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Background: The CLN2 Clinical Rating Scale evaluates disease progression in CLN2 disease, an ultra-rare, neurodegenerative disorder with late infantile onset. To validate the Clinical Rating Scale, a comparison with the Pediatric Quality of Life Inventory (PedsQL) was conducted utilising clinical trial data investigating cerliponase alfa use in CLN2 disease. Methods: Linear regression and mixed effects models were used to investigate the relationship between the Clinical Rating Scale and PedsQL using open-label, single-arm, phase 1/2 (NCT01907087) and ongoing extension study (NCT02485899) data of 23 children with CLN2 disease treated with cerliponase alfa for ≥96 weeks. Results: Correlations between the four Clinical Rating Scale domains were low. Linear mixed effects analyses showed significant correlation between PedsQL and Clinical Rating Scale (Total score or motor-language [ML] score adjusted p-values <0.05), driven by the relationship with the PedsQL Physical domain. A statistically significant relationship was identified between the Clinical Rating Scale motor domain and PedsQL (Total score: adjusted p-value = 0.048, parameter estimate [PE] = 8.10; Physical domain score: adjusted p-value = 0.012; PE = 13.79). Conclusions: Each domain of the Clinical Rating Scale provides unique information on disease state. Validity of the scale is supported by its relationship with the PedsQL. Among the four domains of the Clinical Rating Scale, motor has the highest correlation to PedsQL, suggesting motor function as a driver of patients' quality of life. The lack of association between the remaining domains of the Clinical Rating Scale and PedsQL suggests that additional disease-specific measures may be needed to fully capture the quality of life impact of CLN2 disease. Trial registration: NCT01907087, NCT02485899. [ABSTRACT FROM AUTHOR]

Objectives: To assess the reliability and validity of two disease-specific questionnaires that assess the quality of life (QoL) among patients with Systemic Lupus Erythematosus (SLE); SLEQoL and LupusQoL in Malay language. This study also identified the factors affecting each domain of the questionnaires. Methods: This cross-sectional study was conducted from June 2021 until April 2022, and SLE patients were recruited to complete the SLEQoL, LupusQoL and Short Form Health Survey (SF-36) in Malay language. Disease activity were recorded using the modified SLE Disease Activity Index (M- SLEDAI) and British Isles Lupus Assessment Group 2004 (BILAG-2004) index. Presence of organ damage was determined using the SLICC Damage index. Cronbach's alpha was calculated to determine internal consistency while exploratory factor analysis was done to determine the construct validity. Concurrent validity was evaluated using correlation with SF-36. Multiple linear regression analysis was deployed to determine the factors affecting each domain of SLEQoL and LupusQoL. Results: A total of 125 subjects were recruited. The Cronbach's α value for the Malay-SLEQoL (M-SLEQoL) and Malay-LupusQOL (M-LupusQoL) was 0.890 and 0.944 respectively. Exploratory factor analysis found formation of similar number of components with the original version of questionnaires and all items have good factor loading of >0.4. Both instruments also had good concurrent validity with SF-36. M-SLEQoL had good correlations with BILAG-2004 and M-SLEDAI scores. Musculoskeletal (MSK) involvement was independently associated with lower M-SLEQoL in physical function, activity and symptom domains. Meanwhile, MSK and NPSLE were associated with fatigue in M-LupusQoL. Conclusion: Both M-SLEQoL and M-LupusQoL are reliable and valid as disease -specific QoL instruments for Malaysian patients. The M-Lupus QoL has better discriminative validity compared to the M-SLEQoL. SLE patients with MSK involvement are at risk of poor QoL in multiple domains including physical function, activity, symptoms and fatigue. [ABSTRACT FROM AUTHOR]

