Comparison of the sensitivity of patient-reported outcomes for detecting the benefit of biologics in severe asthma

Abstract

Background The sensitivity of patient-reported outcomes (PROs) to detect the effects of treatment change depends on the match between the change in items of the PRO and the change that takes place in a sample of people. The aim of this study is to compare the sensitivity of different PROs in detecting changes following the initiation of biologic treatment in asthma. Methods: Patients starting a biologic treatment as part of clinical care completed the Asthma Control Questionnaire (ACQ-6), the Severe Asthma Questionnaire (SAQ and SAQ-global scores) and the EQ5D (EQ-5D-5L and EQ5D-VAS) at baseline. They completed the ACQ-6, SAQ, SAQ-global and a retrospective global rating of change (GRoC) scale at weeks 4, 8 and 16 and completed the EQ-5D-5L and EQ5D-VAS at week 16. The SAQ-global and EQ5D-VAS differ but both are single item 100-point questions. Sensitivity was measured by Cohen’s D effect size at each of the three time points. Results: 110 patients were recruited. Depending on the time of assessment, effect size varied between 0.45 and 0.64 for the SAQ, between 0.50 and 0.77 for the SAQ-global; between 0.45 and 0.69 for ACQ-6; between 0.91 and 1.22 for GRoC; 0.32 for EQ-5D-5L and 0.49 for EQ5D-VAS. Conclusion: The sensitivity to change of a questionnaire varies with the time of measurement. The three asthma-specific prospective measures (SAQ, SAQ-global and ACQ-6) have similar sensitivity to change. The single-item EQ5D-VAS was less sensitive than the asthma specific measures and less sensitive than the single-item SAQ-global. The EQ-5D-5L was least sensitive.

Development and validation of an electronic daily control score for asthma (e-DASTHMA): a real-world direct patient data study

“…24,25 In fact, the EQ-5D might not be the best quality-of-life measure for asthma, 24 because it does not react sensitively to small changes in asthma control 26 and its VAS is less sensitive than ACQ-6 for assessing asthma control. 27 e-DASTHMA also showed strong test-retest reliability considering all assessed comparators. The COSMIN guidelines show that coecients of more than 0•7 (observed in all analyses) indicate good reliability.…”

“…11 This study followed the Consensusbased Standards for the selection of health Measurement Instruments (COSMIN) and the Transparent Reporting of a multivariable prediction model for Individual Prognosis Or Diagnosis recommen dations. 12 The protocol of this study is available in the appendix (pp [21][22][23][24][25][26][27][28][29].…”

Control of Allergic Rhinitis and Asthma Test: A systematic review of measurement properties and COSMIN analysis

“…Indeed, when performing sensitivity analysis and quantitatively pooling the results only from studies including patients with asthma, we found that the heterogeneity was greatly decreased. Regarding the lower correlation with the EQ‐5D VAS, it is important to note that CARAT and EQ‐5D measure related but dissimilar constructs, and that EQ‐5D may not be the best quality of life measure to be used in asthma 57 as (i) it does not react very sensitively to small changes in asthma control, 58 (ii) VAS EQ‐5D is less sensitive than ACQ‐6 to assess asthma control, 59 and (iii) it incompletely represents the deficits of quality of life in severe asthma 60 . Therefore, CARAT shows good correlation with asthma PROMs, even in patients without AR.…”

Roles of real-world evidence in severe asthma treatment: challenges and opportunities

“…However, it has not been frequently measured in prospective real-world studies ( table 2 ). Furthermore, although the EuroQoL five-dimensional instrument (EQ-5D) is one of the most widely used tools to measure general health-related QoL, the items are not specific to asthmatic patients’ experiences and may not be sufficiently sensitive to capture clinical changes before versus after biological treatments [ 143 , 144 ]. Therefore, tools that were designed to measure SA patients’ experiences, such as the Severe Asthma Questionnaire, are becoming more popular in real-world studies [ 145 ].…”