Health and Quality of Life Outcomes最新文献

{"title":"A sex-oriented analysis concerning skeletal muscle quantity and quality and associations to quality of life in hospitalized patients with cirrhosis","authors":"Jie Yang, Gaoyue Guo, Fang Yang, Chaoqun Li, Han Wang, Wanting Yang, Ziyi Yang, Qing Liu, Qian Li, Chao Sun","doi":"10.1186/s12955-024-02295-2","DOIUrl":"https://doi.org/10.1186/s12955-024-02295-2","url":null,"abstract":"There is a paucity of data regarding sex-oriented analyses of connection between muscle quantity and quality and health-related quality of life (HRQoL), taking into account the pathophysiological differences of sarcopenia/myosteatosis in males versus females. We sought to investigate the associations between skeletal muscle index (SMI)-defined sarcopenia and intramuscular adipose tissue content (IMAC)-defined myosteatosis and EuroQol-5D (EQ-5D)-defined HRQoL in patients with decompensated cirrhosis concerning sex disparities. Totally, 382 patients were enrolled. The relationship between SMI/IMAC and HRQoL was evaluated with restricted cubic spline and Pearson correlation analyses. Furthermore, association between SMI or sarcopenia and EQ-5D utility index was determined by multiple linear regression, adjusted for age, BMI and concurrent disease severity. The study population comprised evenly distributed male and female patients (190: 192), mean age 61.9 years. The prevalence of sarcopenia (40.5 versus 9.9%, P < 0.001) and SMI (48.8 versus 42.2 cm2/m2, P < 0.001) were significantly higher in males relative to females, with comparable myosteatosis prevalence (15.3 versus 16.7%, P = 0.708). Self-care, usual activities and pain within EQ-5D scale were more prevalent in the sarcopenia compared with non-sarcopenia groups across entire population and stratified by sex. The SMI values exhibited a significantly linear correlation with EQ-5D utility index in male but not female patients (P for non-linearity = 0.281). In multiple analysis, SMI or the presence of sarcopenia was both significantly associated with EQ-5D utility index. Subgroup analyses unveiled no discernible interactions between sarcopenia and EQ-5D utility index. Muscle quantity measured by SMI was associated with declined HRQoL in males rather than females, whereas no associations were found regarding muscle quality measured by IMAC in both sexes. It is tempting to manage sarcopenia by increasing SMI levels as high as possible in hopes of achieving better health consequence. Our findings represent the importance of connecting CT-demarcated body composition abnormalities to meaningful patient-centered outcomes. Future targeted studies with sizable multi-center populations are warranted to clarify this causality, and in consequence develop optimized intervention against sarcopenia/myosteatosis or key determinants concerning impaired HRQoL.","PeriodicalId":12980,"journal":{"name":"Health and Quality of Life Outcomes","volume":"9 1","pages":""},"PeriodicalIF":3.6,"publicationDate":"2024-09-12","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142203862","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Dynamic changes in quality of life in older patients with chronic obstructive pulmonary disease: a 7-year follow up","authors":"Chun-Hsiang Yu, Sheng-Han Tsai, Jo-Ying Hung, Pei-Fang Su, Chih-Hui Hsu, Xin-Min Liao, Tzuen-Ren Hsiue, Chiung-Zuei Chen","doi":"10.1186/s12955-024-02296-1","DOIUrl":"https://doi.org/10.1186/s12955-024-02296-1","url":null,"abstract":": Chronic obstructive pulmonary disease (COPD) is a major cause of the rapid decline of health-related quality of life (HRQoL), associated with accelerated frailty in older populations. This study aimed to analyse the long-term dynamic changes of HRQoL and the predictive factors for the rapid decline of HRQoL in older patients with COPD. Overall 244 patients with COPD, aged ≧ 65 years from one medical centre were enrolled between March 2012 and July 2020. Further, we prospectively assessed HRQoL scores with utility values, using EuroQol Five-Dimension (EQ-5D) questionnaires. Additionally, long-term dynamic changes in HRQoL were analysed using the Kernel smoothing method and examined the factors contributing to the deterioration of HRQoL using a linear mixed effects model. Older patients with COPD with forced expiration volume (FEV1) < 50% of prediction entered the phase of rapid and continuous decline of HRQoL ~ 2 years after enrolment, but patients with FEV1 ≥ 50% of prediction without rapidly declined HRQoL during 7 years follow up. Therefore, FEV1 < 50% of prediction is a novel predictor for the rapid decline of HRQoL. The course of rapidly declining HRQoL occurred, initially