Initial development and validation of a mitochondrial disease quality of life scale.
Ontology highlight
ABSTRACT:
SUBMITTER: Elson JL
PROVIDER: S-EPMC3841574 | biostudies-literature | 2013 Apr
REPOSITORIES: biostudies-literature
Publications
Initial development and validation of a mitochondrial disease quality of life scale.
Elson J L JL Cadogan M M Apabhai S S Whittaker R G RG Phillips A A Trennell M I MI Horvath R R Taylor R W RW McFarland R R McColl E E Turnbull D M DM Gorman G S GS
Neuromuscular disorders : NMD 20130220 4
Mitochondrial diseases are a clinically diverse group of genetic disorders that often present to neurologists. Health related quality of life (HRQOL) is increasingly recognised as a fundamental patient based outcome measure in both clinical intervention and research. Generic outcome measures have been extensively validated to assess HRQOL across populations and different disease states. However, due to their inclusive construct, it is acknowledged that not all relevant aspects of a specific illn ...[more]
Similar Datasets
Project description:PurposeThe development and initial validation of patient-derived, health-related quality-of-life (HRQOL) questionnaires for intermittent exotropia (IXT).DesignCross-sectional study.ParticipantsIn a development phase, 27 children (age, 2-17 years) with IXT and 1 of their parents. In an initial validation phase, 33 children with IXT and 49 control children (age, 5-17 years), along with 1 parent for each child. Children in the control group had no strabismus or amblyopia.MethodsIndividual patient interviews generated 35 items for child and proxy (parental assessment of child's HRQOL) questionnaires and 46 items for a parent questionnaire. To reduce to a feasible number of items, questionnaires were administered to 5- to 17-year-old children with IXT (n = 15) and parents of 2- to 17-year-old children with IXT (n = 27). Responses were analyzed using standard item reduction methodology. Three final derived IXT questionnaires (IXTQ): child, proxy, and parent (12, 12, and 17 items, respectively) were administered to children with IXT and control children and to parents of IXT and control children. Likert-type scales ranging from never (100, best HRQOL) to almost always (0, worst HRQOL) were used.Main outcome measuresMedian scores for IXT and control groups, compared using Wilcoxon tests.ResultsMedian child scores were significantly lower (worse HRQOL) in the IXT group compared with the control group: 85 (quartiles, 73-92) versus 92 (quartiles, 79-96; P = 0.04). Median proxy IXTQ scores were significantly lower for IXT children than controls: 83 (quartiles, 75-94) versus 98 (quartiles, 92-100; P<0.0001). Median parent IXTQ scores also were significantly lower in the IXT group compared with the control group: 68 (quartiles, 56-79) versus 93 (quartiles, 87-99; P<0.0001).ConclusionsA new 3-part patient-derived HRQOL questionnaire for children with IXT and their parents has been developed and validated, comprising child, proxy, and parent questionnaires. These questionnaires detect reduced HRQOL in children with IXT as reported by the children themselves and perceived by their parents (proxy report). Childhood IXT also seems to affect parent HRQOL. The IXTQ HRQOL questionnaires may prove useful in the clinical assessment of IXT and for clinical trials.Financial disclosure(s)The author(s) have no proprietary or commercial interest in any materials discussed in this article.
| S-EPMC2815177 | biostudies-literature
Project description:In a recent work, we introduced a theoretical model for fulfillment in life that covers cognitive and affective components and distinguishes different time frames. The present study evaluates this model and describes the construction of the Fulfilled Life Scale (FLS) to assess fulfillment regarding the whole lived life retrospectively. We investigated the scale in two samples (Sample 1: N = 282 adults aged 50-93 years; Sample 2: N = 406 adults aged 40-85 years). The model of the cognitive component combines three sources of fulfillment (self, life, impact/legacy) with three criteria (wholeness, fit, value), yielding nine facets. Employing hierarchical factor analysis, we inspected all solutions between one and nine. We identified three optimal factors, which we labeled unfolded self and life, the worthwhile life, and positive impact and legacy. Next, we selected marker items and replicated the factor structure in Sample 2. The three scales were positively intercorrelated and showed good internal consistency in both samples. For the affective component, exploratory and confirmatory factor analyses established a one-factor structure in both samples, and high internal consistency was obtained. Across a range of related constructs, we demonstrated construct and criterion validity. Notably, cognitive and affective fulfillment incrementally predicted a global rating of a fulfilled life and mental well-being, even after controlling for subjective and eudaimonic well-being. Overall, the study proves that the FLS is necessary to capture people's experience of a fulfilled life, which could not be assessed sufficiently with previous well-being measures. Both cognitive and affective fulfillment were able to predict additional variance in mental well-being. Moreover, the study reveals psychometric support for the FLS and presents the first evidence on its validity. Lastly, applications in research and practice are discussed, especially in the context of living and aging well in the second half of life.
