Pediatric fungal network study of rare invasive fungal diseases in immunocompromised pediatric patients (PFN-stride) - pilot/feasibility governance core abstract the pediatric fungal network study…

The National Institute of Child Health and Human Development awarded a $1.727 million cooperative agreement to The Children's Hospital of Philadelphia on September 24, 2025, to support the Pediatric Fungal Network Study of Rare Invasive Fungal Diseases in Immunocompromised Pediatric Patients (PFN-STRIDE) Rare Diseases Clinical Research Consortium, a multi-institutional effort funded under CFDA 93.865 (Child Health and Human Development Extramural Research).

PFN-STRIDE advances diagnostic and management approaches for rare invasive fungal diseases (IFD) in immunocompromised pediatric patients through three linked clinical and translational research projects. The work addresses a critical gap: invasive fungal diseases carry case fatality rates exceeding 30% in pediatric immunocompromised populations, yet diagnostic approaches are severely limited and management protocols lack evidence. The award funds a Pilot/Feasibility Governance Core that will solicit and fund pilot projects from investigators across clinical and translational disciplines, build consensus on research topics through input from clinicians, researchers, patients, and caregivers, and support new investigators entering pediatric IFD research to advance clinical trial readiness.

Performance occurs in Pennsylvania with ultimate completion on July 31, 2030. The cooperative agreement carried an obligated amount of $1.727 million as of the award date.

Five sub-recipients deliver specialized research components. Duke University co-leads aims 2 and 3 of Project 3, developing host response signatures using bulk RNA sequencing and establishing a biomarker repository for invasive fungal disease. The University of California, San Francisco leads patient-centered outcome research through qualitative interviews and longitudinal capture of patient and caregiver-reported experiences and outcomes in newly diagnosed IFD. The University of Texas Health Science Center at San Antonio performs antifungal susceptibility testing on up to 150 clinical pathogen isolates, generating minimum inhibitory concentrations for up to seven antifungal agents per species and analyzing relationships between pathogen susceptibility and clinical outcomes. Emory University leads Project 3 overall, developing host response signatures via bulk RNA sequencing, exploring radiomics and artificial intelligence for pediatric IFD radiographic decision support, and forming a biomarker and imaging repository. Arkansas Children's Research Institute participates in Project 1 as a clinical research site, oversees patient enrollment and specimen collection, and leads the Career Enhancement Core supporting junior investigators and fellows in pediatric fungal disease research.

Pediatric fungal network study of rare invasive fungal diseases in immunocompromised pediatric patients (PFN-stride) - pilot/feasibility governance core abstract the pediatric fungal network study of rare invasive fungal diseases in immunocompromised pediatric patients (PFN-stride) rare diseases clinical research consortium (rdcrc) pilot/feasibility governance core will aim to increase the generation of high-quality preliminary data on rare pediatric invasive fungal diseases (IFD) by investigators with a broad range of expertise to advance the overarching goals of the PFN-stride. The pilot/feasibility core will accomplish its aims by soliciting new projects and supporting new investigators. DR. Anna huppler will oversee these efforts through leadership of the pilot program committee, which will interface directly with the PFN-stride executive committee and the PFN-stride multi-PIS. Members of the patient advocacy group, representatives from the other cores, and site PIS will provide input for the selection of pilot topics and awardees. The overall organization and administration of the pilot/feasibility core will ensure integration with all projects and other cores for the PFN-stride. The activities of the pilot/feasibility core will occur in several stages. The first three stages comprise the activities of aim 1: to solicit, review, and award pilot/feasibility projects that address critical gaps in knowledge in pediatric IFD clinical trial readiness. The first stage is consensus building for pilot topics including input from clinicians, researchers, patients and their families and caregivers. The next stage is solicitation and outreach for applications and investigators occurring in parallel with review committee formation with inclusion of junior investigators. A goal of this stage will be the recruitment of a broad range of investigators to support entry into the investigation of rare pediatric IFD. The third stage will be application review and award selection. Aim 2 of the pilot/feasibility core will implement two stages to establish infrastructure to support pilot/feasibility award recipients before and during their project period. Aim 2's first stage will ensure awardees have support for regulatory compliance, facilitate awardee connection to PFN-stride resources, and integrate pilot/feasibility data with PFN-stride data. In the second stage of aim 2, milestone-based progress reports will be reviewed to measure output of key preliminary data in pediatric IFD clinical trial readiness. The expected outcome of the pilot/feasibility core is the development of new lines of investigation and investigators to address the needs of immunocompromised pediatric patients experiencing rare IFD.