Table 2.
Efficacy outcomes at week 16 in patients in the full analysis set and Investigator’s Global Assessment (IGA) > 1 subgroup
| Full analysis set (n = 251) | IGA > 1 subgroup (n = 214) | |||||
|---|---|---|---|---|---|---|
| Placebo (n = 85) | Dupilumab 300 mg q4w (n = 84) |
Dupilumab 200 or 300 mg q2w (n = 82) | Placebo (n = 83) | Dupilumab 300 mg q4w (n = 69) |
Dupilumab 200 or 300 mg q2w (n = 62) | |
| EASI LS mean change from baseline (SE) | − 9.2 (1.8) |
− 22.6 (1.5) p < 0.0001 |
− 22.2 (1.3) p < 0.0001 |
− 8.8 (1.9) |
− 21.9 (1.7) p < 0.0001 |
− 19.7 (1.6) p < 0.0001 |
| EASI LS mean percent change from baseline (SE) | − 23.6 (5.5) |
− 64.8 (4.5) p < 0.0001 |
− 65.9 (4.0) p < 0.0001 |
−20.7 (5.6) |
− 58.4 (5.5) p < 0.0001 |
− 55.0 (4.9) p < 0.0001 |
| EASI-50, n (%) | 11 (13) |
46 (55) p < 0.0001 |
50 (61) p < 0.0001 |
9 (11) |
31 (45) p < 0.0001 |
30 (48) p < 0.0001 |
| EASI-75, n (%) | 7 (8) |
32 (38) p < 0.0001 |
34 (41) p < 0.0001 |
5 (6) |
17 (25) p = 0.0013 |
14 (23) p = 0.0047 |
| EASI ≤ 7 at week 16, n (%) | 7 (8) |
28 (33) p < 0.0001 |
34 (41) p < 0.0001 |
5 (6) |
13 (19) p = 0.0049 |
14 (23) p = 0.0034 |
| SCORAD total score LS mean change from baseline (SE) | − 13.2 (2.5) |
− 33.2 (2.2) p < 0.0001 |
− 35.8 (2.2) p < 0.0001 |
− 12.4 (2.4) |
− 29.0 (2.3) p < 0.0001 |
− 29.9 (2.4) p < 0.0001 |
| SCORAD total score LS mean percent change from baseline (SE) | − 17.6 (3.8) |
− 47.5 (3.2) p < 0.0001 |
− 51.6 (3.2) p < 0.0001 |
− 15.8 (3.5) |
−39.4 (3.4) p < 0.0001 |
− 41.3 (3.5) p < 0.0001 |
| BSA LS mean change from baseline (SE) | − 11.7 (2.7) |
−33.4 (2.3) p < 0.0001 |
− 30.1 (2.3) p < 0.0001 |
− 10.8 (2.7) |
− 31.3 (2.7) p < 0.0001 |
− 24.5 (2.8) p = 0.0002 |
| Peak Pruritus NRS score LS mean change from baseline (SE) | − 1.5 (0.3) |
− 3.4 (0.3) p < 0.0001 |
− 3.7 (0.3) p < 0.0001 |
− 1.5 (0.3) |
− 3.2 (0.3) p < 0.0001 |
− 3.5 (0.3) p < 0.0001 |
| Peak Pruritus NRS score LS mean percent change from baseline (SE) | − 19.0 (4.1) |
− 45.5 (3.5) p < 0.0001 |
− 47.9 (3.4) p < 0.0001 |
− 17.4 (4.2) |
− 41.2 (4.1) p < 0.0001 |
− 44.2 (4.0) p < 0.0001 |
| Peak Pruritus NRS ≥ 3-point improvement from baseline, n/N (%) | 8/85 (9) |
32/83 (39) p < 0.0001 |
40/82 (49) p < 0.0001 |
6/83 (7.2) |
21/69 (30.4) p = 0.0001 |
27/62 (43.5) p < 0.0001 |
| Peak Pruritus NRS ≥ 4-point improvement from baseline, n/N (%) | 4/84 (5) |
22/83 (27) p = 0.0001 |
30/82 (37) p < 0.0001 |
3/82 (4) |
13/69 (19) p = 0.0025 |
21/62 (34) p < 0.0001 |
| SCORAD—Pruritus VAS score LS mean change from baseline (SE) | − 2.1 (0.4) |
− 4.0 (0.3) p = 0.0002 |
− 4.4 (0.3) p < 0.0001 |
− 2.0 (0.4) |
− 3.4 (0.4) p = 0.0124 |
− 3.8 (0.4) p = 0.0012 |
| SCORAD—Sleep VAS score LS mean change from baseline (SE) | − 1.1 (0.4) |
− 3.0 (0.3) p = 0.0001 |
− 3.6 (0.3) p < 0.0001 |
− 1.2 (0.4) |
− 2.7 (0.4) p = 0.0071 |
− 3.4 (0.4) p < 0.0001 |
| POEM score LS mean change from baseline (SE) | − 3.8 (1.0) |
− 9.5 (0.9) p < 0.0001 |
− 10.1 (0.8) p < 0.0001 |
