Rapid regression of large cardiac rhabdomyomas in neonates after sirolimus therapy

Cardiol Young. 2018 Mar;28(3):485-489. doi: 10.1017/S104795111700244X. Epub 2017 Dec 13.

Abstract

Cardiac rhabdomyomas are the most common tumours in children and are typically seen in association with the tuberous sclerosis complex. Although benign and often associated with spontaneous regression, in rare circumstances surgical resection is indicated to relieve obstruction or other mass-related effects. Recent clinical trials have demonstrated the benefits of mammalian target of rapamycin inhibitors for the treatment of other tumour sub-types associated with tuberous sclerosis. Here we report rapid regression of several massive cardiac rhadomyomas in two neonates with the use of the mammalian target of rapamycin inhibitor sirolimus.

Keywords: Rhabdomyoma; sirolimus; tuberous sclerosis complex.

Publication types

  • Case Reports

MeSH terms

  • Echocardiography
  • Heart Neoplasms / diagnostic imaging
  • Heart Neoplasms / drug therapy*
  • Humans
  • Infant, Newborn
  • Rhabdomyoma / diagnostic imaging
  • Rhabdomyoma / drug therapy*
  • Sirolimus / therapeutic use*
  • Tuberous Sclerosis / complications

Substances

  • Sirolimus