Evidence map›Paper›PMID 37867433›Full record

Observational studyEpilepsia open2024

Efficacy and tolerability of add-on stiripentol in real-world clinical practice: An observational study in Dravet syndrome and non-Dravet developmental and epileptic encephalopathies.

Antonio Gil-Nagel, Angel Aledo-Serrano, Álvaro Beltrán-Corbellini, Laura Martínez-Vicente, Adolfo Jimenez-Huete, Rafael Toledano-Delgado, Irene Gacía-Morales, Adrián Valls-Carbó

Open access · goldAbstract readObservational Study
In one paragraph

Observational study in Epilepsia open, 2024. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 14 papers, 1 of them a synthesis that pooled it.

0numbers the graph read from it
0cells of the map it votes in
14citing papers in PubMed, 1 pooled it
4.2field-weighted citation impact, top 5% 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.

Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.

3 · Its place in the literature

Who cites it

14 citing papers in PubMed, 1 synthesis or guideline pooled it, 19 citations in OpenAlex.

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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

8 authors at 3 institutions in 1 country.

Antonio Gil-NagelDepartment of Neurology, Hospital Ruber Internacional, Madrid, Spain.ORCID 0000-0003-4515-0793
Angel Aledo-SerranoDepartment of Neurology, Hospital Ruber Internacional, Madrid, Spain.ORCID 0000-0003-4889-3365
Álvaro Beltrán-CorbelliniDepartment of Neurology, Hospital Ruber Internacional, Madrid, Spain.ORCID 0000-0003-2743-0978
Laura Martínez-VicenteDepartment of Neurology, Hospital Universitario La Fe, Valencia, Spain.
Adolfo Jimenez-HueteDepartment of Neurology, Hospital Ruber Internacional, Madrid, Spain.
Rafael Toledano-DelgadoDepartment of Neurology, Hospital Ruber Internacional, Madrid, Spain.ORCID 0000-0002-9387-1088
Irene Gacía-MoralesDepartment of Neurology, Hospital Ruber Internacional, Madrid, Spain.
Adrián Valls-CarbóFundación INCE (Iniciativa para las Neurociencias), Madrid, Spain.ORCID 0000-0002-4831-1276
Hospital Ruber Internacional · ESFundacion Centro De Investigacion De Enfermedades Neurologicas · ESHospital Universitari i Politècnic La Fe · ES

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

objectiveTo assess efficacy and tolerability of stiripentol (STP) as adjunctive treatment in Dravet syndrome and non-Dravet refractory developmental and epileptic encephalopathies (DREEs).

methodsRetrospective observational study of all children and adults with DREE and prescribed adjunctive STP at Hospital Ruber Internacional from January 2000 to February 2023. Outcomes were retention rate, responder rate (proportion of patients with ≥50% reduction in total seizure frequency relative to baseline), seizure freedom rate, responder rate for status epilepticus, rate of adverse event and individual adverse events, reported at 3, 6, and 12 months and at final visit. Seizure outcomes are reported overall, and for Dravet and non-Dravet subgroups.

resultsA total of 82 patients (55 Dravet syndrome and 27 non-Dravet DREE) were included. Median age was 5 years (range 1-59 years), and median age of epilepsy onset was younger in the Dravet group (4.9 [3.6-6] months) than non-Dravet (17.9 [6-42.3], P < 0.001). Median follow-up time STP was 24.1 months (2 years; range 0.3-164 months) and was longer in the Dravet group (35.9 months; range 0.8-164) than non-Dravet (17 months range 0.3-62.3, P < 0.001). At 12 months, retention rate, responder rate and seizure free rate was 68.3% (56/82), 65% [48-77%] and 18% [5.7-29%], respectively. There were no statistically significant differences between groups on these seizure outcomes. Adverse events were reported in 46.3% of patients (38/82), without differences between groups. SIGNIFICANCE: In this population of patients with epileptic and developmental encephalopathies, outcomes with adjunctive STP were similar in patients with non-Dravet DREE to patients with Dravet syndrome.

Indexed as

DioxolanesEpilepsies, MyoclonicAdolescentAdultAnticonvulsantsChildChild, PreschoolHumansInfantMiddle AgedRetrospective StudiesSeizuresYoung AdultAnticonvulsantsDioxolanesstiripentolantiseizure medicationdevelopmental and epileptic encephalopathyDravet syndromestatus epilepticusstiripentol

Identifiers

PMID37867433
PMCPMC10839358
OpenAlexW4387866136

What OpenQuestion holds

Textmetadata
LicenceCC BY-NC-ND
Read underepoch 390

Registered trials

None linked

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.