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Background: For many decision makers in Health Technology Assessment the EQ-5D-5L is the standard measure of health-related quality of life (HRQL). However, evidence has shown the limitations of the measure in certain disease areas, including dermatology. Alopecia areata (AA) is associated with a significant HRQL impact, partly due to the emotional impact of hair loss. Objectives: This study explores the psychometric properties of the EQ-5D-5L in people with AA in reference to the short-form 36 version 2 (SF-36v2), the Alopecia Areata Patient Priority Outcomes (AAPPO), the Severity of Alopecia Tool (SALT) and the Patient Global Impressions of Change (PGI-C). Methods: Data from participants with AA enrolled in the ALLEGRO-2b/3 trial (NCT03732807) of ritlecitinib were analysed. Participants completed the AAPPO measure (an AA-specific measure assessing emotional symptoms and activity limitations), PGI-C, EQ-5D-5L and SF-36v2 across 48-weeks of follow up. Extent of scalp hair loss was assessed using the SALT. Ceiling effects, known groups validity, convergent validity and responsiveness were examined. Known groups were defined by SALT score and a PGI-C defined response from baseline. Exploratory factor analysis was also performed. Results: Data were available from 612 adult participants. Ceiling effects were observed for the EQ-5D-5L (55.3–61.2%) and analyses suggested that the EQ-5D did not capture important differences between patients that the SF-36v2 did. The EQ-5D-5L very weakly correlated with SALT score, whereas the AAPPO correlated more strongly with the extent of hair loss. Compared with the EQ-5D-5L, the AAPPO was better able to discriminate between known groups defined by SALT and PGI-C. An exploratory factor analysis suggested that the EQ-5D-5L had limitations in content validity compared with the AAPPO. Conclusions: The EQ-5D-5L may not adequately measure the burden of AA on patients' HRQL. Insensitivity to the burden of AA suggests that the EQ-5D-5L may not measure treatment-related benefit with hair regrowth. Data from other measures could be considered if they are shown to be more relevant. Plain Language Summary (PLS): Do frequently used health-related quality of life questionnaires accurately measure the impact of alopecia areata? Alopecia areata (AA) is a disease that causes hair loss on the scalp and, in some cases, other parts of the body. It affects 18.4 million people worldwide. We know that AA can have a significant impact on a person's health-related quality of life (HRQL). Understanding the impact of AA on HRQL is important, but frequently used questionnaires to assess HRQL may not accurately measure the impact of the condition. This study uses data from a clinical trial (ALLEGRO-2b/3 trial) conducted in patients with AA from multiple countries to examine whether frequently used HRQL questionnaires can measure the impact of AA. We compared the HRQL of people with different levels of hair loss to see how well these questionnaires measure the impact of AA. We used data from 612 participants who took part in the trial. We found that some of the frequently used questionnaires did not detect differences between people with different levels of hair loss or those who thought their condition had improved compared with those who did not, suggesting that they may not accurately measure the impact of AA. Overall, some frequently used questionnaires to assess HRQL may not be appropriate for use in people with AA. Other ways of measuring HRQL may be more appropriate for understanding the full impact of AA. [ABSTRACT FROM AUTHOR]

Introduction: The EQ Health and Wellbeing (EQ-HWB) is a new questionnaire for measuring quality of life (QoL) from a broad perspective. The items of the EQ-HWB were derived based on a 'qualitative review' of literature, which reported primarily on Western studies. It can be argued that the QoL is a cultural-related concept and therefore people from China have a different understanding of the QoL. This study aimed to explore whether Chinese citizens could understand the EQ-HWB's candidate items and what they thought of those items. In doing so, we wanted to examine the face validity of the candidate items and explore if further cultural adaptation is necessary. Methods: This research was part of the E-QALY project, in which 36 candidate items were selected for the EQ-HWB from a 97-item pool. In China, three interviewers investigated the face validity of these EQ-HWB candidate items in semi-structured qualitative face-to-face interviews. Respondents were invited to report 'problems' with regard to the interpretation of the items and these problems were grouped into themes. We explored to what extent those themes related to specific cultural aspects in China. We also classified the rates of reported problems for each item into three groups: 1) less than 20%, 2) from 20–50%, and 3) over 50%. Results: For 17 items the rate of reported problems was less than 20%, 15 items fell into the second group (with 20 − 50%) and for 4 items the rate of problems reported was more than 50%. The thematic analysis revealed eight themes: ambiguous problems in the interpretation of 16 items; difficult to understand (11); contained a complex negative expression (10); examples used seemed inappropriate (7); misleading connotation in Chinese (2); long and complex (2); complex response options (1); and use of non-colloquial language (1). Discussion: Our research shows that EQ-HWB candidate items require careful examination to make them more comprehensible. Most of the reported problem themes were generic problems related to the items, and only a few face validity issues appeared to relate to specific cultural aspects in China, even though most of the items were based on Western studies. Our findings are reassuring for the instrument's international application, especially in China. [ABSTRACT FROM AUTHOR]

Background: The St. George's Respiratory Questionnaire (SGRQ) and its idiopathic pulmonary fibrosis (IPF) version (SGRQ-I) are widely used to assess health-related quality of life in IPF. However, the psychometric properties of these questionnaires in the IPF population have not been thoroughly evaluated. This systematic review assessed and summarized the available evidence on the psychometric properties of the SGRQ and SGRQ-I in IPF patients. Methods: Seven electronic databases were searched to identify relevant studies. The eligibility criteria included full-text studies focusing on the psychometric properties of the SGRQ and SGRQ-I in patients with IPF. The risk of bias, criteria for good measurement properties, and strength of evidence were assessed based on the COnsensus-based Standards for the selection of health Measurement INstruments (COSMIN). The inverse-variance heterogeneity (IVhet) model was used to pool results for construct validity. Results: A total of 24 studies were included; 19 assessed the psychometric properties of the SGRQ, and seven evaluated the SGRQ-I. Structural validity was assessed in one study for the SGRQ-I. Most domains of the SGRQ and SGRQ-I, except for the symptom domain of the SGRQ, had acceptable internal consistency. Both questionnaires demonstrated adequate test–retest reliability, known-groups validity, and responsiveness. Regarding construct validity, there were moderate to strong correlations with respiratory-specific measures, yet weaker correlations were observed with pulmonary function tests. Interpretability analysis revealed minor floor and ceiling effects. Conclusion: The SGRQ and SGRQ-I have the potential to capture essential dimensions of health-related quality of life (HRQoL) in IPF. However, further research should focus on ensuring that the items and domains of these questionnaires truly reflect the unique challenges and experiences faced by patients with IPF through content validity studies. [ABSTRACT FROM AUTHOR]

Background: The EuroQol Group recently developed two new instruments, the EQ Health and Wellbeing (EQ-HWB) and the EQ Health and Wellbeing short version (EQ-HWB-S). The EQ-HWB and EQ-HWB-S are intended to capture a broad range of health and broader quality of life aspects, which may be relevant to general public members, patients, their families, social care users and informal carers. This study assesses the content validity of the Italian version of the two instruments in a sample of Italian patients, social care users and informal carers. Methods: Participants were recruited using a convenience sampling approach. One-on-one interviews were carried out using video-conferencing interviews. A semi-structured topic guide was used to guide the interview procedures, with open-ended questions supplemented by probes. Participants were asked to explain important aspects of their health and quality of life, to complete the questionnaires and verbalize their thoughts. Results: Twenty participants comprising of patients (n = 9), informal carers (n = 6), and members of the general public (n = 5) participated to the study. Content validity was summarized into six main themes: comprehension, interpretation, acceptability, relevance, response options and recall period. All participants found the instruments easy or quite easy to understand and to respond to. Items were relevant for all three groups of participants, and response options appropriate. Conclusions: The Italian version of the EQ-HWB showed content validity in measuring health and wellbeing in a mixed Italian population. [ABSTRACT FROM AUTHOR]

Background: Half of the patients with Neurofibromatosis type 1 (NF1) develop one or more tumours called plexiform neurofibromas, which can have a significant impact on Quality of Life (QoL). The PlexiQoL questionnaire is a disease-specific QoL measure for adults with NF1-associated plexiform neurofibromas. The aim of this study was to adapt and validate a Dutch version of the PlexiQoL for the Netherlands. Methods: The PlexiQoL was translated using the dual-panel methodology, followed by cognitive debriefing interviews to assess face and content validity. The psychometric properties were evaluated by administering the questionnaire on two separate occasions to a sample of adults with NF1 and plexiform neurofibromas. Feasibility was evaluated by the presence of floor/ceiling effects. Reliability was assessed by evaluating Cronbach's alpha coefficient and test-retest reliability, using Spearman's rank correlation coefficients. Mann-Whitney U tests were used to check for known group validity. The Nottingham Health Profile (NHP) questionnaire was used as comparator questionnaire to evaluate convergent validity. Results: The translation and cognitive debriefing interviews resulted in a Dutch version of the PlexiQoL that reflected the original concept and underlying semantic meanings of the UK English version. Forty participants completed the validation survey. The Dutch PlexiQoL demonstrated excellent internal consistency (Cronbach's α 0.825) and test-retest reliability (Spearman correlation coefficient 0.928). The questionnaire detected differences in PlexiQoL scores between participants based on self-reported general health and disease severity. Convergent validity was confirmed for relevant NHP subsections. Conclusions: The Dutch PlexiQoL demonstrated excellent psychometric properties and can be reliably used to measure plexiform neurofibroma-related QoL in adults with NF1 in the Netherlands. [ABSTRACT FROM AUTHOR]

