Disease-specific Health-related Quality Of Life Measure Research Articles

A comprehensive systematic review of health-related quality of life measures in short stature paediatric patients

This systematic review investigates Patient-reported Outcome Measures (PROMs) and Observed Reported Outcome Measures (ObsROMs) pertinent to assessing Health-Related Quality of Life (HRQoL) in short-stature paediatric patients, focusing on Achondroplasia (ACH), Growth Hormone Deficiency (GHD), Isolated Growth Hormone Deficiency (IGHD), and Small-for-Gestational-Age (SGA) diagnoses. Utilising rigorous selection criteria, 53 studies published from 1998 to 2023 were analysed, revealing a predominance of European-based research. Notably, the review elucidated the utilisation of disease-specific and generic HRQoL measures, showcasing the multifaceted nature of short-stature conditions and their impact across physical, emotional, and social domains. The Quality of Life in Short Stature Youth (QoLISSY), Paediatric Quality of Life Inventory (PedsQL), and KIDSCREEN emerged as frequently employed instruments, offering nuanced insights into HRQoL perceptions across diverse age demographics. Additionally, the review highlighted the adaptation of adult HRQoL measures for adolescent populations, signalling a need for age-appropriate assessment tools. Furthermore, integrating PROMs and ObsROMs in HRQoL assessment underscored a comprehensive approach, considering both subjective patient perspectives and observed outcomes. Future research directions encompass comprehensive search strategies, longitudinal studies with diverse populations, and the development of age-appropriate HRQoL assessment tools. In conclusion, this review emphasises the importance of comprehensive HRQoL assessment to address the diverse needs of short-stature paediatric patients effectively.

Integrating the assessment of quality of life in care and research in pulmonary fibrosis.

Pulmonary fibrosis (PF) negatively influences health-related quality of life (HRQOL). Patients living with PF have voiced the desire for a focus on symptoms and HRQOL in both disease monitoring and treatment decisions. Currently available disease modifying treatments do little to impact HRQOL. Newer studies evaluating pharmacologic and nonpharmacologic therapies targeting symptoms and HRQOL in PF have been conducted with some promising results. There is increasing recognition of the importance of incorporating HRQOL as a higher tier endpoint in clinical trials. Disease-specific measure of HRQOL have been developed for those living with PF, and there is ongoing work to better understand the validity and reliability characteristics of these tools. In addition to research, there is recognition of the potential benefits of measuring HRQOL and symptoms in clinical practice in facilitate integrating patient perspective into care and allow for more personalized treatment approaches. There is increased momentum to discover treatments that impact HRQOL in PF. More work is desperately needed to identify better treatment targets, and to incorporate HRQOL and symptoms as higher tier endpoints in clinical trials. Further work is also needed to address the practicalities of integrating HRQOL measurement into clinical care.

Raising the standards of patient-centered outcomes research in myelodysplastic syndromes: Clinical utility and validation of the subscales of the QUALMS from the MDS-RIGHT project.

Clinical decision-making for patients with myelodysplastic syndromes (MDS) is challenging, and both disease and treatment effects heavily impact health-related quality of life (HRQoL) of these patients. Therefore, disease-specific HRQoL measures can be critical to harness the patient voice in MDS research. We report a prospective international validation study of the Quality of Life in Myelodysplasia Scale (QUALMS) with a main focus on providing information on the psychometric characteristics of its three subscales: physical burden (QUALMS-P), emotional burden (QUALMS-E), and benefit finding (QUALMS-BF). The analysis is based on patients enrolled from three European countries and Israel, participating to the MDS-RIGHT Project. The scale structure and psychometric properties of the QUALMS were assessed. Overall, 270 patients with a median age of 74 years were analyzed and the majority of them (60.3%) had a low MDS-Comorbidity Index score. Results of the confirmatory factor analysis supported the underlying scale structure of the QUALMS, which, in addition to a total score, includes three subscales: QUALMS-P, QUALMS-E, and the QUALMS-BF. The QUALMS-P exhibited the highest Cronbach's alpha coefficients. Discriminant validity analysis indicated good results with the QUALMS-P and QUALMS-E distinguishing between patients with different performance status, comorbidity, anemia, and transfusion dependency status. No floor and ceiling effects were observed. Responsiveness to change analysis supported the validity of the measure. Patients with a hemoglobin (Hb) level of <11 g/dL at study entry, who subsequently showed an improvement in their Hb levels, also reported a mean score change of 9 and 8 points (scales ranging between 0 and 100) in the expected direction of the QUALMS-E and QUALMS-P, respectively. Our study provides additional validation data on the QUALMS from the international MDS-RIGHT Project. The use of this disease-specific HRQoL measure may contribute to raise quality standards of patient-centered outcomes research in MDS.

