Real-world outcomes in refractory chronic graft-versus-host disease: the Italian multicenter experience with belumosudil
Year: 2026
Type: article
Study design: Observational Study
Abstract: Chronic graft-versus-host disease (cGvHD) remains a major late complication of allogeneic hemato-poietic stem cell transplantation, leading to impaired quality of life and late non-relapse mortality. Belumosudil, an oral ROCK2 inhibitor with immunomodulatory and antifibrotic properties, represents a novel treatment for steroid-refractory or steroid-dependent cGvHD. We conducted a retrospective, multicenter study of 80 patients treated with belumosudil across 29 Italian transplant centers through a compassionate use program. Patients were heavily pretreated (74% with ≥3 prior lines, 84% previously exposed to ruxolitinib), with severe disease in 86% and a median of three organs involved. The best overall response rate was 62.5%, while overall response rates were 52.6%, 57.6%, and 55.0% at three, six, and 12 months, respectively. Median time to response was three months, and the 12-month duration of response was 83.4%. Failure-free survival at 12 months was 67.5%. Responses were observed in all involved organs. Patient-reported outcomes assessed through the National Institutes of Health Severity Index Score (0-10 scale) showed meaningful improvements (≥-2 points) in 33%, 36%, and 29% of patients at three, six, and 12 months, respectively. Treatment was well tolerated. These real-world findings confirm the effectiveness of belumosudil in patients with cGvHD refractory to multiple lines of immunosuppressive therapy.
Source: Haematologica
Authors: Francesca Bonifazi, Marcello Roberto, Maria Teresa Lupo Stanghellini, Patrizia Chiusolo, Nicola Polverelli, Antonella Geromin, Lucia Prezioso, Mattia Algeri, Giorgia Mancini, Giulia Prunotto, Alessandra Algarotti, Chiara Nozzoli, Michele Malagola, Nicola Mordini, Marilena Fedele, Lidia Santoro, Francesco Zallio, Luca Castagna, Alessandra Picardi, Alessandro Busca, Luisa Giaccone, Cristina Skert, Francesca Elice, Luca Facchini, Jacopo Mariotti, Antonio Pierini, Alessandro Spina, Roberta De Marchi, Dalila Salvatore, Massimo Martino, Manuela Tumino, Alessandro Rambaldi, Franco Locatelli
Institutions: Istituto di Ematologia di Bologna, University of Bologna, IRCCS Ospedale San Raffaele, Università Cattolica del Sacro Cuore, Agostino Gemelli University Polyclinic, Istituti di Ricovero e Cura a Carattere Scientifico, Policlinico San Matteo Fondazione, Azienda sanitaria universitaria Friuli Centrale, Magna Graecia University, Bambino Gesù Children's Hospital, Azienda Ospedaliero-Universitaria delle Marche, Ospedale Papa Giovanni XXIII, Azienda Ospedaliero-Universitaria Careggi, Azienda Socio Sanitaria Territoriale degli Spedali Civili di Brescia, University of Brescia, Azienda Sanitaria Ospedaliera S.Croce e Carle Cuneo, Ospedale Vincenzo Cervello, University of Rome Tor Vergata, Ospedale Antonio Cardarelli, Azienda Ospedaliera Citta' della Salute e della Scienza di Torino, University of Turin, Ospedale San Bortolo, Azienda ULSS 8 Berica, Azienda Sanitaria Unità Locale di Reggio Emilia, IRCCS Humanitas Research Hospital, University of Perugia, Ospedale A. Perrino, Istituto Oncologico Veneto, Casa Sollievo della Sofferenza, Azienda ospedaliera "Bianchi-Melacrino-Morelli", Azienda Ospedale - Università Padova, University of Milan
Language: English
Subfield: Hematology
Field: Medicine
Domain: Health Sciences
Open Access status: gold
Estimated APC paid: $2,157
Funder: Ministero della Salute
OpenAlex ID: W7130511790