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Table 2 Changes from baseline to week 16 in primary and secondary efficacy endpoints

From: A multicenter randomized, double-blind, placebo-controlled pilot study to assess the efficacy and safety of riociguat in systemic sclerosis-associated digital ulcers

  Placebo (N = 8) Riociguat (N = 7) Treatment difference (95% CI) p value
Net ulcer burden, LS mean* − 0.98 − 1.22 − 0.24 (− 1.46 to 0.99) 0.706
Patient global assessment for overall disease, LS mean − 1.19 0.31 1.50 (− 1.30 to 4.30) 0.27
Patient assessment, LS mean
 Severity of RP − 1.41 − 3.47 − 2.06 (− 4.63 to 0.51) 0.11
 Severity of DU − 4.00 − 4.63 − 0.63 (− 3.68 to 2.41) 0.66
 Pain during RP attack (0–100) − 7.01 − 0.30 6.71 (− 14.01 to 27.43) 0.49
 Numbness during RP attack (0–100) − 15.44 − 19.73 − 4.28 (− 33.44 to 24.87) 0.75
 Tingling during RP attack (0–100) − 7.49 1.18 8.67 (− 13.75 to 31.09) 0.41
Raynaud’s Condition Score, LS mean − 0.82 − 1.15 − 0.33 (− 2.60 to 1.94) 0.76
Number of Raynaud’s attacks per day, LS mean − 0.96 − 1.24 − 0.28 (− 1.36 to 0.79) 0.57
Duration of Raynaud’s attacks (minutes), LS mean 150.3 − 44.8 − 195.1 (− 683.7 to 293.5) 0.40
Physician global assessment for overall disease, LS mean − 0.66 − 1.17 − 0.51 (− 2.27 to 1.25) 0.54
Physician assessment of the severity of RP, LS mean − 1.86 − 3.00 − 1.15 (− 3.51 to 1.22) 0.32
Physician assessment of severity of DU, LS mean − 3.81 − 3.54 0.27 (− 2.55 to 3.10) 0.84
SHAQ-DI (VAS range 0–150), LS mean
 VAS overall disease − 35.74 − 50.35 − 14.60 (− 45.48 to 16.27) 0.32
 VAS burden of DU − 53.47 − 43.09 10.38 (− 58.03 to 78.79) 0.75
 VAS Raynaud’s − 25.40 − 23.78 1.62 (− 54.56 to 57.80) 0.95
 VAS GI − 7.39 16.72 24.11 (− 25.22 to 73.44) 0.31
 VAS breathing − 12.69 8.35 21.04 (− 8.05 to 50.13) 0.14
PROMIS-29, LS mean
 Anxiety − 1.50 −3.11 − 1.60 (− 9.77 to 6.56) 0.68
 Depression − 0.25 − 3.35 − 3.10 (− 8.65 to 2.45) 0.25
 Fatigue 0.46 0.64 0.19 (− 6.26 to 6.63) 0.95
 Physical function − 2.24 − 2.46 − 0.22 (− 3.96 to 3.53) 0.90
 Sleep disturbance 0.94 − 0.47 − 1.41 (− 4.76 to 1.95) 0.38
 Pain interference − 3.06 − 3.69 − 0.62 (− 6.54 to 5.30) 0.82
 Pain intensity − 1.63 − 2.74 − 1.11 (− 3.59 to 1.37) 0.35
 Ability to participate in social activities − 0.46 − 1.68 − 1.22 (− 4.80 to 2.36) 0.47
HAQ-DI, LS mean
 Overall − 0.06 − 0.01 0.04 (− 0.44 to 0.53) 0.84
 Dressing and grooming 0.05 − 0.16 − 0.21 (− 0.96 to 0.54) 0.55
 Hygiene − 0.27 − 0.39 − 0.11 (− 1.00 to 0.78) 0.79
 Arising 0.02 0.36 0.34 (− 0.41 to 1.09) 0.34
 Reach 0.01 0.37 0.36 (− 0.46 to 1.19) 0.36
 Eating 0.03 − 0.53 − 0.56 (− 1.08 to − 0.03) 0.04
 Grip − 0.18 0.16 0.34 (− 0.37 to 1.04) 0.31
 Walking − 0.12 0.35 0.47 (− 0.18 to 1.12) 0.14
 Common daily activities 0.04 − 0.29 − 0.33 (− 1.17 to 0.51) 0.40
 HDISS-DU, LS mean − 0.32 − 0.47 − 0.14 (− 1.75 to 1.46) 0.85
  1. LS mean least squares mean from an ANCOVA model with treatment and baseline value as covariates, SE standard error, DU digital ulcer, RP Raynaud’s phenomenon, SHAQ-DI Scleroderma Health Assessment Questionnaire Disability Index, VAS visual analog scale, GI gastrointestinal, PROMIS Patient-Reported Outcomes Measures Information System, HAQ-DI Health Assessment Questionnaire Disability Index, HDISS-DU Hand Disability in Systemic Sclerosis Digital Ulcer
  2. *DU net burden is defined as the total number of active and painful indeterminate digital ulcers at an assessment; †Estimates and p-values are from an ANCOVA model, adjusting for baseline values of the outcome