S403 Dupilumab Reduces the Emotional and Dysphagia-Related Impacts of Eosinophilic Esophagitis to Improve Health-Related Quality of Life

Introduction: Eosinophilic esophagitis (EoE) is a chronic, type 2 inflammatory disease of the esophagus which substantially impairs quality of life (QoL). Dupilumab, a fully human mAb, blocks the shared receptor component for IL-4/IL-13, key drivers of type 2 inflammation in EoE. In the 3-part LIBERTY-EoE-TREET phase 3 trial (NCT03633617), Parts A and B demonstrated that weekly (qw) dupilumab 300mg improved clinical, symptomatic, histologic, and endoscopic aspects of EoE and was generally well tolerated in patients (pts) ≥12 years at Wk 24. The objective of this post-hoc analysis was to assess the effect of dupilumab vs placebo on improving aspects of QoL using the EoE Impact Questionnaire (EoE-IQ), a novel disease-specific measure of health-related QoL in EoE patients, during the 24-wk double-blind treatment period in Parts A and B. Methods: In Part A, 42 pts received dupilumab 300mg qw, and 39 received placebo. In Part B, 80 pts received dupilumab 300mg qw, and 79 received placebo for 24 wks. The EoE-IQ is a patient-reported 11-item questionnaire that measures the impact of EoE on emotional, social, work and school, and sleep aspects of a patient (over a 7-day recall period). It was developed in line with best practices in the field of clinical outcomes assessment and includes the most relevant QoL impacts of EoE as identified through literature review and discussion with therapeutic area experts and confirmed via patient interviews. Response to each item is on a 5-point scale ranging from 1-5 with a higher score indicating a more negative impact on QoL. Results: The most burdensome impacts of EoE at baseline were related to overall emotional impact and anxiety around dysphagia (Table). Dupilumab showed a nominally significant reduction vs placebo in 6 items in Parts A and B: ‘bothered’, ‘worried about swallowing’, ‘worried about choking’, ‘worried about swallowing in public’, ‘social activities’, ‘sleep disruption’ (all nominal p< 0.05), and in 1 additional item in Part B (‘embarrassed’, nominal p< 0.05). The largest placebo adjusted change from baseline to Wk 24 was observed in items relating to overall emotional impact and anxiety, including ‘worried about swallowing in public’ (Part A: –0.90; Part B: –0.45), ‘bothered’ (–0.64; –0.53), ‘worried about swallowing’ (–0.73; –0.65), and ‘worried about choking’ (–0.61; –0.57). Conclusion: EoE QoL was improved among pts on dupilumab 300mg qw, with improvement driven by emotional and social impacts, and sleep. Table 1. - EoE-IQ at baseline and after 24 weeks treatment with weekly dupilumab 300mg or placebo. Values after first rescue treatment use were set to missing (censoring), then multiple imputation was used to impute missing values. Mean absolute value at baseline Mean absolute value at Week 24 EoE-IQ Item Dupilumab300 mg qw Dupilumab 300 mg qw LS Mean Difference vs placebo P value Part A 1. Bothered 3.08 1.80 –0.64 0.0043 2. Worried swallowing 2.88 1.69 –0.73 0.0011 3. Worried choking 2.42 1.60 –0.61 0.0040 4. Embarrassed 1.91 1.34 –0.21 0.2336 5. Worried swallowing public 2.80 1.46 –0.90 0.0001 6. Social activities 2.06 1.40 –0.43 0.0311 7. Family 1.44 1.29 –0.20 0.2560 8. Friends 1.39 1.28 –0.31 0.0813 9. Keep up work/school 1.48 1.30 –0.28 0.1379 10. Miss work/school 1.37 1.34 –0.09 0.5504 11. Sleep disruption 1.82 1.21 –0.42 0.0027 Part B 1. Bothered 3.50 1.97 –0.53 0.0004 2. Worried swallowing 3.10 1.60 –0.65 < 0.0001 3. Worried choking 2.72 1.45 –0.57 < 0.0001 4. Embarrassed 2.21 1.28 –0.28 0.0141 5. Worried swallowing public 2.96 1.53 –0.45 0.0020 6. Social activities 2.34 1.35 –0.30 0.0117 7. Family 1.72 1.22 0.01 0.8765 8. Friends 1.68 1.19 –0.06 0.4409 9. Keep up work/school 1.81 1.32 –0.08 0.4170 10. Miss work/school 1.32 1.18 –0.09 0.3390 11. Sleep disruption 1.94 1.36 –0.22 0.0409 EoE-IQ, Eosinophilic Esophagitis Impact Questionnaire; LS, least squares; qw, weekly.

