Very low-dose Everolimus therapy diminishes cardiac tumours in tuberous sclerosis complex disease

Abstract

Tuberous sclerosis complex is syndrome that affects several organs. Cardiac manifestations include rhabdomyoma, which could lead to intracardiac obstruction of blood flow. In the present case, the so far lowest documented Everolimus blood level of 2–3 ng/ml led to tumour regression. Repeated Everolimus stopping and restarting for clinical reasons serves as a proof-of-concept for Everolimus therapy in tuberous sclerosis complex.