Personalizing liver targeted treatments and transplantation for patients with alpha-1 antitrypsin deficiency

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Introduction: There is currently no specific treatment for liver disease due to alpha-1 antitrypsin deficiency (AATD) other than care applied for other liver diseases including transplantation. This review describes the personalized approaches to liver disease in AATD, and the current stage of development of new therapeutic agents.

Areas covered: We review the pathology, presentation and progression of AATD liver disease and the approaches being taken to understand the natural history of the disease to aid future therapeutic advances. Peri- and post-transplant care is described and we highlight the reasons that alternative approaches to avoid the need for liver transplantation are being explored. The role of patient selection for new therapies is addressed as this is likely to be of paramount importance to achieve better outcomes.

Expert opinion: Treatments directed at both liver, and lung and liver combined, are now being trialed in patients with AATD. The next 5–10 years may determine a more reliable noninvasive measurement of liver fibrosis, together with predictors of who is most likely to progress and develop cirrhosis. Personalized approaches could optimize when and how to effectively manage an individual with a cost-effective treatment and avoid progression to liver transplantation.
Original languageEnglish
Pages (from-to)65-78
Number of pages14
JournalExpert Review of Precision Medicine and Drug Development
Issue number1
Early online date10 Dec 2020
Publication statusPublished - 2 Jan 2021

Bibliographical note

Publisher Copyright:
© 2020 Informa UK Limited, trading as Taylor & Francis Group.


  • Alpha 1-antitrypsin deficiency
  • cirrhosis
  • precision medicine

ASJC Scopus subject areas

  • Molecular Medicine
  • Genetics
  • Pharmacology
  • Drug Discovery


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