(2024) Patient-Centered Outcomes & the PN Patient Registry

Patient-Centered Outcomes & the PFIC Network Patient Registry

2024 PFIC Family & Scientific Conference Family Session

Speaker: Dr. Gitta Lubke. Click to read more about Dr. Lubke

Professor Emerita-University of Notre Dame

Dr. Lubke works with the PFIC Network as a Science Advisor to help develop a quality research program, write grant proposals, and maintain a patient registry. She is also Co-Lead of the ongoing Projet IMPACT. The goal of IMPACT is to prepare the PFIC community for patient-centered outcomes research by July 2025. IMPACT is funded through a Patient-Centered Outcomes Research Institute (PCORI) Eugene Washington Engagement Award (EASO-30455).

This presentation gives an update on the PFIC Network Patient Registry.
The registry started two years ago: The registry launched during the PFIC Family & Scientific Conference in 2022, and have about 120 participants!
The registry collects data every 6 months: Multiple waves permit to see how a patient fares over time. For instance, several participants had liver transplant (LT) after they enrolled in the registry, and we can see the effect of LT on itch, sleep, family quality of life, and general health.
The registry collects high quality data: We use validated survey questions and measures so that we can show for instance in which percentile of severity a patient falls with respect to itch or growth retardation.
The registry is patient-focused and patient-driven: When patient families indicated the importance to look at the financial burden associated with PFIC we added several questions to the registry to address this issue.
The registry is an open science project: Researchers can apply for access to de-identified data. All requests are evaluated by a committee of patient families and researchers/clinicians.

This presentation was recorded at the 2024 PFIC Family & Scientific Conference hosted in partnership with Cincinnati Childrens Hospital. Find out more about PFIC Network Conferences!