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Nontransplant surgical interventions in progressive familial intrahepatic cholestasis
Adam Rahn Davis1, Philip Rosenthal, Thomas B Newman
1Division of Pediatric Gastroenterology, Hepatology and Nutrition, Department of Pediatrics, University of California, San Francisco, CA 94143, USA. davisa@peds.ucsf.edu
Insights
Nontransplant surgery offers successful outcomes for most Progressive Familial Intrahepatic Cholestasis (PFIC) patients, halting disease progression. Further research and standardized outcome measurements are needed to compare surgical techniques for this rare childhood liver disease.
Area of Science:
- Hepatology
- Pediatric Surgery
- Medical Genetics
Background:
- Progressive Familial Intrahepatic Cholestasis (PFIC) comprises rare, fatal childhood liver diseases.
- Liver transplantation was the only cure until nontransplant surgical interventions became standard care.
- Various surgical techniques exist, but outcomes have not been systematically reviewed.
Purpose of the Study:
- To review the outcomes of nontransplant surgical interventions in patients with PFIC.
- To identify trends and success rates of current surgical approaches for PFIC.
Main Methods:
- Systematic literature search for studies on nontransplant surgical interventions in PFIC.
- Abstraction and summarization of data from case series and case reports.
Main Results:
- No clinical trials were identified; analysis included 11 case series/reports.
- Nontransplant surgery demonstrated an 81% success rate, halting disease progression and resolving symptoms.
- Treatment failures were linked to advanced stages of the disease.
Conclusions:
- No single nontransplant surgical intervention is proven superior for PFIC.
- A patient registry and standardized outcome metrics are proposed for better comparative analysis.
Background:
Progressive familial intrahepatic cholestasis (PFIC) is a family of rare childhood diseases that was universally fatal until the development of liver transplant. In the last 20 years, the use of nontransplant surgery to treat PFIC has become the standard of care. There are various surgical techniques that have been performed. There are no reviews evaluating the outcome of these operations.
Methods:
A systematic search of the literature for articles evaluating the outcome of nontransplant surgical interventions in PFIC patients was performed. Data from these studies was abstracted and summarized.
Results:
No trials have been performed addressing nontransplant surgical interventions in PFIC patients. We analyzed 11 case series and case reports. Generally, patients had successful outcomes (81%) with cessation of progression of disease and resolution of symptoms. Treatment failures were often associated with more advanced disease.
Discussion:
There is no evidence to demonstrate a superiority of one type of nontransplant surgical intervention in PFIC patients. We propose the development of a registry and standardization of outcomes measurements to allow improved comparison of results.
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