Trial reportPediatric nephrology (Berlin, Germany)2018
The effect of everolimus on renal angiomyolipoma in pediatric patients with tuberous sclerosis being treated for subependymal giant cell astrocytoma.
Trial report in Pediatric nephrology (Berlin, Germany), 2018. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 22 papers, 1 of them a synthesis that pooled it.
What it found
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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
22 citing papers in PubMed, 1 synthesis or guideline pooled it, 43 citations in OpenAlex.
- Rapamycin and rapalogs for tuberous sclerosis complex.The Cochrane database of systematic reviews · 2023Pooled it
- The Burden of Epilepsy: The Contribution of Seizures (AES 2025 Presidential Symposium).Epilepsy currents · 2026Article
- Features and management of kidney manifestations of tuberous sclerosis complex: a real-world study in a Brazilian patient cohort.BMC nephrology · 2026Observational
- Successful Treatment of Growing Renal Angiomyolipomas With Low-Dose Everolimus in a School-Aged Child With Tuberous Sclerosis Complex.Nephrology (Carlton, Vic.) · 2025Article
- Sirolimus can promote the disappearance of renal angiomyolipoma associated with tuberous sclerosis complex: a prospective cohort study.World journal of pediatrics : WJP · 2024Article
- Clinical practice recommendations for kidney involvement in tuberous sclerosis complex: a consensus statement by the ERKNet Working Group for Autosomal Dominant Structural Kidney Disorders and the ERA Genes & Kidney Working Group.Nature reviews. Nephrology · 2024Review
- A case report and review of rheumatoid arthritis co-occurring with tuberous sclerosis complex, a rare occurrence.Frontiers in immunology · 2024Review
- Tuberous Sclerosis Complex and the kidneys: what nephrologists need to know.Jornal brasileiro de nefrologia · 2024Review
- Review
- MicroRNA Expression Profile in TSC Cell Lines and the Impact of mTOR Inhibitor.International journal of molecular sciences · 2022Article
- Perivascular epithelioid cell tumor of the lung: A case report and literature review.Thoracic cancer · 2022Review
- mTOR Inhibitor Treatment in Patients with Tuberous Sclerosis Complex Is Associated with Specific Changes in microRNA Serum Profile.Journal of clinical medicine · 2022Article
- Perfect match: mTOR inhibitors and tuberous sclerosis complex.Orphanet journal of rare diseases · 2022Review
- Cutaneous Adverse Events to Targeted Therapies and Immuno-therapies in Children: A Retrospective Study of 103 Patients from Two Tertiary Haemato-Oncology Referral Centres.Acta dermato-venereologica · 2021Article
- Renal tumors in tuberous sclerosis complex.Pediatric nephrology (Berlin, Germany) · 2021Review
- The metformin in tuberous sclerosis (MiTS) study: A randomised double-blind placebo-controlled trial.EClinicalMedicine · 2021Article
- The paradox of autophagy in Tuberous Sclerosis Complex.Genetics and molecular biology · 2021Article
- Rare inherited kidney diseases: an evolving field in Nephrology.Jornal brasileiro de nefrologia · 2020Article
- Establishment of a Regional Interdisciplinary Medical System for Managing Patients with Tuberous Sclerosis Complex (TSC).Scientific reports · 2018Article
- Renal progression factors in young patients with tuberous sclerosis complex: a retrospective cohort study.Pediatric nephrology (Berlin, Germany) · 2018Article
Corrections and comments
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Authors and funding
9 authors at 8 institutions in 3 countries.
Funding
No grant is acknowledged in the PubMed record.
Abstract
backgroundPatients with tuberous sclerosis complex (TSC) often have multiple TSC-associated hamartomas, particularly in the brain and kidney.
methodsThis was a post hoc analysis of pediatric patients being treated for subependymal giant cell astrocytomas (SEGAs) during the phase 3, randomized, double-blind, placebo-controlled EXIST-1 trial. Patients were initially randomly assigned to receive everolimus 4.5 mg/m
resultsOverall, this analysis included 33 patients. Renal angiomyolipoma response was achieved by 75.8% of patients (95% confidence interval, 57.7-88.9%), with sustained mean reductions in renal angiomyolipoma volume over nearly 4 years of treatment. In addition, most (≥80%) achieved clinically relevant reductions in angiomyolipoma volume (≥50%), beginning at week 24 and continuing for the remainder of the study. Everolimus was generally well tolerated in this subgroup, with most adverse events being grade 1 or 2 in severity.
conclusionsAlthough everolimus is currently not indicated for this use, this analysis from EXIST-1 demonstrates its long-term efficacy and safety for the treatment of renal angiomyolipoma in pediatric patients undergoing treatment for TSC-associated SEGA.
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