NEUROLOGIE PRO PRAXI / Neurol. praxi. 2024;25(4):315-320 / www.neurologiepropraxi.cz 320 FARMAKOTERAPIE Fenfluramin LITERATURA 1. Bishop KI, Isquith PK, Gioia GA, et al. Improved everyday executive functioning following profound reduction in sei‑ zure frequency with fenfluramine: Analysis from a phase 3 longterm extension study in children/young adults with Dra‑ vet syndrome. Epilepsy Behav. 2021;121:108024. 2. Hatini PG and Commons KG. A 5-HT1D‑receptor ago‑ nist protects Dravet syndrome mice from seizure and early death. Eur. J. Neurosci. 2020;52:4370-4374. doi:10.1111/ejn.14776 3. Knupp KG, Scheffer IE, Ceulemans B, et al. Efficacy and Safety of Fenfluramine for the Treatment of Seizures Asso‑ ciated With Lennox‑Gastaut Syndrome: A Randomized Cli‑ nical Trial. JAMA Neurol. 2022;79(6):55-564. doi:10.1001/jama‑ neurol.2022.0829 4. Knupp KG, Scheffer IE, Ceulemans B, et al. Fenfluramine provides clinically meaningful reduction in frequency of drop seizures in patients with Lennox‑Gastaut syndrome: Interim analysis of an open‑label extension study. Epilepsia. 2023;64(1):139-151. doi: 10.1111/epi.17431. Epub 2022 Nov 9. PMID: 36196777; PMCID: PMC10099582. 5. Lagae L, Sullivan J, Knupp KG, et al (FAiRE DS Study Group). Fenfluramine hydrochloride for thetreatment of seizures in Dravet syndrome: a randomised, double‑blind, placebo con‑ trolled trial. Lancet. 2019;394(10216):2243-54. 6. Martin P, Czerwiń ski M, Limaye PB, et al. In vitro evaluation suggests fenfluramine and norfenfluramine are unlikely to act as perpetrators of drug interactions. Pharmacol Res Perspect. 2022;10:e00959. 7. Martin P, de Witte PAM, Maurice T, et al. Fenfluramine acts as a positive modulator of sigma-1 receptors. Epilepsy & Behav. 2020;105:106989. doi:10.1016/j.yebeh.2020.106989 8. Martin P, Reeder T, Sourbron J, et al. An emerging role for sigma-1 receptors in the treatment of developmental and epi‑ leptic encephalopathies. Int J Mol Sci. 2021;22:8416. 9. Nabbout R, Mistry A, Zuberi S, et al. Fenfluramine for treatment‑resistant seizures in patients with Dravet syndro‑ me receiving stiripentol‑inclusive regimens: a randomized clinical trial. JAMA Neurol. 2020;77(3):300-8. 10. Reeder T, Martin P, Sourbron J, et al. Dual Activity of Fen‑ fluramine (Fintepla®) as a Serotonin Receptor Agonist and Positive Sigma-1 Receptor Modulator: Implication for Di‑ sease Modification in Developmental and Epileptic Ence‑ phalopathies. Poster presented at the American Epilepsy Society Annual Meeting, Chicago, IL, 2021;December 3–7. 11. Schoonjans A‑N, Lagae L, Ceulemans B. Low‑dose fen‑ fluramine in the treatment of neurologic disorders: experien‑ ce in Dravet syndrome. Ther Adv Neurol Disord. 2015; 8: 328–38. 12. Sourbron J, Lagae L. Serotonin receptors in epilepsy: Novel treatment targets? Epilepsia Open 7. 2022; 31-246. doi:10.1002/ epi4.12580 13. Sourbron J, Smolders I, de Witte P, et al. Pharmacological ana‑ lysis of the anti‑epileptic mechanisms of fenfluramine in scn1a mutant zebrafish. Front Pharmacol. 2017;8:191. 14. Sullivan J, Scheffer IE, Lagae L, et al. Fenfluramine HCl (Fintep‑ la®) provides longterm clinically meaningful reduction in seizure frequency: Analysis of an ongoing open‑label extension study. Epilepsia. 2020;61(11):2396-404. 15. Tupal S, Faingold CL. Serotonin 5-HT4 receptors play a critical role in the action of fenfluramine to block seizure induced sudden death in a mouse model of SUDEP. Epilepsy Res. 2021;177:106777. 16. Wirrell EC, Hood V, Knupp KG, et al. International consensus on diagnosis and management of Dravet syndrome. Epilepsia. 2022;63(7):1761-1777. doi: 10.1111/epi.17274. Epub 2022 May 12. PMID: 35490361; PMCID: PMC9543220. ODBORNÝ GARANT: doc. MUDr. Irena Doležalová, Ph.D. POŘADATEL: Společnost SOLEN, s. r. o., ve spolupráci s I. neurologickou klinikou LF MU a FN u sv. Anny ZLATÝ PARTNER Registrace ZDARMA TERMÍN květen 2024 až duben 2025 ON LINE KURZ OBSAH KURZU Co víme o syndromu Dravetové u dospělých? – prof. MUDr. Petr Marusič, Ph.D. Na co ještě myslet u dětí? – MUDr. Martin Kudr, Ph.D. Cesta k diagnóze – dialog s genetikem – MUDr. Katalin Štěrbová Cesta k diagnóze – dialog s genetikem – MUDr. Jana Zárubová Cesta k diagnóze – dialog s genetikem – MUDr. Markéta Vlčková, Ph.D. Mechanismus účinku a protizáchvatová aktivita fen uraminu – MUDr. Ondřej Horák, doc. MUDr. Irena Doležalová, Ph.D. Zaostřeno na syndrom Dravetové dostupný na online.solen.cz
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