Table 2.
Summary of stiripentol effectiveness and tolerability
| Case ID | Concurrent AEDs initially associated with stiripentol | Seizure frequency before the introduction of stiripentol | Maximum dose of stiripentol (mg) | Concomitant AEDs modification with introduction of stiripentol | Seizure frequency while on stiripentol | More than 50% reduction in frequency of GTCs | Reduction of episodes of status epilepticus | Adverse effects | Withdrawal (reason) | Time of exposure to stiripentol until withdrawala or last follow‐up (months) |
|---|---|---|---|---|---|---|---|---|---|---|
| 1 | Clobazam, sodium valproate, zonisamide | 4–17 GTCs per month | 750 | No | Exacerbation of seizures (GTCs on a daily basis, increase of myoclonic seizures) and several episodes of status | No | No | None | Yes (lack of efficacy) | 3a |
| 2 | Clobazam, topiramate | Daily | 1750 | na | Daily | No | na | None | Yes (lack of efficacy) | 72a |
| 3 | Carbamazepine, sodium valproate | 3–4 GTCs monthly, 2–3 focal seizures daily | 2000 | Reduction of sodium valproate (carbamazepine was withdrawn 5 months after introduction of stiripentol) | 1–2 GTCs monthly, 5–8 focal szs monthly | Yes | na | Unsteadiness and sedation (1000 mg daily), lethargy (1500 mg), loss of appetite and weight (1750) | Ongoing | 48 |
| 4 | Levetiracetam, sodium valproate | 3 GTCs monthly, myoclonic seizures daily | na | Withdrawal of oxcarbazepine | Unchanged | no | na | Anorexia and weight loss | Yes (lack of efficacy and adverse effects) | 6a |
| 5 | Sodium valproate, zonisamide | Weekly, clusters every few weeks | 1500 | Reduction of zonisamide and sodium valproate | No clusters, reduction in hospitalisations/emergency room visits | Yes | Yes | Weight loss | Ongoing | 10 |
| 6 | Clobazam, sodium valproate | Weekly | 3000 | na | Initially 1–2 GTCs per month and 45 days free of GTCs, more recently free of GTCs for about 12 months | Yes (seizure‐free) | na | Nausea, unsteadiness, abdominal pain, myelodysplasia (thrombocytopenia and neutropenia) | Ongoing, with dose adjustments, haematological review and regular monitoring | 80 |
| 7 | Sodium valproate | 5–19 seizures per month | 750 | na | 9–33 seizures per month | No | No | None | Yes (lack of efficacy) | 31a |
| 8 | Lamotrigine, levetiracetam | Weekly | 2000 | Withdrawal of carbamazepine | Weekly | No | Yes | None | Ongoing | 34 |
| 9 | Sodium valproate | Focal seizures daily | 1000 | Reduction of sodium valproate | Focal seizures daily | No | na | Increased aggression and diarrhoea | Yes (adverse effects) | 3a |
| 10 | Sodium valproate, topiramate | 4 seizures per month | 750 | No | 11 seizures per month | No | No | Loss of appetite, unsteadiness and increased tremor | Yes (adverse effects) | 4a |
| 11 | Clobazam, levetiracetam, phenytoin, sodium valproate | Daily | 2250 | Reduction of clobazam (phenytoin was withdrawn 8 months after introduction of stiripentol) | Reduction of seizure frequency, mainly of clusters of GTCs | No | Yes | Loss of appetite and weight, tiredness (when on maximum dose) | Ongoing | 42 |
| 12 | Clobazam, topiramate | Daily | 2500 | Withdrawal of topiramate and introduction of sodium valproate | Focal seizures daily, GTCs monthly | Yes | Yes | None | Ongoing | 139 |
| 13 | Levetiracetam, sodium valproate | Weekly | 1000 | Reduction of sodium valproate | Initial slight reduction of seizure frequency | No | Yes | None | Ongoing | 79 |
na, Data not available; GTCs, generalized tonic‐clonic seizures; a, cases for which stiripentol was withdrawn.