Augmentation therapy with alpha1-antitrypsin: patterns of use and adverse events.

Study Objectives: To describe patterns of prescribing augmentation therapy, and types and rates of adverse events in the National Heart, Lung, and Blood Institute Registry for Individuals with Severe Deficiency of Alpha(1)-Antitrypsin.

Design: Observational cohort study with follow-up visits every 6 to 12 months for up to 7 years.

Measurements: The rate and dosing frequency with which Registry participants were prescribed to receive augmentation therapy by their managing physicians, and the type and frequency of adverse events, classified in two ways: severity of self-reported symptoms, and actions taken as a consequence of the symptom.

Asthma features in severe alpha1-antitrypsin deficiency: experience of the National Heart, Lung, and Blood Institute Registry.

Study Objectives: To describe asthma features in a cohort with alpha(1)-antitrypsin (AAT) deficiency, and determine the impact of asthma on FEV(1) decline.

Background: Asthma may be common in those with AAT deficiency, and may lead to accelerated airflow obstruction.

Design: Analysis of data obtained from a 5-year, prospective National Heart, Lung, and Blood Institute registry.