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Purpose: This study compared a tablet PC questionnaire with a paper method for reliability and patient preferences in the acquisition of patient-reported outcomes (PROs) for patients treated with radiotherapy. By comparing the two modes of PRO administration, we aimed to evaluate the adequacy of using tablet PC questionnaires in future clinical use.Methods: Patients were randomized in a crossover study design using two different methods for PRO entry. A group of 89 patients answered a paper questionnaire followed by the tablet PC version, whereas 89 patients in another group completed the tablet PC questionnaire followed by the paper version. Surveys were performed four times per patient throughout the course of the radiotherapy. The Korean versions of the M.D. Anderson Symptom Inventory (MDASI-K) and the Brief Fatigue Inventory (BFI-K) were used. The primary endpoint of our current study was an assessment of patient preference for the survey method. The proportions of patients preferring each mode of questionnaire were evaluated.Results: The proportion of patients who preferred the tablet PC version, paper form, or who had no preference was 52.2, 22.1, and 25.7 %, respectively. More than half of the patients preferred the tablet PC to the paper version in all four surveys. Age, gender, educational status, prior experience of using a tablet PC, and the order of paper to tablet PC administration did not impact patient preferences. Inter-class correlation coefficients (ICCs) between the modes were 0.92 for MDASI-K and 0.94 for BFI-K and ranged from 0.91 to 0.96 on both instruments during the four surveys.Conclusions: A tablet PC-based PRO is an acceptable and reliable method compared with paper-based data collection for Korean patients receiving radiotherapy. [ABSTRACT FROM AUTHOR]

In 2014, the European Medicines Agency (EMA) released for comment a draft reflection paper on the use of patient-reported outcome (PRO) measures in oncology studies. A twelve-member International Society for Quality of Life Research (ISOQOL) taskforce was convened to coordinate the ISOQOL response. Twenty-one ISOQOL members provided detailed comments and suggestions on the paper: 81 % from academia and 19 % from industry. Taskforce members consolidated and further refined these comments and shared the recommendations with the wider ISOQOL membership. A final response was submitted to the EMA in November 2014. The impending publication of the EMA reflection paper presents a valuable opportunity for ISOQOL to comment on the current direction of EMA PRO guidance and strategy. The EMA paper, although focused on cancer, could serve as a model for using PROs in other conditions, as it provides a useful update surrounding some of the design issues common to all trial research including PRO endpoints. However, we believe there are a number of additional areas in need of greater consideration. The purpose of this commentary is therefore to highlight the strengths of this timely and potentially useful document, but also to outline areas that may warrant further discussion. [ABSTRACT FROM AUTHOR]

Purpose: An Internet-based tool for reporting and analysing patient-reported outcomes (PROs) has been developed. The tool enables merging PROs with blood test results and allows for computation of treatment responses. Data may be visualized by graphical analysis and may be exported for downstream statistical processing. The aim of this study was to investigate, whether patients with myeloproliferative neoplasms (MPNs) were willing and able to use the tool and fill out questionnaires regularly.Methods: Participants were recruited from the outpatient clinic at the Department of Haematology, Roskilde University Hospital, Denmark. Validated questionnaires that were used were European Organisation for Research and Treatment of Cancer Quality of Life Questionnaire-Core 30, Myeloproliferative Neoplasm Symptom Assessment Form, Brief Fatigue Inventory and Short Form 36 Health Survey. Questionnaires were filled out ≥ 6 months online or on paper according to participant preference. Regularity of questionnaire submission was investigated, and participant acceptance was evaluated by focus-group interviews.Results: Of 135 invited patients, 118 (87 %) accepted participation. One hundred and seven participants (91 %) preferred to use the Internet-based tool. Of the 118 enrolled participants, 104 (88 %) submitted PROs regularly ≥ 6 months. The focus-group interviews revealed that the Internet-based tool was well accepted.Conclusion: The Internet-based approach and regular collection of PROs are well accepted with a high participation rate, persistency and adherence in a population of MPN patients. The plasticity of the platform allows for adaptation to patients with other medical conditions. [ABSTRACT FROM AUTHOR]

Purpose: Different health state utility (HSU) instruments produce different utilities for the same individuals, thereby compromising the intended comparability of economic evaluations of health care interventions. When developing crosswalks, previous studies have indicated nonlinear relationships. This paper inquires into the degree of nonlinearity across the four most widely used HSU-instruments and proposes exchange rates that differ depending on the severity levels of the health state utility scale.Methods: Overall, 7933 respondents from six countries, 1760 in a non-diagnosed healthy group and 6173 in seven disease groups, reported their health states using four different instruments: EQ-5D-5L, SF-6D, HUI-3 and 15D. Quantile regressions investigate the degree of nonlinear relationships between these instruments. To compare the instruments across different disease severities, we split the health state utility scale into utility intervals with 0.2 successive decrements in utility starting from perfect health at 1.00. Exchange rates (ERs) are calculated as the mean utility difference between two utility intervals on one HSU-instrument divided by the difference in mean utility on another HSU-instrument.Results: Quantile regressions reveal significant nonlinear relationships across all four HSU-instruments. The degrees of nonlinearities differ, with a maximum degree of difference in the coefficients along the health state utility scale of 3.34 when SF-6D is regressed on EQ-5D. At the lower end of the health state utility scale, the exchange rate from SF-6D to EQ-5D is 2.11, whilst at the upper end it is 0.38.Conclusion: Comparisons at different utility levels illustrate the fallacy of using linear functions as crosswalks between HSU-instruments. The existence of nonlinear relationships between different HSU-instruments suggests that level-specific exchange rates should be used when converting a change in utility on the instrument used, onto a corresponding utility change had another instrument been used. Accounting for nonlinearities will increase the validity of the comparison for decision makers when faced with a choice between interventions whose calculations of QALY gains have been based on different HSU-instruments. [ABSTRACT FROM AUTHOR]

