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Published ahead of print on February 5, 2003, doi:10.1164/rccm.200209-1035OC

Am. J. Respir. Crit. Care Med., Volume 167, Number 10, May 2003, 1387-1392

A more recent version of this article appeared on May 15, 2003
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Submitted on September 13, 2002
Accepted on February 3, 2003

Cost-Effectiveness Analysis of Augmentation Therapy for Severe Alpha 1-Antitrypsin Deficiency

Thomas R Gildea1*, Kenneth M Shermock2, Mendel E Singer3, and James K Stoller1

1 Pulmonary and Critical Care Medicine and Pharmacy, The Cleveland Clinic Foundation, Cleveland, OH, USA, 2 Center for Pharmaceutical Outcomes and Policy, The Johns Hopkins Hospital, Baltimore, MD, USA, 3 Center for Quality Improvement Research, Cleveland Department of Veterans Affairs Medical Center, Cleveland, OH, USA; Institute for Public Health Sciences, MetroHealth Medical Center, Cleveland, OH, USA

* To whom correspondence should be addressed. E-mail: gildeat{at}ccf.org.

A Markov-based decision model was created to assess the cost-effectiveness of augmentation therapy (Aug) for severe alpha 1-antitrypsin (AAT) deficiency, comparing strategies of: 1. No Aug, 2. Aug for life, and 3. Aug until FEV1 < 35% predicted. A hypothetical cohort of 46 year old patients with FEV1 49% predicted was followed over time using Monte Carlo simulation across 5 possible health states: 1. FEV1 50-79% predicted, 2. FEV1 35-49% predicted, 3. FEV1 < 35% predicted, 4. Status-post lung transplantation, and 5. Dead. Treatment for life yielded 7.19 quality-adjusted life-years (QALYs) and cost $895,243. Treating until FEV1 < 35% predicted cost $511,930 and produced 6.64 QALYs. "No Aug" cost $92,091 with 4.62 QALYs. The incremental cost-effectiveness ratio (ICER) was $207,841/QALY for Aug until FEV1 < 35% predicted and $312,511/QALY for the "Aug for life" strategy. In all sensitivity analyses, the ICER for "Aug for life" exceeded $100,000. The cost of Aug needed to be reduced from $54,765 to $4,900 for the "Aug for life" strategy to become cost-effective. We conclude that, compared with other conventionally used health interventions, Aug is less cost-effective. These results should encourage the development of more clinically and cost-effective therapies for AAT deficiency.
Key words: cost-effectivness, alpha 1-antitrypsin defiency, decision analysis




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