The CONVINCE randomized trial found positive effects on quality of life for patients with chronic kidney disease treated with hemodiafiltration.

In the CONVINCE trial, the primary analysis demonstrated a survival benefit for patients receiving high-dose hemodiafiltration (HDF) as compared with high-flux hemodialysis (HD). A secondary objective was to evaluate effects on health-related quality of life (HRQoL); assessed in eight domains (physical function, cognitive function, fatigue, sleep disturbance, anxiety, depression, pain interference, social participation) applying instruments from the Patient-Reported Outcome Measurement Information System (PROMIS) before randomization and every three months thereafter. In total 1360 adults with dialysis-dependent chronic kidney disease, eligible to receive high-flux HDF (23 liters or more), were randomized (1:1); 84% response rate to all questionnaires. Both groups reported a continuous deterioration in all HRQoL domains. Overall, raw score changes from baseline were more favorable in the HDF group, resulting in a significant omnibus test after a median observation period of 30 months. Most relevant single raw score differences were reported for cognitive function. Patients receiving HDF reported a decline of -0.95 units (95% confidence interval - 2.23 to +0.34) whereas HD treated patients declined by -3.90 units (-5.28 to - 2.52). A joint model, adjusted for mortality differences, utilizing all quarterly assessments, identified a significantly slower HRQoL decline in physical function, cognitive function, pain interference, and social participation for the HDF group. Their physical health summary score declined -0.46 units/year slower compared to the HD group. Thus, the CONVINCE trial showed a beneficial effect of high-dose hemodiafiltration for survival as well as a moderate positive effect on patients' quality of life, most pronounced with respect to their cognitive function. REGISTRATION: NTR7138 on the International Clinical Trials Registry Platform.

Using a measurement type-independent metric to compare patterns of determinants between patient-reported versus performance-based physical function in hemodialysis patients.

PURPOSE: We applied a previously established common T-score metric for patient-reported and performance-based physical function (PF), offering the unique opportunity to directly compare measurement type-specific patterns of associations with potential laboratory-based, psychosocial, sociodemographic, and health-related determinants in hemodialysis patients. METHODS: We analyzed baseline data from the CONVINCE trial (N = 1,360), a multinational randomized controlled trial comparing high-flux hemodialysis with high-dose hemodiafiltration. To explore the associations of potential determinants with performance-based versus patient-reported PF, we conducted multiple linear regression (backward elimination with cross-validation and Lasso regression). We used standardized T-scores as estimated from the PROMIS PF short-form 4a (patient-reported PF) and the Physical Performance Test (performance-based PF) as dependent variables. RESULTS: Performance-based and patient-reported PF were both significantly associated with a laboratory marker-based indicator of muscle mass (simplified creatinine index), although the effects were relatively small (partial f2 = 0.04). Age was negatively associated with PF; the effect size was larger for performance-based (partial f2 = 0.12) than for patient-reported PF (partial f2 = 0.08). Compared to performance-based PF, patient-reported PF showed a stronger association with self-reported health domains, particularly pain interference and fatigue. When using the individual difference between patient-reported and performance-based T-scores as outcome, we found that younger age and more fatigue were associated with lower patient-reported PF compared to performance-based PF (small effect size). CONCLUSION: Patient-reported and performance-based assessments were similarly associated with an objective marker of physical impairment in hemodialysis patients. Age and fatigue may result in discrepancies when comparing performance-based and patient-reported scores on the common PF scale. Trial Registration CONVINCE is registered in the Dutch Trial Register (Register ID: NL64750.041.18). The registration can be accessed at: https://onderzoekmetmensen.nl/en/trial/52958 .

Frequently Used Patient-Reported Outcome Measures of General Physical Function Were Highly Correlated With a Multitask Performance Outcome Test Battery.

