Health-related Quality of Life, Symptom Severity, and Pain Among Patients With PIK3CA-related Overgrowth Spectrum: A Mixed-methods Observational Study

This study will collect qualitative data through interviews with patients and caregivers and qualitative data through a web-based longitudinal survey.

Both parts of the study are designed to collect information on HRQoL, symptom severity, and pain among patients with PROS who are receiving treatment with alpelisib in the US. In addition, patients who have not been treated with alpelisib will also participate in the quantitative part of the study by providing data on HRQoL, symptom severity, and pain, collected from a single administration of the web-based survey.

Fifteen participants treated with alpelisib will be recruited to take part in an hour-long, one-on-one qualitative interview scheduled by GP and conducted by a trained QM qualitative researcher. This subset of 15 participants will complete the first online assessment 1 week after the interview to prevent biasing of their survey responses due to the interview experience. Those that do not take part in the qualitative interview will be directed to complete the online survey upon enrollment (i.e., Day 1, Month 2, Month 4 in reference to time since enrollment in quantitative portion, which is independent of time since the start of treatment). All participants will be directed to complete the online survey which will be accessed through a web link.