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CME: Navigating the progressive familial intrahepatic cholestasis (PFIC) landscape: unmet needs and treatment options

Activity Description / Statement of Need:

In this online, self-learning activity:

Progressive familial intrahepatic cholestasis (PFIC) is comprised of a group of liver diseases notable for high serum bile salt concentrations and serum bilirubin levels in neonates. It is caused by autosomal recessive mutations in the genes that encode proteins expressed mainly in the bile acid membrane of the hepatocytes and alters hepatocytic bile secretion. PFIC accounts for approximately 9%−13% of cases of neonatal cholestasis liver failure or splenomegaly and 10%−15% of liver transplantation (LT) cases in childhood. Although its exact prevalence is unknown, it is estimated at 1–2 per 100,000 births.

Target Audience:

HCPs including: pediatricians and pediatric gastroenterologists and hepatologists; nurse practitioners, physician assistants, and pharmacists who practice in pediatrics; and those who otherwise commonly care for or clinically encounter patients with PFIC.

Commercial Support Disclosure: This program is supported by an educational grant from Ipsen. 

This activity is free of charge.

Release Date: July 12, 2024 -- Expiration Date: July 12, 2026

Faculty: William Balistreri, MD


Faculty introduction and disclosures

Introductory content: review of PFIC

· Definition and clinical features

· Subtypes 

· Genetics and pathophysiology 

· Epidemiology 

· Differential diagnosis, and diagnostic principles

Medical management for PFIC [Learning Objectives #2 & 3] 

· Treatment goals 

o Short-term

o Long-term

· Potential approaches; the roles of: 

o   UDCA 

o   Rifamycin 

o   Cholestyramine 

o   Hydroxyzine, diphenhydramine 

o   Naltrexone 

o   Sertraline 

o   IBAT inhibitors 

· Role of surgery and LT

· Future Options 

· Ongoing challenges 

Patient case(s)

Summary, conclusion, and best practice gap 

Learning Objectives

By the end of the session the participant will be able to:

  • Define Progressive Familial Intrahepatic Cholestasis (PFIC).
  • Summarize the clinical burden of patients with PFIC & challenges to optimal management.
  • Review potential therapies for PFIC and their roles in the treatment paradigm.
  • Develop a treatment plan for a patient with PFIC.


ACCME Activity #202797276

This activity has been planned and implemented in accordance with the accreditation requirements and policies of the Accreditation Council for Continuing Medical Education through ScientiaCME. ScientiaCME is accredited by the ACCME to provide continuing medical education for physicians.

Credit Designation: ScientiaCME designates this educational activity for a maximum of 1.0 AMA PRA Category 1 Credit(s)™ toward the AMA Physician's Recognition Award. Physicians should only claim credit commensurate with the extent of their participation in the activity.

ABP MOC Recognition Statement: Successful completion of this CME activity, which includes participation in the evaluation component, enables the participant to earn up to 1.0 MOC points in the American Board of Pediatrics' (ABP) Maintenance of Certification (MOC) program. It is the CME activity provider's responsibility to submit participant completion information to ACCME for the purpose of granting ABP MOC credit.

ABP MOC Credit Type: Lifelong Learning and Self-Assessment

Physicians: For maintenance of certification (MOC) points, you must enter your board certification ID # and birth date correctly.  It is the learner's responsibility to provide this information completely and accurately at the completion of the activity. Without providing it, the learner will NOT receive MOC points for this activity. By providing this data, you acknowledge that it will be shared with ACCME and the applicable certifying board. Please note: Not all activities on this site provide MOC points. If this activity does not specify that it provides MOC points in this section, then it does NOT provide MOC points. This activity provides MOC points only for ABP.


ScientiaCME is accredited by the Accreditation Council for Pharmacy Education (ACPE) as a provider of continuing pharmacy education. This activity is approved for 1.0 contact hours  of continuing pharmacy education credit. Proof of participation will be posted to your NABP CPE profile within 4 to 6 weeks to participants who have successfully completed the post-test. Participants must participate in the entire presentation and complete the course evaluation to receive continuing pharmacy education credit. ACPE #0574-0000-24-019-H01-P. This is an Application (A)-type activity. 

