Determination of the Minimal Clinically Important Difference Scores for the Cystic Fibrosis Questionnaire-Revised Respiratory Symptom Scale in Two Populations of Patients With Cystic Fibrosis and Chronic Pseudomonas aeruginosa Airway Infection
Determination of the Minimal Clinically Important Difference Scores for the Cystic Fibrosis Questionnaire-Revised Respiratory Symptom Scale in Two Populations of Patients With Cystic Fibrosis and Chronic Pseudomonas aeruginosa Airway Infection
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DOI：
10.1378/chest.08-1190
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发表时间：
2009-06-01
期刊：
影响因子：
9.6
通讯作者：
Montgomery, A. Bruce
中科院分区：
文献类型：
--
作者：
Quittner, Alexandra L.;Modi, Avani C.;Montgomery, A. Bruce
背景：囊性纤维化问卷修订（CFQ-R）是一个经过验证的患者报告结果（PRO），包含囊性纤维化（CF）的通用量表和特异性量表。PRO的最小临床重要差异（MCID）评分对应于患者可以检测到的最小临床相关变化。CFQ-R呼吸症状（CFQ-R- respiratory）量表的MCID评分采用CF和慢性铜绿假单胞菌气道感染患者的两项28天开放标签tobramycin吸入溶液（TIS）研究的数据。研究入组时，研究1加重组患者有肺加重症状（n = 84; < 14岁，31例；>= 14岁，53例）；研究2-stable患者呼吸道症状稳定（n = 140; < 14岁，14例；>= 14岁，126例）。方法：基于锚定的方法采用全球变化评级问卷（GRCQ），评估患者对TIS治疗后呼吸道症状变化的看法。从基线CFQ-R-Respiratory评分的平均变化被映射到GRCQ来估计MCID。两种基于分布的方法分别为：(1)cfq - r呼吸评分（基线至TIS治疗结束）平均变化0.5 SD；(2)基线CFQ-R-Respiratory评分1 SEM。三角测量或这三个估计值定义了mcd。结果：研究1-恶化组患者的MCID评分（8.5分）高于研究2-稳定组患者（4.0分），可能反映了这些研究之间患者疾病状态（恶化/稳定）的差异。结论：可以通过CFQ-R-Respiratory评分的变化来评估患者从新的和当前的CF治疗中获益。使用MCID提供了一种系统的方法来解释这些变化，并有助于识别改善症状和生理变量的CIF治疗，从而可能导致更好的治疗依从性和临床结果。试验注册（研究1-恶化）：澳大利亚-新西兰临床试验注册标识：ACTRN 12605000602628试验注册（研究2-稳定）：ClinicalTrials.gov标识：NCT00104520 （CHEST 2009; 135:1610-1618）
Background: The Cystic Fibrosis Questionnaire-Revised (CFQ-R) is a validated patient-reported outcome (PRO) containing both generic scales and scales specific to cystic fibrosis (CF). The minimal clinically important difference (MCID) score for a PRO corresponds to the smallest clinically relevant change a patient can detect. MCID scores for the CFQ-R respiratory symptom (CFQ-R-Respiratory) scale were determined using data from two 28 day, open-label, tobramycin inhalation solution (TIS) studies in patients with CF and chronic Pseudomonas aeruginosa airway infection. At study enrollment, patients in the study 1-exacerbation had symptoms indicative of pulmonary exacerbation (n = 84; < 14 years of age, 31 patients; >= 14 years of age, 53 patients); patients in study 2-stable had stable respiratory symptoms (n = 140; < 14 years of age, 14 patients; >= 14 years, 126 patients).Methods: The anchor-based method utilized a global rating-of-change questionnaire (GRCQ) that assessed patients' perceptions of change in their respiratory symptoms after TIS treatment. The mean change from baseline CFQ-R-Respiratory scores were mapped onto the GRCQ to estimate the MCID. The two distribution-based methods were as follows: (1) 0.5 SD of mean change in CFQ-R-Respiratory Scores (baseline to end of TIS treatment); and (2) 1 SEM for baseline CFQ-R-Respiratory scores. Triangulation or these three estimates defined the MCIDs.Results: MCID scores were larger for patients in study 1-exacerhation (8.5 points) than for those in study 2-stable (4.0 points), likely reflecting differences in patient disease status (exacerbation/stable) between these studies.Conclusions: Patient benefit from new and current CF therapies can be evaluated using changes in CFQ-R-Respiratory scores. Using the MCID provides a systematic way to interpret these changes, and facilitates the identification of CIF treatments that improve both symptoms and physiologic variables, potentially leading to better treatment adherence and clinical outcomes.Trial registration (study 1-exacerbation): Australian-New Zealand Clinical Trials Registry Identifier: ACTRN 12605000602628 Trial registration (study 2-stable):ClinicalTrials.gov Identifier: NCT00104520 (CHEST 2009; 135:1610-1618)