The estimation of a preference-based measure of health from the SF-36

This paper reports on the findings of a study to derive a preference-based measure of health from the SF-36 for use in economic evaluation. The SF-36 was revised into a six-dimensional health state classification called the SF-6D. A sample of 249 states defined by the SF-6D have been valued by a representative sample of 611 members of the UK general population, using standard gamble. Models are estimated for predicting health state valuations for all 18,000 states defined by the SF-6D. The econometric modelling had to cope with the hierarchical nature of the data and its skewed distribution. The recommended models have produced significant coefficients for levels of the SF-6D, which are robust across model specification. However, there are concerns with some inconsistent estimates and over prediction of the value of the poorest health states. These problems must be weighed against the rich descriptive ability of the SF-6D, and the potential application of these models to existing and future SF-36 data set

Measures of Health Related quality of life in an imperfect world: a comment on Dowie

Professor Dowie has written an interesting and thought provoking paper on a long lasting debate in the literature on measuring health related quality of life. The debate between generic and condition specific measures (CSMs) has not progressed a great deal with time and he is right to question a purely psychometric approach that currently tends to focuses on effect sizes. He has also presented an interesting challenge to the compromise solution suggested by a number of psychometricians to adopt both types of measure

Interpreting and Reporting Results Based on Patient-Reported Outcomes

AbstractThis article deals with the incorporation of patient-reported outcomes (PROs) into clinical trials and focuses on issues associated with the interpretation and reporting of PRO data. The primary focus and context of this information relates to the evidentiary support and reporting for a labeling or advertising claim of a PRO benefit for a new or approved pharmaceutical product. This manuscript focuses on issues associated with assessing clinical significance and common pitfalls to avoid in presenting results related to PROs. Specifically, the questions addressed by this manuscript involve: What are the best methods to assess clinical significance for PROs? How should investigators present PRO data most effectively in a Food and Drug Administration (FDA) application? In labeling or in a scientific publication? Guidelinesfor interpreting clinical significance of PROs and for comprehensively reporting on the methods, measures and results of clinical trials that incorporate PROs are important for clinicians, regulatory agencies, and most of all to patients. Clear specifications for considering a finding on a PRO measure, as clinically meaningful, need to be determined by instrument developers and psychometricians; they need to be reported for all clinical trials involving PRO end points. Clinical trial reports need to be comprehensive, clear, and sufficient to enable any reader to understand the methods, PRO measures, statistical analysis, and results

Impact of brodalumab treatment on psoriasis symptoms and health-related quality of life: use of a novel patient-reported outcome measure, the Psoriasis Symptom Inventory

Background: Psoriasis symptoms have a significant negative impact on health-related quality of life, impairing physical functioning and well-being. Objective: To evaluate the impact of brodalumab, a human anti-interleukin-17R monoclonal antibody, on psoriasis symptom severity as measured by a novel patient-reported outcome measure, the Psoriasis Symptom Inventory, and dermatology-specific health-related quality of life as measured by the Dermatology Life Quality Index (DLQI). Methods: This was a secondary analysis of a phase II, randomized, double-blind, placebo-controlled clinical study of patients with moderate-to-severe psoriasis (n = 198) treated with brodalumab or placebo. This analysis assessed Psoriasis Symptom Inventory scores and DLQI scores over time. Analyses were conducted on all patients who were randomized and received one or more injections of the study drug according to intention to treat using last observation carried forward to impute missing data. Results: At week 12, subjects in the brodalumab groups had significant improvements in mean Psoriasis Symptom Inventory total scores [8·5 (70 mg), 15·8 (140 mg), 16·2 (210 mg) and 12·7 (280 mg)] compared with placebo (4·8). Mean improvements in DLQI were clinically meaningful (≥ 5·7) in the brodalumab groups (6·2, 9·1, 9·6 and 7·1, respectively) and significantly greater than placebo (3·1). Improvements in Psoriasis Symptom Inventory were observed as early as week 2 and in DLQI by week 4. All eight Psoriasis Symptom Inventory item scores improved significantly among the brodalumab groups by week 12. Conclusions: Results were from a single randomized clinical trial and may not generalize to broader patient populations. However, treatment with brodalumab provided significant improvement in psoriasis symptoms in patients with moderate-to-severe psoriasis

The development of a new measure of quality of life in the management of gastro-oesophageal reflux disease: the Reflux Questionnaire.

