Trial reportNeurology2016
Long-term treatment of epilepsy with everolimus in tuberous sclerosis.
Trial report in Neurology, 2016. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 59 papers, 3 of them syntheses that pooled 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.
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.
Who cites it
59 citing papers in PubMed, 3 syntheses or guidelines pooled it, 157 citations in OpenAlex.
- Everolimus as a therapeutic option in refractory epilepsy in children with tuberous sclerosis: a systematic review.Arquivos de neuro-psiquiatria · 2023Pooled it
- Systematic review of mTOR inhibitor treatment, biomarkers and prophylaxis for tuberous sclerosis complex-associated seizures.Clinical medicine (London, England) · 2022Pooled it
- Efficacy and safety of mTOR inhibitors (rapamycin and its analogues) for tuberous sclerosis complex: a meta-analysis.Orphanet journal of rare diseases · 2019Pooled it
- Influence of seizures on early development in tuberous sclerosis complex.Epilepsy & behavior : E&B · 2017Trial
- Whole-exome sequencing in pediatric drug-resistant epilepsy: diagnostic yield and clinical impact in a resource-limited setting.Neurogenetics · 2026Article
- Prenatal diagnosis of cardiac rhabdomyoma: implications for predicting tuberous sclerosis complex and guiding perinatal management.Ultrasound in obstetrics & gynecology : the official journal of the International Society of Ultrasound in Obstetrics and Gynecology · 2026Article
- Genetic Testing and Counselling for Hereditary Renal Carcinoma: What Do Clinicians Need to Know?European urology open science · 2025Review
- Application of population pharmacokinetic modeling of SVG-101 to evaluate proper dose selection.Translational and clinical pharmacology · 2025Article
- AMPK as a Therapeutic Target: Advancing Epilepsy Management Through Metabolic Modulation.Molecular neurobiology · 2025Review
- Chloride deregulation and GABA depolarization in MTOR-related malformations of cortical development.Brain : a journal of neurology · 2025Article
- The translation initiation factor eIF2α regulates lipid homeostasis and metabolic aging.Aging cell · 2025Article
- An interpretable machine learning approach for predicting drug-resistant epilepsy in children with tuberous sclerosis complex.Frontiers in neurology · 2025Article
- NS-Pten knockout mice exhibit sex and hippocampal subregion-specific increases in microglia/macrophage density.Epilepsy research · 2024Article
- Preventative treatment of tuberous sclerosis complex with sirolimus: Phase I safety and efficacy results.Annals of the Child Neurology Society · 2024Article
- Blazing a trail for the clinical use of rapamycin as a geroprotecTOR.GeroScience · 2023Article
- Understanding the impact of tuberous sclerosis complex: development and validation of the TSC-PROM.BMC medicine · 2023Article
- Targeting the biology of aging with mTOR inhibitors.Nature aging · 2023Review
- Successful Prenatal Treatment of Cardiac Rhabdomyoma in a Fetus with Tuberous Sclerosis.Pediatric reports · 2023Article
- DEPDC5-dependent mTORC1 signaling mechanisms are critical for the anti-seizure effects of acute fasting.Cell reports · 2022Article
- Immunity, Ion Channels and Epilepsy.International journal of molecular sciences · 2022Review
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
9 authors at 1 institution in 1 country.
Funding
Abstract
objectiveTo evaluate the long-term benefit and safety of everolimus for the treatment of medically refractory epilepsy in patients with tuberous sclerosis complex (TSC).
methodsEverolimus was titrated over 4 weeks and continued an additional 8 weeks in a prospective, open-label, phase I/II clinical trial design. Participants demonstrating initial benefit continued treatment until study completion (48 months). The primary endpoint was percentage of patients with a ≥50% reduction in seizure frequency compared to baseline. Secondary endpoints assessed absolute seizure frequency, adverse events (AEs), behavior, and quality of life.
resultsOf the 20 participants who completed the initial study phase, 18 continued extended treatment. Fourteen of 18 (78%) participants completed the study, all but 1 of whom reported ≥50% reduction in seizure frequency at 48 months. All participants reported at least 1 AE, the vast majority (94%) of which were graded mild or moderate severity. Improvements in behavior and quality of life were also observed, but failed to achieve statistical significance at 48 months.
conclusionsImproved seizure control was maintained for 4 years in the majority of patients with TSC with medically refractory epilepsy treated with everolimus. Long-term treatment with everolimus is safe and well-tolerated in this population. Everolimus may be a therapeutic option for refractory epilepsy in TSC. CLASSIFICATION OF EVIDENCE: This study provides Class IV evidence that for patients with TSC with medically refractory epilepsy everolimus improves seizure control.
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What OpenQuestion holds
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.