Exacerbations in subjects with alpha-1 antitrypsin deficiency receiving augmentation therapy.
BACKGROUND
The frequency, characteristics and impact of acute exacerbations in patients with alpha-1 antitrypsin deficiency (AATD) and COPD who are on intravenous alpha-1 antitrypsin augmentation therapy have not been described.


METHODS
922 subjects with AATD and COPD on augmentation therapy (mean age 54.5 years) were followed with monthly telephone surveys to record exacerbation characteristics, as well as healthcare resource utilization and health-related quality of life (HRQoL). Exacerbations were defined by symptom-based and healthcare resource utilization (HRU) criteria.


RESULTS
During the 1-year follow-up, 91.5% of participants experienced at least one exacerbation (mean 2.4 exacerbations per subject, median 2, and mean duration 17 days per episode, regardless of the definition used). Most exacerbations were categorized as severe by symptoms and moderate by HRU criteria. Subjects who had 3 or more exacerbations (48.6%) were younger, had higher medication use and had higher tobacco consumption compared with subjects with less exacerbations. Subjects with frequent exacerbations had the worst baseline HRQoL scores, as well as more physician visits, emergency room visits, and hospitalizations. Although most subjects received augmentation therapy on a weekly basis, other infusion schedules were more commonly observed in subjects with fewer exacerbations.


CONCLUSIONS
COPD exacerbations occur frequently and are associated with significant disease burden in subjects with AATD receiving augmentation therapy.
Campos
Michael A
MA
University of Miami Miller School of Medicine, Division of Pulmonary and Critical Care Medicine, P.O. Box 016960 (R-47), Miami, FL 33101, USA. mcampos1@med.miami.edu
Alazemi
Saleh
S
Zhang
Guoyan
G
Wanner
Adam
A
Salathe
Matthias
M
Baier
Horst
H
Sandhaus
Robert A
RA
D018450:Disease Progression; D005260:Female; D006304:Health Status; D006801:Humans; D008297:Male; D008875:Middle Aged; D029424:Pulmonary Disease, Chronic Obstructive / Q000150:complications / Q000503:physiopathology* / Q000628:therapy; D011788:Quality of Life; D012907:Smoking / Q000009:adverse effects*; D000515:alpha 1-Antitrypsin / Q000627:therapeutic use*; D019896:alpha 1-Antitrypsin Deficiency / Q000209:etiology / Q000503:physiopathology* / Q000628:therapy
D000515:alpha 1-Antitrypsin