{"entity":{"id":"paper-rockstar-blood-2021","kind":"paper","name":"Belumosudil for chronic graft-versus-host disease after 2 or more prior lines of therapy: the ROCKstar Study","aka":[],"tldr":"The primary report of ROCKstar: about three in four patients with chronic graft-versus-host disease that had failed at least two treatments responded to belumosudil at either dose, and responses lasted about a year.","summary":"Phase 2 randomised multicentre registration study of belumosudil, an oral selective ROCK2 inhibitor that reduces type 17 and follicular T helper cells and enhances regulatory T cells, at 200 mg daily (66 patients) and 200 mg twice daily (66) in chronic graft-versus-host disease after 2 to 5 prior lines of therapy. The primary endpoint was best overall response rate; duration of response, Lee Symptom Scale change, failure-free survival, corticosteroid dose reduction and overall survival were also evaluated.\n\nAt a median follow-up of 14 months the best overall response rate was 74 percent (95% CI 62 to 84) with 200 mg daily and 77 percent (95% CI 65 to 87) with 200 mg twice daily, with high response rates in all subgroups and complete responses in all affected organs. Median duration of response was 54 weeks and 44 percent of patients remained on therapy for at least a year. Symptom reduction was reported in 59 and 62 percent. Sixteen patients (12 percent) discontinued because of possible drug-related adverse events.","asOf":"2026-09-24","links":[{"label":"Blood 2021","url":"https://doi.org/10.1182/blood.2021012021"},{"label":"PubMed","url":"https://pubmed.ncbi.nlm.nih.gov/34265047/"},{"label":"ClinicalTrials.gov NCT03640481","url":"https://clinicaltrials.gov/study/NCT03640481"}],"tags":[],"related":[],"cancers":[],"sections":[],"technologies":[],"targets":[],"drugs":["belumosudil"],"companies":["sanofi"],"institutions":[],"pathways":[],"terms":["gvhd","allogeneic-transplant"],"trials":["rockstar"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":["blood"],"dependsOn":[],"notes":[],"journal":"Blood","year":2021,"doi":"10.1182/blood.2021012021","pmid":"34265047","authors":"Cutler C, Lee SJ, Arai S, et al.","paperType":"rct","findings":["Best overall response rate 74% (95% CI 62 to 84) with 200 mg daily and 77% (95% CI 65 to 87) with 200 mg twice daily.","Median duration of response 54 weeks; 44% of patients on therapy for at least 1 year.","Lee Symptom Scale reduction in 59% and 62%; 12% discontinued for possible drug-related adverse events."],"whatItMeans":"This is the trial behind belumosudil's July 2021 US approval for chronic graft-versus-host disease after two or more lines. For patients it means a once-daily pill with a high response rate in a condition where earlier treatments had failed; the FDA's own analysis, counting responses only through cycle 7 day 1, gave 75 percent.","caveats":["Both arms received belumosudil; there was no comparator, and response was assessed by site investigators.","The 74% and 77% are best response at any time; the FDA's stricter through-cycle-7 analysis and its 1.9-month median duration of response measure different things.","The registry records the study as terminated in 2023 because adolescents were hard to recruit, after the adult analysis and approval."],"changedPractice":true,"participants":132},"route":"/key-papers/paper-rockstar-blood-2021/","neighbours":{"drug":[{"id":"belumosudil","kind":"drug","name":"Belumosudil","route":"/drugs/belumosudil/"}],"company":[{"id":"sanofi","kind":"company","name":"Sanofi","route":"/companies/sanofi/"}],"term":[{"id":"allogeneic-transplant","kind":"term","name":"Allogeneic stem cell transplant (allo-SCT)","route":"/terms/allogeneic-transplant/"},{"id":"gvhd","kind":"term","name":"Graft-versus-host disease (GVHD) and graft-versus-leukaemia","route":"/terms/gvhd/"}],"trial":[{"id":"rockstar","kind":"trial","name":"ROCKstar","route":"/trials/rockstar/"}],"journal":[{"id":"blood","kind":"journal","name":"Blood","route":"/journals/blood/"}]}}