ReviewItalian journal of pediatrics2013
Is mTOR inhibition a systemic treatment for tuberous sclerosis?
Review in Italian journal of pediatrics, 2013. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT03767660 (Efficacy of Rapamycin), which is not on this map. Cited by 20 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.
Efficacy of Rapamycin (Sirolimus) in the Treatment of Blue Rubber Bleb Nevus Syndrome, Hereditary or Sporadic Venous Malformation
Who cites it
20 citing papers in PubMed, 54 citations in OpenAlex.
- Long-Term Use of Everolimus in Patients with Tuberous Sclerosis Complex: Final Results from the EXIST-1 Study.PloS one · 2016Trial
- Review
- A Case Report of Pediatric Patient with Tuberous Sclerosis and Radiologically Isolated Syndrome.Journal of clinical medicine · 2023Article
- Subependymal Giant Cell Astrocytomas in Tuberous Sclerosis Complex-Current Views on Their Pathogenesis and Management.Journal of clinical medicine · 2023Review
- The Efficacy and Safety of Rapamycin in Children with Tuberous Sclerosis: A Cross-sectional Study.Iranian journal of child neurology · 2023Article
- Trends in survival and treatment of SEGA: National Cancer Database Analysis.Neuro-oncology practice · 2021Article
- Innovative Strategies for the Management of a Massive Neonatal Rhabdomyoma.Journal of pediatric intensive care · 2018Article
- Observational
- Successful use of sirolimus for refractory atrial ectopic tachycardia in a child with cardiac rhabdomyoma.Annals of noninvasive electrocardiology : the official journal of the International Society for Holter and Noninvasive Electrocardiology, Inc · 2017Article
- Novel therapeutic approaches for disease-modification of epileptogenesis for curing epilepsy.Biochimica et biophysica acta. Molecular basis of disease · 2017Review
- Insights into molecular therapy of glioma: current challenges and next generation blueprint.Acta pharmacologica Sinica · 2017Review
- Unique findings of subependymal giant cell astrocytoma within cortical tubers in patients with tuberous sclerosis complex: a histopathological evaluation.Child's nervous system : ChNS : official journal of the International Society for Pediatric Neurosurgery · 2017Article
- mTOR-Dependent Cell Proliferation in the Brain.BioMed research international · 2017Review
- Efficacy and safety of Everolimus in children with TSC - associated epilepsy - Pilot data from an open single-center prospective study.Orphanet journal of rare diseases · 2016Article
- Renal involvement in tuberous sclerosis complex with emphasis on cystic lesions.La Radiologia medica · 2016Observational
- Review
- Recent treatment advances and novel therapeutic approaches in epilepsy.F1000prime reports · 2015Review
- Angiomyolipomas, renal cell carcinomas and pulmonary lymphangioleiomyomatosis.Journal of clinical and diagnostic research : JCDR · 2014Article
- Treating epilepsy in tuberous sclerosis with everolimus: getting closer.Epilepsy currents · 2014Article
- Implementing a Multidisciplinary Approach to Treating Tuberous Sclerosis Complex: A Case Report.Child neurology openArticle
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
4 authors at 2 institutions in 1 country.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Tuberous sclerosis complex (TSC) is a genetic multisystem disorder characterized by the development of hamartomas in several organs. Mutations in the TSC1 and TSC2 tumor suppressor genes determin overactivation of the mammalian target of rapamycin (mTOR) signaling pathway and subsequent abnormalities in numerous cell processes. As a result, mTOR inhibitors such as sirolimus and everolimus have the potential to provide targeted therapy for TSC patients. Everolimus has been recently approved as a pharmacotherapy option for TSC patients with subependymal giant-cell astrocytomas (SEGAs) or renal angiomyolipomas (AMLs). However, clinical evidence suggests that this treatment can benefit other TSC-associated disease manifestations, such as skin manifestations, pulmonary lymphangioleiomyomatosis, cardiac rhabdomyomas, and epilepsy. Therefore, the positive effects that mTOR inhibition have on a wide variety of TSC disease manifestations make this a potential systemic treatment option for this genetic multifaceted disorder.
Indexed as
Identifiers
What OpenQuestion holds
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.