Stiripentol and vigabatrin current roles in the treatment of epilepsy

References

• Chiron C, Kassai B, Dulac O, et al. A revisited strategy for antiepileptic drug development in children: designing an initial exploratory step. CNS Drugs. 2013;27(3):185–195.
   PubMed Web of Science ®Google Scholar
• The European Guidelines on the treatment of epileptic disorders. CHMP/EWP/566/98. 22 July 2010
   Google Scholar
• Fisher JL. The anti-convulsant stiripentol acts directly on the GABA(A) receptor as a positive allosteric modulator. Neuropharmacology. 2009;56:190–197.
   PubMed Web of Science ®Google Scholar
• Quilichini PP, Chiron C, Ben-Ari Y, et al. Stiripentol, a putative antiepileptic drug, enhances the duration of opening of GABA-receptor channels. Epilepsia. 2006;47:704–716.
   PubMed Web of Science ®Google Scholar
• Chiron C, Marchand MC, Tran A, et al. Stiripentol in severe myoclonic epilepsy in infancy: a randomised placebo-controlled syndrome-dedicated trial. STICLO Study Group. Lancet. 2000;356(9242):1638–42.
   PubMed Web of Science ®Google Scholar
• Guerrini R, Tonnelier S, d’Athis P, et al. Stiripentol in severe myoclonic epilepsy in infancy (SMEI): a placebo-controlled Italian trial. Epilepsia. 2002;43(Suppl 8):S155.
   Google Scholar
• Kassaï B, Chiron C, Augier S, et al. Severe myoclonic epilepsy in infancy: A systematic review and a meta-analysis of individual patient data. Epilepsia. 2008;49:343–348.
   PubMed Web of Science ®Google Scholar
• Brigo F, Storti M. Antiepileptic drugs for the treatment of severe myoclonic epilepsy in infancy. Cochrane Database Syst Rev. 2013;19(11):CD010483.
   Google Scholar
• Thanh TN, Chiron C, Dellatolas G, et al. Long-term efficacy and tolerance of stiripentol in severe myoclonic epilepsy of infancy (Dravet’s syndrome). Arch Pediatr. 2002;9:1120–1127.
   PubMed Web of Science ®Google Scholar
• Inoue Y, Ohtsuka Y. STP-1 Study Group. Effectiveness of add-on stiripentol to clobazam and valproate in Japanese patients with Dravet syndrome: additional supportive evidence. Epilepsy Res. 2014 May;108(4):725–731.
   PubMed Web of Science ®Google Scholar
• Inoue Y, Ohtsuka Y. STP-1 Study Group. Long-term safety and efficacy of stiripentol for the treatment of Dravet syndrome: A multicenter, open-label study in Japan. Epilepsy Res. 2015;113:90–97.
   PubMed Web of Science ®Google Scholar
• Wirrell EC, Laux L, Franz DN, et al. Stiripentol in Dravet syndrome: results of a retrospective U.S. study. Epilepsia. 2013;54(9):1595–1604.
   PubMed Web of Science ®Google Scholar
• Moreland TA, Astoin J, Lepage F, et al. The metabolic fate of stiripentol in man. Drug Metab Dispos. 1986;14(6):654–662.
   PubMed Web of Science ®Google Scholar
• Levy RH, Loiseau P, Guyot M, et al. Michaelis–Menten kinetics of stiripentol in normal humans. Epilepsia. 1984;25(4):486–491.
   PubMed Web of Science ®Google Scholar
• Peigné S, Rey E, Le Guern ME, et al. Reassessment of stiripentol pharmacokinetics in healthy adult volunteers. Epilepsy Res. 2014;108(5):909–916.
   PubMed Web of Science ®Google Scholar
• May TW, Boor R, Mayer T, et al. Concentrations of stiripentol in children and adults with epilepsy: the influence of dose, age, and comedication. Ther Drug Monit. 2012;34(4):390–397.
   PubMed Web of Science ®Google Scholar
• Tran A, Rey E, Pons G, et al. Influence of stiripentol on cytochrome P450-mediated metabolic pathways in humans: in vitro and in vivo comparison and calculation of in vivo inhibition constants. Clin Pharmacol Ther. 1997;62(5):490–504.
