Alpha-1 Research Program, Division of Pulmonary, Critical Care and Sleep Medicine, Department of Internal Medicine, University of Florida, Gainesville, Florida, USA.
Int J Chron Obstruct Pulmon Dis. 2009;4:443-52. doi: 10.2147/copd.s8577. Epub 2009 Dec 29.
Intravenous augmentation therapy with purified intravenous alpha-1 antitrypsin replaces the deficient protein and is the only currently approved treatment for alpha-1 antitrypsin deficiency (AATD) related lung disease. While augmentation therapy has been available for more than 20 years, there are a limited number of studies evaluating the effect of augmentation on lung function.
We examined the decline in forced expiratory volume in one second (FEV(1)) in patients enrolled in the Alpha-1 Foundation DNA and Tissue Bank in relation to the use or not of alpha-1 antitrypsin augmentation therapy. For the purpose of our analysis we included 164 patients with AATD and PI ZZ genotype.
Mean age of the patients was 60 years, 52% were females, 94% were white and 78% ex-smokers. The mean FEV(1) at baseline was 1.7 L and the mean FEV(1) % of predicted was 51.3%. The mean follow-up time was 41.7 months. A total of 124 (76%) patients received augmentation therapy (augmented group) while 40 patients (24%) did not received it (non-augmented group). When adjusted by age at baseline, sex, smoking status, baseline FEV(1) % of predicted, the mean overall change in FEV(1) was 47.6 mL/year, favoring the augmented group (DeltaFEV(1) 10.6 +/- 21.4 mL/year) in comparison with the non-augmented group (DeltaFEV(1) -36.96 +/- 12.1 mL/year) (P = 0.05). Beneficial DeltaFEV(1) were observed in ex-smokers and the group with initial FEV(1) % of predicted of <50%. No differences were observed in mortality.
In conclusion, augmentation therapy improves lung function in subjects with AATD when adjusted by age, gender, smoking status and baseline FEV(1) % of predicted. The beneficial effects were noted in ex-smoker subjects with FEV(1) below 50% of predicted.
静脉内补充纯化的静脉内α-1 抗胰蛋白酶可替代缺乏的蛋白质，是目前唯一批准用于治疗α-1 抗胰蛋白酶缺乏症（AATD）相关肺病的方法。尽管静脉内补充治疗已经有 20 多年的历史，但评估补充治疗对肺功能影响的研究数量有限。
我们检查了参加 Alpha-1 基金会 DNA 和组织库的患者中用力呼气量（FEV1）的下降与是否使用α-1 抗胰蛋白酶补充治疗的关系。为了进行我们的分析，我们纳入了 164 名具有 AATD 和 PI ZZ 基因型的患者。
患者的平均年龄为 60 岁，52%为女性，94%为白人，78%为曾经吸烟者。基线时的平均 FEV1 为 1.7 L，预测的 FEV1%为 51.3%。平均随访时间为 41.7 个月。共有 124 名（76%）患者接受了补充治疗（补充治疗组），40 名（24%）未接受补充治疗（未补充治疗组）。通过基线时的年龄、性别、吸烟状况、基线时的 FEV1%预测值进行调整后，FEV1 的总体平均变化为 47.6 mL/年，补充治疗组（DeltaFEV1 为 10.6 +/- 21.4 mL/年）较未补充治疗组（DeltaFEV1 为-36.96 +/- 12.1 mL/年）更有利（P = 0.05）。在曾经吸烟者和初始 FEV1%预测值<50%的患者中观察到有益的 DeltaFEV1。两组间死亡率无差异。
总之，调整年龄、性别、吸烟状况和基线 FEV1%预测值后，补充治疗可改善 AATD 患者的肺功能。在初始 FEV1%预测值低于 50%的曾经吸烟者中观察到有益的效果。
