title: Inhaled alpha 1-antitrypsin: gauging patient interest in a new treatment.
abstract: 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.
authors.affiliation: Division of Pulmonary, Critical Care, Allergy and Sleep Medicine, Department of Medicine, Medical University of South Carolina, Charleston, SC 29425-6300, USA. monk@musc.edu
authors.forename: Richard
authors.initials: R
authors.lastname: Monk
authors.forename: Michael
authors.initials: M
authors.lastname: Graves
authors.forename: Pamela
authors.initials: P
authors.lastname: Williams
authors.forename: Charlie
authors.initials: C
authors.lastname: Strange
mesh_terms: D000280:Administration, Inhalation; D000328:Adult; D000368:Aged; D000369:Aged, 80 and over; D058995:Dry Powder Inhalers; D005260:Female; D006801:Humans; D008297:Male; D008875:Middle Aged; D009330:Nebulizers and Vaporizers; D057240:Patient Preference; D011795:Surveys and Questionnaires; D014361:Trypsin Inhibitors / Q000008:administration & dosage* / Q000191:economics; D000515:alpha 1-Antitrypsin / Q000008:administration & dosage* / Q000191:economics; D019896:alpha 1-Antitrypsin Deficiency / Q000188:drug therapy*
chemical_list: D014361:Trypsin Inhibitors; D000515:alpha 1-Antitrypsin