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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