Guyatt's Responsiveness Statistic Research Articles

Development of the Presbyopia Impact and Coping Questionnaire.

IntroductionPresbyopia is a progressive, age-related visual condition that is characterized by reduced ability to focus on near/close objects, causing impacts on individuals’ daily function and health-related quality of life. The Presbyopia Impact and Coping Questionnaire (PICQ) is a new patient-reported outcome (PRO) instrument that assesses presbyopia impact and use of coping behaviors among presbyopic individuals.MethodsTo document the impacts of presbyopia and associated coping behaviors, concept elicitation (CE) interviews were conducted with 20 presbyopic participants. Results from the CE interviews were used to develop draft items for additional testing. Following item generation, the draft PICQ was cognitively debriefed with 20 participants. Data from a phase 2 controlled clinical trial were used for psychometric analyses of the PICQ. The PICQ was administered at site visits throughout a 28-day treatment period. Confirmatory factor analysis (CFA) methods were used to guide the development of the scoring algorithm. The reliability (internal consistency, test–retest), construct validity (convergent and discriminant validity, known-groups methods), and responsiveness (Guyatt's responsiveness statistic [GRS]) of the PICQ scores were evaluated. Finally, anchor-based and distribution-based methods were used to inform thresholds for interpreting meaningful within-patient change.ResultsCE interviews identified the important and relevant presbyopia-related impacts and coping behaviors and 22 items were drafted and cognitively debriefed. Following minor revisions and item addition/deletion, a version of the PICQ including 23 items was subjected to psychometric testing. The analysis sample included 151 participants. The CFA established two PICQ domain scores, Coping and Impact, on 0-to-4 scales that demonstrate good model fit (root mean square error of approximation = 0.06, comparative fit index = 0.98, Tucker-Lewis index = 0.98, standardized root mean square = 0.07). Cronbach’s alphas for the Coping and Impact scores were 0.89 and 0.84, respectively. Test–retest intraclass correlation coefficients were 0.77 for Coping and 0.67 for Impact. The pattern of results assessing construct validity was acceptable for the PICQ Coping and Impact scores, with the magnitude of correlations and effect sizes generally meeting a priori expectations. The corresponding GRS effect sizes for the PICQ Coping scores were −1.23 (i.e., large) for Patient Global Impression of Change (PGIC) and −0.72 (i.e., medium) for uncorrected near visual acuity (UNVA). The GRS effect sizes for the PICQ Impact scores were −0.60 (i.e., medium) for PGIC and −0.35 (i.e., small) for UNVA. Across three sets of anchor-based analyses for interpreting individual-level change, a responder threshold of −1.00 was identified for both PICQ Coping and PICQ Impact scores.ConclusionsThe totality of evidence from the qualitative and quantitative research establishes that the PICQ scores produced are valid and reliable measures of presbyopia impacts and coping behaviors that are important and relevant for assessing presbyopia treatment outcomes.ClinicalTrials.gov identifierNCT02780115; date of registration May 19, 2016.

Validation of the Impact Index: can we measure disease effects on quality of life in patients with hip and knee osteoarthritis?

To validate the Impact Index, a short, publicly available scale that measures the extent to which a respondent's health problem adversely impacts their quality of life. Secondary analysis of patients with hip or knee osteoarthritis surveyed after visiting a surgeon at baseline (N = 322) and about 6months after the visit (N = 283). Patients responded to the Impact Index and previously validated questionnaires about overall health, pain, and function. The Impact Index includes four questions that ask how much the respondent is bothered, worried, limited, or in pain due to their health condition over the past 30days. Total scores range from 0 to 12; higher scores indicate more deleterious impact. Patients were mostly female (55%), majority white (95%), had an average age of 65 (SD = 9), and most had surgery (64%). The baseline Impact Index score was 9.48 (SD = 2.63); at follow up 4.75 (SD = 3.54). Impact Index was related to overall health at baseline (r = - 0.49). For knee patients at baseline, Impact Index was negatively related to their knee symptoms (r = - 0.49) and knee pain (r = - 0.67). For hip patients at baseline, Impact Index was negatively related to the Harris Hip score (r = - 0.62). Scale directions varied; however, the signs of all correlations were as hypothesized. The Impact Index was predictive of surgical choice (p < .001, OR = 1.45), however, overall health (p = .88) and comorbidity (p = .24) measures were not. Reliability was acceptable (α = 0.85). Responsiveness statistics suggested overall health, pain, function, and Impact Index measures reflected improvement patients experienced from surgery. The Impact Index had the largest effect sizes (> - 3.4) and Guyatt Responsiveness Statistics (> - 2.3). The Impact Index demonstrated strong evidence of validity, reliability, and responsiveness in hip or knee osteoarthritis patients.

