High Ceiling Effects Research Articles

Comparison of the early childhood oral health impact scale (ECOHIS-4D) and EuroQol-5D-Y for measuring oral health-related quality of life utility in children.

The psychometric properties of the Early Childhood Oral Health Impact Scale (ECOHIS-4D), an oral health-related quality-of-life tool for children, and EuroQol 5D-Young (EQ-5D-Y), a commonly used generic quality-of-life tool for children, were compared across a clinical severity index to determine which tool is better for measuring oral health-related quality of life in children. Delayed and filled surfaces score (dfs) was calculated for under six-year-old children at the recruitment and one year later. ECOHIS-4D and EQ-5D were filled out on both occasions. The following properties were examined: (i) Content validity by comparing mean utility values using the Wilcoxson Signed Rank Test and Bland Altman Test, (ii) Construct validity (ability to discriminate between groups of different clinical severity) by examining effect size between severity groups (iii) Responsiveness (for changes in health status) using floor and ceiling effect, standard response mean and linear regression (iv) Correlation between the utilities and dfs. Respectively, 287 and 189 children were examined at the baseline and follow-up. Mean utility estimates from the tools were significantly different. All items of EQ-5D-Y showed high ceiling effects compared to items of ECOHIS-4D. Only the utility scores of ECOHIS-4D showed a significant association with the dfs score (β=-0.003, 95%CI=-0.004 to -0.002) in the linear regression models. Utility values form ECOHIS-4D, but not EQ-5D-Y correlated with the dfs (spearman's r=-0.33, 95%CI -0.43 to -0.23). ECOHIS-4D performs better than EQ-5D-Y in assessing young children's oral health-related quality of life.

Mapping of the health of the Nation Outcomes Scale (HoNOS) and Positive and Negative Symptom Scale (PANSS) to the EQ-5D-3L in psychosis patients using the PHAMOUS study

ABSTRACT Objectives When health outcomes relevant for economic evaluations are unavailable, algorithms can be developed to map utilities using available clinical outcome measures. This study aims to develop two mapping algorithms estimating EuroQol-5 dimension-3 level (EQ-5D-3 L) utilities using the clinician-rated Health of the Nation Outcome Scores (HoNOS) and Positive and Negative Syndrome Scale (PANNS). Methods A dataset with 2,029 observations of patients with psychotic disorders included EQ-5D-3 L, HoNOS, PANSS item scores, and demographics. Correlations between instruments were evaluated. Least Absolute Shrinkage and Selection Operator (LASSO) regression and random forest (RF) algorithms with various predictor variable sets were applied. Model performance was cross-validated using R-squared and Root Mean Square Error (RMSE). Results High ceiling effects were observed for EQ-5D-3 L, with weak to moderate negative correlations between EQ-5D and HoNOS (r = -0.34) and PANSS (r = -0.27). Overall, LASSO models outperformed RF models, with individual item models performing best for the HoNOS and PANSS, with the best observed RMSEs of 0.241 and 0.231, respectively. Conclusions The HoNOS and PANSS could be mapped onto EQ-5D-3 L utilities but lack accuracy for individual patient predictions. However, in the absence of alternatives, they could adequately predict population-based utility score differences for health economic evaluations.

Longitudinal Evaluation Using the Forgotten Joint Score-12 for Double-Bundle Primary Anterior Cruciate Ligament Reconstruction: A Retrospective Observational Study.

Introduction There are several postoperative evaluation methods for anterior cruciate ligament reconstruction (ACLR), and patient-reported outcome measurement (PROM) is one of the most important for evaluating postoperative clinical results. However, conventional PROMs have a high risk ofa ceiling effect at one and two years postoperatively and do not accurately reflect functional improvement over time. Therefore, a longitudinal evaluation using PROM with a low risk of ceiling effect is necessary. The forgotten joint score-12 (FJS) was developedfor clinical evaluation after arthroplasty. It is considered an evaluation method after arthroplasty with a low risk of demonstrating a ceiling effect. However, few studies have used the FJS as an evaluation method after ACLR, particularly those tracking changes over time. This study aimed to longitudinally evaluate FJS at one and two years after ACLR. Methods This is a retrospective observational study of postoperative patients using existing data and a questionnaire-based survey.This studyincluded patients who underwent primary double-bundle ACLRbetween August 2017 and August 2021. We compared the FJS, knee injury and osteoarthritis outcome score (KOOS), and Lysholm knee scale (LKS) scores at one and two years post-surgery using the Wilcoxon signed-rank test. The ceiling effect for each PROM was calculated at one and two years post-surgery.A ceiling was defined as obtaining a perfect score in PROMs, and the risk of demonstrating a ceiling effect was the percentage of perfect scores among all cases. The risk of demonstrating a ceiling effect for each PROM was compared using the McNemar test. To identify factors influencing FJS one-year post-ACLR across all cases, multivariate linear regression analysis was conducted for the FJS. Results Finally, 87 patients were included in this study.Fifty-six participants were women, and 31 were men, with an average age of 28.5 ± 11.8 years at the time of surgery and a BMI of 23.2 ± 3.7 kg/m2. Fifty-eight patients with meniscus injuries requiring treatment were observed. A total of 54 patients were used to compare the results at one and two years, while 87 patients were included in the multivariate analysis for FJS at one year.The median of FJS at one and two years post-surgery were 90.6 and 95.8, respectively. The FJS showed a statistically significant improvement from one to two years (p = 0.033). Question #12 in FJS showed a significant improvement from the first to the second year postoperatively (1.74 ± 1.46 vs 1.15 ± 1.25 at one year vs two years, respectively; p = 0.0016). FJS had a lower risk of demonstrating a ceiling effect than KOOS ADL at one and two years (FJS vs KOOS ADL; at first year: 20.4% and 48.1%, p < 0.001; at second year: 33.3% and 63.0%, p = 0.0013). There was no difference compared to the other PROMs. According to the multivariate linear regression analysis,predictive factors for higher FJS scoresat one year post-surgery were younger age and limb symmetry index of single-leg hop test(SLH-LSI) on the affected side that was close to that of the healthy side (SLH-LSI > 0.9). Conclusions The FJS continued to improve over two years after ACLR. The FJS post-ACLR was higher in younger individuals and those with SLH-LSI of 0.9 or higher.

