Fig. 1: Selected efficacy evaluations at the end of the Japanese clinical trial.

a Best ORR at key time points; b DOR rate (belumosudil responders only); c treatment status by individual patient; d best ORR by organ; e changes in steroid dose and LSS overall scores over time in the adolescent patient. In panel b + indicates censored patients. In panel c the symbols indicating the start and end of response refer to the start and end of periods judged to be CR/PR. Since the end of response could refer to a situation in which one affected organ had worsened, but other organs were still responding (i.e., not an overall evaluation of progressive disease), treatment was continued after the end of response with the aim of maintaining any persisting responses (in order to preserve the treatment benefit in the organ(s) that continue to respond), whilst also ensuring patient safety. Reasons for discontinuation are indicated as follows: a = withdrawal of consent; b = physician’s judgment; c = progression/recurrence of malignancy; d = progression of cGVHD; e = protocol deviation. The adolescent patient is indicated by #14. In panel e the steroid dose is shown as a plane and corresponds to the treatment ongoing in panel c. At baseline, the corticosteroid dose was 10.0 mg/day; this was reduced to 5.0 mg/day on the day following Week 24, 2.5 mg/day on the day following Week 52, 1.5 mg/day on the day following Week 61, and treatment was discontinued on the day following Week 65. The LSS score was improved by 19.0 points from baseline (score 24.8 points) to Week 2 (score 5.8 points), and clinically meaningful improvements (a decrease of ≥7 points from baseline, indicated by the blue dotted line) endured between Weeks 2–84. BL baseline, CI confidence interval, CR complete response, EOT end of treatment, GI gastrointestinal, LR-M lack of response-mixed, NIH National Institutes of Health, PR partial response.