Introduction: Port-wine stains (PWS) are congenital capillary abnormalities caused by immature, venule-like vasculature that progressively dilates due to poor endothelial cell differentiation. PWS affects between 0.3% and 0.9% of newborns, with 90% of cases occurring on the face. Individuals with facial PWS and their parents had a significant negative impairment on their quality of life (QoL) and also suffered from psychological disabilities. Methods: This was a cross-sectional questionnaire-based survey study in Thailand from July 2021 to April 2022. The questionnaires included demographic data, subjective evaluation (SE), and the Dermatology Life Quality Index (DLQI). The questionnaire was performed with a full scale and adjusted scale of validity and reliability test of DLQI using factor analysis and Cronbach's alpha. The study outcome was a subjective evaluation and DLQI in patients who received pulsed dye laser (PDL) treatment. Results: Of the 54 patients, 35.2% (19) are male, and 64.8% (35) are female. Regarding age groups, 64.8% (35) are below 5 years old, and 35.2% (19) are older than 5 years. SE results showed that males evaluated an improvement of the facial PWS lesion significantly better than females (P < 0.05). The older age group graded the percentage of improvement better than the younger age group (P < 0.01). The result of the DLQI showed no difference in DLQI between gender. Older age result resulted in a significantly different DLQI compared with younger age (P < 0.01). Parent-reported DLQI improvement was less than self-reported DLQI improvement in patients with PWS treated with PDL (P < 0.05). Concerning the instrument of the study, the validity and reliability analysis of the DLQI questionnaire using factor analysis and Cronbach's alpha have been performed. The adjusted scale with the 5-item DLQI questionnaire is more appropriate in terms validity and reliability. Conclusion: This study demonstrates that facial PWS reduces the QoL as measured by DLQI. We discovered that the QoL of patients and parents with PWS was significantly impaired. The main influencing factors were older age, the improved perception between gender, and PDL treatments. In addition, we found only five questions that are reliable for PWS. The adjusted five-item DLQI questionnaires are more appropriate regarding validity and reliability. Trial Registration Number: TCTR20230210001, COA no. si 1059/2020. [ABSTRACT FROM AUTHOR]

The purpose of the present study is to develop and validate the hypertension scale of the Quality of Life Instruments (QoL) for Chronic Diseases system, QLICD-HY (V2.0). The QLICD-HY (V2.0) was developed via a programmed decision method with several focus groups, nominal discussions and pilot testing. The data was collected from 370 hypertensive inpatients and measured their QoL three times before and after treatment. Using correlation, factor analyses, as well as t-tests, the psychometric properties of the scale were assessed with regard to validity, reliability and responsiveness. Correlation and factor analysis supported good construct validity and criterion-related validity when using Short Form 36 as a criterion. Test–retest reliability coefficients for the overall scale score and all domains, with the exception of the psychological and social domain (0.77, 0.78), were greater than 0.80, with a range of 0.77–0.92. The internal consistency for all domains was higher than 0.70. With the exception of the psychological domain and social domain, the overall score and scores for the majority of aspects within each domain underwent statistically significant changes (t-tests) after the treatment. The QLICD-HY (V2.0) has good validity, reliability and responsiveness and can be used as a QoL measure for hypertensive patients. [ABSTRACT FROM AUTHOR]

Introduction: Sleep is often disturbed in patients with prurigo nodularis (PN). To address the lack of validated patient-reported outcome (PRO) measures for quantifying sleep disturbance in PN, we evaluated the Sleep Disturbance Numerical Rating Scale (SD NRS) as a single-item PRO measure for quantifying sleep disturbance in PN. Methods: Adults with PN participated in qualitative interviews, which included concept elicitation and cognitive debriefing of the SD NRS. The SD NRS was evaluated psychometrically using data from a phase 2 randomized trial in adults with PN (NCT03181503). Other PRO assessments included the Average Pruritus (AP) NRS, AP Verbal Rating Scale (VRS), peak pruritus (PP) NRS, PP VRS, and Dermatology Life Quality Index (DLQI). Reliability, validity, and responsiveness of the SD NRS were evaluated, and meaningful within-patient change was estimated from qualitative interview responses and quantitative trial data. Results: All interview participants (N = 21) experienced sleep disturbance and most (95%) understood the SD NRS as intended. The SD NRS demonstrated test–retest reliability based on intra-class correlation coefficients for itch-stable participants of 0.87 for the AP VRS and 0.76 for the PP VRS. At baseline, Spearman's rank-order correlation coefficients were moderate to strong (0.3–0.8) between the SD NRS and the AP NRS, AP VRS, PP NRS, PP VRS, and DLQI. Known-groups validity was demonstrated by higher (worse) SD NRS scores in participants with worse scores on the AP NRS, AP VRS, PP VRS, and DLQI. Improvements in SD NRS scores were greater in participants classified as "improved" versus "worsened/unchanged" on the anchor PROs. A 2- to 4-point decrease on the 11-point SD NRS scale was identified as a meaningful within-patient change. Conclusion: The SD NRS is a well-defined, reliable, and valid PRO measure that can be used in daily practice and clinical trials to capture sleep disturbance in adults with PN. Plain Language Summary: Patients with the skin disease prurigo nodularis often sleep badly because their skin is very itchy in the evening and at night. In assessing the effectiveness of new treatments for prurigo nodularis, it is useful to know whether patients sleep better when taking them. Here, we tested whether the "Sleep Disturbance Numerical Rating Scale" (SD NRS), a tool for measuring sleep, can be used by patients with prurigo nodularis. The SD NRS asks patients to score their sleep loss during the previous night on a scale of 0 ("no sleep loss") to 10 ("I did not sleep at all"). When we interviewed 21 patients with prurigo nodularis, 19 of them had problems falling asleep and 18 of them woke up every night. Most of the patients found the SD NRS easy to use. To further explore whether the SD NRS is suitable for patients with prurigo nodularis, we also analyzed data from a European clinical study in which patients with prurigo nodularis received nemolizumab, a new treatment being developed for the disease. Patients completed the SD NRS each morning during the clinical study. We found that the SD NRS has the necessary properties to be useful for assessing sleep on a daily basis. We also determined that a 2- to 4-point decrease on the SD NRS scale would represent a meaningful improvement in sleep for patients with prurigo nodularis. We conclude that the SD NRS is a useful tool for assessing sleep in prurigo nodularis patients participating in clinical studies. [ABSTRACT FROM AUTHOR]

The aim of the present study was to translate into Spanish and evaluate the psychometric evidence of the Impact on Quality of Life (COV19-QoL) applied to a sample of Peruvian older adults (N = 298; 58.1% women, 41.9% men, mean age 65.34 years [SD = 11.33]). The study used techniques from the Classical Test Theory (CTT) and Item Response Theory (IRT). The findings confirmed the single factor structure of the COV19-QoL, high internal consistency reliability, measurement invariance by gender, and all items demonstrated adequate discrimination and difficulty indices. In this sense, the items allow adequate discrimination between low, medium and high levels of the impact of the COVID-19 pandemic on quality of life. In addition, a greater perceived impact of the pandemic on quality of life is necessary to answer the higher response options of the COV19-QoL. In conclusion, the COV19-QoL is a valid measurement scale of the impact of the COVID-19 pandemic on the quality of life of Peruvian older adults. [ABSTRACT FROM AUTHOR]

Individuals with stroke are at high malnutrition risk in both the acute and chronic phases. This study aimed to assess the validity of different malnutrition screening tools for stroke patients in rehabilitation phase. Participants in this study were 304 stroke patients from three hospitals in the East-Coast region of Peninsular Malaysia from May–August 2019. The concurrent validity of the Malnutrition Risk Screening Tool-Hospital (MRST-H), Mini Nutritional Assessment-Short Form (MNA-SF), Malnutrition Screening Tool (MST), Malnutrition Universal Screening (MUST) and Nutritional Risk Screening (NRS-2002) was assessed with the diagnostic criteria for malnutrition proposed by the Global Leadership Initiative on Malnutrition (GLIM-DCM). Sensitivity, specificity, positive predictive value, negative predictive value, and the area under the curve were computed. MUST and MRST-H demonstrated good validity regardless of different age groups (> 80% sensitivity and specificity); meanwhile, MST and MNA-SF had fair validity, yet NRS-2002 had poor to fair validity with GLIM-DCM. Only MRST-H and NRS-2002 were significantly correlated with all anthropometric indices, dietary energy intake, and health-related quality of life in both age groups. In conclusion, MRST-H and MUST showed good concurrent validity with GLIM-DCM and can be considered as appropriate malnutrition screening tool in discriminating malnutrition among stroke individuals attending rehabilitation centre in Malaysia regardless of their age groups. [ABSTRACT FROM AUTHOR]

