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

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  • Monika Sugalska

    (Department of Pediatric Neurology, Medical University of Warsaw, 02-091 Warsaw, Poland)

  • Anna Tomik

    (Department of Pediatric Cardiology, Medical University of Warsaw, 02-091 Warsaw, Poland)

  • Sergiusz Jóźwiak

    (Department of Pediatric Neurology, Medical University of Warsaw, 02-091 Warsaw, Poland)

  • Bożena Werner

    (Department of Pediatric Cardiology, Medical University of Warsaw, 02-091 Warsaw, Poland)

Abstract

Background: Cardiac 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. Methods: The 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. Results: Thirty 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. Conclusions: mTORi 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.

Suggested Citation

  • Monika Sugalska & Anna Tomik & Sergiusz Jóźwiak & Bożena Werner, 2021. "Treatment of Cardiac Rhabdomyomas with mTOR Inhibitors in Children with Tuberous Sclerosis Complex—A Systematic Review," IJERPH, MDPI, vol. 18(9), pages 1-22, May.
    • Handle: RePEc:gam:jijerp:v:18:y:2021:i:9:p:4907-:d:548897
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    References listed on IDEAS

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    1. David Moher & Alessandro Liberati & Jennifer Tetzlaff & Douglas G Altman & The PRISMA Group, 2009. "Preferred Reporting Items for Systematic Reviews and Meta-Analyses: The PRISMA Statement," PLOS Medicine, Public Library of Science, vol. 6(7), pages 1-6, July.
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