Dry eye disease (DED) is a common and growing eye problem worldwide. Chronic DED symptoms can, subsequently, affect the patients' quality of life (QOL). This prospective cross-sectional study aimed to assess the reliability, validity, and responsiveness of the Thai version of the Dry Eye-Related Quality-of-Life Score (DEQS-Th) questionnaire and to evaluate its accuracy in DED screening. Psychometric validation was conducted on DED participants. All participants completed the DEQS-Th and other measurements including the Ocular Surface Disease Index (OSDI) and the 5-level EQ-5D (EQ-5D-5L). Internal consistency, concurrent validity, convergent, and discriminant validity were evaluated. The standardized response mean (SRM) was used to evaluate the responsiveness of the DEQS-Th. The optimal cut-off score of DEQS-Th for DED screening was assessed. Among 100 participants with a mean age of 50.9 ± 14.4 years, and 89.0% female, the internal consistency of the DEQS-Th was excellent (Cronbach's alpha: 0.80–0.92). The test-retest intraclass correlation was 0.82–0.92. It showed concurrent validity with the OSDI (r = 0.694, p <.001) and EQ-5D-5L index scores (r = -0.578, p <.001). DED is suspected if the DEQS-Th score ≥ 18.33 (AUC = 0.897, sensitivity 90.0%, specificity 76.7%) or its Short Form score ≥ 3 (AUC 0.857, sensitivity 93.0%, specificity 63.3%). The SRM of the symptom subscale of DEQS-Th was 0.82, indicating relatively large responsiveness, whereas the impact on daily life subscale and the summary score was small. In conclusion, the DEQS-Th is valid and reliable for evaluating the multifaceted effects of DED on a patient's QOL. It can be useful for primary assessment and monitoring of DED in routine clinical practice. [ABSTRACT FROM AUTHOR]

Objective: Transcultural adaptation and validation of the FACT-C questionnaire to Serbian language. Methods: The study included 131 patients with colorectal cancer. Translation included standard forward and backward translation from original language to Serbian and back. Pilot testing of the questionnaire was conducted on 10 patients with diagnosed colorectal cancer. The questionnaires EORTC-QLQ-C30 and DASS will be used as validated tools to evaluate validity of examined, FACT-C questionnaire. Results: The FACT-C demonstrated satisfactory construct validity using Cronbach's alpha. Satisfactory concurrent validity was demonstrated using correlations with EORTC-QLQ-C30 and DASS questionnaires. High reproducibility was demonstrated using repeated questionnaires on 30 patients two weeks after the first interview. Conclusion: The Serbian version of the FACT-C was demonstrated to have satisfactory applicability, reliability and validity in Serbian patients with colorectal cancer. It can be considered as a valid colorectal cancer specific health related quality of life tool for the Serbian population. [ABSTRACT FROM AUTHOR]

Objective: Lifestyle is considered as a key factor that affects one's health and quality of life, and it has become the focus of increasing research interest worldwide. Research has shown that lifestyle is an important health determinant in preventive health care. However, lifestyle is a multifaceted construct and there is limited evidence regarding lifestyle assessment, which evaluates individuals' multi-domain lifestyle factors. This study developed evaluation items for measuring the multifaceted lifestyle profile of community-dwelling older adult to prevent chronic disease and improve their health and quality of life. Methods: Opinions from 21 experts with experience in older adults and lifestyle research were collected from December 2019 to January 2020. Three Delphi surveys were carried out, based on previous research. The first survey gathered opinions using a mix of open- and closed-ended questions regarding items of the older adults' multifaceted lifestyle profile. The second was conducted after adding and modifying several items based on the first Delphi survey. In the third survey, after the results of the second one were presented to the expert panels, final opinions from the experts were converged. Results: In total, 59 items were selected as the first Delphi results; 62 items were selected as the second results after adding and modifying the values below a content validity ratio of.42; and 62 items were selected as the third Delphi results. The average content validity ratio of the final Delphi survey was.92, the stability was.18, and the consensus was.80, which were all high. Conclusions: This study verified the content validity of the evaluation items for community-dwelling older adults' multifaceted lifestyle profile. In the future, it is expected that after verifying the validity and reliability, this will be used as a standardized assessment tool in clinical environments. [ABSTRACT FROM AUTHOR]

