Thank you for joining us!
April 25-27, 2024
The 2024 PFIC Family & Scientific Conference, held in partnership with Cincinnati Children’s Hospital and Medical Center, was our biggest conference yet, with 138 in-person and 76 virtual participants. We appreciate all attendees making time in their busy lives to engage in important conversations about PFIC research, treatments and the patient experience.
This conference is supported by our generous sponsors:
Presenting Sponsors
Gold Sponsor
Virtual Platform- Conference Session Recordings
Recordings of the Family Sessions will be available to all attendees for 30 days post-conference in the virtual conference platform. Use your conference registration email to log in.
Conference Agenda
The conference format includes family-oriented sessions, research workshops, and combined roundtable discussions. Family-oriented sessions will include informative talks by field experts with ample time for audience Q&A. These talks will be complemented by immersive roundtable discussions that foster collaborative dialogue between patients, families, clinicians, and researchers. The research workshop sessions are designed for researchers, physicians and industry to share and learn about new disease models, connect with each other to build next projects and to highlight problems important to patients.
Session Descriptions
Check out a detailed description of each presentation and read poster abstracts:
Conference Speakers
We’ve got an incredible roster of PFIC experts lined up for the conference!
Topics
Family Session Topics
- Current & Emerging Treatments for PFIC
- Current Prognosis and Prospects of Liver Transplant
- Pre-Transplant Diet & Nutrition
- Patient Centered Outcomes & the PFIC Network Patient Registry
- PFIC in Adults
- Future Treatments for PFIC
Roundtable Discussion Topics
- Project IMPACT and Patient Centered Outcomes Research
- Understanding Risk/Benefit of PFIC Treatments, Present and Future
Research Workshop Topics
- Understanding and mitigating hepatic bile acid accretion in pediatric cholestatic diseases
- A rapid mouse model system for PFIC by CRISPR-based multigenic knockout
- Novel mouse models for PFIC
- IPSC Model-New Type of PFIC
- Using complementary experimental models to study new PFIC genes and variants
- FIC1-ko iPSC model
- Alagille model and development of new RNAi therapy
- Gene therapy targeting cholangiocytes
- In vivo selection for cell and gene therapy in the liver
Schedule
Day One (Friday April 26, 2024)
Cincinnati Children’s Hospital – Sabin Auditorium – 3333 Burnet Ave. Cincinnati, OH 45229
| 7:00- 8:00 am | Breakfast and Registration |
| 8:00 – 8:20am | Plenary & Family Oriented Sessions: Welcome Address E. Ventura, A. Asai, C. Ventura |
| 8:20-8:40am | Patient/Family Presentation Opener S. Munn, L.Susoi, A. Perez, E. Ventura |
| 8:40-9:20am | Current & Emerging Treatment Landscape (P. Hertel) |
| 9:20-10:00am | PFIC in Adults: Diagnosis & Onset (S. Vilarinho) |
| 10:00-10:20am | Break |
| 10:20-11:00am | Pre-Transplant Diet & Nutrition Panel Presentation & Discussion (R. Sokol, E. Tamayo, Patient & Parent Representatives, Moderator: L. Bull) |
| 11:00-11:30am | Patient-Centered Outcomes & the PFIC Network Patient Registry (G. Lubke) |
| 11:30-12:15pm | Lunch |
| 12:15-2:15pm | Family-Researcher Roundtable #1 “Patient-Centered Outcomes Research Priorities in PFIC” -Facilitators: J. Squires, A. Perez (PFIC parent) -IMPACT Project Team Members: G. Lubke, M. Kochanowsky, P. Catalla |
| 2:15-2:30pm | Break |
| Optional Family Activity: free Cincinnati Zoo visit @ 3pm Research Workshop: | |
| 2:30 – 3:10 pm | Keynote Address: Understanding and mitigating hepatic bile acid accretion in pediatric cholestatic diseases (S.Karpen) |