Estimating the association between physical activity and health-related quality of life in individuals with multiple sclerosis

BackgroundHigher levels of total physical activity (PA) are associated with better health-related quality of life (HRQOL) in individuals with multiple sclerosis (MS). The benefits of PA across the activity continuum have not been well-studied. The goal of this study was to compare the associations between total PA, strenuous PA, moderate PA, and mild PA and HRQOL in a large cohort of individuals with MS using both generic and neurologic disease-specific questionnaires. Longitudinal changes in PA and HRQOL over two years were also examined MethodsSubjects enrolled in SysteMS completed the Godin Leisure Time Exercise Questionnaire (GLTEQ) to measure PA. Subjects also completed generic (SF-36) and neurologic disease-specific (NeuroQoL) HRQOL measures. GLTEQ and HRQOL measures were administered at baseline and 24 months. The associations between the GLTEQ total leisure activity (TLA), strenuous PA, moderate PA and mild PA and scores on NeuroQoL and SF-36 were estimated using Spearman's correlation coefficient and partial Spearman's correlation coefficient adjusting for age, sex and Expanded Disability Status Scale (EDSS) measured by a provider. To further investigate the associations between mild PA and HRQOL measures, the associations between mild PA and HRQOL were estimated in participants who reported no moderate or strenuous PA in the last week. The changes in GLTEQ TLA scores and each component score were compared to the changes in NeuroQoL and SF-36 over 24 months using Spearman's correlation coefficient and partial Spearman's correlation coefficient adjusting for age, sex and EDSS. ResultsStatistically significant weak correlations were observed between GLTEQ TLA and NeuroQoL and SF-36 domains, with higher levels of TLA being associated with better HRQOL outcomes. After adjusting for age, sex and EDSS, all correlations were attenuated. Strenuous and moderate levels of PA were similarly associated with many HRQOL outcomes, but mild PA was only weakly correlated with NeuroQoL Lower Extremity Function. There was limited change in PA over 24 months. In a subgroup of participants who reported mild PA, but no moderate or strenuous PA, there were no significant associations with NeuroQoL or SF-36 domains at baseline, but increases in mild PA over two years were moderately associated with improvement on NeuroQoL Upper Extremity Function and SF-36 Mental Health and Mental Component Summary. ConclusionThere were weak associations between TLA and HRQOL across a wide range of HRQOL variables. In addition, both strenuous PA and moderate PA were weakly associated with many HRQOL outcomes, but mild PA was only associated with lower extremity function. Increases in mild PA in a subgroup of individuals who reported no strenuous or moderate PA at baseline were associated with improvements in HRQOL at 24 months. These findings suggest that programs aimed at increasing PA across the activity continuum may lead to improvements in multiple areas of HRQOL in individuals with MS.

Evaluation and validation of a patient-reported quality-of-life questionnaire for Parkinson\u2019s disease

BackgroundParkinson’s disease (PD) is a chronic, progressive illness with a profound impact on the health-related quality of life (HRQoL). Disease-specific patient-reported HRQoL measures, such as PDQ-39 and its short version PDQ-8, are increasingly used in clinical practice to address the consequences of PD on everyday life. Due to limitations in the content, especially in non-motor symptoms and sleep disturbances of PDQ-8, PDQoL7, a 7-item, short-term, self-reported, PD-specific HRQoL questionnaire was developed.MethodsA representative sample of 60 adults with idiopathic PD completed the PDQoL7 questionnaire and the existing validated PDQ-8 and EQ-5D-5L questionnaires (all in Greek).ResultsPDQoL7 summary index strongly correlated with PDQ-8 (rs = 0.833, P < 0.001) and EQ-5D-5L (rs = − 0.852, P < 0.001). The correlation between PDQoL7 and EQ-5D-5L was statistically significantly stronger compared to PDQ-8 and EQ-5D-5L (rs = − 0.852 vs rs = − 0.789 respectively, P < 0.001). The internal consistency of PDQoL7 was not affected by item deletion (positive item to total correlations: 0.29–0.63). No redundant items (with inter-item correlation coefficients greater than 0.80) were identified. Cronbach’s α for PDQoL7 was comparable to PDQ-8 (0.804 versus 0.799 respectively). As PDQoL7 had three-dimensional structure, omega coefficient analysis confirmed its reliability (omega total: 0.88; omega hierarchical: 0.58).ConclusionsPDQoL7 is an acceptable, easy to use, valid and reliable tool for the determination of HRQoL in PD patients that is potentially more comprehensive than PDQ-8 based on the available evidence. PDQoL7 could allow for a more thorough evaluation of the impact of PD and contribute to guiding healthcare decisions. This will be confirmed in subsequent analysis on larger patient cohorts.