OBJECTIVES: This study aimed to determine the relationship between frequently used patient-reported outcome (PRO) measures and a multitask performance outcome (PerfO) measure of general physical function (PF) and to examine the association of these measures with depressive mood, pain, and age. METHODS: Frequently used PRO measures of general PF (Patient-Reported Outcomes Measurement Information System [PROMIS] PF item bank, PROMIS PF Short Form 20a, Short Form 36 Physical Function Scale) and a PerfO test battery, namely, the Physical Performance Test (PPT), were administered to 78 adult patients from 3 inpatient clinics (cardiology and angiology, rheumatology and clinical immunology, and psychosomatic medicine) at Charité - Universitätsmedizin Berlin. Pearson correlations were used to investigate the associations between PRO measures and the PPT. To explore the predictive value of age, depressive symptoms, and pain intensity, we conducted multiple linear regression analysis for each PF measure. RESULTS: We found strong linear relationships between PRO measures and PPT sum scores. Correlations between PPT sum scores and PROMIS PF T-scores were r > 0.75. For all PRO and PerfO measures, age was a predictor of general PF whereas depressive mood was not found to be a relevant predictor. Moreover, pain intensity was found to be a significant predictor of PRO measures but not for PPT sum scores. CONCLUSIONS: Our findings suggest that frequently used PRO measures and a multitask PerfO measure of general PF can be used to measure a common PF construct. Nevertheless, PF scores based on PRO measures should ideally be controlled for self-rated pain intensity.

[Empirical Assessment of Patient-Reported Outcomes and Exemplary Introduction to Health-Related Quality of Life]. / Empirische Erfassung subjektiver Gesundheitsmerkmale am Beispiel der gesundheitsbezogenen Lebensqualität.

Health-related quality of life (HRQoL) acquires increased importance as a target parameter for different stakeholders in healthcare, e. g. to assess treatment outcome in chronically ill patients. In this educational article, we explain the levels of the health-related quality of life construct and associated main dimensions including physical, mental and social health. State-of-the-art approaches for assessment of patient-reported outcomes (PROs) are introduced on the basis of the HRQoL model. Furthermore, modern test-theoretical approaches and their applications are presented, which are aimed at standardizing and improving the assessment of PROs (e. g., computer-adaptive testing). Finally, we present 2 major international PRO initiatives (PROMIS® and EORTC Quality of Life Group) that have been influential in advancing the assessment of patient outcomes over the last few years.

Development and Psychometric Validation of the 27 Item Zarit Caregiver Interview for Alzheimer's Disease (ZCI-AD-27).

BACKGROUND: Caring for an individual with Alzheimer's disease (AD) is an allencompassing challenge that affects daily life. Assessment of the care partner experience is needed to support the development and evaluation of successful interventions for people with AD and their care partners. We developed the 27-item Zarit Caregiver Interview for Alzheimer's Disease (ZCI-AD-27) to assess the impact of informal caregiving in the context of AD. OBJECTIVE: We assessed the psychometric validity of the ZCI-AD-27 in a population of care partners for individuals with moderate AD, and established thresholds for meaningful score change. METHODS: Secondary data were obtained from informal care partners of participants in a clinical trial (NCT01677754). Psychometric analyses were conducted to assess validity, reliability, and responsiveness of the ZCI-AD-27. Anchor-based and distribution-based methods were performed to determine clinically meaningful score change. RESULTS: The ZCI-AD-27 had a 12-domain factor structure, including a second-order domain termed Humanistic impact that included four key domains (Physical, Emotional, Social, and Daily life) as confirmed by confirmatory factor analysis with the adequate fit. Internal consistency (Cronbach's alpha ranging from 0.66 to 0.93 for domains), convergent validity, and discriminant validity indicated the good performance of the ZCI-AD-27. Known-groups validity analyses showed a greater impact on care partners with increasing disease severity. Responsiveness results demonstrated that the ZCI-AD- 27 is sensitive to change over time and meaningful change analyses indicated a range of meaningful score changes in this population. CONCLUSION: The ZCI-AD-27 is a comprehensive, psychometrically valid measure to assess the impact of caring for individuals with moderate AD.

Physical performance tasks were linked to the PROMIS physical function metric in patients undergoing hemodialysis.