The present study examined the psychometric properties of the 12-item Stroke-Specific Quality of Life Scale (SSQOL-12) in 184 stroke survivors in Hong Kong. The participants completed a self-report questionnaire including the SSQOL-12, SF-12 health survey, and validating variables at baseline and 148 stroke survivors completed SSQOL-12 two months later. Confirmatory factor analysis investigated the construct validity, reliability, and measurement invariance of SSQOL-12 across two months. Concurrent, convergent, and divergent validity was examined with respect to SF-12, hope, self-esteem, functional disability, anxiety, and depression. The original 2-factor model did not reveal a superior fit over the 1-factor model and a modified 1-factor model provided an acceptable fit to the data in both waves. The SSQOL-12 factor displayed substantial factor loadings (λ = 0.40–0.87), good internal consistency (Ω = 0.88), temporal stability (r = 0.70), and scalar measurement invariance across time. Stroke-specific quality of life was significantly associated with higher levels of SF-12, hope, and self-esteem and lower levels of functional disability, anxiety, and depression. Most correlations remained significant after controlling for demographic covariates and SF-12. The present findings offered empirical support for the validity and reliability of the unidimensional structure for SSQOL-12 as a measure of stroke-specific quality of life among stroke survivors. [ABSTRACT FROM AUTHOR]

The purpose of this review was to identify cross-cultural and psychometric characteristics of the European Organization for Research and Treatment of Cancer Head and neck cancer questionnaire (EORTC QLQ-H&N35) in various languages. A literature search was performed for original papers in PubMed, EMBASE, and Google scholar electronic databases on validation, psychometric properties of the EORTC-H&N35 questionnaire for patients with head and neck cancer. A total of 17 papers had been reviewed systematically. The studies were conducted in 28 countries and EORTC QLQ-H&N35 questionnaire was validated in 21 different languages. The majority of papers reported high reliability having Cronbach's coefficient above 0.70. Low reliability was reported for senses and speech problems, pain, and less sexuality. Moderate to good convergent validity was found as the correlation coefficient was above 0.40 except for speech problems and social contact trouble. Discriminant validity (weak correlations < 0.70) was confirmed in 14 papers. This review provides comprehensive information on cross-cultural and psychometric properties of EORTC QLQ-H&N35 and can be recommended to implement in oncological practice. [ABSTRACT FROM AUTHOR]

Background: This study aimed to translate and validate an Arabic version of the Functional Assessment of Cancer Therapy Head and Neck Scale (FACT-H&N, v-4) for use among Sudanese oral cancer patients. Methods: The instrument underwent translation and validation following the standard FACT translation methodology. The translated instrument was pre-tested for face validity and content validity using semi-structured, in-depth interviews with ten oral cancer patients to assess acceptability. The questionnaire was pilot tested with 60 patients; reliability was tested for internal consistency with Cronbach's alpha while construct validity was tested using 'known-group validity'. Results: The pre-test study revealed no major issues, apart from a reluctance to answer questions on sexual satisfaction. The FACT-H&N demonstrated good internal consistency, it considered five particular constructs: PWB, SWB, EWB, FWB and FACT-H&N, their Cronbach's α values were positive and close to 1 with values of 0.85, 0.788, 0.86, 0.895 and 0.703 respectively, indicating that the questionnaire was valid and the responses consistent. Sixty patients were asked the global health-related quality of life item, 36.3% rated their QOL as very good or good (36.3%), while 41.7% rated it as average, and 21.7% as poor or very poor. Then FACT subscale mean scores were tabulated against three categories; patients with very poor/poor recorded significantly lower scores indicating construct validity. Some psychometric properties were consistent with other FACT-H&N translations such as the Chinese, French, Pakistani and Malaysian. Conclusions: This study validates the Arabic version of the FACT-H&N. It is a reliable tool and, will assist further QoL research in other Arabic-speaking countries. Plain English Summary: While survival rates are routinely used to evaluate treatment outcomes in oral cancer, increased understanding of the human cost of this disease highlights the need to also consider the impact of the disease and therapy on patients' quality of life (QoL). Measurement of quality of life outcomes requires a validated QoL questionnaire with proven applicability in that specific context. Little research has been undertaken on the QoL of cancer patients in Sudan and no published research exists to date on the QoL of Sudanese oral cancer patients. While an Arabic version of the FACT-G general questionnaire exists, the FACT-H&N module was not previously available in Arabic. This paper describes the translation of the FACT-H&N from English into Arabic to suit Sudanese patients and other Arabic-speaking patients, with a detailed pre-test review by 10 patients, a pilot study with 60 patients and further validation with 130 patients. This study represents the first QoL research undertaken in oral cancer patients in Sudan and provides some insight into the impact of oral cancer on their lives. Findings from this study indicate that the Arabic version of the FACT-H&N questionnaire is a valid, reliable and acceptable method for assessing health related QoL in Arabic-speaking Sudanese oral cancer patients. This work will benefit future researchers working with Arabic-speaking oral cancer patients. [ABSTRACT FROM AUTHOR]

Introduction: Vitiligo can be associated with a psychological burden, stigmatization and impaired quality of life. Tools to assess the impact of vitiligo exist; however, none were developed in line with the FDA's patient-reported outcome (PRO) Guidance for Industry. This study aimed to explore the content validity of two newly developed PRO measures to assess the impact of facial and total body vitiligo on how patients feel and function. Methods: Draft PRO measures were developed from existing literature and input from PRO experts, a patient advocate and a clinical expert. Qualitative interviews were conducted with US participants living with vitiligo and international dermatologists with vitiligo expertise. Concept elicitation methodology explored the relevance of concepts in the draft PRO, while cognitive debriefing assessed conceptual relevance and understanding/interpretation. Items were iteratively amended/added throughout the interview study. Results: The 60 participants included adults (n = 48, 63% female, 18–62 years old) and adolescents (n = 12, 67% female, 12–17 years old) with Fitzpatrick Skin Types I–VI. Expert dermatologists from the US (n = 8), EU (n = 4), India (n = 1) and Egypt (n = 1) participated. Concept elicitation was utilized to confirm the signs/symptoms of vitiligo and the associated impact on emotional/psychological wellbeing, social functioning, daily life and work/school. Conceptual saturation was achieved. Most participants reported impacts on their emotional/psychological wellbeing (n = 57, 95%), e.g. feeling self-conscious (n = 35, 58%). Participants reported impacts on social functioning (n = 53, 88%), e.g. vitiligo being noticed by others (n = 42, 70%). There was general consensus between participants and expert dermatologists. Cognitive debriefing confirmed that the items were well understood. Most items were conceptually relevant; feeling self-conscious and feeling frustrated were highly endorsed. Items were removed based on low conceptual relevance (feeling abandoned, skin roughness) and expected redundancy (four items), resulting in two measures with three proposed domain scores: Emotional/Psychological Wellbeing; Social Functioning; and Physical Sensation. No comprehension concerns were observed in relation to the 7-day recall period or the item response scale/options. Eight dermatologists reviewed the PRO measures, confirming comprehensiveness and relevance. Conclusion: The draft Vitiligo Patient Priority Outcomes (ViPPO) measures evaluate the impact of facial (ViPPO-F) and total body (ViPPO-T) vitiligo on emotional/psychological and social functioning. The ViPPO measures are well understood, comprehensive and content valid for adults and adolescents with vitiligo. [ABSTRACT FROM AUTHOR]

Introduction: This study explored patients' and dermatologists' priority outcomes for treatment to address, clinical outcome assessments (COA) for use in vitiligo clinical trials, and perceptions of within-patient meaningful change in facial and total body vitiligo. Methods: Semistructured, individual, qualitative interviews were conducted with patients living with non-segmental vitiligo in the USA and with expert dermatologists in vitiligo. Concept elicitation discussions included open-ended questions to identify patient priority outcomes. Vitiligo COAs were reviewed by dermatologists. Tasks were completed by patients to explore their perceptions of meaningful changes in vitiligo outcomes; dermatologists' opinions were elicited. Data were analyzed using thematic methods; meaningful change tasks were descriptively summarized. Results: Individuals with vitiligo (N = 60) included adults (n = 48, 63% female) and adolescents (n = 12, 67% female). All Fitzpatrick Skin Types were represented. Eight (13%) were first- or second-generation immigrants to the USA. Expert dermatologists (N = 14) participated from the USA (n = 8), EU (n = 4), India (n = 1), and Egypt (n = 1). All individuals with vitiligo reported experiencing skin depigmentation; an observable clinical sign of vitiligo. Most confirmed that lesion surface area (n = 59/60, 98%) and level of pigmentation (n = 53/60, 88%) were important to include in disease assessments. Following an explanation, participants (n = 49/60, 82%) felt that the Facial Vitiligo Area Scoring Index (F-VASI) measurement generally made sense and understood that doctors would use it to assess facial vitiligo. Most participants felt that a 75% (n = 47/59, 80%) or 9 0% improvement in their facial vitiligo would be indicative of treatment success (n = 55/59, 93%). In the context of evaluating a systemic oral treatment for vitiligo, dermatologists perceived a 75% improvement on the F-VASI as successful (n = 9/14, 64%). Regarding the Total VASI (T-VASI) score, n = 30 participants considered 33% improvement as treatment success; an additional n = 10 endorsed 50% improvement and a further n = 5 endorsed 75% improvement. Clinicians most frequently identified 50% (n = 6/14, 43%) or 75% (n = 4/14, 29%) improvement in T-VASI as successful. Conclusion: Repigmentation is a priority outcome for patients. The VASI was considered an appropriate tool to assess the extent of vitiligo. A minimum 75% improvement from baseline in the F-VASI and minimum 50% improvement from baseline in the T-VASI were identified as within-patient clinically meaningful thresholds. [ABSTRACT FROM AUTHOR]

