Summary of efficacy outcomes at week 24 in the enriched population
| Placebo (n=33) | 10 mg (n=30) | 50 mg (n=38) | |
|---|---|---|---|
| SRI response rate, n/N (%)* | 8/30 (27.7) | 15/21 (73.1) | 12/28 (43.1) |
| OR vs placebo (90% CI) | 7.09 (2.11 to 23.85) | 1.98 (0.67 to 5.86) | |
| p Value | 0.004 | 0.151 | |
| BICLA response rate, n/N (%)* | 6/30 (19.7) | 12/21 (55.7) | 10/28 (34.7) |
| OR vs placebo (90% CI) | 5.11 (1.56 to 16.72) | 2.16 (0.71 to 6.59) | |
| p Value | 0.012 | 0.127 | |
| Patients with disease flares, n (%) | |||
| BILAG flares (new BILAG A or two new BILAG B organ domain scores) | 5/33 (15.2) | 0/30 (0.0)† | 0/38 (0.0)† |
| Severe SFI flares | 8/33 (24.2) | 0/28 (0.0)† | 2/35 (5.7)† |
| Placebo (n=33) | 10 mg (n=28) | 50 mg (n=37) | |
| LS mean change in SF-36 score from baseline (SE) | |||
| PCS score | 2.80 (1.4) | 7.60 (1.5)‡ | 5.07 (1.3) |
| MCS score | 2.04 (1.6) | 2.69 (1.7) | 1.56 (1.5) |
| Physical functioning | 4.62 (4.0) | 15.09 (4.4) | 12.44 (3.8) |
| Role physical | 8.44 (3.9) | 14.63 (4.3) | 13.83 (3.7) |
| Body pain | 6.43 (3.9) | 17.79 (4.2) | 11.56 (3.7) |
| General health | 6.80 (2.7) | 14.13 (3.0) | 4.59 (2.6) |
| Vitality | 3.45 (3.5) | 12.05 (3.8) | 4.19 (3.3) |
| Social functioning | 5.80 (4.1) | 10.02 (4.5) | 9.54 (3.9) |
| Role emotional | 3.62 (3.7) | 5.89 (4.1) | 8.98 (3.6) |
| Mental health | 4.20 (3.0) | 6.78 (3.3) | 2.75 (2.9) |
| LS mean change in EQ-5D VAS score from baseline (SE) | 2.30 (3.2) | 11.47 (3.5) | 6.10 (3.0) |
| LS mean change in FACIT-Fatigue score from baseline (SE) | 1.16 (1.8) | 5.28 (2.0) | 3.68 (1.8) |
Bold italic text denotes changes that were greater than the minimum clinically important difference (SF-36 PCS and MCS >2.5-point change from baseline;17 SF-36 domain scores >5-point change from baseline;17 EQ-5D >10-point change from baseline; FACIT-Fatigue score >4-point change from baseline).
*Estimates from generalised linear mixed model. n/N represents the observed number of responders (n) for patients who completed through week 24 (N). Patients who discontinued from the study were not included in the denominator. Estimates from the generalised linear mixed model include all available data from completed and discontinued patients.
†p<0.01 for combined 10 mg and 50 mg groups versus placebo (Fisher's exact test).
‡p<0.05 vs placebo.
BICLA, BILAG-based Composite Lupus Assessment; BILAG, British Isles Lupus Assessment Group; EQ-5D, European Quality of Life 5 Dimensions; FACIT, Functional Assessment of Chronic Illness Therapy; LS, least squares; MCS, mental component summary; PCS, physical component summary; SF-36, 36-item Short Form Health Survey; SFI, modified Safety of Estrogens in Lupus Erythematosus: National Assessment (SELENA)-Systemic Lupus Erythematosus Disease Activity Index (SLEDAI) Flare Index; SRI, Systemic Lupus Erythematosus Responder Index; VAS, visual analogue scale.