Background: The efficacy of IV augmentation therapy
with human α1-protease inhibitor (α1-Pi) in
patients with severe α1-Pi deficiency is still under
Study objectives: To evaluate the progression
of emphysema in patients with α1-Pi deficiency before and
during a period in which they received treatment withα
retrospective cohort study.
clinics of 26 university clinics and pulmonary hospitals.
Patients: Ninety-six patients with severeα
1-Pi deficiency receiving weekly augmentation therapy
with human α1-Pi, 60 mg/kg of body weight, had a minimum
of two lung function measurements before and two lung function
measurements after augmentation therapy was started. Lung function data
were followed up for a minimum of 12 months both before and during
treatment (mean, 47.5 months and 50.2 months, respectively).
Measurements and results: Patients were grouped according
to the severity of their lung function impairment. The change in
FEV1 was compared during nontreatment and treatment
periods. In the whole group, the decline in FEV1 was
significantly lower during the treatment period (49.2 mL/yr vs 34.2
mL/yr, p = 0.019). In patients with FEV1 > 65%, IVα
1-Pi treatment reduced the decline in FEV1
by 73.6 mL/yr (p = 0.045). Seven individuals had a rapid decline of
FEV1 before treatment, and the loss in FEV1
could be reduced from 256 mL/yr to 53 mL/yr (p = 0.001).
Conclusion: Some patients with severe α1-Pi
deficiency and well-preserved lung function show a rapid decline in
FEV1. These patients profit from weekly IV therapy with
human α1-Pi and have less rapid decline if treated. Early
detection of patients at risk and early start of augmentation therapy
may prevent accelerated loss of lung tissue.