Purpose: This paper aims to estimate the comorbidity of mental disorders and chronic physical conditions and to describe the impact of these conditions on health-related quality of life (HRQoL) in a sample of older primary care (PC) attendees by gender.Methods: Cross-sectional survey, conducted in 77 PC centres in Catalonia (Spain) on 1192 patients over 65 years old. Using face-to-face interviews, we assessed HRQoL (SF-12), mental disorders (SCID and MINI structured clinical interviews), chronic physical conditions (checklist), and disability (Sheehan disability scale). We used multivariate quantile regressions to model which factors were associated with the physical component summary-short form 12 and mental component summary-short form 12.Result: The most frequent comorbidity in both men and women was mood disorder with chronic pain and arthrosis. Mental disorders mainly affected 'mental' QoL, while physical disorders affected 'physical' QoL. Mental disorders had a greater impact on HRQoL than chronic physical conditions, with mood and adjustment disorders being the most disabling conditions. There were some gender differences in the impact of mental and chronic physical conditions on HRQoL. Anxiety disorders and pain had an impact on HRQoL but only in women. Respiratory diseases had an effect on the MCS in women, but only affected the PCS in men.Conclusions: Mood and adjustment disorders had the greatest impact on HRQoL. The impact profile of mental and chronic physical conditions differs between genders. Our results reinforce the need for screening for mental disorders (mainly depression) in older patients in PC. [ABSTRACT FROM AUTHOR]

Objective: This paper evaluates the effectiveness of motivational enhancement therapy plus cognitive behavioural therapy on depressive symptoms, glycosylated haemoglobin, fasting glucose, body mass index (BMI), and health-related quality of life in type II diabetes patients.Methods: A controlled trial was conducted to compare patients who received the behavioural intervention with untreated controls on measures of health outcomes. A total of 31 intervention group participants and 30 controls were selected from patients that met the inclusion criteria from a hospital-based endocrinology outpatient department. The outcome measures including depressive symptoms, glycosylated haemoglobin, fasting glucose, BMI, and both physical and mental quality of life were collected before (T1), after (T2), and after 90 days (T3) following the intervention.Results: The experimental group showed a significant reduction in glycosylated haemoglobin, fasting glucose, and depressive symptoms and a significant increase in physical quality of life and mental quality of life at T2 and T3, while patients in the control group with usual care showed no changes over time.Conclusion: The behavioural intervention facilitated a significant improvement in psychological adjustment and glycemic control, thus strengthening diabetes control skills and leading to healthy outcomes. It is feasible that nurses and psychiatrists can deliver the behavioural intervention for diabetes patients to decrease their depressive symptoms. Sharing discussion and problem-solving experiences is particularly helpful method for self-control, and these will be beneficially influential on further research. [ABSTRACT FROM AUTHOR]

Background: A faecal or urinary ostomy may be lifesaving. However, it involves significant bodily change, and the adjustment process to life with an ostomy includes a broad spectre of physical and psychosocial challenges. Thus, new interventions are needed to improve adaptation to living with an ostomy. This study aimed to examine experiences and outcomes using a new clinical feedback system with patient-reported outcome measures in ostomy care. Methods: In this longitudinal explorative study, 69 ostomy patients were followed by a stoma care nurse in an outpatient clinic, using a clinical feedback system postoperatively at 3, 6 and 12 months. The patients responded electronically to the questionnaires before each consultation. The Generic Short Patient Experiences Questionnaire was used to measure patient experiences and satisfaction with follow-up. The Ostomy Adjustment Scale (OAS) measured adjustment to life with an ostomy, and the Short Form-36 (SF-36) assessed the patient's health-related quality of life. Longitudinal regression models with time as an explanatory (categorical) variable were used to analyse changes. The STROBE guideline was applied. Results: The patients were satisfied with their follow-up (96%). Especially, they felt they received sufficient and individualised information, were involved in treatment decisions, and benefited from the consultations. The OAS subscale scores for 'daily activities', 'knowledge and skills' and 'health' improved over time (all p < 0.05), as did the physical and mental component summary scores of the SF-36 (all p < 0.05). Effect sizes of changes were small (0.20–0.40). Sexuality was the most challenging factor reported. Conclusions: The clinical feedback system could be helpful because outpatient follow-ups for ostomy patients may be more tailored when clinicians use clinical feedback systems. However, further development and testing are needed. Plain English summary: Studies show that patients struggle with adjustment to the ostomy in a broad spectre of physical and psychosocial factors, and stoma care nurse follow-up is necessary. Mapping each patient's most important challenges during a short outpatient follow-up consultation may be difficult. Thus, missing information on individual factors affecting adjustment and quality of life can result in underreported problems and unclear communication between patients and stoma care nurse. The study aimed to explore patient satisfaction and experiences using digital questionnaires before each consultation in stoma care nurse ostomy follow-up and to study adjustment to ostomy and health-related quality of life in routine follow-up 3,6 and 12 months after ostomy surgery. The study results showed that the patients were satisfied with their follow-up using questionnaires before consultations (96%). Especially, they felt they received sufficient and individualised information, were involved in treatment decisions, and benefited from the consultations. Especially the life areas "daily activities", having "knowledge and skills", "health", and the patient's health-related quality of life improved during the first year after the operation. Thus, patient reported outcomes/clinical feedbacksystem is a promising method for follow-up of ostomy patients because it may promote better discussions during the consultation and tailor the patient's adjustment trajectory more precisely than without such a system. [ABSTRACT FROM AUTHOR]

Background: Atopic dermatitis (AD) is a common skin disorder characterized by chronic inflammation, altered skin barrier function, and inflammatory cell skin infiltration that decreases health-related quality of life (HRQoL). The study objective was to understand the patient perspective of AD burden and determine suitable patient-reported outcome (PRO) measures. Methods: This mixed methods study involved the collection of qualitative and quantitative information from adults (≥ 18 years old) and adolescents (12 – 17 years old) with clinician-confirmed AD regarding their experiences of AD symptoms and its impact on HRQoL. The first part of the study included three stages: in-person concept elicitation (CE) interviews, a 2-week daily electronic diary (eDiary) study, and in-person cognitive debriefing (CD) interviews. An Itch numeric rating scale (NRS) (v1.0) and a Skin Pain NRS (v1.0) evaluation during CD interviews required participants to think about their 'worst' itch and 'worst' skin pain in the past 24 h. Other PRO measures allowed for psychometric testing. The second part of the study involved telephone-depth interviews (TDIs) and qualitative feedback from participants who had not participated in the CD interviews. Qualitative data were thematically analyzed. Psychometric evaluation of NRS measures was performed using eDiary data. Results: In the CE interviews, itch and/or itching and skin pain were the most prevalent symptoms consistently discussed by participants. Both NRS measures demonstrated strong psychometric reliability and were applicable across ages with suitable concurrent validity. During the CD interviews, some participants focused their answers on their 'average' itch/itching in the past 24 h, rather than their 'worst' itch. Some participants answered the Skin Pain NRS thinking about general pain or other types of pain, rather than skin pain specifically. Consequently, modifications to both measures addressed these issues and re-tested as paper-and-pen versions in subsequent TDIs. Itch NRS (v2.0) modifications helped participants focus on their worst itching. Most participants preferred Skin Pain NRS v2.0b, which included skin pain descriptors. Conclusions: Itching and skin pain are the most important and relevant AD symptoms. The Itch NRS (v2.0) and Skin Pain NRS (v2.0b) appear to be appropriate endpoints for the assessment of itching and skin pain severity for clinical trials with adults and adolescents with AD. [ABSTRACT FROM AUTHOR]