   PubMed Web of Science ®Google Scholar
• Giraud C, Treluyer JM, Rey E, et al. In vitro and in vivo inhibitory effect of stiripentol on clobazam metabolism. Drug Metab Dispos. 2006;34:608–611.
   PubMed Web of Science ®Google Scholar
• Jullien V, Chhun S, Rey E, et al. Pharmacokinetics of clobazam and N-desmethylclobazam in children with Dravet syndrome receiving concomitant stiripentol and valproic acid. Clin Pharmacokinet. 2015;54(5):527–536.
   PubMed Web of Science ®Google Scholar
• Poisson M, Huguet F, Savattier A, et al. A new type of anticonvulsant, stiripentol. Pharmacological profile and neurochemical study. Arzneimittelforschung. 1984;34(2):199–204.
   PubMed Web of Science ®Google Scholar
• Grosenbaugh DK, Mott DD. Stiripentol is anticonvulsant by potentiating GABAergic transmission in a model of benzodiazepine-refractory status epilepticus. Neuropharmacology. 2013;67:136–143.
   PubMed Web of Science ®Google Scholar
• Auvin S, Lecointe C, Dupuis N, et al. Stiripentol exhibits higher anticonvulsant properties in the immature than in the mature rat brain. Epilepsia. 2013;54(12):2082–2090.
   PubMed Web of Science ®Google Scholar
• Fisher JL. Interactions between modulators of the GABA(A) receptor: Stiripentol and benzodiazepines. Eur J Pharmacol. 2011;654:160–165.
   PubMed Web of Science ®Google Scholar
• Cao D, Ohtani H, Ogiwara I, et al. Efficacy of stiripentol in hyperthermia-induced seizures in a mouse model of Dravet syndrome. Epilepsia. 2012;53(7):1140–1145.
   PubMed Web of Science ®Google Scholar
• Baraban SC, Dinday MT, Hortopan GA. Drug screening in Scn1a zebrafish mutant identifies clemizole as a potential Dravet syndrome treatment. Nat Commun. 2013;4:2410.
   PubMed Web of Science ®Google Scholar
• Inoue Y, Ohtsuka Y, Oguni H, et al. Stiripentol open study in Japanese patients with Dravet syndrome. Epilepsia. 2009;50:2362–2368.
   PubMed Web of Science ®Google Scholar
• Rosander C, Hallböök T. Dravet syndrome in Sweden: a population-based study. Dev Med Child Neurol. 2015 Mar 13. doi:10.1111/dmcn.12709.
   PubMed Web of Science ®Google Scholar
• Strzelczyk A, Schubert-Bast S, Reese JP, et al. Evaluation of health-care utilization in patients with Dravet syndrome and on adjunctive treatment with stiripentol and clobazam. Epilepsy Behav. 2014 May;34:86–91.
   PubMed Web of Science ®Google Scholar
• Dressler A, Trimmel-Schwahofer P, Reithofer E, et al. Efficacy and tolerability of the ketogenic diet in Dravet syndrome - Comparison with various standard antiepileptic drug regimen. Epilepsy Res. 2015;109:81–89.
   PubMed Web of Science ®Google Scholar
• Kouga T, Shimbo H, Iai M, et al. Effect of CYP2C19 polymorphisms on stiripentol administration in Japanese cases of Dravet syndrome. Brain De. 2015;37(2):243–249.
   Web of Science ®Google Scholar
• Yamamoto Y, Takahashi Y, Imai K, et al. Influence of CYP2C19 polymorphism and concomitant antiepileptic drugs on serum clobazam and N-desmethyl clobazam concentrations in patients with epilepsy. Ther Drug Monit. 2013;35(3):305–312.
   PubMed Web of Science ®Google Scholar
• Nabbout R, Chiron C. Stiripentol: an example of antiepileptic drug development in childhood epilepsies. Eur J Paediatr Neurol. 2012;16(Suppl 1):S13–S17.
   PubMedGoogle Scholar
• Coppola G, Capovilla G, Montagnini A, et al. Topiramate as add-on drug in severe myoclonic epilepsy in infancy: an Italian multicenter open trial. Epilepsy Res. 2002;49(1):45–48.
