Efficacy and safety of sirolimus in the treatment of complicated vascular anomalies
DM Adams, CC Trenor, AM Hammill, AA Vinks… - …, 2016 - publications.aap.org
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-
one patients were enrolled; 57 patients were evaluable for efficacy at the end of course 6 …
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-
one patients were enrolled; 57 patients were evaluable for efficacy at the end of course 6 …