− 3.5 (1.0) |
− 8.4 (1.0) p = 0.0005 |
− 8.5 (0.9) p = 0.0001 |
| POEM ≥ 6-point improvement from baseline, n/N (%) | 8/84 (10) |
39/84 (46) p < 0.0001 |
52/82 (63) p < 0.0001 |
7/82 (9) |
27/69 (39) p < 0.0001 |
33/62 (53) p < 0.0001 |
| CDLQI LS mean change from baseline (SE) | − 5.1 (0.6) |
− 8.8 (0.5) p < 0.0001 |
− 8.5 (0.5) p < 0.0001 |
− 5.6 (0.7) |
− 8.5 (0.6) p = 0.0022 |
− 8.4 (0.6) p = 0.0023 |
| CDLQI ≥ 6-point improvement from baseline, n/N (%) | 15/76 (20) |
42/71 (59) p < 0.0001 |
43/71 (61) p < 0.0001 |
14/74 (19) |
30/59 (51) p = 0.0002 |
32/56 (57) p < 0.0001 |
| CDLQI ≤ 6 at week 16, n/N (%) | 13/73 (18) |
32/69 (46) p = 0.0003 |
36/69 (52) p < 0.0001 |
12/71 (17) |
21/57 (37) p = 0.0104 |
25/56 (45) p = 0.0007 |
| HADS total score LS mean change from baseline (SE) | − 2.5 (0.8) |
− 5.2 (0.7) p = 0.0133 |
− 3.8 (0.7) p = 0.2203 |
− 2.3 (0.8) |
− 4.2 (0.8) p = 0.0939 |
− 3.7 (0.8) p = 0.2076 |
| HADS-A score LS mean change from baseline (SE) | − 1.6 (0.5) |
− 2.7 (0.5) p = 0.1229 |
− 2.3 (0.4) p = 0.2980 |
− 1.5 (0.5) |
− 1.9 (0.5) p = 0.5757 |
− 2.2 (0.5) p = 0.3043 |
| HADS-D score LS mean change from baseline (SE) | − 0.8 (0.4) |
− 2.4 (0.4) p = 0.0016 |
− 1.4 (0.3) p = 0.1691 |
− 0.7 (0.4) |
− 2.2 (0.4) p = 0.0076 |
− 1.4 (0.4) p = 0.1892 |
| PGADS “no” or “mild” symptoms, n (%) | 11 (13) |
33 (39) p < 0.0001 |
42 (51) p < 0.0001 |
10 (12) |
19 (28) p = 0.0149 |
26 (42) p < 0.0001 |
| PGADS “moderate” symptoms, n (%) | 9 (11) |
15 (18) p = 0.1703 |
19 (23) p = 0.0304 |
8 (10) | 14 (20) | 15 (24) |
| PGADS “severe” symptoms, n (%) | 10 (12) |
6 (7) p = 0.3007 |
6 (7) p = 0.3070 |
10 (12) | 6 (9) | 6 (10) |
| PGADS “very severe” symptoms, n (%) | 55 (65) | 30 (36) | 15 (18) | 55 (66) | 30 (43) | 15 (24) |
| Use of ≥ 1 rescue medication, n (%) | 50 (59) | 27 (32) | 17 (21) | 50 (60) | 27 (39) | 17 (27) |
| Use of ≥ 1 systemic rescue medication, n (%) | 8 (9) | 0 | 1 (1) | 8 (10) | 0 | 2 (3) |
AD atopic dermatitis, BMI body mass index (calculated as weight in kilograms divided by height in meters squared), BSA body surface area, CDLQI Children’s Dermatology Life Quality Index, EASI Eczema Area and Severity Index, FAS full analysis set, HADS Hospital Anxiety and Depression Scale, HADS-A Hospital Anxiety and Depression Scale—Anxiety, HADS-D Hospital Anxiety and Depression Scale—Depression, LS least-squares, N number of patients with baseline Peak Pruritus NRS score ≥ 3 or ≥ 4, POEM score ≥ 6, or CDLQI ≥ 6, NRS Numerical Rating Scale, PGADS Patient Global Assessment of Disease Severity, POEM Patient-Oriented Eczema Measure, q2w every 2 weeks, q4w every 4 weeks, SCORAD SCORing Atopic Dermatitis, SE standard error, TARC thymus and activation-regulated chemokine, VAS visual analog scale
FAS data of the pre-specified endpoints also reported by Simpson et al. (2019) [30]
For PGADS, values after the first rescue treatment were set to censor/missing, and all missing data were imputed to the worst category