INTRODUCTION This paper reports on the development of a new measure of health-related quality of life for use among patients with gastro-oesophageal reflux disease (GORD), funded as part of the REFLUX trial. This is a large UK multi centre trial that aims to compare the clinical and cost effectiveness of minimal access surgery with best medical treatment for patients with GORD within the NHS. Method Potential items were identified via a series of interviews and focus groups carried out with patients who were receiving/had received medical or surgical treatment for GORD. The final measure consisted of 31 items covering 7 categories (Heartburn; Acid reflux; Wind; Eating and swallowing; Bowel movements; Sleep; Work, physical and social activities). The measure produced two outputs: a quality of life score (RQLS) and five Reflux symptom scores. Reliability (internal consistency), criterion validity with the SF-36 and, sensitivity to change in terms of relationship with reported change in prescribed medication were assessed amongst a sample of 794 patients recruited into the trial. RESULTS The measure was shown to be internally consistent, to show criterion validity with the SF-36 and sensitive to changes in patients use of prescribed medication at baseline and 3 month follow-up. DISCUSSION The Reflux questionnaire is a new self-administered questionnaire for use amongst patients with GORD. Initial findings suggest that the new measure is valid, reliable, acceptable to respondents and simple to administer in both a clinical and research context

The Fatigue Associated with Depression Questionnaire (FAsD): responsiveness and responder definition

PURPOSE: The Fatigue Associated with Depression Questionnaire (FAsD) was developed to assess fatigue and its impact among patients with depression. The purpose of this study was to examine the questionnaire’s responsiveness to change and identify a responder definition for interpretation of treatment-related changes. METHODS: Data were collected at baseline and at 6 weeks from patients with depression starting treatment with a new antidepressant. RESULTS: Of the 96 participants, 55.2% were women, with a mean age of 43.4 years. The total score and both subscales demonstrated statistically significant change with moderate to large effect sizes (absolute values ≥0.76). FAsD change scores were significantly correlated with change on the Brief Fatigue Inventory (r ≥ 0.73; p < 0.001). FAsD mean change scores discriminated among patient subgroups differing by degree of improvement in patient- and clinician-reported fatigue and depression. Responder definition for the two subscales and total score (0.67, 0.57, 0.62) was estimated primarily based on mean change among patients who reported a small but important improvement in fatigue. DISCUSSION: The FAsD was responsive to change, and the responder definition may be used when interpreting treatment-related change. Results add to previous findings suggesting the FAsD is a useful measure of fatigue among patients with depression

Content validity and test-retest reliability of patient perception of intensity of urgency scale (PPIUS) for overactive bladder

BACKGROUND: The Patient Perception of Intensity of Urgency Scale (PPIUS) is a patient-reported outcome instrument intended to measure the intensity of urgency associated with each urinary or incontinence episode. The objectives of this study were to assess the content validity, test-retest reliability, and acclimation effect of the PPIUS in overactive bladder (OAB) patients. METHODS: Patients undergoing treatment for OAB were recruited to participate in a non-interventional study by completing a three-day micturition diary including the PPIUS for three consecutive weeks. Following completion of the three-week study, participants from two select sites also completed a cognitive interview to assess their comprehension of the PPIUS. RESULTS: Thirty-nine participants successfully completed the three-week test-retest study; twelve of these participants completed the cognitive interview. Test-retest reliability was high based on intra-class correlation coefficient of 0.95. Among stable patients, the difference between the mean ratings of any two weeks was non-significant. Among the twelve interview participants, nine found it simple to choose a PPIUS rating for each of their micturition episodes and most found the urgency rating definitions consistent with their urgency experiences. CONCLUSIONS: The results demonstrated content validity based on qualitative interviews, and excellent test-retest reliability among stable patients. In addition, no acclimation effect was observed among stable patients. These findings support the use of the PPIUS as a reliable measure of urgency in both clinical trial and real life settings. The validity of PPIUS could be further established with future studies investigating the relationship between discretely graded urgency and incontinence continuum

