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Article

Response to Alpelisib in Clinically Distinct Pediatric Patients With PIK3CA -related Disorders.

Journal of pediatric hematology/oncology - 1 Nov 2022

Kolitz Elysha, Fernandes Neil J, Agim Nnenna G, Ludwigl Kathleen

Abstract excerpt

PIK3CA -related disorders include vascular malformations, potential overgrowth of various tissues, limb abnormalities, disordered soft tissue, and/or fatty hyperplasia that often leads to significant morbidity. Alpelisib, a targeted inhibitor of p110α, an enzyme encoded by the PIK3CA gene, has demonstrated success in a cohort of patients with PIK3CA -driven overgrowth syndromes. We describe the clinical course of...

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PMID: 35129141
DOI: 10.1097/MPH.0000000000002418

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