'Aix-Marseille, Inserm, CBT 1409, Marseille, France
Dazzi, F ; Comprehensive Cancer Centre, King's College London, London, UK
Ruggeri, A ; San Raffaele Scientific Institute, Hematology and Bone marrow Transplantation Unit, Milan, Italy
Kuball, J ; Department of Hematology, University Medical Centre Utrecht, Utrecht, The Netherlands. j.h.e.kuball@umcutrecht.nl
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