PharmacistsYou must enter your NABP # and birth date correctly so that proof of participation can be posted to your NABP CPE profile. It is the learner's responsibility to provide this information completely and accurately at the completion of the activity. Without providing it, the learner will NOT receive CPE credit for this activity.

Nurses: The American Nurses Credentialing Center (ANCC) accepts AMA PRA Category 1 Credit(s)™ from organizations accredited by the ACCME. This activity is designated for up to 1.0 AMA PRA Category 1 Credit(s)™.

Physician Assistants: The American Academy of Physician Assistants (AAPA) accepts AMA PRA Category 1 Credit™ assigned by organizations accredited by the ACCME as satisfying Category 1 CME for National Commission on Certification of Physician Assistants (NCCPA) national certification maintenance. This activity is designated for up to 1.0 AMA PRA Category 1 Credit(s)™.

Nurse Practitioners: The American Academy of Nurse Practitioners Certification Board (AANPCB) states that continuing education providers accredited by the ACCME may provide acceptable, accredited Advanced Practice Provider content. This activity is designated for up to 1.0 AMA PRA Category 1 Credit(s)™.

Faculty Disclosure and Resolution of COI

As a provider of continuing medical education, it is the policy of ScientiaCME to ensure balance, independence, objectivity, and scientific rigor in all of its educational activities. In accordance with this policy, faculty and educational planners must disclose any significant relationships with commercial interests whose products or devices may be mentioned in faculty presentations, and any relationships with the commercial supporter of the activity. The intent of this disclosure is to provide the intended audience with information on which they can make their own judgments. Additionally, in the event a conflict of interest (COI) does exist, it is the policy of ScientiaCME to ensure that the COI is resolved in order to ensure the integrity of the CME activity. For this CME activity, any COI has been resolved thru content review by ScientiaCME.

Disclosures of Faculty: William F. Balistreri, MD, Dorothy M. M. Kersten, Professor of Pediatrics, Cincinnati Children's Hospital Medical Center, has received financial compensation from Mirum for advisory board work.

Disclosures of Educational Planners: Charles Turck, PharmD, BCPS, BCCCP, President of ScientiaCME, has no relevant financial disclosures.  

Faculty WILL NOT discuss off-label uses of a commercial product. 

All relevant financial relationships have been mitigated. 

ScientiaCME adheres to the ACCME’s Standards for Integrity and Independence in Accredited Continuing Education. Any individuals in a position to control the content of a CE activity, including faculty, planners, reviewers or others are required to disclose all relevant financial relationships with ineligible entities (commercial interests). All relevant conflicts of interest have been mitigated prior to the commencement of the activity.

Commercial Support Disclosure: This program is supported by an educational grant from Ipsen. 


  • Read the learning objectives above
  • Take the Pre-Test (optional). Completion of the pre-test will help us evaluate the knowledge gained by participating in this CME activity.
  • View the online activity. You may view this is in more than one session, and may pause or repeat any portion of the presentation if you need to.
  • Minimum participation threshold: Take the post-test. A score of 70% or higher is required to pass and proceed to the activity evaluation.
  • Complete the activity evaluation and CME registration. A CE certificate will be emailed to you immediately.

Cultural/Linguistic Competence & Health Disparities

System Requirements

Windows 7 or above
Internet Explorer 8
*Adobe Acrobat Reader
Mac OS 10.2.8
Safari or Chrome or Firefox
*Adobe Acrobat Reader
Internet Explorer is not supported on the Macintosh

*Required to view Printable PDF Version

Perform Pre-Test (optional)

Please take a few minutes to participate in the optional pre-test. It will help us measure the knowledge gained by participating in this activity.

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Acute hepatic porphyria: optimizing pharmacotherapeutic management strategies