Belumosudil is indicated to treat individuals aged 12 years and older with chronic graft-versus-host disease who have received at least 2 prior systemic therapy treatments without success.
Data from a pooled analysis of oral medication belumosudil (Rezurock; Kadmon) showed that certain organ clinical responses correlated with clinically meaningful changes in patient-reported outcomes for the treatment of chronic graft-versus-host disease (cGVHD), Sanofi said in a statement.
Kadmon, a Sanofi subsidiary, will present the results of the analysis at the 2022 American Society for Transplantation and Cellular Therapy and the Center for International Blood & Marrow Transplant Research Tandem Meetings.
“These belumosudil data reinforce the importance of patient-reported outcome measures by showing their correlation to clinical response. Knowing the impact that chronic graft-versus-host disease has on patients, the results add support for use of patient-reported outcomes to assess responses in clinical care,” Jonathan Ieyoub, head of Sanofi US Rezurock Medical, said in a statement.
The analysis was of the ROCKstar and KD025-208 studies, which demonstrated a strong, though not universal, correlation between organ clinical response measures and clinically meaningful changes in patient-reported outcomes for those treated with belumosudil. The clinically meaningful changes in patient-reported outcomes were specifically reported in the eye, lung, mouth, skin, upper gastrointestinal (GI), and overall measures.
The data support use of patient-reported outcomes for response assessment in cGVHD clinical trials, as well as patient care, because it helps capture the individual’s perspective on disease activity.
The pooled analysis included data on 170 individuals enrolled in the 2 belumosudil studies. They had a baseline patient reported outcome, at least 1 follow-up patient-reported outcome, and at least 1 response assessment.
The median age of participants in the study was aged 54.5 years, and 58% were men. The median number of prior treatments was 3, and approximately 70% of individuals had severe cGVHD. The median duration of follow-up at the time of data cut-off was 15 months.
The organ responses, which were defined as complete and partial responses, were 41% in eyes, 68% in joints, 23% in lungs, 57% in mouth, 35% in skin, 87% in lower GI, 73% in upper GI, and 77% overall.
For all the organs, except for esophagus, joints, and lower GI, there was at least 1 patient-reported outcome that was statistically correlated at p<0.05 with the National Institutes of Health (NIH) organ response.
The NIH clinical lung response, but not the lung response based on FEV1% change, was associated with the lung patient-reported response.
Belumosudil is indicated to treat individuals aged 12 years and older, with cGVHD who have received at least 2 prior systemic therapy treatments without an adequate response. It is also the first and only approved therapy inhibiting Rho-associated coiled-coil kinase 2. It is unknown if the drug is effective and safe for children younger than aged 12 years.
cGVHD is a complication that can occur following allogeneic stem cell transplantation, resulting in significant morbidity and mortality, according to the statement.
Transplanted immune cells attack the individual’s cells, which leads to inflammation and fibrosis in multiple tissues.
Rezurock (belumosudil) patient-reported outcomes correlated with clinical response in chronic graft-versus-host disease. Sanofi. News release. April 21, 2022. Accessed April 21, 2022. https://www.news.sanofi.us/2022-04-21-Rezurock-R-belumosudil-patient-reported-outcomes-correlated-with-clinical-response-in-chronic-graft-versus-host-disease