Pediatric Fungal Network's (PFN) STudy of Rare Invasive Fungal DisEases in Immunocompromised Pediatric Patients (STRIDE) Project 1

Children'S Hosp Of Philadelphia, Philadelphia PA

Investigators

Abstract

PROJECT 1 SUMMARY/ABSTRACT The proposed project will be a natural history study intended to garner knowledge on the epidemiology of invasive fungal disease (IFD) in immunocompromised pediatric patients, including patient-centered outcome data. This study will fill gaps in the current literature on IFDs presenting in neonates, children, and adolescents. Existing data from descriptive epidemiology studies of pediatric IFD are limited by small numbers of participants, short observational periods, homogeneity in underlying immunocompromised conditions studied, and little to no patient-centered data. The first aim will be a natural history study to describe the epidemiology of IFD, including management approaches and outcomes one year after diagnosis, in pediatric patients, by pathogen type and immunocompromising condition. It will compare one-year morbidity and mortality between patients diagnosed with IFD and matched controls with similar immunocompromising conditions who do not have clinical concern for IFD. Aim 1 will also establish a central data source for outcomes of patients who receive novel antifungal agents for IFD under expanded-use protocols. This resource will be shared with the FDA to address a dearth in data on use of these agents in pediatric patients. Aim 2 will utilize qualitative interviews and self-report surveys to capture patient-centered outcome data from patients and caregivers who have and are experiencing IFD, guided by patient advisory group and advocacy partner engagement. Data gathered in this natural history study will inform the other proposed projects in this Pediatric Fungal Network STudy of Rare Invasive Fungal DisEases in Immunocompromised Pediatric Patients (PFN-STRIDE) Rare Diseases Clinical Research Consortium (RDCRC). These aims will also generate a breadth of data on IFD in pediatric patients that does not currently exist due to the rarity of these conditions, creating a path toward evidence-based practice and clinical trial readiness.