Ontology highlight
ABSTRACT:
SUBMITTER: Singh S
PROVIDER: S-EPMC10841299 | biostudies-literature | 2024 Jan
REPOSITORIES: biostudies-literature
Singh Sonia S Bradford Diana D Li Xiaoxue X Mishra-Kalyani Pallavi S PS Shen Yuan-Li YL Wang Lingshan L Zhao Hong H Xiong Ye Y Liu Jiang J Charlab Rosane R Kraft Jeffrey J Khasar Sachia S Miller Claudia P CP Rivera Donna R DR Kluetz Paul G PG Pazdur Richard R Beaver Julia A JA Singh Harpreet H Donoghue Martha M
Clinical cancer research : an official journal of the American Association for Cancer Research 20240101 1
On April 5, 2022, FDA granted accelerated approval to alpelisib for the treatment of adult and pediatric patients 2 years of age and older with severe manifestations of PIK3CA-related overgrowth spectrum (PROS) who require systemic therapy. Efficacy was evaluated using real-world data (RWD) from EPIK-P1 (NCT04285723), a single-arm clinical study in patients 2 years of age and older with severe or life-threatening PROS who received alpelisib as part of an expanded access program (EAP) for compass ...[more]