Single-item measure for assessing quality of life in children with drug-resistant epilepsy.

SummaryObjectiveThe current study investigated the psychometric properties of a single‐item quality of life (QOL) measure, the Global Quality of Life in Childhood Epilepsy question (G‐QOLCE), in children with drug‐resistant epilepsy.MethodData came from the Impact of Pediatric Epilepsy Surgery on Health‐Related Quality of Life Study (PESQOL), a multicenter prospective cohort study (n = 118) with observations collected at baseline and at 6 months of follow‐up on children aged 4–18 years. QOL was measured with the QOLCE‐76 and KIDSCREEN‐27. The G‐QOLCE was an overall QOL question derived from the QOLCE‐76. Construct validity and reliability were assessed with Spearman's correlation and intraclass correlation coefficient (ICC). Responsiveness was examined through distribution‐based and anchor‐based methods.ResultsThe G‐QOLCE showed moderate (r ≥ 0.30) to strong (r ≥ 0.50) correlations with composite scores, and most subscales of the QOLCE‐76 and KIDSCREEN‐27 at baseline and 6‐month follow‐up. The G‐QOLCE had moderate test‐retest reliability (ICC range: 0.49–0.72) and was able to detect clinically important change in patients' QOL (standardized response mean: 0.38; probability of change: 0.65; Guyatt's responsiveness statistics: 0.62 and 0.78). Caregiver anxiety and family functioning contributed most strongly to G‐QOLCE scores over time.SignificanceResults offer promising preliminary evidence regarding the validity, reliability, and responsiveness of the proposed single‐item QOL measure. The G‐QOLCE is a potentially useful tool that can be feasibly administered in a busy clinical setting to evaluate clinical status and impact of treatment outcomes in pediatric epilepsy.

Responsiveness of Endoscopic Indices of Disease Activity for Crohn's Disease.

The Crohn's Disease Endoscopic Index of Severity (CDEIS) and the Simple Endoscopic Score for Crohn's Disease (SES-CD) are commonly used to assess Crohn's disease (CD) activity; however neither instrument is fully validated. We evaluated the responsiveness to change of the SES-CD and CDEIS using data from a trial of adalimumab, a drug therapy of known efficacy. Paired video recordings (N=112) of colonoscopies (baseline and week 8-12) obtained from patients with CD who participated in a trial of adalimumab therapy were reviewed in random order, in duplicate, by four central readers (56 pairs of videos by 2 groups of readers). Responsiveness of the SES-CD and the CDEIS was evaluated by comparing correlations between the observed and pre-specified predictions of change scores for these endoscopic indices with a global endoscopic evaluation of severity (GELS), a patient reported outcome (PRO2), and the Crohn's disease activity index (CDAI), and by calculation of the standardized effect size, and Guyatt's Responsiveness statistic (GRS) using 2 definitions of change; (1) treatment assignment and (2) an absolute change in total PRO2 of 50. The potential application of effect size estimates was demonstrated by calculating hypothetical sample sizes for comparing two independent groups. The impact of removing stenosis as an index item and adjusting for the number of segments observed was also assessed. Changes in both endoscopic instruments and the GELS were highly correlated. The SES-CD displayed numerically higher effect sizes for both definitions of change. The standardized effect size and GRS estimates (95% confidence interval) for the SES-CD based on treatment assignment were 0.84 (0.53, 1.15) and 0.79 (0.48, 1.09). Corresponding values for the CDEIS were 0.72 (0.42, 1.02) and 0.75 (0.45, 1.06). The standardized effect size and GRS estimates for the SES-CD based on an absolute change in total PRO2 of 50 points or greater were 0.76 (0.49, 1.02) and 0.93 (0.64, 1.21). Corresponding values for CDEIS were 0.70 (0.44, 0.97), 0.83 (0.55, 1.10). Removal of stenosis as an index item and adjusting for observed segments did not improve responsiveness estimates. Although both the SES-CD and CDEIS are valid measures of endoscopic disease activity that are moderately responsive to changes in endoscopic disease activity, the SES-CD displayed numerically greater responsiveness in this data set.

Global assessment of the severity of epilepsy (GASE) Scale in children: Validity, reliability, responsiveness.

