Biotech Values

[genisi]

Meanwhile, here are two responses to that paper:

Prof. Albert Farrugia from the Plasma Protein Therapeutics Association:

Statement on Review - Intravenous Alpha-1 Antitrypsin Augmentation Therapy

http://www.pptaglobal.org/news/news.aspx?nid=61

The authors of the ‘Review’ claim that their analysis is limited to just their two trials with a total of 140 patients because these are the only studies which were classifiable as randomized clinical trials (RCTs). The authors dismiss the meta-analysis of five trials, four of which were non-randomized, performed in 2009 by Professor Chapman in Toronto, Canada, which shows that treatment of 1500 patients confers benefit in alpha-1 antitrypsin deficiency. It has been pointed out by many eminent medical authorities that performing RCTs on small numbers of patients with rare disorders is fraught with statistical limitations and ethical problems. The use of non-randomized studies, such as in Professor Chapman’s meta-analysis, is an entirely valid approach to studying treatments in these patients. Excluding the majority of a patient population in the name of dogma is not conducive to optimal patient care.

It is interesting to note, however, that the two studies which were included by the authors of the ‘Review’ both stated that there is a benefit in AAT augmentation for patients with AAT deficiency. The investigators comment on the difficulty in getting a statistically valid result with the small number of patients at their disposal, but note that emphysema was delayed in patients on AAT, and that exacerbation severity in treated patients was reduced. The investigators also validated the use of computed tomography (CT) densitometric assessment of lung tissue, using this technique to assess lung structure and function. They show that CT analysis is a more sensitive indication of lung status, than the parameters used by the ‘Review’ authors.

In summary, this ‘Review’ represents an arbitrary and dogmatic interpretation of clinical findings in AAT augmentation therapy, much of which is challengeable by the limited studies cited and by the much wider experience in the treater community.

The Alpha-1 Foundation:

Cochrane study poorly designed, ignores wealth of data, does disservice to rare disease patients, says Alpha-1 Foundation

http://www.alpha-1foundation.org/news/cochrane-study-poorly-designed-ignores-wealth-of-data-does-disservice-to-rare-disease-patients-says-alpha-1-foundation

“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.”