Patient-Reported Outcomes and Organ Responses in Belumosudil Studies

According to Stephanie Lee, MD, MPH, the association between organ responses and patient-reported outcomes (PROs) changes during belumosudil therapy for chronic graft-versus-host disease (cGVHD) is unclear. Dr. Lee and colleagues examined clinical trial data and concluded that “there is a very strong but not universal correlation between organ clinical response measures and clinically meaningful changes in PROs recommended by the National Institutes of Health consensus conference in patients treated with belumosudil.” Their findings were presented at the Transplantation & Cellular Therapy 2022 Tandem Meetings.

Specifically, Dr. Lee reported that “there is excellent correlation of the skin, mouth, eye, upper GI, lung (clinical but not [forced expiratory volume in one second, or] FEV1%), and overall measures.” Conversely, the study found that joint, esophagus, lower gastrointestinal (GI), and FEV1% measures exhibited a poor correlation with clinical organ responses, which the authors theorized could mean these PROs “may be measuring different manifestations, or the clinician or PRO measures may be inadequately sensitive to change in cGVHD.”

The analysis included PROs from all dosage groups in both belumosudil trials (NCT02841995 and NCT03640481), and the generalized estimating equation (GEE) was used to account for missing or repeated data. The final assessment included 170 patients, 70% of which had severe cGVHD. The study did not analyze liver and genital tract measures due to a lack of PROs specific to those sites.

Over a median follow-up of 15 months (range: 0.6–44 months), organ responses—complete or partial—were observed in 48/135 patients (35%) for skin, 84/124 (67.7%) for joint, 56/99 (57%) for mouth, 52/128 (41%) for eye, 19/26 (73%) for upper GI, 13/15 (87%) for lower GI, and 14/61 (23%) for lung, with an overall total of 130/170 (77%). These authors identified the described PROs as having meaningful associations to organ response.

“Overall, these data support use of PROs for response assessment in cGVHD clinical trials and patient care to help capture the patient’s perspective on cGVHD disease activity,” the researchers proposed in closing.