Heidenreich, Silke and Egger-Heidrich, Katharina and Halter, Joerg P. and Jost, Lasse and Stoelzel, Friedrich and Perl, Markus and Denk, Alexander and Edinger, Matthias and Herr, Wolfgang and Kroeger, Nicolaus and Wolff, Daniel and Ayuk, Francis and Fante, Matthias A. (2025) Safety and efficacy of the ROCK-2-inhibitor Belumosudil in cGvHD treatment - a retrospective, German-Swiss multicenter real-world data analysis. BONE MARROW TRANSPLANTATION, 60 (4). pp. 439-446. ISSN 0268-3369, 1476-5365
Full text not available from this repository. (Request a copy)Abstract
Belumosudil is a first in class ROCK2-inhibitor approved by the FDA for the 3rd line treatment of chronic graft-versus-host disease (cGvHD). In this retrospective real-world analysis, we report safety and efficacy data of belumosudil treatment from 5 German/Swiss transplant centers. A total of 33 adult patients (median age 59 years) with moderate (n = 2) or severe (n = 31) cGvHD were treated on individual request due to lack of EMA approval. The patient cohort had a long history of cGvHD (median 44 months) and was heavily pretreated (median 4 prior lines). The overall response rate was 42% (95%CI, 25-60%) including organ responses in all organs except the liver (n = 2). The median time to response was 3 months (range, 1-9 months) and 8 of 14 patients (57%) had a durable response at last follow-up. One-third of patients had at least a 50% reduction in concomitant corticosteroid dosage. Median failure-free survival and median overall survival were 16.5 and 23.1 months, respectively. Adverse events >= CTCAE grade 3 were reported in 27% of patients, with a predominance of infectious events, including one fatal course. The results are consistent with previous prospective trials including a favorable safety profile, while acknowledging the challenges of a heavily pretreated patient cohort.
| Item Type: | Article |
|---|---|
| Uncontrolled Keywords: | VERSUS-HOST-DISEASE; CONSENSUS DEVELOPMENT PROJECT; CLINICAL-TRIALS; CHRONIC GVHD; CRITERIA; DIAGNOSIS; TARGETS; IV.; |
| Subjects: | 600 Technology > 610 Medical sciences Medicine |
| Divisions: | Medicine > Lehrstuhl für Innere Medizin III (Hämatologie und Internistische Onkologie) |
| Depositing User: | Dr. Gernot Deinzer |
| Date Deposited: | 15 Sep 2026 10:11 |
| Last Modified: | 15 Sep 2026 10:11 |
| URI: | https://pred.uni-regensburg.de/id/eprint/66658 |
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