alpha1-Antitrypsin augmentation therapy for PI*MZ heterozygotes: a cautionary note |
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Authors: | Sandhaus Robert A,Turino Gerard,Stocks James,Strange Charlie,Trapnell Bruce C,Silverman Edwin K,Everett Sarah E,Stoller James K Medical Scientific Advisory Committee of the Alpha- Foundation |
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Affiliation: | National Jewish Medical and Research Center, Denver, CO, USA. rasandhaus@alphaone.org |
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Abstract: | The use of IV augmentation therapy with plasma-derived alpha1-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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