Background: The Developmental Coordination Disorder Questionnaire’07 (DCDQ’07) is a parent-report measure to identify children at risk for Developmental Coordination Disorder (DCD). We developed a French version of the DCDQ’07 (DCDQ-FE) that has shown excellent inter-language reliability (intraclass correlation coefficient (ICC) = 0.91) and is culturally relevant for use in European countries. The aims of this study were to examine the internal consistency, test-retest reliability, construct validity of the Developmental Coordination Disorder Questionnaire-French European (DCDQ-FE), as well as establish a cut-off score. Methods: The psychometric properties of the DCDQ-FE were examined with a clinical group of 30 children (mean age: 9.4 years, SD = 2.6) and a control group of 43 children (mean age: 9.1 years, SD = 2.4). Their parents (n = 73) filled out the DCDQ-FE at a first sitting and 70 of them filled it out 38 days later in average for test-retest reliability. The children were assessed using the Movement Assessment Battery for Children-2 (MABC-2) so as to measure the convergent validity of the DCDQ-FE. The cut-off score was determined with an additional sample of 42 children according to scores on the MABC-2 (≥ 16th percentile) (n = 115). Results and implications: Internal consistency of the DCDQ-FE was excellent (Cronbach’s alpha = 0.96) and test-retest reliability was good (ICC = 0.956) with no differences between scores obtained at the two sittings (p > 0.05). Differences in scores between children in the clinical and control groups (Z = -6.58, p < 0.001) provide evidence of construct validity. The correlation obtained between DCDQ-FE and MABC-2 scores (Spearman’s rho correlation coefficient = 0.802, p < 0.001) supports convergent validity. Using a cut-off of 56, overall sensitivity and specificity were 85.0% and 81.6% respectively (area under the curve = 0.896). The DCDQ-FE is a reliable and valid questionnaire for detecting children who are at risk for DCD in a European-French population of children aged 5 to 15 years old. [ABSTRACT FROM AUTHOR]

Quality of life is an important outcome indicator to evaluate whether treatment is successful or not. Chronic gastritis leads to ongoing deterioration of subjectively perceived quality of life. There are several generic measures, but they are not developed particularly to assess chronic gastritis problems. The Quality of Life Instruments for Chronic Diseases—Chronic Gastritis (QLICD-CG V2.0) questionnaire is a 39-item, multi-dimensional, self-report instrument to assess chronic gastritis patients’ perception of their health related quality of life in four domains. The instrument was developed in China. The current study aimed to evaluate the psychometric properties of the QLICD-CG V2.0. 194 patients with chronic gastritis were enrolled from 4 hospitals in China. The QLICD-CG V2.0 was administered to patients by trained research assistants. In addition, their demographic characteristics were also recorded. The psychometric testing included construct validity, convergent validity, discriminant validity, test-retest, and responsiveness. The results showed good internal consistency and acceptable floor and ceiling effects (Cronbach’s alpha range from 0.80 to 0.93). CFA showed that the instrument structure has a reasonable fitness (RMSEA = 0.063, 95%CI = [0.057 0.079], CFI = 0.93, GFI = 0.95, SRMR = 0.028). The convergent validity was considered appropriate, with 38 of the 39 items correlated stronger with their assigned scale than a competing scale, except for GPS1. Known groups comparisons showed that the QLICD-CG V2.0 discriminated well between subgroups on the basis of gender, marriage status, and economy status, thus providing evidence of discriminative validity. Convergent validity testing revealed that the QLICD-CG V2.0 domain scores correlated significantly with SF-36 dimension scores, which ranged from 0.21 to 0.58. Test-retest coefficients were satisfactory. A majority of intraclass correlation coefficients were above 0.70, except the psychological domain (0.60) and the items of social support/security (0.61). Responsiveness was tested on 157 patients. Significant differences were found on all QLICD-CG V2.0 domains, between baseline responses and after a treatment, except for the items of appetite and sleep. Robust sensitivity to change was observed. The QLICD-CG V2.0 appears to be a valid and reliable instrument to measure QOL in chronic gastritis patients. Scores were reproducible. [ABSTRACT FROM AUTHOR]

