UCSF

Cardiac rhabdomyoma is the most common primary cardiac tumour during childhood and is usually associated with tuberous sclerosis complex (TSC). These tumours are generally considered benign, and spontaneous regression occurs commonly. However, when the tumours cause significant symptoms, the current standard treatment is surgical resection. Everolimus is an mammalian target of rapamycin (mTOR) complex 1 inhibitor that has been successfully used to treat subependymal giant cell astrocytomas and renal angiomyolipomas associated with TSC. A few case reports have described the effectiveness of everolimus therapy in treating cardiac rhabdomyomas as well. We report a case of a newborn who had near complete resolution of multiple rhabdomyomas within a month of receiving everolimus therapy for non-cardiac masses. To the best of our knowledge, this is the fastest resolution of cardiac rhabdomyomas associated with everolimus therapy to date. Everolimus may be a promising alternative for high-risk surgical candidates with haemodynamically significant cardiac rhabdomyomas.