Purpose: Behavioral interventions have been used with breast cancer survivors (BCS) in cancer pain management and post-treatment quality of life (QOL) studies. We studied the effects of an anti-inflammatory dietary intervention on QOL in BCS.Methods: One hundred fifty-three overweight and obese (body mass index [BMI] ≥ 25 kg/m2), early stage (0-III), English-speaking BCS who had completed all cancer treatment 2 or more months prior to enrollment were recruited into a two-arm randomized controlled trial with a 2 (group) by 3 (time) repeated measures design. Intervention components included six monthly food-preparation workshops and twelve motivational interviewing telephone calls. Endpoints included the Perceived Stress Scale (PSS), the Functional Assessment of Cancer Therapy-General (FACT-G) and Breast Cancer (FACT-B), and the Center for Epidemiologic Studies Depression Scale (CES-D). Repeated measures analysis using PROC MIXED in SAS version 9.4 was used.Results: On repeated measures analysis (intent to treat), there were no differences between groups on any of the QOL outcomes except the PSS total scores. These were significantly different in the intervention group (IG; n = 76) compared to control group (CG; n = 77), showing a main effect of assignment but no effect of time and no interaction effects.Conclusion: There was an impact on QOL as measured by the PSS between groups. The intervention reduced perceived stress at 6-month follow-up, but the effects dissipated by 12 months. Sources and stress and stress reduction should be a focus of future studies. Future research should also identify appropriate QOL measures that are sensitive to changes brought about by behavioral interventions. [ABSTRACT FROM AUTHOR]

Background: Papulopustular rash is a common class effect of epidermal growth factor receptor inhibitors (EGFRI) that can affect patients' health-related quality of life and cause disruptions to treatment. SWOG S1013 (NCT01416688) is a multi-center study designed to validate the Functional Assessment of Cancer Therapy EGFRI 18 (FACT-EGFRI 18) using 7-items from the National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) version 4.0 to assess EGFRI-induced skin-related toxicities and their impact on functional status. Methods: Patients with a diagnosis of colorectal or lung cancer to receive EGFRI therapies for at least 6 weeks were enrolled. Patient self-assessments using the FACT-EGFRI 18 were completed prior to undergoing CTCAE assessment by trained clinicians at baseline, weekly × 6, and then monthly × 3. The psychometric properties of the FACT-EGFRI 14 (skin toxicity items only) and 18 (plus 2 nail and 2 hair items) were established based on criterion validity, known groups validity, internal consistency reliability, and responsiveness to change. Results: Of the 146 registered patients, 124 were evaluable. High Cronbach's alpha (> 0.70) for both FACT-EGFRI 14 and FACT-EGFRI 18 scores across assessment times were observed. Although agreement (i.e. criterion validity) between individual and summary scales of the FACT-EGFRI 18 for assessing skin toxicity was good, agreement with the clinician-reported CTCAE was only fair. The minimal important difference was determined to be 3 points. The results also demonstrated responsiveness to symptom change. Discussion: Based on the results of this multi-center validation study, the FACT-EGFRI 18 patient-reported outcome instrument provided data from the patient's perspective yielding unique information as well as complementing clinician-rated CTCAE grades, especially for the symptoms of pain, pruritus, and paronychia. Conclusions: Good to excellent psychometric properties for the FACT-EGFRI 18 were demonstrated, supporting further use of this patient-reported outcomes measure. Additional validation with a more diverse group of patients should be conducted. [ABSTRACT FROM AUTHOR]

Purpose: To compare self-rated health among the United States general population in 2002 and 2017.Methods: Secondary data were analyzed from two EQ-5D valuation studies conducted in 2002 and 2017. Both studies included the EQ-5D-3L self-classifier and visual analog scale (VAS), where health is rated from 0 (worst imaginable health) to 100 (best imaginable health). VAS scores were compared between time points using regression models, adjusting for sociodemographic factors (Model 1), plus illness (Model 2), and health problems according to the EQ-5D classifier (Model 3).Results: Mean VAS scores in 2002 [84.4 (SD = 16.1)] were not different from 2017 [84.6 (SD = 14.5)] (p = 0.63), nor different after adjusting for demographics (Model 1) or illness (Model 2). However, 2017 VAS mean scores were significantly higher than 2002 [2.2 (95% CI 1.36-3.10)] upon adjusting for the presence of dimension-specific health problems.Conclusions: Self-rated health of the general US adult population in 2017 was similar to 2002, but after adjusting for health problems, scores were slightly higher in 2017. Sociodemographic shifts in age and education explain some of the differences in scores, and by removing health and sociodemographic factors, we found the VAS reveals self-rated health is slightly better in 2017 than 2002. [ABSTRACT FROM AUTHOR]

Purpose: Early detection and improved treatment have increased lung cancer survival. Lung cancer survivors have more symptom distress and lower function compared with other cancer survivors; however, few interventions are available to improve health-related quality of life (HR-QOL). Lifestyle risk reduction interventions have improved HR-QOL in other cancer survivors. The purpose of this study was to explore lung cancer survivor perspectives on making behavioral changes in the context of a lifestyle risk reduction intervention.Methods: Twenty-two lung cancer survivors participated in interviews after completing the Healthy Directions (HD) intervention. Interviews were audiotaped, transcribed, and analyzed using inductive content analysis. Demographic and clinical characteristics were gathered through a survey and analyzed using descriptive statistics.Results: Five main themes were identified: (1) the diagnosis was a motivator for behavior change, (2) participants had to deal with disease consequences, (3) the coach provided guidance, (4) strategies for change were initiated, and (5) social support sustained behavioral changes. Other important subthemes were the coach helped interpret symptoms, which supported self-efficacy and goal setting, and survivors employed self-monitoring behaviors. Several participants found the recommended goals for physical activity were difficult and were discouraged if unable to attain the goal. Findings underscore the need for individualized prescriptions of physical activity, especially for sedentary survivors.Conclusions: Lung cancer survivors described the benefits of coaching to enhance their engagement in behavioral change. Additional research is needed to validate the benefit of the HD intervention to improve HR-QOL among this vulnerable and understudied group of cancer survivors. [ABSTRACT FROM AUTHOR]

