Medical Status of 219 Children with Biliary Atresia Surviving Long-Term with Their Native Livers: Results from a North American Multicenter Consortium

• Published in: The Journal of pediatrics Vol. 165; no. 3; pp. 539 - 546.e2
• Main Authors: Ng, Vicky Lee, MD, Haber, Barbara H., MD, Magee, John C., MD, Miethke, Alexander, MD, Murray, Karen F., MD, Michail, Sonia, MD, Karpen, Saul J., MD, Kerkar, Nanda, MD, Molleston, Jean P., MD, Romero, Rene, MD, Rosenthal, Philip, MD, Schwarz, Kathleen B., MD, Shneider, Benjamin L., MD, Turmelle, Yumirle P., MD, Alonso, Estella M., MD, Sherker, Averell H., MD, Sokol, Ronald J., MD
• Format: Journal Article
• Language: English
• Published: United States Mosby, Inc 01.09.2014
• Subjects: Biliary Atresia - surgery; Canada; Child; Enterostomy; Female; Health Status; Humans; Liver - surgery; Male; Pediatrics; Quality of Life; Survivors; Time Factors; United States; Canada; United States; Pediatric Quality of Life Inventory; Childhood Liver Disease Research and Education Network; LT; ALT; γ-glutamyl transpeptidase; INR; ChiLDREN; BASIC; Hepatoportoenterostomy; Portal hypertension; Chronic liver disease; PHT; Health-related quality of life; AST; Biliary atresia; Albumin; TB; GGT; Alb; International normalized ratio; HRQOL; Alanine aminotransferase; Aspartate aminotransferase; Biliary Atresia Study of Infants and Children; CLD; HPE; PedsQL; Total bilirubin; BA; Liver transplantation
• ISSN: 0022-3476; 1097-6833
• DOI: 10.1016/j.jpeds.2014.05.038

Objectives To examine the medical status of children with biliary atresia (BA) with their native livers after hepato- portoenterostomy (HPE) surgery. Study design The Childhood Liver Disease Research and Education Network database was utilized to examine subjects with BA living with their native livers 5 or more years after HPE and to describe the prevalence of subjects with BA with an “ideal” outcome, defined as no clinical evidence of chronic liver disease, normal liver biochemical indices (aspartate aminotransferase, alanine aminotransferase, γ-glutamyl transpeptidase, platelet count, total bilirubin, international normalized ratio, and albumin), and normal health-related quality of life 5 or more years after HPE. Results Children with BA (n = 219; 43% male) with median age 9.7 years were studied. Median age at HPE was 56 (range 7-125) days. Median age- and sex-adjusted height and weight z-scores at 5-year follow-up were 0.487 (IQR −0.27 to 1.02) and 0.00 (IQR −0.74 to 0.70), respectively. During the 12 preceding months, cholangitis and bone fractures occurred in 17% and 5.5%, respectively. Health-related quality of life was reported normal by 53% of patients. However, only 1.8% met the study definition of “ideal” outcome. Individual tests of liver synthetic function (total bilirubin, albumin, and international normalized ratio) were normal in 75%, 85%, and 73% of the study cohort. Conclusion Cholangitis and fractures in long-term survivors underscore the importance of ongoing medical surveillance. Over 98% of this North American cohort of subjects with BA living with native livers 5 or more years after HPE have clinical or biochemical evidence of chronic liver disease.