Sirolimus for the treatment of complicated vascular anomalies in children

Adrienne M. Hammill*, Marysue Wentzel, Anita Gupta, Stephen Nelson, Anne Lucky, Ravi Elluru, Roshni Dasgupta, Richard G. Azizkhan, Denise M. Adams

*Corresponding author for this work

Research output: Contribution to journalArticlepeer-review

473 Scopus citations

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. Procedure: 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.

Original languageEnglish (US)
Pages (from-to)1018-1024
Number of pages7
JournalPediatric Blood and Cancer
Volume57
Issue number6
DOIs
StatePublished - Dec 1 2011
Externally publishedYes

Keywords

  • Kaposiform hemangioendothelioma
  • Kasabach-Merritt phenomenon
  • Lymphatic malformation
  • Rapamycin
  • Sirolimus
  • Vascular anomalies
  • Vascular malformations

ASJC Scopus subject areas

  • Hematology
  • Oncology
  • Pediatrics, Perinatology, and Child Health

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