Treatment of relapsed/refractory multiple myeloma (RRMM) aims to prolong survival while maintaining health-related quality of life (HRQoL) by managing disease-related symptoms and complications-one of the most frequent and debilitating being bone pain. In the ELOQUENT-2 study (NCT01239797), which evaluated the addition of elotuzumab to lenalidomide plus dexamethasone versus lenalidomide plus dexamethasone, pain and HRQoL were assessed in patients with relapsed/refractory disease using the Brief Pain Inventory-Short Form (BPI-SF) and the European Organisation for Research and Treatment of Cancer (EORTC) Quality of Life Questionnaire-Core 30 module (QLQ-C30) and myeloma-specific module (QLQ-MY20). Mean baseline pain scores were low and remained so throughout treatment with both regimens; mean HRQoL scores did not change substantially from baseline. A significantly higher proportion of patients with objective response than without had clinically meaningful improvements in worst pain over two consecutive treatment cycles (29 versus 12%; p < 0.001). Patients with very good partial response (VGPR) or better reported reduced scores for pain severity and worst pain; those with progressive disease reported increased scores for these domains and pain interference. These findings show that previously reported improvements in progression-free survival and response rate with elotuzumab are achieved without detriment to HRQoL, which is maintained over time. [ABSTRACT FROM AUTHOR]

Purpose: The objective of this study was to analyze the clinical factors associated with changes in HRQoL in outpatients with schizophrenia using both generic and condition-specific HRQoL scales.Methods: Adult outpatients with schizophrenia at least 18 years of age who did not have an acute psychotic exacerbation in the 3 months prior to baseline were recruited. PANSS dimensions were calculated based on Lindenmayer et al.'s five factors. HRQoL data were assessed by patients using the Schizophrenia Quality of Life Scale (SQLS), the Short Form-36 (SF-36), and the EuroQol-5 Dimension (EQ-5D) questionnaires.Results: Out of the 1345 patients included at baseline, 1196 (89%) were evaluated at 12 months. Regression models showed that the factor most consistently associated with HRQoL at endpoint was change in the PANSS negative symptoms score. A decrease in the PANSS negative symptoms score from baseline to 1 year was associated with a decrease in HRQoL during the same period. There were also significant associations of the change in PANSS excitatory factor with all the HRQoL scales except the SF-36 PCS. Female gender was associated with a decrease in all HRQoL ratings. There was also a relationship between years since onset and HRQoL. The longer the time since illness onset, the larger the decrease in HRQoL.Conclusions: This study has found that, in outpatients with schizophrenia, changes in negative and excitement symptoms may have a greater an association with HRQoL than changes in positive, cognitive and depressive symptoms. [ABSTRACT FROM AUTHOR]

Purpose: Gynecologic Cancer Intergroup Symptom Benefit Study (GCIG-SBS) Stage 2 aimed to review, revise, and validate a patient-reported outcome measure (PROM), the Measure of Ovarian Symptoms and Treatment concerns (MOST), developed in GCIG-SBS Stage 1 (MOSTv1, 35 items), and document recurrent ovarian cancer (ROC) symptom burden and benefit.Methods: GCIG-SBS Stage 2 recruited patients with platinum-resistant/refractory ROC (PRR-ROC) or potentially platinum-sensitive ROC with ≥ 3 lines of prior chemotherapy (PPS-ROC ≥ 3). Patients completed MOSTv1, QLQ-C30, QLQ-OV28, and FACT-O/FOSI at baseline and before cycle 3 of chemotherapy (pre-C3), and global assessments of change (MOST-Change) pre-C3. Clinicians rated patients' cancer-related symptoms, performance status, and adverse events. Convergent and divergent validity (Spearman's correlations), discriminative validity (effect sizes between groups classified by clinician-rated characteristics), and responsiveness (paired t tests in patients expected to experience clinically meaningful change) were assessed.Results: Of 948 recruits, 903 completed PROMs at baseline and 685 pre-C3. Baseline symptom burden was substantial for PRR-ROC and PPS-ROC ≥ 3. MOSTv2 has 24 items and five multi-item scales: abdominal symptoms (MOST-Abdo), disease or treatment-related symptoms (MOST-DorT), chemotherapy-related symptoms (MOST-Chemo), psychological symptoms (MOST-Psych), and MOST-Well-being. Correlations confirmed concurrent and divergent validity. Discriminative validity was confirmed by effect sizes that conformed with a priori hypotheses. MOST-Abdo was responsive to improvements in abdominal symptoms and MOST-Chemo detected the adverse effects of chemotherapy.Conclusions: The MOSTv2 validly quantifies patient-reported symptom burden, adverse effects, and symptom benefit in ROC, and as such is fit-for-purpose for clinical trials of palliative chemotherapy in ROC. Further research is required to assess test-retest reliability. [ABSTRACT FROM AUTHOR]

Purpose: To test whether the systematic monitoring of health-related quality of life (HRQOL) in clinical practice in Spanish pediatric patients with T1DM helps improve their daily life in a multicenter longitudinal study.Methods: One hundred thirty-six patients participated, recruited from five centers in Barcelona, Spain (72 girls, mean age 13.4 years). Complete data were collected for 119 patients (85%). Pediatricians were randomly assigned to the HRQOL intervention (n = 70), or control group (n = 49). The intervention group discussed the results of HRQOL face to face with the physician, quarterly over a year. The control group received care as usual. HRQOL was assessed using KIDSCREEN-27 collected online. Standardized mean differences (effect size, ES) and generalized estimating equation (GEE) were computed to compare group differences between baseline and follow-up, taking into account sociodemographic and clinical variables.Results: Statistically significant higher scores were seen in the intervention group at follow-up for the dimensions of Psychological well-being (ES = 0.56), School environment (ES = 0.56), and the KIDSCREEN-10 index (ES = 0.63). No differences were found in the control group. GEE analysis showed an improvement in HRQOL at follow-up with statistically significant association of the intervention on Psychological well-being (B = 4.32; p 0.03 for the interaction of group by follow-up) and School environment (B = 4.64; p 0.02 for the same interaction term).Conclusions: Routine assessment and face-to-face patient-physician discussion of HRQOL results improved HRQOL scores after a year of follow-up, especially in Psychological well-being and school environment. The results support the routinary use of HRQOL assessment in clinical practice. [ABSTRACT FROM AUTHOR]

