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

Quittner, AL, Modi, AC, Wainwright, C, Otto, K, Kirihara, J and Montgomery, AB (2009) 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. Chest, 135 6: 1610-1618. doi:10.1378/chest.08-1190


Author Quittner, AL
Modi, AC
Wainwright, C
Otto, K
Kirihara, J
Montgomery, AB
Title 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
Formatted title
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
Journal name Chest   Check publisher's open access policy
ISSN 0012-3692
1931-3543
1970-4917
1578-0023
Publication date 2009-06-01
Year available 2009
Sub-type Article (original research)
DOI 10.1378/chest.08-1190
Volume 135
Issue 6
Start page 1610
End page 1618
Total pages 9
Editor Richard S Irwin
Place of publication United States
Publisher American College of Chest Physicians
Collection year 2010
Language eng
Subject C1
920115 Respiratory System and Diseases (incl. Asthma)
110203 Respiratory Diseases
Abstract 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 of these three estimates defined the MCIDs. Results MCID scores were larger for patients in study 1-exacerbation (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 CF treatments that improve both symptoms and physiologic variables, potentially leading to better treatment adherence and clinical outcomes.
Keyword quality of life
Cystic Fibrosis
Chronic Pseudomonas Aeruginosa
Airway Infection
respiratory
Q-Index Code C1
Q-Index Status Confirmed Code

Document type: Journal Article
Sub-type: Article (original research)
Collections: 2010 Higher Education Research Data Collection
School of Medicine Publications
 
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Created: Thu, 03 Sep 2009, 18:00:38 EST by Mr Andrew Martlew on behalf of Paediatrics & Child Health - RBWH