   PubMed Web of Science ®Google Scholar
• Nieto-Barrera M, Candau R, Nieto-Jimenez M, et al. Topiramate in the treatment of severe myoclonic epilepsy in infancy. Seizure. 2000;9(8):590–594.
   PubMed Web of Science ®Google Scholar
• Ceulemans B, Boel M, Claes L, et al. Severe myoclonic epilepsy in infancy: toward an optimal treatment. J Child Neurol. 2004;19(7):516–521.
   PubMed Web of Science ®Google Scholar
• Kröll-Seger J, Portilla P, Dulac O, et al. Topiramate in the treatment of highly refractory patients with Dravet syndrome. Neuropediatrics. 2006;37:325–329.
   PubMed Web of Science ®Google Scholar
• Neal EG, Chaffe H, Schwartz RH, et al. The ketogenic diet for the treatment of childhood epilepsy: a randomised controlled trial. Lancet Neurol. 2008;7(6):500–506.
   PubMed Web of Science ®Google Scholar
• Kang HC, Kim YJ, Kim DW, et al. Efficacy and safety of the ketogenic diet for intractable childhood epilepsy: Korean multicentric experience. Epilepsia. 2005;46(2):272–279.
   PubMed Web of Science ®Google Scholar
• Caraballo RH. Nonpharmacologic treatments of Dravet syndrome: focus on the ketogenic diet. Epilepsia. 2011;52(Suppl 2):S79–S82.
   PubMed Web of Science ®Google Scholar
• Laux L, Blackford R. The ketogenic diet in Dravet syndrome. J Child Neurol. 2013;28(8):1041–1044.
   PubMed Web of Science ®Google Scholar
• Nabbout R, Copioli C, Chipaux M, et al. Ketogenic diet also benefits Dravet syndrome patients receiving stiripentol: a prospective pilot study. Epilepsia. 2011;52(7):e54–7.
   PubMed Web of Science ®Google Scholar
• Akiyama M, Kobayashi K, Yoshinaga H, et al. A long-term follow-up study of Dravet syndrome up to adulthood. Epilepsia. 2010;51(6):1043–1052.
   PubMed Web of Science ®Google Scholar
• Catarino CB, Liu JY, Liagkouras I, et al. Dravet syndrome as epileptic encephalopathy: evidence from long-term course and neuropathology. Brain. 2011;134(Pt 10):2982–3010.
   PubMed Web of Science ®Google Scholar
• Takayama R, Fujiwara T, Shigematsu H, et al. Long-term course of Dravet syndrome: a study from an epilepsy center in Japan. Epilepsia. 2014;55(4):528–538.
   PubMed Web of Science ®Google Scholar
• Chiron C, An I. Pharmacology aspects during transition and at transfer in patients with epilepsy. Epilepsia. 2014;55(Suppl 3):8–11.
   PubMed Web of Science ®Google Scholar
• Diacomit. EPAR - Product Information 2013 Available from: http://www.ema.europa.eu/docs/en_GB/document_library/EPAR_Product_Information/human/000664/WC500036518.pdf
   Google Scholar
• Depienne C, Bouteiller D, Keren B, et al. Sporadic infantile epileptic encephalopathy caused by mutations in PCDH19 resembles Dravet syndrome but mainly affects females. PLoS Genet. 2009;5(2):e1000381.
   Web of Science ®Google Scholar
• Trivisano M, Specchio N, Vigevano F. Extending the use of stiripentol to other epileptic syndromes: a case of PCDH19-related epilepsy. Eur J Paediatr Neurol. 2015;19(2):248–250.
   PubMed Web of Science ®Google Scholar
• Chiron C, Tonnelier S, Rey E, et al. Stiripentol in childhood partial epilepsy: randomized placebo-controlled trial with enrichment and withdrawal design. J Child Neurol. 2006;21(6):496–502.
   PubMed Web of Science ®Google Scholar
• Brigo F, Storti M. Stiripentol for focal refractory epilepsy. Cochrane Database Syst Rev. 2014 31;1:CD009887.
   Google Scholar
• Perez J, Chiron C, Musial C, et al. Stiripentol: efficacy and tolerability in children with epilepsy. Epilepsia. 1999;40:1618–1626.