Validation of the Qualms Questionnaire to Assess Health-Related Quality of Life in European and Israeli Patients with Myelodysplastic Syndromes: Results from the MDS-Right Project

Validation of the Qualms Questionnaire to Assess Health-Related Quality of Life in European and Israeli Patients with Myelodysplastic Syndromes: Results from the MDS-Right Project

Understanding and Measuring Key Symptoms and Health-Related Quality of Life in Patients with Chronic Immune Thrombocytopenia

Introduction Immune thrombocytopenia (ITP) is a rare autoimmune disorder in which there is an increase in platelet destruction and a decrease in platelet production, resulting in low platelet count, high bleeding risk and impact on quality of life (QoL). A critical appraisal of the literature was conducted to understand the patient experience with ITP and its treatment, and to assess which clinical outcome assessment (COA) instruments can best capture the ITP patient experience and changes due to disease management. Methods A focused PubMed search was performed for qualitative studies describing patient experience with ITP published prior to June 2019. The search paired population terms (ITP) with qualitative study design terms ("qualitative", "focus group/s", "interview/s", "blog"). Grey literature (congresses, patient advocacy group websites) was also consulted. Key patient-reported concepts of interest for measurement in clinical trials were identified. A second review was conducted to identify COA instruments used in ITP to measure the key concepts using PROQOLID (no limits; prior to November 2019), Clinicaltrials.gov (Phase 2 and 3; November 2009-2019), and PubMed (October 2009-2019). COA instruments were critically appraised based on their published psychometric properties and use in regulatory labels and health technology assessment (HTA) appraisal decisions. Results Of the 52 articles screened in the initial search, 2 relevant articles were identified. One reported the development of ITP-patient assessment questionnaire (ITP-PAQ), a disease-specific measure of health-related QoL (HRQoL) in adults with ITP (Mathias et al. Health and Quality of Life Outcomes 2007); the second introduced a conceptual model informed by literature, clinical expertise and qualitative research to describe the impact of ITP and its treatment on patients' HRQoL (Mathias et al. Health and Quality of Life Outcomes 2008). This conceptual model proposed 1 biological variable (low platelet count), 2 main determinants of HRQoL (symptoms of ITP, treatment side-effects) and 5 conceptual domains of HRQoL (emotional health, functional health, social and leisure, reproductive health, work). Fatigue and bleeding/bruising symptoms were considered the main determinants of HRQoL change for patients with ITP. However, the literature supporting this conceptual model was over 15 years old, from one country (US) and conducted in one clinical setting (tertiary care). A third recent publication reporting an interim analysis from the ITP World Impact Survey in 1,400 patients was identified from grey literature (Cooper et al. EHA Library 2018; PF654). Fatigue and bleeding (including petechiae) were the most frequently reported symptoms, at diagnosis and at time of survey completion, and fatigue was reported as one of the most severe symptoms. Twenty-two COA instruments used in ITP were identified for assessments of bleeding/bruising, fatigue/tiredness, anxiety/fear, and/or avoidance of physical activity. From this search, 8 instruments were analysed in depth: ITP-PAQ; Functional Assessment of Cancer Therapy - Thrombocytopenia 6 (FACT-Th6); FACT-Th11/18; Functional Assessment of Chronic Illness - Fatigue (FACIT-F); WHO Bleeding Score; ITP Bleeding Score; ITP-specific bleeding assessment tool (ITP-BAT); Short Form 36 (SF-36). The WHO Bleeding Scale is widely used in ITP and mentioned in at least one product label for ITP treatments (eltrombopag). The FACIT-F is the most widely-used fatigue instrument and was utilized in non-ITP related regulatory labels in the US and EU. It has also been considered by HTA agencies for ITP and its psychometric properties were validated in ITP. The ITP-PAQ was developed and validated in ITP for HRQoL evaluation; it was used for clinical development of romiplostim and was well considered by the National Institute for Health and Care Excellence (NICE). Conclusion The studies identified in these literature searches indicate that fatigue and bleeding are key determinants of HRQoL in patients with ITP. Three COA instruments (WHO Bleeding scale, FACIT-F and ITP-PAQ) were identified using evidence from the existing literature as potential valid tools to measure these concepts of interest in future ITP clinical trials. Disclosures Joly: Sanofi: Current Employment, Current equity holder in publicly-traded company. Reaney:IQVIA: Current Employment; Sanofi: Research Funding. Daak:Sanofi: Current Employment, Current equity holder in publicly-traded company. Venerus:Sanofi: Research Funding; IQVIA: Current Employment. Roborel de Climens:Sanofi: Current Employment, Current equity holder in publicly-traded company.