Responsiveness of the Various Short-Form Versions of the Knee Injury and Osteoarthritis Outcome Score Between 2 and 5 Years After Anterior Cruciate Ligament Reconstruction

Background: Various short-form versions of the Knee injury and Osteoarthritis Outcome Score (KOOS) have been developed in an attempt to minimize responder burden. However, the responsiveness of these short-form measures in patients who have undergone anterior cruciate ligament (ACL) reconstruction has not been compared at midterm follow-up. Purpose: To determine the responsiveness of 3 short-form versions of the KOOS (KOOS-12, KOOS-Global, and KOOS-ACL) in patients who have undergone ACL reconstruction. Study Design: Cohort study (diagnosis); Level of evidence, 3. Methods: In 276 patients (149 male, 127 female), we administered the KOOS and a measure of overall knee function at both 2 and 5 years after ACL reconstruction. From the full KOOS, the following short-form versions were calculated: KOOS-12, KOOS-Global, and KOOS-ACL. Responsiveness was assessed using several distribution and anchor-based methods for each of the short-form versions. From distribution statistics the standardized response mean (SRM) and smallest detectable change (SDC) were calculated. Using the anchor-based method, the minimally important change (MIC) that was associated with an improvement in knee function was determined using receiver operating characteristic (ROC) analysis. Results: High ceiling effects were present for all measures. KOOS-Global scores increased significantly over time, whereas KOOS-12 and KOOS-ACL did not change. The KOOS-Quality of Life (QOL) subscale, which can be derived from both KOOS-Global and KOOS-12, also increased significantly between assessments. Both these increases were associated with a small (0.2-0.3) SRM. The MIC was smallest for KOOS-Global (3.2 points) and largest for KOOS-QOL (9.4 points), and, for all measures, the MIC was larger than the SDC at a group level. KOOS-Global was the only measure for which the mean difference between the 2- and 5-year assessments exceeded both the SDC (group level) and the MIC. Conclusion: Of the 3 short-form versions of the KOOS currently available, the KOOS-Global had the greatest responsiveness to change between the 2- and 5-year assessments after ACL reconstruction. High ceiling effects were present for all versions.

Validation testing of OUTPATSAT-35RT questionnaire to assess satisfaction in patients on outpatient radiotherapy in a large Indian cohort

ABSTRACT Background: This study reports the psychometric properties of OUTPASAT-35RT questionnaire from a large Indian cancer cohort treated with ambulatory radiotherapy (RT) at a tertiary-care comprehensive cancer center. Materials and Methods: Adults with a pathologically proven diagnosis of cancer undergoing fractionated RT (≥10 fractions) with the ability to read and understand English, Hindi, or Marathi were accrued after written informed consent. Patients were administered a language-appropriate version of OUTPATSAT-35RT along with European Organization for Research and Treatment of Cancer (EORTC) generic quality-of-life questionnaire (QLQ-C30) at conclusion of RT (±3 days). Psychometric properties of OUTPATSAT-35RT were analyzed. Results: A total of 314 patients were accrued between 2018 and 2020. A negligible floor effect (&lt;2%) but a high ceiling effect (&gt;15%) was observed for OUTPATSAT-35RT. Multi-trait analysis revealed satisfactory validity and reliability of the questionnaire. Pearson’s correlation coefficient (PCC) of individual item score with own scale was &gt;0.4 suggesting good convergent validity. Discriminant validity was satisfactory as the PCC with its own scale/own domain was greater than with other scales of the same domain and other domains of OUTPATSAT-35RT, respectively. Correlation of scales of OUTPATSAT-35RT with scales of QLQ-C30 demonstrated acceptable divergent validity (all values of PCC &lt;0.3). Cronbach’s alpha was &gt;0.7 suggesting good internal consistency. Test–retest reliability was good with intra-class coefficient being &gt;0.7. Economic status was the only socio-demographic factor correlating significantly with OUTPATSAT-35RT scores; satisfaction of patients with the delivery of care increased with increasing family income. Conclusion: The psychometric properties of OUTPATSAT-35RT were found to be acceptable and satisfactory during validation testing in a large Indian cohort.

