Effect of ivacaftor treatment in patients with cystic fibrosis and the G551D-CFTR mutation: patient-reported outcomes in the STRIVE randomized, controlled trial.
Effect of ivacaftor treatment in patients with cystic fibrosis and the G551D-CFTR mutation: patient-reported outcomes in the STRIVE randomized, controlled trial.
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DOI：
10.1186/s12955-015-0293-6
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发表时间：
2015-07-02
影响因子：
3.6
通讯作者：
Vera-Llonch M
中科院分区：
文献类型：
--
作者：
Quittner A;Suthoff E;Rendas-Baum R;Bayliss MS;Sermet-Gaudelus I;Castiglione B;Vera-Llonch M
囊性纤维化（CF）是一种罕见的常染色体隐性遗传病，可导致慢性衰弱性疾病和高过早死亡率。通过囊性纤维化问卷修订（CFQ-R）（一种广泛使用的患者报告结果（PRO）测量方法），我们评估了ivvacaftor治疗对CF症状、功能和健康的影响。STRIVE是一项双盲、安慰剂对照的随机试验，评估了ivacaftor （150 mg）对12岁以上G551D-CFTR突变CF患者48周的疗效。治疗效果分析采用混合效应重复测量模型。治疗效益分析应用累积分布函数和变化分数的分类分析（“改善”、“无变化”或“下降”）。基于内容的解释检验了治疗对特定项目反应的影响。分析了152例基线CFQ-R评估患者的数据。治疗效果分析表明，在身体形象、饮食、健康感知、身体功能、呼吸、社会功能、治疗负担和活力量表上，ivacaftor治疗优于安慰剂。研究结果得到了分类变化分析的支持。在所有CFQ-R量表中，ivacaftor改善的患者百分比更高。在基于内容的分析中，治疗益处的特点是在广泛的领域中得分更高。结果表明，在许多领域，如CFQ-R测量的呼吸道症状、身体和社会功能、健康观念和活力，ivacaftor治疗都有广泛的益处。与其他疗法相比，改善的广度反映了ivacaftor的系统作用机制。研究结果支持患者报告的在该患者群体中使用干扰素治疗的价值。本文的在线版本（doi:10.1186/s12955-015-0293-6）包含补充材料，授权用户可获得。
Cystic fibrosis (CF) is an inherited, rare autosomal recessive disease that results in chronically debilitating morbidities and high premature mortality. We evaluated how ivacaftor treatment affected CF symptoms, functioning, and well-being, as measured by the Cystic Fibrosis Questionnaire-Revised (CFQ-R), a widely-used patient-reported outcome (PRO) measure. STRIVE, a double-blind, placebo-controlled randomized trial, evaluated ivacaftor (150 mg) in CF patients aged 12+ with the G551D-CFTR mutation for 48 weeks. Treatment effect analysis used a mixed-effects repeated measures model. Treatment benefit analyses applied the cumulative distribution function and a categorical analysis of change scores (“improvement,” “no change,” or “decline”). Content-based interpretation examined treatment effect on specific item responses. Data from 152 patients with a baseline CFQ-R assessment were analyzed. The treatment effect analysis favored treatment with ivacaftor over placebo on the Body Image, Eating, Health Perceptions, Physical Functioning, Respiratory, Social Functioning, Treatment Burden, and Vitality scales. Findings were supported by the analysis of categorical change. On all CFQ-R scales, the percentage of patients who improved was greater for ivacaftor. In the content-based analysis, the treatment benefit was characterized by better scores across a broad range of domains. Results illustrate broad benefits of ivacaftor treatment across many domains: respiratory symptoms, physical and social functioning, health perceptions, and vitality, as measured by the CFQ-R. The breadth of improvements reflects the systemic mechanism of action of ivacaftor compared to other therapies. Findings support the patient-reported value of ivacaftor treatment in this patient population. ClinicalTrials.gov NCT00909532 The online version of this article (doi:10.1186/s12955-015-0293-6) contains supplementary material, which is available to authorized users.