Table 3.
Summary of adverse events during the dose adjustment and primary treatment period, and maintenance period
| Incidence, n (%) | NNS/CANDLE (N = 5) n (%) |
SAVI (N = 3) n (%) |
AGS (N = 1) n (%) |
Total (N = 9) n (%) |
|---|---|---|---|---|
| TEAEa | 5 (100.0) | 3 (100.0) | 1 (100.0) | 9 (100.0) |
| Severe TEAEsb | 2 (40.0) | 1 (33.3) | 0 (0.0) | 3 (33.3) |
| Moderate TEAEsb | 2 (40.0) | 0 (0.0) | 0 (0.0) | 2 (22.2) |
| Mild TEAEsb | 1 (20.0) | 2 (66.7) | 1 (100.0) | 4 (44.4) |
| Deathc | 0 (0.0) | 1 (33.3)c | 0 (0.0) | 1 (11.1) |
| SAE | 2 (40.0) | 1 (33.3)c | 0 (0.0) | 3 (33.3) |
| Discontinuation from study and study treatment due to AE | 1 (20.0) | 1 (33.3)c | 0 (0.0) | 2 (22.2) |
AE adverse event, AGS Aicardi-Goutières Syndrome, n number of patients with at least one adverse event per event type, NNS/CANDLE Nakajo-Nishimura syndrome/chronic atypical neutrophilic dermatosis with lipodystrophy and elevated temperature, SAE serious adverse event, SAVI STING-associated vasculopathy with onset during infancy, STING stimulator of interferon genes, TEAE treatment-emergent adverse event
aPatients may be counted in more than one category
bPatients with multiple occurrences of the same event are counted under the highest severity
cThe death event was included in SAE and discontinuations due to AE