AUGMENTATION THERAPY--CONGENITAL ALPHA1 PROTEINASE INHIB

这项多中心的D类研究已经完成。这项研究旨在评估一种名为ATC 97-01的新药，该药旨在增强遗传性缺乏这一重要血浆酶的患者的α-1抗胰蛋白酶活性。新药与现有的制剂(Prolastin)进行了比较。患者被随机分配到新药或对照药物组，为期10周，之后所有受试者都接受新药治疗，总研究持续时间为两年。在过去的十年里，对遗传性α-1抗胰蛋白酶缺陷患者的治疗有了显著的进步。已经证明，巴氏杀菌的混合人类产品可以将血浆中的α-1抗胰蛋白酶水平提高到生物意义上的水平。除了这一生化证据外，两项大型研究表明，这种疗法具有临床疗效。欧洲的一项研究表明，强化治疗影响了肺功能的重要参数，最近在美国进行的一项研究表明，由于遗传性α1抗胰蛋白酶缺陷而接受强化治疗的患者具有生存优势。由于巴氏杀菌的人源酶的供应不足以满足患者的需求，一种新的产品被开发出来，这项研究正在测试该产品。这种新产品也是由混合的人血浆制成的，使用溶剂洗涤剂处理方法制备，大大增强了现有制剂的病毒安全性。

This multi-center, category D study has been completed. The study was designed to evaluate a new drug, ATC 97-01, designed to augment alpha-1 antitrypsin activity in patients with an inherited deficiency in this important plasma enzyme. The new drug was compared with an existing formulation (Prolastin). Patients were randomly allocated to the new drug or the comparator drug for ten weeks, after which all subjects received the new drug for a total study duration of two years. Over the last ten years, there have been significant improvements in the therapy of patients with inherited deficiencies of alpha-1 antitrypsin. It has been demonstrated that pasteurized, pooled human products can increase plasma levels of alpha-1 antitrypsin to biologically significant levels. In addition to this biochemical evidence, two large studies have suggested that the therapy has clinical efficacy. A European study demonstrated that augmentation therapy affected important parameters of lung function and, more recently, a study done in the United States demonstrated a survival advantage for patients receiving augmentation therapy for their inherited deficiency of alpha-1 antitrypsin. Because the supply of pasteurized, pool human enzyme has not been sufficient to meet patient demands, a new product was developed, and this study is testing that product. The new product, also made from pooled human plasma, is prepared using a solvent detergent treatment method, which greatly enhances viral safety over existing preparations.