title: Sirolimus for the treatment of complicated vascular anomalies in children.
abstract: BACKGROUND
  Vascular anomalies comprise a diverse group of diagnoses. While infantile hemangiomas are common, the majority of these conditions are quite rare and have not been widely studied. Some of these lesions, though benign, can impair vital structures, be deforming, or even become life-threatening. Vascular tumors such as kaposiform hemangioendotheliomas (KHE) and complicated vascular malformations have proven particularly difficult to treat.
  
  
  METHODS
  Here we retrospectively evaluate a series of six patients with complicated, life-threatening vascular anomalies who were treated with the mTOR inhibitor sirolimus for compassionate use at two centers after failing multiple other therapies.
  
  
  RESULTS
  These patients showed significant improvement in clinical status with tolerable side effects.
  
  
  CONCLUSIONS
  Sirolimus appears to be effective and safe in patients with life-threatening vascular anomalies and represents an important tool in treating these diseases. These findings are currently being further evaluated in a Phase II safety and efficacy trial.
authors: Hemangioma and Vascular Malformation Center, Cincinnati Children's Hospital Medical Center, Cincinnati, Ohio, USA. adrienne.hammill@cchmc.org
authors: Adrienne M
authors: AM
authors: Hammill
authors: MarySue
authors: M
authors: Wentzel
authors: Anita
authors: A
authors: Gupta
authors: Stephen
authors: S
authors: Nelson
authors: Anne
authors: A
authors: Lucky
authors: Ravi
authors: R
authors: Elluru
authors: Roshni
authors: R
authors: Dasgupta
authors: Richard G
authors: RG
authors: Azizkhan
authors: Denise M
authors: DM
authors: Adams
mesh_terms: D000293:Adolescent; D002648:Child; D002675:Child, Preschool; D057176:Compassionate Use Trials; D005260:Female; D006801:Humans; D007166:Immunosuppressive Agents / Q000009:adverse effects / Q000627:therapeutic use*; D007223:Infant; D008297:Male; D012189:Retrospective Studies; D020123:Sirolimus / Q000009:adverse effects / Q000627:therapeutic use*; D016896:Treatment Outcome; D054079:Vascular Malformations / Q000150:complications* / Q000188:drug therapy*
chemical_list: D007166:Immunosuppressive Agents; D020123:Sirolimus