Diagnosis and Monitoring Pathways Using Non‐Invasive Tests in Patients With Alpha‐1 Antitrypsin Deficiency‐Associated Liver Disease: Results From an Expert Delphi Panel

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Abstract
Background and Aims: The severe alpha‐1 antitrypsin deficiency (AATD) genotype Pi*ZZ increases the risk of liver disease (AATD‐LD) and lung disease. While non‐invasive tests (NITs) are widely used for fibrosis stage and monitoring of all liver diseases, the consensus for use in AATD‐LD is limited. A Delphi panel study was conducted to address this need.Method: Healthcare providers who managed at least two patients with AATD‐LD in the past two years participated. Two iterative surveys were developed and administered. The second survey clarified the results from the first and provided deeper feedback. As follow‐up, a real‐time consensus‐building exercise focused on survey topics without consensus. Controlled feedback was anonymous.Results: A total of 20 AATD experts (hepatology [n = 9], pulmonology [n = 6], transplant hepatology [n = 3], gastroenterology [n = 1], and hepatology and transplant hepatology [n = 1]) completed the study. A strong consensus was achieved around the use and evaluation of NITs for risk stratification and monitoring. All panelists agreed that vibration‐controlled transient elastography (VCTE) is the preferred NIT for the initial assessment of AATD‐LD owing to its accessibility and reliability. Magnetic resonance elastography and enhanced liver fibrosis tests were also considered valuable. Most (85%) agreed that VCTE < 8 kPa could indicate no or mild fibrosis and VCTE ≥ 8 kPa could indicate clinically significant fibrosis, which may correspond to fibrosis stage ≥ F2 on the METAVIR scale. Most (85%) agreed that VCTE ≥ 13 kPa may indicate cirrhosis.Conclusion: Utilizing the Delphi technique, this study identified a clinically applicable framework for the diagnosis and monitoring of AATD‐LD. A high level of agreement emerged regarding preferred NITs and their usage, risk stratification and monitoring in the context of AATD‐LD management. The results provide a foundation for future efforts into NIT validation and the development of clinical guidelines for AATD‐LD.
Funding Information
  • Deutsche Forschungsgemeinschaft (SFB1382(ID403224013),STR1095/6‐1)
  • Takeda Pharmaceuticals U.S.A.
  • National Institute for Health and Care Research
  • National Center for Advancing Translational Sciences (5UL1TR001442)
  • National Institute of Diabetes and Digestive and Kidney Diseases (P30DK120515, R01DK106419, R01DK121378, R01DK124318, U01DK061734, U01DK130190)
  • National Heart, Lung, and Blood Institute (P01HL147835)

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