Long-term evolution of lung function in individuals with alpha-1 antitrypsin deficiency from the Spanish registry (REDAAT)

Abstract

Background

The clinical course of alpha-1 antitrypsin deficiency (AATD) is very heterogeneous. It is estimated that 60% of individuals with severe AATD (Pi*ZZ) develop emphysema. The main objective of this study was to describe the outcomes of long-term lung function in individuals with AATD-associated emphysema after at least 8 years of follow-up.

Materials and methods

We performed a retrospective analysis of longitudinal follow-up data of AATD PiZZ patients from the Spanish registry (AATD Spanish Registry [REDAAT]). The main follow-up outcome was the annual rate of decline in forced expiratory volume in 1 second (FEV₁) calculated using the FEV₁ values at baseline and in the last post-bronchodilator spirometry available.

Results

One hundred and twenty-two AATD PiZZ patients were analyzed. The median follow-up was 11 years (interquartile range =9–14). The mean FEV₁ decline was 28 mL/year (SD=54), with a median of 33 mL/year. Tobacco consumption (β=19.8, p<0.001), previous pneumonia (β=27.8, p=0.026) and higher baseline FEV₁% (β=0.798, p=0.016) were independently related to a faster FEV₁ decline.

Conclusion

In this large cohort with a long follow-up, we observed a very variable decline of FEV₁. However, the mean FEV₁ decline was similar to that observed in large cohorts of smoking-related COPD. Tobacco consumption, previous pneumonia and better lung function at baseline were related to a faster decline in FEV₁. These results highlight the importance of early diagnosis and effective treatment.

Keywords:

• alpha-1 antitrypsin deficiency
• lung function
• registers

Acknowledgments

The Alpha-1 Antitrypsin Deficiency Spanish Registry (REDAAT) is supported in part by an unrestricted grant from Grifols (Barcelona, Spain). Part of the results of this study have been presented in the form of an abstract to the European Respiratory Society (ERS) International Congress 2015 (Amsterdam, the Netherlands).

Disclosure

Sonia Serreri was supported by an Erasmus placement 2014–2015 from Università di Sassari (Sassari, Italy). Marc Miravitlles has received speaker fees from Boehringer Ingelheim, Chiesi, Cipla, Menarini, Grifols and Novartis, and consulting fees from Boehringer Ingelheim, Glaxo-SmithKline, Gebro Pharma, CSL Behring, Novartis and Grifols. Miriam Barecheguren has received speaker fees from Menarini, GlaxoSmithKline and Gebro Pharma. The authors report no other conflicts of interest in this work.