Evidence map›Paper›PMID 34062963›Full record

SynthesisInternational journal of environmental research and public health2021

Treatment of Cardiac Rhabdomyomas with mTOR Inhibitors in Children with Tuberous Sclerosis Complex-A Systematic Review.

Monika Sugalska, Anna Tomik, Sergiusz Jóźwiak, Bożena Werner

Open access · goldAbstract readSystematic Review
In one paragraph

Synthesis in International journal of environmental research and public health, 2021. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 31 papers, 1 of them a synthesis that pooled it.

0numbers the graph read from it
0cells of the map it votes in
31citing papers in PubMed, 1 pooled it
4.0field-weighted citation impact, top 5% 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

31 citing papers in PubMed, 1 synthesis or guideline pooled it, 55 citations in OpenAlex.

  1. Pooled it
  2. Trial
  3. Early use of everolimus for neonatal cardiac rhabdomyoma: a pharmacotherapeutic case with pharmacist-led monitoring.Saudi pharmaceutical journal : SPJ : the official publication of the Saudi Pharmaceutical Society · 2026
    Article
  4. Article
  5. Impact of prenatal sirolimus on cardiac rhabdomyomas and brain tubers.Ultrasound in obstetrics & gynecology : the official journal of the International Society of Ultrasound in Obstetrics and Gynecology · 2026
    Article
  6. Article
  7. Article
  8. Article
  9. Review
  10. Insights into Tuberous Sclerosis Complex : From Genes to Clinics.Journal of Korean Neurosurgical Society · 2025
    Article
  11. Diagnosis and treatment of cardiac tumors.Medical oncology (Northwood, London, England) · 2025
    Review
  12. Review
  13. Article
  14. Review
  15. Review
  16. Cardiac Tumors: Review.Brazilian journal of cardiovascular surgery · 2024
    Review
  17. Review
  18. Article
  19. Article
  20. Cardiac Masses Discovered by Echocardiogram; What to Do Next?Structural heart : the journal of the Heart Team · 2023
    Review
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

4 authors at 1 institution in 1 country.

Monika SugalskaDepartment of Pediatric Neurology, Medical University of Warsaw, 02-091 Warsaw, Poland.
Anna TomikDepartment of Pediatric Cardiology, Medical University of Warsaw, 02-091 Warsaw, Poland.
Sergiusz JóźwiakDepartment of Pediatric Neurology, Medical University of Warsaw, 02-091 Warsaw, Poland.ORCID 0000-0003-3350-6326
Bożena WernerDepartment of Pediatric Cardiology, Medical University of Warsaw, 02-091 Warsaw, Poland.ORCID 0000-0002-6416-4106
Medical University of Warsaw · PL

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

backgroundCardiac rhabdomyomas (CRs) are the earliest sign of tuberous sclerosis complex (TSC). Most of them spontaneously regress after birth. However, multiple and/or large tumors may result in heart failure or cardiac arrhythmia. Recently, the attempts to treat CRs with mTOR inhibitors (mTORi) have been undertaken. We reviewed the current data regarding the effectiveness and safety of mTORi in the treatment of CRs in children with TSC.

methodsThe review was conducted according to the PRISMA guidelines. Medline, Embase, Cochrane library, and ClinicalTrial.gov databases were searched for original, full-text articles reporting the use of mTORi (everolimus or sirolimus) in the treatment of CRs in children with TSC.

resultsThirty articles describing 41 patients were identified (mostly case reports, no randomized or large cohort studies). Thirty-three children (80.5%) had symptomatic CRs and mTORi therapy resulted in clinical improvement in 30 of them (90.9%). CRs size reduction was reported in 95.1%. Some CRs regrew after mTORi withdrawal but usually without clinical symptoms recurrence. The observed side effects were mostly mild.

conclusionsmTORi may be considered as a temporary and safe treatment for symptomatic CRs in children with TSC, especially in high-risk or inoperable tumors. However, high-quality, randomized trials are still lacking.

Indexed as

RhabdomyomaTuberous SclerosisChildEverolimusHumansNeoplasm Recurrence, LocalTOR Serine-Threonine KinasesEverolimusMTOR protein, humanTOR Serine-Threonine Kinasescardiac rhabdomyomachildreneverolimusheartmTOR inhibitorsirolimustuberous sclerosis complextumor

Identifiers

PMID34062963
PMCPMC8124908
OpenAlexW3157370820

What OpenQuestion holds

Textmetadata
LicenceCC BY
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.