| S-EPMC8787361 | biostudies-literature
Project description:BackgroundFabry disease is a rare X-linked lysosomal storage disorder. It is associated with physical distress and social challenges that may affect adults differently compared to pediatric patients. However, there is no disease-specific quality of life (QOL) scale that can provide a detailed assessment of QOL for adults with Fabry disease. Therefore, we aimed to determine the factor structure and assess the validity of a scale that was created to assess the QOL of adult patients with Fabry disease. This study was conducted in two phases. First, scale feasibility was confirmed through a questionnaire survey of nine patients. Second, a cross-sectional questionnaire survey of patients (aged ≥ 18 years) diagnosed with Fabry disease was conducted. Item development and refinement were conducted based on guidelines for scale development. Exploratory factor analysis was used to clarify the factor structure and confirm internal consistency. As a measure of QOL, construct validity was of the scale was verified based on its correlations with the Short Form-8 (SF-8) scale.ResultsThe newly created Adult Fabry Disease QOL (AFQOL) scale comprises 39 items that cover five factors: "neuropathic pain and abdominal symptoms," "impact on work and school," "relationship challenges," "ophthalmologic and otolaryngologic symptoms," and "cardiovascular and renal symptoms." Cronbach's alpha coefficient for all factors was above 0.8, and the AFQOL total scores were significantly correlated with the physical and mental components of the SF-8 (rs = - 0.508 and - 0.400, respectively).ConclusionsThe AFQOL scale assesses physical symptoms and social difficulties experienced by adult patients with Fabry disease. A strength of the scale is its ability to assess the impact of work and relationships on patients. The scale can be useful in objectively assessing QOL for a group or for individual patients. Future research should explore further aspects of the scale's validity and reliability.
| S-EPMC9672224 | biostudies-literature
Project description:BACKGROUND:Advances in medical treatment for HIV are driving major changes in HIV policy and practice, including the encouragement of intake and adherence to HIV antiretroviral treatment (ART) by people living with HIV (PLHIV) for both personal and public health benefits. However, there is increasing recognition that achieving these goals will require a concurrent focus on the broader psychological and social wellbeing of PLHIV. Increasingly calls are being been made to incorporate a stronger focus on quality of life (QoL) of PLHIV into HIV prevention policy. In order to achieve this goal, HIV community, support and healthcare services need a valid, short and practical way to evaluate QoL of PLHIV accessing their programs. Current QoL measures are either long, complex, restricted in their use, or expensive. To address these shortcomings, the PozQoL study aimed to develop, test and validate a short and freely available scale assessing QoL among PLHIV. METHODS:Drawing on a literature review, the prioritisation of domains and development of the initial pool of items was conducted in consultation with PLHIV community organisations in Australia. The items covered health concerns, psychological, social, and functional wellbeing. Testing involved a baseline and a follow-up survey of 465 adult Australians living with HIV. Participants were recruited through social media and various community organizations nationwide. The survey included the pilot PozQoL scale and other validated measures of health and wellbeing. RESULTS:Guided by an Exploratory Factor Analysis and conceptual considerations, a 13-item scale was developed. The PozQoL scale demonstrated high levels of fit in a Confirmatory Factor Analysis, very good internal consistency, test-retest reliability, and concurrent validity with other measures that approximated different aspects of QoL. CONCLUSION:The PozQoL scale has been tested in a diverse sample of adult PLHIV living in Australia, demonstrating very good reliability and validity. The insights from PLHIV and other stakeholders supported the balancing of statistical rigour and conceptual accuracy. The scale is now ready to be implemented and field-tested across a range of community, support and healthcare programs for PLHIV. This will make a significant contribution to the evaluation and enhancement of programs for PLHIV.
