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Revista Portuguesa de Pneumologia

versão impressa ISSN 0873-2159


COSTA, Carla Alves  e  SANTOS, Cristina. Alpha -1 antitrypsin deficiency. The experience of Pulido Valente Hospital with augmentation therapy. Rev Port Pneumol [online]. 2009, vol.15, n.3, pp.473-482. ISSN 0873-2159.

Alpha -1 antitrypsin (AAT) is synthesised in the liver and has half -life of 4 -5 days. AAT has antiprotease activity, with particular affinity for neutrophil elastase. Its deficiency leads to a lack of effective lung protection against activated neutrophil enzymes. Deficiency of AAT is a genetic disorder that occurs as a result of the inheritance of two protease inhibitor deficient alleles. Of the deficient alleles, Pi*Z is the most common, and the homozygous form Pi*ZZ results in the lowest serum levels, usually below 50 mg/dl. The “protective threshold” is 80 mg/dl. Smoking increases the risk of emphysema. The current goal of augmentation therapy is to raise the plasma levels, above protective threshold and slow disease progression. The authors present the experience of the Day Care Hospital of the Pulido Valente Hospital with five male patients presenting emphysema due to AAT deficiency, receiving weekly intravenous treatment with ProlastinR. We performed a clinical, respiratory functional and radiological evaluation between 2003 and 2007. The results point to a slower progression of the disease, with clinical and radiological stability and a reduced rate of FEV1 decline. Augmentation therapy is an expensive treatment and its use is lacking supportive evidence of efficacy by randomized controlled clinical trials. Evidence that it confers benefits is based on observational studies. Our experience is positive, showing clinical, radiological and functional benefits. The literature available points to a decrease in mortality, but we could not affirm so in our small population.

Palavras-chave : Alpha -1 antitrypsin; augmentation therapy; emphysema.

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