Evidence map›Paper›PMID 31912454›Full record

ReviewPaediatric drugs2020

Update on Drug Management of Refractory Epilepsy in Tuberous Sclerosis Complex.

Emma van der Poest Clement, Floor E Jansen, Kees P J Braun, Jurriaan M Peters

2 registry-linked trialsAbstract readReview
PubMed Publisher
In one paragraph

Review in Paediatric drugs, 2020. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to 2 registered trials, which are not on this map. Cited by 32 papers, 1 of them a synthesis that pooled it.

0numbers the graph read from it
0cells of the map it votes in
32citing papers in PubMed, 1 pooled it
5.8field-weighted citation impact, top 3% of its field
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

NCT02098759 naunknown statusnot on this map

Long-term, Prospective Study Evaluating Clinical and Molecular Biomarkers of Epileptogenesis in a Genetic Model of Epilepsy - Tuberous Sclerosis Complex

TypeinterventionalSponsorSergiusz JozwiakRan2013 to 2018Enrolled100ConditionsTSC, Tuberous Sclerosis Complex, EpilepsyArmsepilepsy early diagnosis protocol
NCT02849457 phase2completednot on this map

Preventing Epilepsy Using Vigabatrin In Infants With Tuberous Sclerosis Complex (PREVeNT Trial) A Randomized, Double-blind, Placebo-controlled Seizure Prevention Clinical Trial for Infants With TSC

TypeinterventionalSponsorMartina BebinRan2016 to 2023Enrolled84ConditionsTuberous Sclerosis ComplexArmsEarly Vigabatrin, Delayed Vigabatrin (Placebo)
3 · Its place in the literature

Who cites it

32 citing papers in PubMed, 1 synthesis or guideline pooled it, 64 citations in OpenAlex.

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  18. Tuberous sclerosis complex with skin lesions as the initial presentation: A case report and multidisciplinary discussion.Zhong nan da xue xue bao. Yi xue ban = Journal of Central South University. Medical sciences · 2022
    Article
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4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

4 authors at 2 institutions in 2 countries.

Emma van der Poest ClementDepartment of Child Neurology, University Medical Center Utrecht Brain Center, Utrecht, The Netherlands.
Floor E JansenDepartment of Child Neurology, University Medical Center Utrecht Brain Center, Utrecht, The Netherlands.
Kees P J BraunDepartment of Child Neurology, University Medical Center Utrecht Brain Center, Utrecht, The Netherlands.
Jurriaan M PetersDivision of Epilepsy and Clinical Neurophysiology, Boston Children's Hospital and Harvard Medical School, 300 Longwood Ave, FE9, Boston, 02115, USA. jurriaan.peters@childrens.harvard.edu.ORCID http://orcid.org/0000-0002-6725-2814
University Medical Center Utrecht · NLBoston Children's Hospital · US

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Tuberous sclerosis complex (TSC) is a genetic neurocutaneous disorder with epilepsy as a common and early presenting symptom. The neurological phenotype, however, is variable and unpredictable. Early and refractory seizures, infantile spasms in particular, are associated with a poor neurological outcome. Preliminary data suggests early and aggressive seizure control may mitigate the detrimental neurodevelopmental effects of epilepsy. For infantile spasms, vigabatrin is the first line of treatment, and steroids and classic antiepileptic drugs (AEDs) are suitable for second line. Based on retrospective data, vigabatrin should be considered for other indications, especially in infants with focal seizures, as this may prevent infantile spasms, but also in children and adults with epileptic spasms and tonic seizures. Otherwise, for most seizure types, treatment is similar to that for patients without TSC, including the use of novel AEDs, although limited data are available. Three major developments are changing the field of epilepsy management in TSC. First, final recommendations on preventive treatment with vigabatrin will result from two multicenter trials in the US (PREVeNT, clinicaltrials.gov #NCT02849457) and Europe (EPISTOP, clinicaltrials.gov #NCT02098759). Second, treatment with everolimus, an inhibitor of the mechanistic target of rapamycin (mTOR), reduced seizures when compared to placebo. Further, mTOR inhibitors may have an overall disease-modifying effect. Third, the role of cannabidiol in the treatment of refractory seizures in TSC is yet to be established. With treatment recommendations in TSC, we keep an eye on the prize for the broader field of pediatric epilepsy: the lessons learned from TSC are likely applicable to other epileptic encephalopathies.

Indexed as

AdultAnticonvulsantsDrug Resistant EpilepsyFemaleHumansMaleRetrospective StudiesTuberous SclerosisYoung AdultAnticonvulsants

Identifiers

PMID31912454
OpenAlexW2999248741

What OpenQuestion holds

Textmetadata
Read underepoch 390

Registered trials

Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.