Assessing Treatment Burden in Pediatric Hemophilia: Validation of the Observer-Reported Child Hemophilia Treatment Experience Measure (Child Hemo-TEM)

Background: Despite treatment safety and efficacy advancements for children with hemophilia (CwH), home treatment can be complicated and burdensome. Treatment burdens are major contributing factors to adherence issues. The Child Hemo-TEM is a patient-centric outcome measure developed following the United States (US) Food and Drug Administration guidance for patient-reported outcome measure development. Designed to assess the treatment burden for CwH, the measure is a caregiver observer-reported outcome (ObsRO) measure where questions are answered by the caregiver about the child's treatment experience based on what the caregiver has seen, heard, or been told by the child. Aim: The purpose of this study was to psychometrically validate the Child Hemo-TEM ObsRO. Methods: Data was collected through a cross-sectional, web survey from a population of caregivers of CwH aged 2 to &amp;lt; 12 years in the US. The survey consisted of the Child Hemo-TEM, sociodemographic items, child's hemophilia medical history, and supporting items/measures required to assess the measure's psychometric properties. The survey took up to 45 minutes to complete and was administered at a single time point. To be eligible for the study, caregivers needed to be: aged ≥ 18 years; a caregiver of a male child with a diagnosis of severe to moderate hemophilia A or B, with a factor level of &amp;lt; 2%, with or without an inhibitor, and currently receiving either prophylactic or on-demand treatment; and living in the same residence as the child over the past 2 weeks. Recruitment was conducted by a patient advocacy group and a professional market research firm. Recruitment quotas were targeted to ensure a diverse population and mix of frequency of prophylactic treatment(s) and treatment administration type. All psychometric analyses were conducted following an a-priori statistical analysis plan, which included examination of measurement characteristics, item reduction, factor analysis, measure scoring, internal consistency, reliability, and validity. Results: Caregivers ( n=187) of CwH completed the web survey. The respondents were, on average, 37.8 years of age, mothers (84.0%), and predominantly White/Caucasian (74.3%). Twelve items were dropped from the validation-ready, 19-item measure: 5 items due to high ceiling effects at baseline (&amp;gt;50%) mirrored by the Rasch results and 1 item as it was highly correlated with a retained item. Further, as there were only a small number of children who self-injected, 6 items designed to assess child self-injection were removed. The remaining items underwent further psychometric testing. An exploratory factor analysis generated a single, 7-item factor. The Child Hemo-TEM was found to be internally consistent with a Cronbach's α of 0.855. A-priori validity hypotheses were adapted to match the final factor structure. Hypothesized associations were predominantly confirmed with a few exceptions. For convergent validity (Pearson's r significance set at α = 0.05), a few of the associations, although significant, were below 0.30 (Table 1). The Child Hemo-TEM was able to discriminate between levels of self-complaining about their infusions (p &amp;lt; 0.001), psychological stress reported by parent (p &amp;lt; 0.001), and ease/difficulty of their medication in its current form, among other variables. Based on psychometric findings and taking into consideration learnings from the concept elicitation during measure development, the brief, 7-item Child Hemo-TEM was finalized with 1 domain capturing information from all the hypothesized concepts of treatment concerns (3 items), adherence (1 item), physical impact (1 item), and interference with daily life (2 items) (Figure 1). A five-point Likert response scale with ranges from “Never” to “Always”, or a “Don't know” option, is used. Conclusion: The Child Hemo-TEM ObsRO has been found to be conceptually sound with adequate evidence to support its reliability and validity. Incorporation of the measure into both clinical and research settings will help assess the pediatric patient's experience of living with hemophilia as well as aid clinicians in tailoring treatments to patient characteristics and situations. Future studies are needed to assess test-retest reproducibility, sensitivity to change, and meaningful change thresholds of this new measure.

Reliability and validity of the Tinetti performance oriented mobility assessment in Chinese community-dwelling older adults

ObjectiveThe Tinetti Performance Oriented Mobility Assessment (POMA) has been used for assessing mobility limitations and predicting falling risk among older adults. This study aimed to evaluate the reliability and validity of the POMA in Chinese community-dwelling older adults. MethodsWe used data from a cross-sectional study in which a sample of 627 older adults completed the POMA. Reliability was tested using internal consistencies and test-retest reliability analyses, while validity was assessed using confirmatory factor analysis and known-group approach. Floor and ceiling effects were also tested. ResultsThe POMA and its two subscales had good internal consistency reliability and interrater reliability. Confirmatory factor analysis indicated the POMA had a two-factor structure. The POMA and its subscales exhibited moderate-to-good discriminant validity. A high ceiling effect was detected. ConclusionsThe POMA had satisfactory reliability and validity among Chinese older adults. Nevertheless, a high ceiling effect may limit its use in community settings.

Measuring glaucoma quality of life in an Asian population using item banking: psychometric evaluation and computerized adaptive testing simulations.