Background: Despite bearing the largest HIV-related burden, little is known of the Health-Related Quality of Life (HRQoL) among people living with HIV in sub-Saharan Africa. One of the factors contributing to this gap in knowledge is the lack of culturally adapted and validated measures of HRQoL that are relevant for this setting. Aims: We set out to adapt the Functional Assessment of HIV Infection (FAHI) Questionnaire, an HIV-specific measure of HRQoL, and evaluate its internal consistency and validity. Methods: The three phase mixed-methods study took place in a rural setting at the Kenyan Coast. Phase one involved a scoping review to describe the evidence base of the reliability and validity of FAHI as well as the geographical contexts in which it has been administered. Phase two involved in-depth interviews (n = 38) to explore the content validity, and initial piloting for face validation of the adapted FAHI. Phase three was quantitative (n = 103) and evaluated the internal consistency, convergent and construct validities of the adapted interviewer-administered questionnaire. Results: In the first phase of the study, we identified 16 studies that have used the FAHI. Most (82%) were conducted in North America. Only seven (44%) of the reviewed studies reported on the psychometric properties of the FAHI. In the second phase, most of the participants (37 out of 38) reported satisfaction with word clarity and content coverage whereas 34 (89%) reported satisfaction with relevance of the items, confirming the face validity of the adapted questionnaire during initial piloting. Our participants indicated that HIV impacted on their physical, functional, emotional, and social wellbeing. Their responses overlapped with items in four of the five subscales of the FAHI Questionnaire establishing its content validity. In the third phase, the internal consistency of the scale was found to be satisfactory with subscale Cronbach’s α ranging from 0.55 to 0.78. The construct and convergent validity of the tool were supported by acceptable factor loadings for most of the items on the respective sub-scales and confirmation of expected significant correlations of the FAHI subscale scores with scores of a measure of common mental disorders. Conclusion: The adapted interviewer-administered Swahili version of FAHI questionnaire showed initial strong evidence of good psychometric properties with satisfactory internal consistency and acceptable validity (content, face, and convergent validity). It gives impetus for further validation work, especially construct validity, in similar settings before it can be used for research and clinical purposes in the entire East African region. [ABSTRACT FROM AUTHOR]

Background: The Global Program to Eliminate Lymphatic Filariasis aims to interrupt transmission of lymphatic filariasis and manage morbidity in people currently living with the disease. A component of morbidity management is improving health-related quality of life (HRQoL) in patients. Measurement of HRQoL in current management programs is varied because of the lack of a standard HRQoL tool for use in the lymphatic filariasis population. Methodology/Principal Findings: In this study, the psychometric properties of three health status measures were compared when used in a group of lymphatic filariasis patients and healthy controls. The World Health Organization Disability Assessment Schedule 2.0 (WHODAS 2.0), the Dermatology Life Quality Index (DLQI), and the Lymphatic Filariasis Quality of Life Questionnaire (LFSQQ) were administered to 36 stage II and stage III lymphatic filariasis subjects and 36 age and sex matched controls in Kerala, India. All three tools yielded missing value rates lower than 10%, suggesting high feasibility. Highest internal consistency was seen in the LFSQQ (α = 0.97). Discriminant validity analysis demonstrated that HRQoL was significantly lower in the LF group than in controls for the WHODAS 2.0, DLQI, and LFSQQ, but total HRQoL scores did not differ between stage II and stage III lymphedema subjects. The LFSQQ total score correlated most strongly with the WHODAS 2.0 (r = 0.91, p<0.001) and DLQI (r = 0.81, p<0.001). Conclusions/Significance: The WHODAS 2.0, DLQI, and LFSQQ demonstrate acceptable feasibility, internal consistency, discriminate validity, and construct validity. Based on our psychometric analyses, the LFSQQ performs the best and is recommended for use in the lymphatic filariasis population. Author Summary: Lymphatic filariasis affects approximately 120 million people and is the second leading cause of life-long disability worldwide. Because lymphatic filariasis is one of the World Health Organization's six eradicable diseases, much effort has been placed into reducing transmission of the disease and managing morbidity. Novel interventions frequently use health-related quality of life as an outcome measure to monitor efficacy of the intervention. In an effort to delineate the strengths and weaknesses of health status measures and recommend use of a single tool in the lymphatic filariasis population, we compared the use of three health status tools (The World Health Organization Disability Assessment Schedule 2.0, the Dermatology Life Quality Index, and the Lymphatic Filariasis Quality of Life Questionnaire) in lymphatic filariasis subjects and healthy controls in Kerala, India. The Lymphatic Filariasis Quality of Life Questionnaire performed the best by discriminating well between subjects and controls, possessing significant content overlap with the other two tools, yielding a low missing value rate, and being internally consistent. This is the first study to compare health status measures in lymphatic filariasis subjects and provides insight into the use of the tools in quality of life analysis. [ABSTRACT FROM AUTHOR]