The Global Assessment of Severity of Epilepsy (GASE) Scale is a single-item, 7-point global rating scale designed for neurologist-report of overall severity of epilepsy in children. Building on previous preliminary evidence of its validity and reliability for research and clinical use, this study evaluated the GASE Scale's construct validity, reliability, and responsiveness to changes in severity of epilepsy. Data used for the study arose from the Health-Related Quality of Life in Children with Epilepsy Study (HERQULES), a 2-year multicenter prospective cohort study (n = 374) with observations taken at baseline, and 6, 12, and 24 months after diagnosis. Construct validity and reliability were quantified using Spearman's correlation and intraclass correlation coefficient (ICC). Responsiveness was assessed using both distribution-based and anchor-based indices. The GASE Scale was at least moderately correlated (r ≥ 0.30) with several key clinical aspects and most strongly correlated with frequency and intensity of seizures and interference of epilepsy or drugs with daily activities (r > 0.30). Total variation in GASE Scale scores explained by seven core clinical aspects of epilepsy increased over time (R(2) = 28% at baseline to R(2) = 70% at 24 months). The GASE Scale had modest test-retest reliability (ICC range: 0.52-0.64) and was responsive to changes in clinical criteria (standardized response mean range: 0.49-0.68; probability of change range: 0.69-0.75; Guyatt's responsiveness statistic range: 0.56-0.84). The GASE Scale showed potential to discriminate "stable" and "changed" patients according to select criteria and to a composite score (area under the receiver operating characteristic [ROC] curve range: 0.50-0.67). Results offer additional evidence in support of the GASE Scale's validity, reliability, as well as responsiveness to changes in severity of epilepsy in children. We conclude that the GASE Scale is a potentially useful tool for assessing the severity of epilepsy in both clinical and research settings.

Development and validation of a histological index for UC

ObjectiveAlthough the Geboes score (GS) and modified Riley score (MRS) are commonly used to evaluate histological disease activity in UC, their operating properties are unknown. Accordingly, we developed an alternative...

Feasibility and validity of the pediatric ulcerative colitis activity index in routine clinical practice.

The Pediatric Ulcerative Colitis Activity Index (PUCAI) is a noninvasive disease activity index developed as a clinical trial endpoint. More recently, practice guidelines have recommended the use of PUCAI in routine clinical care. We therefore sought to evaluate the feasibility, validity, and responsiveness of PUCAI in a large, diverse collection of pediatric gastroenterology practices. We extracted data from the 2 most recent encounters for patients with ulcerative colitis in the ImproveCareNow registry. Feasibility was determined by the percentage of patients for whom all PUCAI components were recorded, validity by correlation of PUCAI scores across physician global assessment (PGA) categories, and responsiveness to change by the correlation between the change in PUCAI and PGA scores between visits. A total of 2503 patients were included (49.5% boys, age 15.2 ± 4.1 years, disease duration 3.7 ± 3.2 years). All items in the PUCAI were completed for 96% of visits. PUCAI demonstrated excellent discriminatory ability between remission, mild, and moderate disease; discrimination between moderate and severe disease was less robust. There was good correlation with PGA (r = 0.76 [P < 0.001] and weighted kappa κ = 0.73 [P < 0.001]). The PUCAI change scores correlated well with PGA change scores (P < 0.001). Test-retest reliability of the PUCAI was good (intraclass correlation coefficient 0.72 [95% confidence interval 0.70-0.75], P < 0.001). Guyatt responsiveness statistic was 1.18, and the correlation of ΔPUCAI with ΔPGA was 0.69 (P < 0.001). The PUCAI is feasible to use in routine clinical settings. Evidence of its validity and responsiveness supports its use as a clinical tool for monitoring disease activity for patients with ulcerative colitis.

Comparing and Validating Simple Measures of Patient-Reported Peripheral Neuropathy for Oncology Clinical Trials: NCCTG N0897 (Alliance) A Pooled Analysis of 2440 Patients.

