title: Rapid resolution of cardiac rhabdomyomas following everolimus therapy.
abstract: 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.
authors.affiliation: Department of Pediatric Cardiology, Washington University School of Medicine, St Louis, Missouri, USA.
authors.forename: Swati
authors.initials: S
authors.lastname: Choudhry
authors.affiliation: Department of Pediatric Cardiology, Washington University School of Medicine, St Louis, Missouri, USA.
authors.forename: Hoang Hiep
authors.initials: HH
authors.lastname: Nguyen
authors.affiliation: Department of Pediatric Cardiology, Washington University School of Medicine, St Louis, Missouri, USA.
authors.forename: Shafkat
authors.initials: S
authors.lastname: Anwar
mesh_terms: D000970:Antineoplastic Agents / Q000627:therapeutic use*; D000068338:Everolimus / Q000627:therapeutic use*; D006338:Heart Neoplasms / Q000150:complications / Q000188:drug therapy*; D006801:Humans; D007231:Infant, Newborn; D008297:Male; D012207:Rhabdomyoma / Q000150:complications / Q000188:drug therapy*; D016896:Treatment Outcome
chemical_list: D000970:Antineoplastic Agents; D000068338:Everolimus