To assess the psychometric properties of glaucoma-specific health-related quality of life (HRQoL) item banks (IBs), and explore their efficiency using computerized adaptive testing (CAT) simulations. In this cross-sectional, clinical study, 300 Asian glaucoma patients answered 221 items within seven IBs: Ocular Comfort Symptoms (OS); Activity Limitation (AL); Lighting (LT); Mobility (MB); Glaucoma Management (GM); Psychosocial (PSY); and Work (WK). Rasch analysis was conducted to assess each IB's psychometric properties (e.g., item "fit" to the construct; unidimensionality) and a set of analytic performance criteria guiding decision making relating to retaining or dropping domains and items was employed. CAT simulations determined the mean number of items for 'high' and 'moderate' measurement precision (stopping rule: SEM 0.3 and 0.387, respectively). Participants' mean age was 67.2 ± 9.2years (62% male; 87% Chinese). LT, MB, and GM displayed good psychometric properties overall. To optimize AL's psychometric properties, 16 items were deleted due to poor "fit", high missing data, item bias, low discrimination and/or a low clinical/patient importance rating. To resolve multidimensionality in PSY, we rehomed 16 items into a "Concern (CN)" domain. PSY and CN required further amendment, including collapsing of response categories, and removal of poorly functioning items (N = 7). Due to poor measurement precision, low applicability and high ceiling effect, low test information indices, and low item separation index the WK IB was not considered further. In CAT simulations on the final seven IBs (n = 182 items total), an average of 12.1 and 15.7 items per IB were required for moderate and high precision measurement, respectively. After reengineering our seven IBs, they displayed robust psychometric properties and good efficiency in CAT simulations. Once finalized, GlauCAT™-Asian may enable comprehensive assessment of the HRQoL impact of glaucoma and associated treatments.

Excellent Correlation of the Patient-Reported Outcomes Measurement Information System Upper Extremity Score With Legacy Outcome Scores Preoperatively and at 1 Year After Arthroscopic Rotator Cuff Repair

To assess the preoperative and postoperative performance of the Patient-Reported Outcomes Measurement Information System Upper Extremity (PROMIS-UE, version 2.0) outcome score in comparison to the American Shoulder and Elbow Surgeons (ASES) and Western Ontario Rotator Cuff Index (WORC) instruments in patients undergoing rotator cuff repair. This prospective longitudinal study included 91 patients undergoing rotator cuff repair. Patients completed the PROMIS-UE, ASES, and WORC instruments preoperatively and postoperatively at 2 weeks, 6 weeks, 3 months, and 12 months. The Pearson correlation coefficient (r) between these tools was calculated at each time point. Correlations were graded as excellent (>0.7), excellent-good (0.61-0.7), good (0.4-0.6), or poor (<0.4). Responsiveness to change was assessed using the effect size and the standardized response mean. Floor and ceiling effects for each instrument were also assessed. The PROMIS-UE instrument showed good to excellent correlation with the legacy instruments at all time points. There were variations in the measured effect sizes of the various instruments, with the PROMIS-UE instrument showing responsiveness to change at 3 and 12 months but the ASES and WORC instruments showing responsiveness at 6 weeks, 3 months, and 12 months. Both PROMIS-UE and ASES scores displayed ceiling effects at 12 months. The PROMIS-UE instrument shows excellent correlation with the ASES instrument and a rotator cuff-specific outcome instrument-the WORC instrument-preoperatively and at 1 year after arthroscopic rotator cuff repair. Variations in the measured effect sizes at different postoperative time points and high ceiling effects of the PROMIS-UE instrument at the 1-year time point may limit its utility in the early postoperative phase and at long-term follow-up after rotator cuff repair. The performance of the PROMIS-UE outcome measure after arthroscopic rotator cuff repair was investigated.

The Effect of Social Deprivation on Patient Satisfaction in Otolaryngology Clinics.

Multiple factors have been associated with lower satisfaction scores. We hypothesize that patients with a more deprived socioeconomic status will have different patient satisfaction scores than patients of higher socioeconomic status. We reviewed Press Ganey satisfaction scores for new, outpatient visits between January 1, 2014, and December 31, 2018. Due to the high ceiling effects of the survey, "satisfaction" was defined as achieving a perfect score of 100. We determined social deprivation using the 2015 Area Deprivation Index (ADI) which serves as a proxy for socioeconomic status derived from zip codes. Patient satisfaction was defined as a binary variable. Univariate and multivariate binary logistic regression analyses were used to identify factors correlated with patient satisfaction. There were 3239 unique new patients with completed surveys. Univariate analysis demonstrated decreased odds of achieving satisfaction for both the Total Score and Provider Sub-Score for each decile increase in ADI (OR 0.94; CI = 0.908-0.981; P = .003 and OR 0.94; CI = 0.91-0.98; P = .002 respectively). Multivariate analysis revealed the odds for reporting satisfaction for each decile increase in ADI were 0.96 for Total Score (CI = 0.921-0.998; P = .038) and 0.96 for Provider Sub-Score (CI = 0.92-0.993; P = .019). Patients in the most deprived quartile, compared to the least deprived, were significantly less likely to be satisfied with their care for both Total Score (OR 0.70; CI = 0.564-0.865; P = .001) and Provider Sub-Score (OR 0.69; CI = 0.558-0.852; P = .001). Increased social deprivation was an independent predictor of lower patient satisfaction in otolaryngology outpatients using the Press Ganey survey. This non-modifiable outcome should be taken into consideration when evaluating patient satisfaction scores and offers further support to the need of addressing and improving healthcare discrepancies in the field of otolaryngology. Level 3.

