Study objectives: To propose an hypothesis that
antiprotease augmentation therapy reduces the incidence of lung
infections in α1-antitrypsin (AAT)-deficient patients,
and to present supporting data.
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.
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 humanα
1-proteinase inhibitor (α1-PI) responded,
as did 47 similar patients not receiving augmentation therapy.
Results: Seventy-four of 89 patients who had receivedα
1-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 α1-PI infusions.
Before starting α1-PI, the majority of patients had three
to five infections per year, dropping to zero to one infection per year
during α1-PI therapy (p < 0.001).
Conclusions: Replacement therapy for AAT
deficiency-associated emphysema appears to be associated with a marked
reduction in the frequency and severity of lung infections. This
association must be evaluated further in future, more rigid,
prospective studies of AAT augmentation therapy. Findings support the
hypothesis that antiprotease therapy with α1-PI reduces
the incidence of lung infections in addition to slowing the
deterioration of lung function and causing a reduction in