Abstract
Background Studies on the efficacy and tolerability of rufinamide in infants and young children are scarce. Here we report on an open, retrospective, and pragmatic study about safety and efficacy of rufinamide in children aged less than four years, in terms of seizures types and epilepsy syndromes. Methods Forty children (mean age 39.5 months; range 22-48) were enrolled in the study. The mean follow-up period was 12.2 months (range 5-21). Rufinamide was initiated at a mean age of 26.7 months (range 12-42). Final rufinamide mean dosage was 31.5 mg/kg/day if associated with valproic acid and 44.2 mg/kg/day if not. Results The highest seizure reduction rate was observed in the epileptic spasms (46%) and drop attacks (42%) groups. Seizure reduction was also observed in tonic seizures (35%) and in the focal seizure (30%) groups. In terms of epilepsy syndrome, rufinamide was effective in Lennox-Gastaut syndrome. Results were very poor for those affected by Dravet's syndrome. Globally, responder rate was 27.5%, including two (5%) patients seizure-free. Adverse reactions occurred in 37.5% of children and were mainly represented by vomiting, drowsiness, irritability, and anorexia. Discontinuation rate due to treatment-emergent adverse events was 15%. Conclusion The present study concludes that rufinamide may be a safe and effective drug for a broad range of seizures and epilepsy syndromes in infants and young children and represents a valid therapeutic option in this population.
| Original language | English |
|---|---|
| Pages (from-to) | 641-645 |
| Number of pages | 5 |
| Journal | European Journal of Paediatric Neurology |
| Volume | 18 |
| Issue number | 5 |
| DOIs | |
| Publication status | Published - 2014 |
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Keywords
- Antiepileptic drugs
- Drug-resistant epilepsy
- Epilepsy syndromes
- Epileptic encephalopathy
- Pediatrics
ASJC Scopus subject areas
- Clinical Neurology
- Pediatrics, Perinatology, and Child Health
- Medicine(all)
Cite this
Efficacy and safety of rufinamide in children under four years of age with drug-resistant epilepsies. / Grosso, Salvatore; Coppola, Giangennaro; Dontin, Serena Donetti; Gobbi, Giuseppe; Pruna, Dario; Accorsi, Patrizia; Verrotti, Alberto; Parisi, Pasquale; Balestri, Paolo.
In: European Journal of Paediatric Neurology, Vol. 18, No. 5, 2014, p. 641-645.Research output: Contribution to journal › Article
}
TY - JOUR
T1 - Efficacy and safety of rufinamide in children under four years of age with drug-resistant epilepsies
AU - Grosso, Salvatore
AU - Coppola, Giangennaro
AU - Dontin, Serena Donetti
AU - Gobbi, Giuseppe
AU - Pruna, Dario
AU - Accorsi, Patrizia
AU - Verrotti, Alberto
AU - Parisi, Pasquale
AU - Balestri, Paolo
PY - 2014
Y1 - 2014
N2 - Background Studies on the efficacy and tolerability of rufinamide in infants and young children are scarce. Here we report on an open, retrospective, and pragmatic study about safety and efficacy of rufinamide in children aged less than four years, in terms of seizures types and epilepsy syndromes. Methods Forty children (mean age 39.5 months; range 22-48) were enrolled in the study. The mean follow-up period was 12.2 months (range 5-21). Rufinamide was initiated at a mean age of 26.7 months (range 12-42). Final rufinamide mean dosage was 31.5 mg/kg/day if associated with valproic acid and 44.2 mg/kg/day if not. Results The highest seizure reduction rate was observed in the epileptic spasms (46%) and drop attacks (42%) groups. Seizure reduction was also observed in tonic seizures (35%) and in the focal seizure (30%) groups. In terms of epilepsy syndrome, rufinamide was effective in Lennox-Gastaut syndrome. Results were very poor for those affected by Dravet's syndrome. Globally, responder rate was 27.5%, including two (5%) patients seizure-free. Adverse reactions occurred in 37.5% of children and were mainly represented by vomiting, drowsiness, irritability, and anorexia. Discontinuation rate due to treatment-emergent adverse events was 15%. Conclusion The present study concludes that rufinamide may be a safe and effective drug for a broad range of seizures and epilepsy syndromes in infants and young children and represents a valid therapeutic option in this population.
AB - Background Studies on the efficacy and tolerability of rufinamide in infants and young children are scarce. Here we report on an open, retrospective, and pragmatic study about safety and efficacy of rufinamide in children aged less than four years, in terms of seizures types and epilepsy syndromes. Methods Forty children (mean age 39.5 months; range 22-48) were enrolled in the study. The mean follow-up period was 12.2 months (range 5-21). Rufinamide was initiated at a mean age of 26.7 months (range 12-42). Final rufinamide mean dosage was 31.5 mg/kg/day if associated with valproic acid and 44.2 mg/kg/day if not. Results The highest seizure reduction rate was observed in the epileptic spasms (46%) and drop attacks (42%) groups. Seizure reduction was also observed in tonic seizures (35%) and in the focal seizure (30%) groups. In terms of epilepsy syndrome, rufinamide was effective in Lennox-Gastaut syndrome. Results were very poor for those affected by Dravet's syndrome. Globally, responder rate was 27.5%, including two (5%) patients seizure-free. Adverse reactions occurred in 37.5% of children and were mainly represented by vomiting, drowsiness, irritability, and anorexia. Discontinuation rate due to treatment-emergent adverse events was 15%. Conclusion The present study concludes that rufinamide may be a safe and effective drug for a broad range of seizures and epilepsy syndromes in infants and young children and represents a valid therapeutic option in this population.
KW - Antiepileptic drugs
KW - Drug-resistant epilepsy
KW - Epilepsy syndromes
KW - Epileptic encephalopathy
KW - Pediatrics
UR - http://www.scopus.com/inward/record.url?scp=84906938103&partnerID=8YFLogxK
UR - http://www.scopus.com/inward/citedby.url?scp=84906938103&partnerID=8YFLogxK
U2 - 10.1016/j.ejpn.2014.05.001
DO - 10.1016/j.ejpn.2014.05.001
M3 - Article
C2 - 24912730
AN - SCOPUS:84906938103
VL - 18
SP - 641
EP - 645
JO - European Journal of Paediatric Neurology
JF - European Journal of Paediatric Neurology
SN - 1090-3798
IS - 5
ER -