SynthesisOrphanet journal of rare diseases2019
Efficacy and safety of mTOR inhibitors (rapamycin and its analogues) for tuberous sclerosis complex: a meta-analysis.
Synthesis in Orphanet journal of rare diseases, 2019. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 35 papers.
What it found
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Who cites it
35 citing papers in PubMed, 78 citations in OpenAlex.
- A randomized controlled trial of everolimus for neurocognitive symptoms in PTEN hamartoma tumor syndrome.Human molecular genetics · 2022Trial
- Sirolimus excretion in breast milk: a preliminary investigation.Frontiers in pharmacology · 2026Article
- Nanotechnology strategies for endometrium health: Are we on the right track?Bioactive materials · 2025Review
- Antiseizure Medications: Advancements, Challenges, and Prospects in Drug Development.Current neuropharmacology · 2025Review
- mTOR signaling is required for phagocyte free radical production, GLUT1 expression, and control ofmBio · 2024Article
- Tuberous Sclerosis Complex and the kidneys: what nephrologists need to know.Jornal brasileiro de nefrologia · 2024Review
- Unveiling the growing significance of metabolism in modulating immune cell function: exploring mechanisms and implications; a review.Annals of medicine and surgery (2012) · 2023Article
- Safety of Sirolimus in Patients with Tuberous Sclerosis Complex under Two Years of Age-A Bicenter Retrospective Study.Journal of clinical medicine · 2023Article
- Is Size All That Matters? New Predictors of Complications and Bleeding in Renal Angiomyolipoma.Research and reports in urology · 2023Article
- GENE TARGET: A framework for evaluating Mendelian neurodevelopmental disorders for gene therapy.Molecular therapy. Methods & clinical development · 2022Review
- Nervous system (NS) Tumors in Cancer Predisposition Syndromes.Neurotherapeutics : the journal of the American Society for Experimental NeuroTherapeutics · 2022Review
- Using AI-Based Evolutionary Algorithms to Elucidate Adult Brain Tumor (Glioma) Etiology Associated with IDH1 for Therapeutic Target Identification.Current issues in molecular biology · 2022Article
- Review
- Transcranial sonography depicts a larger substantia nigra echogenic area in renal transplant patients on calcineurin inhibitors than on rapamycin.BMC nephrology · 2022Article
- The importance of imaging in tuberous sclerosis complex (tsc) in children: Two cases.Radiology case reports · 2022Article
- Review
- Analysis of Clinical Features and Next-Generation Sequencing of 12 Tuberous Sclerosis Families in China.Frontiers in medicine · 2022Article
- Orphan Drug Use in Patients With Rare Diseases: A Population-Based Cohort Study.Frontiers in pharmacology · 2022Article
- Genetic Testing in Pediatric Epilepsy.Indian journal of pediatrics · 2021Review
- Everolimus versus sirolimus for angiomyolipoma associated with tuberous sclerosis complex: a multi-institutional retrospective study in China.Orphanet journal of rare diseases · 2021Article
Corrections and comments
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Authors and funding
8 authors at 2 institutions in 1 country.
Funding
Abstract
backgroundThe treatment of tuberous sclerosis complex (TSC) using mammalian target of rapamycin (mTOR) inhibitors is clinically promising. The aim of the present study was to evaluate the efficacy and safety of mTOR inhibitors for improving the clinical symptoms of TSC.
methodsWe performed a systematic search of major electronic databases (PubMed, EMBASE, Cochrane Library and WanFang, CNKI, and VIP databases) to identify randomized controlled trials (RCTs) and quasi-randomized studies from the date of database inception to November 2017; the Chinese Food and Drug Administration and clinicaltrials.gov were also searched for unpublished studies. The endpoints of the study were the tumor response rate and seizure frequency response rate (the proportion of patients achieving a ≥ 50% reduction relative to the baseline). Two researchers screened articles, assessed the risk of bias and extracted data independently. The included RCTs were analyzed using RevMan 5.3, which was provided by the Cochrane Collaboration.
resultsCompared with the placebo, mTOR inhibitors significantly reduced tumor volume in both angiomyolipoma (AML) (RR = 24.69, 95% CI = 3.51,173.41, P = 0.001) and subependymal giant cell astrocytoma (SEGA) (RR = 27.85, 95% CI = 1.74,444.82, P = 0.02). Compared with the placebo, mTOR inhibitors significantly reduced seizure frequency (RR = 2.12, 95% CI = 1.41,3.19, P = 0.0003). Regarding safety, compared with patients who did not receive mTOR inhibitors, those who did had a higher risk of suffering stomatitis (RR = 3.20, 95% CI = 1.49,6.86, P = 0.003). In contrast, patients who did and did not receive mTOR inhibitors experienced similar adverse events, such as upper respiratory tract infections (RR = 1.08, 95% CI = 0.81,1.45, P = 0.59) and nasopharyngitis (RR = 0.86, 95% CI = 0.60,1.21, P = 0.38).
conclusionIn view of the efficacy and safety associated with tumor and seizure frequency in the TSC patients, mTOR inhibitors is a good therapeutic choice. Unlike the risks of upper respiratory tract infections and nasopharyngitis, mTOR inhibitors seem to increase the risk of stomatitis, mostly grade 1 and 2.
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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.