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Literature review of international mammalian target of rapamycin inhibitor use in the non-surgical management of haemodynamically significant cardiac rhabdomyomas

Published online by Cambridge University Press:  11 June 2020

Aoife Cleary Open the ORCID record for Aoife Cleary [Opens in a new window]  and
Colin J. McMahon Open the ORCID record for Colin J. McMahon [Opens in a new window]
Aoife Cleary
Affiliation:
Department of Paediatric Cardiology, Evelina London Children’s Hospital, SE1 7EHEngland, UK
Colin J. McMahon*
Affiliation:
Children’s Health Ireland, Crumlin, Dublin 12, Ireland
*
Author for correspondence: Prof. Colin McMahon, MD, MHPE, FACC, Department of Paediatric Cardiology, Children’s Health Ireland, Crumlin, Dublin 12, Ireland. Tel: +3531 4096160; Fax: +35314096181. E-mail: [email protected]
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Abstract

Cardiac rhabdomyomas represent the most common primary paediatric cardiac tumour and typically regresses over time in the majority of patients. Among those who are symptomatic, surgical resection or catheterisation procedures have traditionally proven effective. More recently, those invasive or challenging tumours have been successfully treated with mammalian target of rapamycin inhibitors, typically everolimus and sirolimus. This review outlines the current medical literature of the state-of-the-art medical treatment of these tumours. We specifically focus on dosing regimens, duration of therapy, and side-effect profiles of mammalian target of rapamycin inhibitors among this population. Although the majority of cases responded to mammalian target of rapamycin inhibition, standardised guidelines for dosing and duration of treatment remain to be defined.

Keywords

Cardiac tumoureverolimusrhabdomyomasirolimustuberous sclerosis
Type
Review Article
Information
Cardiology in the Young , Volume 30 , Issue 7 , July 2020 , pp. 923 - 933
Copyright
© The Author(s), 2020. Published by Cambridge University Press

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