Abstract: TH-PO472
Qualitative Analysis and Comparison of Externally Led, Patient-Focused Drug Development (EL-PFDD) Concepts for Autosomal Recessive Polycystic Kidney Disease (ARPKD) against Standardized Outcomes in Nephrology (SONG) Initiatives
Session Information
- Cystic Kidney Diseases: Clinical Assessment and Therapeutic Directions
October 24, 2024 | Location: Exhibit Hall, Convention Center
Abstract Time: 10:00 AM - 12:00 PM
Category: Genetic Diseases of the Kidneys
- 1201 Genetic Diseases of the Kidneys: Cystic
Authors
- Soyfer, Belle Alice, Critical Path Institute, Tucson, Arizona, United States
- Fedeles, Sorin V., Critical Path Institute, Tucson, Arizona, United States
- Vanasco, Wendy, Critical Path Institute, Tucson, Arizona, United States
- Perrone, Ronald D., Tufts Medical Center, Boston, Massachusetts, United States
- Liebau, Max Christoph, Universitatsklinikum Koln Klinische Infektiologie, Koln, Nordrhein-Westfalen, Germany
- Hartung, Erum Aftab, The Children's Hospital of Philadelphia, Philadelphia, Pennsylvania, United States
- Dell, Katherine MacRae, Cleveland Clinic Children's Hospital, Cleveland, Ohio, United States
- Guay-Woodford, Lisa M., The Children's Hospital of Philadelphia, Philadelphia, Pennsylvania, United States
- Hoover, Elise, PKD Foundation, Kansas City, Missouri, United States
- Oberdhan, Dorothee, Otsuka Seiyaku Kabushiki Kaisha, Chiyoda-ku, Tokyo, Japan
Background
ARPKD is an area of huge unmet need with no approved treatments. An EL-PFDD meeting in 2023 collected patient and caregiver concepts regarding burden of disease and treatment preferences. The Standardized Outcomes in Nephrology (SONG) initiatives previously captured qualitative data from large international cohorts including pediatric CKD, but the EL-PFDD focused specifically on individuals and families with ARPKD.
Methods
A thematic synthesis and comparison among SONG-Transplant (Tx), SONG-Children and Adolescents (SONG-Kids), SONG-Polycystic Kidney Disease (PKD, focused on autosomal dominant PKD), and the ARPKD EL-PFDD was conducted.
Results
All participants from the EL-PFDD and SONG-PKD had a diagnosis of ADPKD, compared to 6% in SONG-Kids and 27% in SONG-Tx. The EL-PFDD represented patients and caregivers while the SONG had additional concepts from healthcare professionals, as well. Common themes included disease inevitability, multi-organ involvement, treatment complications, blood pressure, kidney function, graft function and loss, self-esteem, desire for normality, mental health, and life participation. Exploratory findings between the EL-PFDD and SONG-Kids found disease inevitability and uncertainty, treatment complications and risks, blood pressure, kidney function, life participation, treatment, and failure to thrive. Concepts unique to the EL-PFDD included heterogeneous disease, body image related to extended abdomen and height, deficiencies of currently available treatment options, and goals for future research. Additionally, the EL-PFDD discussed supportive ARPKD therapies, unmet needs of currently available treatment options, and goals for future research.
Conclusion
The EL-PFDD meeting captured patient and caregiver insights that will impact the advancement of biomarkers and drugs whose benefits are aligned with the patient experience. The EL-PFDD highlighted the need for ARPKD-specific standardized outcomes due to its unique pathophysiology distinct from pediatric CKD etiologies. The analysis of combined elements from the SONG initiatives and EL-PFDD will facilitate future development of outcome tools and treatment plan designs.
Funding
- Other U.S. Government Support – Otsuka Pharmaceuticals