N/A
N=57
Retrospective Chart Review Study of Patients With PIK3CA-Related Overgrowth Spectrum Who Have Received Alpelisib
PIK3CA-Related Overgrowth Spectrum (PROS)
Bottom Line
View on ClinicalTrials.gov: NCT04285723 ↗Enrolled (actual)
57
Serious AEs
36.8%
Results posted
Feb 2023
Primary outcome: Primary: Percentage of Patients Responders and Non-responders at Week 24 — 30.4; 55.6; 69.6; 44.4 Percentage of participants
Study Design & Population
- Study type
- Observational
- Phase
- N/A
- Interventions
- alpelisib (Other)
- Age
- Pediatric, Adult, Older Adult · 2+ yrs
- Sex
- All
- Sponsor
- Novartis Pharmaceuticals
- Primary completion
- Apr 2021
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Percentage of Patients Responders and Non-responders at Week 24 |
30.4; 55.6; 69.6; 44.4 | — |
| SECONDARY Percent Change in the Sum of Measurable Target Lesion Volume |
-11.64; -11.78; -18.59; -19.77; -11.20; -19.69 | — |
| SECONDARY Percent Change in the Sum of All Measurable Lesion Volume |
-11.64; -11.78; -18.59; -19.77; -11.20; -19.69 | — |
| SECONDARY Percent Change in the Sum of All Measurable Non-target Lesion Volume |
— | — |
| SECONDARY Mean Duration of Response (DoR) |
— | — |
| SECONDARY Reasons for Discontinuation of Concomitant PROS-related Non-drug Treatments |
12; 2; 4; 7; 2; 1 | — |
| SECONDARY Participants With Concomitant PROS-related Medications Over Time |
34; 30; 25 | — |
| SECONDARY Number of PROS-related Completed Surgeries During the Study Period |
4; 2; 2; 1; 3; 0 | — |
| SECONDARY Number of Patients With Improvement in Most Frequent PROS-related Signs and Symptoms |
22; 10; 20; 10; 11; 5 | — |
| SECONDARY Change in Performance Status Score |
9; 1; 8; 6; 0; 0 | — |
| SECONDARY Functional Status - Mobility Assessment |
0; 1; 0; 1; 1; 2 | — |
| SECONDARY Change From Index Date in Functional Status - School Status During the Study Period |
1; 0; 28; 4; 0; 0 | — |
| SECONDARY Change From Index Date in Functional Status - Work Status |
0; 1; 6; 1; 5; 0 | — |
| SECONDARY Health Resource Utilization (HRU) - Number of Hospitalizations Per Patient |
1.8; 1.7; 1.1; 1.0 | — |
| SECONDARY Number of Patients With Grade 3/4 on Laboratory Assessments: Hematology |
0; 0; 0; 0; 2; 1 | — |
| SECONDARY Number of Patients With Grade 3/4 on Clinical Assessments - Clinical Chemistry |
0; 0; 0; 0; 0; 0 | — |
| SECONDARY Notable Vital Sign Values During the Study Period - Pediatric Patients |
17; 3; 12; 2; 15; 10 | — |
| SECONDARY Notable Vital Sign Values During the Study Period - Adult Patients |
17; 0; 3; 0; 12; 2 | — |
| SECONDARY Number of Participants With Notable ECG Values. |
1; 1; 1; 0; 0; 1 | — |
| SECONDARY Growth and Development in Pediatric Population |
-0.1; 0.1; 0.8; -0.2 | — |
| SECONDARY Overview of Number of Patients With Adverse Events (AEs) |
31; 16; 4; 9; 10; 11 | — |
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.
Eligibility 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)
Data sourced from ClinicalTrials.gov (NCT04285723). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.