in the usual activities and pain/discomfort domains, followed by the morbidity, self-care, and depression/anxiety domains ~ 2 and 4 years after enrolment, respectively. The mixed effects model indicated that both FEV1 < 50% of prediction and a history of severe acute exacerbation (SAE) requiring hospitalisation were contributing factors for deterioration in HRQoL . Both FEV1 < 50% of prediction and exacerbations requiring hospitalisation were contributing factors for the deterioration of HRQoL in long-term follow up. Additionally, FEV1 < 50% of prediction was a novel predictor for patients entering the phase of rapid decline of HRQoL.","PeriodicalId":12980,"journal":{"name":"Health and Quality of Life Outcomes","volume":"12 1","pages":""},"PeriodicalIF":3.6,"publicationDate":"2024-09-11","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142203816","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Validation of the parents’ version of the KINDLR and Kiddy Parents questionnaire in a South African context","authors":"Elmari Deacon, Esmé Jansen van Vuren, Elizabeth Bothma, Chanelle Volschenk, Ruan Kruger","doi":"10.1186/s12955-024-02292-5","DOIUrl":"https://doi.org/10.1186/s12955-024-02292-5","url":null,"abstract":"This study aimed to assess the usefulness of the parent version of the KINDLR and the additional items of the Kiddy Parents questionnaire in the South-African context and to validate it as an appropriate tool for measuring health-related quality of life (HRQoL). The ExAMIN Youth SA study was designed to investigate lifestyle behaviours, including psychosocial factors that may adversely impact on cardiovascular health of children. Construct validity was examined by using exploratory and confirmatory factor analysis, while internal consistency was tested by Cronbach’s alpha. The final factor structure was confirmed by model fit indices. The study included children (n = 1088) aged between 5 and 10 years in North-West, South Africa. The reliability coefficients of the original factors could not be reproduced in this data set, with the Cronbach’s alphas ranging between 0.46 and 0.78. With exploratory factor analysis, including the additional items, our data supported a 7-factor structure with acceptable internal consistency (Cronbach’s alpha: 0.68–0.79; Omega: 0.75–0.85) and acceptable model fit indices (CFI: 0.91; TLI: 0.90; RMSEA: 0.05; SRMR: 0.07). Two factors (emotional wellbeing and everyday functioning) further split into separate factors for positive and negative experiences related to each of these dimensions. We confirmed a new factor structure of the parent version of the KINDLR and the additional items of the Kiddy Parents questionnaire, which can be used in the African context. Although the new factor structure has great overlap with the original structure, some items did not contribute to the factors as expected. Language and cultural differences between the original German group and the current South African study group resulted in a different factor structure.","PeriodicalId":12980,"journal":{"name":"Health and Quality of Life Outcomes","volume":"402 1","pages":""},"PeriodicalIF":3.6,"publicationDate":"2024-09-11","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142203815","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"How are maternal and fetal outcomes incorporated when measuring benefits of interventions in pregnancy? Findings from a systematic review of cost-utility analyses","authors":"Lucy Abel, Helen Dakin, Ting Cai, Richard J. McManus, Abigail McNiven, Oliver Rivero-Arias","doi":"10.1186/s12955-024-02293-4","DOIUrl":"https://doi.org/10.1186/s12955-024-02293-4","url":null,"abstract":"Medical interventions used in pregnancy can affect the length and quality of life of both the pregnant person and fetus. The aim of this systematic review was to identify and describe the theoretical frameworks that underpin outcome measurement in cost-utility analyses of pregnancy interventions. Searches were conducted in the Paediatric Economic Database Evaluation (PEDE) database (up to 2017), as well as Medline, Embase and EconLit (2017–2019). We included all cost-utility analyses of any intervention given during pregnancy, published in English. We conducted a narrative synthesis of: study design; outcome construction (life expectancy, quality adjustment, discount rate); and whether the Incremental Cost-Effectiveness Ratio (ICER) was constructed using maternal or fetal outcomes. Where both outcomes were included, methods for combining them were extracted. We identified 127 cost-utility analyses in pregnancy, of which 89 reported QALYs and 38 DALYs. Outcomes were considered solely for