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. 2024 Mar 26;22:142. doi: 10.1186/s12916-024-03348-5

Table 2.

Response rates in the mITT population

Response
Data are n (%) unless stated otherwise
Belumosudil 200 mg once daily (N = 30)
ORR 22 (73.3%)
 95% CI 54.1–87.7%
 PR 22 (73.3%)
ORR for responses occurring within 6 months of treatment 20 (66.7%)
 95% CI 47.2–82.7%
 PR 20 (66.7%)
Median DOR, weeks (95% CI) NR (20.3–NR)
Median TTR, weeks (range) 4.3 (3.9–48.1)
Improvement from baseline based on LSSa
 Overall 15 (50.0%)
 Responder, n/N (%) 12/22 (54.5%)
 Non-responder, n/N (%) 3/8 (37.5%)
Median duration of improvement, weeks (range) 16 (4–69)
  ≥ 16 weeks 9 (30.0%)
  ≥ 24 weeks 5 (16.7%)
  ≥ 32 weeks 5 (16.7%)
Improvement from baseline based on LSS on two consecutive visitsa
 Overall 10 (33.3%)
 Responder, n/N (%) 9/22 (40.9%)
 Non-responder, n/N (%) 1/8 (12.5%)
FFS, % (95% CI)
 Median, months NR (7.8–NR)
 6 months 73 (54–86%)
 12 months 56 (37–72%)
TTNT, % (95% CI)
 Median, months NR (8.9–NR)
 6 months 77 (57–88%)
 12 months 63 (43–77%)
OS, % (95% CI)
 Median, months NR (NR–NR)
 6 months 97 (79–100%)
 12 months 87 (68–95%)

CI confidence interval, DOR duration of response, FFS failure-free-survival, LSS Lee symptom scale, NR not reached, ORR overall response rate, OS overall survival, PR partial response, TTNT time-to-next-treatment, TTR time-to-response

aClinically meaningful improvement in cGVHD symptom burden defined as a decrease ≥ 7 points in LSS score from baseline