Louie Stan Gee, Sclar David Alexander, Gill Mark Anthony
Department of Pharmacy, University of Southern California, Los Angeles, CA 90089-9121, USA.
Ann Pharmacother. 2005 Nov;39(11):1861-9. doi: 10.1345/aph.1E061. Epub 2005 Oct 11.
To review the epidemiology, pathogenesis, and management of patients with alpha-antitrypsin (AAT) deficiency syndrome and compare Aralast with Prolastin, 2 of the 3 available human plasma-derived AAT agents.
Articles were identified using a MEDLINE (1966-September 2005) search with MESH headings that included alpha-antitrypsin and emphysema.
All papers from peer-reviewed journals on the laboratory or clinical efficacy of plasma-derived AAT (eg, Prolastin, Aralast) for patients with this autosomal recessive disorder were reviewed.
Clinical trials found that AAT augmentation prevents progression of AAT-deficient emphysema and thus its associated morbidity and mortality. Treatment with Aralast has been shown to be safe and well tolerated, with a low incidence of mild to moderate adverse events. Pharmacoeconomics studies of AAT augmentation demonstrated that the use of Aralast was cost-effective as lifelong augmentation therapy for AAT-deficient emphysema.
Because of its effectiveness and extra safety measure compared with Prolastin, Aralast should be recommended for formulary inclusion.
回顾α1-抗胰蛋白酶(AAT)缺乏综合征患者的流行病学、发病机制及治疗,并比较Aralast与Prolastin这两种现有的人类血浆源性AAT制剂中的两种。
使用MEDLINE(1966年至2005年9月)检索,检索词为包含α1-抗胰蛋白酶和肺气肿的医学主题词,以此来识别文章。
对所有来自同行评审期刊的关于血浆源性AAT(如Prolastin、Aralast)对这种常染色体隐性疾病患者的实验室或临床疗效的论文进行了回顾。
临床试验发现,AAT补充疗法可防止AAT缺乏性肺气肿的进展,从而预防其相关的发病率和死亡率。已证明使用Aralast是安全且耐受性良好的,轻度至中度不良事件的发生率较低。AAT补充疗法的药物经济学研究表明,使用Aralast作为AAT缺乏性肺气肿的终身补充疗法具有成本效益。
由于与Prolastin相比,Aralast具有有效性和额外的安全措施,因此应推荐将其纳入处方药物目录。