the fetus in 59 studies (47%), solely for the pregnant person in 13 studies (10%), and for both in 49 studies (39%). The choice to include or exclude one or both sets of outcomes was not consistent within particular clinical areas. Where outcomes for both mother and baby were included, methods for combining these outcomes varied. Twenty-nine studies summed QALYs/DALYs for maternal and fetal outcomes, with no adjustment. The remaining 20 took a variety of approaches designed to weigh maternal and fetal outcomes differently. These include (1) treating fetal outcomes as a component of maternal quality of life, rather than (or in addition to) an independent individual health outcome; (2) treating the maternal-fetal dyad as a single entity and applying a single utility value to each combination of outcomes; and (3) assigning a shorter time horizon to fetal outcomes to reduce the weight of lifetime fetal outcomes. Each approach made different assumptions about the relative value of maternal and fetal health outcomes, demonstrating a lack of consistency and the need for guidance. Methods for capturing QALY/DALY outcomes in cost-utility analysis in pregnancy vary widely. This lack of consistency indicates a need for new methods to support the valuation of maternal and fetal health outcomes.","PeriodicalId":12980,"journal":{"name":"Health and Quality of Life Outcomes","volume":"27 1","pages":""},"PeriodicalIF":3.6,"publicationDate":"2024-09-11","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142203863","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Health state utility values of type 2 diabetes mellitus and related complications: a systematic review and meta-regression.","authors":"Yubo Wang, Yueru Xu, Huiting Shan, Huimin Pan, Ji Chen, Jianhua Yang","doi":"10.1186/s12955-024-02288-1","DOIUrl":"10.1186/s12955-024-02288-1","url":null,"abstract":"<p><strong>Background: </strong>This study aimed to synthesize and quantitatively examine Health State Utility Values (HSUVs) for Type 2 Diabetes Mellitus (T2DM) and its complications, providing a robust meta-regression framework for selecting appropriate HSUV estimates.</p><p><strong>Method: </strong>We conducted a systematic review to extract HSUVs for T2DM and its complications, encompassing various influencing factors. Relevant literature was sourced from a review spanning 2000-2020, supplemented by literature from PubMed, Embase, and the Web of Science (up to March 2024). Multivariate meta-regression was performed to evaluate the impact of measurement tools, tariffs, health status, and clinical and demographic variables on HSUVs.</p><p><strong>Results: </strong>Our search yielded 118 studies, contributing 1044 HSUVs. The HSUVs for T2DM with complications varied, from 0.65 for cerebrovascular disease to 0.77 for neuropathy. The EQ-5D-3L emerged as the most frequently employed valuation method. HSUV differences across instruments were observed; 15-D had the highest (0.89), while HUI-3 had the lowest (0.70) values. Regression analysis elucidated the significant effects of instrument and tariff choice on HSUVs. Complication-related utility decrement, especially in diabetic foot, was quantified. Age <70 was linked to increased HSUVs, while longer illness duration, hypertension, overweight and obesity correlated with reduced HSUVs.</p><p><strong>Conclusion: </strong>Accurate HSUVs are vital for the optimization of T2DM management strategies. This study provided a comprehensive data pool for HSUVs selection, and quantified the influence of various factors on HSUVs, informing analysts and policymakers in understanding the utility variations associated with T2DM and its complications.</p>","PeriodicalId":12980,"journal":{"name":"Health and Quality of Life Outcomes","volume":"22 1","pages":"74"},"PeriodicalIF":3.2,"publicationDate":"2024-09-07","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11380328/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142145577","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Psychometric properties of computerized adaptive testing for chronic obstructive pulmonary disease patient-reported outcome measurement.","authors":"Jiajia Wang, Yang Xie, Zhenzhen Feng, Jiansheng Li","doi":"10.1186/s12955-024-02291-6","DOIUrl":"10.1186/s12955-024-02291-6","url":null,"abstract":"<p><strong>Background: </strong>Computerized adaptive testing (CAT) is an effective way to reduce time, repetitious redundancy, and respond burden, and has been used to measure outcomes in many diseases. This study aimed to develop and validate a comprehensive disease-specific CAT for chronic obstructive pulmonary disease (COPD) patient-reported outcome measurement.