PMID- 29446109
OWN - NLM
STAT- MEDLINE
DCOM- 20190531
LR  - 20210109
IS  - 1365-2036 (Electronic)
IS  - 0269-2813 (Linking)
VI  - 47
IP  - 7
DP  - 2018 Apr
TI  - Systematic review: the natural history of alpha-1 antitrypsin deficiency, and 
      associated liver disease.
PG  - 877-885
LID - 10.1111/apt.14537 [doi]
AB  - 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.
CI  - (c) 2018 John Wiley & Sons Ltd.
FAU - Townsend, S A
AU  - Townsend SA
AUID- ORCID: 0000-0002-6811-3939
AD  - National Institute for Health Research Liver Biomedical Research Unit, University 
      Hospitals Birmingham, NHS Foundation Trust, University of Birmingham, Birmingham, 
      UK.
AD  - Centre for Liver Research, Institute of Immunology and Immunotherapy, University 
      of Birmingham, Birmingham, UK.
AD  - Liver Unit, BirminghamInstitute of Inflammation and Ageing, University Hospitals 
      Birmingham NHS Foundation Trust, University of Birmingham, Birmingham, UK.
FAU - Edgar, R G
AU  - Edgar RG
AD  - Centre for Liver Research, Institute of Immunology and Immunotherapy, University 
      of Birmingham, Birmingham, UK.
AD  - Institute of Applied Health Research, University of Birmingham, Birmingham, UK.
FAU - Ellis, P R
AU  - Ellis PR
AUID- ORCID: 0000-0003-3817-3882
AD  - Heart of England NHS Foundation Trust, Birmingham, UK.
FAU - Kantas, D
AU  - Kantas D
AD  - Centre for Liver Research, Institute of Immunology and Immunotherapy, University 
      of Birmingham, Birmingham, UK.
FAU - Newsome, P N
AU  - Newsome PN
AUID- ORCID: 0000-0001-6085-3652
AD  - National Institute for Health Research Liver Biomedical Research Unit, University 
      Hospitals Birmingham, NHS Foundation Trust, University of Birmingham, Birmingham, 
      UK.
AD  - Centre for Liver Research, Institute of Immunology and Immunotherapy, University 
      of Birmingham, Birmingham, UK.
AD  - Liver Unit, BirminghamInstitute of Inflammation and Ageing, University Hospitals 
      Birmingham NHS Foundation Trust, University of Birmingham, Birmingham, UK.
FAU - Turner, A M
AU  - Turner AM
AD  - Institute of Applied Health Research, University of Birmingham, Birmingham, UK.
AD  - Heart of England NHS Foundation Trust, Birmingham, UK.
LA  - eng
GR  - CDRF-2014-05-044/DH_/Department of Health/United Kingdom
PT  - Journal Article
PT  - Research Support, Non-U.S. Gov't
PT  - Systematic Review
DEP - 20180215
PL  - England
TA  - Aliment Pharmacol Ther
JT  - Alimentary pharmacology & therapeutics
JID - 8707234
SB  - IM
CIN - Aliment Pharmacol Ther. 2018 Jul;48(2):232-233. PMID: 29939411
CIN - Aliment Pharmacol Ther. 2018 Jul;48(2):233. PMID: 29939419
MH  - Adult
MH  - Child
MH  - Disease Progression
MH  - Humans
MH  - Liver Cirrhosis/epidemiology/etiology/*pathology/therapy
MH  - Liver Transplantation
MH  - Prevalence
MH  - Treatment Outcome
MH  - alpha 1-Antitrypsin Deficiency/complications/epidemiology/*pathology/therapy
EDAT- 2018/02/16 06:00
MHDA- 2019/06/01 06:00
CRDT- 2018/02/16 06:00
PHST- 2017/10/19 00:00 [received]
PHST- 2017/11/21 00:00 [revised]
PHST- 2018/01/03 00:00 [accepted]
PHST- 2018/02/16 06:00 [pubmed]
PHST- 2019/06/01 06:00 [medline]
PHST- 2018/02/16 06:00 [entrez]
AID - 10.1111/apt.14537 [doi]
PST - ppublish
SO  - Aliment Pharmacol Ther. 2018 Apr;47(7):877-885. doi: 10.1111/apt.14537. Epub 2018 
      Feb 15.