Table 2.
Summary of efficacy parameters by subgroup
| NNS/CANDLE (N = 5) | SAVI (N = 3) | AGS (N = 1) | ||||||
|---|---|---|---|---|---|---|---|---|
| Parameters | Period | Week | Observed | Change | Observed | Change | Observed | Change |
| Mean DDS, mean (SD) | Pre-treatmenta | 0.64 (0.32) | - | - | ||||
| Baseline | 0 | 0.92 (0.35) | 0.79 (0.28) | 1.25 (NA) | ||||
| Primary treatmentb | 20/32 | 0.70 (0.51) | -0.22 (0.59) | 0.57 (0.23) | -0.21 (0.25) | 1.3 (NA) | 0.07 (NA) | |
| Maintenance treatment | 52 | 0.74 (0.57) | -0.18 (0.64) | 0.52 (0.28) | -0.27 (0.34) | 1.29 (NA) | 0.04 (NA) | |
| Proportion of days with < 0.5 in mean DDS, mean (SD) | Pre-treatmenta | 0.36 (0.24) | - | - | ||||
| Baseline | 0 | 0.0 (0) | 0.23 (0.39) | 0 (NA) | ||||
| Primary treatmentb | 20/32 | 0.40 (0.55) | 0.40 (0.55) | 0.32 (0.56) | 0.10 (0.17) | 0 (NA) | 0.0 (NA) | |
| Maintenance treatment | 52 | 0.36 (0.50) | 0.36 (0.50) | 0.63 (0.55) | 0.41 (0.47) | 0 (NA) | 0.0 (NA) | |
| Physician’s Global Assessment of disease activity, mean (SD) | Pre-treatmenta | 5.45 (1.33) | - | - | ||||
| Baseline | 0 | 5.80 (1.15) | 3.83 (1.26) | 2.50 (NA) | ||||
| Primary treatment | 20/32 | 3.70 (1.10) | -2.10 (0.82) | 2.50 (1.80) | -1.33 (1.76) | 2.0 (NA) | -0.50 (NA) | |
| Maintenance treatment | 52 | 4.20 (2.80) | -1.60 (1.85) | 2.50 (1.80) | -1.33 (1.76) | 1.50 (NA) | -1.0 (NA) | |
| Dose of systemic corticosteroid with prednisone equivalent (mg/kg/day)c | Baseline, mean (SD) | 0 | 0.19 (0.09) | 0.39 (NA) | - | |||
| Primary treatment, mean (SD) | 20/32 | 0.15 (0.15) | -0.04 (0.10) | 0.15 (NA) | -0.24 (NA) | - | - | |
| Primary treatment, % change | -23.5% (41.5%) | -61.2% (NA) | ||||||
| Maintenance treatment, mean (SD) | 52 | 0.17 (0.15) | -0.03 (0.11) | 0.14 (NA) | -0.24 (NA) | - | - | |
| Maintenance treatment, % change | -18.4% (43.4%) | -62.9% (NA) | ||||||
AGS Aicardi-Goutières syndrome, DDS daily diary score, N total number of patients, NA not applicable, NNS/CANDLE Nakajo-Nishimura syndrome/chronic atypical neutrophilic dermatosis with lipodystrophy and elevated temperature, SAVI STING-associated vasculopathy with onset during infancy, STING stimulator of interferon genes
Patients with SAVI and AGS had no pre-treatment scores
aAveraged scores collected during pre-treatment period were used
bWeek 20 for NNS/CANDLE; Week 32 for SAVI and AGS
cPatients on systemic corticosteroid at baseline were included