117
Views
0
CrossRef citations to date
0
Altmetric
Review

Stiripentol for the treatment of Dravet syndrome

Pages 29-38 | Published online: 02 Apr 2014

References

  • Dravet C. Les epilepsies graves de l’enfant. Vie Med. 1978;8:543–548. French.
  • Dravet C, Oguni H. Dravet syndrome (severe myoclonic epilepsy in infancy). Handb Clin Neurol. 2013;111:627–633.
  • Claes L, Del-Favero J, Ceulemans B, Lagae L, Van Broeckhoven C, De Jonghe P. De novo mutations in the sodium-channel gene SCN1A cause severe myoclonic epilepsy of infancy. Am J Hum Genet. 2001;68:1327–1332.
  • Nabbout R, Gennaro E, Dalla Bernardina B, et al. Spectrum of SCN1A mutations in severe myoclonic epilepsy of infancy. Neurology. 2003;60(12):1961–1967.
  • Escayg A, Heils A, MacDonald BT, Haug K, Sander T, Meisler MH. A novel SCN1A mutation associated with generalized epilepsy with febrile seizures plus – and prevalence of variants in patients with epilepsy. Am J Hum Genet. 2001;68(4):866–873.
  • Harkin LA, McMahon JM, Iona X, et al; Infantile Epileptic Encephalopathy Referral Consortium, Sutherland G, Berkovic SF, Mulley JC, Scheffer IE. The spectrum of SCN1A-related infantile epileptic encephalopathies. Brain. 2007;130:843–852.
  • 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.
  • [No authors listed]. Proposal for revised classification of epilepsies and epileptic syndromes. Commission on Classification and Terminology of the International League Against Epilepsy. Epilepsia. 1989;30(4):389–399.
  • Tanabe T, Awaya Y, Matsuishi T, et al. Management of and prophylaxis against status epilepticus in children with severe myoclonic epilepsy in infancy (SMEI; Dravet syndrome) – a nationwide questionnaire survey in Japan. Brain Dev. 2008;30(10):629–635.
  • Lotte J, Haberlandt E, Neubauer B, Staudt M, Kluger GJ. Bromide in patients with SCN1A-mutations manifesting as Dravet syndrome. Neuropediatrics. 2012;43(1):17–21.
  • Guerrini R, Dravet C, Genton P, Belmonte A, Kaminska A, Dulac O. Lamotrigine and seizure aggravation in severe myoclonic epilepsy. Epilepsia. 1998;39:508–512.
  • 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. French.
  • Mueller A, Boor R, Coppola G, et al. Low long-term efficacy and tolerability of add-on rufinamide in patients with Dravet syndrome. Epilepsy Behav. 2011;21(3):282–284.
  • Chipaux M, Villeneuve N, Sabouraud P, et al. Unusual consequences of status epilepticus in Dravet syndrome. Seizure. 2010;19:190–194.
  • Okumura A, Uematsu M, Imataka G, et al. Acute encephalopathy in children with Dravet syndrome. Epilepsia. 2012;53(1):79–86.
  • Nieto-Barrera M, Candau R, Nieto-Jimenez M, Correa A, del Portal LR. Topiramate in the treatment of severe myoclonic epilepsy in infancy. Seizure. 2000;9:590–594.
  • 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;49:45–48.
  • Kröll-Seger J, Portilla P, Dulac O, Chiron C. Topiramate in the treatment of highly refractory patients with Dravet syndrome. Neuropediatrics. 2006;37:325–329.
  • Striano P, Coppola G, Pezella M, et al. An open-label trial of levetiracetam in severe myoclonic epilepsy of infancy. Neurology. 2007;69:250–254.
  • Chhun S, Troude P, Villeneuve N, et al. A prospective open-labeled trial with levetiracetam in pediatric epilepsy syndromes: continuous spikes and waves during sleep is definitely a target. Seizure. 2011;20:320–325.
  • Ceulemans B, Boel M, Leyssens K, et al. Successful use of fenfluramine as an add-on treatment for Dravet syndrome. Epilepsia. 2012;53(7):1131–1139.
  • Laux L, Blackford R. The ketogenic diet in Dravet syndrome. J Child Neurol. 2013;28(8):1041–1044.
  • Caraballo RH. Nonpharmacologic treatments of Dravet syndrome: focus on the ketogenic diet. Epilepsia. 2011;52 Suppl 2:S79–S82.
  • Kang HC, Kim YJ, Kim DW, Kim HD. Efficacy and safety of the ketogenic diet for intractable childhood epilepsy: Korean multicentric experience. Epilepsia. 2005;46(2):272–279.
  • 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–e57.
  • Chiron C, Dulac O. The pharmacologic treatment of Dravet syndrome. Epilepsia. 2011;52(Suppl 2):S72–S75.
  • Nabbout R, Chiron C. Stiripentol: an example of antiepileptic drug development in childhood epilepsies. Eur J Paediatr Neurol. 2012; 16 Suppl 1:S13–S17.
  • Plosker GL. Stiripentol: in severe myoclonic epilepsy of infancy (dravet syndrome). CNS Drugs. 2012;26(11):993–1001.