Current standard evaluation of Peripheral Neuropathy (PN) is based on an investigator-reported classification system that is commonly unable to correctly reflect the subjective symptoms for patients. Thus more reliable methods to assess PN are needed. This study assessed alternative methods of assessing patient-reported PN in 5 North Central Cancer Treatment Group (NCCTG) clinical trials. Two single-item assessments relating to numbness and tingling were used to measure PN. Patients' Quality Of Life (QOL) was also assessed using the Uniscale, Symptom Distress Scale (SDS), Profile of Mood States (POMS), Brief Pain Inventory (BPI) and Subject Global Impression of Change (SGIC). Wilcoxon tests compared QOL scores between patients with PN (score > 50) vs. no PN (score ≤ 50). Changes from baseline in QOL were compared by Wilcoxon rank sum test with a 20-point change in PN defined as clinically meaningful. Both distribution-based and anchor-based approaches were used to derive estimates of Minimal Important Differences (MID). Standardized Response Means (SRM), Effect Sizes (ES) and Guyatt's responsiveness statistic were used to measure responsiveness. The proportion of patients reporting numbness (tingling) at baseline was 10.7% (10.0%) and 18.4% (17.8%) at last assessment. The correlation between numbness and tingling at baseline was 0.81, and at last assessment was 0.83. Patients with substantial PN reported an average of 10 points lower overall QOL, mood and worse symptom distress and 20 points lower in the BPI interference items. Patients having a ≤ 20 point worsening in PN score reported significantly worse in symptom distress and BPI worst pain, but not in POMS or overall QOL. The MID estimates were similar between numbness and tingling items but varied depending on the approach used. Responsiveness statistics indicated that the two PN assessments are sensitive and responsive instruments for cancer patients with PN. The two PN items for numbness and tingling were redundant. Evidence of criterion validity and responsiveness indicates that these simple measures of PN can be used successfully in cancer clinical trials.

Sa1197 Ulcerative Colitis Patients After Pouch Operation Are Serologically More Comparable to Crohn's Disease Patients

characteristic (ROC) curves. The strength of relationship was evaluated according to conventional thresholds whereby 0.2, 0.5, and 0.8 indicate small, moderate, and large degrees of responsiveness, respectively.1 Results: Among 121 patients, 29 were clinically unchanged and 92 were changed (Table 1). Between weeks 0 and 6 or 10, the effect sizes and Guyatt's responsiveness statistics (95% confidence intervals [CIs]) based on mean scores for the MMCS, MBS, and UCEIS were 0.49 (0.28, 0.71), 0.49 (0.28, 0.71), and 0.58 (0.36, 0.81) and 0.32 (0.11, 0.53), 0.33 (0.12, 0.54), and 0.47 (0.25, 0.69), respectively. The corresponding estimates (95% CI) for the areas under the ROC curves were 0.66 (0.55, 0.78), 0.65 (0.54, 0.77), and 0.68 (0.58, 0.79) The ROC curves are displayed in Figure 1. Conclusion: Although the UCEIS had greater numerical values, the MMCS, MBS, and UCEIS displayed similar, small-to-moderate, responsiveness for the assessment of UC disease activity. These results have important implications for sample size in early drug development.

Measurement properties of the benign prostatic hyperplasia impact index in tadalafil studies

BackgroundTo assess the measurement properties of the Benign Prostatic Hyperplasia Impact Index (BII) for use in men with Lower Urinary Tract Symptoms (LUTS) secondary to Benign Prostatic Hyperplasia (BPH) treated with tadalafil.MethodsData from a dose-titration (Study 1) and a dose-finding placebo-controlled (Study 2) tadalafil studies of men 45 years of age or older with moderate to severe LUTS (N = 281; N = 1053) were included in this post-hoc analysis. Measures included the BII, International Prostate Symptom Score (IPSS), IPSS Quality of Life Index (IPSS-QoL), LUTS Global Assessment Question, uroflowmetry measure peak flow rate (Qmax) and postvoid residual volume (PVR). Spearman rank and Pearson correlation coefficients were computed between the BII score and the other measures at each visit. Wilcoxin two-sample tests, t-tests and general linear modeling compared BII scores of subjects with global ratings of improvement versus no improvement, and subjects taking tadalafil versus placebo. Effect size, standardized response mean and Guyatt's responsiveness statistic were calculated for BII and IPSS change scores.ResultsThere were high correlations between BII and IPSS & IPSS-QoL and low correlations between BII and Qmax & PVR at each visit. There were significant differences in BII at the End-of-Study Visit between subjects reporting improvement versus subjects reporting no improvement (Studies 1 and 2, P < .0001) and subjects taking tadalafil versus subjects taking placebo (Study 1, P = .0045; Study 2, P = .0064). The BII and IPSS were both responsive to change.ConclusionsResults show that the BII is reliable, shows responsiveness to change in patients with BPH-LUTS, and demonstrates construct validity.

