Systematic review: the natural history of alpha-1 antitrypsin deficiency, and associated liver disease

Autor:	Paul R. Ellis, Philip N. Newsome, Sarah Townsend, Alice M Turner, Dimitris Kantas, Ross Edgar
Rok vydání:	2018
Předmět:	0301 basic medicine medicine.medical_specialty Cirrhosis Alpha 1-antitrypsin deficiency Hepatology business.industry medicine.medical_treatment Gastroenterology Liver transplantation medicine.disease Penetrance Natural history Transplantation Clinical trial 03 medical and health sciences Liver disease 030104 developmental biology 0302 clinical medicine Internal medicine medicine 030211 gastroenterology & hepatology Pharmacology (medical) business
Zdroj:	Alimentary Pharmacology & Therapeutics. 47:877-885
ISSN:	0269-2813
DOI:	10.1111/apt.14537
Popis:	BACKGROUND Alpha-1 antitrypsin deficiency (AATD) is estimated to affect three million people worldwide. It causes liver disease in a proportion of carriers of the PiS and PiZ allele due to the formation and retention of polymers within the endoplasmic reticulum of hepatocytes. The reason for this selective penetrance is not known. Although clinical trials are underway, liver transplantation is the only effective treatment for liver disease due to AATD. AIMS To report the prevalence and natural history of liver disease among individuals with AATD, and assess the outcomes of liver transplantation through systematic review. METHODS A comprehensive search was conducted across multiple databases. Two independent authors selected the articles and assessed bias using the Newcastle-Ottawa Scale. Data were pooled for analysis, where comparable outcomes were reported. RESULTS Thirty-five studies were identified related to disease progression and 12 for the treatment of AATD. Seven per cent of children were reported to develop liver cirrhosis, with 16.5% of individuals presenting in childhood requiring liver transplantation. Of those surviving to adulthood, 10.5% had liver cirrhosis and 14.7% required transplantation. Liver transplantation was the only effective treatment reported and outcomes compare favourably to other indications, with 5-year survival reported as over 90% in children and over 80% in adults. DISCUSSION The clinical course of liver disease in individuals with AATD remains poorly understood, but affects about 10% of those with AATD. More research is required to identify those patients at risk of developing liver disease at an early stage, and to provide alternative treatments to liver transplantation.
Databáze:	OpenAIRE
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