Efficacy and Safety of Sirolimus in the Treatment of Complicated Vascular Anomalies.

ABSTRACT:

Background and objectives

Complicated vascular anomalies have limited therapeutic options and cause significant morbidity and mortality. This Phase II trial enrolled patients with complicated vascular anomalies to determine the efficacy and safety of treatment with sirolimus for 12 courses; each course was defined as 28 days.

Methods

Treatment consisted of a continuous dosing schedule of oral sirolimus starting at 0.8 mg/m(2) per dose twice daily, with pharmacokinetic-guided target serum trough levels of 10 to 15 ng/mL. The primary outcomes were responsiveness to sirolimus by the end of course 6 (evaluated according to functional impairment score, quality of life, and radiologic assessment) and the incidence of toxicities and/or infection-related deaths.

Results

Sixty

SUBMITTER: Adams DM 

PROVIDER: S-EPMC4732362 | biostudies-literature | 2016 Feb

REPOSITORIES: biostudies-literature