Appraisal of the Pediatric Crohn's Disease Activity Index on Four Prospectively Collected Datasets: Recommended Cutoff Values and Clinimetric Properties

The Pediatric Crohn's Disease Activity Index (PCDAI) is the outcome measure of choice in clinical trials of pediatric Crohn's disease. The aim of this study was to provide knowledge on its performance and accuracy of different cutoff scores. Longitudinal data prospectively generated from four sources were used, including the REACH and budesonide trials, a North-American inflammatory bowel diseases (IBD) registry, and a cohort aimed at evaluating growth. Cutoff values of disease activity were determined by physician global assessment from the pooled cohort using serial receiver operator characteristic curves and area under the curve (AUC) as well as comparing the overall accuracy. Test-retest reliability and responsiveness were ascertained by comparing the baseline and follow-up scores, using an external anchor. A total of 437 children were included (268 (61%) males, mean age 12.9+/-2.6 years). To define remission, a composite definition of <10 points or <7.5 points without the height item had the highest accuracy; this addressed the limitation that height is not a responsive item. The best cutoff of 10-27.5 was determined for mild disease, 30-37.5 for moderate disease, 40-100 for severe disease, and a change of >12.5 points for response (AUC 0.8-0.9; P<0.001). Ninety children whose disease remained unchanged showed fair test-retest reliability (intraclass correlation coefficient=0.74-0.8; P<0.001). The PCDAI showed good responsiveness, as reflected from the correlational (r=0.7; P<0.001), distributional (Guyatt's responsiveness statistics=0.9), and diagnostic utility analysis (AUC 0.85 (95% confidence interval 0.81-0.88). The clinimetric properties of the PCDAI are sufficient to support its use in clinical research. Cutoff values suggested by this study differ slightly from those previously published on much smaller cohorts.

The Drooling Impact Scale: a measure of the impact of drooling in children with developmental disabilities

To describe the development and clinimetric properties of a new scale to evaluate changes in the impact of drooling in children with developmental disabilities. After examining the properties of potential items, 10 items were retained for inclusion in the final Drooling Impact Scale. The clinimetric properties of the scale were evaluated using data from two convenience samples of children attending a saliva-control clinic: a stable group (n=31, 22 males, nine females; mean age 10y 7mo, SD 4y 5mo, range 3y 6mo-18y 3mo; cerebral palsy [CP] n=17, intellectual disability n=10; non-ambulatory n=13, nonverbal n=12) and an intervention group (n=49, 29 males, 20 females; mean age 11y, SD 3y 6mo, range 3y 4mo-16y 10mo; CP n=31, intellectual disability n=15; non-ambulatory n=27, nonverbal n=28). To assess validity, changes in scores on the Drooling Impact Scale over time were compared with a carer's global rating of change using Pearson's correlations and t-tests. A concordance correlation coefficient was used to compute the level of agreement between assessments 1 month apart in stable children. Effect size, standardized response mean, Guyatt responsiveness statistic, and an unpaired t-test were used to estimate responsiveness. The correlation between the global rating and change in Drooling Impact Scale scores was 0.69 (p<0.001). The concordance correlation coefficient was 0.85. An effect size of 1.8, standardized response mean of 1.5, Guyatt responsiveness statistic of 1.4, and mean group difference of 23.5 (95% confidence interval 17.4-29.6) were obtained. The Drooling Impact Scale is a valid and reliable subjective measure that is responsive to change.

Lysholm scale and WOMAC index were responsive in prospective cohort of young general practice patients

Objective To determine the construct validity and responsiveness of the Lysholm knee scoring scale and the WOMAC osteoarthritis index in adolescents and young adults with knee complaints in general practice. Study Design and Setting In the framework of a prospective cohort study with 1-year follow-up, we included 314 patients aged 12–35 years consulting the general practitioner for incident knee complaints. Subgroup analyses of traumatic and nontraumatic knee complaints and of adolescents and adults were performed. Results Construct validity was adequate for both questionnaires both in traumatic and nontraumatic patients (aged 12–35) and in adolescents (12–17) and young adults (18–35). Effect size (ES) and standardized response mean (SRM) for both Lysholm and WOMAC global scores were moderate in nontraumatic patients and high in traumatic patients. Guyatt's responsiveness statistic was high in both subpopulations. Adolescents showed high responsiveness with all measures on the Lysholm scale, and moderate (Guyatt's statistic) to high responsiveness (ES and SRM) on the WOMAC index. Young adults showed high responsiveness with all measures on both instruments. Conclusion Although neither of the scales was developed for use in adolescents and young adults in general practice, both scales show adequate responsiveness, content, and construct validity in this population.