Physiotherapist-led suprascapular nerve blocks for persistent shoulder pain: Evaluation of a new service in the UK.

INTRODUCTION: This service evaluation explored and reported findings from a new physiotherapist-led service offering suprascapular nerve blocks (SSNBs) to patients with persistent shoulder pain. METHODS: We collected data before the SSNB injection and at the 6-weeks and 6-month follow-up from consecutive patients with persistent shoulder pain being treated by physiotherapists or an anaesthetist. Outcomes were patient-reported pain (numerical rating scale [NRS 0 to 10]), patient-specific functional score (PSFS) and health-related quality of life [the EuroQol five dimensions questionnaire (EQ5D-5 L)]. Exploratory analyses compared baseline and follow-up scores within each clinician delivery group (physiotherapists, anaesthetist). RESULTS: Forty patients (mean age 57 years [standard deviation {SD} 12]; 63% female) received an SSNB from a physiotherapist, eight patients (mean age 59 years [SD 11]; female 88%) received an SSNB from an anaesthetist. At the 6-week follow-up, the physiotherapy group showed a mean reduction in pain (on the NRS): 2.2 (95% confidence interval [CI] 1.3 to 3.0) and an improvement in function (on the PSFS): -1.3 (95% CI -1.9 to -0.4). Similar changes were found in those treated by the anaesthetist (pain: 1.3 [95% CI -1.18 to 3.80]; function: -1.4 (95% CI -3.18 to 0.35]). Very small changes, that were not statistically significant, were found in EQ5D-5 L scores. At the 6-month follow-up, the mean reduction in pain (NRS) was maintained at 2.0 (95% CI 0.99 to 2.95) for the physiotherapy group. CONCLUSION: The results provide early, exploratory evidence that patients with persistent shoulder pain treated by physiotherapists using palpation-guided SSNBs achieve clinically important changes in pain and function in the short and medium term

Development and evaluation of the Children's Experiences of Dental Anxiety Measure

Background Existing measures of children's dental anxiety have not been developed with children or based on a theoretical framework of dental anxiety. Aim To develop the children's experiences of dental anxiety measure (CEDAM) and evaluate the measure's properties. Design The measure was developed from interviews with dentally anxious children. Children recruited from a dental hospital and secondary school completed the CEDAM and Modified Child Dental Anxiety Scale (MCDAS). A subgroup of children completed the CEDAM before and after receiving an intervention to reduce dental anxiety to examine the measure's responsiveness. Rasch and Classical test analyses were undertaken. Results Children were aged between 9 and 16 years (N = 88 recruited from a dental hospital and N = 159 recruited from a school). Rasch analysis confirmed the measure's unidimensionality. The CEDAM correlated well with the MCDAS (rho = 0.67, P < 0.01) and had excellent internal consistency (Cronbach's alpha = 0.88) and test–retest reliability (ICC = 0.98). The CEDAM was also able to detect changes in dental anxiety following the intervention (baseline mean = 22.36, SD = 2.57 and follow-up mean = 18.88, SD = 2.42, t(df = 37) = 9.54, P < 0.01, Cohen's d = 1.39). Conclusions The results support the reliability, validity and responsiveness of the CEDAM. Initial findings indicate it has potential for use in future intervention trials or in clinical practice to monitor children's dental anxiety