Purpose: Community treatment orders (CTOs) are widely used internationally despite a lack of evidence supporting their effectiveness. Most effectiveness studies are relatively short (12-months or less) and focus on clinical symptoms and service data, while a little attention is given to patients' social outcomes and broader welfare. We tested the association between the duration of CTO intervention and patients' long-term social outcomes.Methods: A sub-sample (n = 114) of community-based patients from the Oxford Community Treatment Order Evaluation Trial (OCTET) were interviewed 48 months after randomisation. Multivariate regression models were used to examine the association between the duration of the CTO intervention and social outcomes as measured by the social network schedule, Objective Social Outcomes Index, Euro-Qol EQ-5D-3L (EQ-5D), and Oxford Capabilities Questionnaire for Mental Health.Results: No significant association was found between the duration of CTO intervention and social network size (IRR = 0.996, p = .63), objective social outcomes (B = -0.003, p = .77), health-related quality of life (B = 0.001, p = .77), and capabilities (B = 0.046, p = .41). There were no between-group differences in social outcomes when outcomes were stratified by original arm of randomisation. Patients had a mean of 10.2 (SD = 5.9) contacts in their social networks, 42% of whom were relatives.Conclusions: CTO duration was not associated with improvements in patients' social outcomes even over the long term. This study adds to growing concerns about CTO effectiveness and the justification for their continued use. [ABSTRACT FROM AUTHOR]

Background: Human papillomavirus (HPV)-related cancers have been associated with different demographic profiles and disease characteristics than HPV-unrelated cancers in head and neck patients, but distress and other symptoms have not been compared. The aim of this study was to assess whether distress levels, fatigue, pain, anxiety, depression, and common psychological and practical problems differ between head and neck cancer patients with HPV-related vs. HPV-unrelated carcinomas (using oropharyngeal carcinoma (OPC) and non-OPC cancers as surrogates for HPV status).Methods: Distress, depression, anxiety, fatigue, pain, and common problems were examined in 56 OPC and 90 non-OPC patients at 4 timepoints during the first year following diagnosis. Two-level hierarchical linear modeling was used to examine effects.Results: The HPV-related OPC group was more likely to be younger (p = 0.05), Caucasian (p = 0.001), non-smokers (p = 0.01), earn more (p = 0.04), and present with more advanced stage (p < 0.0001). At baseline, OPC patients reported only higher pain scores (p = 0.01) than non-OPC patients. Total problems decreased more in the OPC group (p = 0.08) than the non-OPC group from baseline to 12-month follow-up. In both groups, scores on distress, depression, psychosocial problems, and practical problems decreased similarly over time.Conclusions: Despite a difference in the clinico-demographic characteristics of HPV-related vs. HPV-unrelated patients, only baseline pain levels and total problems over time differed between the two groups. [ABSTRACT FROM AUTHOR]

Introduction: To date, there is little research on health-related quality of life (HRQoL) in Down syndrome (DS), and existing research is variable with regard to reported HRQoL in DS. There are also no HRQoL measures developed specifically to be used with individuals with Down syndrome.Methods: A multi-national, longitudinal, 24-week non-interventional study was conducted in adolescents and adults with DS. HRQoL was assessed (n = 90) using the parent-report KIDSCREEN-27 questionnaire.Results: HRQoL domain scores were found to be similar to those in the KIDSCREEN-27 European normative group data set on the Physical Well-being, Psychological Well-being, Autonomy and Parent Relations domains. Compared with the normative data set, the adolescent participants with DS in the current study were found to have lower scores on the Social Support and Peers domain and higher scores than the normative group on the School Environment domain. The test-retest reliability of the KIDSCREEN-27 was also examined using the intraclass correlation coefficient (ICC) in a subgroup of stable participants. The KIDSCREEN-27 demonstrated poor-to-moderate test-retest reliability; however, test-retest reliability was assessed using a long time interval between assessment time points.Conclusion: The findings of this study underline that further research is needed to better understand the nature of HRQoL in DS. Further research using a shorter time interval between assessment time points to examine test-retest reliability is also required.Funding: F. Hoffmann-La Roche Ltd. [ABSTRACT FROM AUTHOR]

Purpose: To validate and compare the psychometric properties of the EQ-5D-3L with the EQ-5D-5L classification systems in Greece.Methods: Participants (n = 2279) over 40 years old, sampled from the greater area of Athens using a multistage stratified quota sampling method, completed both EQ-5D versions, while information was also collected on socio-demographics and health-related characteristics. The EQ-5D-5L and EQ-5D-3L were evaluated in terms of agreement, ceiling effects, redistribution and inconsistency, informativity, and convergent and known-groups validity.Results: The agreement between the EQ-5D-3L and EQ-5D-5L was high (ICC = 0.85). Ceiling effects decreased significantly in the EQ-5D-5L in all domains (P < 0.001), with "usual activities" (-21.4 %) and "self-care" (-20.1 %) showing the highest absolute and "anxiety/depression" the highest relative reduction (-32.46 %). Inconsistency was low (5.7 %). The increase in prevalence of problems was larger than the decrease in their severity, resulting in a lower mean health utility for the EQ-5D-5L. Overall absolute and relative informativity improved by 70.5 % and 16.4 %, respectively, in the EQ-5D-5L. Both instruments exhibited good convergent and known-groups validity, with evidence of a considerably better convergent performance and discriminatory ability of the EQ-5D-5L.Conclusions: Both EQ-5D versions demonstrated good construct validity and had consistent redistribution. The EQ-5D-5L system may be preferable to the EQ-5D-3L, as it exhibited superior performance in terms of lower ceiling effects, higher absolute and relative informativity, and improved convergent and known-groups validity efficiency. [ABSTRACT FROM AUTHOR]

Purpose: The goal of chemotherapy for metastatic breast cancer (MBC) is to prolong survival and maintain health-related quality of life. This study aimed to evaluate long-term health status of patients with MBC who participated in the phase III randomized SELECT BC trial.Methods: In the SELECT BC trial, patients were randomly allocated to the S-1 or taxane (paclitaxel or docetaxel) arm. Health status was assessed by EQ-5D at pre-treatment, 3 and 6 months after randomization, and every 6 months thereafter to the extent possible. Least square mean scores were assessed to compare EQ-5D index values between groups. Time to deterioration analysis was also performed by defining the minimally important difference of EQ-5D as 0.05 or 0.1.Results: The number of patients for EQ-5D analysis was 175 and 208 in the taxane and S-1 arms, respectively. Least square mean EQ-5D index values up to 60 months were 0.741 (95 % CI [0.713-0.769]) in the taxane arm and 0.748 [0.722-0.775] in the S-1 arm. The EQ-5D index value during PFS up to 12 months in the S-1 was superior to the corresponding index value in the taxane (0.812 [0.789-0.834] vs. 0.772 [0.751-0.792], P = 0.009). Time to deterioration analysis also revealed that S-1 significantly delayed the deterioration of EQ-5D index value during the period before progression (P = 0.002 and 0.003).Conclusions: Our findings suggest that the EQ-5D index value was higher in patients treated with S-1 during first-line chemotherapy. Considering non-inferiority of S-1 in terms of OS, obtained quality-adjusted life years may be greater in the S-1 arm. [ABSTRACT FROM AUTHOR]

