Table 1.
Summary of demographic and baseline characteristics
| Parameter | NNS/CANDLE (N = 5) |
SAVI (N = 3) |
AGS (N = 1) |
Total (N = 9) |
|---|---|---|---|---|
| Age, years | ||||
| Mean (SD) | 39.2 (13.8) | 9.7 (9.8) | 9.0 (NA) | 26.0 (19.1) |
| Range (minimum, maximum) | 15–48 | 4–21 | 9–9 | 4–48 |
| Sex, n (%) | ||||
| Female | 1 (20.0) | 3 (100.0) | 1 (100.0) | 5 (55.6) |
| eGFR (mL/min/1.73m2) | ||||
| Mean (SD) | 124.7 (19.3) | 109.4 (22.5) | 106.7 (NA) | 117.6 (19.6) |
| Weight, kg | ||||
| Mean (SD) | 45.8 (5.9) | 22.7 (17.3) | 14.30 (NA) | 34.6 (16.6) |
| Weight, n (%) | ||||
| ≥ 10 to < 20 kg | 0 | 2 (66.7) | 1 (100.0) | 3 (33.3) |
| ≥ 40 to < 50 kg | 4 (80.0) | 1 (33.3) | 0 | 5 (55.6) |
| ≥ 50 to < 60 kg | 1 (20.0) | 0 | 0 | 1 (11.1) |
| Height (cm) | ||||
| Mean (SD) | 150.9 (11.2) | 112.9 (31.2) | 112.2 (NA) | 133.9 (26.6) |
| BMI (kg/m2) | ||||
| Mean (SD) | 20.2 (2.3) | 15.7 (3.2) | 11.4 (NA) | 17.7 (4.0) |
| Corticosteroid use | ||||
| n (%) | 5 (100.0) | 1 (33.3) | 0 | 6 (66.7) |
| Corticosteroid total daily dose (mg)a | ||||
| Mean (SD) | 8.9 (4.3) | 4 (NA) | 0 | 8.08 (4.3) |
| Corticosteroid dose per weight (mg/kg)a | ||||
| Mean (SD) | 0.2 (0.1) | 0.4 (NA) | 0 | 0.2 (0.1) |
AGS Aicardi-Goutières syndrome, BMI body mass index, eGFR estimated glomerular filtration rate, n number of patients with non-missing data, N number of patients in the safety population within each disease subpopulation, 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, SD standard deviation, STING stimulator of interferon genes
aThe mean corticosteroid doses are reported as prednisone equivalent doses in patients taking corticosteroids