Health-related quality of life and estimation of the minimally important difference in the Functional Assessment of Cancer Therapy-Endocrine Symptom (FACT-ES) score in postmenopausal ER-positive, HER2- negative metastatic breast cancer with low sensitivity to endocrine therapy

Background The HORSE-BC study previously demonstrated that second-line endocrine therapy (ET) for patients with acquired endocrine-resistant metastatic breast cancer (MBC) still provided a clinically meaningful benefit. Herein, we investigated the health-related quality of life (HR-QOL) in the HORSE-BC study. Methods Patients with acquired endocrine-resistant MBC who were scheduled for second-line ET were recruited. The HR-QOL was assessed at baseline, and 1 and 3 months after second-line ET initiation. To investigate the minimally important difference (MID) in the Functional Assessment of Cancer Therapy-Endocrine Symptoms (FACT-ES), we evaluated the means and standard deviations for the distribution-based method, and differences in the change in HR-QOL for the anchor-based method. We also investigated the association between FACT-ES total scores and clinical benefit. Results Overall, 56 patients were enrolled. Of these, 47 were analyzed. When defined as 1/3 standard deviation estimates based on the distribution method, the calculated MID was 5.9. The MIDs of the FACT-ES total scores based on the anchor method were 7.7 for decline and 4.1 for improvement. The MID decline proportions were 6.1% and 14.7% lower in patients who experienced clinical benefits than in those who did not at 1 and 3 months, respectively. The ratios of MID improvement in patients who experienced clinical benefits were 18.3% and 3.2% higher, respectively; the mean change in the FACT-ES total score from baseline improved in patients who experienced clinical benefits. Conclusions Maintaining the HR-QOL as determined by FACT-ES may be associated with clinical benefits in patients with acquired endocrine-resistant MBC treated with ET.

Health-related quality of life and estimation of minimally important difference in the Functional Assessment of Cancer Therapy-Endocrine Symptom (FACT-ES) score in postmenopausal ER-positive, HER2-negative metastatic breast cancer with low sensitivity to endocrine therapy

Purpose The HORSE-BC study previously demonstrated that 2nd-line endocrine therapy (ET) for metastatic breast cancer (MBC) patients with acquired endocrine resistance still provided a clinically meaningful benefit. Herein, we investigated health-related quality of life (HR-QOL) in HORSE-BC. Methods MBC patients with acquired endocrine resistance who were scheduled for 2nd-line ET were recruited. HR-QOL was assessed at baseline and 1 and 3 months after initiation of 2nd-line ET. To investigate the minimally important difference (MID) in the Functional Assessment of Cancer Therapy-Endocrine Symptoms (FACT-ES), we evaluated the means and standard deviations (SDs) for the distribution-based method, and differences in the change in HR-QOL for the anchor-based method. We also investigated the association between FACT-ES total scores and clinical benefit. Results Overall, 56 patients were enrolled. Of these, 47 were analyzed. When defined as 1/3 SD estimates based on the distribution method, the calculated MID was 5.9. The MIDs of the FACT-ES total scores based on the anchor method were 7.7 for decline and 4.1 for improvement. The proportions of MID decline were 6.1% and 14.7% lower in patients who experienced clinical benefits than in those who did not at 1 month and 3 months, respectively. The respective ratios of MID improvement in patients who experienced clinical benefits were 18.3% and 3.2% higher, respectively; mean change in FACT-ES total score from baseline was improved in patients who experienced clinical benefits. Conclusion Maintaining HR-QOL as determined by FACT-ES may be associated with clinical benefits in patients with acquired endocrine-resistant MBC treated with ET.

Interpretation of change scores for the National Eye Institute Visual Function Questionnaire-25: the minimally important difference

AimTo estimate the minimally important difference (MID) in change in National Eye Institute Visual Function Questionnaire-25 (VFQ-25) composite score using methods aligned with patient perception.MethodsRetrospective analysis of prospectively collected data from adults with primary angle closure or primary angle closure glaucoma enrolled in the Effectiveness, in Angle-closure Glaucoma, of Lens Extraction study. We included data from 335 participants with patient reported visual function (VFQ-25) and health status measured by the EQ-5D-3L over 36 months. We used the recommended anchor-based methods (receiver operating characteristic (ROC), predictive modelling and mean change) to determine the MID of the VFQ-25. EQ-5D-3L anchor change was defined as none (<0.065); minimal (0.065≤EQ-5D-3L change ≤0.075 points) and greater change (>0.075 points).ResultsMean baseline VFQ-25 score was 87.6 (SD 11.8). Estimated MIDs in the change in VFQ-25 scores (95% CI) were 10.5 (1.9 to 19.2); 3.9 (−2.3 to 10.1); 5.8 (1.9 to 7.2) and 8.1 (1.7 to 14.8) for the ‘within-patient’, ‘between-patient’ change, ROC and predictive modelling anchor methods respectively. Excluding estimates from the methodologically weaker ‘within-patient’ method, the MID of a change in VFQ-25 composite score is 5.8 (median value).ConclusionsEstimates of the MID using multiple methods assist in the interpretation of the VFQ scores. In the context of early glaucoma related visual disability, a change score of around six points on the VFQ-25 is likely to be important to patients. Further confirmatory research is required. Studies comparing changes in patient-reported outcome measure scores with a global measure of patients’ perceived change are required.