Observational Study to Assess Quality of Life in Patients with Pancreatic Neuroendocrine Tumors Receiving Treatment with Everolimus: The OBLIQUE Study (UK Phase IV Trial)

Background/Aims: To assess health-related quality of life (HRQoL), treatment patterns, and clinical outcomes of adult (≥18 years) patients with advanced (unresectable or metastatic) pancreatic neuroendocrine neoplasms (PanNENs) treated with everolimus in routine clinical practice. Methods: In a prospective, non-interventional, multi-center study patients administered at least one 10 mg dose of everolimus were evaluated for change in HRQoL (EORTC QLQ-C30 Global Health Status scale) from baseline after 6 months treatment (primary endpoint). Secondary endpoints included disease-specific HRQoL measures (EORTC QLQ-G.I.NET21), clinical outcomes, everolimus treatment patterns, and safety. Results: Forty-eight patients were recruited (between August 2013 and March 2015); the median treatment duration was 27.8 months. EORTC QLQ-C30 Global Health score was not significantly different from baseline after 6 months of treatment (mean difference –1.9 points, p = 0.660, n = 30). In pairwise analyses, the only significant changes in HRQoL from baseline were for EORTC QLQ-C30 physical functioning score at month 3 (adjusted mean difference –8.8 points, p = 0.002, n = 36) and the EORTC QLQ-G.I.NET21 disease-related worries scores at months 1 and 2 (adjusted mean differences: –11.5 points [p = 0.001, n = 44] and –8.8 points [p = 0.017, n = 43], respectively). Disease progression or death was recorded in 44.4% (n = 20/45) patients during follow-up; median progression-free survival was 25.1 months and the cumulative survival rate at 3 years was 71%. No new safety signals were detected. Conclusions: The OBLIQUE study demonstrates that HRQoL is maintained in patients with PanNENs during treatment with everolimus in a UK real-world setting. This study adds to the limited HRQoL data available in this patient group.

Systematic Literature Review of the Cost-Effectiveness of Treatments, Costs/Resource Use, and Quality of Life in Patients with Mantle Cell Lymphoma

Systematic Literature Review of the Cost-Effectiveness of Treatments, Costs/Resource Use, and Quality of Life in Patients with Mantle Cell Lymphoma

Development of a patient-oriented disease specific outcome measure of health-related quality of life (HRQOL) for musculoskeletal oncology patients

BackgroundAccording to improved functional outcome and life expectancy in orthopaedic oncology patients, there has been a growing interest in not only oncologic and functional outcomes but also health-related quality of life (HRQOL), including body image, mental status, or social activities, after surgery. However, there has been a lack of disease-specific measures focusing on the ability of orthopaedic oncology patients to evaluate their HRQOL comprehensively. Therefore, our aims in the present study were 1) to develop a patient-oriented disease-specific outcome measure of HRQOL for musculoskeletal oncology patients (COMMON-LE), and 2) to examine the practical applicability, reliability and validity of the COMMON-LE for patients with musculoskeletal tumors in the lower extremity. MethodsThe COMMON-LE was developed by expert committee of orthopaedic oncology and rehabilitation. A total of 101 patients were surveyed using the COMMON-LE, as well as the TESS, the MSTS score, and the SF-36, to assess their psychometric characteristics, including reliability, validity, and responsiveness. ResultsThe COMMON-LE showed no marked floor and ceiling effects. Test-retest reliability and internal consistency determined using the intraclass correlation coefficient (0.928) and Cronbach's alpha (0.948–0.968), respectively, were excellent. Each domain of the COMMON-LE (pain, ADL, socioemotional condition and general health) was well correlated with the scores of the standard measures (SF-36, TESS, MSTS score). Factor analysis and the AIC network showed the questionnaire items of the COMMON-LE were clearly separable into three clusters according to their content, corresponding to each domain of the questionnaire. ConclusionsWe have successfully developed and validated a disease-specific measure, the COMMON-LE, to evaluate not only physical function, but also various aspects of HRQOL in patients with musculoskeletal tumors. The COMMON-LE has sufficient reliability and internal consistency, and good validity, and appears to be practically applicable to this group of patients.