| S-EPMC5910603 | biostudies-literature
Project description:Objective To develop a psychometrically reliable instrument to assess psychological distress during the COVID-19 pandemic across Arab countries. Design The new instrument was developed through the review of relevant literature. We adapted multiple items from the following tools: The Fear of COVID-19 Scale, Social Phobia Inventory, Health Anxiety Inventory, Swine Influenza Anxiety Scale and the Arabic Scale of Death Anxiety to design our new assessment tool which is called COVID-19 Psychological Distress Scale (CPDS). For psychometric analyses and validation, we conducted a cross-sectional study that solicited data through a web-based survey using the newly developed CPDS. Setting and participants This validation study was conducted in four Arab countries, including Algeria, Kuwait, Saudi Arabia and Yemen. A total of 1337 participants from these countries have voluntarily responded to our survey questionnaire that included the newly developed scale. Results The final version of the CPDS comprised 12 items. Participants from Algeria (n=447), Kuwait (n=437), Saudi Arabia (n=160) and Yemen (n=293) have completed the 12-item CPDS. Exploratory factor analysis (used on the Algerian sample) suggested a two-factor structure of the CPDS. The two-factor structure was then supported by the confirmatory factor analysis with an independent sample. Additionally, Rasch analyses showed that all the items fit well in their embedded construct; only one item showed somewhat substantial differential item functioning across gender and country. Conclusion The 12-item CPDS was found to be measurement invariant across country and gender. The CPDS, with its promising psychometric properties, might help healthcare professionals to identify people with COVID-19-induced psychological distress.
| S-EPMC8212153 | biostudies-literature
Project description:The present study describes the development and initial validation of a new obesity-specific, self-report measure of health-related quality of life (HRQOL) for children aged 5-13 years. Participants included 141 obese children (mean age=9.2 years, 67% female, 55% black, mean zBMI=2.52) and their primary caregivers. Children completed Sizing Me Up (obesity-specific HRQOL) and the PedsQL (generic HRQOL). Item content for Sizing Me Up was based on the published child obesity and HRQOL literatures and expert opinion. Items use phrasing to orient children to respond to questions in context of his/her size (e.g., "were teased by other kids because of your size"). Caregivers completed Sizing Them Up, a parallel parent-proxy, obesity-specific HRQOL measure. Initial psychometric evaluation of Sizing Me Up was completed by conducting a factor analysis and determining internal consistency, test-retest reliability, and convergent and construct validity. Sizing Me Up is a 22-item measure with five scales (i.e., Emotional Functioning, Physical Functioning, Social Avoidance, Positive Social Attributes, and Teasing/Marginalization) that account for 57% of the variance and a total HRQOL score. Internal consistency coefficients range from 0.68 to 0.85. Test-retest reliabilities range from 0.53 to 0.78. Good convergent validity was demonstrated with the PedsQL (rs=0.35-0.65) and the parent-proxy Sizing Them Up (rs=0.22-0.44). Sizing Me Up represents the first obesity-specific HRQOL measure developed specifically for younger school-aged children (aged 5-13 years) with preliminary evidence of strong psychometric properties that likely has both clinical and research utility in a variety of settings.
| S-EPMC3483635 | biostudies-literature
Project description:Purpose:Current instruments to assess thyroid eye disease (TED) quality of life (QoL) were not developed using modern psychometric theory and may not be applicable to Asian populations. Therefore, we developed a psychometrically robust questionnaire, the Singapore Thyroid Eye Disease Quality of Life questionnaire (STED-QoL), for assessing QoL in Asian patients. Methods:This cross-sectional study was conducted at the Singapore National Eye Centre between 2012 and 2015. In Phase 1, content for the questionnaire was developed using qualitative methods. A total of 20 patients participated in three different focus groups. Thematic analysis was conducted to identify relevant themes from which 12 items, rated on a 5-point Likert-type scale, were generated. In Phase 2, the pilot instrument was administered to 59 TED patients and psychometric assessment of the STED-QoL was conducted using Rasch analysis. Results:After collapsing categories from five to four and deleting two misfitting items, we generated a 10-item STED-QoL befitting the Rasch model. The scale showed good criterion validity, with scores decreasing as severity of TED worsened: mild (1.78 logits), moderate (0.27 logits), and severe (0.92 logits). A 'Psychosocial' subscale also had adequate psychometric properties and psychosocial scores were significantly worse in those who underwent surgery for TED compared to those who had not (0.41 vs. 1.82 logits, P = 0.021). Conclusions:The STED-QoL is a robust 10-item questionnaire specifically developed to measure the impact of TED on QoL and psychosocial well-being in an Asian population. Translational Relevance:QoL assessment is important for holistic management of TED patients.
