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Randomized Controlled Trial
. 2020 Nov 23;21(1):957.
doi: 10.1186/s13063-020-04874-2.

Corticosteroids versus clobazam in epileptic encephalopathy with ESES: a European multicentre randomised controlled clinical trial (RESCUE ESES*)

Bart van den Munckhof  1 , Alexis Arzimanoglou  2   3 , Emilio Perucca  4 , Heleen C van Teeseling  5 , Frans S S Leijten  6 , Kees P J Braun  1 , Floor E Jansen  7 ; RESCUE ESES study group
Collaborators, Affiliations
Randomized Controlled Trial

Corticosteroids versus clobazam in epileptic encephalopathy with ESES: a European multicentre randomised controlled clinical trial (RESCUE ESES*)

Bart van den Munckhof et al. Trials. .

Abstract

Background: Epileptic encephalopathy with electrical status epilepticus in sleep (ESES) is an epilepsy syndrome occurring almost exclusively in children, usually at an age between 4 and 12 years. It is characterised by abundant sleep-induced epileptic activity in the electroencephalogram (EEG) and by acquired cognitive and behavioural deficits. The goal of treatment is to prevent further decline or even improve cognitive functioning. Based on mostly small and retrospective studies, corticosteroids and clobazam are regarded by many clinicians as the most effective pharmacological treatments. This European multicentre randomised controlled trial is designed to compare the effects of corticosteroids and clobazam on cognitive functioning after 6 months. Secondary outcomes include cognitive functioning after 18 months, EEG abnormalities in sleep, safety and tolerability, and seizure frequency. We also aimed at investigating whether treatment response in epileptic encephalopathy with ESES can be predicted by measurement of inflammatory mediators and autoantibodies in serum.

Methods: The pragmatic study will be performed in centres with expertise in the treatment of rare paediatric epilepsy syndromes across Europe. A total of 130 patients, 2 to 12 years of age, with epileptic encephalopathy with ESES will be enrolled and randomised in a 1:1 ratio to receive either corticosteroids (monthly intravenous methylprednisolone pulses or daily oral prednisolone) or oral clobazam for 6 months according to an open-label parallel-group design. Follow-up visits with clinical assessment, EEGs, and neuropsychological testing are scheduled for up to 18 months. Blood samples for cytokine and autoantibody testing are obtained before treatment and 8 months after treatment initiation.

Discussion: The treatment of epileptic encephalopathy with ESES aims at improving cognitive outcome. This randomised controlled study will compare the most frequently used treatments, i.e. corticosteroids and clobazam. If the study proves superiority of one treatment over the other or identifies biomarkers of treatment response, results will guide clinicians in the early treatment of this severe epilepsy syndrome.

Trial registration: ISRCTN, ISRCTN42686094 . Registered on 24 May 2013.

Keywords: CSWS; Clobazam; Cognition; Corticosteroids; ESES; Epilepsy; Neuropsychology; Sleep.

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Conflict of interest statement

The authors declare that they have no competing interests.

Figures

Fig. 1
Fig. 1
Schedule of enrolment, interventions, and assessments for participants
See this image and copyright information in PMC

References

    1. Patry G, Lyagoubi S, Tassinari CA. Subclinical “electrical status epilepticus” induced by sleep in children. Arch Neurol. 1971;24(3):242–252. doi: 10.1001/archneur.1971.00480330070006. - DOI - PubMed
    1. Landau WM, Kleffner FR. Syndrome of acquired aphasia with convulsive disorder in children. Neurology. 1957;7(8):1241. doi: 10.1212/WNL.7.8.523. - DOI - PubMed
    1. Fernández IS, Chapman KE, Peters JM, et al. The tower of Babel: survey on concepts and terminology in electrical status epilepticus in sleep and continuous spikes and waves during sleep in North America. Epilepsia. 2013;54(4):741–750. doi: 10.1111/epi.12039. - DOI - PMC - PubMed
    1. Nickels K, Wirrell E. Electrical status epilepticus in sleep. Semin Pediatr Neurol. 2008;15(2):50–60. doi: 10.1016/j.spen.2008.03.002. - DOI - PubMed
    1. Sánchez Fernández I, Loddenkemper T, Peters JM, Kothare SV. Electrical status epilepticus in sleep: clinical presentation and pathophysiology. Pediatr Neurol. 2012;47(6):390–410. doi: 10.1016/j.pediatrneurol.2012.06.016. - DOI - PubMed

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