Human a1-proteinase inhibitor for patients with a1-antitrypsin deficiency

Chen S, Farahati F, Marciniuk D, Mayers I, Boudreau R, Keating T
Record ID 32007000119
English
Authors' objectives:

The objectives were to assess the clinical and economic implications of human a1-proteinase inhibitor (a1-PI) intravenous infusion for patients with a1- antitrypsin (AAT) deficiency, with or without a diagnosis of chronic obstructive pulmonary disease.

Authors' results and conclusions: One randomized controlled trial, four retrospective cohort studies, and eight case series reports provided evidence of clinical effect. Three cost-effectiveness and two cost studies conducted outside Canada were identified.
Authors' recommendations: Evidence showing health improvement is inconclusive. In controlled trials, augmentation therapy has not shown reduced lung function impairment in patients with AAT deficiency and COPD, compared with normal care. Conversely, in observational studies, a1-PI is associated with outcomes suggestive of therapeutic benefit in patients with severe AAT deficiency and moderate airflow obstruction. Severe adverse events from treatment have been reported in ~1% of study populations. Use in patients without COPD is experimental. No evidence was found evaluating the use of a1-PIs in patients with AAT deficiency and no lung function impairment.
Authors' methods: Systematic review
Details
Project Status: Completed
Year Published: 2007
English language abstract: An English language summary is available
Publication Type: Not Assigned
Country: Canada
MeSH Terms
  • alpha 1-Antitrypsin Deficiency
  • Protease Inhibitors
Contact
Organisation Name: Canadian Agency for Drugs and Technologies in Health
Contact Address: 600-865 Carling Avenue, Ottawa, ON K1S 5S8 Canada. Tel: +1 613 226 2553; Fax: +1 613 226 5392;
Contact Name: requests@cadth.ca
Contact Email: requests@cadth.ca
Copyright: Canadian Agency for Drugs and Technologies in Health (CADTH)
This is a bibliographic record of a published health technology assessment from a member of INAHTA or other HTA producer. No evaluation of the quality of this assessment has been made for the HTA database.