Evidence map›Paper›PMID 41046308›Full record

ArticleBone marrow transplantation2025

Real-world experience of belumosudil and belumosudil/ruxolitinib combination in steroid-refractory chronic graft-versus-host disease.

Gagan Raju, Moneeza Walji, David Nemirovsky, Sean Devlin, Ilan Goldstein, Amandeep Singh, Pamela Susman, Natasia Rodriguez, Miguel-Angel Perales, Doris M Ponce

Erratum issuedAbstract read
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. An erratum has been issued. Not yet cited in PubMed.

0numbers the graph read from it
0cells of the map it votes in
0citing papers in PubMed
–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

0 citing papers in PubMed.

No citing paper in PubMed yet.

4 · The record

Corrections and comments

5 · Who and what money

Authors and funding

10 authors.

Gagan RajuDepartment of Medicine, Adult Bone Marrow Transplant Service, Memorial Sloan Kettering Cancer Center, New York, NY, USA.
Moneeza WaljiDepartment of Medicine, Adult Bone Marrow Transplant Service, Memorial Sloan Kettering Cancer Center, New York, NY, USA.
David NemirovskyDepartment of Epidemiology and Biostatistics, Memorial Sloan Kettering Cancer Center, New York, NY, USA.ORCID 0009-0007-6971-1408
Sean DevlinDepartment of Epidemiology and Biostatistics, Memorial Sloan Kettering Cancer Center, New York, NY, USA.ORCID 0000-0002-6801-720X
Ilan GoldsteinDepartment of Medicine, Adult Bone Marrow Transplant Service, Memorial Sloan Kettering Cancer Center, New York, NY, USA.
Amandeep SinghDepartment of Medicine, Adult Bone Marrow Transplant Service, Memorial Sloan Kettering Cancer Center, New York, NY, USA.
Pamela SusmanDepartment of Medicine, Adult Bone Marrow Transplant Service, Memorial Sloan Kettering Cancer Center, New York, NY, USA.
Natasia RodriguezDepartment of Medicine, Adult Bone Marrow Transplant Service, Memorial Sloan Kettering Cancer Center, New York, NY, USA.ORCID 0000-0003-4183-3845
Miguel-Angel PeralesDepartment of Medicine, Adult Bone Marrow Transplant Service, Memorial Sloan Kettering Cancer Center, New York, NY, USA.ORCID 0000-0002-5910-4571
Doris M PonceDepartment of Medicine, Adult Bone Marrow Transplant Service, Memorial Sloan Kettering Cancer Center, New York, NY, USA. ponced@mskcc.org.ORCID 0000-0002-9422-5766

Funding

X-RAY CRYSTALLOGRAPHYP30CA008748 · NCI · SLOAN-KETTERING INSTITUTE FOR CANCER RES · PI Nicholas D Socci · 1985 to 2026
$347.4M
Intestinal microbiome restoration in allogeneic stem cell transplantationR01HL164902 · NHLBI · SLOAN-KETTERING INST CAN RESEARCH · PI Doris Ponce, Marcel R M van den Brink · 2023 to 2026
$1.8M
Microbiome manipulation through oral live biotherapeutic product (LBP) for the treatment of gut acute graft-versus-host disease (GVHD)R34HL184529 · NHLBI · SLOAN-KETTERING INST CAN RESEARCH · PI Doris Ponce · 2026 to 2026
$282k
NCI NIH HHS P30 CA008748NHLBI NIH HHS R01 HL164902NHLBI NIH HHS R34 HL184529U.S. Department of Health & Human Services | NIH | National Heart, Lung, and Blood Institute (NHLBI) 5R01-HL164902-02
6 · The paper itself

Abstract

Belumosudil is approved after failure of ≥2 lines of therapy in chronic graft-versus-host disease cGVHD. However, real-world data is limited. We conducted a retrospective analysis of 67 patients with steroid-refractory or dependent (SR/SD) cGVHD. At baseline, most patients had advanced multi-organ cGVHD. The 6- and 12-month overall response rate (ORR) was 61%. However, a subset of patients achieved deeper responses with ongoing therapy at 12 months. The 6-month failure-free survival (FFS) was 75% (95%CI: 65-86) whereas the 12-month FFS was 66% (95%CI: 55-78). A low incidence of drug-related grade ≥3 toxicities was observed. A cohort of patients with immune function analysis showed gradual improvement in immune subsets at 1-year post-treatment. The combined bel-rux cohort (n = 14) showed a 6- and 12-month ORR of 64% and 57%, respectively. Overall, belumosudil was associated with high treatment response and survival outcomes. Notably, deeper responses were observed with ongoing therapy, and it was overall well tolerated. In a cohort of patients, immune cell populations had preserved to improved values throughout treatment. Patients who received bel-rux demonstrated efficacy and safety as well. Overall, our real-world study indicates similar findings to the clinical trial and supports the use of belumosudil in cGVHD.

Indexed as

Graft vs Host DiseasePyrazolesAdultAgedChronic DiseaseFemaleHumansIsoquinolinesMaleMiddle AgedNitrilesPyrimidinesRetrospective StudiesSulfonamidesIsoquinolinesK-115NitrilesPyrazolesPyrimidinesSulfonamides

Identifiers

PMID41046308
PMCPMC13405253

What OpenQuestion holds

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Registered trials

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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.