Skip to main content

Table 5 Summary of changes in the efficacy measures at the end of open-label extension

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-riociguat (N = 6) Riociguat-riociguat (N = 6)
Baseline* Week 16 OLE Change from baseline Baseline* Week 16 OLE Change from baseline
Net ulcer burden 2.7 (2.2) 1.5 (1.1) − 1.2 (2.3) 1.67 (0.8) 0 (0) − 1.67 (0.8)
Patient global assessment for overall disease 4.3 (2.2) 3.3 (1.9) − 1.0 (2.3) 4.2 (2.9) 3.5 (1.6) − 0.7 (2.6)
Patient assessment
 Severity of RP 4.8 (2.5) 3 (1.9) − 1.8 (2.3) 7.0 (1.7) 3.0 (2.7) − 4.0 (3.3)
 Severity of DU 6.3 (1.6) 2.8 (1.5) − 3.5 (2.6) 8.3 (1.2) 1.0 (0.9) − 7.3 (1.9)
Physician global assessment for overall disease 4.8 (1.8) 5.2 (1.7) 0.6 (2.9) 6 (1.7) 3.2 (1.7) − 1.8 (2.7)
Physician assessment of severity of RP 6.2 (2.2) 4.7 (2.2) − 1.4 (2.4) 1.3 (5.5) 3.0 (2.2) − 3.0 (1.3)
Physician assessment of severity of DU 5.6 (2.5) 2.8 (2.1) − 2.2 (2.2) 6.7 (2.2) 0.5 (0.8) − 6.2 (2.1)
SHAQ-DI (VAS range 0–150)
 VAS overall disease 94.2 (20.1) 65.5 (24.1) − 28.7 (26.9) 100.5 (24.4) 42.8 (25.8) − 57.7 (32.7)
 VAS burden of DU 64.3 (33.8) 39.2 (35.2) − 25.2 (42.1) 107.2 (21.3) 10.0 (6.3) − 97.2 (24.2)
 VAS Raynaud’s 56.8 (36.7) 37.8 (39.7) − 19.0 (47.5) 69.7 (38.3) 36.7 (40.2) − 33.0 (46.5)
 VAS GI 6.8 (7.5) 46.2 (47.8) 39.3 (47.7) 44.5 (29.9) 45.5 (33.6) 1.0 (28.6)
 VAS breathing 18.5 (29.4) 20.5 (27.5) 2.0 (14.7) 40.3 (22.6) 33.7 (27.2) − 6.7 (24.9)
PROMIS-29
 Anxiety 51.5 (9.8) 51.9 (9.5) 3.2 (9.4) 61.4 (14.3) 56.7 (9.4) − 4.8 (11.4)
 Depression 51.2 (8.8) 51.4 (8.7) 0.2 (4.2) 57.4 (7.1) 55.7 (8.3) − 1.7 (7.8)
 Fatigue 44.7 (8.7) 54.0 (3.8) 9.2 (6.4) 57.6 (6.7) 54.7 (7.0) − 2.9 (9.5)
 Physical function 35.3 (5.4) 33.8 (3.4) − 1.5 (4.1) 34.7 (3.3) 32.0 (3.6) − 2.7 (1.9)
 Sleep disturbance 51.1 (5.1) 49.8 (4.3) − 1.3 (7.9) 52.3 (2.0) 49.9 (1.9) − 2.4 (3.2)
 Pain interference 60.3 (3.4) 49.6 (9.0) − 10.6 (8.3) 63.8 (6.1) 58.5 (3.1) − 5.3 (7.5)
 Pain intensity 6.5 (1.5) 3.7 (2.1) − 2.8 (3.0) 8.7 (0.8) 5.0 (1.1) − 3.7 (1.0)
 Ability to participate in social activities 41.1 (3.0) 40.7 (5.0) − 0.4 (7.2) 45.7 (7.5) 41.8 (3.6) − 4.0 (5.7)
HAQ-DI 1.3 (0.7) 1.5 (0.5) 0.9 (0.5) 1.2 (0.6) 0.9 (0.5) − 0.3 (0.5)
HDISS-DU 5.2 (1.7) 5.5 (1.8) 0.3 (1.0) 4.8 (2.3) 4.2 (1.9) − 0.7 (2.0)
  1. * Baseline data is at the start of double blind study. 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