   PubMed Web of Science ®Google Scholar
• Merdariu D, Delanoë C, Mahfoufi N, et al. Malignant migrating partial seizures of infancy controlled by stiripentol and clonazepam. Brain Dev. 2013;35(2):177–80.
   PubMed Web of Science ®Google Scholar
• Djuric M, Kravljanac R, Kovacevic G, et al. The efficacy of bromides, stiripentol and levetiracetam in two patients with malignant migrating partial seizures in infancy. Epileptic Disord. 2011;13(1):22–26.
   PubMed Web of Science ®Google Scholar
• Tran A, Vauzelle-Kervroedan F, Rey E, et al. Effect of stiripentol on carbamazepine plasma concentration and metabolism in epileptic children. Eur J Clin Pharmacol. 1996;50(6):497–500.
   PubMed Web of Science ®Google Scholar
• Strzelczyk A, Kortland LM, Knake S, et al. Stiripentol for the treatment of super-refractory status epilepticus. Acta Neurol Scand. 2015;132(6):435–439.
   PubMed Web of Science ®Google Scholar
• Eke T, Talbot JF, Lawden MC. Severe persistent visual field constriction associated with vigabatrin. BMJ. 1997 18;314(7075):180–181.
   PubMed Web of Science ®Google Scholar
• Chiron C, Dulac O, Luna D, et al. Vigabatrin in infantile spasms. Lancet. 1990 10;335(8685):363–364.
   PubMed Web of Science ®Google Scholar
• Chiron C, Dumas C, Jambaqué I, et al. Randomized trial comparing vigabatrin and hydrocortisone in infantile spasms due to tuberous sclerosis. Epilepsy Res. 1997;26(2):389–395.
   PubMed Web of Science ®Google Scholar
• Elterman RD, Shields WD, Mansfield KA, et al. Vigabatrin Study Group. Randomized trial of vigabatrin in patients with infantile spasms. Neurology. 2001;57(8):1416–1421.
   PubMed Web of Science ®Google Scholar
• Appleton RE, Peters AC, Mumford JP, et al. Randomised, placebo-controlled study of vigabatrin as first-line treatment of infantile spasms. Epilepsia. 1999;40(11):1627–1633.
   PubMed Web of Science ®Google Scholar
• Lux AL, Edwards SW, Hancock E, et al. The United Kingdom Infantile Spasms Study comparing vigabatrin with prednisolone or tetracosactide at 14 days: a multicentre, randomised controlled trial. Lancet. 2004;364(9447):1773–8.
   PubMed Web of Science ®Google Scholar
• Lux AL, Edwards SW, Hancock E, et al. United Kingdom Infantile Spasms Study. The United Kingdom Infantile Spasms Study (UKISS) comparing hormone treatment with vigabatrin on developmental and epilepsy outcomes to age 14 months: a multicentre randomised trial. Lancet Neurol. 2005;4(11):712–717.
   PubMed Web of Science ®Google Scholar
• Darke K, Edwards SW, Hancock E, et al; Trial steering committee on behalf of participating investigators. Developmental and epilepsy outcomes at age 4 years in the UKISS trial comparing hormonal treatments to vigabatrin for infantile spasms: a multi-centre randomised trial. Arch Dis Child. 2010;95(5):382–6.
   PubMed Web of Science ®Google Scholar
• Elterman RD, Shields WD, Bittman RM, et al. Vigabatrin for the treatment of infantile spasms: final report of a randomized trial. J Child Neurol. 2010;25(11):1340–1347.
   PubMed Web of Science ®Google Scholar
• Djuric M, Kravljanac R, Tadic B, et al. Long-term outcome in children with infantile spasms treated with vigabatrin: a cohort of 180 patients. Epilepsia. 2014;55(12):1918–1925.
   PubMed Web of Science ®Google Scholar
• Riikonen R. Long-term otucome of West syndrome: a study of adults with a history of infantile spasms. Epilepsia. 1996;37(4):367–372.
   PubMed Web of Science ®Google Scholar
• Maguire MJ, Hemming K, Wild JM, et al. Prevalence of visual field loss following exposure to vigabatrin therapy: a systematic review. Epilepsia. 2010;51(12):2423–2431.