Determination of thresholds for minimally important difference and clinically important response on the functional outcomes of sleep questionnaire short version in adults with narcolepsy or obstructive sleep apnea

Purpose This study estimated thresholds for clinically important responses and minimally important differences for two indicators of improvement for the 10-item version of the functional outcomes of sleep questionnaire (FOSQ-10). Methods Participants with excessive daytime sleepiness with narcolepsy or obstructive sleep apnea received 12 weeks of solriamfetol treatment. Participants completed the FOSQ-10 and other patient-reported outcome measures, including the single-item patient global impression of change (PGI-C) assessment. Clinicians completed the single-item clinician global impression of change (CGI-C) for each participant. Data from the two studies were analyzed separately, both without regard to treatment assignment. In total, 690 participants (47% female, mean age 48 years, 77% Caucasian, 91% from North America) were enrolled. Two clinically important changes, defined as a minimally important difference and a clinically important response, were determined using distribution and anchor-based analyses. A receiver operating characteristic analysis was used to determine the optimal FOSQ-10 change threshold. Results Spearman correlations between change in FOSQ-10 scores and PGI-C and CGI-C were − 0.57 and − 0.49 for participants with narcolepsy and − 0.42 and − 0.37 for participants with obstructive sleep apnea. Receiver operating characteristic analysis suggested minimally important difference and clinically important response estimates of 1.7 and 2.5 and 1.8 and 2.2 points in narcolepsy and obstructive sleep apnea, respectively. Conclusions Minimally important difference and clinically important response estimates for the FOSQ-10 for adults with excessive daytime sleepiness in narcolepsy or obstructive sleep apnea will be helpful for interpreting changes over time and defining a clinical responder. ClinicalTrials.gov identifiers NCT02348593 (first submitted January 15, 2015) and NCT02348606 (first submitted January 15, 2015)

Estimation of minimally important difference of the EQ-5D-5L utility scores among patients with either hypertension or diabetes or both: a cross-sectional study in Hong Kong

ObjectiveTo estimate and report the minimally important difference (MID) of the EuroQol five-dimensional five-level questionnaire (EQ-5D-5L) using the Hong Kong (HK) utility score among patients with either hypertension or diabetes or both.DesignData were analysed using secondary data analysis based on a cross-sectional study assessing patients’ experiences in HK.SettingA representative sample was recruited from 26 specialist outpatient clinics in HK.ParticipantsWe analysed data from 2231 and 662 patients who reported having hypertension or diabetes alone, respectively, and 874 patients had these two diseases.InterventionAn instrument-defined approach was applied to estimate MID stratified according to sex and age for the three subpopulations.ResultsThe overall MID (oMID) estimates were 0.089, 0.086 and 0.089 for patients with hypertension or diabetes alone and with these two diseases, respectively. The adjusted MID (aMID) estimate was smaller than the oMID, and the improved MID was larger than the deteriorated MID. Women had larger oMID but smaller aMID than men. Younger respondents had larger aMID than older respondents. Effect sizes ranged from 0.30 to 0.503, which fit our preset criteria.ConclusionFour types of MID of the EQ-5D utility score for patients with hypertension or diabetes alone and with these two diseases were reported. Variations in the MID estimates should be further explored in other populations or using different statistical methods.

Frailty state utility and minimally important difference: findings from the North West Adelaide Health Study

Background frailty is a dynamic condition for which a range of interventions are available. Health state utilities are values that represent the strength of an individual’s preference for specific health states, and are used in economic evaluation. This is a topic yet to be examined in detail for frailty. Likewise, little has been reported on minimally important difference (MID), the extent of change in frailty status that individuals consider to be important. Objectives to examine the relationship between frailty status, for both the frailty phenotype (FP) and frailty index (FI), and utility (preference-based health state), and to determine a MID for both frailty measures. Design and setting population-based cohort of community-dwelling Australians. Participant in total, 874 adults aged ≥65 years (54% female), mean age 74.4 (6.2) years. Measurements frailty was measured using the FP and FI. Utilities were calculated using the short-form 6D health survey, with Australian and UK weighting applied. MID was calculated cross-sectionally. Results for both the FP and FI, frailty was significantly statistically associated (P &lt; 0.001) with lower utility in an adjusted analysis using both Australian and UK weighting. Between-person MID for the FP was identified as 0.59 [standard deviation (SD) 0.31] (anchor-based) and 0.59 (distribution-based), whereas for the FI, MID was 0.11 (SD 0.05) (anchor-based) and 0.07 (distribution-based). Conclusions frailty is significantly associated with lower preference-based health state utility. Frailty MID can be used to inform design of clinical trials and economic evaluations, as well as providing useful clinical information on frailty differences that patients consider important.

An Editorial on Tailoring Patient-Reported Outcomes to the Individual Patient: Precision PROs

Patient-reported outcomes (PROs) have the potential to improve clinical care. One barrier to implementing PROs and maximizing their effectiveness is difficulty individualizing PROs. To address this barrier, we propose a precision PRO methodology. By this approach, patients would first define a minimally important difference for themselves. Patients would then choose which items within a PRO item bank are most important to them; these items would be used to monitor patients’ progress. Patients could be compared to each other – even if they chose different questions – using modern psychometric methods. We propose future research to guide how to determine the simplest and most effective precision PRO methodology.