Comparison of Effects of Endurance and Strength Training Programs in Patients with COPD

ABSTRACTComparisons between endurance and strength training in chronic obstructive pulmonary disease (COPD) patients have produced equivocal findings when examining physical function and health-related quality of life (HRQL). One reason for these differences may be due to individual patient responses to the different training modalities. PURPOSE: To compare changes in physical function and HRQL in a group of COPD patients completing both an endurance and a strength training program. METHODS: Eleven mildly diseased patients completed a three month endurance training program and, approximately 5 years later, completed a three month strength training program. Changes in 6 minute walk distance (6 MW), time to rise from a chair five times (CRT), and the total score and subscores from the SF-36 and Chronic Respiratory Disease Questionnaire (CRQ) were examined. RESULTS: The forced expiratory volume as a percent of predicted remained relatively constant over the 5 years (61.1 ± 5.9 vs. 60.0 ± 10.3). Endurance and strength training increased 6 MW by 48.2 ± 11.2 (p = 0.008) and 39.8 ± 9.8 (p = 0.001) meters, respectively. Endurance and strength training decreased CRT by 4.8 ± 0.7 (p = 0.001) and 1.3 ± 1.2 (p = 0.056) seconds, respectively. Endurance training resulted in greater improvements in HRQL as compared to strength training. CONCLUSION: These results show that walk distance improves as a result of participating in either an endurance or a strength training program. However, an endurance training program leads to greater improvements in both general and disease specific measures of HRQL.

Trajectories of Health-Related Quality of Life in Coronary Artery Disease.

Health-related quality of life (HRQOL) assessment is an important health outcome for measuring the efficacy of treatments and interventions for coronary artery disease (CAD). HRQOL is known to improve over the first year after interventions for CAD, but there is limited knowledge of the changes in HRQOL beyond 1 year. We investigated heterogeneity in long-term trajectories of HRQOL in patients with CAD. Data were obtained from 6226 patients identified from the Alberta Provincial Project for Outcome Assessment in Coronary Heart Disease with at least 1-vessel CAD who underwent their first catheterization between 2006 and 2009. HRQOL was assessed using the Seattle Angina Questionnaire, a 19-item disease-specific measure of HRQOL for patients with CAD. Group-based trajectory analysis was used to identify various subgroups of Seattle Angina Questionnaire trajectories over time while adjusting for missing data through a longitudinal multiple imputation model. Multinomial logistic regression was used to identify the predictors of differences among the identified subgroups. Our analysis revealed significant improvements in HRQOL across all the 5 domains of Seattle Angina Questionnaire overtime for the whole data. Multitrajectory analyses revealed 4 HRQOL trajectory subgroups including high (25.1%), largely increased (32.3%), largely decreased (25.0%), and low (17.6%) trajectories. Age, sex, body mass index, diabetes mellitus, previous history of myocardial infarction, smoking, depression, anxiety, type of treatment received, and perceived social support were significant predictors of differences among these trajectory subgroups. This study highlights variations in longitudinal trajectories of HRQOL in patients with CAD. Despite overall improvements in HRQOL, about a quarter of our cohort experienced a significant decline in their HRQOL over the 5-year period. Understanding these HRQOL trajectories may help personalize prognostic information, identify patients and HRQOL domains on which clinical interventions are most beneficial, and support treatment decisions for patients with CAD.

Response shift in parents' assessment of health-related quality of life of children with new-onset epilepsy

ObjectiveDiagnosis of epilepsy is known to impact health-related quality of life (HRQOL) of children with new-onset epilepsy and can also influence their conceptualization and valuation of HRQOL construct, also known as response shift. This study investigates the presence of response shift in a cohort of children with new-onset epilepsy. MethodsData are from the HEalth-Related QUality of Life in children with Epilepsy Study, a prospective cohort study of 373 children with new-onset epilepsy. Hypotheses about the presence of reconceptualization, reprioritization, and recalibration response shift were tested in the Quality of Life in Childhood Epilepsy (QOLCE-55) Questionnaire, a parent-reported, disease-specific HRQOL measure, using Oort's structural equation model between baseline and 1-year follow-up. Model fit was assessed using log-likelihood ratio test, root mean square error of approximation, and comparative fit index. ResultsSmall positive uniform recalibration response shift effects were observed on physical, emotional, and social functioning domains of the QOLCE-55, but negligibly small negative nonuniform recalibration response shift effect was observed on social functioning domain. There was no significant change in overall QOLCE-55 scores over time after adjusting for response shift effects. SignificanceParents of children with new-onset epilepsy are likely to positively recalibrate (upward bias) their assessments of their children's HRQOL over a 1-year period after diagnosis. This study highlights the potential benefits of response shift as a desired consequence in parents' perception of changes in HRQOL of children with new-onset epilepsy.

