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Review: Human alpha-1-proteinase inhibitor for patients with AAT deficiency


The Canadian Agency for Drugs and Technologies in Health (CADTH) has published a systematic review of human a1-proteinase inhibitor (a1-PI) IV infusion for patients with a1- antitrypsin (AAT) deficiency, with or without a diagnosis of COPD.

AAT deficiency was first described in 1963; pulmonary emphysema presenting as COPD is the most prevalent clinical presentation, and is the major cause of disability and death, with liver disease being the second most frequent clinical complication. Purified a1-PI is used for chronic replacement therapy of individuals having congenital deficiency of a1-PI with clinically demonstrable panacinar emphysema. The review concluded the following:

• Evidence showing health improvement is inconclusive.

• In controlled trials, 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.

• Anticipated health gains may cost more than other funded treatments in Canada.

National Electronic Library for Medicine 06/03/2007


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