Background
Methods
Study design and treatment
Patients
Key inclusion criteria
Key exclusion criteria
Assessments
Statistical analysis
Results
Patients
2 % tofacitinib BID | 1 % tofacitinib BID | Vehicle BID | 2 % tofacitinib QD | 1 % tofacitinib QD | Vehicle QD | |
---|---|---|---|---|---|---|
N = 71 | N = 70 | N = 71 | N = 70 | N = 74 | N = 74 | |
Age (years) | ||||||
Mean (SD) | 47.6 (15.6) | 50.4 (14.5) | 48.8 (15.0) | 50.7 (13.2) | 47.8 (14.0) | 48.9 (13.9) |
Range | 18.0–74.0 | 18.0–77.0 | 21.0–84.0 | 21.0–77.0 | 20.0–85.0 | 20.0–74.0 |
Male (%) | 60.6 | 67.1 | 57.7 | 52.9 | 67.6 | 56.8 |
BMI (kg/m2) | ||||||
Mean (SD) | 31.8 (7.9) | 29.6 (5.6) | 30.2 (8.3) | 28.9 (7.8) | 31.0 (7.2) | 31.1 (6.5) |
Range | 17.2–58.4 | 19.2–51.4 | 17.0–79.8 | 16.4–68.6 | 17.6–50.4 | 20.6–47.4 |
Race (%) | ||||||
White | 93.0 | 85.7 | 94.4 | 90.0 | 91.9 | 97.3 |
Black | 1.4 | 4.3 | 1.4 | 1.4 | 2.7 | 2.7 |
Asian | 4.2 | 7.1 | 2.8 | 5.7 | 2.7 | 0.0 |
Other | 1.4 | 2.9 | 1.4 | 2.9 | 2.7 | 0.0 |
Geographical region (%) | ||||||
Canada | 15.5 | 30.0 | 26.8 | 27.1 | 23.0 | 33.8 |
Denmark | 1.4 | 1.4 | 0.0 | 1.4 | 1.4 | 1.4 |
Poland | 14.1 | 12.9 | 15.5 | 32.9 | 39.2 | 28.4 |
United States | 69.0 | 55.7 | 57.7 | 38.6 | 36.5 | 36.5 |
PGA-C (%) | ||||||
Mild | 28.2 | 30.0 | 29.6 | 32.9 | 27.0 | 27.0 |
Moderate | 71.8 | 70.0 | 70.4 | 67.1 | 73.0 | 73.0 |
PASI score | ||||||
Mean (SD) | 9.5 (5.1) | 8.5 (3.3) | 8.5 (3.6) | 9.9 (4.1) | 10.1 (4.4) | 9.6 (3.8) |
Range | 2.4–29.0 | 3.0–18.0 | 2.4–18.0 | 2.0–19.8 | 2.8–19.8 | 3.2–17.1 |
BSA (%) | ||||||
Mean (SD) | 7.6 (4.6) | 6.4 (3.8) | 6.5 (4.1) | 7.8 (4.3) | 8.4 (4.9) | 8.0 (4.5) |
Range | 2.0–19.0 | 1.5–17.0 | 2.0–20.0 | 2.0–19.0 | 2.4–20.0 | 2.0–19.0 |
ISI scorea | ||||||
Mean (SD) | 5.8 (2.6) | 5.3 (2.4) | 5.4 (2.6) | 6.0 (2.7) | 5.7 (2.9) | 5.4 (3.0) |
Range | 0.0–10.0 | 1.0–10.0 | 0.0–10.0 | 0.0–10.0 | 0.0–10.0 | 0.0–10.0 |
DLQI | ||||||
Mean (SD) | 10.6 (5.9) | 8.6 (5.5) | 9.3 (6.0) | 12.2 (7.4) | 10.9 (7.0) | 10.2 (6.5) |
Range | 0.0–25.0 | 1.0–25.0 | 1.0–24.0 | 1.0–29.0 | 1.0–29.0 | 0.0–26.0 |
Efficacy
Primary endpoints
Secondary endpoints
Endpoint | Week | 2 % tofacitinib BID | 1 % tofacitinib BID | Vehicle BID | 2 % tofacitinib QD | 1 % tofacitinib QD | Vehicle QD |
---|---|---|---|---|---|---|---|
PGA-C, clear (0) or almost clear (1) Responders, % (n/N) Odds ratios (80 % CI) | 8 | 41.8 (26/61) 2.13b (1.29, 3.54) | 20.9 (14/64) 0.79 (0.45, 1.36) | 25.2 (16/55) | 35.9 (23/60) 3.52b (1.97, 6.28) | 23.4 (16/67) 1.92b (1.06, 3.48) | 13.8 (9/58) |
12 | 39.7 (24/58) 1.75b (1.06, 2.90) | 28.4 (18/57) 1.06 (0.62, 1.78) | 27.3 (17/55) | 36.1 (19/53) 1.36 (0.81, 2.28) | 32.9 (22/62) 1.18 (0.71, 1.97) | 29.3 (17/52) | |