Systematic review: health-related quality of life in children and adults with eosinophilic oesophagitis-instruments for measurement and determinant factors.

Several measures have been used to assess the health-related quality of life (HRQoL) of patients with eosinophilic oesophagitis (EoE). To systematically review these HRQoL measures, to appraise measurement properties of specific instruments and to evaluate determinant factors influencing HRQoL in paediatric and adult EoE patients. We searched the PubMed, Embase, Scopus, Web of Science (WOS) and PsycINFO databases for documents providing original information on the development of measurement tools and/or evaluation of HRQoL outcomes in EoE patients of all ages. Of the 596 references identified, data was collected from 34 studies (with only 16 of them being published as full papers) including a total of 1,689 individual patients. Three disease-specific HRQoL measures in EoE covering different aspects of patients' lives and developed in English, were scored positive regarding measurement properties. The PedsQL inventory (including parent and child report forms) and the Peds-QoL EoE module were the generic and specific instruments respectively used in children, while the SF-36 and EoE-QoL-A were the most used questionnaires in adults. Patients with EoE show an impaired HRQoL compared to controls, which greatly depends on symptom severity and disease duration. Severity of endoscopic features and female gender may also determine an impaired HRQoL. The effect of treatments on HRQoL requires further assessment. HRQoL is a relevant outcome that should be considered in clinical practice and research of EoE. Further validation studies in several languages and populations are required to support the use of disease-specific HRQoL measures.

Burden of Keloid Disease: A Cross-sectional Health-related Quality of Life Assessment.

Keloid scars may be painful, itch severely and be cosmetically disturbing. The burden of keloid disease, however, has not yet been determined. This study evaluated the association of keloid disease with health-related quality of life (HRQL) and identified indicators of burden using a cross-sectional survey study, with one disease-specific HRQL measure (Skindex-29) and 2 generic HRQL measures (SF-36 and EQ-5D-5L). A total of 106 keloid patients with no other skin diseases participated in the study. Having keloid disease was associated with a considerable impairment of emotional wellbeing, with most impairment on the emotional and mental HRQL. Pain and itch were the strongest indicators of HRQL impairment in keloid patients. Having painful or itchy keloids was related to low mental and emotional HRQL, implying that patients with keloids require access to effective treatment aimed at alleviating physical symptoms.

Rationale and study design of MOTION: A phase 4, prospective, single-arm, open-label study to measure outcomes in patients with pulmonary arterial hypertension not on active treatment

In clinical trials of treatments for pulmonary arterial hypertension (PAH), objective measures, such as 6-min walk distance (6MWD) are limited in their ability to characterize the impact of PAH therapy from a patient's perspective. Few clinical studies have evaluated the primary effects of pharmacologic treatment on patient-reported outcomes, such as symptoms, health-related quality of life (HRQoL), and productivity. MOTION (NCT02191137) is a prospective, multicenter, single-arm, open-label, phase 4 trial designed to assess whether riociguat monotherapy will improve patient-reported outcomes in patients with PAH in the United States who are not currently on treatment. Following a screening period of up to 14 days, eligible subjects will receive riociguat (0.5–2.5 mg TID) during a 10-week titration phase and a 14-week maintenance phase. The primary endpoint is change from baseline in the Living with Pulmonary Hypertension (LPH) questionnaire, a disease-specific HRQoL measure, after 24 weeks of riociguat treatment. The Short Form-12 Health Survey (SF-12) and the Work Limitations Questionnaire 8 (WLQ-8) will also be utilized to assess patient-reported outcomes. Other variables include change from baseline in World Health Organization functional class, 6MWD, and modified Borg Dyspnea Index. In addition, accelerator band activity will be validated against the 6MWD test. Safety will also be assessed. The MOTION trial will provide information on the effect of riociguat on patient-reported outcomes in PAH patients in the United States who are not currently on active treatment through the use of disease-specific and generic HRQoL measures (LPH and SF-12) and a measure of worker productivity (WLQ-8).

