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Clinical Commentary |

α1-Antitrypsin Augmentation Therapy for PI*MZ Heterozygotes: A Cautionary Note

Robert A. Sandhaus, MD, PhD, FCCP; Gerard Turino, MD; James Stocks, MD, FCCP; Charlie Strange, MD, FCCP; Bruce C. Trapnell, MD; Edwin K. Silverman, MD, PhD; Sarah E. Everett, MEd, JD; James K. Stoller, MD, MS, FCCP; for the Medical and Scientific Advisory Committee of the Alpha-1 Foundation
Author and Funding Information

*From the National Jewish Medical and Research Center (Dr. Sandhaus); Denver, CO; St. Luke's-Roosevelt Hospital Center (Dr. Turino), Columbia University College of Physicians & Surgeons, New York, NY; University of Texas Health Sciences Center at Tyler (Dr. Stocks), Tyler, TX; Medical University of South Carolina (Dr. Strange), Charleston, SC; Cincinnati Children's Hospital Medical Center (Dr. Trapnell), University of Cincinnati School of Medicine, Cincinnati, OH; Channing Laboratory (Dr. Silverman), Brigham and Women's Hospital, Boston, MA; Medical and Scientific Advisory Committee (Ms. Everett), Alpha-1 Foundation, Miami, FL; and Cleveland Clinic Foundation (Dr. Stoller), Cleveland, OH.

Correspondence to: Robert A. Sandhaus, MD, PhD, FCCP, Alpha-1 Foundation, 2937 SW 27th Ave, Maimi, FL 33133; e-mail: rasandhaus@alphaone.org

Dr. Sandhaus has presented talks relating to AAT deficiency at events sponsored by Talecris Biotherapeutics, CSL Behring, Baxter Healthcare, and Dey Pharmaceuticals, and has been a principal investigator for therapeutic clinical trials in AAT deficiency sponsored by Talecris Biotherapeutics, CSL Behring, and Kamada Pharmaceuticals. Dr. Turino has no conflicts of interest to disclose. Dr. Stocks has received compensation for serving on the advisory boards of, and has also been the recipient of research funding from, Bayer, Talecris, CSL Behring, Baxter, and Kamada for AAT drug development. In the past 3 years, Dr. Strange has consulted for Arriva, GTC Biotherapeutics, and CSL Behring, and is on the speaker's bureau for Talecris with total amounts of compensation less than US $10,000. He has held grants from Talecris, the Alpha-1 Foundation, Alpha-1 Association, and the National Institutes of Health for study of AAT deficiency. Dr. Trapnell has no conflicts of interest to disclose. Dr. Silverman received an honorarium for a talk on COPD genetics in 2006, grant support and consulting fees from GlaxoSmithKline for two studies of COPD genetics, an honorarium from Bayer Biologicals for a symposium at the 2005 European Respiratory Society Meeting, and an honorarium for a talk at the Lund Symposium in 2007 and consulting fees from AstraZeneca. Ms. Everett has severe AAT deficiency, receives augmentation therapy, and has been a member of the voluntary leadership of the Alpha-1 Foundation for the past 12 years. Dr. Stoller has served as a consultant to Talecris Biotherapeutics; has given lectures that have been supported by Talecris Biotherapeutics, Baxter Healthcare, Grifols, and CSL Behring; and has served as a member of data monitoring and safety committee for Kamada Pharmaceuticals.


All authors are members of the Alpha-1 Foundation Medical and Scientific Advisory Committee. Additional membership can be found at the Alpha-1 Foundation Web site (www.alphaone.org). Dr. Silverman is also a member of the Alpha-1 Foundation Board of Directors.

In addition to individual disclosures, the authors wish to disclose the following potential conflicts of interest: the Alpha-1 Foundation relies entirely on donations for its operating budget. Included among the major donors to the Alpha-1 Foundation are all the companies that produce the augmentation therapy products mentioned in this commentary.

Reproduction of this article is prohibited without written permission from the American College of Chest Physicians (www.chestjournal.org/misc/reprints.shtml).


Chest. 2008;134(4):831-834. doi:10.1378/chest.08-0868
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The use of IV augmentation therapy with plasma-derived α1-antitrypsin (AAT) has become the standard of care for the treatment of pulmonary disease associated with the severe genetic deficiency of AAT. The Medical and Scientific Advisory Committee of the Alpha-1 Foundation has become aware that physicians are prescribing this expensive blood product for the treatment of individuals with a single abnormal AAT gene, primarily the PI*MZ genotype. We are aware of no evidence that such therapy is effective in this patient population. The most important therapeutic interventions in such patients remain smoking cessation and elimination of other risk factors for lung disease. This commentary discusses the treatment of AAT deficiency and the concerns regarding treatment of PI*MZ individuals. We conclude that clinicians should avoid prescribing augmentation therapy for this heterozygote population.


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