Trial reportAnnals of neurology2015
Everolimus for subependymal giant cell astrocytoma: 5-year final analysis.
Trial report in Annals of neurology, 2015. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT00411619 (Everolimus), which is not on this map. Cited by 79 papers.
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.
Everolimus (RAD001)Therapy of Giant Cell Astrocytomas in Patients With Tuberous Sclerosis Complex
Who cites it
79 citing papers in PubMed, 171 citations in OpenAlex.
- Phase II Study of Samotolisib in Children and Young Adults With Tumors Harboring Phosphoinositide 3-Kinase/Mammalian Target of Rapamycin Pathway Alterations: Pediatric MATCH APEC1621D.JCO precision oncology · 2024Trial
- Longitudinal Effects of Everolimus on White Matter Diffusion in Tuberous Sclerosis Complex.Pediatric neurology · 2019Trial
- Influence of seizures on early development in tuberous sclerosis complex.Epilepsy & behavior : E&B · 2017Trial
- Everolimus long-term use in patients with tuberous sclerosis complex: Four-year update of the EXIST-2 study.PloS one · 2017Trial
- Trial
- Long-Term Use of Everolimus in Patients with Tuberous Sclerosis Complex: Final Results from the EXIST-1 Study.PloS one · 2016Trial
- The Burden of Epilepsy: The Contribution of Seizures (AES 2025 Presidential Symposium).Epilepsy currents · 2026Article
- Genotype and subependymal lesion burden influence volumetric response to everolimus in tuberous sclerosis complex-associated subependymal giant cell astrocytoma: A 10-year real-world study.Neurotherapeutics : the journal of the American Society for Experimental NeuroTherapeutics · 2026Article
- Recent Advances in Targeted Therapies for Adult Gliomas.Current neurology and neuroscience reports · 2026Review
- Neurogenetic tumor syndromes: The current landscape of workup and treatment.Neuro-oncology practice · 2026Review
- mTOR inhibitor everolimus versus conventional chemotherapy in pediatric low-grade gliomas.American journal of cancer research · 2026Article
- The Role of mTOR Signaling in Tumor-Induced Alterations to Neuronal Function in Diffusely Infiltrating Glioma.Biomedicines · 2025Review
- Late failure of Everolimus in a patient with tuberous sclerosis complex after 10 years of effective response: an illustrative case.Child's nervous system : ChNS : official journal of the International Society for Pediatric Neurosurgery · 2025Article
- Exploring the effects of ROS on PI3K/AKT/mTOR signalling in pediatric low-grade glioma and therapeutic strategies.Molecular biology reports · 2025Review
- A bitopic mTORC inhibitor reverses phenotypes in a tuberous sclerosis complex model.Scientific reports · 2025Article
- Precision Medicine for Pediatric Glioma and NF1-Associated Tumors: The Role of Small Molecule Inhibitors.Current oncology (Toronto, Ont.) · 2025Review
- Surgical treatment of subependymal giant cell astrocytoma in patients with tuberous sclerosis complex-an institutional experience and results.Child's nervous system : ChNS : official journal of the International Society for Pediatric Neurosurgery · 2025Article
- Tuberous Sclerosis Complex: New Insights into Pathogenesis and Therapeutic Breakthroughs.Life (Basel, Switzerland) · 2025Review
- New insights into tuberous sclerosis complex: from structure to pathogenesis.Frontiers in cell and developmental biology · 2025Review
- Review
19 more citing papers are in PubMed but not listed here.
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
10 authors at 4 institutions in 2 countries.
Funding
No grant is acknowledged in the PubMed record.
Abstract
objectiveTo analyze the cumulative efficacy and safety of everolimus in treating subependymal giant cell astrocytomas (SEGA) associated with tuberous sclerosis complex (TSC) from an open-label phase II study (NCT00411619). Updated data became available from the conclusion of the extension phase and are presented in this ≥5-year analysis.
methodsPatients aged ≥ 3 years with a definite diagnosis of TSC and increasing SEGA lesion size (≥2 magnetic resonance imaging scans) received everolimus starting at 3mg/m(2) /day (titrated to target blood trough levels of 5-15ng/ml). The primary efficacy endpoint was reduction from baseline in primary SEGA volume.
resultsAs of the study completion date (January 28, 2014), 22 of 28 (78.6%) initially enrolled patients finished the study per protocol. Median (range) duration of exposure to everolimus was 67.8 (4.7-83.2) months; 12 (52.2%) and 14 (60.9%) of 23 patients experienced SEGA volume reductions of ≥50% and ≥30% relative to baseline, respectively, after 60 months of treatment. The proportion of patients experiencing daily seizures was reduced from 7 of 26 (26.9%) patients at baseline to 2 of 18 (11.1%) patients at month 60. Most commonly reported adverse events (AEs) were upper respiratory tract infection and stomatitis of mostly grade 1 or 2 severity. No patient discontinued treatment due to AEs. The frequency of emergence of most AEs decreased over the course of the study.
interpretationEverolimus continues to demonstrate a sustained effect on SEGA tumor reduction over ≥5 years of treatment. Everolimus remained well-tolerated, and no new safety concerns were noted.
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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.