  • Brigo F, Storti M. Antiepileptic drugs for the treatment of severe myoclonic epilepsy in infancy. Cochrane Database Syst Rev. 2013;19(11):CD010483.
  • Perez J, Chiron C, Musial C, et al. Stiripentol: efficacy and tolerability in children with epilepsy. Epilepsia. 1999;40:1618–1626.
  • 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;356:1638–1642.
  • Guerrini R, Tonnelier S, d’Athis P, Rey E, Vincent J, Pons G. Stiripentol in severe myoclonic epilepsy in infancy (SMEI): a placebo-controlled Italian trial. Epilepsia. 2002;43(Suppl 8):S155.
  • 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.
  • Inoue Y, Ohtsuka Y, Oguni H, et al. Stiripentol open study in Japanese patients with Dravet syndrome. Epilepsia. 2009;50:2362–2368.
  • Vari MS, Prato G, Siri L, et al. Long term efficacy of stiripentol in Dravet syndrome: an open label trial of 6 cases. Bollettino Lega italiana contro L’Epilessia. 2010;140:215–216.
  • Wirrell EC, Laux L, Franz DN, et al. Stiripentol in Dravet syndrome: results of a retrospective US study. Epilepsia. 2013;54(9):1595–1604.
  • Diacomit: EPAR – Product Information 2013;http://www.ema.europa.eu/docs/en_GB/document_library/EPAR_-_Summary_for_the_public/human/000664/WC500036519.pdf.
  • Levy RH, Lin HS, Blehaut HM, Tor JA. Pharmacokinetics of stiripentol in normal man: evidence of nonlinearity. J Clin Pharmacol. 1983;23(11–12):523–533.
  • Levy RH, Loiseau P, Guyot M, Blehaut HM, Tor J, Moreland TA. Michaelis-Menten kinetics of stiripentol in normal humans. Epilepsia. 1984;25(4):486–491.
  • 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.
  • 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.
  • Cazali N, Tran A, Treluyer JM, et al. Inhibitory effect of stiripentol on carbamazepine and saquinavir metabolism in human. Br J Clin Pharmacol. 2003;56(5):526–536.
  • 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.
  • 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.
  • Mott DD, Grosenbaugh DK, Fisher JL. Polytherapy with stiripentol: consider more than just metabolic interactions. Epilepsy Behav. 2013;29(3):585.
  • Poisson M, Huguet F, Savattier A, Bakri-Logeais F, Narcisse G. A new type of anticonvulsant, stiripentol. Pharmacological profile and neurochemical study. Arzneimittelforschung. 1984;34(2):199–204.
  • Quilichini PP, Chiron C, Ben-Ari Y, Gozlan H. Stiripentol, a putative antiepileptic drug, enhances the duration of opening of GABA-receptor channels. Epilepsia. 2006;47:704–716.
  • Fisher JL. The anti-convulsant stiripentol acts directly on the GABA(A) receptor as a positive allosteric modulator. Neuropharmacology. 2009;56:190–197.
  • Fisher JL. The effects of stiripentol on GABA(A) receptors. Epilepsia. 2011;52 Suppl 2:76–78.
  • Fisher JL. Interactions between modulators of the GABA(A) receptor: stiripentol and benzodiazepines. Eur J Pharmacol. 2011;654:160–165.
  • Grosenbaugh DK, Mott DD. Stiripentol in refractory status epilepticus. Epilepsia. 2013;54 Suppl 6:103–105.
  • Grosenbaugh DK, Mott DD. Stiripentol is anticonvulsant by potentiating GABAergic transmission in a model of benzodiazepine-refractory status epilepticus. Neuropharmacology. 2013;67:136–143.
  • 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.
  • Laurie DJ, Wisden W, Seeburg PH. The distribution of thirteen GABAA receptor subunit mRNAs in the rat brain. III. Embryonic and postnatal development. J Neurosci. 1992;12(11):4151–4172.
  • 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.
  • 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.
  • Kosaki K, Tamura K, Sato R, Samejima H, Tanigawara Y, Takahashi T. A major influence of CYP2C19 genotype on the steady-state concentration of N-desmethylclobazam. Brain Dev. 2004;26:530–534.
  • Desta Z, Zhao X, Shin JG, Flockhart DA. Clinical significance of the cytochrome P450 2C19 genetic polymorphism. Clin Pharmacokinet. 2002;41:913–958.
  • Chiron C, Kassai B, Dulac O, Pons G, Nabbout R. A revisited strategy for antiepileptic drug development in children: designing an initial exploratory step. CNS Drugs. 2013;27(3):185–195.
  • Brigo F, Ausserer H, Tezzon F, Nardone R. When one plus one makes three: the quest for rational antiepileptic polytherapy with supraadditive anticonvulsant efficacy. Epilepsy Behav. 2013;27:439–442.