Table 1 Baseline demographic and clinical characteristics of all of the randomized patients
Characteristics | Double-blind phase | Open-label extension | ||||
|---|---|---|---|---|---|---|
PBO (n = 8) | RIO (n = 9) | All patients (n = 17) | PBO-RIO (n = 6) | RIO-RIO (n = 6) | All patients (n = 12) | |
Age in years, mean (SD) | 61 (17) | 43 (14) | 51 (18) | 61 (20) | 44 (14) | 52 (19) |
Gender, n (%) | ||||||
 Male | 3 (38) | 1 (11) | 4 (24) | 3 (50) | 0 (0) | 3 (25) |
 Female | 5 (63) | 8 (89) | 13 (76) | 3 (50) | 6 (100) | 9 (75) |
Race, n (%) | ||||||
 Caucasian | 7 (88) | 6 (67) | 13 (76) | 5 (83) | 5 (83) | 10 (83) |
 African-American | 1 (13) | 2 (22) | 3 (18) | 1 (17) | 0 (0) | 1 (8) |
 Others | 0 (0) | 1 (11) | 1 (6) | 0 (0) | 1 (17) | 1 (8) |
SSc subset, n (%) | ||||||
 Limited cutaneous SSc | 4 (50) | 5 (56) | 9 (53) | 2 (33) | 4 (67) | 6 (50) |
 Diffuse cutaneous SSc | 4 (50) | 4 (44) | 8 (47) | 4 (67) | 2 (33) | 6 (50) |
Time since SSc diagnosis, in years, mean (SD)†| 15.0 (8.2) | 6.2 (5.8) | 10.4 (8.2) | 14.3 (8.0) | 5.2 (6.0) | 9.7 (8.2) |
Time since first non-RP symptom, in years, mean (SD)††| 17.5 (11.2) | 7.1 (6.0) | 12 (10.1) | 16.9 (12.1) | 5.7 (5.8) | 11.3 (10.8) |
Time since first RP symptom, in years, mean (SD)††| 14.5 (7.9) | 7.5 (6.6) | 11 (7.9) | 13.2 (6.7) | 6.9 (6.9) | 10.1 (7.3) |
Time since first DU,, in years†| 8.0 (6.8) | 5.4 (4.6) | 6.7 (5.7) | 9.8 (7.0) | 3.5 (3.1) | 6.7 (6.1) |
Number of DU, mean (SD)††| 2.5 (1.7) | 2.7 (1.8) | 2.6 (1.7) | 2.7 (1.8) | 1.7 (0.8) | 2.2 (1.5) |
 Number of active DU | 1.4 (1.1) | 1.1 (1.0) | 1.2 (1.0) | 1.2 (1.0) | 0.5 (0.5) | 0.8 (0.8) |
 Number of indeterminate DU | 1.1 (1.4) | 1.6 (1.3) | 1.3 (1.3) | 1.5 (1.4) | 1.2 (1.2) | 1.3 (1.2) |
 Net ulcer burden | 2.5 (2.0) | 2.4 (1.4) | 2.5 (1.7) | 2.7 (2.3) | 1.7 (0.8) | 2.2 (1.7) |
Characteristics of Raynaud’s attacks | ||||||
 Raynaud’s Condition Score (0–10 Likert scale), mean (SD)†| 3.4 (2.2) | 5.4 (1.6) | 4.5 (2.1) | 4.0 (1.8) | 5.1 (1.9) | 4.6 (1.8) |
 Number of Raynaud’s attacks per day, mean (SD)†| 2.2 (1.7) | 4.3 (1.7) | 3.3 (2.0) | 2.4 (1.8) | 3.6 (1.4) | 3.0 (1.7) |
 Pain during a RP attack (0–100 VAS scale), mean (SD)†| 37.2 (24.6) | 54.9 (13.1) | 46.6 (20.7) | 41.7 (23.6) | 53.4 (14.2) | 47.5 (19.5) |
 Numbness during a RP attack (0–100 VAS scale), mean (SD)†| 32.0 (30.2) | 40.5 (15.9) | 36.5 (23.2) | 35.6 (31.4) | 43.7 (16.3) | 39.7 (24.2) |
 Tingling during a RP attack (0–100 VAS scale), mean (SD)†| 26.9 (16.1) | 34.8 (16.6) | 31.1 (16.3) | 29.7 (15.7) | 37.7 (15.9) | 33.7 (15.6) |
 Duration of RP attacks, in minutes, mean (SD)†| 47.9 (51.6) | 101.4 (117.3) | 76.4 (93.8) | 55.0 (52.6) | 112.5 (136.4) | 83.7 (103.0) |
Patient assessment of RP (0–10 Likert scale) | ||||||
 Severity of RP, mean (SD)†| 4.2 (2.7) | 7.1 (1.4) | 5.8 (2.5) | 4.8 (2.5) | 7.0 (1.7) | 5.9 (2.3) |
 Severity of DU, mean (SD)†| 6.7 (1.9) | 8.0 (1.5) | 7.4 (1.8) | 6.3 (1.6) | 8.3 (1.2) | 7.3 (1.7) |
SHAQ: DUs interfere with daily activities in past week (theoretical range, 0–150), median (IQR) | 55 (36–102) | 116 (98–125) | 98 (62–124) | 64.3 (33.8) | 107.2 (21.3) | 85.7 (35.0) |
Physician assessment of RP (0–10 Likert scale) | ||||||
 Severity of RP, mean (SD)†| 5.4 (3.0) | 6.1 (1.0) | 5.8 (2.1) | 6.2 (2.2) | 6.0 (1.3) | 6.1 (1.7) |
 Severity of DU, mean (SD)†| 6.3 (2.6) | 6.4 (1.9) | 6.4 (2.1) | 5.6 (2.5) | 6.7 (2.5) | 6.2 (2.3) |
SSc-related antibodies, n (%)‡ | ||||||
 Anti-centromere B | 3 (38) | 3 (33) | 6 (35) | 2 (33) | 3 (50) | 5 (42) |
 Anti-topoisomerase I | 3 (38) | 2 (22) | 5 (29) | 3 (50) | 0 (0) | 3 (25) |
 Anti-RNA polymerase III | 0 (0) | 1 (11) | 1 (6) | 0 (0) | 1 (17) | 1 (8) |
 Not done | 2 (25) | 3 (33) | 5 (29) | 1 (17) | 2 (33) | 3 (25) |
Baseline use of medications, n (%) | ||||||
 Vasodilators | 1 (13) | 1 (11) | 2 (12) | 0 (0) | 1 (17) | 1 (8) |
 Prednisone | 1 (13) | 0 (0) | 1 (6) | 1 (17) | 0 (0) | 1 (8) |
 Immunosuppressive | 0 (0) | 0 (0) | 0 (0) | 0 (0) | 0 (0) | 0 (0) |