Long-term Evaluation Parameters in GNE Myopathy: A Five-year Observational Follow-up Natural History Study

BackgroundA number of clinical trials targeting GNE myopathy patients have been conducted. However, useful clinical parameters for post-marketing surveillance and long-term clinical observation have not yet been established. ObjectiveWe conducted a 5-year observational follow-up natural history study to identify evaluation parameters which may be useful for the long-term observation of GNE myopathy patients. MethodsThirty-three genetically-confirmed GNE myopathy patients were recruited and evaluated at study entry (baseline) and yearly in a 5-year follow-up. Hand-held dynamometer measurements of knee extension strength, grip power, and pinch power, summed Manual Muscle Testing (MMT) score of 17 muscles, Gross Motor Function Measure (GMFM), 6-minute walk test, percent vital capacity and percent force vital capacity (%FVC), lean body mass (whole body, arms, and legs), creatine kinase (CK), Barthel Index, modified Rankin Scale, and SF-36 national standard scores were examined. ResultsOf the 33 patients, 22 (66%) completed evaluations for the entire 5-year follow-up period. These patients had a significant reduction in summed MMT score (p=0.001), GMFM (p=0.001), grip power (p=0.013), pinch power (p<0.001), CK (p=0.030), %FVC (p<0.001), leg lean body mass (p=0.040), and the Physical Functioning subscale score of the SF-36 (p=0.015) at the 5th year evaluation relative to baseline. Among these parameters, summed MMT score, GMFM, pinch power, and %FVC showed significant changes even in non-ambulant patients.ConclusionsMMT, GMFM, pinch power, CK, %FVC, lean body mass, and Physical Functioning subscale score of the SF-36 are useful parameters for the long-term evaluation of GNE myopathy patients.

Cross-Cultural Adaptation of the General Functioning Subscale of the McMaster Family Assessment Device

Background: The aim of the study was to conduct a cross-cultural adaptation and validation of the General Functioning Subscale (GF12) of the McMaster Family Assessment Device (FAD) into the Malay language. Methods: The translation and adaptation procedure of the Malay GF12 was based on the dual-panel methodology. This involved conducting a bilingual panel (providing the initial translation into Malay language) followed by a lay panel (where items are assessed for comprehension and acceptability). A mixed-methods approach with exploratory sequential study design was employed.Results: Two hundred and fifty-one families who have children in pre-schools (Tadikas) answered the Malay GF12. The KMO value for sampling adequacy was 0.81, indicating the sample was adequate for factor analysis. The Malay GF12 demonstrated high internal consistency (Cronbach’s alpha coefficient > 0.80). In the confirmatory factor analysis, the original 12-item model did not fit very well. Alternatively, a 2-factor-6-item model showed a sufficient fit. The two constructs comprised Positive Items and Negative Items. Conclusions: The results indicate the feasibility of administering 6 of the 12 items of Malay GF12 (3 positively worded and 3 negatively worded) to measure family functioning in the Malaysian population.

International relevance of two measures of awareness of age-related change (AARC)

Background A questionnaire assessing awareness of positive and negative age-related changes (AARC gains and losses) was developed in the US and Germany. We validated the short form of the measure (AARC-10 SF) and the cognitive functioning subscale from the 50-item version of the AARC (AARC-50) questionnaire in the UK population aged 50 and over. Methods Data from 9410 participants (Mean (SD) age = 65.9 (7.1)) in the PROTECT cohort were used to explore and confirm the psychometric properties of the AARC measures including: validity of the factor structure; reliability; measurement invariance across men and women, individuals with and without a university degree, and in middle age, early old age, and advanced old age; and convergent validity with measures of self-perception of aging and mental, physical, and cognitive health. We explored the relationship between demographic variables (age, sex, marital status, employment, and university education) and AARC. Results We confirmed the two-factor structure (gains and losses) of the AARC-10 SF and the AARC-50 cognitive functioning subscale. Both scales showed good reliability and good convergent validity for AARC losses, but weak convergent validity for AARC gains. For both scales metric invariance was held for the two subgroups defined by education level and age. For the AARC-50 subscale, but not for the AARC-10 SF, strong invariance was also held for the two subgroups defined by sex. Age, sex, marital status, employment, and university education predicted AARC gains and losses. Conclusions The AARC-10 SF and AARC-50 cognitive functioning subscale identify UK individuals who perceive age-related changes in their mental, physical, and cognitive health.

International relevance of Two Measures of Awareness of Age-Related Change (AARC)

Background: A questionnaire assessing awareness of positive and negative age-related changes (AARC gains and losses) was developed in the US and Germany. We validated the short form of the measure (AARC-10 SF) and the cognitive functioning subscale from the 50-item version of the AARC (AARC-50) questionnaire in the UK population aged 50 and over. Methods: Data from 9,410 participants (Mean (SD) age= 65.9 (7.1)) in the PROTECT cohort were used to explore and confirm the psychometric properties of the AARC measures including: validity of the factor structure; reliability; measurement invariance across men and women, individuals with and without a university degree, and in middle age, early old age, and advanced old age; and convergent validity with measures of self-perception of aging and mental, physical, and cognitive health. We explored the relationship between demographic variables (age, sex, marital status, employment, and university education) and AARC. Results: We confirmed the two-factor structure (gains and losses) of the AARC-10 SF and the AARC-50 cognitive functioning subscale. Both scales showed good reliability and good convergent validity for AARC losses, but weak convergent validity for AARC gains. For both scales metric invariance was held for the two subgroups defined by education level and age. For the AARC-50 subscale, but not for the AARC-10 SF, strong invariance was also held for the two subgroups defined by sex. Age, sex, marital status, employment, and university education predicted AARC gains and losses. Conclusions: The AARC-10 SF and AARC-50 cognitive functioning subscale identify UK individuals who perceive age-related changes in their mental, physical, and cognitive health.