Validation and reliability testing of the BREAST-Q expectations questionnaire in Swedish

Knowledge about preoperative expectations and how they affect satisfaction with breast reconstruction are key in improving care. A prerequisite for such studies are methodologically sound ways to define and measure expectations. The aims of this study were to translate and culturally adapt BREAST-Q Expectations for Sweden, and to perform a psychometric evaluation of the questionnaire. A cross-sectional study was performed. BREAST-Q expectations was translated according to current guidelines and sent to all patients on the waiting list for a breast reconstruction in our department. Internal consistency was assessed by Cronbach’s α. Inter-item correlations were calculated, and convergent validity was evaluated using a subjective comparator. Bland–Altman plots were drawn to evaluate test–retest reliability. Floor and ceiling effects were calculated. The questionnaire was sent to 198 patients, of which 129 responded (65%). Internal consistency was acceptable for all domains (Cronbach’s α 0.71–0.85) and all except one inter-item correlations were within the predefined intervals. Bland–Altman plots indicated that the agreement is variable. Ceiling effects were high for most domains. The results of the study support that the Swedish version of BREAST-Q expectations has a good content and face validity and internal consistency. Convergent validity and known-group validation cannot be adequately examined for expectations. Further studies are needed regarding test–retest reliability. High ceiling effects indicate that the instrument cannot discriminate between patients with high expectations and very high expectations. More studies are needed on how we can evaluate if expectations are realistic or not. Trial registration ClinicalTrials.gov Identifier NCT04714463, 19 January 2021 Patient and public contribution: All the data were generated directly from patients.

Validation of Single-Outcome Questionnaire in Primary TKA and THA

BackgroundPatient-reported outcome measures can be used to evaluate post-operative health care quality and patient satisfaction. The Patient’s Joint Perception (PJP) question gathers a single patient-reported outcome to measure how patients appraise their joint. This study compares PJP to the Forgotten Joint Score (FJS) at 21 months post-operation to assess its value. MethodsA retrospective review was performed at an orthopedic specialty hospital for patients who completed both PJP and FJS questionnaires in 2020-2021 and underwent either a unilateral elective primary Total Knee Arthroplasty (TKA) or Total Hip Arthroplasty (THA). Spearman’s correlation coefficients and P-values were calculated to determine external validity of PJP. Floor and ceiling effects were analyzed and considered present if ≥ 15% of patients achieved the worst or best score (0-4 for PJP and 0-100 for FJS). ResultsIn total, 534 patients (327 THA and 207 TKA) were surveyed at 21 months post-operation. External validity against FJS was assessed for both TKA (r = 0.66, P < .01) and THA (r = 0.69, P < .01). For TKA, the floor and ceiling effects were 0.97% and 25.12% for PJP and 3.86% and 4.83% for FJS, respectively. For THA, the floor and ceiling effects were 0.92% and 50.46% for PJP and 2.47% and 20.50% for FJS, respectively. ConclusionThe PJP was correlated with FJS moderately for both TKA and THA and can be collected with lesser burden. However, ceiling effects were higher in both TKA and THA for PJP compared to FJS. Further studies are needed to investigate the questionnaires at additional time points and to evaluate the implications of high ceiling effects.

Psychometric study of the SF-36v2 in hereditary angioedema due to C1 inhibitor deficiency (C1-INH-HAE)

BackgroundThe generic 36-item Short-Form Health Survey (SF-36v2) has been used to assess health related quality of life in adult patients with hereditary angioedema due to C1-inhibitor deficiency (C1-INH-HAE) even though it has not yet been validated for use in this specific disease.ObjectiveThis study aims to validate the SF-36v2 for use in adult patients with C1-INH-HAE.ResultsThere was a very low item non-response rate (1–3.4%), with a high ceiling effect in 25/35 items and a low floor effect in 3/35 items. A moderate ceiling effect was observed in 5/8 dimensions of the SF-36v2, whereas no floor effect was noticed in any of the dimensions. Internal consistency was good to excellent with Cronbach's alpha coefficient ranging between 0.82 and 0.93 for the different dimensions. Construct validity was good: seven out of the 8 hypotheses defined on clinical criteria were confirmed, discriminant validity assessment showed significant differences among patients with different C1-INH-HAE severity, convergent validity showed a good correlation among the physical and mental component summaries of the SF-36v2 and the HAE-QoL total score (0.45 and 0.64 respectively, P < 0.001). Test–retest reliability was high with intraclass correlation coefficient varying from 0.758 to 0.962. The minimal clinically important difference was calculated by distribution methods and small differences in the domain scores and in the component summaries scores were shown to be meaningful. ConclusionsThe psychometric properties of the SF-36v2 show it can be a useful tool to assess HRQoL in adult patients with C1-INH-HAE, although with some content validity limitation.MethodsThe psychometric properties of the SF-36v2 were evaluated in an international setting based on responses from 290 adult C1-INH-HAE patients in 11 countries.

