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Heidenreich, Silke ; Egger-Heidrich, Katharina ; Halter, Jörg P. ; Jost, Lasse ; Stölzel, Friedrich ; Perl, Markus ; Denk, Alexander ; Edinger, Matthias ; Herr, Wolfgang ; Kröger, Nicolaus ; Wolff, Daniel ; Ayuk, Francis ; Fante, Matthias A.

Safety and efficacy of the ROCK-2-inhibitor Belumosudil in cGvHD treatment - a retrospective, German-Swiss multicenter real-world data analysis

Heidenreich, Silke, Egger-Heidrich, Katharina, Halter, Jörg P., Jost, Lasse, Stölzel, Friedrich, Perl, Markus, Denk, Alexander , Edinger, Matthias , Herr, Wolfgang, Kröger, Nicolaus, Wolff, Daniel , 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.

Date of publication of this fulltext: 28 Jan 2025 05:27
Article
DOI to cite this document: 10.5283/epub.74676


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 ...

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.



Involved Institutions


Details

Item typeArticle
Journal or Publication TitleBone Marrow Transplantation
Publisher:Springer Nature
Open Access Type:DEAL (Springer)
Date14 January 2025
InstitutionsMedicine > Lehrstuhl für Innere Medizin III (Hämatologie und Internistische Onkologie)
Identification Number
ValueType
10.1038/s41409-024-02507-9DOI
Dewey Decimal Classification600 Technology > 610 Medical sciences Medicine
StatusPublished
RefereedYes, this version has been refereed
Created at the University of RegensburgPartially
URN of the UB Regensburgurn:nbn:de:bvb:355-epub-746760
Item ID74676

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