Accelerated regression of cardiac rhabdomyoma by mTOR inhibitors in a neonate with heart failure

Krishna Prasad, Parag Barwad, Pruthvi,C.R, Krishna Santosh, JyothiVijay, SanjeevNaganur.  Accelerated regression of cardiac rhabdomyoma by mTOR inhibitors in a neonate with heart failure: A case report.  Indian Heart Journal, in press.  https://doi.org/10.1016/j.ihjccr.2020.08.008

Abstract

Cardiac Rhabdomyomas (CR) are a rare occurrence with an incidence of 0.1% and are the most common primary cardiac tumors in the fetus and pediatric age groups. Although largely known as “spontaneously regressing tumor” very rarely they cause symptoms of heart failure due to ventricular dysfunction or outflow tract obstruction, or arrhythmias. When associated with tuberous sclerosis complex (TSC) they are usually multiple in number. mTOR inhibitors were recently approved by FDA for the treatment of TSC. We report a case of fetal and neonatal rhabdomyoma presenting as heart failure refractory to medical management and was treated with mTOR inhibitors (Everolimus) with which the patient had improvement in symptoms and accelerated regression of tumor. We conclude that rhabdomyomas though asymptomatic in most, can present with refractory heart failure and mTOR inhibitors can be helpful in such cases.