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Research Project

Descriptive Analysis of the Expert Opinion Regarding the Initiation of Augmentation Therapy in Alpha-1-Antitrypsin-Deficiency

Principal Investigator:
Timm Greulich
University Medical Center Giessen and Marburg
Marburg (Germany)
Start date:
November 2018
Finished and published
Contact E-mail:
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Although clear indications for augmentation therapy exists, there is a great variability in the indication of this treatment in patients with alpha-1 antitrypsin deficiency. This survey investigates the criteria used for experts in real clinical practice.
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To identify the clinical and functional criteria used by experts for the indication of augmentation therapy.

Inclusion criteria

Fifty participants from 14 different European countries answered the questionnaire. The majority of respondents were between 40 and 60 years of age. The five countries with the highest number of participating physicians were Germany, United Kingdom, Spain, Portugal and Italy.

Brief summary

We constructed and sent out a questionnaire to AATD experts to find out which factors influence the decision of AATD experts to start augmentation treatment. Therefore, we provided 50 experts with ten (different) virtual cases with limited information. The experts were asked whether they would recommend augmentation therapy (at this stage of the disease).
In a second part of the questionnaire, we asked the participants to rank available clinical information according to their importance.