A New Hypothesis With Supporting Data

Study objectives: To propose an hypothesis that antiprotease augmentation therapy reduces the incidence of lung infections in a1-antitrypsin (AAT)-deficient patients, and to present supporting data. Design: The proposed concept is based on a survey taken via the Internet of patients receiving augmentation therapy for 1 to 10 years compared to similar patients not receiving such therapy. Setting: A questionnaire was submitted to patients with a ZZ phenotype for AAT deficiency to determine whether those receiving antitrypsin augmentation therapy were aware of any personal benefit, and whether the therapy had an effect on the frequency of lung infections. Patients: Ninety-six adult patients receiving human a1-proteinase inhibitor (a1-PI) responded, as did 47 similar patients not receiving augmentation therapy. Results: Seventy-four of 89 patients who had received a1-PI infusions for> 1 year believed that they had definitely benefited from such therapy. Fifty-six of the 74 patients claiming a benefit attributed this to a reduction in the number of lung infections since starting therapy with a1-PI infusions. Before starting a1-PI, the majority of patients had three to five infections per year, dropping to zero to one infection per year during a1-PI therapy (p < 0.001). Conclusions: Replacement therapy for AAT deficiency-associated emphysema appears to b