Indication for a Pneumocystis Prophylaxis Therapy in Patients with Vascular Anomalies Treated with PIK3/AKT/mTOR Pathway Inhibitors: Experts' Opinion and Systematic Review from the Literature.

Background: Vascular anomalies (VAs) are increasingly being treated with PI3K/AKT/mTOR pathway inhibitors. These drugs have immunosuppressive properties and thus theoretically overexpose patients to opportunistic infections, especially Pneumocystis jirovecii pneumonia (PJP). PJP prophylaxis use lacks consensus.

Patient and public involvement is suboptimal in randomized controlled trials addressing a chronic condition.

Objectives: To describe patient and public involvement (PPI) in randomized controlled trials (RCTs) addressing a chronic condition and to analyze whether PPI is associated with trial characteristics.

Study Design And Setting: We used PubMed search to identify RCTs addressing a chronic condition and published in journals with a mandatory PPI statement.

Results: Across 101 RCTs; 40 (40%) reported PPI at any stage of the research process.