Pediatr Res. 2025 Apr 26. doi: 10.1038/s41390-025-04043-8. Online ahead of print.
ABSTRACT
BACKGROUND AND OBJECTIVES: Cardiac 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.
METHODS: This 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.
RESULTS: Thirty-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.
CONCLUSIONS: mTOR 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.
PMID:40287604 | DOI:10.1038/s41390-025-04043-8
