Should Off Label Antiepileptic Drugs Be Used for Treatment of Infancy and Childhood Epilepsy? Discussions Based on a Dravet Syndrome Case Report
Epilepsy represents a burdensome neurological disorder with higher incidence before the age of 18 years. The treatment is medical and involves long-term administration of antiepileptic drugs (AED). There are known high-resistant syndromes with onset in infancy and childhood, Dravet syndrome, being one of them. It is a well-known fact that early seizures treatment prevents associated comorbidities, cognitive and motor disabilities, and improve long-term prognosis. There are several AEDs available but not all of them are approved for use in infants. This is due to the need for additional toxicology studies at this age and for development of suitable formulations.
A 14-month-old girl with Dravet syndrome was presented here. Prompt diagnosis was made based on clinical features and confirmed by the genetic tests. She partially responded to valproate and clobazam but continued to have prolonged febrile seizures. We added stiripentol after consulting reports of studies in infants younger than 2 years and after obtaining family consent. She responded well with decrease in episodes of status epilepticus and improvement in psychomotor development and stiripentol was tolerated well. Off label use of certain AEDs can benefit infants when there are no major pharmacokinetic differences in comparison to older children.
Received: 22 March 2020
Accepted: 02 April 2020
28 May 2020 (online)
© 2020. Thieme. All rights reserved.
Georg Thieme Verlag KG
Stuttgart · New York
- 1 Fiest KM, Sauro KM, Wiebe S. , et al. Prevalence and incidence of epilepsy: a systematic review and meta-analysis of international studies. Neurology 2017; 88 (03) 296-303
- 2 Hanaya R, Arita K. The new antiepileptic drugs: their neuropharmacology and clinical indications. Neurol Med Chir (Tokyo) 2016; 56 (05) 205-220
- 3 Reimers A, Brodtkorb E. Second-generation antiepileptic drugs and pregnancy: a guide for clinicians. Expert Rev Neurother 2012; 12 (06) 707-717
- 4 Kuchenbuch M, Chemaly N, Henniene KM, Kaminska A, Chiron C, Nabbout R. Off-label use and manipulations of antiepileptic drugs in children: analysis of the outpatient prescriptions in a tertiary center. Epilepsy Behav 2018; 82: 133-139
- 5 Wirrell EC. Treatment of Dravet syndrome. Can J Neurol Sci 2016; 43 (Suppl. 03) S13-S18
- 6 Patients DP, Food US. Anticonvulsant medications: use in pediatric patients. 2013 ;(August):1–15. Available at: https://www.cms.gov/Medicare-Medicaid-Coordination/Fraud-Prevention/Medicaid-Integrity-Education/Pharmacy-Education-Materials/Downloads/ac-pediatric-factsheet11-14.pdf . Accessed May 5, 2020
- 7 Chiron C. Stiripentol for the treatment of Dravet syndrome. Orphan Drugs Res Rev 2014; 4: 29-38
- 8 Pellock JM, Carman WJ, Thyagarajan V, Daniels T, Morris DL, D'Cruz O. Efficacy of antiepileptic drugs in adults predicts efficacy in children: a systematic review. Neurology 2012; 79 (14) 1482-1489
- 9 Ollivier C, Mulugeta YL, Ruggieri L, Saint-Raymond A, Yao L. Paediatric extrapolation: a necessary paradigm shift. Br J Clin Pharmacol 2019; 85 (04) 675-679