Abstract P4-12-11: Eq-5d-5l utility scores for patients with breast cancer

Abstract Background: The EuroQol- 5 Dimension (EQ-5D) is a generic patient-reported outcome measure widely used to capture meaningful change in health-related quality of life between treatments to inform drug and health technology reimbursement decision making. The EQ-5D-5L (5 level) consists of 5 dimensions (mobility, self-care, usual activities, pain/discomfort, and anxiety/depression) and a visual analogue scale (VAS). Each dimension has 5 levels, ranging from 1 “no problems” to 5 “extreme problems”, generating 3125 distinct health states (HS). The VAS score ranges from 0 (worst health imaginable) to 100 (best health imaginable). We used Canadian Index values to calculate community-weighted utility scores for 5 pre-defined breast cancer (BC) disease states.Methods: This cross-sectional study included adult women diagnosed with stage I to IV BC, who completed EQ-5D-5L, during outpatient clinic visits at two academic cancer centres in Toronto. Participants were classified into 5 disease states, considered relevant both to clinical practice and economic modeling, ranging from primary breast cancer to metastatic disease. EQ-5D-5L scores were described for each disease state, and mean utility scores and VAS scores were calculated.Results: 549 women were included; the mean age was 57 (SD 12) years; 72% had a Charlson Comorbidity Index of 0. A majority reported “Slight” to “Moderate” problems for the “Pain/Discomfort” (60%) and “Anxiety/Depression” (55%) dimensions of EQ-5D-5L; in all other dimensions, most patients reported “No problems”. As expected, patients with metastatic BC had the highest proportion of reported problems. The mean EQ-5D-5L index score was 0.83 (SD 0.13; range 0.36 to 0.95), with a distribution skewed towards full health and a ceiling effect of 20% (n=110; Table 1). Only 126 different self-reported health states were observed in our patient population. Mean VAS was 75 (SD 17.5; range 10 to 100). For early BC, mean index scores were: 0.87 (n=49; SD 0.07) for patients on chemotherapy (+/-targeted therapy); 0.85 (n=212; SD 0.12) for patients on endocrine treatment (+/-targeted therapy); 0.79 (n=16; SD 16) for patients on targeted therapy; and 0.84 (n=129; SD 0.14) for patients not receiving systemic therapy. For metastatic BC, mean index scores were: 0.79 (n=36, SD 14) for patients on chemotherapy (+/-targeted therapy); 0.80 (n=79, SD 0.14) for patients on endocrine treatment (+/-targeted therapy); 0.69 (n=8; SD 0.19) for patients on targeted therapy; and 0.77 (n=20; SD 0.14) for patients not receiving systemic therapy.Conclusions: BC had an impact mainly in the “Pain/Discomfort” and “Anxiety/Depression” dimensions of EQ-5D-5L. A high ceiling effect was observed in this patient population, despite using the 5L version of EQ-5D. EQ-5D-5L health utilities were significantly lower for metastatic BC, but no significant differences were found among various early-stage BC states. Table 1.- Mean EQ-5D-5L Index and VAS scoresEQ-5D-5LIndex ScoresVASDisease StateN (%)Mean (SD)Min-MaxMean (SD)Min-Max1st year after primary BC146 (27)0.85 (0.12)*0.23-0.9576.5(16.6)10-1001st year after recurrence or primary BC13 (2)0.78 (0.21)0.13-0.9568.9(23.3)20-1002nd-5th year after a primary BC or recurrence treated with curative intent185 (34)0.84 (0.11)*0.21-0.9578.3(14.7)36-1006th and following years after a primary BC or recurrence treated with curative intent62 (11)0.86 (0.14)*0.36-0.9581.0(15.6)20-100Metastatic BC143 (26)0.78 (0.14)0.29-0.9567.3(19.2)21-100Total549 (100)0.83 (0.13)0.13-0.9575.0(17.5)10-100*significant difference was found between these disease states and metastatic diseases (P &amp;lt; 0.05). Citation Format: Sofia Torres, Ahmed Bayoumi, Maureen Trudeau, Ana Beatriz Kinupe Abrahao, Nicholas Mitsakakis, Murray Krahn, Geoffrey Liu. Eq-5d-5l utility scores for patients with breast cancer [abstract]. In: Proceedings of the 2021 San Antonio Breast Cancer Symposium; 2021 Dec 7-10; San Antonio, TX. Philadelphia (PA): AACR; Cancer Res 2022;82(4 Suppl):Abstract nr P4-12-11.

Analyzing the future time perspective scale in the baseline period in the Anti‐Amyloid treatment in Asymptomatic Alzheimer's Disease (A4) Study

AbstractBackgroundLearning an Alzheimer’s disease (AD) biomarker result can impact a person’s expectations for their future. We assessed the behavior of the Future Time Perspective (FTP) scale in a sample of cognitively unimpaired older adults undergoing AD biomarker testing and disclosure.Method5665 participants who were screened for a multisite randomized preclinical AD trial learned their result of an amyloid PET scan: 1182 elevated and 3310 not elevated. Each completed the FTP scale before and after learning the result. The FTP is a 10‐item scale that measures perceived remaining time. Items are rated on a 7‐point scale. Correlations, factor analysis, within‐subject comparisons, and polytomous analyses explored relationships between FTP and amyloid status. In multinomial logistic regression (MLR) analyses, the base outcome was no FTP change (50th%tile interquartile range of change score distribution). Decreased FTP was the lowest quartile of change score distribution while increased FTP was the upper quartile.ResultsUsing items that showed high uniqueness scores (&lt;0.40) and low ceiling effects (&lt;20% endorsing maximum scale scores), we examined a 3‐item FTP subscale (FTP‐3, score range 1 to 7). Mean change in persons with elevated amyloid was 0.12 (95%CI 0.06 to 0.18) and 0.20 (95%CI 0.17 to 0.24) in those with a not elevated result.In MLR analysis, the mean in the decreased FTP‐3 group was ‐0.89 points (95%CI ‐0.92 to ‐0.86), while the mean increased FTP‐3 was 1.26 (95%CI 1.22 to 1.29). Elevated amyloid results (0.22, 95%CI 0.06 to 0.39) and better self‐rated cognitive function (CFI, ‐0.03, 95%CI ‐0.05 to 0.01) made decreased FTP‐3 more likely than no FTP‐3 change. In the same analysis, lower MMSE scores (‐0.08, 95%CI ‐0.14 to ‐0.03) made increased FTP‐3 more likely than no FTP‐3 change.ConclusionThe 3‐item FTP scale offers an option for a brief measure with limited ceiling effects. Additionally, while mean FTP change due to AD biomarker result disclosure is small (mean=0.18), our findings from polytomous analysis support the hypothesis that AD biomarker result knowledge may have notable effects on FTP (∼2SD). FTP warrants further study in preclinical AD with attention to associations with disease progression, well‐being, and life plans.

