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Rapid regression of large cardiac rhabdomyomas in neonates after sirolimus therapy

Published online by Cambridge University Press:  13 December 2017

M. David Weiland ,
Kristin Bonello  and
Kevin D. Hill
M. David Weiland*
Affiliation:
Department of Pediatrics, Division of Pediatric Cardiology, Duke University Hospital and Health Center, Durham, NC, United States of America
Kristin Bonello
Affiliation:
Department of Pediatrics, Duke University Hospital and Health System, Durham, NC, United States of America
Kevin D. Hill
Affiliation:
Department of Pediatrics, Division of Pediatric Cardiology, Duke University Hospital and Health Center, Durham, NC, United States of America
*
Correspondence to: M. D. Weiland, MD, Department of Pediatrics, Division of Pediatric Cardiology, Duke University Health System, DUMC Box 3090, Durham, NC 27710, United States of America. Tel: +1 919 684 8111; Fax: +919 681 7892; E-mail: michael.weiland@duke.edu
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Abstract

Cardiac rhabdomyomas are the most common tumours in children and are typically seen in association with the tuberous sclerosis complex. Although benign and often associated with spontaneous regression, in rare circumstances surgical resection is indicated to relieve obstruction or other mass-related effects. Recent clinical trials have demonstrated the benefits of mammalian target of rapamycin inhibitors for the treatment of other tumour sub-types associated with tuberous sclerosis. Here we report rapid regression of several massive cardiac rhadomyomas in two neonates with the use of the mammalian target of rapamycin inhibitor sirolimus.

Keywords

Rhabdomyomasirolimustuberous sclerosis complex
Type
Brief Report
Information
Cardiology in the Young , Volume 28 , Issue 3 , March 2018 , pp. 485 - 489
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Copyright
© Cambridge University Press 2017 

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