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Research Project

Defining Rapid Decliners : Implications for identifying clinical benefit in AATD

Principal Investigator:
Robert A. Stockley
University Hospitals Birmingham NHS Foundation Trust
Birmingham (UK)
Start date:
December 2021
Contact E-mail:
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Although cigarette smoking generally accelerates lung function decline in AATD never smokers and ex-smokers have variable rates of progression with respect to FEV1, gas transfer and CT densitometry. Many patients retain normal lung function and smoking cessation alone can stabilise lung function in others. The limited clinical trials to date based on cross sectional demographic recruitment parameters have supported a benefit of augmentation therapy on progression of lung densitometry although not lung function or general health status. For this reason augmentation has remained unavailable in many countries and may be overprescribed in others. It is likely that the generalised inclusion of patients based on cross sectional static inclusion parameter hides potential benefits particularly in the limited numbers able to be recruited. For instance, in studies of therapy for exacerbations it is easier to demonstrate an effect of treatment when patients with a history of such episodes are recruited. Hence a physiological outcome in augmentation trials will only be shown to be beneficial in those in whom it is rapidly declining at the point of entry.


The purpose of the study is to characterise “rapid progression” and its clinical impact prospectively and explore the possibility of developing composite scoring to determine and define the benefits of augmentation in a highly characterised and monitored group of AATD subjects.


Inclusion criteria

Patients with ZZ or Znull AAT genotypes will be recruited from centres where augmentation therapy is not currently available or where the baseline physiology is not within current guidelines for treatment.

Brief summary

This project requires EARCO participant centres meeting the following characteristics

  1. Enrolled at least 5 patients not receiving augmentation therapy and >30yrs. It is especially valuable if such patients are in the early stages of the disease (normal FEV1 or mild Gold stage COPD), but not essential.
  2. Access and experience in CT acquisition for densitometry analysis
  3. Access to full, post bronchodilator lung function including gas transfer and lung volumes routine exercise testing.
Experience in clinical trial delivery to ensure consistency in highest quality data collection.