International relevance of Two Measures of Awareness of Age-Related Change (AARC)

Background A questionnaire assessing awareness of positive and negative age-related changes (AARC gains and losses) was developed in the US and Germany. We validated the short form of the measure (AARC-10 SF) and the cognitive functioning subscale from the 50-item version of the AARC (AARC-50) questionnaire in the UK population aged 50 and over. Methods Data from 14,797 participants in the PROTECT cohort were used to explore and confirm the psychometric properties of the AARC measures including: validity of the factor structure; reliability; measurement invariance across males and females and across individuals with and without a university degree; and convergent validity with measures of self-perception of aging and mental, physical, and cognitive health. We also explored the relationship between demographic variables and AARC. Results We confirmed the two-factor structure (gains and losses) of the AARC-10 SF and the AARC-50 cognitive functioning subscale. Both scales showed good reliability and convergent validity. The meaning of AARC gains and losses was the same across males and females and across individuals with and without a university degree. Items composing AARC scales had the same meaning across individuals with and without a university degree. Items composing the AARC-50 cognitive functioning subscale had the same meaning across males and females. Two items in the AARC-10 SF had different meaning across males and females. Demographic variables significantly predicted AARC gains and losses. Conclusions The AARC-10 SF and AARC-50 cognitive functioning subscale can help to identify UK individuals who perceive age-related changes in their mental, physical, and cognitive health.

International relevance of Two Measures of Awareness of Age-Related Change (AARC)

Background: A questionnaire assessing awareness of positive and negative age-related changes (AARC gains and losses) was developed in the US and Germany. We validated the short form of the measure (AARC-10 SF) and the cognitive functioning subscale from the 50-item version of the AARC (AARC-50) questionnaire in the UK population aged 50 and over. Methods: Data from 9,410 participants (Mean (SD) age= 65.9 (7.1)) in the PROTECT cohort were used to explore and confirm the psychometric properties of the AARC measures including: validity of the factor structure; reliability; measurement invariance across males and females and across individuals with and without a university degree; and convergent validity with measures of self-perception of aging and mental, physical, and cognitive health. We also explored the relationship between demographic variables and AARC. Results: We confirmed the two-factor structure (gains and losses) of the AARC-10 SF and the AARC-50 cognitive functioning subscale. Both scales showed good reliability. Convergent validity was good for AARC losses, but weaker for AARC gains. The meaning of AARC gains and losses was the same across males and females and across individuals with and without a university degree. Items in the AARC scales had the same meaning across individuals with and without a university degree. Items in the AARC-50 cognitive functioning subscale had the same meaning across males and females. Single items in the AARC-10 SF had different meaning across males and females. Demographic variables predicted AARC gains and losses. Conclusions: The AARC-10 SF and AARC-50 cognitive functioning subscale can help to identify UK individuals who perceive age-related changes in their mental, physical, and cognitive health.

Assessing functioning in adolescents with chronic fatigue syndrome: psychometric properties and factor structure of the School and Social Adjustment Scale and the Physical Functioning Subscale of the SF36

Background:Chronic fatigue syndrome (CFS) has a major impact on functioning. However, no validated measures of functioning for this population exist.Aims:We aimed to establish the psychometric properties of the 5-item School and Social Adjustment Scale (SSAS) and the 10-item Physical Functioning Subscale of the SF-36 in adolescents with CFS.Method:Measures were completed by adolescents with CFS (n = 121).Results:For the Physical Functioning Subscale, a 2-factor solution provided a close fit to the data. Internal consistency was satisfactory. For the SSAS, a 1-factor solution provided an adequate fit to the data. The internal consistency was satisfactory. Inter-item and item-total correlations did not indicate any problematic items and functioning scores were moderately correlated with other measures of disability, providing evidence of construct validity.Conclusion:Both measures were found to be reliable and valid and provide brief measures for assessing these important outcomes. The Physical Functioning Subscale can be used as two subscales in adolescents with CFS.

International relevance of Two Measures of Awareness of Age-Related Change (AARC)

Background: A questionnaire assessing awareness of positive and negative age-related changes (AARC gains and losses) was developed in the US and Germany. We validated the short form of the measure (AARC-10 SF) and the cognitive functioning subscale from the 50-item version of the AARC (AARC-50) questionnaire in the UK population aged 50 and over.Methods: Data from 14,797 participants in the “blind for review” cohort were used to explore and confirm the psychometric properties of the AARC measures including: validity of the factor structure; reliability; measurement invariance across males and females and across individuals with and without a university degree; and convergent validity with measures of self-perception of aging and mental, physical, and cognitive health. We also explored the relationship between demographic variables and AARC.Results: We confirmed the two-factor structure (gains and losses) of the AARC-10 SF and the AARC-50 cognitive functioning subscale. Both scales showed good reliability and convergent validity. The meaning of AARC gains and losses was the same across males and females and across individuals with and without a university degree. Items composing AARC scales had the same meaning across individuals with and without a university degree. Items composing the AARC-50 cognitive functioning subscale had the same meaning across males and females. Two items in the AARC-10 SF had different meaning across males and females. Demographic variables significantly predicted AARC gains and losses.Conclusions: The AARC-10 SF and AARC-50 cognitive functioning subscale can help to identify UK individuals who perceive age-related changes in their mental, physical, and cognitive health.