OBJECTIVES: To investigate whether a multi-item performance outcome measure, the physical performance test (PPT), can be calibrated to a common scale with patient-reported outcome measures, using the Patient-Reported Outcomes Measurement Information System (PROMIS) physical function (PF) metric. STUDY DESIGN AND SETTING: We analyzed baseline data (N = 1,113) from the CONVINCE study, an international trial in end-stage kidney disease patients comparing high-dose hemodiafiltration with high-flux hemodialysis. Assumptions of item response theory (IRT) modelling were investigated for the combined set of the nine-item PPT and a four-item PROMIS PF short form (PROMIS-PF4a). We applied unidimensional IRT linking for calibrating the PPT to the PROMIS PF metric. RESULTS: Although some evidence for multidimensionality was found, classical test statistics (Cronbach's Alpha = 0.93), Mokken (Loevinger's H = 0.50), and bifactor analysis (explained common variance = 0.65) indicated that PPT and PROMIS-PF4a items can be used to assess a common PF construct. On the group level, the agreement between PROMIS-PF4a and linked PPT scores was stable across several subsamples. On the individual level, scores differed considerably. CONCLUSION: We found preliminary evidence that the PPT can be linked to the PROMIS PF metric in hemodialysis patients, enabling group comparisons across patient-reported outcome and performance outcome measures. Alternative linking methods should be applied in future studies using a more comprehensive PROMIS PF item set.

Patient-reported outcomes feedback report for knee arthroplasty patients should present selective information in a simple design - findings of a qualitative study.

BACKGROUND: Technical innovation to assess patient-reported outcomes (PROs) facilitates their implementation in clinical practice. In particular, mobile applications (apps) allow PROs to be assessed outside of the clinical setting. A patient's health status can be remotely monitored and evaluated after discharge, and their recovery process tracked. This is of particular interest for patients after knee arthroplasty, as the recovery phase after surgery usually takes place in an outpatient setting and requires a high level of patient engagement. Providing results of PRO assessments to patients in the form of a feedback report could increase patient engagement and may improve communication between health care professionals and patients. The aim of the study is to develop a PRO feedback report for mobile devices that is comprehensible and provides valuable information for patients after knee arthroplasty. RESULTS: In an iterative development process, our expert group developed two preliminary feedback reports (a text-based version and a graphical display) based on previous research results and practical experience. In a second step, we discussed these reports with orthopedic patients (n = 8) in terms of comprehensibility and value using semi-structured interviews and cognitive debriefing methods. Participants assessed the reports as informative, but had some difficulties in fully comprehending all of the information provided. Based on the feedback from patients, we modified both versions and reduced complexity to increase comprehensibility. CONCLUSIONS: A PRO feedback report for patients for mobile app use has to take account of the heterogeneous user group, particularly demographics such as age and experience with mobile devices. Information should be presented in a simple way to be comprehensible and of value to patients. Technological advancements allow a simple default report to be set, something which enables patients interested in additional information to make customizations.

Benefits and harms of high-dose haemodiafiltration versus high-flux haemodialysis: the comparison of high-dose haemodiafiltration with high-flux haemodialysis (CONVINCE) trial protocol.

INTRODUCTION: End-stage kidney disease (ESKD) is a major public health problem affecting more than 2 million people worldwide. It is one of the most severe chronic non-communicable diseases. Haemodialysis (HD) is the most common therapeutic option but is also associated with a risk of cardiovascular events, hospitalisation and suboptimal quality of life. Over the past decades, haemodiafiltration (HDF) has become available. Although high-dose HDF has shown some promising survival advantage compared to conventional HD, the evidence remains controversial. A Cochrane systematic review found, in low-quality trials, with various convective forms of dialysis, a reduction in cardiovascular, but not all-cause mortality and the effects on non-fatal cardiovascular events and hospitalisation were uncertain. In contrast, an individual patient data analysis suggested that high-dose HDF reduced both all-cause and cardiovascular mortality compared to HD. In view of these discrepant results, a definitive trial is required to determine whether high-dose HDF is preferable to high-flux HD. The comparison of high-dose HDF with high-flux HD (CONVINCE) study will assess the benefits and harms of high-dose HDF versus a conventional high-flux HD in adults with ESKD. METHODS AND ANALYSIS: This international, prospective, open label, randomised controlled trial aims to recruit 1800 ESKD adults treated with HD in nine European countries. Patients will be randomised 1:1 to high-dose HDF versus continuation of conventional high-flux HD. The primary outcome will be all-cause mortality at 3 years' follow-up. Secondary outcomes will include cause-specific mortality, cardiovascular events, all-cause and infection-related hospitalisations, patient-reported outcomes (eg, health-related quality of life) and cost-effectiveness. ETHICS AND DISSEMINATION: The CONVINCE study will address the question of benefits and harms of high-dose HDF compared to high-flux HD for kidney replacement therapy in patients with ESKD with a focus on survival, patient perspectives and cost-effectiveness. TRIAL REGISTRATION NUMBER: Netherlands National Trial Register (NTR 7138).

