Cancer drug shows promise for rare blood vessel disorders in kids and young adults

This study tests the drug alpelisib, already approved for some breast cancers, in people aged 2 months to 30 years with certain blood vessel growth disorders caused by PIK3CA or TIE2 gene changes. Participants take the drug for 2-3 years and attend up to 16 clinic visits to check if their condition improves through scans, quality-of-life surveys, and clinical exams. The goal is to see if alpelisib can shrink abnormal vessels and ease symptoms, but ongoing treatment is needed.