Prevention of Epilepsy in Infants with Tuberous Sclerosis Complex in the EPISTOP Trial.

Autor: Kotulska K; Department of Neurology and Epileptology, The Children's Memorial Health Institute, Warsaw, Poland., Kwiatkowski DJ; Brigham and Women's Hospital, Harvard Medical School, Boston, MA, USA., Curatolo P; Child Neurology and Psychiatry Unit, Systems Medicine Department, Tor Vergata University, Rome, Italy., Weschke B; Department of Child Neurology, Charité University Medicine Berlin, Berlin, Germany., Riney K; Neurosciences Unit, Queensland Children's Hospital, South Brisbane, QLD, Australia.; School of Medicine, University of Queensland, St Lucia, QLD, Australia., Jansen F; Department of Child Neurology, Brain Center, University Medical Center Utrecht, Utrecht, The Netherlands., Feucht M; Department of Pediatrics, University Hospital Vienna, Vienna, Austria., Krsek P; Motol University Hospital, Charles University, Prague 5, Czech Republic., Nabbout R; Department of Pediatric Neurology, Reference Centre for Rare Epilepsies, Necker- Enfants Malades Hospital, University Paris Descartes, Imagine Institute, Paris, France., Jansen AC; Pediatric Neurology Unit-UZ Brussel, Brussels, Belgium.; Neurogenetics Research Group, Vrije Universiteit Brussel, Brussels, Belgium., Wojdan K; Transition Technologies, Warsaw, Poland.; Warsaw University of Technology, Institute of Heat Engineering, Warsaw, Poland., Sijko K; Department of Neurology and Epileptology, The Children's Memorial Health Institute, Warsaw, Poland.; Transition Technologies, Warsaw, Poland., Głowacka-Walas J; Department of Neurology and Epileptology, The Children's Memorial Health Institute, Warsaw, Poland.; Warsaw University of Technology, The Faculty of Electronics and Information Technology, Warsaw, Poland., Borkowska J; Department of Neurology and Epileptology, The Children's Memorial Health Institute, Warsaw, Poland., Sadowski K; Department of Neurology and Epileptology, The Children's Memorial Health Institute, Warsaw, Poland., Domańska-Pakieła D; Department of Neurology and Epileptology, The Children's Memorial Health Institute, Warsaw, Poland., Moavero R; Child Neurology Unit, Neuroscience and Neurorehabilitation Department, Bambino Gesù Children's Hospital, IRCCS, Rome, Italy., Hertzberg C; Department of Child Neurology, Charité University Medicine Berlin, Berlin, Germany., Hulshof H; Department of Child Neurology, Brain Center, University Medical Center Utrecht, Utrecht, The Netherlands., Scholl T; Department of Pediatrics, University Hospital Vienna, Vienna, Austria., Benova B; Motol University Hospital, Charles University, Prague 5, Czech Republic., Aronica E; Amsterdam UMC, University of Amsterdam, Department of (Neuro)Pathology, Amsterdam Neuroscience, Amsterdam, The Netherlands., de Ridder J; Department of Development and Regeneration-Section Pediatric Neurology, University Hospitals KU Leuven, Leuven, Belgium., Lagae L; Department of Development and Regeneration-Section Pediatric Neurology, University Hospitals KU Leuven, Leuven, Belgium., Jóźwiak S; Department of Neurology and Epileptology, The Children's Memorial Health Institute, Warsaw, Poland.; Department of Child Neurology, Medical University of Warsaw, Warsaw, Poland.
Jazyk: angličtina
Zdroj: Annals of neurology [Ann Neurol] 2021 Feb; Vol. 89 (2), pp. 304-314. Date of Electronic Publication: 2020 Nov 27.
DOI: 10.1002/ana.25956
Abstrakt: Objective: Epilepsy develops in 70 to 90% of children with tuberous sclerosis complex (TSC) and is often resistant to medication. Recently, the concept of preventive antiepileptic treatment to modify the natural history of epilepsy has been proposed. EPISTOP was a clinical trial designed to compare preventive versus conventional antiepileptic treatment in TSC infants.
Methods: In this multicenter study, 94 infants with TSC without seizure history were followed with monthly video electroencephalography (EEG), and received vigabatrin either as conventional antiepileptic treatment, started after the first electrographic or clinical seizure, or preventively when epileptiform EEG activity before seizures was detected. At 6 sites, subjects were randomly allocated to treatment in a 1:1 ratio in a randomized controlled trial (RCT). At 4 sites, treatment allocation was fixed; this was denoted an open-label trial (OLT). Subjects were followed until 2 years of age. The primary endpoint was the time to first clinical seizure.
Results: In 54 subjects, epileptiform EEG abnormalities were identified before seizures. Twenty-seven were included in the RCT and 27 in the OLT. The time to the first clinical seizure was significantly longer with preventive than conventional treatment [RCT: 364 days (95% confidence interval [CI] = 223-535) vs 124 days (95% CI = 33-149); OLT: 426 days (95% CI = 258-628) vs 106 days (95% CI = 11-149)]. At 24 months, our pooled analysis showed preventive treatment reduced the risk of clinical seizures (odds ratio [OR] = 0.21, p = 0.032), drug-resistant epilepsy (OR = 0.23, p = 0.022), and infantile spasms (OR = 0, p < 0.001). No adverse events related to preventive treatment were noted.
Interpretation: Preventive treatment with vigabatrin was safe and modified the natural history of seizures in TSC, reducing the risk and severity of epilepsy. ANN NEUROL 2021;89:304-314.
(© 2020 The Authors. Annals of Neurology published by Wiley Periodicals LLC on behalf of American Neurological Association.)
Databáze: MEDLINE
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