Evidence map›Paper›PMID 40169928›Full record

ArticleBone marrow transplantation2025

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.

David Michonneau, Florent Malard, Sophie Le Grand, Leonardo Magro, Maud D'Aveni, Jean Jacques Tudesq, Alban Villate, Mathieu Meunier, Natacha Maillard, Cristina Castilla-Llorente and 8 more

Abstract readMulticenter Study
In one paragraph

Article in Bone marrow transplantation, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 9 papers, 1 of them a synthesis that pooled it.

0numbers the graph read from it
0cells of the map it votes in
9citing papers in PubMed, 1 pooled it
–field-weighted citation impact
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.

3 · Its place in the literature

Who cites it

9 citing papers in PubMed, 1 synthesis or guideline pooled it.

  1. Pooled it
  2. Article
  3. Review
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  8. Review
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4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

18 authors.

David MichonneauHematology and transplantation unit, Saint Louis hospital, Paris, France. david.michonneau@aphp.fr.ORCID http://orcid.org/0000-0003-4553-3065
Florent MalardSorbonne Université, Centre de Recherche Saint-Antoine, INSERM UMRs938, Service d'Hématologie Clinique et de Thérapie Cellulaire, Hôpital Saint Antoine, AP-HP, Paris, France.
Sophie Le GrandHematology and transplantation unit, Saint Louis hospital, Paris, France.ORCID http://orcid.org/0000-0002-9985-465X
Leonardo MagroHematology unit, Claude Huriez hospital, Lille, France.
Maud D'AveniHematology unit, Brabois hospital, Nancy University hospital, Vandoeuvre-les-Nancy, France.ORCID http://orcid.org/0000-0002-5909-2742
Jean Jacques TudesqHematology unit, Montpellier university hospital, Montpellier, France.ORCID http://orcid.org/0000-0003-4879-8106
Alban VillateHematology unit, Tours university hospital, Tours, France.
Mathieu MeunierHematology unit, Grenoble university hospital, La Tronche, France.
Natacha MaillardHematology unit, Poitiers university hospital, Poitiers, France.
Cristina Castilla-LlorenteHematology unit, Gustave Roussy Cancer Campus Grand Paris, Villejuif, France.
Ambroise MarçaisHematology unit, Necker Enfants-Malades hospital, Paris, France.ORCID http://orcid.org/0000-0002-6844-7920
Quentin CabreraHematology unit, Saint Pierre University hospital, La Réunion, France.
Anne HuynhHematology unit, Institut Universitaire de Cancerologie de Toulouse, Oncopole, Toulouse, France.
Anne-Lise MenardHematology unit, CRLCC Henri Becquerel, Rouen, France.
Edouard ForcadeService d'Hématologie Clinique et Thérapie Cellulaire, CHU Bordeaux, F-33000, Bordeaux, France.ORCID http://orcid.org/0000-0002-8873-2868
Hélène Labussière-WalletHematology unit, Lyon university hospital, Lyon, France.
Nicole RausSociété Francophone de Greffe de Moelle et de Thérapie Cellulaire, Lyon, France.
Michael LoschiHematology unit, Nice university hospital, Nice, France.ORCID http://orcid.org/0000-0002-4460-5939

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

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.

Indexed as

Graft vs Host DiseaseHematopoietic Stem Cell TransplantationAdolescentAdultAgedAllograftsChronic DiseaseCompassionate Use TrialsFemaleFranceHumansMaleMiddle AgedRetrospective StudiesYoung Adult

Identifiers

PMID40169928
PMCPMC12151857

What OpenQuestion holds

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Registered trials

None linked

Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.