Retrospective Chart Review Study of Patients With PIK3CA-Related Overgrowth Spectrum Who Have Received Alpelisib (EPIK-P1)

Novartis

Status

Completed

Conditions

PIK3CA-Related Overgrowth Spectrum (PROS)

Treatments

Other: alpelisib

Study type

Observational

Funder types

Industry

Identifiers

NCT04285723

CBYL719F12002

Details and patient eligibility

About

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.

Full description

The index date (baseline) is defined as the date of alpelisib initiation. The study period is the period from the index date up to the most recent data available at the time of the cut-off date.

The maximum follow-up was 187 weeks.

Enrollment

57 patients

Sex

All

Ages

2+ years old

Volunteers

No Healthy Volunteers

Inclusion and exclusion criteria

Inclusion Criteria:

Patient (adult or pediatric) is ≥ 2 years of age
Patient has a physician confirmed/documented diagnosis of PROS
Patient has a documented evidence of a mutation in the PIK3CA gene
Patient's condition was assessed by the treating physician as severe or life threatening and treatment was deemed necessary
Patient has been treated with at least one dose of alpelisib, initiated on or before 23-Sep-2019 (i.e. at least 24 weeks before the cut-off date of the 09-Mar-2020)
Patient has medical chart history available during enrollment in the Novartis MAP
Patient (or parent/guardian in case of pediatric patient) consented to participate in the study (as required by local ethics regulations) Inclusion criteria for MAP enrollment (assessed at the time of alpelisib initiation)