Background: Childhood asthma is an important unmet need. To date, patient-reported outcome measures (PROMs) for children with asthma have used a combination of caregiver or proxy-reported and self-reported measures. No comprehensive measure is available to assess the severity and impact of daytime and nighttime asthma symptoms and rescue medication use for self-completion by children aged 6–11 years. This study aimed to develop a novel, interactive, electronic Pediatric Asthma Symptom Diary (ePASD) measuring self-reported key symptom severity and proximal impacts of asthma in young children with varying reading ability and disease severity, consistent with US Food and Drug Administration (FDA) PRO guidance and the International Society for Health Economics and Outcomes Research (ISPOR) good research practices. Methods: A targeted literature review and clinician interviews were undertaken to characterize symptoms and impacts experienced by children with mild-to-severe asthma. Concept elicitation interviews (CEIs) were conducted with 44 children and their caregivers (30 US; 14 UK). Following item and digital application development, the ePASD was assessed for relevance, understanding, and interpretability through cognitive debriefing interviews (CDIs) with 21 US children. Face validity/translatability assessments were also performed. Results: Key measurement concepts included cough, wheeze, difficulty breathing, chest tightness/discomfort, nighttime awakening, and daytime activity limitations. Concept saturation was reached during CEIs for primary asthma-related daytime and nighttime symptoms and core impacts. Most CDI participants found the ePASD items clear, understandable, and comprehensive. Standardized training is anticipated to facilitate reliable child self-report. Conclusion: The ePASD, a novel PROM for children aged 6–11 years with asthma, uses an innovative multimedia approach and has been developed in accordance with FDA PRO guidance and ISPOR good research practices, directly capturing the child's self-reported asthma symptoms, impacts on daily activities and nighttime awakening, and rescue medication use. [ABSTRACT FROM AUTHOR]

Background: Fatigue is a common and often disturbing sequela of serious chronic health conditions. In the widely applied HRQL instrument, the EQ-5D, this aspect is not included directly, for its assumed lack of additional information. We investigated the validity of this assumption by determining the gain—if any—of an additional fatigue item to the EQ-5D-5L in a general population sample. Methods: A Dutch general population sample (including diseased people) completed a web-based survey including the EQ-5D-5L and the Rivermead Post-Concussion Symptoms Questionnaire (RPQ). The RPQ fatigue item was used to create the EQ-5D-5L + Fatigue. We head-to-head compared the psychometric performance contrasting the EQ-5D-5L and EQ-5D-5L + Fatigue: distribution (e.g. ceiling), informativity cf. Shannon's indices, convergent validity, domain dependency, and explanatory power. Results were compared between subgroups with and without ≥ 1 chronic health condition. Results: The study population consisted of 3027 persons of whom 52% had a chronic health condition. The mean EQ-5D-5L utility score was 0.83 and 48% experienced some degree of fatigue. Adding the fatigue item to the EQ-5D-5L decreased the ceiling effect, increased absolute informativity (Hʹ = 6.44 vs. Hʹ = 4.90) and relative informativity (Jʹ = 0.46 vs. Jʹ = 0.42). The extra fatigue item slightly increased convergent validity (Spearman's rank correlation coefficient = − 0.61 vs. − 0.62). Domain dependency analysis showed that all EQ-5D-5L domains are dominant over the fatigue item. Explanatory power of the EQ-5D-5L + Fatigue was higher compared to the EQ-5D-5L (R2 = 0.42 vs. 0.39). The gain is substantially larger in the subgroup with chronic health conditions. Conclusions: Adding a fatigue item to the EQ-5D-5L improved all psychometric performance criteria of the enriched instrument in the general population. Effects are substantially larger in the subgroup with chronic health conditions, indicating that adding a fatigue item to the EQ-5D-5L is especially relevant in evaluating the HRQL of diseased people. [ABSTRACT FROM AUTHOR]

Background: There has been a growing emphasis on health-related quality of life (HRQoL) as an important outcome in rare disease drug development, although its assessment may be useful outside the drug development context, including in clinical applications or natural history studies. Central to assessing quality of life in health research is utilizing outcome measures that capture symptoms and impacts of the disease and treatment that are important and relevant to patients. Identifying and implementing valid and reliable tools to measure HRQoL in rare diseases poses unique challenges that often require creative solutions. Main body: In this commentary, we explore some of the challenges in HRQoL assessment in rare disease, propose solutions, and consider regulatory issues. Some of the solutions discussed entail the use of item banks, adapting existing measures from phenotypically similar disease contexts, use of multi-domain measurement indices, and adapting methods for assessing content validity of existing measures. Current regulatory considerations are discussed and resources outlined. Conclusion: Quality of life may be the most important endpoint for patients with rare diseases, and the challenges of valid assessment require effort and innovative thinking specific to each context to improve measurement and clinical outcomes. [ABSTRACT FROM AUTHOR]

Background: Fatigue is a key symptom in patients with systemic lupus erythematosus (SLE), and regulatory bodies recommend its assessment in clinical trials of SLE therapies. Methods: This post hoc pooled analysis of the three BeLimumab In Subjects with Systemic lupus erythematosus (BLISS) Phase 3 randomised, double-blind, parallel-group controlled trials evaluated the measurement properties of the Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue. Patients (N = 2520) completed the FACIT-Fatigue every 4 weeks from baseline until the end of each study period. Internal consistency, test–retest reliability, convergent validity, and ability to detect changes in SLE were evaluated for the FACIT-Fatigue. Results: The FACIT-Fatigue showed good internal consistency reliability (Cronbach's alpha > 0.90), very good test–retest reliability (0.76 ≤ intraclass correlation coefficient ≤ 0.92), and moderate-strong convergent validity (0.49 ≤ |r| ≤ 0.86) against scale and summary measure scores from the Short Form 36 Health Survey Version 2. Correlations between FACIT-Fatigue and British Isles Lupus Assessment Group (BILAG) General/Musculoskeletal scores (0.24 ≤ |r| ≤ 0.43) supported convergent validity. Correlations between FACIT-Fatigue and the Safety of Estrogens in Lupus Erythematosus National Assessment-Systemic Lupus Erythematosus Disease Activity Index (SELENA-SLEDAI) scores and SLE annualised flare rate were weak but in the expected direction (ranging from − 0.02 to − 0.25). Known-groups validity testing showed that the FACIT-Fatigue can significantly discriminate between patient groups with differing scores for SELENA-SLEDAI, BILAG (General and Musculoskeletal) ratings, and Physician's Global Assessment (PGA). Patients showing improvement in PGA and meeting the BILAG responder criteria had significantly higher mean improvement in FACIT-Fatigue scores than those without improvements in either measure (Week 52 mean score difference [95% confidence interval]: − 4.0 [− 5.0, − 3.0] and −2.2 [−3.1, −1.2], respectively; both p < 0.0001). The range of important (i.e. meaningful) change in FACIT-Fatigue, based on multiple anchors, was 3–6 points. Conclusions: The FACIT-Fatigue demonstrated adequate psychometric properties in patients with SLE. The body of evidence from the three BLISS trials (both pooled and individually) supports the FACIT-Fatigue as a reliable and valid measure of SLE-related fatigue in clinical trials. Clinical trial identifiers: BLISS-SC (NCT01484496), BLISS-52 (NCT00424476), and BLISS-76 (NCT00410384). [ABSTRACT FROM AUTHOR]

