Table 2

Summary of efficacy outcomes at week 24

Placebo (n=45)10 mg (n=45)50 mg (n=47)
SRI response rate, n/N (%)*16/42 (40.1)20/35 (59.9)14/36 (39.2)
 OR vs placebo (90% CI)2.2 (0.89 to 5.62)0.96 (0.38 to 2.41)
 p Value0.0760.528
BICLA response rate, n/N (%)*11/42 (25.1)18/35 (49.7)15/36 (40.5)
 OR vs placebo (90% CI)2.95 (1.18 to 7.41)2.03 (0.82 to 5.06)
 p Value0.0260.10
Treatment failures, n/N (%)11/45 (24.4)1/45 (2.2)4/47 (8.5)
 p Value0.0050.031
Patients with disease flares, n/N (%)
 Severe BILAG flares (new BILAG A or two new BILAG B organ domain scores)5/45 (11.1)2/43 (4.7)0/44 (0.0)
 Severe SFI flares8/45 (17.8)0/43 (0.0)†2/44 (4.5)
Proportion of patients with reductions in anti-dsDNA from baseline, n/N (%)‡
 ≥10% reduction7/17 (41.2)9/15 (60.0)11/18 (61.1)
 ≥30% reduction3/16 (18.8)7/14 (50.0)6/18 (33.3)
 ≥50% reduction1/15 (6.7)4/14 (28.6)1/16 (6.3)
Mean change in C3 concentration from baseline, g/L (SD)§−0.021 (0.176)−0.100 (0.163)−0.169 (0.161)
Mean change in C4 concentration from baseline, g/L (SD)§0.0002 (0.0417)−0.0096 (0.0516)−0.0551 (0.0491)
Patients whose corticosteroid dose was reduced by ≥25% from baseline, and to ≤7.5 mg/day, for at least one visit up to and including week 24, n/N (%)¶2/23 (8.7)4/15 (26.7)5/24 (20.8)
Placebo (n=45)10 mg (n=43)50 mg (n=46)
LS mean change in SF-36 score from baseline (SE)
 PCS score3.08 (1.2)6.04 (1.2)5.67 (1.2)
 MCS score2.95 (1.4)2.94 (1.4)2.12 (1.4)
 Physical functioning4.87 (3.4)10.96 (3.5)12.49 (3.4)
 Role physical10.62 (3.4)15.28 (3.5)16.06 (3.3)
 Body pain7.92 (3.3)13.38 (3.3)13.94 (3.2)
 General health7.01 (2.5)12.53 (2.6)5.15 (2.5)
 Vitality6.42 (3.0)10.30 (3.1)7.45 (3.0)
 Social functioning7.62 (3.5)6.78 (3.5)9.58 (3.4)
 Role emotional6.49 (3.4)6.65 (3.5)10.80 (3.3)
 Mental health4.90 (2.5)6.96 (2.6)2.72 (2.5)
LS mean change in EQ-5D VAS score from baseline (SE)5.99 (2.8)10.30 (2.9)6.18 (2.7)
LS mean change in FACIT-Fatigue score from baseline (SE)2.82 (1.5)4.43 (1.6)3.47 (1.5)
  • 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).

  • ‡Patients with baseline anti-dsDNA above 31 IU/mL were included in the ≥10% reduction analysis (n=50); patients with baseline anti-dsDNA above 40 IU/mL were included in the ≥30% reduction analysis (n=48); patients with baseline anti-dsDNA above 54 IU/mL were included in the ≥50% reduction analysis (n=45).

  • §Patients with complement data were included in the analyses of changes in C3 and C4 concentrations (placebo, n=41; 10 mg, n=39; 50 mg, n=38).

  • ¶Patients with a baseline corticosteroid dose >7.5 mg/day were included in the corticosteroid reduction analysis (n=62).

  • BICLA, BILAG-based Composite Lupus Assessment; BILAG, British Isles Lupus Assessment Group; dsDNA, double-stranded DNA; 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.