Table 2.
Summary of published pediatric rufinamide studies.
| Reference | Study design | Population | Countable seizure outcome | Adverse events: Total patients, most common |
|---|---|---|---|---|
| Glauser et al. [2008] | Randomized | 74 rufinamide patients | ≥50% decrease: 31.1% | Total 81.1% |
| Double-blind | Age 4–35 years | Seizure-free: none | -Somnolence 24.3% | |
| Placebo-controlled | LGS | -Vomiting 21.6% | ||
| Kluger et al. [2009] | Retrospective | 60 patients | ≥50% decrease 46.7% | Total 58.3% |
| 12-week Observational | Age 1–50 years | Seizure-free 8.3% | -Fatigue 18.3% | |
| Mixed epilepsy types | -Vomiting 13.3% | |||
| -Loss of appetite 10% | ||||
| Coppola et al. [2010] | Prospective | 43 patients | ≥50% decrease: 60.5% | Total 23.2% |
| Open-label | Age 4–34 years | Seizure-free: 9.3% | -Vomiting 13.5% | |
| LGS | -Irritability 6.9% | |||
| -Drowsiness 2.3% | ||||
| Kluger et al. [2010a] | Open-label extension study | 124 patients | ≥50% decrease: | Total 91.1% |
| Age 4–37 years | 41% during last 12 months | -Vomiting 30.6% | ||
| LGS | Seizure-free: none | -Pyrexia 25.8% | ||
| -Somnolence 21% | ||||
| Kluger et al. [2010b] | Retrospective | 52 patients | ≥50% decrease 26.7% | Total 61.6% |
| 18-month Observational | Age 1–50 years | Seizure-free: 1.6% | -Fatigue 18.3% | |
| Mixed epilepsy types | -Vomiting 15% | |||
| -Loss of appetite 10% | ||||
| Vendrame et al. [2010] | Retrospective | 77 patients | ≥50% decrease: 51% | Total 29% |
| Age 1–27 years | Seizure-free: not reported | -Drowsiness 13% | ||
| Mixed epilepsy types | -Rash 6% | |||
| Coppola et al. [2011] | Prospective | 38 patients | ≥50% decrease: 39.5% | Total: 28.9% |
| Open-label | Age 4–34 years | Seizure-free: 2.6% | -Vomiting 13.1% | |
| Non-LGS epileptic encephalopathies | -Irritability 5.3% | |||
| -Drowsiness 5.3% | ||||
| Hausler et al. [2011] | Retrospective | 3 patients | ≥50% decrease: 100% | ‘Transient and mild’ |
| Age 2–4 years | Seizure-free: 66.6% | |||
| EMA | ||||
| Joseph et al. [2011] | Retrospective | 45 patients | ≥50% decrease: 46% | Total: not reported |
| Age 1–20 years | Seizure-free: none | -Vomiting 4.4% | ||
| Mixed epilepsy types | -Loss of appetite 4.4% | |||
| -Agitation 4.4% | ||||
| Mueller et al. [2011] | Retrospective | 20 patients | ≥50% decrease at 6 months: 20% | Total 40% |
| Age 3–23 years | -Decreased appetite 15% | |||
| Dravet syndrome | ≥50% decrease at 18 months: 5% | -Fatigue 10% | ||
| -Behavior change 10% | ||||
| Olson et al. [2011] | Retrospective | 38 patients | ≥50% decrease in spasms: 53% | Total 37% |
| 1–23 years | -Decreased appetite 7.9% | |||
| Epileptic spasms | >99% decrease: 5% | -Sedation 7.9% | ||
| Vendrame et al. [2011] | Retrospective | 5 patients | ≥50% decrease: 40% | Total: 40% |
| Age 2–3 years | -Vomiting 20% | |||
| MMPEI | Seizure-free: none | -Loss of appetite 20% | ||
| Kim et al. [2012] | Prospective | 128 patients | ≥50% decrease: 35.9% | Total 32.8% |
| Open-label | Age 1–19 years | Seizure-free:7.8% | -Fatigue 11.7% | |
| Observational | LGS | -Loss of appetite 7% | ||
| Lee et al. [2012] | Retrospective | 88 patients | ≥50% decrease: 54,6% | Total: 31.8% |
| Age 2–43 years | Seizure-free: 2.3% | - Loss of appetite 8% | ||
| Mixed epilepsy types | - Somnolence 6.8% | |||
| Moavero et al. [2012] | Prospective | 70 patients | ≥50% decrease: 38.5% | Total 24.3% |
| Open-label | Age 3–21 years | Seizure-free: 4.3% | - Drowsiness 22.8% | |
| Partial-onset seizures | - Vomiting 10% | |||
| von Stulpnagel et al. [2012] | Retrospective | 8 patients | ≥50% decrease: 75% at 6 months, 62.5% at 12 months | Total 25% |
| Age 3–20 years | -Sleepiness 12.5% | |||
| Doose syndrome | Seizure-free: none | -Decreased appetite 12.5% |
EMA, epilepsy with myoclonic absences; LGS, Lennox–Gastaut syndrome; MMPEI, malignant migrating partial epilepsy of infancy.