Introduction: Fatigue is highly prevalent and burdensome in systemic lupus erythematosus (SLE). The Functional Assessment of Chronic Illness Therapy-Fatigue (FACIT-Fatigue) is a patient-reported questionnaire that measures physical and mental fatigue and consequent impact on daily living. Qualitative evidence of content validity in SLE is limited. This study (GSK Study 209226) assessed the content validity of the FACIT-Fatigue for SLE and explored patients' experiences of SLE-related fatigue using qualitative methods. Methods: Fatigue-related themes were identified through semi-structured, hybrid cognitive debriefing and concept elicitation interviews and evaluated for concordance with the FACIT-Fatigue. Results: Fatigue was experienced regularly by all participants (N = 15, 86.7% female) and was rated as the most bothersome symptom of SLE by 11/15 participants. All participants reported emotional impacts of fatigue, while 14/15 and 9/15 participants also reported impacts on social life and physical functioning, respectively. Most (12/15) reported that fatigue interfered with their ability to fulfill work- or school-related roles, and activities of daily living were limited in all participants. All (14/14) reported that a meaningful change in their level of fatigue would be the ability to have a more active and normal lifestyle. Concept mapping showed that all 13 FACIT-Fatigue items mapped directly onto concepts spontaneously mentioned by participants. Cognitive debriefing revealed that 13/15 participants found the instructions easy to understand and 11/15 participants endorsed the recall period (7 days) as appropriate. Participants found the FACIT-Fatigue items were clear and relevant. Most participants (11/15) reported that all response options adequately captured their experience of fatigue. Conclusions: Qualitative evaluation of the content validity of the FACIT-Fatigue supports it as an appropriate measure for assessing the impact of fatigue on daily living of patients with SLE. The tool is easily understood by patients and a valuable resource for measuring a common and debilitating symptom of this condition. [ABSTRACT FROM AUTHOR]

Background: Pectus excavatum (PE) is the most common congenital chest wall deformity. Most individuals with PE suffer from psychosocial problems, with low self-esteem and poor body image. Correctional surgery for PE is available, the most widely used is the Nuss procedure. The Nuss procedure has previously been reported to improve self-esteem, body image and health-related quality of life (HRQoL). To assess HRQoL among individuals with PE, the Nuss Questionnaire modified for Adults (NQ-mA) and Single Step Questionnaire (SSQ) has been developed. The aim of the current study was to translate and culturally adapt NQ-mA and SSQ to fit a Swedish context, and to evaluate the psychometric properties, and validate the culturally adapted versions. Methods: Individuals who had undergone the Nuss procedure for pectus excavatum were invited to participate in a multicentre study with cross-sectional design. HRQoL was assessed by NQ-mA, SSQ and RAND-36. Psychometric properties for NQ-mA and SSQ were evaluated by content validity index and construct validity (exploratory factor analysis, confirmatory factor analysis, and inter-scale correlations). Known-groups validity, as well as floor and ceiling effects, were evaluated. Internal consistency reliability was examined using Cronbach's alpha. Results: In total 236 individuals participated in the study. Content validity index for NQ-mA showed satisfactory results, except for two items. Factor analysis for NQ-mA suggested a two-factor model, with exclusion of two items. Subscales correlated adequately with RAND-36's domains. Known-groups validity for NQ-mA demonstrated high sensitivity for between-group differences. Ceiling effects were present in several items. Cronbach's alpha for NQ-mA was.89. Confirmatory factor analysis for SSQ resulted in a three-factor model, with exclusion of five items. However, this model was shown to be unstable through further exploratory factor analysis testing, and no further psychometric tests were conducted for SSQ. Conclusion: The 10-item Swedish version of NQ-mA was shown to be valid for research and clinical assessment of HRQoL in individuals with pectus excavatum. The Swedish version of SSQ requires revision of items before further validation can be performed. [ABSTRACT FROM AUTHOR]

Background: Hemophagocyti.c lymphohistiocytosis (HLH) is a rare and severe disorder characterized by abnormal activation of the immune system. Primary HLH causes prolonged fever, spleen and liver enlargement, and organ dysfunction, usually in infancy and early childhood and is fatal if left untreated. As effective treatment options emerge, such as emapalumab-lzsg, Health Technology Assessment bodies around the world will assess them in terms of cost-effectiveness. This study was designed to estimate quality of life weights (utilities) for such analyses. Methods: Vignettes were developed describing HLH treatment related health states. Health states included active HLH, HLH plus neurological symptoms, receiving chemotherapy, undergoing stem cell transplant (SCT), graft versus host disease (GVHD), cure and end of life care. The vignettes were based on information from in depth interviews with clinical specialists; and qualitative research with four parents of children with primary HLH aged between 1 and 18 years old. The vignettes were then assessed in time trade off (TTO) interviews with members of the UK general public in one on one face to face interviews with trained, experienced interviewers. Preference data were analysed using the generalised estimating equations framework. Results: Detailed qualitative data captured the substantial burden of this disease for young children. One hundred participants completed the TTO interviews. The utility score for Active HLH was estimated as 0.32 (95% CI, 0.24 to 0.39). Values for other states were HLH plus neurological symptoms (0.27, 95%CI 0.18–0.35), receiving chemotherapy (0.26, 95%CI 0.17–0.34), undergoing SCT (0.18, 95%CI 0.07–0.28), GVHD (0.07, 95%CI -0.04-0.17), cure (0.72, 95%CI 0.67–0.77) and end of life care (− 0.17, 95%CI -0.27- -0.07). Conclusions: This study has estimated utility weights for seven different HLH related states which are based on detailed input from carers and physicians and have good face validity. There are few other options for collecting these data in an ultra-rare setting. [ABSTRACT FROM AUTHOR]

Background: In oncology practice, eliciting the patient's perspective on their quality of life (QOL) adds important information and value to their treatment and care. The European Organization for Research and Treatment of Cancer QOL questionnaire (EORTC QLQ-C30) is the most commonly used tool for this purpose but has not been validated in Kenya. The present study aimed to conduct a preliminary assessment of the QOL among Kenyan cancer patients and examine the psychometric properties of the tool in this population. One hundred patients with heterogeneous types of cancer were enrolled in this cross-sectional study between July and August 2019. The EORTC QLQ-C30 questionnaire was administered to patients using either the English or Kiswahili official version. Descriptive statistics were used to assess patient demographics and clinical characteristics. The psychometric properties of the EORTC QLQ-C30 were evaluated in terms of acceptability, internal consistency, and construct validity using statistical software packages, STATA and SPSS. Results: The EORTC QLQ-C30 was found to be acceptable for use in our patient population as indicated by high compliance and low missing responses. Of the 100 patients, 66 were able to self-administer the questionnaire. The average time for completion was 13 min. Preliminary QOL assessment indicated an average QOL in Kenyan cancer patients (53 ± 27). Among the function scales, participants scored the lowest on the social function scale (51 ± 36) whereas among the symptom scales, participants scored the highest on the financial difficulties scale (79 ± 31). Cronbach's alpha coefficient values ranged from 0.72–0.95, illustrating the reliability of the scales measured. Interscale correlations were statistically significant (p < 0.05), indicating clinical validity of the data collected. The magnitudes of the correlations between the physical functioning scale and the role functioning, pain, and fatigue scales were consistent with the values published in other studies across different geographical populations, further cross-validating the results from our study. Conclusion: The results from this study provide important first insights into using EORTC QLQ-C30 in the Kenyan population. We conclude that the questionnaire is an acceptable, reliable, and valid instrument for measuring the QOL in cancer patients in Kenya and recommend its use in clinical practice. [ABSTRACT FROM AUTHOR]

Background: Patients suffering from exsudative retinal diseases may experience severe central vision loss and this might have impact on their daily activities and quality of life. To measure the disabilities these patients may have, the use of the Impact of Vision Impairment Profile questionnaire is recommended. The aim of this study was to translate the original English 28-item Impact of Vision Impairment Profile (IVI) into the Dutch language and evaluate its comprehensibility, comprehensiveness and relevance as evidence of content validity. The translation process was performed using standardized methods. Content validity was assessed by cognitive debriefing using a Three-Step Test-Interview (TSTI) method for participants diagnosed with exudative retinal diseases. Step 1 and 2 focused on assessment of comprehensibility of items, step 3 on comprehensiveness and relevance. Audio-recorded qualitative data was analyzed using Atlas.ti. Data regarding comprehensibility problems was further categorized into item-specific problems and general problems. Results: Few minor discrepancies in wording were found after translation. After conducting 12 cognitive interviews, data saturation was reached. All participants reported comprehensibility problems resulting from specific items, these were; sentence structure, vocabulary and formulation, influence of conditions or composite items and influence of comorbid disorders. Several general comprehensibility problems resulting from instructions or response categories were detected. The main general comprehensibility problem resulted from the layout of the Dutch-IVI. Most participants considered the included items as relevant and indicated that they covered the problems that occur due to vision impairment. Conclusions: Minor problems in the Dutch translation were detected and adjusted. The layout and instructions of the Dutch-IVI resulted in some comprehensibility problems. The Dutch-IVI appeared to be at risk of being interpreted as a generic patient reported outcome measure, instead of a disease-specific instrument, mainly due to the influence of co-morbidities. Adaptations should improve validity and reliability of the Dutch-IVI, however, cross-cultural comparisons may be at stake. [ABSTRACT FROM AUTHOR]

