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Inhaled alpha 1-antitrypsin: gauging patient interest in a new treatment
Richard Monk1, Michael Graves, Pamela Williams
1Division of Pulmonary, Critical Care, Allergy and Sleep Medicine, Department of Medicine, Medical University of South Carolina, Charleston, SC 29425-6300, USA. monk@musc.edu
Background:
Given the high cost of plasma derived intravenous alpha 1-antitrypsin (AAT), a more efficient method of delivery to the lungs is desirable. Inhaled AAT has been shown feasible for the treatment of alpha 1-antitrypsin deficiency (AATD) and is currently in clinical trials. To better understand patient preferences about possible inhaled AAT therapy, a survey was conducted to explore patient attitudes.
Methods:
We conducted an email based survey of patients in the Alpha-1 Foundation Research Registry with AATD on intravenous AAT replacement. Respondents were asked to rate their interest in hypothetical nebulized or dry powder inhaled AAT.
Results:
Respondents reported high levels of interest in both dried powder inhaler and nebulizer delivered inhaled AAT. The interest in dried powder inhaled was higher than interest in nebulized AAT (71% vs 64%, p = 0.0001). The interest in dried powder inhaled AAT was particularly high in respondents currently on bronchodilator therapy (p = 0.0053). Patients were just as likely to use or not use the product if it required 20% more out of pocket cost.
Conclusions:
There is a high level of patient interest in the development of a commercially available inhaled AAT replacement product.
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