PASI75 Responders, % (n/N) Odds ratios (80 % CI) | 8 | 15.2 (10/61) 2.06 (0.96, 4.45) | 9.1 (6/64) 1.15 (0.50, 2.66) | 8.0 (5/55) | 17.9 (11/60) 2.40b (1.14, 5.05) | 7.2 (5/67) 0.85 (0.36, 2.01) | 8.3 (5/58) |
12 | 20.3 (12/58) 1.42 (0.76, 2.64) | 14.4 (9/57) 0.94 (0.48, 1.83) | 15.2 (9/55) | 23.0 (11/53) 3.11b (1.47, 6.55) | 12.1 (8/62) 1.44 (0.65, 3.19) | 8.8 (5/52) | |
PASI, % change from baseline LSM, % (N) Differencea (80 % CI) | 8 | −31.8 (61) −8.3 (−17.3, 0.6) | −26.7 (64) −3.2 (−12.1, 5.7) | −23.5 (55) | −28.3 (60) −9.2b (−17.1, −1.4) | −25.5 (67) −6.4 (−14.1, 1.3) | −19.1 (58) |
12 | −33.9 (58) −6.5 (−17.1, 4.1) | −32.6 (57) −5.2 (−15.7, 5.3) | −27.4 (55) | −33.4 (53) −12.3b (−21.8, −2.8) | −27.0 (62) −5.9 (−15.2, 3.3) | −21.1 (52) | |
BSA, % change from baseline LSM, % (N) Differencea (80 % CI) | 8 | −22.1 (61) −4.2 (−13.1, 4.7) | −20.9 (64) −3.0 (−11.8, 5.8) | −17.9 (55) | −12.5 (60) −8.0 (−16.7, 0.7) | −10.1 (67) −5.7 (−14.2, 2.8) | −4.5 (58) |
12 | −31.2 (58) −4.5 (−13.8, 4.9) | −26.0 (57) 0.8 (−8.5, 10.0) | −26.7 (55) | −22.8 (53) −20.0b (−31.4, −8.7) | −12.3 (62) −9.5 (−20.6, 1.6) | −2.8 (52) |
Patient-reported outcomes
Safety
All adverse events
2 % tofacitinib BID | 1 % tofacitinib BID | Vehicle BID | 2 % tofacitinib QD | 1 % tofacitinib QD | Vehicle QD | |
---|---|---|---|---|---|---|
N = 71 | N = 70 | N = 71 | N = 70 | N = 74 | N = 74 | |
Number of AEs | 47 | 51 | 54 | 66 | 65 | 62 |
Patients with treatment-emergent AEs, n (%) | 30 (42.3) | 30 (42.9) | 28 (39.4) | 34 (48.6) | 28 (37.8) | 40 (54.1) |
Patients with application site AEs, n (%) | 4 (5.6) | 0 (0.0) | 4 (5.6) | 8 (11.4) | 7 (9.5) | 12 (16.2) |
Patients with SAEs, n (%) | 0 (0.0) | 5 (7.1) | 2 (2.8) | 0 (0.0) | 2 (2.7) | 1 (1.4) |
Patients discontinued due to AEs, n (%) | 0 (0.0) | 1 (1.4)a | 4 (5.6) | 6 (8.6) | 3 (4.1) | 7 (9.5) |
Deaths, n (%) | 0 (0.0) | 1 (1.4)a | 0 (0.0) | 0 (0.0) | 0 (0.0) | 0 (0.0) |
Application site adverse events
Laboratory assessments
Criterion, % (n/N) | 2 % tofacitinib BID | 1 % tofacitinib BID | Vehicle BID | 2 % tofacitinib QD | 1 % tofacitinib QD | Vehicle QD |
---|---|---|---|---|---|---|
Any criterion | 2.9 (2/70) | 2.9 (2/70) | 1.4 (1/69) | 5.8 (4/69) | 4.1 (3/73) | 1.4 (1/73) |
Hemoglobina | 1.5 (1/68) | 0.0 (0/70) | 0.0 (0/69) | 0.0 (0/67) | 4.1 (3/73) | 1.4 (1/72) |
Neutrophil countb | 0.0 (0/68) | 0.0 (0/69) | 0.0 (0/69) | 1.5 (1/67) | 0.0 (0/73) | 0.0 (0/72) |
Lymphocyte countc | 0.0 (0/68) | 0.0 (0/70) | 0.0 (0/69) | 0.0 (0/67) | 0.0 (0/73) | 0.0 (0/71) |
Platelet countd | 0.0 (0/68) | 0.0 (0/69) | 0.0 (0/69) | 0.0 (0/67) | 0.0 (0/73) | 0.0 (0/72) |
Serum creatininee | 1.4 (1/69) | 0.0 (0/70) | 0.0 (0/69) | 1.5 (1/67) | 0.0 (0/73) | 0.0 (0/72) |
AST/ALTf | 0.0 (0/69) | 2.9 (2/70) | 1.4 (1/69) | 1.5 (1/67) | 0.0 (0/73) | 0.0 (0/72) |
CPKg | 0.0 (0/69) | 0.0 (0/70) | 1.4 (1/69) | 3.0 (2/67) | 0.0 (0/73) | 0.0 (0/72) |