Assessing responsiveness of generic and specific health related quality of life measures in heart failure

BackgroundResponsiveness, or sensitivity to clinical change, is an important consideration in selection of a health-related quality of life (HRQL) measure for trials or clinical applications. Many approaches can be used to assess responsiveness, which may affect the interpretation of study results. We compared the relative responsiveness of generic and heart failure specific HRQL instruments, as measured both by common psychometric indices and by external clinical criteria.MethodsWe analyzed data collected at baseline and 6-weeks in 298 subjects with heart failure on the following HRQL measures: EQ-5D (US, UK, and VAS Scoring), Kansas City Cardiomyopathy Questionnaire (KCCQ) (Clinical and Overall Summary Score), and RAND12 (Physical and Mental Component Summaries). Three external indicators of clinical change were used to classify subjects as improved, deteriorated, or unchanged: 6-minute walk test, New York Heart Association (NYHA) class, and physician global rating of change. Four responsiveness statistics (T-test, effect size, Guyatt's responsiveness statistic, and standardized response mean) were used to evaluate the responsiveness of the select measures. The median rank of each HRQL measure across responsiveness indices and clinical criteria was then determined.ResultsAverage age of subjects was 60 years, 75 percent were male, and had moderate to severe heart failure symptoms. Overall, the KCCQ Summary Scores had the highest relative ranking, irrespective of the responsiveness index or external criterion used. Importantly, we observed that the relative ranking of responsiveness of the generic measures (i.e. EQ-5D, RAND12) was influenced by both the responsive indices and external criterion used.ConclusionThe disease specific KCCQ was the most responsive HRQL measure assessing change over a 6-week period, although generic measures provide information for which the KCCQ is not suitable. The responsiveness of generic HRQL measures may be affected by the index used, as well as the external criterion to classify patients who have clinically change or remained stable.

Sensitivity to Change and Internal Consistency of the Northwick Park Neck Pain Questionnaire and Derivation of a Minimal Clinically Important Difference

To estimate the sensitivity to change and internal consistency of the Northwick Park Neck Pain Questionnaire (NPQ) and derive its minimal clinically important difference (MCID). Data on 311 participants in a randomized controlled trial of physical therapy for neck pain were analyzed, between baseline and 6-month follow-up. Cronbach alpha was used to estimate internal consistency. Three methods of evaluating the NPQ's sensitivity to change were used; Cohen's effect size, standardized response mean, and Guyatt's responsiveness statistic. The smallest change on the NPQ that can be construed as clinically important (the MCID) was obtained by (1) comparing values derived from different methods that account for the measurement error of the questionnaire with improvements in other neck pain-specific measures, and then (2) examining the impact of the addition of an independent measure ("anchor") relating to participants' subjective rating of improvement. Cronbach alpha was 0.79 for the whole scale. Using all participants, Cohen's d was 0.78, standardized response mean was 0.71, and Guyatt's responsiveness statistic was 0.93. Using only participants who stated they had improved, the estimates were 1.35, 1.27, 1.45, respectively. The MCID for the NPQ was determined as a 25% reduction in score from baseline together with a patient's global rating of their neck pain of at least "better." These results show high internal consistency and sensitivity to change for the NPQ, and provide an MCID that allows participants with varying levels of severity to demonstrate improvement.

