Complications Following Liver Transplantation for Progressive Familial Intrahepatic Cholestasis

Berumen, Jennifer; Feinberg, Elyssa; Todo, Tsuyoshi; Bonham, Clark A.; Concepcion, Waldo; Esquivel, Carlos O.

doi:10.1007/s10620-014-3220-5

Cited by 10 publications

(9 citation statements)

References 7 publications

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“…Extrahepatic manifestation post-LT as a potential complication, including pancreatic disease, is more frequently described after LT in PFIC1 [ 4 , 21 , 22 , 23 , 24 ]. Pancreatic disease may be associated with LT or be due to the progression of extrahepatic disease.…”

Section: Introductionmentioning

confidence: 99%

“…In addition, diarrhea is also frequently described, as PFIC1 is strongly expressed in the small and large intestine of healthy individuals. Gastrointestinal dysfunction and pancreatic injury may contribute to diarrhea-associated malnutrition, poor growth, prolonged delay of puberty and low albumin and hemoglobin levels after LT in PFIC1 [ 4 , 23 , 25 ].…”

Section: Introductionmentioning

confidence: 99%

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Long-Term Outcome after Liver Transplantation for Progressive Familial Intrahepatic Cholestasis

et al. 2021

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Background and Objectives: Progressive familial intrahepatic cholestasis (PFIC) is a rare autosomal recessive inherited disease divided into five types (PFIC 1-5). Characteristic of all types is early disease onset, which may result clinically in portal hypertension, fibrosis, cirrhosis, hepatocellular carcinoma (HCC), and extrahepatic manifestations. Liver transplantation (LT) is the only successful treatment approach. Our aim is to present the good long-term outcomes after liver transplantation for PFIC1, focusing on liver function as well as the occurrence of extrahepatic manifestation after liver transplantation. Materials and Methods: A total of seven pediatric patients with PFIC1 underwent liver transplantation between January 1999 and September 2019 at the Department of Surgery, Charité Campus Virchow Klinikum and Charité Campus Mitte of Charité-Universitätsmedizin Berlin. Long-term follow-up data were collected on all patients, specifically considering liver function and extrahepatic manifestations. Results: Seven (3.2%) recipients were found from a cohort of 219 pediatric patients. Two of the seven patients had multilocular HCC in cirrhosis. Disease recurrence or graft loss did not occur in any patient. Two patients (male, siblings) had persistently elevated liver parameters but showed excellent liver function. Patient and graft survival during long-term follow-up was 100%, and no severe extrahepatic manifestations requiring hospitalization or surgery occurred. We noted a low complication rate during long-term follow-up and excellent patient outcome. Conclusions: PFIC1 long-term follow-up after LT shows promising results for this rare disease. In particular, the clinical relevance of extrahepatic manifestations seems acceptable, and graft function seems to be barely affected. Further multicenter studies are needed to analyze the clinically inhomogeneous presentation and to better understand the courses after LT.

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Section: Introductionmentioning

confidence: 99%

Section: Introductionmentioning

confidence: 99%

Long-Term Outcome after Liver Transplantation for Progressive Familial Intrahepatic Cholestasis

et al. 2021

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“…There have been rare reports regarding the occurrence of HCC in patients with PFIC, which emphasizes on the risk of HCC in the patients with defects in bile export proteins, i.e. PFIC (9,10).…”

Section: Discussionmentioning

confidence: 99%

Pediatric Hepatocellular Carcinoma, a Single Center Study from the South of Iran: Case Series

et al. 2017

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Background: Hepatocellular carcinoma is a rare tumor in children, thus, there are not many studies regarding clinicopathologic findings of this tumor and no report regarding this tumor in this age group from Iran. Objectives: In this study, we tried to find out the clinicopathologic findings including outcome of hepatocellular carcinoma in the patients under the age of 18. Methods: During the last 10 years (2005 -2016), we collected all hepatocellular carcinoma cases in the affiliated hospitals of Shiraz University of Medical Sciences (as the largest referral center in the South of Iran and the only center of pediatric liver transplantation in the whole country) from the archives of Pathology department. Next all clinical data were subsequently extracted from the patients' hospital charts. Results: We collected 30 cases of hepatocellular carcinoma with the age range of 9 months to 18 years with no significant sex preference. All of the patients, except 3, showed abnormal levels of AFP. The most common underlying cause of liver tumorigenesis was type I tyrosinemia. Conclusions: Hepatocellular carcinoma in children is a rare tumor and is mostly secondary to an underlying liver disease, which is tyrosinemia in our center. Hepatitis B related hepatocellular carcinoma is rare in our country, which seems to be related to successful early neonatal vaccination against this viral disease. This tumor has a poor outcome in children and without liver transplantation, has a very short survival rate.

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“…High concentrations of bile salts in the colon may result in diarrhea which however can be diminished by treatment with bile adsorptive resin. Furthermore, significant hepatic steatosis often occurs after transplantation which can progress to cirrhosis and may require re-transplantation[ 37 , 38 ]. The pathogenesis of this steatosis is not totally clear, but it might also be secondary to the continued intestinal malfunction of ATP8B1 since it coincides with diarrhea in practically all patients[ 37 ].…”

Section: Current Therapeutic Regimenmentioning

confidence: 99%

Current and future therapies for inherited cholestatic liver diseases

Woerd¹,

Houwen²,

Graaf³

2017

WJG

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Familial intrahepatic cholestasis (FIC) comprises a group of rare cholestatic liver diseases associated with canalicular transport defects resulting predominantly from mutations in ATP8B1, ABCB11 and ABCB4. Phenotypes range from benign recurrent intrahepatic cholestasis (BRIC), associated with recurrent cholestatic attacks, to progressive FIC (PFIC). Patients often suffer from severe pruritus and eventually progressive cholestasis results in liver failure. Currently, first-line treatment includes ursodeoxycholic acid in patients with ABCB4 deficiency (PFIC3) and partial biliary diversion in patients with ATP8B1 or ABCB11 deficiency (PFIC1 and PFIC2). When treatment fails, liver transplantation is needed which is associated with complications like rejection, post-transplant hepatic steatosis and recurrence of disease. Therefore, the need for more and better therapies for this group of chronic diseases remains. Here, we discuss new symptomatic treatment options like total biliary diversion, pharmacological diversion of bile acids and hepatocyte transplantation. Furthermore, we focus on emerging mutation-targeted therapeutic strategies, providing an outlook for future personalized treatment for inherited cholestatic liver diseases.

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Complications Following Liver Transplantation for Progressive Familial Intrahepatic Cholestasis

Cited by 10 publications

References 7 publications

Long-Term Outcome after Liver Transplantation for Progressive Familial Intrahepatic Cholestasis

Long-Term Outcome after Liver Transplantation for Progressive Familial Intrahepatic Cholestasis

Pediatric Hepatocellular Carcinoma, a Single Center Study from the South of Iran: Case Series

Current and future therapies for inherited cholestatic liver diseases

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