Efficacy and safety of rufinamide in children under four years of age with drug-resistant epilepsies

Salvatore Grosso, Giangennaro Coppola, Serena Donetti Dontin, Giuseppe Gobbi, Dario Pruna, Patrizia Accorsi, Alberto Verrotti, Pasquale Parisi, Paolo Balestri

Research output: Contribution to journalArticle

13 Citations (Scopus)

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 languageEnglish
Pages (from-to)641-645
Number of pages5
JournalEuropean Journal of Paediatric Neurology
Volume18
Issue number5
DOIs
Publication statusPublished - 2014

Fingerprint

Seizures
Safety
Epilepsy
Myoclonic Epilepsy
Sleep Stages
Spasm
Syncope
Valproic Acid
Anorexia
rufinamide
Drug Resistant Epilepsy
Vomiting
Retrospective Studies
Therapeutics
Pharmaceutical Preparations
Population

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 journalArticle

Grosso, S, Coppola, G, Dontin, SD, Gobbi, G, Pruna, D, Accorsi, P, Verrotti, A, Parisi, P & Balestri, P 2014, 'Efficacy and safety of rufinamide in children under four years of age with drug-resistant epilepsies', European Journal of Paediatric Neurology, vol. 18, no. 5, pp. 641-645. https://doi.org/10.1016/j.ejpn.2014.05.001
Grosso, Salvatore ; Coppola, Giangennaro ; Dontin, Serena Donetti ; Gobbi, Giuseppe ; Pruna, Dario ; Accorsi, Patrizia ; Verrotti, Alberto ; Parisi, Pasquale ; Balestri, Paolo. / Efficacy and safety of rufinamide in children under four years of age with drug-resistant epilepsies. In: European Journal of Paediatric Neurology. 2014 ; Vol. 18, No. 5. pp. 641-645.
@article{df59c04df874408bb1ae58ec5bf86b44,
title = "Efficacy and safety of rufinamide in children under four years of age with drug-resistant epilepsies",
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.",
keywords = "Antiepileptic drugs, Drug-resistant epilepsy, Epilepsy syndromes, Epileptic encephalopathy, Pediatrics",
author = "Salvatore Grosso and Giangennaro Coppola and Dontin, {Serena Donetti} and Giuseppe Gobbi and Dario Pruna and Patrizia Accorsi and Alberto Verrotti and Pasquale Parisi and Paolo Balestri",
year = "2014",
doi = "10.1016/j.ejpn.2014.05.001",
language = "English",
volume = "18",
pages = "641--645",
journal = "European Journal of Paediatric Neurology",
issn = "1090-3798",
publisher = "W.B. Saunders Ltd",
number = "5",

}

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 -