ArticleJournal of the American Academy of Dermatology2015
Improvement of tuberous sclerosis complex (TSC) skin tumors during long-term treatment with oral sirolimus.
Article in Journal of the American Academy of Dermatology, 2015. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 27 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
27 citing papers in PubMed, 52 citations in OpenAlex.
- Tuberous sclerosis complex.Nature reviews. Disease primers · 2026Review
- Cutaneous manifestations-associated with tuberous sclerosis complex and the use of topical rapamycin in the United States: a sub-analysis of an international survey of caregivers and patients.Orphanet journal of rare diseases · 2025Article
- The role and mechanism of TSC in kidney diseases: a literature review.BMC nephrology · 2025Review
- Insights into Tuberous Sclerosis Complex : From Genes to Clinics.Journal of Korean Neurosurgical Society · 2025Article
- Tuberous Sclerosis Complex: New Insights into Pathogenesis and Therapeutic Breakthroughs.Life (Basel, Switzerland) · 2025Review
- Canagliflozin as a Potential Preclinical Therapy for Tuberous Sclerosis Complex: Inhibition ofDrug design, development and therapy · 2025Article
- Case Report: Tuberous sclerosis complex-associated hemihypertrophy successfully treated with mTOR inhibitor sirolimus.Frontiers in pediatrics · 2024Article
- Review
- Characterization and management of facial angiofibroma related to tuberous sclerosis complex in the United States: retrospective analysis of the natural history database.Orphanet journal of rare diseases · 2022Article
- Efficacy, Retention and Tolerability of Everolimus in Patients with Tuberous Sclerosis Complex: A Survey-Based Study on Patients' Perspectives.CNS drugs · 2021Article
- Long-Term Effects of Sirolimus on Human Skin TSC2-Null Fibroblast‒Like Cells.The Journal of investigative dermatology · 2021Article
- Multiple Koenen Tumors, a Rare Entity: Combination Treatment with 1% Topical Sirolimus Electrofulguration and Excision.Skin appendage disorders · 2021Article
- Population Pharmacokinetics and Initial Dose Optimization of Sirolimus Improving Drug Blood Level for Seizure Control in Pediatric Patients With Tuberous Sclerosis Complex.Frontiers in pharmacology · 2021Article
- Initial Dosage Recommendation for Sirolimus in Children With Tuberous Sclerosis Complex.Frontiers in pharmacology · 2020Article
- mTOR-dependent upregulation of xCT blocks melanin synthesis and promotes tumorigenesis.Cell death and differentiation · 2019Article
- The Lymphangioleiomyomatosis Lung Cell and Its Human Cell Models.American journal of respiratory cell and molecular biology · 2018Review
- Tuberous sclerosis complex: review based on new diagnostic criteria.Anais brasileiros de dermatologia · 2018Review
- Fibrous Plaque of the Eyelid in a Patient with Tuberous Sclerosis Responding to Everolimus.Annals of dermatology · 2018Article
- Sclerotic bone lesions as a potential imaging biomarker for the diagnosis of tuberous sclerosis complex.Scientific reports · 2018Article
- Cutaneous manifestations of tuberous sclerosis complex and the paediatrician's role.Archives of disease in childhood · 2017Review
Corrections and comments
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Authors and funding
7 authors at 3 institutions in 1 country.
Funding
Abstract
backgroundOral mechanistic target of rapamycin inhibitors have been shown to reduce visceral tumor volume in patients with tuberous sclerosis complex (TSC).
objectiveWe sought to evaluate the cutaneous response to oral sirolimus in patients with TSC and an indication for systemic treatment, including long-term effects.
methodsA retrospective analysis of 14 adult patients with TSC prescribed sirolimus to treat lymphangioleiomyomatosis was performed. Serial photographs of angiofibromas, shagreen patches, and ungual fibromas taken before, during, and after the treatment period were blinded, then assessed using the Physician Global Assessment of Clinical Condition (PGA). Microscopic and molecular studies were performed on skin tumors harvested before and during treatment.
resultsSirolimus significantly improved angiofibromas (median treatment duration 12 months; median PGA score 4.5 [range 1.5-5]; Wilcoxon signed rank test, P = .018) and shagreen patches (median treatment duration 10 months; median PGA score 4.5 [range 3.5-5]; Wilcoxon signed rank test, P = .039), whereas ungual fibromas improved in some patients (median treatment duration 6.5 months; median PGA score 4.66 [range 2.75-5]; Wilcoxon signed rank test, P = .109). Clinical, immunohistochemical, or molecular evidence of resistance was not observed (range 5-64 months of treatment). LIMITATIONS: This was a retrospective analysis limited to adult women with lymphangioleiomyomatosis.
conclusionOral sirolimus is an effective long-term therapy for TSC skin tumors, particularly angiofibromas, in patients for whom systemic treatment is indicated.
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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.