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Is augmentation therapy recommended for MZs?


  • There is no scientific evidence (or even compelling suspicion) that it helps.
  • There are both clinical and economic adverse effects. 
  • It depletes insurance money that could be used for other treatments, such as for COPD and other common morbidities.

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.

Chest Journal, October 2008, Volume 134, Issue 4, Pages 831–834
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

Intravenous augmentation therapy is not recommended for: Individuals with the MZ genotype of AATD.

Clinical Practice Guidelines – The Diagnosis and Management of Alpha-1 Antitrypsin Deficiency in the Adult