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1. Alpha-1 antitrypsin deficiency

The patient was diagnosed with alpha-1 antitrypsin (AAT-1) deficiency. Although there are reports of second-hand smoke causing lung disease, the presentation is classic for alpha-1 antitrypsin deficiency other than the patient is presenting somewhat later in life than usual. Topamax does not cause emphysema.

In AAT-1 deficiency circulating levels of AAT-1 are reduced due to an inherited deficiency usually seen in patients of Northern European origin. AAT-1 normally binds elastase released by neutrophils in the lung preventing tissue breakdown and emphysema. The obvious treatment is to replace circulating AAT-1 by infusion. However, when this was first proposed in the early 1980's it was dismissed as foolish and impractical. However, time has shown that AAT-1 replacement results in a reduction in the rate of decline of FEV1. However, the rate of decline varies between patients and AAT-1 replacement is quite expensive (usually over $80,000/year) and will not reverse severe emphysema. Lung transplant or lung volume reduction surgery can be performed on those with more severe emphysema.

Our patient had an AAT-1 level of 27.7 mg/dL (normal 150-350) and was phenotyped as ZZ rather than the normal MM. She is currently receiving alpha-1 replacement therapy and doing well.

References

  1. American Thoracic Society, European Respiratory Society. American Thoracic Society/European Respiratory Society statement: standards for the diagnosis and management of individuals with alpha-1 antitrypsin deficiency. Am J Respir Crit Care Med. 2003;168:818–900. [CrossRef] [PubMed]
  2. The Alpha-1-Antitrypsin Deficiency Registry Study Group. Survival and FEV1 decline in individuals with severe deficiency of alpha1-antitrypsin. Am J Respir Crit Care Med. 1998;158(1):49-59. [CrossRef] [PubMed]
  3. Chapman KR, Burdon JG, Piitulainen E, et al. Intravenous augmentation treatment and lung density in severe α1 antitrypsin deficiency (RAPID): a randomised, double-blind, placebo-controlled trial. Lancet. 2015;386(9991):360-8. [CrossRef] [PubMed]

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