MIAMI, FL – The Alpha-1 Foundation today challenged a newly published review which questions the value of augmentation therapy for Alpha-1 Antitrypsin Deficiency (Alpha-1).
The article, Intravenous alpha-1 antitrypsin augmentation therapy for treating patients with alpha-1 antitrypsin deficiency and lung disease by Peter C. Gøtzsche and Helle Krogh Johansen, was published this week by the Cochrane Library.
“The Cochrane Library has been respected in the scientific community for carefully performed reviews based on solid evidence,” said Foundation President and CEO John Walsh.
“But this report is so flawed in its methodology that it may threaten the reputation of the Cochrane Library. The report does a disservice to rare disease patients everywhere. We hope that therapies for other rare conditions won't become victims of the same poorly designed analysis.”
Robert Stockley, MD, Director of Research and Development at Queen Elizabeth Hospital, Birmingham, UK, had this critique:
“This conclusion was based on retrospective analysis of published data from only two small pilot placebo-controlled studies that were not powered to evaluate the effectiveness of augmentation therapy. This flies in the face of carefully crafted guidelines from the American Thoracic Society, the European Respiratory Society, the American College of Chest Physicians, and the American Association for Respiratory Care – all prestigious organizations that recommend augmentation therapy for the treatment of patients with lung disease due to Alpha-1. The guidelines are based on the totality of the evidence, scientific understanding of the disease, correcting the biochemical defect, and a wealth of observational studies.”
Danish researcher Asger Dirksen, MD, originally listed as a co-author of the Gøtzsche review, had his name removed before publication.
Dirksen, who was the lead author of both augmentation studies cited by Gøtzsche, said today:
“After seeing the first draft I realized that our points of view were so far apart that collaboration with Peter Gøtzsche and his wife (Helle Krogh Johansen) would not be possible.”