Health-related quality of life in patients with Gilles de la Tourette syndrome at the transition between adolescence and adulthood

Gilles de la Tourette syndrome (GTS) is a neurodevelopmental condition characterised by tics and co-morbid behavioural problems, affecting predominantly male patients. Tic severity typically fluctuates over time, with a consistent pattern showing improvement after adolescence in a considerable proportion of patients. Both tics and behavioural co-morbidities have been shown to have the potential to affect patients’ health-related quality of life (HR-QoL) in children and adults with persisting symptoms. In this study, we present the results of the first investigation of HR-QoL in patients with Gilles de la Tourette syndrome at the transition between adolescence and adulthood using a disease-specific HR-QoL measure, the Gilles de la Tourette Syndrome-Quality of Life-Children and Adolescents scale. Our results showed that patients with GTS and more severe co-morbid anxiety symptoms reported lower HR-QoL across all domains, highlighting the impact of anxiety on patient’s well-being at a critical stage of development. Routine screening for anxiety symptoms is recommended in all patients with GTS seen at transition clinics from paediatric to adult care, to implement effective behavioural and pharmacological interventions as appropriate.

Health-related quality of life and its association with medication adherence in active pulmonary tuberculosis- a systematic review of global literature with focus on South Africa.

IntroductionTuberculosis (TB) is a leading cause of morbidity and mortality in South Africa. Clinical parameters are important objective outcomes in TB; however they often are not directly correlated with subjective well-being of the patient, but can be assessed using patient-reported outcome (PRO) measures. Health-related quality of life (HRQOL) is a specific PRO generally multi-dimensional in nature and includes physical, mental and social health domains. The inclusion of HRQOL PROs in trials and clinical practice can provide additional information beyondclinical and microbiological parameters. Furthermore, HRQOL may be associated with medication adherence. This review focuses on patient-reported HRQOL and its association with medication adherence in TB patients in South Africa.MethodsA comprehensive search strategy was developed focusing on the impact of TB on patient-reported HRQOL,the existence of a conceptual framework of TB-specific HRQOL, determinants of medication adherence and the association of HRQOL with medication adherence. Data were extracted from all identified articles and additionaldata extraction was performed by two independent reviewers with special focus on longitudinal studies in order to understand changes of HRQOL and adherence over time. Research gaps were identified with regard to patient-reported HRQOL and medication adherence.ResultsA total of 66 articles met the eligibility criteria. Ten HRQOL studies and one adherence study used a longitudinal design, none of these in South Africa. A variety of different generic and disease-specific HRQOL measures were identified in the articles. In South Africa four HRQOL and five adherence studies (non-longitudinal) were published. Similar factors (socio-demographic, socio-economic, disease-related, therapy-related and psycho-social aspects) affect HRQOL and adherence. Although standard TB treatment improved all health domains, psychological well-being and social functioning remained impaired in microbiologically cured patients after treatment.ConclusionWhile evidence of TB impact on HRQOL and medication adherence and their association exists, it is verylimited for the South African situation. No valid and reliable TB-specific HRQOL measures were identified in this systematicreview. An assessment of HRQOL in TB patients in South Africa is required as this may assist with improving current disease management programmes, medication adherence and national treatment guidelines.Electronic supplementary materialThe online version of this article (doi:10.1186/s12955-016-0442-6) contains supplementary material, which is available to authorized users.

Unhealthy weight control behaviors mediate the association between weight status and weight-specific health-related quality of life in treatment-seeking youth who are obese.

Examine whether unhealthy and extreme weight control behaviors (WCBs) mediate the relationship between youth weight status and disease-specific health-related quality of life (HRQOL) in treatment-seeking youth who are overweight and obese (OV/OB). 82 youth 10-17years of age who were OV/OB and attending an outpatient obesity-related medical appointment completed measures assessing unhealthy and extreme WCBs and disease-specific HRQOL. Parents completed a demographic questionnaire and medical staff measured youth height and weight. Regression analyses revealed that unhealthy WCBs mediated the associations between youth weight status and emotional and social avoidance disease-specific HRQOL, such that higher body mass index (BMI) predicted unhealthy WCBs, which were ultimately associated with poorer emotional and social HRQOL. Mediation analyses were not significant for total, physical, teasing/marginalization, and positive attributes disease-specific HRQOL. In addition, extreme WCBs did not mediate the association between youth weight status and any subscales of the disease-specific HRQOL measure. Weight status is an important predictor of disease-specific HRQOL in OV/OB youth; however, the association with emotional and social HRQOL is partially accounted for by youth engagement in unhealthy WCBs. Clinicians and researchers should assess WCBs and further research should explore and evaluate appropriate intervention strategies to address unhealthy WCBs in pediatric weight management prevention and treatment efforts.