ArticleIranian journal of child neurology2023
The Efficacy and Safety of Rapamycin in Children with Tuberous Sclerosis: A Cross-sectional Study.
Article in Iranian journal of child neurology, 2023. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 2 papers.
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.
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Who cites it
2 citing papers in PubMed, 2 citations in OpenAlex.
- mTORC1 inhibition by sirolimus as adjunctive treatment in experimental pneumococcal meningitis.Brain communications · 2026Article
- Developmental mechanisms underlying pediatric epilepsy.Frontiers in neurology · 2025Review
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Authors and funding
5 authors at 1 institution in 1 country.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Objectives: Mutations in TSC1 or TSC2 genes have been proposed as the main causative factors responsible for developing Tuberous Sclerosis Complex (TSC). Given the effect of these two genes on the mTOR pathway, rapamycin has emerged as a novel therapeutic agent. The present study evaluated the effectiveness and safety of rapamycin on the multiple manifestations of TSC. Materials & Methods: Twenty-three eligible children were enrolled in the present cross-sectional study. They were prescribed rapamycin 1mg tablet twice daily for the first two weeks of treatment and then once daily for at least one year. Periodic evaluations through follow-up visits were performed. Besides, growth and developmental statuses were evaluated. All data, including the number and size of brain tuberomas, size of renal angiomyolipomas, and skin lesions, were gathered and recorded, and then analyzed. Results: During the study period, the mean number of epileptic episodes significantly reduced (p<0.0001), and nine cases were seizure-free at the final visit. The mean number of brain tuberomas decreased from 19.3±11.0 at the initial visit to 11.1±5.6 and 8.2±3.2 in the subsequent visits (p<0.001). The mean size of brain tuberomas similarly decreased from 17.9±18.5 cm at enrollment to 13.7±5.1 cm and 6.9±5.1 cm in the second and third visits, respectively (p=0.029). The mean size of renal angiomyolipomas significantly decreased (p<0.001). A significant trend toward a decrease in the number of skin lesions was observed (p<0.0001). No relationship was observed between the effects of rapamycin and the patient's age or sex (p>0.05). Changes in patients' growth and developmental features were not statistically significant through subsequent visits (p=0.507). Conclusion: This study revealed the effectiveness and safety of rapamycin on TSC among our patients.
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