Purpose: To validate the Dutch version of the EORTC QLQ-CR29 quality of life questionnaire for colorectal cancer.Methods: We translated and pilot-tested the original questionnaire in the Netherlands, following EORTC guidelines. We assessed factor structure, reliability and construct validity in different samples of patients from four hospitals.Results: Of 296 patients, 236 (80 %) returned the questionnaire, and 27 out of 48 patients returned the retest questionnaire. In addition to the original three scales, we found a reliable bowel functioning scale (α = 0.80), reducing the number of individual items by five. Two of the other scales had sufficient to good reliability (urinary frequency, α = 0.71, original α = 0.75, body image α = 0.80, original α = 0.84), the third, blood and mucus in stool, only moderate (α = 0.56, original α = 0.69). Item functioning was sufficient to excellent for all but two items (urinary incontinence and dysuria). Construct validity was similar to that in earlier studies.Conclusion: We found a very satisfactory scale for bowel problems, in patients both with and without stoma. The body image and urinary incontinence scales were reliable, and construct validity was sufficient. We suggest the questionnaire to be adapted to decrease the number of individual items, improve the scales, and therefore increase reliability of the entire questionnaire. [ABSTRACT FROM AUTHOR]

Purpose: This study sought to compare the association between health-related quality of life (HRQoL) and four body health types by gender.Methods: The study included 6217 men and 8243 women over 30 years of age chosen from a population-based survey. Participants were grouped by body mass index and metabolic abnormality into four types: metabolically healthy normal weight, metabolically abnormal but normal weight (MANW), metabolically healthy obesity (MHO), and metabolically abnormal obesity (MAO). HRQoL was measured using the EQ-5D health questionnaire. The outcomes encompassed five dimensions (mobility, self-care, usual activity, pain/discomfort, and anxiety/depression), and the impaired HRQoL dichotomized by the EQ-5D preference score. Complex sample multivariate binary logistic regression analyses were conducted to adjust for sociodemographic variables, lifestyle factors, and disease comorbidity.Results: Among men, those in the MANW group presented worse conditions on all dimensions and the impaired HRQoL compared to other men. However, no significant effect remained after adjusting for relevant covariates. For women, those in the MAO group had the most adversely affected HRQoL followed by those females in the MHO group. The domain of mobility and impaired HRQoL variable of the MAO and MHO groups remained significant when controlling for all covariates in the model.Conclusions: The MANW is the least favorable condition of HRQoL for men, suggesting that metabolic health may associate with HRQoL more than obesity for males. In women, the MAO and MHO groups had the most adversely affected HRQoL, implying that MHO is not a favorable health condition and that obesity, in general, may be strongly associated with HRQoL in women. [ABSTRACT FROM AUTHOR]

Background: The aim of this study was to determine the social/economic costs and health-related quality of life (HRQOL) of patients with epidermolysis bullosa (EB) in eight EU member states.Methods: We conducted a cross-sectional study of patients with EB from Bulgaria, France, Germany, Hungary, Italy, Spain, Sweden and the United Kingdom. Data on demographic characteristics, health resource utilisation, informal care, labour productivity losses, and HRQOL were collected from the questionnaires completed by patients or their caregivers. HRQOL was measured with the EuroQol 5-domain (EQ-5D) questionnaire.Results: A total of 204 patients completed the questionnaire. Average annual costs varied from country to country, and ranged from €9509 to €49,233 (reference year 2012). Estimated direct healthcare costs ranged from €419 to €10,688; direct non-healthcare costs ranged from €7449 to €37,451 and labour productivity losses ranged from €0 to €7259. The average annual cost per patient across all countries was estimated at €31,390, out of which €5646 accounted for direct health costs (18.0 %), €23,483 accounted for direct non-healthcare costs (74.8 %), and €2261 accounted for indirect costs (7.2 %). Costs were shown to vary across patients with different disability but also between children and adults. The mean EQ-5D score for adult EB patients was estimated at between 0.49 and 0.71 and the mean EQ-5D visual analogue scale score was estimated at between 62 and 77.Conclusion: In addition to its negative impact on patient HRQOL, our study indicates the substantial social/economic burden of EB in Europe, attributable mostly to high direct non-healthcare costs. [ABSTRACT FROM AUTHOR]

Objective: The aim of this study was to determine the economic burden from a societal perspective and the health-related quality of life (HRQOL) of patients with Duchenne muscular dystrophy (DMD) in Europe.Methods: We conducted a cross-sectional study of patients with DMD from Bulgaria, France, Germany, Hungary, Italy, Spain, Sweden, and the UK. Data on demographic characteristics, healthcare resource utilization, informal care, labor productivity losses, and HRQOL were collected from the questionnaires completed by patients or their caregivers. HRQOL was measured with the EuroQol 5-domain (EQ-5D) questionnaire. Costs have been estimated from a societal perspective adopting a bottom-up approach.Results: A total of 422 questionnaires were included in the study; 268 of which were collected from patients with DMD and 154 from caregivers. The average annual cost per person in 2012 ranged from €7657 in Hungary to €58,704 in France. Direct non-healthcare costs are the main component of whole costs and informal care is the main driver of non-healthcare costs. Costs are also shown to differ between children and adults. With regard to HRQOL of adult patients, the EQ-5D VAS score and EQ-5D index scores were 50.5 and 0.24, respectively. The corresponding EQ-5D VAS and EQ-5D index scores for caregivers were 74.7 and 0.71, respectively.Conclusions: We have estimated the average annual cost per patient with DMD in eight European countries adopting a social perspective, and to our knowledge this is the first study with such a wide perspective. The results on costs show a considerable gap between Eastern and Western European countries. Non-healthcare costs range from 64 to 89 % of overall costs and informal care is to a great extent the main driver of this cost category. The HRQOL of people with DMD is much lower than that of the general population. [ABSTRACT FROM AUTHOR]

