Re: Researchers Say Drugs for AAT Deficiency Are Useless
I have read the review by Gotzsche and Johansen and here are a few points I would note:
They only looked at 2 studies that were not powered to show statsig efficacy, were quite different (even AAT dose was not the standard), had many dropouts in the placebo groups, they mainly looked at FEV1, which is not the best endpoint in the disease and doesn't reflect loss of lung tissue, and did not report mortality data (mortality is a very important outcome in this disease). So, while I agree that clinical trials with proper end points are needed to demonstrate efficacy of AAT augmentation therapy in alfa-1 antitrypsin deficiency, I certainly object to the authors' conclusion that the treatment is usless.
