Review of augmentation therapy for alpha-1 antitrypsin deficiency

Augmentation therapy has become somewhat controversial. A recent Cochrane review, concluding that augmentation therapy was ineffective, was simplistic and heavily criticized. A new review published in Therapeutic Advances in Respiratory Disease provides a careful look at the evidence. The level of evidence regarding augmentation therapy is not as high as we might like. We have to go with the best evidence we have, which suggests that augmentation therapy does impact the progression of the disease and provides meaningful clinical benefits for selected patients. As the review pointed out there are areas of controversy:

Areas where controversy exists regarding the use of AAT augmentation therapy include: (1) indications for treatment, (2) selection of specific AAT augmentation therapy, (3) appropriate dose and interval of administration, (4) cost effectiveness, (5) frequency and mode of follow up of treated patients, (6) use of augmentation therapy after lung transplantation, (7) use of recombinant AAT supplementation, (8) alternative delivery routes, and (9) genetic therapy. 

These are decisions that require special expertise, suggesting to me that patients with COPD who might have AAT should be screened and appropriate candidates referred to centers where such expertise is available.