At a glance
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Retrospective Chart Review Study of Patients With PIK3CA-Related Overgrowth Spectrum (PROS) Who Have Received Alpelisib as Part of a Compassionate Use Program (EPIK-P1)
In Brief
An observational study evaluating alpelisib for PIK3CA-Related Overgrowth Spectrum (PROS). Completed, enrolled 57 participants across 7 sites in 5 countries.
Detailed Summary
The study was a site-based retrospective non-interventional medical chart review of pediatric and adult male and female patients with PIK3CA-Related Overgrowth Spectrum (PROS) who initiated alpelisib at least 24 weeks before the cut-off date at a MAP site. The study cut-off date was 09-Mar-2020. Patient-level data were abstracted from medical charts of all eligible patients at all participating sites. Study completion date refers to the last date data was extracted. Information from patients treated with alpelisib was used to describe the efficacy and safety of alpelisib in PROS patients.
Study Details
Timeline
Interventions
Retrospective observational case-only study. There is no treatment allocation. Patients with severe or life-threatening PROS who have received alpelisib as part of a compassionate use program were invited to participate.