Evidence map›Paper›PMID 26927950›Full record

ReviewDrugs2016

The Role of mTOR Inhibitors in the Treatment of Patients with Tuberous Sclerosis Complex: Evidence-based and Expert Opinions.

Paolo Curatolo, Marit Bjørnvold, Patricia E Dill, José Carlos Ferreira, Martha Feucht, Christoph Hertzberg, Anna Jansen, Sergiusz Jóźwiak, J Christopher Kingswood, Katarzyna Kotulska and 4 more

Abstract readReview
PubMed Publisher
In one paragraph

Review in Drugs, 2016. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 37 papers, 2 of them syntheses that pooled it.

0numbers the graph read from it
0cells of the map it votes in
37citing papers in PubMed, 2 pooled it
8.4field-weighted citation impact, top 2% of its field
1 · What the graph read from it

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.

2 · The registry

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.

3 · Its place in the literature

Who cites it

37 citing papers in PubMed, 2 syntheses or guidelines pooled it, 78 citations in OpenAlex.

  1. Pooled it
  2. Pooled it
  3. Trial
  4. Article
  5. NEAT1 Promotes Epileptogenesis in Tuberous Sclerosis Complex.Advanced science (Weinheim, Baden-Wurttemberg, Germany) · 2026
    Article
  6. Article
  7. Article
  8. Article
  9. Review
  10. Review
  11. Article
  12. Article
  13. Review
  14. Everolimus as a possible prenatal treatment of in utero diagnosed subependymal lesions in tuberous sclerosis complex: a case report.Child's nervous system : ChNS : official journal of the International Society for Pediatric Neurosurgery · 2021
    Article
  15. Review
  16. Article
  17. Observational
  18. Review
  19. Article
  20. Article
4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

14 authors at 14 institutions in 12 countries.

Paolo CuratoloDepartment of Neurosciences, Child Neurology and Psychiatry Unit, Tor Vergata University Hospital of Rome, Rome, Italy. curatolo@uniroma2.it.
Marit BjørnvoldNational Center for Rare Epilepsy-related Disorders, National Center of Epilepsy, Oslo University Hospital, Oslo, Norway.
Patricia E DillDepartment of Pediatric Neurology and Developmental Medicine, University Children's Hospital Basel, University of Basel, Basel, Switzerland.
José Carlos FerreiraNeuro Pediatra, Centro Hospitalar Lisboa Ocidental, Hospital São Francisco Xavier, Lisbon, Portugal.
Martha FeuchtDepartment of Paediatrics, University Hospital Vienna, Vienna, Austria.
Christoph HertzbergDiagnose und Behandlungszentrum für Kinder und Jugendliche, Vivantes Klinikum Neukölln, Berlin, Germany.
Anna JansenPediatric Neurology Unit-UZ Brussel, Brussels, Belgium.
Sergiusz JóźwiakDepartment of Child Neurology, Medical University of Warsaw, Warsaw, Poland.
J Christopher KingswoodSussex Renal Unit, Royal Sussex County Hospital, Brighton, UK.
Katarzyna KotulskaDepartment of Neurology and Epileptology, The Children's Memorial Health Institute, Warsaw, Poland.
Alfons MacayaServei de Neurologia Pediàtrica, Hospital Universitari Vall d'Hebron, Barcelona, Spain.
Romina MoaveroDepartment of Neurosciences, Child Neurology and Psychiatry Unit, Tor Vergata University Hospital of Rome, Rome, Italy.
Rima NabboutDepartment of Pediatric Neurology, Reference Centre for Rare Epilepsies and Tuberous Sclerosis Complex, Necker-Enfants Malades Hospital, University Paris Descartes, Paris, France.
Bernard A ZonnenbergUniversity Medical Center Utrecht, Utrecht, The Netherlands.
Centro Hospitalar de Lisboa Ocidental · PTChildren's Memorial Health Institute · PLHôpital Necker-Enfants Malades · FRInserm · FRIstituti di Ricovero e Cura a Carattere Scientifico · ITMedical University of Warsaw · PLOslo University Hospital · NORoyal Sussex County Hospital · GBUniversitair Ziekenhuis Brussel · BEUniversity Medical Center Utrecht · NLUniversity of Rome Tor Vergata · ITVall d'Hebron Hospital Universitari · ESVienna General Hospital · ATVivantes Klinikum · DE

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Tuberous sclerosis complex (TSC) is a genetic disorder arising from mutations in the TSC1 or TSC2 genes. The resulting over-activation of the mammalian target of rapamycin (mTOR) signalling pathway leaves patients with TSC susceptible to the growth of non-malignant tumours in multiple organs. Previously, surgery was the main therapeutic option for TSC. However, pharmacological therapy with mTOR inhibitors such as everolimus and sirolimus is now emerging as an alternate approach. Everolimus and sirolimus have already been shown to be effective in treating subependymal giant cell astrocytoma (SEGA) and renal angiomyolipoma (AML), and everolimus is currently being evaluated in treating TSC-related epilepsy. In November 2013 a group of European experts convened to discuss the current options and practical considerations for treating various manifestations of TSC. This article provides evidence-based recommendations for the treatment of SEGA, TSC-related epilepsy and renal AML, with a focus on where mTOR inhibitor therapy may be considered alongside other treatment options. Safety considerations regarding mTOR inhibitor therapy are also reviewed. With evidence of beneficial effects in neurological and non-neurological TSC manifestations, mTOR inhibitors may represent a systemic treatment for TSC.

Indexed as

AngiomyolipomaAstrocytomaEpilepsyHumansKidney NeoplasmsProtein Kinase InhibitorsSignal TransductionTOR Serine-Threonine KinasesTuberous SclerosisProtein Kinase InhibitorsTOR Serine-Threonine Kinases

Identifiers

PMID26927950
OpenAlexW2291704585

What OpenQuestion holds

Textmetadata
Read underepoch 390

Registered trials

None linked

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.