Baseline analysis of patient reported outcomes in the progressive familial intrahepatic cholestasis patient registry
The PNPR fills a previously identified gap in PFIC research-the lack of PROs-and reveals the negative impact of disease and pruritus on patient and family function, QoL indicators, finances, and measures of general health.
Authors
- James E. Squires (ORCID: https://orcid.org/0000-0001-6979-8987)
- Akihiro Asai (ORCID: https://orcid.org/0000-0003-3029-5754)
- Wálter Pérez (ORCID: https://orcid.org/0009-0003-3637-8793)
- R. Daryl Fischer
- Melissa Kochanowsky
- Benjamin L. Shneider
- Emily Ventura
- Gitta H. Lubke
Institutions
- University of Notre Dame (US)
- Cincinnati Children's Hospital Medical Center (US)
- Children's Mercy Hospital (US)
- Baylor College of Medicine (US)
- Kentucky Educational Television (US)
- Children's Hospital of Pittsburgh (US)
- Texas Children's Hospital (US)
Publication Details
- Journal
- Journal of Pediatric Gastroenterology and Nutrition
- Published
- 2026-01-29
- DOI
- https://doi.org/10.1002/jpn3.70366
- Primary Topic
- Drug Transport and Resistance Mechanisms
- Type
- article
- Field-Weighted Citation Impact
- 0.00