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Alloveda Liver Update: Neonatal Cholestasis is a Poor Predictor of Portal Hypertension in young patients with Alpha-1 antitrypsin deficiency

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eMediNexus    11 September 2020

Alpha-1 antitrypsin deficiency (AAT deficiency) is an inherited condition which is associated with liver diseases. The current study, The Childhood Liver Disease Research Network (ChiLDReN) Longitudinal Observational Study of Genetic Causes of Intrahepatic Cholestasis (LOGIC), was conducted to assess the predictors of portal hypertension, liver transplantation, and death in young population with AAT deficiency.

This study enrolled PIZZ and PISZ subjects with pediatric cholestatic liver diseases having AAT deficiency, 0-25 years of age reported since November 2007 at 17 tertiary care centers. Medical records, history, physical examination, and laboratory studies were utilized for obtaining data from standard-of-care baseline and annual follow-up visits. This data was also useful to recognize patients with portal hypertension.

The outcome suggested that among 350 participants with native liver enrolled, 79% had no portal hypertension in the beginning of the study. However, 18 developed portal hypertension during follow-up. A total of 30 candidates needed liver transplantation while two died during 1077 person-years of follow-up. Moreover, similar data was gathered from participants with or without preceding neonatal cholestasis progressing to transplantation or death during the study (12% versus 7%), or in experiencing portal hypertension (28% versus 21%). Additionally, development of portal hypertension was related to decreased height Z-score.

The results show that portal hypertension with AAT deficiency in young population influences growth measures. Although rate of patients requiring liver transplantation is slow with rare mortality, risk of adverse effects and advancement to severe liver disease is a concern throughout childhood. Furthermore, a history of neonatal cholestasis is a weak predictor of severe liver disease.

Source: Teckman J, Rosenthal P, Hawthorne K, et al. Longitudinal Outcomes in Young Patients with Alpha-1-Antitrypsin Deficiency with Native Liver Reveal that Neonatal Cholestasis is a Poor Predictor of Future Portal Hypertension [published online ahead of print, 2020 Jul 11]. J Pediatr. 2020;S0022-3476(20)30874-X. 

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