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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