Efficacy and safety of belumosudil for treatment of cGVHD: multicenter retrospective analysis of the French cohort of the compassionate use program, on behalf of the French Society of Bone Marrow Transplantation and Cellular Therapy

Chronic graft versus host disease is a major cause of morbidity after allogeneic haematopoietic cell transplantation. Belumosudil has recently been approved for the treatment of cGVHD refractory after two lines of treatment. However, few data are available to evaluate its efficacy and safety in real life. 68 patients with cGVHD received belumosudil through a compassionate access program in France. The median follow-up was 337 days from belumosudil initiation. Eighty-two percent of patients had severe cGVHD with a median of three organs involved. Patients had received a median of three prior treatment lines. Median treatment duration was 251 days. The best overall response rate (ORR) was 57.3%, including 14.7% complete remission (CR) and 42.6% partial response (PR). The ORR at three and six months was 47% and 45.6%, respectively. Liver and mouth involvement showed the highest response rates (72.7% and 70.4%), while lung involvement had the lowest (17.2%). Median failure-free survival (FFS) was not reached, with 6- and 12-month FFS rates of 89.1% and 80.4%, respectively. Nine patients died, mainly from GVHD (n = 5). Ten adverse events were reported, leading to treatment discontinuation in three cases. These results support the efficacy and safety of belumosudil in refractory cGVHD.

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Publication Details

Journal
Bone Marrow Transplantation
Published
2025-04-01
DOI
https://doi.org/10.1038/s41409-025-02554-w
Citations
10
Primary Topic
Hematopoietic Stem Cell Transplantation
Type
article
Field-Weighted Citation Impact
17.70
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article

Efficacy and safety of belumosudil for treatment of cGVHD: multicenter retrospective analysis of the French cohort of the compassionate use program, on behalf of the French Society of Bone Marrow Transplantation and Cellular Therapy

Michael L. Loschi, Sophie Le Grand, Anne Huynh, David Michonneau et al.
10 citations
Bone Marrow Transplantation
Hematopoietic Stem Cell Transplantation
17.70
article

Efficacy and safety of belumosudil for treatment of cGVHD: multicenter retrospective analysis of the French cohort of the compassionate use program, on behalf of the French Society of Bone Marrow Transplantation and Cellular Therapy

Michael L. Loschi, Sophie Le Grand, Anne Huynh, David Michonneau, Cristina Castilla‐Llorente, Florent Malard, Quentin Cabrera, Nicole Raus, Jean‐Jacques Tudesq, Mathieu Meunier, Maud D’Aveni, Ambroise Marçais, Édouard Forcade, Alban Villate, Anne‐Lise Ménard, Natacha Maillard, Léonardo Magro, Hélène Labussière‐Wallet
article en
10 citations

Abstract

Chronic graft versus host disease is a major cause of morbidity after allogeneic haematopoietic cell transplantation. Belumosudil has recently been approved for the treatment of cGVHD refractory after two lines of treatment. However, few data are available to evaluate its efficacy and safety in real life. 68 patients with cGVHD received belumosudil through a compassionate access program in France. The median follow-up was 337 days from belumosudil initiation. Eighty-two percent of patients had severe cGVHD with a median of three organs involved. Patients had received a median of three prior treatment lines. Median treatment duration was 251 days. The best overall response rate (ORR) was 57.3%, including 14.7% complete remission (CR) and 42.6% partial response (PR). The ORR at three and six months was 47% and 45.6%, respectively. Liver and mouth involvement showed the highest response rates (72.7% and 70.4%), while lung involvement had the lowest (17.2%). Median failure-free survival (FFS) was not reached, with 6- and 12-month FFS rates of 89.1% and 80.4%, respectively. Nine patients died, mainly from GVHD (n = 5). Ten adverse events were reported, leading to treatment discontinuation in three cases. These results support the efficacy and safety of belumosudil in refractory cGVHD.

Bone Marrow TransplantationVol. 60(6)
Université de Tours (FR), Hôpital Necker-Enfants Malades (FR), Inserm (FR), Lyon College (US), Université Côte d'Azur (FR), Université de Lyon (FR), Université Paris Cité (FR), Institut Gustave Roussy (FR), Centre Hospitalier Universitaire de Nice (FR), Université de Poitiers (FR), Sorbonne Université (FR), Institut Necker Enfants Malades (FR), Centre Hospitalier Universitaire de La Réunion (RE), Centre Hospitalier Universitaire de Montpellier (FR), Assistance Publique – Hôpitaux de Paris (FR), Institut universitaire du cancer de Toulouse Oncopole (FR), Centre Hospitalier Régional et Universitaire de Nancy (FR), Hôpital Saint-Antoine (FR), Polyclinique de Bordeaux-Tondu (FR), Centre de Recherche Saint-Antoine (FR), Peuplements végétaux et bioagresseurs en milieu tropical (RE), Laboratoire National Henri Becquerel (FR), Agir Pour les Maladies Chroniques (FR), Hôpital Saint-Louis (FR), Société Francophone d’Arthroscopie (FR), Hôpital Claude Huriez (FR), Société Francophone du Diabète (FR), Université Grenoble Alpes (FR)
Good health and well-being
Openalex Percentile: Top 1%
Hematopoietic Stem Cell Transplantation
17.70
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