Baylor College of Medicine, Houston, Texas, USA.
Pediatr Pulmonol. 2010 Nov;45(11):1121-34. doi: 10.1002/ppul.21301.
Chronic airway infection with Pseudomonas aeruginosa (PA) causes morbidity and mortality in patients with cystic fibrosis (CF). Additional anti-PA therapies are needed to improve health status and health-related quality of life. AIR-CF3 was an international 18-month, open-label study to evaluate the safety and efficacy of repeated courses of aztreonam for inhalation solution (AZLI, now marketed as Cayston®) in patients aged ≥ 6 years with CF and PA infection who previously participated in one of two Phase 3 studies: AIR-CF1 or AIR-CF2. Patients received up to nine courses (28 days on/28 days off) of 75 mg AZLI two (BID) or three times daily (TID) based on randomization in the previous trials. 274 patients, mean age 28.5 years (range: 8-74 years), participated. Mean treatment adherence was high (92.0% BID group, 88.0% TID group). Hospitalization rates were low and adverse events were consistent with CF. With each course of AZLI, FEV(1) and scores on the Cystic Fibrosis Questionnaire-Revised Respiratory Symptom scale improved and bacterial density in sputum was reduced. Benefits waned in the 28 days off therapy, but weight gain was sustained over the 18 months. There were no sustained decreases in PA susceptibility. A dose response was observed; AZLI TID-treated patients demonstrated greater improvements in lung function and respiratory symptoms over 18 months. Repeated intermittent 28-day courses of AZLI treatment were well tolerated. Clinical benefits in pulmonary function, health-related quality of life, and weight were observed with each course of therapy. AZLI is a safe and effective new therapy in patients with CF and PA airway infection.
慢性气道铜绿假单胞菌(PA)感染导致囊性纤维化(CF)患者的发病率和死亡率。需要额外的抗 PA 治疗方法来改善健康状况和健康相关的生活质量。AIR-CF3 是一项国际性的 18 个月、开放性标签研究,旨在评估重复使用吸入用氨曲南溶液(AZLI,现已上市的 Cayston®)治疗方案在既往参加过两项 3 期研究(AIR-CF1 或 AIR-CF2)的年龄≥6 岁 CF 合并 PA 感染患者中的安全性和疗效。患者根据既往试验中的随机分组接受最多 9 个疗程(28 天用药/28 天停药)的 75mg AZLI,每日两次(BID)或每日三次(TID)。共有 274 名患者,平均年龄 28.5 岁(范围:8-74 岁)参与了该研究。治疗依从性高(BID 组 92.0%,TID 组 88.0%)。住院率较低,不良事件与 CF 相符。接受每一个 AZLI 疗程治疗后,FEV1 和囊性纤维化问卷-修订呼吸症状量表评分均有所改善,痰液中的细菌密度降低。在停药的 28 天内疗效逐渐减弱,但体重在 18 个月内持续增加。PA 敏感性没有持续降低。观察到剂量反应,TID 组患者在 18 个月内肺功能和呼吸道症状改善更明显。重复间歇性 28 天 AZLI 治疗方案耐受性良好。在每个疗程的治疗中均观察到肺功能、健康相关生活质量和体重方面的临床获益。AZLI 是 CF 合并 PA 气道感染患者的一种安全有效的新型治疗方法。