Reliability and Validity of the Patient Activation Measure in Kidney Disease: Results of Rasch Analysis.

Despite the increasing prioritization of the promotion of patient activation in nephrology, its applicability to people with CKD is not well established. Before the Patient Activation Measure is universally adopted for use in CKD, it is important to critically evaluate this measure. The aim of this study was to describe the psychometric properties of the Patient Activation Measure in CKD. A survey containing the 13-item Patient Activation Measure was completed by 942 patients with CKD, not treated with dialysis. Data quality was assessed by mean, item response, missing values, floor and ceiling effects, internal consistency (Cronbach's alpha and average interitem correlation), and item-rest correlations. Rasch modeling was used to assess item performance and scaling (item statistics, person and item reliability, rating scale diagnostics, factorial test of residuals, and differential item functioning). The item response was high, with a small number of missing values (<1%). Floor effect was small (range 1%-5%), but the ceiling effect was above 15% for nine items (range 15%-38%). The Patient Activation Measure demonstrated good internal consistency overall (Cronbach α=0.925, and average interitem correlation 0.502). The difficulty of the Patient Activation Measure items ranged from -0.90 to 0.86. Differential item functioning was found for disease type (item 3) and age (item 12). The person separation index was 9.48 and item separation index was 3.21. The 13-item Patient Activation Measure appears to be a suitably reliable and valid instrument for assessing patient activation in CKD. In the absence of a kidney-specific instrument, our results support the 13-item Patient Activation Measure as a promising measure to assess activation in those with CKD, although consideration for several items is warranted. The high ceiling effect may be a problem when using the 13-item Patient Activation Measure to measure changes over time.

PIH38 Performance of Proxy-reported EQ-5D Youth Version 5-Level (EQ-5D-Y-5L) in Comparison with Three-Level (EQ-5D-Y-3L) in Chinese Patients with Adolescent Idiopathic Scoliosis

Adolescent idiopathic scoliosis (AIS) has significant influence on patients' health-related quality of life (HRQoL). Three-level and five-level EQ-5D-Y (hereafter named Y-3L and Y-5L) are validated utility instruments to assess HRQoL but psychometric properties of the proxy versions remain unknown. This study aims to assess the performance of the proxy-reported Y-5L compared with Y-3L for assessing AIS patients. 129 AIS patient-proxy dyads were recruited from a hospital in Hong Kong, China. Patients' caregivers were asked to complete proxy-reported Y-3L and Y-5L. All participants' sociodemographic and patients' clinical characteristics were collected. We assessed redistribution property and ceiling effects from Y-3L to Y-5L. Subgroup analysis was also conducted, where chi-squared test was used to detect differences in proportion of reporting problems. Ability of 'full health' defined by Y-3L and Y-5L to predict clinical outcomes was examined using area under curve (AUC). Mean age of patients was 13.3±1.4 years, 90.7% were female. Proxies were mostly mothers (71.1%) and only 28.6% received tertiary education or above. Consistencies between Y-3L and Y-5L responses were high for all dimensions (93.8-99.2%). All dimensions showed high ceiling effect (Y-3L:77.5-97.7%; Y-5L:77.5-96.9%) while largest reduction was observed in 'Doing usual activities' (absolute reduction: 2.3%; relative reduction: 2.5%) and 'Feeling worried/sad/unhappy' (absolute reduction: 2.3%; relative reduction: 2.9%). In 'Doing usual activities' dimension, less patients treated with bracing reported full-health status compared to those under observation in Y-3L (P-value: 0.08) and Y-5L (P-value: 0.034), proxies with secondary school or below education and those as mothers (P-value: 0.04 and 0.036 respectively) were more likely to report problems. In AUS analysis, 'full health' defined by Y-3L and Y-5L was not found to predict clinical outcomes. The overall performance of proxy-reported Y-3L and Y-5L in AIS patients was appeared to be similar. However, Y-5L is potentially more discriminative among mothers and proxies with lower education levels.

Validity and Reliability of the German Version of the Locomotor Capabilities Index-5 (LCI-5).