| 3:10 – 3:30 pm | A rapid mouse model system for PFIC by CRISPR-based multigenic knockout (H.Hayashi) |
| 3:30 – 3:50 pm | Novel mouse models for PFIC (H. Verkade) |
| 3:50-4:10pm | Discussion |
| 4:10- 4:20 pm | Break |
| 4:20- 5:15 pm | Research Workshop: –IPSC Model-New Type of PFIC (S. Vilarinho) |
| 4:20-4:40pm | Using complementary experimental models to study new PFIC genes and variants (C.Yin) |
| 4:40-5:00pm | Discussion |
| 6:30- 8 pm | Dinner @ Cincinnati Zoo *Must arrive by 7pm. Zoo gates will be closed after |
Day Two (Saturday April 27th, 2024)
Cincinnati Children’s Hospital – Sabin Auditorium – 3333 Burnet Ave. Cincinnati, OH 45229
| 7:00 – 8:00 am | Breakfast and Registration |
| 8:00 – 8:20am | Research Workshop: FIC1-ko iPSC model (R. Florentino) |
| 8:20-8:40am | Alagille model and development of new RNAi therapy (S. Huppert) |
| 8:40-9:00am | Discussion |
| 9:00-9:10am | Break |
| 9:10-9:30am | In vivo selection for cell and gene therapy in the liver (M. Grompe) |
| 9:30-9:50am | Gene therapy targeting cholangiocytes (H. Willenbring) |
| 9:50-10:10am | Discussion |
| 10:10-10:30 am | Break |
| 10:30-11:10am | Plenary & Family Oriented Sessions: State of the Art: Current Prognosis and Prospects of Liver Transplant ( H. Verkade) |
| 11:10-11:30pm | Group Picture |
| 11:30 – 12:30pm | Lunch |
| 12:30-1:10pm | Future Treatments of PFIC (A.Asai) |
| 1:10-1:20pm | Break |
| 1:20-3:20pm | Family-Researcher Roundtable #2 “The Benefits and Risks of PFIC Clinical Research” -Facilitators: R. Thompson, L. Susoi (PFIC parent) |
| 3:20-3:35pm | Break |
| 3:35-4:05pm | Plenary & Family Oriented Sessions: Summary Report of Research Workshop (C. Yin, W. Balistreri) |
| 4:05-4:20pm | Closing Remarks + Conference End (E. Ventura) |
*Times and running order subject to change
Continuing Education
In support of improving patient care, this activity has been planned and implemented by Cincinnati Children’s and PFIC Network. Cincinnati Children’s is jointly accredited by the Accreditation Council for Continuing Medical Education (ACCME), the Accreditation Council for Pharmacy Education (ACPE), and the American Nurses Credentialing Center (ANCC), to provide continuing education for the healthcare team.
- CME: This activity has been approved for AMA PRA Category 1 Credit™.
- Nursing: This activity is approved for continuing nursing education (CNE) contact hours.
- IPCE: This activity was planned by and for the healthcare team, and learners will receive Interprofessional Continuing Education (IPCE) credits for learning and change.
If you need proof of attendance or credit, please visit the CCHMC CME portal to make your profile to receive credit.
All attendees who would like credit will be required to text attendance for this course. You must have your cell phone number listed in your profile in order for it to record your attendance. Please follow the directions below to update your profile with your most current information and phone number. Plan ahead and save 513-409-9506 under the contact name: CCHMC CME Credit when it’s time to text your attendance.
Note: If you do not have a U.S. cell phone number, you will be instructed to complete a different online form to receive credit.
Update Your Account – CCHMC employees
1. Click here: https://cchmc.cloud-cme.com
2. Click“Sign In” on the top-left side of page
3. Click “Sign in/Create an Account with Cincinnati Children’s email” – left side
4. Login using your CCHMC credentials
5. Click on MY Account – Profile
Update Your Account – External
1. Click here: https://cchmc.cloud-cme.com
2. Click “Sign In” on the top-left side of page
3. Click “Sign In/Create an account with my email and password” – right side
4. Click on “MY Account” – “Profile”