SynthesisPediatric research2025
Everolimus and sirolimus in the treatment of cardiac rhabdomyomas in neonates.
Synthesis in Pediatric research, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 6 papers, 1 of them a synthesis that pooled it.
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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.
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.
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Who cites it
6 citing papers in PubMed, 1 synthesis or guideline pooled it.
- The dual role of mTOR in multiple sclerosis pathophysiology: a systematic review.Journal of neurology · 2026Pooled it
- Neonatal right ventricular rhabdomyoma: Imaging findings and spontaneous regression.Radiology case reports · 2026Article
- Tuberous sclerosis complex.Nature reviews. Disease primers · 2026Review
- 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 · 2026Article
- Rhabdomyomas of the Mitral Valve: Case Series and Conservative Management Approach.Pediatric cardiology · 2026Article
- Fetal cardiac rhabdomyomas susceptible to prenatal treatment with mTOR inhibitors: literature review and proposal of a prenatal management algorithm.Frontiers in medicine · 2025Review
Corrections and comments
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Authors and funding
5 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
BACKGROUND AND
objectivesCardiac rhabdomyoma (CR) is the principal cardiac tumor diagnosed in pediatric age and is commonly associated with tuberous sclerosis complex. In some patients, these masses can cause heart failure and difficult-to-control arrhythmias. There are multiple case reports on use of mammalian target of rapamycin (mTOR) inhibitors, everolimus or sirolimus, in treatment of CRs. We reviewed the current data regarding effectiveness of everolimus and sirolimus in treating of CRs in newborns with hemodynamic repercussions.
methodsThis systematic review was reported according to the PRISMA guidelines. The EBSCO, PubMed, EMBASE, and Lilacs databases were searched for full-text articles reporting the use of everolimus or sirolimus in the treatment of CRs in neonates and infants.
resultsThirty-one articles met inclusion criteria, totaling 48 patients. Hemodynamic instability prompted treatment in 89.5% of cases. Everolimus was used in 83.3% of cases and sirolimus in 16.6%. The median treatment duration was 67 days, with a 57 ± 23% average CR size reduction. Common adverse events included hypertriglyceridemia, infections, and hematological abnormalities.
conclusionsmTOR inhibitors appear effective and safe for treating CRs in neonates and infants. The average daily doses were 1.03 mg/m²/day for everolimus and 1.37 mg/m²/day for sirolimus. Randomized controlled clinical trials are necessary to confirm these findings and establish optimal treatment protocols. IMPACT: Currently, there are no results from randomized clinical trials evaluating the efficacy of mammalian target of rapamycin inhibitors in patients with symptomatic cardiac rhabdomyomas. This is the first systematic review that evaluates the efficacy and safety of the use of everolimus and sirolimus in the non-surgical treatment of cardiac rhabdomyomas with hemodynamic repercussions in neonates. Everolimus and sirolimus may be particularly useful in the neonatal period when the hemodynamic complications caused by cardiac rhabdomyomas are more severe.
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