| |
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)
|
- * 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
