No Relief by Alpha-1 Antitrypsin: Study Says
Review by Cochrane Researchers on the new treatment for alpha-1 antitrypsin deficiency, genetic disorder has revealed that suggested treatment has proved to be no good. The disorder is the cause of lung disease.
One out of 1,600 people suffer from deficiency Alpha-1 antitrypsin. Inherited disorder causes low level of the protein alpha-1 antitrypsin, also called alpha-1 proteinase inhibitor, shields lung tissues from white blood cells.
The data was collected from the survey conducted on 140 people suffering from disorder, who were at the high risk of developing chronic lung disease. During trial patients were administered with intravenous alpha-1 antitrypsin, every four weeks for three years and another trial was conducted for 2 years. Results revealed no difference in the condition of the two set of patients. The review authors concluded that treatment costing $150,000 is of no use.
Lead researcher Peter Gotzsche of the Nordic Cochrane Center at Rigshospitalet in Copenhagen, Denmark said, "The drug has not shown any clinical benefit, is extremely costly and has important adverse effects."
Robert A. Sandhaus, MD, PhD, Foundation Clinical Director said, "While large randomized, controlled, blinded studies remain the gold standard in the evaluation of therapeutics, performing such studies in a population of patients with a rare genetic condition presents almost insurmountable obstacles."
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