Table 1.
Review of tolerability and adverse events in pediatric patients treated with rufinamide
| References | Number of patients | Age range (years) median or mean | Study design | Type of population | Adverse events on RUF no of pts (%) most common AEs (%) | Responder rate (percentage of patients with a total seizure reduction of ≥50%) |
|---|---|---|---|---|---|---|
| Glauser et al6 | 138 | 4–37 (12.0) | Double-blind, randomized, placebo-controlled trial | LGS | 41/74 (55.4) somnolence (24.3) and vomiting (21.6) | 31.1 |
| Kluger et al13 | 60 | 1–50 (14.5 ± 11.6) | Retrospective observational study | Focal or generalized refractory epilepsy | 25/60 (41.7) fatigue (18.3) vomiting (13.3) |
46.7 |
| Olson et al8 | 38 | 1.5–23 (7.0) | Retrospective analysis RUF as add-on | Epileptic spasms | 14/38 (37.0) increased seizure frequency (13.1) loss of appetite (7.8) sedation (7.8) |
53.0 |
| Kluger et al19 | 52 | 14.9 ± 12.1 | Long-term extension phase study RUF as adjunctive drug | Focal or generalized childhood-onset refractory epilepsy | 37/60 (51.7) fatigue (18.3) vomiting (15.0) loss of appetite (10.0) |
46.7 > 3 months 26.7 > 18 months |
| Coppola et al15 | 43 | 4–34 (15.0) | Prospective, add-on, open label treatment study | LGS | 10/43 (23.2) vomiting (13.5) irritability (6.9) drowsiness (2.3) rash (2.3) |
60.5 |
| Kluger et al14 | 124 | 4–37 (14.2) | Long-term, open label, extension phase (432 days) | LGS | 87/124 (70.2) vomiting (30.6) pyrexia (25.8) URTI (25.8) somnolence (21.8) |
41.0 |
| Coppola et al7 | 38 | 3–34 (12.5) | Prospective, add-on open label therapy study | Refractory childhood epileptic encephalopathies | 11/38 (39.5) vomiting (28.9) drowsiness (5.3) irritability (5.3) |
39.5 |
| Vendrame et al16 | 77 | 1–27 (12.0) | Retrospective analysis as add-on drug | Focal or generalized epilepsy | 23/77 (29.0) drowsiness (13.0) rash (6.0) anorexia (2.0) |
LGS (38.4) WS (14.3) FCS (83.3) FSS (31.3) |
| Mueller et al11 | 20 | 3–23 (12.2) | Retrospective European multicenter study | Dravet syndrome | 8/20 (40.0) fatigue (10.0) gait disorders (5.0) | 20.0 > 6 months 5.5 > 34 months |
| Von Stupnagel et al9 | 8 | 3.7–20.2 (7.3) | Retrospective analysis | Doose syndrome | 2/8 (25.0) decreased appetite (50.0) sleepiness (12.5) | 87.5 > 3 months 33.0 > 18 months |
| Vendrame et al10 | 5 | 2.1–3.11 (2.6) | Retrospective analysis | Malignant migrating partial seizures in infancy | 3/5 (60.0) vomiting (20.0) loss of appetite (20.0) | 40.0 |
Abbreviations: AEs, adverse events; URTI, upper respiratory tract infections; PLB, placebo; RUF, rufinamide; LGS, Lennox-Gastaut syndrome; WS, West syndrome; FCS, focal cryptogenic seizures; FSS, focal symptomatic seizures.