Journal
FRONTIERS IN PHARMACOLOGY
Volume 14, Issue -, Pages -Publisher
FRONTIERS MEDIA SA
DOI: 10.3389/fphar.2023.1291677
Keywords
alpha-1 antitrypsin deficiency; disease burden; augmentation therapy; self-administration; independence; satisfaction
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Self-administered augmentation therapy is a successful alternative for individuals with alpha-1 antitrypsin deficiency, providing increased independence, satisfaction, and no interference with clinical stability.
Intravenous augmentation therapy with human alpha-1 proteinase inhibitor for the management of respiratory disease is recommended for people with alpha-1 antitrypsin deficiency (AATD) who are nonsmokers or former smokers. Augmentation therapy usually requires weekly administration at the hospital or clinic and poses an additional burden for patients due to interference with daily life, including work and social activities. Self-administration is a useful alternative to overcome this limitation, but there is a lack of published information on clinical outcomes. We report two cases of individuals with AATD at different stages of the disease who were successfully managed with self-administered augmentation therapy, with increased satisfaction because of the independence gained, lack of interference with clinical stability, and no relevant safety issues.
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