Goals, Mission & Vision

Overall Goals

The overarching goals of PFN-STRIDE are to incorporate long-term outcomes and patient and caregiver perspectives into management of rare invasive fungal diseases (IFDs) (Project 1), to better characterize the genetic and susceptibility phenotypes of pathogens causing IFD so that future therapeutic discovery and antifungal clinical trials can be more intentional (Project 2), and to more fully understand how the host immune system responds to IFD so that novel targeted diagnostic strategies can be developed (Project 3). This will allow us to directly guide new evidence-based clinical management approaches and inform design of future diagnostic and therapeutic trials.

Research Project 1 Goals

We will conduct a prospective multi-center, pediatric observational natural history study to capture clinical outcomes and patient and caregiver perspectives for children and adolescents with a rare IFD in the setting of an underlying immunocompromising condition. This project will establish the foundation of epidemiological evidence for one-year outcomes of IFD from four groups of rare fungal pathogens. The infrastructure of this natural history study will also provide the unique opportunity to centrally capture the experience of pediatric patients receiving several novel antifungal therapies. These are critically important data for practicing clinicians to understand experiences with these new agents, but currently are not systematically collected or analyzed to benefit the community. We will identify and longitudinally measure patient and caregiver perspectives on IFD outcomes and healthcare experiences. We expect that this aim will capture patient-centered outcomes and experiences that will inform future study design and interventions aimed at optimizing IFD outcomes and patient experiences.

Research Project 2 Goals

This project will establish a central pathogen biorepository to receive and process rare fungal pathogens obtained from immunocompromised pediatric patients with newly diagnosed IFD from Project 1, and to establish a comprehensive system that innovatively integrates genomic, phenotypic susceptibility, and clinical data. The pathogen biorepository will be supplemented by fungal pathogens previously collected into existing pathogen repositories at the Children’s Hospital of Philadelphia and the University of Texas at San Antonio. Our holistic approach will harness the power of genomic interrogation alongside clinically relevant fungal pathogen features such as comprehensive antifungal susceptibility testing of approved and newer antifungal agents. These genotypic and phenotypic data will be linked to clinical features and outcomes. We will gain important insights on antifungal resistance and how this resistance may impact clinical outcomes and what genomic features may be associated with emerging resistance patterns.

Research Project 3 Goals

Our first goal is to identify host RNA transcriptomic signatures that discriminate IFD from other infection etiologies (bacterial or viral) in immunocompromised children and adolescents. Secondly, we will employ AI modeling to identify IFD radiological patterns in a multi-center cohort. The significance of this project lies in the promise to develop non-invasive diagnostic approaches informed by host response to a broad range of IFD. These novel tools can supplement existing diagnostic tools to reduce time to diagnosis and complications from invasive procedures.

Pilot Research Program Goals

The first goal of the pilot research program is to solicit, review, and award pilot projects that address critical gaps in knowledge in pediatric IFD clinical trial readiness as identified by clinicians, researchers, patients and their families and caregivers. Secondly, the pilot research program will support pilot grant awardees to ensure ethical compliance, integration of data management with other PFN-STRIDE studies and maximize the development of key preliminary data necessary for pediatric IFD clinical trial design readiness. Ultimately, the pilot research program will develop new lines of investigation and investigators in rare pediatric IFD. Administration of this program in the context of the greater PFN-STRIDE RDCRC research priorities will ensure advancements in the science that directly addresses the needs of immunocompromised pediatric patients experiencing rare IFD.

Career Enhancement Program Goals

The first goal of the career enhancement program is to educate clinicians and investigators about pediatric IFD through innovative seminars and research conference presentations to increase awareness and stimulate research interest in these rare diseases. Secondly, we will establish a clinical investigator training pathway in the area of pediatric IFD to train and support the next generation of clinical researchers through purposeful mentorship and involvement in existing and future studies. The overall goal is to develop a robust pathway for trainees and junior faculty interested in pediatric IFD to develop into successful scientists.

Patient Advocacy Goals

The patient advocacy goals of PFN-STRIDE will capture perspectives of pediatric patients (and their caregivers) experiencing a rare IFD. We will accomplish this initially through qualitative interviews which will allow us to identify self-reported outcomes considered relevant to patients and their caregivers who have experienced an IFD. Secondly, we will determine the feasibility of longitudinally collecting relevant patient and caregiver reported outcomes in patients with newly diagnosed IFD at 3, 6, 9, and 12 months after IFD diagnosis. We expect to capture patient-centered outcomes and experiences that will inform future study design and interventions aimed at optimizing IFD outcomes and patient experiences.