A two-year randomized, placebo-controlled trial of dornase alfa in young patients with cystic fibrosis with mild lung function abnormalities
A two-year randomized, placebo-controlled trial of dornase alfa in young patients with cystic fibrosis with mild lung function abnormalities
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DOI：
10.1067/mpd.2001.118570
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发表时间：
2001-12-01
影响因子：
5.1
通讯作者：
Konstan, MW
中科院分区：
文献类型：
--
作者：
Quan, JM;Tiddens, HAWM;Konstan, MW
目的：我们的目的是确定长期使用dornase alfa治疗囊性纤维化（CF）的年轻患者是否能维持肺功能并减少呼吸道恶化。研究设计：这是一项为期96周的随机、双盲、安慰剂对照试验，涉及49个CF中心。纳入标准为6 ~ 10岁，强迫肺活量大于或等于预测的85%。排除2个月内因CF并发症住院和6个月内使用dornase alfa的患者。患者每天服用一次dornase α 2.5 mg或安慰剂，并使用喷射雾化器和压缩机。结果：患者是随机的，239人服用dornase alfa， 235人服用安慰剂。基线时平均年龄为8.4岁，1秒内平均用力呼气量预测95%，平均用力呼气流量、呼气中期预测85%，平均用力肺活量预测102%。在96周时，与安慰剂相比，dornase alfa治疗的预期收益百分比（平均SE）为：1秒用力呼气量为3.2 +/- 1.2 (P = 0.006)，用力呼气流量在25%至75%之间为7.9 +/- 2.3 (P = 0.0008)，用力肺活量为0.7 +/- 1.0 （P = 0.51）。在接受dornase alfa治疗的患者中，呼吸道恶化的风险降低了34%（相对风险0.66,P = 0.048）。两组间年龄体重百分位数变化无统计学差异。治疗组的不良事件概况相似。结论：在96周的时间内，使用dornase alfa治疗年轻CF患者可维持肺功能并降低病情恶化的风险。
Objective: Our objective was to determine whether long-term treatment of young patients with cystic fibrosis (CF) with dornase alfa maintains lung function and reduces respiratory tract exacerbations.Study design: This was a 96-week, randomized, double-blind, placebo-controlled trial involving 49 CF centers. Inclusion criteria were age 6 to 10 years and forced vital capacity greater than or equal to 85% predicted. Patients were excluded for hospitalization for complications of CF within 2 months and use of dornase alfa within 6 months. Patients were treated with dornase alfa 2.5 mg or placebo once daily with a jet nebulizer and a compressor.Results: Patients were randomized, 239 to dornase alfa and 235 to placebo. At baseline the mean age was 8.4 years, the mean forced expiratory volume in I second 95% predicted, the mean forced expiratory flow, midexpiratory phase 85% predicted, and the mean forced vital capacity 102% predicted. At 96 weeks the treatment benefit for dornase alfa compared with placebo in percent predicted (mean SE) was 3.2 +/- 1.2 for forced expiratory volume in I second (P = .006), 7.9 +/- 2.3 for forced expiratory flow between 25% and 75% of vital capacity (P = .0008), and 0.7 +/- 1.0 for forced vital capacity (P = .51). The risk of respiratory tract exacerbation was reduced by 34% in patients who received dornase alfa (relative risk 0.66, P = .048). There was no statistically significant difference between the groups in changes in weight-for-age percentile. Adverse event profiles for the treatment groups were similar.Conclusions: Treatment of young patients with CF,,vith dornase alfa maintains lung function and reduces the risk of exacerbations over a 96-week period.