Genetically-caused emphysema: treatment is effective

Although augmentation therapy against genetically-caused emphysema has been available for 25 years, it is rarely used. Physicians are uncertain whether or not it works. A Canadian study published in “The Lancet” now confirms the treatment’s efficacy.

Researchers from Toronto Western Hospital recruited 180 patients with alpha-1-antitrypsin deficiency from 13 countries for their study. One group of patients was treated with augmentation therapy for two years, the other group received a placebo.

With alpha-1-antitrypsin deficiency, the AAT protein that develops in the liver does not reach the lungs to protect them, but instead accumulates in the liver. Augmentation therapy therefore involves regular infusions with purified AAT protein to raise its level in the blood and lungs.

Whether or not the treatment was effective, was examined using CT-scans to measure lung density. It clearly showed that the treatment worked: the annual rate of lung density change in the augmentation-group was significantly lower than that of the control group. Adverse effects occurred in both groups to the same extent.

“Augmentation therapy not only preserves lung structure, but likely adds years of life”, said study author Kenneth Chapman. “Patients with this condition need access to timely diagnosis and treatments to ensure they receive the best possible care”, he emphasised. However, only persons with genetically-caused emphysema benefit from this treatment.


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