Development and Validation of the Effectiveness of Auditory Rehabilitation Scale

To develop a new scale of hearing-related function and quality of life in patients with hearing aids that addresses overlooked concerns, such as hearing-aid comfort, convenience, and cosmetic appearance, that may influence hearing-aid adherence while maintaining brevity and sensitivity to clinical change. Prospective, multicenter instrument validation. Four diverse sites in Washington State, including 2 private practices, 1 university setting, and 1 Veterans Affairs hospital. Seventy-eight patients with hearing aids. We created 2 modules in the Effectiveness of Auditory Rehabilitation (EAR) scale. The first module (Inner EAR) covers intrinsic hearing issues such as hearing in quiet and hearing in noise and is administered both before and after treatment. The second module (Outer EAR) covers extrinsic (hearing-aid related) issues such as comfort, appearance, and convenience and is administered after hearing-aid fitting. Both scales were developed and validated in 3 stages. Stage 1 used a qualitative approach from multiple data sources to develop preliminary instruments. Stage 2 used approaches from classic test theory to reduce the number of items and psychometrically validate the instruments. Stage 3 examined the responsiveness or sensitivity to clinical change. A 10-item Inner EAR module and a 10-item Outer EAR module were created and validated. Internal consistency of individual domains (Cronbach alpha = 0.85 and 0.72, respectively) and test-retest reliability (intraclass correlation coefficients = 0.76 and 0.81, respectively) were excellent. Evidence of construct validity included concurrent validity with other hearing scales and global visual analog scales, discriminant validity with dizziness handicap, correlation with hearing-aid adherence, and confirmatory factor analyses. Both scales had strong evidence of responsiveness (sensitivity to change), with higher effect sizes and Guyatt responsiveness statistics than the 2 widely used hearing scales in this study. The scales took an average of 5 minutes to complete. The EAR scale is a valid and reliable measure of the effectiveness of amplification in the treatment of sensorineural hearing loss. It addresses the range of issues that are of importance to hearing-aid patients. The scales have excellent psychometric properties, are more responsive than several widely used hearing scales, and are minimally burdensome for patients to complete. The EAR may be a valuable outcome measure in future studies of both existing hearing aids and newer hearing-aid technologies, such as bone-anchored aids or middle ear implants.

The 13- and 20-item Hopkins Symptom Checklist Depression Scale: psychometric properties in primary care patients with minor depression or dysthymia.

Depression scales that are responsive to changes in clinical symptoms are important for clinical monitoring and outcomes assessment in longitudinal studies. We evaluated the psychometric properties and responsiveness to clinical change of the 13- and 20-item versions of the Hopkins Symptom Checklist Depression Scale (HSCL-D). A secondary data analysis from a large 11-week, multicenter clinical trial, comparing three treatments was performed. Adult patients with minor depression or dysthymia and a score of > or = 10 on the Hamilton Depression Rating Scale (HDRS) were recruited from primary care clinics. Item-total correlations and Cronbach alphas were computed for HSCL-D-13 and HSCL-D-20. Clinical response at 11 weeks was defined by a Hamilton Depression Rating Scale (HDRS) < 10, clinical remission by a HDRS < 7, and criterion symptom remission by < or = 1 DSM-III-R criterion symptoms. Standardized effect sizes and Guyatt's responsiveness statistic were determined for the 13- and 20-item HSCL-D. Of the 656 subjects enrolled, 511 (77.9%) had complete data and were included in the analysis. Patients were 61.1 +/- 15.0 years old; minor depression was diagnosed in 238, dysthymia in 273. Both scales had good internal consistency; Cronbach's alpha = 0.835 and 0.859 for the 13- and 20-items questionnaires respectively. Standardized effect sizes for clinical response (0.62 for the HSCL-D-13; 0.66 for the HSCL-D-20), clinical remission (0.69 and 0.70), and criterion symptom remission (0.65 and 0.67) showed moderate to large effects and did not differ significantly for the two versions. Responsiveness was virtually identical for patients with minor depression and dysthymia but responsiveness was substantially lower for ethnic minorities. The HSCL-D-13 and 20-item versions have similar responsiveness to change. For use in European Americans, we recommend the HSCL-D-13 if response burden is the preeminent consideration. To more fully capture DSM criterion symptoms, we recommend the HSCL-D-20.

Measurement of global functional performance in patients with rheumatoid arthritis using rheumatology function tests

Outcome assessment in patients with rheumatoid arthritis (RA) includes measurement of physical function. We derived a scale to quantify global physical function in RA, using three performance-based rheumatology function tests (RFTs). We measured grip strength, walking velocity, and shirt button speed in consecutive RA patients attending scheduled appointments at six rheumatology clinics, repeating these measurements after a median interval of 1 year. We extracted the underlying latent variable using principal component factor analysis. We used the Bayesian information criterion to assess the global physical function scale's cross-sectional fit to criterion standards. The criteria were joint tenderness, swelling, and deformity, pain, physical disability, current work status, and vital status at 6 years after study enrolment. We computed Guyatt's responsiveness statistic for improvement according to the American College of Rheumatology (ACR) definition. Baseline functional performance data were available for 777 patients, and follow-up data were available for 681. Mean ± standard deviation for each RFT at baseline were: grip strength, 14 ± 10 kg; walking velocity, 194 ± 82 ft/min; and shirt button speed, 7.1 ± 3.8 buttons/min. Grip strength and walking velocity departed significantly from normality. The three RFTs loaded strongly on a single factor that explained ≥70% of their combined variance. We rescaled the factor to vary from 0 to 100. Its mean ± standard deviation was 41 ± 20, with a normal distribution. The new global scale had a stronger fit than the primary RFT to most of the criterion standards. It correlated more strongly with physical disability at follow-up and was more responsive to improvement defined according to the ACR20 and ACR50 definitions. We conclude that a performance-based physical function scale extracted from three RFTs has acceptable distributional and measurement properties and is responsive to clinically meaningful change. It provides a parsimonious scale to measure global physical function in RA.

