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Belumosudil for the treatment of chronic graft-versus-host disease: a single center real-world experience

Bone Marrow Transplantation volume 60, pages 555–557 (2025)Cite this article

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Chronic graft-versus-host-disease (cGVHD) remains a major cause of morbidity and mortality after allogeneic hematopoietic stem cell transplantation (HCT) [1,2,3,4]. Belumosudil is an oral selective rho-associated coiled-coil kinase 2 (ROCK) inhibitor approved by the US FDA in 2021 for the treatment of cGVHD after failure of at least two prior lines of systemic therapy in patients 12 years or older [5]. The ROCKstar study evaluated efficacy of belumosudil in patients with moderate to severe cGVHD, with 2–5 prior lines of therapy, showing an overall response rate of 74% (95% CI: 62–84%). Clinical responses were seen in all subgroups of clinical interest including patients with severe cGVHD, > 3 prior lines of therapy, and those with prior exposure to ruxolitinib or ibrutinib [6, 7]. Herein, we report our single-institution experience with using belumosudil in a real-world setting.

We conducted a retrospective analysis of 45 patients who were treated with belumosudil 200 mg daily (twice daily if on a proton-pump inhibitor) for treatment of cGVHD City of Hope Medical Center (COH) between August 2021 to November 2022. Baseline characteristics are summarized in Table 1 supplemental appendix. Briefly, the median age of patients prior to initiating belumosudil was 59 years (range 19–78). Donor type was predominantly matched-unrelated donors (42.2%) or mismatched-unrelated (26.7%) using peripheral blood stem cell grafts (91.1%) and tacrolimus and sirolimus (71.1%) for GVHD prophylaxis based on the current standard at COH [8]. Patients with cGVHD most often had skin (75.6%), eyes (48.9%), mouth (46.7%), and joints (37.8%). Baseline severity of GVHD based on organ and global scores are summarized in Table 2 supplementary appendix. Based on the NIH consensus criteria, 46.7% had severe GVHD, and 37.8% had moderate cGVHD prior to initiating belumosudil. Median prior lines of therapy were 3 (range 1–6) with majority of patients previously treated with ruxolitinib (71.1%). The time from HCT to onset of cGVHD was 24 months (range 0–147) and time from HCT to belumosudil start was 35 months (range 6–160). There were 23 patients (51.1%) treated concurrently with ruxolitinib and had received a median 461 days (range 78–1226) of ruxolitinib prior to initiating belumosudil. In addition, 78% of patients were on prednisone, with median dose of 10 mg/day (approximately 0.15 mg/kg/day, range 1–40 mg/day) upon initiating salvage with belumosudil.

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Fig. 1: Response rate to belumosudil by organ involvement and global response rate as defined by NIH Consensus Response Criteria Working Group.

Data availability

The datasets generated during and/or analyzed during the current study are available from the corresponding author on reasonable request.

References

  1. DeFilipp Z, Alousi AM, Pidala JA, Carpenter PA, Onstad LE, Arai S, et al. Nonrelapse mortality among patients diagnosed with chronic GVHD: an updated analysis from the Chronic GVHD Consortium. Blood Adv. 2021;5:4278–84.

    Article  CAS  PubMed  PubMed Central  Google Scholar 

  2. Arai S, Arora M, Wang T, Spellman SR, He W, Couriel DR, et al. Graft-versus-Host Disease Working Committee of the CIBMTR. Increasing incidence of chronic graft-versus-host disease in allogeneic transplantation: a report from the Center for International Blood and Marrow Transplant Research. Biol Blood Marrow Transplant. 2015;21:266–74.

    Article  PubMed  Google Scholar 

  3. Malard F, Mohty M. Updates in chronic graft-versus-host disease management. Am J Hematol. 2023;98:1637–44.

    Article  PubMed  Google Scholar 

  4. Hamilton BK. Updates in chronic graft-versus-host disease. Hematology Am Soc Hematol Educ Program. 2021;2021:648–54.

    Article  PubMed  PubMed Central  Google Scholar 

  5. U.S. Food & Drug Administration. “FDA approves belumosudil for chronic graft-versus-host disease.” https://www.fda.gov/drugs/resources-information-approved-drugs/fda-approves-belumosudil-chronic-graft-versus-host-disease. Last updated 2/1/2022. Accessed 8/9/2024.

  6. Cutler C, Lee SJ, Arai S, Rotta M, Zoghi B, Lazaryan A, et al. Belumosudil for chronic graft-versus-host disease after 2 or more prior lines of therapy: the ROCKstar Study. Blood. 2021;138:2278–89.

