Patient demographics and clinical characteristics
| Variable | Paediatric n=47 | FCAS n=30 | MWS n=103 | NOMID* n=32 | Overall n=166 |
|---|---|---|---|---|---|
| Baseline age, years, n (%) | |||||
| <18 | 47 (100) | 5 (16.7) | 23 (22.3) | 18 (56.3) | 47(28.3) |
| ≥18 | – | 25 (83.3) | 80 (77.7) | 14 (43.7) | 119 (71.7) |
| Sex, n (%) | |||||
| Female | 30 (63.8) | 18 (60.0) | 58 (56.3) | 21 (65.6) | 97 (58.4) |
| Male | 17 (36.2) | 12 (40.0) | 45 (43.7) | 11 (34.4) | 69 (41.6) |
| Age, years | |||||
| Mean (SD) | 10.2 (4.12) | 34.8 (20.94) | 34.3 (17.59) | 17.2 (10.80) | 30.9 (18.43) |
| Cohort, n (%) | |||||
| Patients from phase II study | 5 (10.6) | 1 (3.3) | 21 (20.4) | 2 (6.3) | 24 (14.5) |
| Patients from phase III study | 4 (8.5) | 0 | 29 (28.2) | 4 (12.5) | 33 (19.9) |
| Canakinumab-naive patients | 38 (80.9) | 29 (96.7) | 53 (51.5) | 26 (81.3) | 109 (65.7) |
| Race, n (%) | |||||
| Caucasian | 47 (100) | 30 (100) | 99 (96.1) | 31 (96.9) | 161 (97.0) |
| Others | 0 | 0 | 4 (3.9) | 1 (3.1) | 5 (3.0) |
| Molecular diagnosis of NLRP3 mutation, n (%) | |||||
| Positive | 41 (87.2) | 30 (100) | 100 (97.1) | 26 (81.3) | 156 (94.0) |
| Negative† | 6 (12.8) | 0 | 3 (2.9) | 6 (18.8) | 10 (6.0) |
| Physician's global assessment of disease activity, n (%) | |||||
| Absent | 6 (12.8) | 4 (13.3) | 24 (23.3) | 5 (15.6) | 34 (20.5) |
| Minimal | 10 (21.3) | 6 (20.0) | 24 (23.3) | 8 (25.0) | 38 (22.9) |
| Mild | 17 (36.2) | 6 (20.0) | 32 (31.1) | 9 (28.1) | 47 (28.3) |
| Moderate | 12 (25.5) | 12 (40.0) | 19 (18.4) | 9 (28.1) | 40 (24.1) |
| Severe | 2 (4.3) | 2 (6.7) | 3 (2.9) | 1 (3.1) | 6 (3.6) |
| Missing | 0 | 0 | 1 (1.0) | 0 | 1 (0.6) |
| Assessment of skin disease, n (%) | |||||
| Absent | 16 (34.0) | 7 (23.3) | 59 (57.3) | 11 (34.4) | 78 (47.0) |
| Minimal | 7 (14.9) | 4 (13.3) | 15 (14.6) | 4 (12.5) | 23 (13.9) |
| Mild | 9 (19.1) | 7 (23.3) | 14 (13.6) | 4 (12.5) | 25 (15.1) |
| Moderate | 12 (25.5) | 9 (30.0) | 14 (13.6) | 8 (25.0) | 31 (18.7) |
| Severe | 3 (6.4) | 3 (10.0) | 1 (1.0) | 5 (15.6) | 9 (5.4) |
↵* Including 18 patients with MWS/NOMID overlap.
↵† Patients with either a negative or indeterminate NLRP3 mutation are classified as negative for a molecular diagnosis of NLRP3 mutation.
FCAS, familial cold autoinflammatory syndrome; MWS, Muckle–Wells syndrome; NOMID, neonatal-onset multisystem inflammatory disease.