Objectives: Our goal was to provide data on the economic burden and health-related quality of life (HRQOL) of patients with cystic fibrosis (CF) and their caregivers in Europe.Methods: A cross-sectional study was carried out on adults and children with CF in eight European countries. Patients completed an anonymous questionnaire regarding their socio-demographic characteristics, use of healthcare services and presence of a caregiver. Costs were calculated with a bottom-up approach using unit costs from each participating country, and HRQOL was assessed using EQ-5D. The principal caregiver also answered a questionnaire on their characteristics, HRQOL and burden.Results: A total of 905 patients with CF was included (399 adults and 506 children). The total average annual cost per patient varied from €21,144 in Bulgaria to €53,256 in Germany. Adults had higher direct healthcare costs than children, but children had much higher informal care costs (P < 0.0001). Total costs increased with patients' level of dependence. In adults, mean utility fell between 0.640 and 0.870, and the visual analogue scale ranged from 46.0 to 69.7. There was no difference in caregiver HRQOL regardless of whether they cared for an adult or a child. However, caregivers who looked after a child had a significantly higher burden (P = 0.0013).Conclusions: Our study highlights the burden of CF in terms of costs and decreased HRQOL for both patients and their caregivers throughout Europe. [ABSTRACT FROM AUTHOR]

Objective: The aim of this study was to determine the economic burden from a societal perspective and health-related quality of life (HRQOL) of patients with Prader-Willi syndrome (PWS) in Europe.Methods: We conducted a cross-sectional study of patients with PWS from Spain, Bulgaria, Hungary, Germany, Italy, the UK, Sweden and France. Data on demographic characteristics, healthcare resource utilisation, informal care, labour productivity losses and HRQOL were collected from questionnaires completed by patients or their caregivers. HRQOL was measured with the EuroQol 5-domain (EQ-5D) questionnaire.Results: A total of 261 patients completed the questionnaire. The average annual costs ranged from € 3937 to € 67,484 between countries; the reference year for unit prices was 2012. Direct healthcare costs ranged from € 311 to € 18,760, direct non-healthcare costs ranged from € 1269 to € 44,035, and loss of labour productivity ranged from € 0 to € 2255. Costs were also shown to differ between children and adults. The mean EQ-5D index score for adult PWS patients ranged between 0.40 and 0.81 and the mean EQ-5D visual analogue scale score ranged between 51.25 and 90.00.Conclusion: The main strengths of this study lie in our bottom-up approach to costing and in the evaluation of PWS patients from a broad societal perspective. This type of analysis is very scarce in the international literature on rare diseases in comparison with other illnesses. We conclude that PWS patients incur considerable societal costs and experience substantial deterioration in HRQOL. [ABSTRACT FROM AUTHOR]

Objective: The aim of this study was to determine the economic burden from a societal perspective and the health-related quality of life (HRQOL) of patients with systemic sclerosis (SSc; scleroderma) in Europe.Methods: We conducted a cross-sectional study of patients with SSc (involving both localised and systemic sclerosis) from Germany, Italy, Spain, France, the UK, Hungary and Sweden. Data on demographic characteristics, healthcare resource utilisation, informal care, labour productivity losses and HRQOL were collected from the questionnaires completed by patients or their caregivers. HRQOL was measured with the EuroQol 5-domain (EQ-5D) questionnaire.Results: A total of 589 patients completed the questionnaire. The rate of patients with localised scleroderma, limited cutan and diffuse cutan SSc were 28, 68 and 4 %, respectively. Average annual costs varied from country to country and ranged from € 4607 to € 30,797 (reference year: 2012). Estimated direct healthcare costs ranged from € 1413 to € 17,300; direct non-healthcare costs ranged from € 1875 to € 4684 and labour productivity losses ranged from € 1701 to € 14,444. The mean EQ-5D index score for adult SSc patients varied from 0.49 to 0.75 and the mean EQ-5D visual analogue scale score was between 58.72 and 65.86.Conclusion: The main strengths of this study lie in our bottom-up approach to costing and our evaluation of SSs patients from a broad societal perspective. This type of analysis is very unusual in the international literature on rare diseases in comparison with other illnesses. We concluded that SSc patients incur considerable societal costs and experience substantial deterioration in HRQOL. [ABSTRACT FROM AUTHOR]

Objectives: To assess the health-related quality of life (HRQOL) of patients with mucopolysaccharidosis (MPS) and their caregivers and to quantify the disease-related costs from a societal perspective.Methods: In the context of a multi-country study of rare diseases (BURQOL-RD project), a cross-sectional survey was performed among MPS patients in seven European countries. Data on demographic characteristics, health resource utilization, informal care, and loss of labor productivity were collected. The EQ-5D, Barthel index (BI), and Zarit burden interview (ZBI) questionnaires were used to assess patients' and their informal caregivers' quality of life, patients' functional ability, and caregivers' burden, respectively.Results: Altogether, 120 patients (children 62 %, females 40 %) and 66 caregivers completed the questionnaire. Patients' mean age was 16.5 years and median age at diagnosis was 3 years. Adult patients' average EQ-5D and EQ VAS scores varied across countries from 0.13 to 0.43 and 30.0 to 62.2, respectively, mean BI was 46.7, and ZBI was 32.7. Mean informal care time was 51.3 h/week. The mean total annual cost per patient (reference year 2012) was €24,520 in Hungary, €25,993 in France, €84,921 in Italy, €94,384 in Spain, and €209,420 in Germany. Costs are also shown to differ between children and adults. Direct costs accounted for most of the costs in all five countries (80, 100, 99, 98, and 93 %, respectively).Conclusions: MPS patients experience substantial loss of HRQOL and their families take a remarkable part in their care. Although utilization of health and social care resources varies significantly across countries, MPS incurs considerable societal costs in all the countries studied. [ABSTRACT FROM AUTHOR]

Objective: The aim of this study was to determine the economic burden from a societal perspective and the health-related quality of life (HRQOL) of patients with histiocytosis in Europe.Methods: We conducted a cross-sectional study of patients with histiocytosis from France, Germany, Italy, Spain, Bulgaria, the UK, and Sweden. Data on demographic characteristics, health resource utilisation, informal care, loss of labour productivity and HRQOL were collected from the questionnaires completed by patients or their caregivers. HRQOL was measured with the EuroQol 5-domain (EQ-5D) questionnaire.Results: A total of 134 patients (35 France, 32 Germany, 30 Italy, 24 Spain, 7 Bulgaria, 4 UK and 2 Sweden) completed the questionnaire. The average annual costs ranged from € 6832 to € 33,283 between countries, the year of reference being 2012. Estimated direct healthcare costs ranged from € 1698 to € 18,213; direct nonhealthcare costs ranged from € 2936 to € 17,622 and labour productivity losses ranged from € 1 to € 8855. The mean EQ-5D score for adult histiocytosis patients was estimated at between 0.32 and 0.85, and the mean EQ-5D visual analogue scale score was estimated at between 50.00 and 66.50.Conclusion: The main strengths of this study lie in our bottom-up approach to costing and in the evaluation of histiocytosis patients from a broad perspective (societal costs). This type of analysis is very scarce in international literature for rare diseases in comparison with other illnesses. We conclude that histiocytosis patients incur considerable societal costs and experience substantial deterioration in HRQOL. [ABSTRACT FROM AUTHOR]