Minimal Associations Between Clinical Data and Children's Self-Reported Health-Related Quality of Life in Children With Chronic Conditions-A Cross-Sectional Study.

Introduction: The improvement-or at least maintenance-of health-related quality of life (HRQoL) in children and adolescents is one of the main aims of chronic disease care. This study examines HRQoL of children and adolescents with three different chronic conditions (i.e., diabetes mellitus, asthma, juvenile arthritis) using the computer-adaptive test Kids-CAT, comprising five HRQoL domains: physical well-being, psychological well-being, parent relations, social support and peers, and school well-being. Further, associations between HRQoL and distinct clinical data and medical assessments are investigated to explore how much variability of the five domains can be explained by these variables. Methods: Cross-sectional data of the Kids-CAT study was analyzed. The Kids-CAT was used in two outpatient clinics in northern Germany gathering data on self-reported HRQoL in n = 309 children and adolescents aged 7-17 years. Additionally, general patient information, clinical data, and pediatrician-reported medical assessments were measured. Multiple regression analyses were conducted to explore associations between HRQoL and selected variables (i.e., disease duration, co-morbidity, disease control, overall health status). Results: Overall, self-reported HRQoL in all five domains were comparable to data of an age- and sex-matched reference population. Results of regression analyses indicated that the investigated variables only minimally explain variance in the five Kids-CAT domains. Sociodemographic, clinical data, and medical assessments explained 18.4% of the variance in physical well-being, 10.7% in psychological well-being, and < 10% of the variance in parent relations, social support and peers, and school well-being. Conclusion: Sociodemographic data, disease duration, co-morbidity, and medical assessments, such as disease control or pediatrician-assessed overall health status show low association with HRQoL of children and adolescents with chronic conditions. Data on self-reported HRQoL delivers valuable information on children's well-being and can improve healthcare professionals' understanding of the subjective well-being of their young patients. The implementation of tools like the Kids-CAT can facilitate the identification of potential problem areas, which should enable healthcare professionals to better address specific healthcare needs. Clinical Trial Registration: identifier: DRKS00006326 (retrospectively registered); Date of registry: August 1st, 2014.

Trajectories of Health-Related Quality of Life and HbA1c Values of Children and Adolescents With Diabetes Mellitus Type 1 Over 6 Months: A Longitudinal Observational Study.

Introduction: To achieve optimized blood glucose concentrations (assessed by HbA1c) and high health-related quality of life (HRQL), children and adolescents with diabetes mellitus type 1 (T1DM) must follow strict disease management strategies. This study aims to investigate HRQL of children and adolescents with T1DM and its association with HbA1c values over the course of 6 months. Methods: Patients aged 7-17 years (n = 203) with T1DM provided HRQL data on a monthly basis. HRQL was measured using the Kids-CAT, a computer-adaptive test (CAT) comprising five generic HRQL domains. HbA1c concentrations were assessed at baseline, at 3 and 6 months. We explored the trajectory of HRQL at the domain level using linear mixed effects models. Further, we investigated the association between HRQL and HbA1c concentrations over time using path analysis models. Results: Children and adolescents with T1DM reported high scores across all HRQL domains over time. However, those with an HbA1c concentrations of >9.0% reported significantly lower scores in physical well-being and parent relations compared with those with an HbA1c concentration of <7.5%. Path analysis models revealed a minimal temporal relationship between HbA1c and HRQL, with a small negative impact of HbA1c on physical well-being, psychological well-being and parent relations. Conclusion: Although observed HRQL of young patients with T1DM was comparable to age-related German-speaking reference population over the course of 6 months, those with an HbA1c concentration >9.0% reported lower scores in selected HRQL domains. Thus, special attention should be drawn to HRQL of children and adolescents with higher HbA1c concentrations. The minimal relationship between HbA1c and HRQL indicates that the two therapy goals, i.e., achievement and maintenance of glycemic targets and high HRQL, should be considered and evaluated independently in clinical routine. Trial Registration: DRKS00006326 (German Clinical Trial Register), date of registration: August 1st, 2014.