Abstract
Alpha-1 antitrypsin (AAT) augmentation therapy is the only specific treatment available for alpha-1 antitrypsin deficiency (AATD) related lung disease. Randomised control trials (RCTs) have consistently demonstrated its benefit in preserving lung density as measured with CT densitometry. These studies were not adequately powered to detect differences in mortality or progression to lung transplant. Use of retrospective data may provide insight into effects of augmentation on survival and progression to lung transplant.
Comparison was made between augmentation naive AATD patients from the AATD UK registry (UK) and patients on augmentation therapy followed up in AlphaNet’s Disease Management and Prevention Program in the US.
Kaplan-Meier survival analysis of 1535 adult patients with severe AATD and associated lung disease was performed with matching for age, sex and smoking status. Time to death or lung transplant was taken from start of augmentation (treatment) or baseline assessment (control). Estimated mean survival was significantly longer in the treatment group: augmentation 20.3 years (95%CI 19.4 to 21.2), control 13.7 years (95%CI 13.1 to 14.3) p<0.001 (Figure 1).
This study suggests treatment with augmentation therapy prolongs survival or time to lung transplantation compared to those who are augmentation naïve and should be considered for AATD patients with lung disease.
Footnotes
Cite this article as: European Respiratory Journal 2019; 54: Suppl. 63, PA3383.
This is an ERS International Congress abstract. No full-text version is available. Further material to accompany this abstract may be available at www.ers-education.org (ERS member access only).
- Copyright ©the authors 2019
