Key result
Mammalian target of rapamycin inhibitors (everolimus and sirolimus) successfully treat haemodynamically significant cardiac rhabdomyomas, though standardized dosing guidelines are lacking.
Why the study?
Standardised guidelines for the dosing and duration of mammalian target of rapamycin inhibitor therapy in symptomatic cardiac rhabdomyomas remain to be defined.
Do mammalian target of rapamycin (mTOR) inhibitors effectively treat haemodynamically significant cardiac rhabdomyomas in pediatric patients?
Do mammalian target of rapamycin (mTOR) inhibitors effectively treat haemodynamically significant cardiac rhabdomyomas in pediatric patients?
mTOR inhibitors such as everolimus and sirolimus are effective for treating invasive or challenging cardiac rhabdomyomas in pediatric patients, though standardized dosing guidelines are still needed.
Supports mTOR inhibitor use for symptomatic cardiac rhabdomyomas; leaves open standardized dosing and prospective trials.
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.
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Cleary et al. (2020) conducted a review in Haemodynamically significant cardiac rhabdomyomas. Mammalian target of rapamycin (mTOR) inhibitors (everolimus and sirolimus) was evaluated. Mammalian target of rapamycin inhibitors (everolimus and sirolimus) successfully treat haemodynamically significant cardiac rhabdomyomas, though standardized dosing guidelines are lacking.
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