   PubMed Web of Science ®Google Scholar
• Wild JM, Chiron C, Ahn H, et al. Visual field loss in patients with refractory partial epilepsy treated with vigabatrin: final results from an open-label, observational, multicentre study. CNS Drugs. 2009;23(11):965–982.
   PubMed Web of Science ®Google Scholar
• Riikonen R. Long-term outcome in children with infantile spasms treated with vigabatrin: A cohort of 180 patients. Epilepsia. 2015;56(5):807–809.
   PubMed Web of Science ®Google Scholar
• Gaily E, Jonsson H, Lappi M. Visual fields at school-age in children treated with vigabatrin in infancy. Epilepsia. 2009;50(2):206–216.
   PubMed Web of Science ®Google Scholar
• Westall CA, Nobile R, Morong S, et al. Changes in the electroretinogram resulting from discontinuation of vigabatrin in children. Doc Ophthalmol. 2003;107(3):299–309.
   PubMed Web of Science ®Google Scholar
• Westall CA, Wright T, Cortese F, et al. Vigabatrin retinal toxicity in children with infantile spasms: an observational cohort study. Neurology. 2014;83(24):2262–8.
   PubMed Web of Science ®Google Scholar
• Wheless JW, Carmant L, Bebin M, et al. Magnetic resonance imaging abnormalities associated with vigabatrin in patients with epilepsy. Epilepsia. 2009;50(2):195–205.
   PubMed Web of Science ®Google Scholar
• Milh M, Villeneuve N, Chapon F, et al. Transient brain magnetic resonance imaging hyperintensity in basal ganglia and brain stem of epileptic infants treated with vigabatrin. J Child Neurol. 2009;24(3):305–315.
   PubMed Web of Science ®Google Scholar
• Fong CY, Osborne JP, Edwards SW, et al. An investigation into the relationship between vigabatrin, movement disorders, and brain magnetic resonance imaging abnormalities in children with infantile spasms. Dev Med Child Neurol. 2013;55(9):862–867.
   PubMed Web of Science ®Google Scholar
• Hemming K, Maguire MJ, Hutton JL, et al. Vigabatrin for refractory partial epilepsy. Cochrane Database Syst Rev. 2013;1:CD007302.
   PubMed Web of Science ®Google Scholar
• Luna D, Dulac O, Pajot N, et al. Vigabatrin in the treatment of childhood epilepsies: a single-blind placebo-controlled study. Epilepsia. 1989;30(4):430–7.
   PubMed Web of Science ®Google Scholar
• Nabbout RC, Chiron C, Mumford J, et al. Vigabatrin in partial seizures in children. J Child Neurol. 1997;12(3):172–177.
   PubMed Web of Science ®Google Scholar
• Lortie A, Plouin P, Chiron C, et al. Characteristics of epilepsy in focal cortical dysplasia in infancy. Epilepsy Res. 2002;51(1–2):133–145.
   PubMed Web of Science ®Google Scholar
• Camposano SE, Major P, Halpern E, et al. Vigabatrin in the treatment of childhood epilepsy: a retrospective chart review of efficacy and safety profile. Epilepsia. 2008;49(7):1186–1191.
   PubMed Web of Science ®Google Scholar
• Parker AP, Agathonikou A, Robinson RO, et al. Inappropriate use of carbamazepine and vigabatrin in typical absence seizures. Dev Med Child Neurol. 1998;40(8):517–519.
   PubMed Web of Science ®Google Scholar
• Lortie A, Chiron C, Dumas C, et al. Optimizing the indication of vigabatrin in children with refractory epilepsy. J Child Neurol. 1997;12(4):253–259.
   PubMed Web of Science ®Google Scholar
• Wilmshurst JM, Gaillard WD, Vinayan KP, et al. Summary of recommendations for the management of infantile seizures: Task Force Report for the ILAE Commission of Pediatrics. Epilepsia. 2015;56(8):1185–1197.
   PubMed Web of Science ®Google Scholar
• Aras LM, Isla J, Mingorance-Le Meur A. The European patient with Dravet syndrome: results from a parent-reported survey on antiepileptic drug use in the European population with Dravet syndrome. Epilepsy Behav. 2015;44:104–109.
