CC BY-NC-ND 4.0 · AJP Rep 2019; 09(03): e213-e217
DOI: 10.1055/s-0039-1692198
Case Report
Thieme Medical Publishers 333 Seventh Avenue, New York, NY 10001, USA.

Effect and Complications of Everolimus Use for Giant Cardiac Rhabdomyomas with Neonatal Tuberous Sclerosis

Yuka Shibata
1  Division of Neonatology, Center for Maternal-Fetal, Neonatal and Reproductive Medicine, Setagaya, Tokyo, Japan
,
Hidehiko Maruyama
1  Division of Neonatology, Center for Maternal-Fetal, Neonatal and Reproductive Medicine, Setagaya, Tokyo, Japan
,
Taiyu Hayashi
2  Division of Cardiology, National Center for Child Health and Development, Setagaya, Tokyo, Japan
,
Hiroshi Ono
2  Division of Cardiology, National Center for Child Health and Development, Setagaya, Tokyo, Japan
,
Yuka Wada
1  Division of Neonatology, Center for Maternal-Fetal, Neonatal and Reproductive Medicine, Setagaya, Tokyo, Japan
,
Hideshi Fujinaga
1  Division of Neonatology, Center for Maternal-Fetal, Neonatal and Reproductive Medicine, Setagaya, Tokyo, Japan
,
Shuhei Fujino
1  Division of Neonatology, Center for Maternal-Fetal, Neonatal and Reproductive Medicine, Setagaya, Tokyo, Japan
,
Junko Nagasawa
1  Division of Neonatology, Center for Maternal-Fetal, Neonatal and Reproductive Medicine, Setagaya, Tokyo, Japan
,
Shoichiro Amari
1  Division of Neonatology, Center for Maternal-Fetal, Neonatal and Reproductive Medicine, Setagaya, Tokyo, Japan
,
Keiko Tsukamoto
1  Division of Neonatology, Center for Maternal-Fetal, Neonatal and Reproductive Medicine, Setagaya, Tokyo, Japan
,
Yushi Ito
1  Division of Neonatology, Center for Maternal-Fetal, Neonatal and Reproductive Medicine, Setagaya, Tokyo, Japan
› Author Affiliations
Funding No financial assistance was received to support this study.
Further Information

Publication History

08 November 2018

05 March 2019

Publication Date:
11 July 2019 (online)

Abstract

Most cardiac rhabdomyomas with tuberous sclerosis (TS) are asymptomatic and spontaneously regress. However, some cases require surgical intervention due to arrhythmia and severe obstruction of cardiac inflow or outflow. We report herein a neonatal case of giant cardiac rhabdomyomas with TS and insufficient pulmonary blood flow from the right ventricle. Lipoprostaglandin E1 was necessary to maintain patency of the ductus arteriosus. We used everolimus, a mammalian target of rapamycin inhibitor, to diminish the cardiac rhabdomyomas. After treatment, the rhabdomyomas shrank rapidly, but the serum concentration of everolimus increased sharply (maximum serum trough level: 76.1 ng/mL) and induced complications including pulmonary hemorrhage, liver dysfunction, and acne. After the everolimus level decreased, the complications resolved. Everolimus may be a viable treatment option for rhabdomyomas, but its concentration requires close monitoring to circumvent complications associated with its use.

Ethical Approval

All the procedures involving human participants were performed in accordance with the ethical standards of the institution and with the 1964 Helsinki declaration and its later amendments. The approval of the ethics review committee and parental consent to use everolimus were obtained. Parental written informed consent was obtained for this report.