Background: Patients with cystic fibrosis (CF) experience significant disease burden, including progressive pulmonary decline and reduced survival. This multicenter qualitative study was conducted to develop a new patient-reported outcome (PRO) measure to assess the impact of CF on patients' quality of life: the Cystic Fibrosis Impact Questionnaire (CF-IQ). Semi-structured qualitative concept elicitation (CE) interviews with patients and caregivers documented CF-related symptoms, impacts, and treatment experiences. Coded interview data were considered alongside existing PROs, published literature, and expert opinion to develop an initial scale. Three rounds of cognitive interviews evaluated respondent comprehension and facilitated refinement of the CF-IQ. Results: Adult (N = 20) and pediatric (N = 22) patients with CF and their parents/caregivers (N = 22) completed CE interviews at 7 US clinics. The sample included patients aged 6–58 years, 57% females, and represented a broad range of disease severity (forced expiratory volume in 1 s range: 22%–127% predicted). Interviews identified 59 unique CF-related impact concepts in domains, including activity limitations (physical, social, leisure), functional limitations (school, work), vulnerability/lack of control, emotional impact, treatment burden, and future outlook. Concept saturation was achieved, and a draft questionnaire was developed. Findings from the cognitive interviews (n = 18) confirmed that instructions, items, and response scales were relevant and clear, and interpreted as intended by patients. Conclusion: The CF-IQ is a 40-item novel PRO scale assessing a comprehensive set of patient-relevant concepts to characterize the multifaceted nature of CF. Qualitative interview data support the content validity of the CF-IQ, which is currently undergoing additional psychometric evaluation in patients with CF. [ABSTRACT FROM AUTHOR]

Background: Burns are a global health problem affecting the survivors and disrupting many aspects of their lives. It is the second most common injury in rural Nepal accounting 5% of disabilities. Burn Specific Health Scale (BSHS) is a valid and most commonly used tool to measure Health Related Quality of Life (HRQoL) of the patient with Burns. BSHS- B (Brief) has been translated, culturally adapted and validated in multiple languages but not in Nepali. Therefore we aim to translate, culturally adapt and validate the BSHS-B in Nepali language (BSHS-B-Np). Methods: Standard guideline was followed to translate the scale into Nepali language. One hundred eleven participants were evaluated to establish the psychometric properties of BSHS-B-Np. Internal consistency, test retest, content validity, discriminant validity and construct validity were assessed using Cronbach's alpha, Interclass correlation coefficient, Factor analysis, Spearman rank test, and Mann- Whitney U test respectively. Results: The Cronbach's alpha for BSHS-B-Np was 0.93. Test retest inter-class correlation coefficient was between 0.92 and 0.98. The principal component factor analysis with varimax rotation resulted in separation of nine factors explaining 75.19% of total variance. BSHS-B-Np showed good discriminant validity in 35 out of 36 domain correlations confirming the construct of the scale. Furthermore, the scale was able to discriminate between face, upper limb and lower limb injury (p < 0.05). Conclusions: BSHS-B-Np is a reliable and valid scale for Nepali burns survivors to assess their health related quality of life. [ABSTRACT FROM AUTHOR]

Background: The FACT-8D is a new cancer-specific, preference-based measure (PBM) of health, derived from the Functional Assessment of Cancer Therapy – General (FACT-G) questionnaire. The FACT-8D's measurement properties have not been tested to date. We assessed it's validity and responsiveness in relapsed/refractory mantle cell lymphoma (RR MCL) and compared the results to the EQ-5D-5L. Methods: Blinded analysis of pooled data from a phase 3 clinical trial. FACT-8D baseline and follow-up data (weeks 4, 7, 16, 31) were scored using Australian preference weights, the first available value-set. Convergent validity was assessed by estimating baseline correlations with the FACT-Lym total score, Trial Outcome Index (TOI), FACT-Lym lymphoma-specific sub-scale (LymS), EQ-5D Visual Analog Scale (VAS), and haemoglobin (HgB). Relevant clinical variables were used to categorise patients to test known groups' validity and responsiveness was investigated using data from baseline (n = 250) and week 31 (n = 130). Results were compared with EQ-5D-5L, scored using the UK 3L crosswalk and the 5L England value-sets. Results: The FACT-8D showed good convergent validity and responsiveness; baseline Pearson correlation coefficients between FACT-8D Index scores and other PRO measures were moderate to very strong (range: 0.49 for the EQ-VAS to 0.79 for FACT TOI) and the size of the change in FACT-8D Index scores at week 31 differed significantly (p < 0.005) between patients categorised as improved, worsened or stable using the FACT-Lym total score, LymS, and HgB. However, when assessing known groups' validity, FACT-8D failed to discriminate between patients categorised by health status on four of the seven variables analysed. Overall, FACT-8D and EQ-5D-5L performed similarly, although EQ-5D-5L showed better known groups' validity. Conclusions: This is the first investigation into the psychometric properties of the FACT-8D. In this RR MCL trial dataset, it showed good convergent validity and responsiveness, but poorer known groups' validity, and EQ-5D performed as well or better on the tests conducted. The FACT-8D may offer an alternative method to generate utilities for the cost-effectiveness analysis of cancer treatments but needs further testing in other types of cancer patients. Evaluation of utility gains may have been limited by high baseline performance status in this RR MCL trial sample. [ABSTRACT FROM AUTHOR]

Background: The Western Ontario Osteoarthritis of the Shoulder index (WOOS) has been introduced as a disease-specific quality of life measurement in patients with glenohumeral arthritis. The aim of the present study was to perform a cross-cultural adaptation of the English version of the WOOS to Italian and to assess its validity, reliability and responsiveness in patients with glenohumeral joint osteoarthritis treated conservatively.Material and Methods: The adaptation process was carried out following the simplified Guillemin criteria. The English version was translated into Italian by two bilingual orthopaedic surgeons and then translated back into English by two different bilingual orthopaedic surgeons. The original version was compared with the back-translation. The questionnaire was prospectively administered to 30 patients with glenohumeral osteoarthritis at baseline and again after 5 days for retest reliability. After 6 months of conservative treatment, the responsiveness of the questionnaire was assessed in a subsample of 20 patients. The level of statistical significance was set at 0.05.Results: The interclass correlation coefficient between test and retest of the WOOS was 0.99 (P < 0.001). Pearson's correlation coefficient between the WOOS and disability of the arm, shoulder and hand (DASH) preoperatively was 0.73 (P < 0.01) and the correlation between the changes of score for the WOOS and DASH was 0.75 (P < 0.01). There were no floor or ceiling effects. Responsiveness, calculated by standardized response mean, was 1.1 and effect size was 1.3.Conclusions: The Italian version of the WOOS questionnaire has shown to be equivalent to its English version and demonstrated good validity, reliability and responsiveness to conservative treatment of glenohumeral osteoarthritis.Level Of Evidence: Level II. [ABSTRACT FROM AUTHOR]

Background: Kashin-Beck Disease (KBD) is an endemic osteoarthropathy in areas which extend from the North-East to the South-West of China. Most of the patients with KBD suffer multiple dysfunctions in major joints causing decreased health status. However because of their low education level and unique living habits, it is hard to find tools to measure the health-related quality of life (HRQOL). European quality of life (EQ-5D-3L) patient-reported instrument is widely used to measure HRQOL. This study aimed to establish the validity and reliability of the Chinese version of the EQ-5D-3L for evaluating HRQOL of KBD individuals in rural area. Methods: 368 individuals who were suffering from KBD were recruited through stratified multistage random sampling from Shaanxi province, China. The EQ-5D-3L and the WHOQOL-BREF were administrated in each individual by face to face interview. Test-retest reliability was assessed at 10-14 days intervals. The test-retest reliability was measured by calculating the Kappa coefficients for EQ-5D-3L five dimensions. For the EQ VAS, the intraclass correlation coefficient (ICC) was computed. Convergent and divergent analysis, construct validity was established using Spearman's rank correlation between the EQ-5D-3L and the WHOQOL-BREF. Known groups' validity was examined by comparing groups with a priori expected differences in health-related quality of life (HRQOL). Results: For 362 individuals (98%), comprehensive data of all the EQ-5D-3L dimensions were available. Kappa values of the EQ-5D-3L five items ranged from 0.324 to 0.554. ICC of the EQ VAS was 0.497. For convergent validity, the three items (self-care, usual activity, and mobility) of EQ-5D-3L, index scores, and VAS showed moderate correlations with the physical health domain of the WHOQOL-BREF (r absolute value ranged from 0.339 to 0.475). For divergent validity, the 5 items of EQ-5D-3L showed weak or no correlations with environment and social relationship domains of WHOQOL-BREF. The Chinese EQ-5D-3L clearly demarcated between groups which were reporting severe disease degree, poorer general health, more number of painful joints with worse HRQOL. Conclusions: The EQ-5D-3L Chinese Version demonstrated fair to moderate levels of test-retest reliability and adequate construct validity in KBD individuals in China. [ABSTRACT FROM AUTHOR]