Before foreign language questionnaires, such as patient reported outcome measures (PROMs), can be scientifically used in the national language, guideline-based translation, cultural adaptation and comprehensive verification of their validity and reliability are needed. The aim of this study was to assess the psychometric properties of the German version of the LCI-5 (LCI-5-D) on a sample of people with lower limb amputation (LLA). The LCI-5 was translated into German (LCI-5-D) based on the ISPOR guidelines for cross-cultural adaptation following pilot testing. The final LCI-5-D was administered to n = 52 lower limb amputees in order to determine the validity and reliability of both the total score and the subscores ("basic items" and "advanced items"). Internal consistency (Cronbach's alpha), test-retest reliability (ICC2,1), standard error of the mean (SEM), minimal detectable change (MDC), item-to-total correlation, and item-to-subscale correlation were calculated. Floor and ceiling effects were checked. For construct validity, the Rivermead Mobility Index (RMI) and timed up and go (TUG) test were correlated, and differences of defined subgroups (amputation height and prosthesis user type) were calculated. On average, the score of the LCI-5-D was 40.13 ± 16.64 points. High ceiling effects were present for the subscale "basic items" (57.7% maximum score). Internal consistency showed excellent results (Cronbach's α = 0.97). A strong correlation to the RMI (r = 0.863; p < 0.001) and to the TUG (r = - 0.714; p < 0.001) demonstrated construct validity. Test-rest reliability was measured after an averaged time span of 5.3 ± 2.47 days and resulted in very high reliability [ICC (2,1) = 0.97; p < 0.001]. MDC was 7.57 points for the total score. The LCI-5-D is a valid and reliable PROM for measuring mobility with prostheses in German-speaking lower limb amputees, which is rather suitable for people with low to moderate mobility abilities. It can be used in a German-speaking population.

Unicompartmental knee arthroplasty: the Italian version of the Forgotten Joint Score-12 is valid and reliable to assess prosthesis awareness.

Unicompartmental Knee Arthroplasty (UKA) recorded an increased incidence of around 30% per year in the United States. Patient's experience and satisfaction after surgery were traditionally assessed by pre, and post-surgical scores and Patient-Reported Outcome Measures (PROMs) scales. Traditional scales as Western Ontario and McMaster University Osteoarthritis Index (WOMAC) and Oxford Knee Score (OKS) reported high ceiling effect. Patients treated by UKA usually perform well; therefore, it is necessary to have a PROMs' scale with a low ceiling effect as the Forgotten Joint Score-12 (FJS-12). PROMs have to be validated in the local language to be used. This study aims to perform a psychometric validation of the Italian version of FJS-12 for UKA for the first time. Between January 2019 and October 2019, 44 patients were included. Each patient completed both the FJS-12 Italian version and the WOMAC Italian version in preoperative follow-up, after 2-week and 1-month, 3-month, and 6-month postoperative follow-up. Cronbach's α, intraclass correlation coefficient (ICC), standard error of measurement (SEM), and minimal detectable change (MDC) were calculated to evaluate the reliability. The Pearson coefficient was used to assess validity. The Effect Size (ES) was used to test the responsiveness. A range of Cronbach's α between 0.90 and 0.95 indicated good internal consistency for the FJS-12. The test-retest reliability was acceptable (i.e., the ICC was higher than 0.7) at each follow-up. The Pearson correlation coefficient between the FJS-12 and WOMAC was - 0.11 (n.s.) at preoperative follow-up, r = 0.47 (P = 0.001) at 1 month, r = 0.57 (P < 0.001) at 3 months, and r = 0.57 (P < 0.001) at 6 months. Therefore, except for the preoperative period, the validity of the FJS-12 score was assessed. The FJS-12 represents a valid and reliable tool with a low ceiling effect to assess the outcomes improvement in UKA patients. Therefore, validating and translating this score in different languages could help perform more accurate studies on outcomes after UKA. Level III, diagnostic study.

Inter-specialty variation of the Press Ganey Outpatient Medical Practice Survey.

Measuring patient satisfaction scores and interpreting factors that impact their variation is of importance as scores influence various aspects of health care administration. Our objective was to evaluate if Press Ganey scores differ between medical specialties.New patient visits between January 2014 and December 2016 at a single tertiary academic center were included in this study. Press Ganey scores were compared between specialties using a multivariable logistic mixed effects model. Secondary outcomes included a comparison between surgical versus non-surgical specialties, and pediatric versus adult specialties. Due to the survey's high ceiling effect, satisfaction was defined as a perfect total score.Forty four thousand four hundred ninety six patients met inclusion criteria. Compared to internal medicine, plastic surgery, general surgery, dermatology, and family medicine were more likely to achieve a perfect overall score, as, with odds ratios of 1.46 (P = .02), 1.29 (P = .002), 1.22 (P = .004), and 1.16 (P = .02) respectively. Orthopaedics, pediatric medicine, pediatric neurology, neurology, and pain management were less likely to achieve satisfaction with odds ratios of 0.85 (P = .047), 0.71 (P < .001), 0.63 (P = .005), 0.57 (P < .001), and 0.51 (P = .006), respectively. Compared to pediatric specialties, adult specialties were more likely to achieve satisfaction (OR 1.73; P < .001). There were no significant differences between surgical versus non-surgical specialties.Press Ganey scores systematically differ between specialties within the studied institution. These differences should be considered by healthcare systems that use patient satisfaction data to modify provider reimbursement.