Long-term outcome and quality of life in patients with biliary atresia

Abstract

Biliary atresia (BA) is a rare neonatal cholestatic disease of unknown etiology and is the commonest cause of neonatal obstructive jaundice with an incidence quoted to be 1 in 10,000 to 1 in 16,700 live births (C. Chardot, 2006; Karrer, Lilly, Stewart, & Hall, 1990a; Livesey et al., 2009; McKiernan, Baker, & Kelly, 2000). It leads to progressive obliterative cholangiopathy, resulting in biliary obstruction and jaundice (Sokol, Mack, Narkewicz, & Karrer, 2003). If left untreated, BA would rapidly progress to cirrhosis, liver failure and mortality within two years, with a median survival of eight months (Bates, Bucuvalas, Alonso, & Ryckman, 1998). Japanese surgeons Morio Kasai and Sozo Suzuki introduced the surgical procedure of hepatoportoenterostomy to achieve restoration of bile flow 50 years ago, nowadays commonly known as the Kasai operation (Kasai & Suzuki, 1959). However, this surgical treatment is far from perfect. A large proportion of post-operative patients will continue to have ongoing cholangitis and liver cirrhosis, leading to malnutrition, ascites, portal hypertension and coagulopathy. As a result, approximately 50% of the affected infants would require liver transplantation within the first two years of life (Nio, Ohi, Miyano, Saeki, Shiraki, Tanaka, et al., 2003; Shneider et al., 2006; Shneider & Mazariegos, 2007). The patients who are spared from transplantation may live for years with their native liver, despite the progression of cirrhosis and chronic liver disease. Many of them will be affected by long-term complications such as repeated cholangitis, portal hypertension and variceal bleeding, contributing to frequent hospital admissions (Bessho, 2015). The disease may also affect the nutritional status of the patient during childhood and adolescence, resulting in malnutrition and growth failure. These morbidities impose a huge impact on the quality of life (QOL) of the patients and their families. The QOL of BA patients surviving with their native liver has not been comprehensively explored in the past literature. In this study, I investigated the long term results of all our patients with biliary atresia in the past 35 years, focusing on the transplant-free survival and the QOL in patients who had Kasai operation for over 20 years.

Objectives: Review long-term transplant-free survival and quality of life of patients with biliary atresia.

Methods: A retrospective study reviewing all patients with Kasai operation between 1980 and 2015 was performed to evaluate the transplant-free survival. Subgroup analysis of patients over 20 years old was carried out to assess the quality of life using the Short Form-36 Health Survey and incidences of disease-related complications. Comparison between patients with native and transplanted liver was performed using two-tailed independent samples t-test (p-value<0.05=significant).

Results: The 20-year Kaplan-Meier transplant-free survival of the 141 patients in our study was 51%. In the subgroup analysis of long-term survivors, 33 patients living with their native liver were divided into three groups by serum bilirubin level and their incidences of long-term complications analyzed, which revealed a trend of increased prevalence of complications like esophageal varices, portal hypertension and recurrent admissions in the patient groups with raised serum bilirubin.

Thirty-one patients were successfully contacted for quality of life assessment, 26 (16 with native liver and 10 with transplanted liver) responded (76.5%). BA patients who were documented to have active complications have a significantly lower vitality score (50.7 versus 57.5, p=0.015). There was no statistically significant difference in the scores between the transplanted group and the normal control group. However, the native liver group achieved a lower score in both the general health section (42.9 versus 49.6, p=0.029) and the overall physical component (49.6 versus 54.4, p=0.037).

Conclusions: A significant proportion of our patients survive with their native liver for over 20 years. These long-term survivors may suffer from complications that impair their quality of life. They require continuous life-long care.