   PubMed Web of Science ®Google Scholar
• Brunklaus A, Ellis R, Reavey E, et al. Prognostic, clinical and demographic features in SCN1A mutation-positive Dravet syndrome. Brain. 2012;135(Pt 8):2329–2336.
   PubMed Web of Science ®Google Scholar
• Lu R, Liu S, Wang Q, et al. Nanoemulsions as novel oral carriers of stiripentol: insights into the protective effect and absorption enhancement. Int J Nanomedicine. 2015 31;10:4937–4946.
   PubMed Web of Science ®Google Scholar
• Sada N, Lee S, Katsu T, et al. Epilepsy treatment. Targeting LDH enzymes with a stiripentol analog to treat epilepsy. Science. 2015;347(6228):1362–1367.
   PubMed Web of Science ®Google Scholar
• Vigevano F, Cilio MR. Vigabatrin versus ACTH as first-line treatment for infantile spasms: a randomized, prospective study. Epilepsia. 1997;38(12):1270–1274.
   PubMed Web of Science ®Google Scholar
• Granström ML, Gaily E, Liukkonen E. Treatment of infantile spasms: results of a population-based study with vigabatrin as the first drug for spasms. Epilepsia. 1999;40(7):950–957.
   PubMed Web of Science ®Google Scholar
• Jambaqué I, Chiron C, Dumas C, et al. Mental and behavioural outcome of infantile epilepsy treated by vigabatrin in tuberous sclerosis patients. Epilepsy Res. 2000;38(2–3):151–160.
   PubMed Web of Science ®Google Scholar
• Gaily E, Appelqvist K, Kantola-Sorsa E, et al. Cognitive deficits after cryptogenic infantile spasms with benign seizure evolution. Dev Med Child Neurol. 1999;41(10):660–664.
   PubMed Web of Science ®Google Scholar
• Eisermann MM, DeLaRaillère A, Dellatolas G, et al. Infantile spasms in Down syndrome - effects of delayed anticonvulsive treatment. Epilepsy Res. 2003;55(1–2):21–27.
   PubMed Web of Science ®Google Scholar
• O’Callaghan FJ, Lux AL, Darke K, et al. The effect of lead time to treatment and of age of onset on developmental outcome at 4 years in infantile spasms: evidence from the United Kingdom Infantile Spasms Study. Epilepsia. 2011;52(7):1359–1364.
   PubMed Web of Science ®Google Scholar
• Jóźwiak S, Kotulska K, Domańska-Pakieła D, et al. Antiepileptic treatment before the onset of seizures reduces epilepsy severity and risk of mental retardation in infants with tuberous sclerosis complex. Eur J Paediatr Neurol. 2011;15(5):424–431.
   PubMed Web of Science ®Google Scholar
• Camfield P, Camfield C, Lortie A, et al. Infantile spasms in remission may reemerge as intractable epileptic spasms. Epilepsia. 2003;44(12):1592–1595.
   PubMed Web of Science ®Google Scholar
• Overwater IE, Bindels-de Heus K, Rietman AB, et al. Epilepsy in children with tuberous sclerosis complex: Chance of remission and response to antiepileptic drugs. Epilepsia. 2015;56(8):1239–1245.
   PubMed Web of Science ®Google Scholar
• Kröll-Seger J, Kaminska A, Moutard ML, et al. Severe relapse of epilepsy after vigabatrin withdrawal: for how long should we treat symptomatic infantile spasms? Epilepsia. 2007;48(3):612–613.
   PubMed Web of Science ®Google Scholar
• Jammoul F, Wang Q, Nabbout R, et al. Taurine deficiency is a cause of vigabatrin-induced retinal phototoxicity. Ann Neurol. 2009;65(1):98–107.
   PubMed Web of Science ®Google Scholar
• Raffo E, Coppola A, Ono T, et al. A pulse rapamycin therapy for infantile spasms and associated cognitive decline. Neurobiol Dis. 2011;43(2):322–329.
   PubMed Web of Science ®Google Scholar
• Briggs SW, Mowrey W, Hall CB. Galanopoulou AS. CPP-115, a vigabatrin analogue, decreases spasms in the multiple-hit rat model of infantile spasms. Epilepsia. 2014;55(1):94–102.
   PubMed Web of Science ®Google Scholar