    Article  CAS  PubMed  PubMed Central  Google Scholar 

  7. Lee SJ, Cutler C, Pavletic SZ, Blazar BR. Belumosudil for Chronic Graft-Versus-Host Disease after 2 or More Prior Lines of Systemic Therapy: 3-Year Follow-up of the Rockstar Study. Transplant Cell Ther. 2024;30:S262–S263.

    Article  Google Scholar 

  8. Rodriguez R, Nakamura R, Palmer JM, Parker P, Shayani S, Nademanee A, et al. A phase II pilot study of tacrolimus/sirolimus GVHD prophylaxis for sibling donor hematopoietic stem cell transplantation using 3 conditioning regimens. Blood. 2010;115:1098–105.

    Article  CAS  PubMed  PubMed Central  Google Scholar 

  9. Pusic I, Lee C, Veeraputhiran M, Minor C, DiPersio JF. Belumosudil and ruxolitinib combination for treatment of refractory chronic graft-versus-host disease. Bone Marrow Transplant. 2024;59:282–4.

    Article  CAS  PubMed  Google Scholar 

  10. Caputo J, Peddireddi A, Bhatta S, Huang Y, Bezerra E, Brammer JE, et al. Combination Ruxolitinib and Belumosudil Is Tolerable and Induces Responses Despite Treatment Failure As Monotherapies. Transplant Cell Ther. 2024;30:S285.

    Article  Google Scholar 

  11. Raju G, Walji M, Nemirovosky D, Rodriguez NT, Perales M, Ponce DM, et al. Combined Belumosudil-Ruxolitinib Therapy for the Treatment of Steroid-Refractory Chronic Graft-Versus-Host Disease (cGVHD) Was Associated with Robust Treatment Response and Was Well-Tolerated. Transplant Cell Ther. 2024;30:S281.

    Article  Google Scholar 

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Funding

This research did not receive any specific grant from funding agencies in the public, commercial, or not-for-profit sectors.

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Author notes

  1. These authors contributed equally: Badri Modi, Dat Ngo.

  2. These authors jointly supervised this work: Haris Ali, Amandeep Salhotra.

Authors and Affiliations

  1. Department of Dermatology, City of Hope, Duarte, CA, USA

    Badri Modi

  2. Department of Pharmacy, City of Hope, Duarte, CA, USA

    Dat Ngo & Jason Chen

  3. Department of Computational and Quantitative Medicine, Division of Biostatistics Core, City of Hope, Duarte, CA, USA

    Dongyun Yang & Haoyue Shan

  4. Department of Hematology and Hematopoietic Cell Transplantation, City of Hope, Duarte, CA, USA

    Karamjeet Sandhu, Nahid Rashid, Idoroenyi Amanam, Salman Otoukesh, Ryotaro Nakamura, Haris Ali & Amandeep Salhotra

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Contributions

Badri Modi and Dat Ngo: study design, extracting and analyzing data, interpreting results, manuscript writing. Jason Chen: extracting data. Dongyun Yang and Haoyue Shan: statistical analysis. Karamjeet Sandhu, Ryotaro Nakamura, Idoroenyi Amanam, Salman Otoukesh, Nahid Rashid: manuscript review and editing. Haris Ali, Amandeep Salhotra: concept, study design, manuscript review and editing.

Corresponding author

Correspondence to Amandeep Salhotra.

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Competing interests

The authors have no competing interests if not stated below. This research did not receive any specific grant from funding agencies in the public, commercial, or not-for-profit sectors. Amandeep Salhotra: Received funding from Orca Bio, Bristol Myers Squibb, Rigel Pharmaceuticals. Serves on advisory board for Incyte Corporation and speaker panel for Sanofi. Haris Ali: Serves on speaker panel for Incyte Corporation.

Ethics approval and consent to participate

This study was approved by our Institutional Review Board (IRB) under registration number #22401 and met the criteria for exemption for ethics approval and consent to participate due to the retrospective nature of this study under Code of Federal Regulations 45CFR46.104 (d). Exempt Category 4 states “secondary research for which consent is not required: secondary use of identifiable private information or identifiable biospecimens, if at least the follow criteria is met: (iii) The research involves only information collection and analysis involving the investigator’s use of identifiable health information when that use is regulated under 45 CFR parts 160 and 164, subparts A and E, for the purposes of “health care operations” or “research” as those are defined as 45 CFR 164.501 or for “public health activities and purposes” as described under 45 CFR 164.512 (b).

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Modi, B., Ngo, D., Chen, J. et al. Belumosudil for the treatment of chronic graft-versus-host disease: a single center real-world experience. Bone Marrow Transplant 60, 555–557 (2025). https://doi.org/10.1038/s41409-024-02498-7

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