Purpose: Heart diseases are often associated with residual injuries, persisting functional restrictions, and long-term sequelae for psychosocial development. Currently, there are no disease-specific instruments to assess the health-related quality of life (HrQoL) of pre-school children. The aims of this study were to develop a parent proxy instrument to measure the HrQoL of children aged 3–7 years with a heart disease and to confirm its validity and reliability. Methods: Items from the Preschool Pediatric Cardiac Quality of Life Inventory (P-PCQLI) were generated through focus groups of caregivers. In a pilot study, comprehensibility and feasibility were tested. Five subdimensions were defined theoretically. Psychometric properties were analysed within a multicentre study with 167 parental caregivers. Results: The final 52-item instrument contains a total score covering five moderately inter-correlated dimensions. The total score of the questionnaire showed a very high internal consistency ( Cronbachs’ α = 0.95). Test–retest correlation was at rtt = 0.96. External validity was indicated by higher correlations ( r = 0.24–0.68) with a generic paediatric quality of life questionnaire (KINDL) compared to the Strengths and Difficulties Questionnaire ( r = 0.17 to 0.59). Low P-PCQLI total scores were significantly associated with inpatient as opposed to outpatient treatment ( t = 6.04, p < .001), with at least moderate disease severity (( t = 5.05, p < .001) NYHA classification) and with poorer prognosis ( t = 5.53, p < .001) as estimated by the physician. Conclusions: The P-PCQLI is reliable and valid for pre-school children with a heart disease. It could be used as a screening instrument in routine care, and for evaluation of HrQoL outcomes in clinical trials and intervention research. [ABSTRACT FROM AUTHOR]

Objective: To evaluate the psychometric properties of the National Eye Institute Visual Function Questionnaire-25 (NEI VFQ-25) and Visual Function Questionnaire Utility Index (VFQ-UI) in patients with non-infectious intermediate and posterior uveitis. Methods: Secondary analysis of pooled data from a 26-week, multicenter, masked, randomized, sham-controlled Phase 3 clinical trial. Health-related quality of life was assessed using the NEI VFQ-25, the EQ-5D, and SF-36. Internal consistency reliability, reproducibility, convergent validity, and known groups of BCVA and vitreous haze severity were assessed. Clinically significant difference was assessed using anchor-based and distribution-based methods. Results: The study included 224 subjects with non-infectious intermediate (80.4 %) or posterior uveitis (19.6 %). The NEI VFQ-25 and the VFQ-UI demonstrated good internal consistency (Cronbach’s alpha 0.87–0.94) and test–retest reliability (ICCs 0.58–0.88). Spearman’s product–moment rank correlations between the NEI VFQ-25 and VFQ-UI scores and the SF-6D, EQ-5D, and BCVA ranged from small to moderate. There was a significant association between visual functioning and known groups of visual acuity ( p < 0.05). Clinical significance, using the anchor-based method (difference between visual acuity groups ≥10–<15 letter better and no change), was 10.2 for change from baseline to week 26 for the NEI VFQ-25 composite score and 0.05 for the VFQ-UI. Using the distribution-based method, the clinical significance was 3.86 for the composite score and 0.04 for the VFQ-UI. Conclusion: The NEI VFQ-25 and the VFQ-UI are reliable and valid measures of vision-related functioning and preference-based status in patients with non-infectious intermediate and posterior uveitis. [ABSTRACT FROM AUTHOR]

Purpose: This review examines psychometric performance of three widely used generic preference-based measures, that is, EuroQol 5 dimensions (EQ-5D), Health Utility Index 3 (HUI3) and Short-form 6 dimensions (SF-6D) in patients with hearing impairments. Methods: A systematic search was undertaken to identify studies of patients with hearing impairments where health state utility values were measured and reported. Data were extracted and analysed to assess the reliability, validity (known group differences and convergent validity) and responsiveness of the measures across hearing impairments. Results: Fourteen studies (18 papers) were included in the review. HUI3 was the most commonly used utility measures in hearing impairment. In all six studies, the HUI3 detected difference between groups defined by the severity of impairment, and four out of five studies detected statistically significant changes as a result of intervention. The only study available suggested that EQ-5D only had weak ability to discriminate difference between severity groups, and in four out of five studies, EQ-5D failed to detected changes. Only one study involved the SF-6D; thus, the information is too limited to conclude on its performance. Also evidence for the reliability of these measures was not found. Conclusion: Overall, the validity and responsiveness of the HUI3 in hearing impairment was good. The responsiveness of EQ-5D was relatively poor and weak validity was suggested by limited evidence. The evidence on SF-6D was too limited to make any judgment. More head-to-head comparisons of these and other preference measures of health are required. [ABSTRACT FROM AUTHOR]

National accounts of subjective well-being are being considered and adopted by nations. In order to be useful for policy deliberations, the measures of life satisfaction must be psychometrically sound. The reliability, validity, and sensitivity to change of life satisfaction measures are reviewed. The scales are stable under unchanging conditions, but are sensitive to changes in circumstances in people's lives. Several types of data indicate that the scales validly reflect the quality of respondents' lives: (1) Differences between nations in life satisfaction associated with differences in objective conditions, (2) Differences between groups who live in different circumstances, (3) Correlations with nonself-report measures of life satisfaction, (4) Genetic and physiological associations with life satisfaction, (5) Systematic patterns of change in the scales before, during, and after significant life events, and (6) Prediction by life satisfaction scores of future behaviors such as suicide. The life satisfaction scales can be influenced by factors such as question order, current mood, and mode of presentation, but in most cases these can be controlled. Our model of life satisfaction judgments points to the importance of attention, values, standards, and top-down effects. Although the scales are useful in research on individual well-being, there are policy questions that need more analysis and research, such as which types of subjective well-being measures are most relevant to which types of policies, how standards influence scores, and how best to associate the scores with current policy deliberations. [ABSTRACT FROM AUTHOR]

Purpose: To assess the construct validity of the CHU9D in an adolescent general population sample. The CHU9D is a new generic preference-based measure of health-related quality of life developed specifically for application in the economic evaluation of health care treatments and interventions for young people. Methods: A web-based survey was developed including the CHU9D and HUI2 instruments and administered to a community-based sample of consenting adolescents ( n = 710) aged 11-17 years. The practicality, face and construct validity of the CHU9D was examined. The relationship between the CHU9D and HUI2 instruments was assessed by a comparison of responses to similar dimensions and the utility scores derived from the two instruments. Results: The CHU9D demonstrated high completion rates. CHU9D was able to discriminate between respondents according to their self-reported general health (Kruskal-Wallis P value <0.001). The mean CHU9D adolescent population utilities were similar to those generated from the HUI2 [Mean (SD) CHU9D utility 0.844 (0.102) and HUI2 utility 0.872 (0.138)], and the intra-class correlation coefficient indicated good levels of agreement overall (ICC = 0.742). Conclusion: The findings from this study provide support for the practicality, face and construct validity of the CHU9D for application with adolescents aged 11-17 years. [ABSTRACT FROM AUTHOR]

Purpose: The objective of this qualitative study was to develop the items and support the content validity of the PedsQL™ Sickle Cell Disease Module for pediatric patients with sickle cell disease (SCD). Methods: The iterative process included multiphase qualitative methodology. A literature review on SCD was conducted to generate domains of interest for the individual in-depth interviews. Ten healthcare experts with clinical experience in SCD participated in the development of the conceptual framework. A total of 13 pediatric patients with SCD ages 5-18 and 18 parents of patients ages 2-18 participated in the individual in-depth interviews. A total of 33 pediatric patients with SCD ages 5-18 and 39 parents of patients ages 2-18 participated in individually conducted cognitive interviews that included both think aloud and cognitive debriefing techniques to assess the interpretability and readability of the item stems. Results: Six domains were derived from the qualitative methods involving patient/parent interviews and expert opinion, with content saturation achieved, resulting in 48 items. The six domains consisted of items measuring Pain Intensity/Location (9 items), Pain Interference (11 items), Worry (7 items), Emotions (3 items), Disease Symptoms/Treatment, (12 items), and Communication (6 items). Conclusions: Qualitative methods involving pediatric patients and parents in the item development process support the content validity for the PedsQL™ SCD Module. The PedsQL™ SCD Module is now undergoing national multisite field testing for the psychometric validation phase of instrument development. [ABSTRACT FROM AUTHOR]

Due to their cognitive impairment, the health-related quality of life (HRQoL) of patients with dementia is often rated by proxies. This study aims to analyse the psychometric properties of the EQ-5D applied to patients with mild to moderate dementia and their family caregivers. Three hundred and ninety patients and their caregivers were asked to assess the patients’ HRQoL using the EQ-5D. The German population-based time trade-off values were used to calculate utility weights. Acceptance, discriminative ability, construct validity, inter-rater agreement and responsiveness were tested. Factors that could have an impact on inter-rater agreement were analysed using a multivariate regression. Five per cent of patients did not fill out the EQ-5D. The response rate of caregivers and of patients with mild dementia was higher than that of patients with moderate dementia. There were no floor or ceiling effects. The test results of the caregivers concerning construct validity and responsiveness were better than those of the patients. The inter-rater reliability was not satisfactory either on the dimension level or on the utility score level. Caregivers gave the patients’ HRQoL significantly lower ratings than did the patients themselves. Better abilities of the patient to perform activities of daily living and a lower subjective burden of the caregiver were associated with a higher inter-rater agreement. The study showed that the EQ-5D is especially applicable to patients with mild dementia and their caregivers as proxies. However, there are important differences between patient and proxy ratings, even in cases of mild dementia, at the dimension level as well as utility score level, which should be considered in the interpretation of quality of life scores. [ABSTRACT FROM AUTHOR]