Purpose: In Norway, 19 % of revisions of non-resurfaced total knee arthroplasties done for knee pain between 1994 and 2011 were Secondary Patella Resurfacing (SPR). It is, however, unclear whether SPR actually resolves the pain. The aim was to investigate prostheses survival and clinical outcomes following SPR.Method: A total of 308 knees (301 patients) with SPR were used to assess implant survival, and a sub-cohort (n = 114 out of 301 patients) with Patient Reported Outcome Measures (PROMs) data were used to assess the clinical outcomes. The EuroQol (EQ-5D), the Knee Injury and Osteoarthritis Outcome Score, and Visual Analogue Scales on satisfaction and pain were used to collect PROM data. Outcomes were analysed by Kaplan-Meier, Cox regression, and multiple linear regression.Results: The five- and ten-year Kaplan-Meier survival percentages were 91 % and 87 %, respectively. Overall, 35 knees were re-revised at a median follow-up of eight years and pain alone (10 knees) was the main cause of re-revision. Younger patients (<60 years) had nearly nine times higher risk of re-revision compared to older patients (>70 years) (RR = 8.6; p < 0.001). Mean EQ-5D index score had improved from 0.41 (SD 0.21) preoperative to 0.56 (SD 0.25) postoperative following SPR. A total of 63 % of patients with PROM data were satisfied with the outcomes of SPR.Conclusion: The long-term prostheses survival following SPR was satisfactory, although not as good as for primary knee replacement. Patients' health related quality of life improved significantly following SPR. Still, more than a third of patients with PROMs data were dissatisfied with the outcomes of the SPR procedure. [ABSTRACT FROM AUTHOR]

Objective: The objective of this study was to evaluate changes in health related quality of life (HRQoL) in patients with metastatic head and neck (H&N) cancer randomized to receive metronomic (methotrexate and celecoxib) or cisplatin chemotherapy.Methods: Patients older than 18 years, with a Karnofsky Performance score of ≥70, and diagnosed with metastatic, locally advanced inoperable or recurrent head and neck (H&N) cancer not amenable to surgery or radiation were randomized (1:1) to receive metronomic or cisplatin chemotherapy. All patients were recruited from the Tata Memorial Hospital, Mumbai, India. In addition to demographic and baseline clinical characteristics, patients were asked to rate their HRQoL using the EORTC QLQ-C30 and the EORTC QLQ-H&N35 questionnaires (Indian versions) at baseline and at the end of each chemo cycle (every 3 weeks) till the end of study or early termination.Results: Of the 110 patients screened, 87 agreed to participate in the study. Mean age of the study population was 47.5 years (S.D. ±10.04) for the metronomic group and 47.2 years (S.D. ±9.89) for the cisplatin group. Overall quality of life was not significantly different between the two treatment groups from baseline to end of treatment. However, there was a statistically significant improvement in Pain QLQ-C30 score from baseline to week 3 (OR = 3.14, p = 0.036) and week 6 (OR = 3.33, p = 0.034) in the metronomic arm compared with the cisplatin arm.Conclusion: In addition to improvements in survival, understanding the impact of treatment options on changes in HRQoL is important as it can aid physicians in making treatment and rehabilitation decisions for patients with advanced inoperable H&N cancer. [ABSTRACT FROM AUTHOR]

Purpose: The chronic liver disease questionnaire (CLDQ) is a frequently used liver-specific quality of life instrument, but it does not provide information on preference-adjusted health status, which is essential for cost-utility analysis. We aimed to develop a mapping function deriving utilities from the CLDQ in primary sclerosing cholangitis (PSC).Methods: Short form-6D (SF-6D) utilities were calculated from SF-36 data collected in a recent prospective study in which unselected patients with PSC also completed the CLDQ. Ordinary least squares (OLS), generalized linear, median, and kernel regression analyses were employed to devise a mapping function predicting utilities. This was validated in three random subsamples of the cohort and in a separate sample of PSC patients following liver transplantation. Adjusted R (2) and root-mean-square error (RMSE) as well as Pearson's r coefficients and mean absolute errors between predicted and observed values were used to determine model performance.Results: Decompensated liver disease and fatigue, systemic symptoms, and emotional distress, assessed with the CLDQ, were related to worse SF-6D utilities. The final OLS prediction model explained 66.3 % of the variance in the derivation sample. Predicted and observed utilities were strongly correlated (r = 0.807, p < 0.001), but the mean absolute error (0.0604) and adjusted RMSE (10.6 %) were of intermediate size. Similar model characteristics were observed after employment of generalized linear and median regression models and at validation.Conclusions: A model has been constructed, showing good validity predicting SF-6D utilities from CLDQ scores at the group level in PSC. Further testing is required to externally validate the model. [ABSTRACT FROM AUTHOR]

Introduction: Assessing the consequences of chronic spontaneous/idiopathic urticaria (CSU) requires the evaluation of health-related quality of life (HRQoL) associated with the severity of CSU signs and symptoms. It is important to understand how signs, symptoms, and HRQoL change over time in CSU. Evidence is lacking on how closely changes in signs and symptoms of CSU are related to changes in HRQoL. The objective of this study was to assess the correlation between changes in patient-reported outcome measures (PROMs) of signs and symptoms, dermatologic quality of life (QoL), and urticaria-specific QoL.Methods: Latent growth models (LGMs) were applied to longitudinal data from three randomized, Phase 3 clinical trials investigating the efficacy and safety of omalizumab in CSU.Results: A near-perfect association between changes in signs and symptoms and changes in dermatologic and urticaria-specific QoLs was identified in each clinical trial when using LGMs (correlation coefficient range 0.88-0.92).Conclusion: Evidence showed that changes in signs and symptoms are closely related to changes in HRQoL. However, analyses were performed on clinical trial results of an extremely effective treatment; a less effective treatment with much smaller changes over time may not show such close correlations. Results suggest that any of these PROMs may be used to understand changes in CSU. [ABSTRACT FROM AUTHOR]