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

Pediatrics
Denise M AdamsRichard G Azizkhan

Abstract

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. 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. Sixty-one patients were enrolled; 57 patients were evaluable for efficacy at the end of course 6, and 53 were evaluable at the end of course 12. No patient had a complete response at the end of course 6 or 12 as anticipated. At the end of course 6, a total of 47 patients had a partial response, 3 patients had stable disease, and 7 patients had progressive disease. Two patients were taken off of study medicine secondary to persistent adverse effects. Grade 3 and higher toxicities attributable to sirolimus includ...Continue Reading

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Citations

Jun 9, 2016·Pediatric Blood & Cancer·Ralph SalloumDenise M Adams
Jun 3, 2016·International Journal of Cancer. Journal International Du Cancer·Xiao Han LiuJia Wei Zheng
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Apr 7, 2017·Pediatric Dermatology·Verónica Alonso ArroyoIsrael Fernandez-Pineda
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