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Table 1 Comparison of study characteristics

From: Therapeutic efficacy of alpha-1 antitrypsin augmentation therapy on the loss of lung tissue: an integrated analysis of 2 randomised clinical trials using computed tomography densitometry

  Danish-Dutch trial EXACTLE trial
Genotype/phenotype PiZZ on IEF PiZZ or severe deficiency with AAT concentrations <11 μM
Lung function, FEV1 30-80% 25-80% and FEV1/VC ≤70% or
Kco NA ≤80% if spirometry normal
Exacerbations NA ≥1 exacerbation in the past 2 years
Smoking history Never or ex-smokers for >6 months Cotinine checked every 4 weeks Never or ex-smokers for >6 months Cotinine checked at 1, 6, 24 and 30 months
Previous augmentation therapy NA Never or ≤1 month in past 2 years
Study design Randomised, double-blind, placebo-controlled Randomised, double-blind, placebo-controlled
AAT dosing 250 mg•kg-1 body weight AAT 60 mg•kg-1 body weight AAT
Treatment interval Every 4 weeks Every week
Placebo 625 mg•kg-1 body weight albumin 2% albumin
Centres 2 (Copenhagen, Leiden) 3 (Copenhagen, Birmingham, Malmö)
Duration of study Minimum 3 years 24 months (optional 6-months extension)
Study period January 1991 to August 1997 November 2003 to December 2006
Primary endpoints FEV1 measured by home spirometry twice daily Change in PD15 measured by CT
Other endpoints Change in PD15 measured by CT Exacerbations
Lung function (FEV1, KCO)
Quality of life (SGRQ)
  1. AAT: alpha-1 antitrypsin; EXACTLE: Exacerbations and Computed Tomography scan as Lung Endpoints; IEF: isoelectric focusing; Kco: carbon monoxide transfer coefficient; NA: not applicable; PD15: 15th percentile lung density; SGRQ: St George's Respiratory Questionnaire; VC: vital capacity. CT: computed tomography.