Is the health utilities index responsive in total hip arthroplasty patients?

Objective The purpose is to examine the responsiveness of the Health Utilities Index Mark 2 (HUI2), Mark 3 (HUI3), and other generic and disease-specific measures in osteoarthritis patients undergoing total hip arthroplasty (THA). Methods Ninety patients (mean age = 68.13; SD = 8.15) on a waiting list for THA completed measures that included the standard gamble, HUI2, HUI3, SF-36, Harris Hip Scale, WOMAC, and MACTAR. before and after THA. Responsiveness statistics (effect size, standardized response mean, Guyatt's responsiveness statistic, paired-sample t-tests, and relative efficiency statistic) were calculated. Results The disease-specific measures were more responsive than the generic measures. Rankings of the degree of responsiveness varied depending on the responsiveness statistic used. Conclusions Disease-specific measures are the most responsive in THA patients. However, the SF-36, HUI2, and HUI3 had summary scores and domain/attributes scores that were also responsive and provided additional information. Among the generic measures, HUI3 was the most responsive.

Randomized, open-label preference study of two cyclosporine capsule formulations (usp modified) in stable solid-organ transplant recipients

Background: Prior research has indicated patient dissatisfaction with the odor, size, and taste of cyclosporine capsules, as well as the halitosis and body odor the capsules can cause. Objectives: The purposes of this investigation were to (1) compare the overall cyclosporine capsule preference (Gengraf TM vs Neoral®) in stable, solid-organ transplant recipients, (2) assess patient preference based on specific capsule attributes, and (3) determine the reliability of the Cyclosporine Capsule SatiSfaCtion Survey (original to this study). Methods: In this multicenter, randomized, open-label, parallel-group, preference study, patients were recruited from 144 centers in North America with established transplant programs. Solid-organ transplant recipients who had taken stable doses of cyclosporine (Neoral) for ≥2 consecutive months were randomized in a 9:1 ratio to receive another cyclosporine formulation (Gengraf) or to remain on Neoral therapy. Patients completed the Cyclosporine Capsule Satisfaction Survey prior to randomization (baseline survey) and after taking the study drug for 4 weeks (final survey). The survey consisted of multiple attribute items with high face validity in assessing patients' perceptions and preferences with regard to their overall experience, as well as specific attributes of cyclosporine capsules known to affect patient acceptance. Results: The intent-to-treat population included 1906 patients (1211 men, 693 women [sex unknown in 2 patients]; mean [SD] age, 50.2 [12.4] years). A total of 1708 patients were switched to Gengraf; 198 continued on Neoral. Based on their overall experience with both capsule formulations, the majority of patients switched to Gengraf (61.9%) responded that they preferred the Gengraf capsule, compared with 13.7% who preferred the Neoral capsule and 24.4% who indicated no preference ( P < 0.001). A similar preference for Gengraf was observed based on capsule odor (66.3%), ease of swallowing (51.5%), taste (57.1%), and impact on breath odor (52.5%) and body odor (48.4%) ( P < 0.001 for each test). The results of internal consistency and reproducibility calculations were high for the Cyclosporine Capsule Satisfaction Survey. Internal consistency ranged from alpha = 0.84 to 0.95 for the subscales and was alpha = 0.95 for the overall score. Ranges for reproducibility in the subscales were r = 0.75 to 0.79, with an overall reproducibility of r = 0.85. Guyatt's responsiveness statistics for the subscale and overall scores were moderately high to very high, indicating that the survey is capable of measuring change in response to treatment. Conclusions: Of the transplant recipients receiving Gengraf in this study, most preferred Gengraf to Neoral based on overall experience, capsule odor, difficulty swallowing, taste, breath odor, and body odor. Among all study patients, fewer patients receiving Gengraf were bothered by capsule odor, difficulty in swallowing, taste, or the impact on breath or body odor compared with patients who continued to receive Neoral. Internal consistency, reproducibility, and responsiveness results show that the Cyclosporine Capsule Satisfaction Survey is a psychometrically valid instrument that is appropriate for use in clinical trials.