Alpelisib in pediatric PIK3CA- and TIE-2–mutant vascular anomalies: a case series on safety, efficacy, and drug exposure

Abstract excerpt

Alpelisib was recently approved by the FDA for the management of pediatric patients with PIK3CA-related overgrowth spectrum. However, this medication was approved in the absence of pediatric pharmacokinetic data, as a fixed 50 mg dose, with no consideration of weight, the primary pharmacokinetically relevant covariate. This raises concerns regarding potential under and over-exposure. Given this gap in...

Topics

Share this publication in a Topic to start or enrich a Post.

• Share to Platelet Disorders and Treatments
• Share to Vascular Malformations and Hemangiomas
• Share to Vascular Tumors and Angiosarcomas