A Dramatic Response to Sirolimus Therapy in a Premature Infant With Massive Cardiac Rhabdomyoma

Claire Lawley; Himanshu Popat; Melanie Wong; Nadia Badawi; Julian Ayer

doi:10.1016/j.jaccas.2019.07.030

A Dramatic Response to Sirolimus Therapy in a Premature Infant With Massive Cardiac Rhabdomyoma

JACC Case Rep. 2019 Oct 16;1(3):327-331. doi: 10.1016/j.jaccas.2019.07.030. eCollection 2019 Oct.

Authors

Claire Lawley^{1

2}, Himanshu Popat^{1

3}, Melanie Wong^{1

4}, Nadia Badawi^{1

3

5}, Julian Ayer^{1

2}

Affiliations

¹ Discipline of Child and Adolescent Health, The Children's Hospital at Westmead Clinical School, Sydney Medical School, Sydney, New South Wales, Australia.
² The Heart Centre for Children, The Children's Hospital at Westmead, Sydney Children's Hospital Network, Sydney, New South Wales, Australia.
³ Grace Centre for Newborn Intensive Care, The Children's Hospital at Westmead, Westmead, New South Wales, Australia.
⁴ Department of Immunology, The Children's Hospital at Westmead, Sydney Children's Hospital Network, Westmead, New South Wales, Australia.
⁵ Cerebral Palsy Alliance Research Institute, Allambie Heights, New South Wales, Australia.

Abstract

Cardiac rhabdomyomas in neonates may cause significant cardiac risk. Recently, sirolimus has been used to treat these lesions. The dose, duration, and monitoring for therapy are unknown. A case of sirolimus use in a premature neonate is presented. No significant adverse effects were seen. Review of published cases is included. (Level of Difficulty: Advanced.).

Keywords: LVOT, left ventricular outflow tract; TSC, tuberous sclerosis complex; cancer; congenital heart defect; echocardiography; mTOR, mammalian target of rapamycin.

Publication types

Case Reports