To establish a reliable and valid scale structure of a patient-reported outcome measuring thyroid-specific quality of life. The 98-item ThyPRO questionnaire was administered to patients with benign thyroid diseases at two university hospitals. Multi-trait scaling was performed, evaluating lack of convergent validity (item-own scale polyserial correlation <0.40) or lack of discriminant validity (item-other scale correlation higher than item-own scale correlation) of the hypothesized scale structure. Analyses were repeated in clinical and sociodemographic subgroups and with Pearson correlations. Reliability was estimated by Cronbach’s α, both conventionally and with polychoric correlations. In total, 904 patients (69%) responded. Initial multitrait scaling analysis identified 25 scaling errors. Twelve items were omitted from the scale structure, and a re-analysis showed complete convergent validity and only two instances of lack of discriminant validity. Pearson correlations yielded similar results. Across all subgroups, convergent validity was complete, and discriminant validity was found in 99.2% of tests. Lack of discriminant validity was mainly between physical symptoms and psychological and disease-impact scales. Cronbach’s α was acceptable (>0.70, >0.80 with polychoric correlations) for all 13 scales. A reliable scale structure displaying complete convergent and almost complete discriminant validity was established in general analyses and in distinct clinical subgroups of patients with benign thyroid diseases. [ABSTRACT FROM AUTHOR]

Content validity, the extent to which a measurement reflects the specific intended domain of content, is a basic type of validity for a valid measurement. It was usually examined qualitatively and relied on experts’ subjective judgments, not on respondents’ responses. Therefore, the purpose of this study was to introduce and demonstrate how to use quantitative methods to examine the content validity for a certain measurement from respondents’ viewpoint with quantitative methods. In the current study, content validity of the WHOQOL-BREF was examined with quantitative methods among 102 undergraduate students and 128 community adults. They were asked to rate the appropriateness of each item with the four domains of the WHOQOL-BREF respectively and also asked to sort the items into the four domains. Then, three quantitative methods on examining content validity were applied to analyze the rating and sorting data, including (1) proportion of substantive agreement, (2) substantive validity, and (3) ANOVA approach. These results were used to compare with the original content structure of the WHOQOL-BREF, to see if the original structure is consistent with the structure of subjects’ judgments. The results showed that the content structure gained from appropriateness rating and item sorting was not totally consistent with the original content structure of the WHOQOL-BREF. Among 24 items, 12 items did not have adequate content validity. More discussion on these items and the issue of content validity were further provided. [ABSTRACT FROM AUTHOR]

Objectives: The Hospital Anxiety and Depression Scale (HADS) is a widely used screening instrument. The purpose of this study was to evaluate reliability and validity of the Hungarian translation.Methods: The English version of the HADS was translated using the 'forward-backward' procedure. The questionnaire was used in a large scale study of 715 Hungarian cancer patients along with other screening measures of psychological state and description of illness.Results: Translated items of the HADS questionnaire showed high internal consistency: Cronbach's alpha values for the subscales were 0.81 (anxiety) and 0.83 (depression). Factor analysis of the Hungarian version yielded an identical two-factor model to the English and German versions. Results of the known groups comparison showed that both subscales of the HADS discriminates well between sub-groups: decreasing performance status and more advanced disease stage showed significantly higher levels of anxiety and depression. Sufficient concurrent validity of the HADS depression subscale was found using five items from the Symptom List and the Hungarian version of the Beck Depression Scale.Conclusions: Based on a detailed analysis of results we found the translated version of the HADS a reliable and valid self-assessment screening tool in medical practice. [ABSTRACT FROM AUTHOR]

Objective: This study examined the construct validity, and improved the test reliability and the estimation accuracy for the correlation between domains of the WHOQOL-BREF using multidimensional Rasch analysis. Method: A total of 13,083 adults were administered the 28-item WHOQOL-BREF Taiwan version, which consists of 4 subscales (domains). The multidimensional form of the partial credit model was used to examine the fit of the 4 subscales. For comparison, each subscale individually was also fitted to the unidimensional partial credit model. Standard item fit statistics and analysis of differential item functioning (DIF) were used to check model-data fit. Results: After excluding 2 overall items and deleting 7 DIF items, the remaining items of each subscale in the WHOQOL-BREF constituted a single construct. The test reliabilities and correlations between domains obtained from the multidimensional approach, (0.82–0.86) and (0.79–0.89), respectively, were much higher than those obtained from the unidimensional approach, (0.67–0.75) and (0.53–0.65), respectively. Conclusion: The 19-item WHOQOL-BREF measures more succinct latent traits than the original design. The multidimensional approach yields not only more accurate estimates for the correlation between domains but also substantially higher reliabilities, than the standard unidimensional approach. [ABSTRACT FROM AUTHOR]

The Low Vision Quality of Life (LVQOL) questionnaire and the Vision-related Quality of Life Core Measure (VCM1) are two of the many vision-related quality of life (QOL) questionnaires that have been developed in recent years. Although psychometric properties of the LVQOL and VCM1 compare well with other vision-related QOL questionnaires, construct and longitudinal validity have not been assessed (adequately). The purpose of this study was to examine the cross-sectional and longitudinal construct validity of these questionnaires by testing specific pre-specified hypotheses about the relations of these questionnaires with other measures. The percentage of hypotheses regarding the cross-sectional construct validity that were refuted for the LVQOL was 22% for the basic aspects of vision subscale, 50% for the mobility subscale, 39% for the adjustment subscale and 17% for the reading and fine work subscale. For the VCM1 this percentage was 57%. For the longitudinal construct validity the percentage of hypotheses that were refuted ranged from 33 to 75% for the LVQOL subscales and was 50% for the VCM1. In conclusion, cross-sectional construct validity was satisfactory for the LVQOL subscales, but seemed poor for the VCM1. In addition, the longitudinal validity of these scales was poor to moderate. [ABSTRACT FROM AUTHOR]

This paper describes the procedure used to translate the COOP Charts into French and provides preliminary information on the instrument's acceptability, reliability and validity. The charts were translated in several steps: seven initial translations were combined into a first pilot version, which was then tested for acceptability, clarity and alternative wordings in two convenience samples taken from the general population (n = 53). The modified version was then reviewed by a lay panel and another translator and submitted by mail to 209 congress participants to test several construct validity hypotheses through known-groups comparisons. A panel of public health professionals discussed the content validity of the charts. Finally, test-retest reliability and concurrent validity with SF-36 Health Survey scores were examined among 65 patients with end-stage renal disease. The translation process identified a wide variability in translation options for several items. The acceptability of the charts was excellent. The test-retest correlations ranged from 0.60 to 0.87. Content validity appeared to be appropriate, except for the chart on 'social support', which combines the questions of need and availability of social support. The utility of illustrations was questioned by some respondents: many claimed not to have used the illustrations in selecting their response, while others found them to be not expressive enough. Most preliminary tests of construct validity were consistent with theory. This French translation of the COOP Charts appears to be ready for more extensive testing in the intended target population of ambulatory patients. [ABSTRACT FROM AUTHOR]

Background: Robust outcome measures are needed to assess and monitor the impact of chronic low back pain (CLBP) on physical functioning. The Roland Morris Disability Questionnaire (RMDQ) is a well-established measure designed to capture the impacts of back pain on everyday functioning, with a particular emphasis on physical functioning. It has documented evaluation of psychometric properties. However, there is no documented qualitative evidence to confirm the content validity of the tool, nor have changes made for electronic administration been debriefed in participants with CLBP. Methods: In-depth, semi-structured, concept elicitation and cognitive debriefing interviews were conducted with 23 US participants with confirmed CLBP. Interviews allowed participants to describe the impact of CLBP on their day-to-day functioning and discuss comprehension and suitability of the RMDQ. Interviews were transcribed verbatim and analyzed using thematic analysis. Results: Concept elicitation and cognitive debriefing revealed the substantial burden associated with CLBP, highlighting 15 key areas of functional impact. These were grouped into overarching themes of mobility (walking, stairs, sitting/standing, bending/kneeling, lifting, lying down), activities (chores/housework, dressing, washing, driving, work) and other (relationships/socializing, mood, sleep, appetite), which are consistent with those evaluated within the RMDQ. All participants found the RMDQ to be relevant with most reporting that the instructions, recall period, and response options were suitable. A few suggested minor changes, however, none were consistent or necessary to support content validity. Updates to the measure for electronic administration and to clarify the response options were well received. Conclusion: The qualitative data from individuals with CLBP confirmed that the RMDQ has content validity and, alongside documented psychometric evidence, supports the use of the RMDQ as a reliable and valid tool to assess the impact of CLBP on physical functioning. [ABSTRACT FROM AUTHOR]