Schwaiblmair M, Vogelmeier C
Department of Internal Medicine, Klinikum Grosshadern, University of Munich, Germany.
Drugs Aging. 1998 Jun;12(6):429-40. doi: 10.2165/00002512-199812060-00002.
Alpha 1-Antitrypsin (alpha 1AT) deficiency is the most common genetic cause of liver disease in children and emphysema in adults. Therapy for pulmonary disease attributable to alpha 1AT deficiency includes alpha 1AT augmentation therapy along with supportive measures. The alpha 1AT preparation that is currently used for therapy is derived from fractionated plasma. The results of clinical trials suggest that augmentation therapy with alpha 1AT slows the progression of emphysema and causes few adverse events. Patients with plasma levels of alpha 1AT that are < 11 mumol/L and who have airway obstruction should be considered for augmentation therapy. Novel approaches include the administration of aerosolised alpha 1AT, recombinant alpha 1AT, gene therapy and synthetic elastase inhibitors.
α1抗胰蛋白酶（α1AT）缺乏症是儿童肝病和成人肺气肿最常见的遗传病因。针对α1AT缺乏所致肺部疾病的治疗包括α1AT补充疗法及支持性措施。目前用于治疗的α1AT制剂源自分馏血浆。临床试验结果表明，α1AT补充疗法可减缓肺气肿进展，且不良事件较少。α1AT血浆水平<11μmol/L且有气道阻塞的患者应考虑接受补充疗法。新方法包括雾化α1AT给药、重组α1AT、基因治疗和合成弹性蛋白酶抑制剂。
