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Surgical outcomes in Alagille syndrome and PFIC: A single institution's 20-year experience
Celia D Flores1, Yangyang R Yu1, Tamir A Miloh2
1Division of Pediatric Surgery, Department of Surgery, Texas Children's Hospital, Houston, TX, United States; Michael E. DeBakey Department of Surgery, Baylor College of Medicine, Houston, TX, United States.
Insights
Surgical management for pediatric Alagille Syndrome (AGS) and Progressive Familial Intrahepatic Cholestasis (PFIC) included liver transplantation and biliary diversion. Biliary diversion offered symptom control for some patients, potentially delaying or avoiding liver transplantation.
Area of Science:
- Pediatric Hepatology
- Surgical Gastroenterology
- Rare Pediatric Diseases
Background:
- Alagille Syndrome (AGS) and Progressive Familial Intrahepatic Cholestasis (PFIC) are rare pediatric cholestatic liver diseases.
- These conditions lead to progressive liver dysfunction and significant morbidity in children.
Purpose of the Study:
- To review the surgical management strategies for pediatric AGS and PFIC.
- To evaluate the outcomes of liver transplantation and biliary diversion in these rare conditions.
Main Methods:
- Retrospective review of pediatric patients diagnosed with AGS or PFIC between 1996 and 2016.
- Data analysis included demographics, surgical interventions (liver transplant, biliary diversion), and postoperative complications.
Main Results:
- 17 out of 37 patients underwent surgical intervention, with liver transplantation being the most common procedure (n=14).
- Biliary diversion (external or internal) was performed in 3 patients, resulting in symptom resolution and no need for transplantation in one AGS case and two PFIC cases.
- PFIC patients were older at transplantation than AGS patients (4.3 vs. 2.4 years).
Conclusions:
- Surgical intervention, including liver transplantation and biliary diversion, is crucial for managing pediatric AGS and PFIC.
- Biliary diversion can be an effective option for controlling pruritus and potentially avoiding or delaying liver transplantation in select cases.
- While liver transplantation is frequently required, biliary diversion offers a valuable alternative for symptom management in these rare pediatric liver diseases.
Background:
Alagille Syndrome (AGS) and Progressive Familial Intrahepatic Cholestasis (PFIC) are rare pediatric biliary disorders that lead to progressive liver disease. This study reviews our experience with the surgical management of these disorders over the last 20years.
Methods:
We retrospectively reviewed the records of children diagnosed with AGS or PFIC from January 1996 to December 2016. Data collected included demographics, surgical intervention (liver transplant or biliary diversion), and complications.
Results:
Of 37 patients identified with these disorders, 17 patients (8 AGS,9 PFIC) underwent surgical intervention. Mean postsurgical follow-up was 6.9±4.7years. Liver transplantation was the most common procedure (n=14). Two patients who were initially thought to have biliary atresia underwent hepatoportoenterostomy, but were subsequently shown to have Alagille syndrome. Biliary diversion procedures were performed in 3 patients (external n=1, internal n=2). PFIC patients tended to be older at the time of liver transplant compared to AGS (4.3±3.9years vs. 2.4±1.1years, p=0.25). The AGS patient with external diversion had resolution of symptoms and no complications (follow-up: 12.5years). Both PFIC patients with internal diversion (conduit between gallbladder and transverse colon) had resolution of pruritus and no progression of liver disease (follow-up: 3.8 and 4.5years).
Conclusions:
AGS and PFIC are rare biliary disorders in children which result in pruritus and progressive liver failure. Three patients in this series (8%) benefited from biliary diversion for control of pruritus and have not to date required transplantation for progressive liver disease. 38% underwent transplantation owing to pruritus and severe liver dysfunction.
Level Of Evidence:
2b.