Real-world outcomes in refractory chronic graft-<i>versus</i>-host disease: the Italian multicenter experience with belumosudil

Chronic graft-versus-host disease (cGvHD) remains a major late complication of allogeneic hematopoietic 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 -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 was 52.6%, 57.6%, and 55.0% at 3, 6, and 12 months, respectively. Median time to response was 3 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 NIH Severity Index Score (0-10 scale) showed meaningful improvements (≥-2 points) in 33%, 36%, and 29% of patients at 3, 6, 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.

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Journal
Haematologica
Published
2026-02-19
DOI
https://doi.org/10.3324/haematol.2025.289302
Primary Topic
Hematopoietic Stem Cell Transplantation
Type
article
Field-Weighted Citation Impact
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article

Real-world outcomes in refractory chronic graft-versus-host disease: the Italian multicenter experience with belumosudil

Maria Teresa Lupo Stanghellini, Nicola Polverelli, Alessandra Algarotti, Giulia Prunotto et al.
Haematologica
Hematopoietic Stem Cell Transplantation
article

Real-world outcomes in refractory chronic graft-versus-host disease: the Italian multicenter experience with belumosudil

Maria Teresa Lupo Stanghellini, Nicola Polverelli, Alessandra Algarotti, Giulia Prunotto, Francesca Elice, Michele Malagola, Giorgia Mancini, Chiara Nozzoli, L. Prezioso, Marcello Roberto, Roberta De Marchi, Marilena Fedele, Federica Locatelli, J. Mariotti, Dalila Salvatore, Lidia Santoro, Alessandro Spina, Francesca Bonifazi, Francesco Zallio, Antonella Geromin, Massimo Martino, Alessandra Picardi, Luca Castagna, Patrizia Chiusolo, Cristina Skert, Antonio Pierini, Luca Facchini, Alessandro Rambaldi, Mattia Algeri, Luisa Giaccone, Manuela Tumino
article en

Abstract

Chronic graft-versus-host disease (cGvHD) remains a major late complication of allogeneic hematopoietic 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 -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 was 52.6%, 57.6%, and 55.0% at 3, 6, and 12 months, respectively. Median time to response was 3 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 NIH Severity Index Score (0-10 scale) showed meaningful improvements (≥-2 points) in 33%, 36%, and 29% of patients at 3, 6, 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.

Haematologica
Università Cattolica del Sacro Cuore (IT), University of Rome Tor Vergata (IT), University of Parma (IT), Vita-Salute San Raffaele University (IT), University of Perugia (IT), Casa Sollievo della Sofferenza (IT), Agostino Gemelli University Polyclinic (IT), Brescia University (US), Magna Graecia University (IT), Azienda Ospedaliera Citta' della Salute e della Scienza di Torino (IT), Azienda Sanitaria Unità Locale di Reggio Emilia (IT), Azienda ospedaliera "Bianchi-Melacrino-Morelli" (IT), Ospedale Vincenzo Cervello (IT), Azienda Socio Sanitaria Territoriale degli Spedali Civili di Brescia (IT), Ospedale A. Perrino (IT), Ospedale Papa Giovanni XXIII (IT), Azienda Ospedaliero Universitaria Ospedali Riuniti (IT), Istituto Oncologico Veneto (IT), Azienda Ospedaliera San Gerardo (IT), University Hospitals Parma Medical Center (US), Azienda Ospedaliero-Universitaria Careggi (IT), Istituto di Ematologia di Bologna (IT), Azienda USL di Bologna (IT), Bambino Gesù Children's Hospital (IT), Ospedale Santa Maria della Misericordia di Udine (IT), Azienda Sanitaria Locale Alessandria (IT), Azienda Sanitaria Ospedaliera S.Croce e Carle Cuneo (IT), Azienda Ospedale - Università Padova (IT), Policlinico San Matteo Fondazione (IT), Ospedale San Bortolo (IT), Istituti di Ricovero e Cura a Carattere Scientifico (IT), Gruppo Italiano per il Trapianto di Midollo Osseo (IT), IRCCS Humanitas Research Hospital (IT), Azienda Ospedaliera Universitaria Pisana (IT), Istituto di Ricovero e Cura a Carattere Scientifico San Raffaele (IT), University of Turin (IT), University of Brescia (IT), University of Bologna (IT)
Ministero della Salute
Good health and well-being
Openalex Percentile: Top 49%
Hematopoietic Stem Cell Transplantation
DOIOpen Access PDFOpenAlex
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