| S-EPMC6166898 | biostudies-literature
Project description:BackgroundNearly one-third of youth are affected by a mental health disorder, and the majority do not receive adequate care. To improve clinical outcomes among youth, efforts have been made to train providers in evidence-based mental health practices, such as cognitive behavioral therapy (CBT). Such efforts call for valid assessment measures that can inform and evaluate training activities.AimsThis study presents the development and validation of the CBT Competence Scale (CCS), a brief self-report measure to assess provider competence for CBT delivery.MethodParticipants were 387 school mental health professionals (SMHPs) working with students in Michigan, USA. Initial items (n=59) were developed to evaluate competence in delivering common elements of CBT, with competence conceptualized as covering domains of knowledge, perception, and use of CBT techniques. CCS validation proceeded in three steps: using item response theory to select the most important items for assessing knowledge, evaluating the factor structure using exploratory and then confirmatory factor analyses, and examining reliability and validity of the resultant measure.ResultsThe validated CCS measure consists of four dimensions of CBT competence across 33 items: Non-behavioral skills, Behavioral skills, Perceptions, and Knowledge. The CCS demonstrated excellent internal consistency and good construct-based validity.ConclusionsThe CCS holds promise as a valid, informative measure of CBT competence appropriate for the school setting, with potential for application in other environments such as mental health clinics.
| S-EPMC9307077 | biostudies-literature
Project description:PurposeTo develop and validate a new disease-specific quality of life measure in hyperhidrosis for use in both routine clinical practice and clinical research.MethodsInterviews and focus group discussions with hyperhidrosis patients, reported elsewhere, provided the content for the measure validated in this study (n = 71). A panel of dermatologists (n = 5) and patients (n = 7) carried out content validation. Further, item reduction and the initial construct validation were carried out in a cross-sectional study (n = 595), using the unidimensional Rasch analysis and exploratory factor analysis. Subsequently, the construct validity, reliability and responsiveness of the revised measure were assessed in a longitudinal study (n = 260). Data collection for the item reduction and the final validation phases was entirely carried out online.ResultsThe expert panels judged the HidroQoL as content valid. Rasch analysis supported the revision of response options from five to three. Following removal of misfitting items, a set of 15 items showed optimal fit to the model (chi-squared statistic = 159.64, p = 0.07). Three additional items were retained on consideration of their importance to patients, resulting in an 18-item instrument. The items were grouped into two subscales, daily life activities and psychosocial life domains, based on results of the factor analysis. In subsequent construct validation, the HidroQoL correlated with the DLQI (r s = 0.6, p < 0.01). Reliability was high (internal consistency, Cronbach's alpha: overall scale = 0.9; test-retest reliability, Intra-class correlation = 0.9). The HidroQoL scores were sensitive to change in patients' disease severity (score change from baseline to follow-up after 15-35 days, Cohen's ES = 0.47).ConclusionThis study has provided the initial evidence supporting measurement properties and the use of the HidroQoL instrument in both routine clinical practice and in research, for assessing quality of life impacts in hyperhidrosis.
| S-EPMC4366556 | biostudies-literature
Project description:The mandatory home confinement of the Spanish population, implemented in response to the COVID-19 pandemic, presents a unique opportunity to study the use and influence of Information and Communication Technologies (ICT) in people's perception of quality of life during this exceptional situation. This article adapts and validates a psychometric scale designed to identify and measure the main dimensions of the Quality of Life construct perceived through ICT use. To this end, an exploratory and transversal study has been carried out in Spain on a sample of 2,346 participants. Data processing has been carried out with SPSS and EQS. The results provide evidence of the reliability and psychometric quality on the scale, which exhibits adequate consistency that facilitates its application. The confirmatory factor analysis showed a hierarchical model of three correlated factors that account for the dimensions "Satisfaction with life", "Emotional support" and "Social support", which have enough correlation to measure the personal perception of quality of life associated with ICT use and are consistent with previous psychometric studies. The results of the TICO scale indicate that more than 70% of the sample feel ICT have united their family during home confinement and more than 45% experience happy feelings when they use ICT. In home confinement, ICT use has improved users' quality of life, mainly their satisfaction with life and social and family support.
| S-EPMC7643959 | biostudies-literature