</p><p><strong>Methods: </strong>The discrimination and difficulty of the items from the modified patient-reported outcome scale for COPD (mCOPD-PRO) were analyzed using item response theory. Then the initial item, item selection method, ability estimation method, and stopping criteria were further set based on Concerto platform to form the CAT. Finally, the reliability and validity were validated.</p><p><strong>Results: </strong>The item discrimination ranged from 1.05 to 2.71, and the item difficulty ranged from - 3.08 to 3.65. The measurement reliability of the CAT ranged from 0.910 to 0.922 using random method, while that ranged from 0.910 to 0.924 using maximum Fisher information (MFI) method. The content validity was good. The correlation coefficient between theta of the CAT and COPD assessment test and modified Medical Research Council dyspnea scale scores using random method was 0.628 and 0.540 (P < 0.001; P < 0.001) respectively, while that using MFI method was 0.347 and 0.328 (P = 0.007; P = 0.010) respectively. About 11 items (reducing by 59.3%) on average were tested using random method, while about seven items (reducing by 74.1%) on average using MFI method. The correlation coefficient between theta of the CAT and mCOPD-PRO total scores using random method was 0.919 (P < 0.001), while that using MFI method was 0.760 (P < 0.001).</p><p><strong>Conclusions: </strong>The comprehensive disease-specific CAT for COPD patient-reported outcome measurement is well developed with good psychometric properties, which can provide an efficient, accurate, and user-friendly measurement for patient-reported outcome of COPD.</p>","PeriodicalId":12980,"journal":{"name":"Health and Quality of Life Outcomes","volume":"22 1","pages":"73"},"PeriodicalIF":3.2,"publicationDate":"2024-09-04","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11373186/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142125558","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Perceptions of adults with type 1 diabetes toward diabetes-specific quality of life measures: a survey-based qualitative exploration.","authors":"Elizabeth Holmes-Truscott, Jasmine Schipp, Debbie D Cooke, Christel Hendrieckx, Elizabeth J Coates, Simon R Heller, Jane Speight","doi":"10.1186/s12955-024-02285-4","DOIUrl":"10.1186/s12955-024-02285-4","url":null,"abstract":"<p><strong>Background: </strong>Diabetes-specific quality of life (QoL) questionnaires are commonly used to assess the impact of diabetes and its management on an individual's quality of life. While several valid and reliable measures of diabetes-specific QoL exist, there is no consensus on which to use and in what setting. Furthermore, there is limited evidence of their acceptability to people with diabetes. Our aim was to explore perceptions of adults with type 1 diabetes (T1D) toward five diabetes-specific QoL measures.</p><p><strong>Methods: </strong>Adults (aged 18 + years) with T1D living in Australia or the United Kingdom (UK) were eligible to take part in 'YourSAY: QoL', an online cross-sectional survey. Recruitment involved study promotion on diabetes-related websites and social media, as well as direct invitation of people with T1D via a hospital client list (UK only). In random order, participants completed five diabetes-specific QoL measures: Audit of Diabetes-Dependent Quality of Life (ADDQoL-19); Diabetes Care Profile: Social and Personal Factors subscale (DCP); DAWN Impact of Diabetes Profile (DIDP); Diabetes-Specific Quality of Life Scale: Burden Subscale (DSQoLS); Diabetes Quality of Life Questionnaire (Diabetes QOL-Q). They were invited to provide feedback on each questionnaire in the form of a brief free-text response. Responses were analysed using inductive, thematic template analysis.</p><p><strong>Results: </strong>Of the N = 1,946 adults with T1D who completed the survey, 20% (UK: n = 216, Australia: n = 168) provided qualitative responses about ≥ 1 measure. All measures received both positive and negative feedback, across four themes: (1) clarity and ease of completion, e.g., difficulty isolating impact of diabetes, dislike of hypothetical questions, and preference for 'not applicable' response options; (2) relevance and comprehensiveness, e.g., inclusion of a wide range of aspects of life to improve personal relevance; (3) length and repetition, e.g., length to be balanced against respondent burden; (4) framing and tone, e.g., preference for respectful language and avoidance of extremes.</p><p><strong>Conclusions: </strong>These findings suggest opportunities to improve the relevance and acceptability of existing diabetes-specific QoL measures, and offer considerations for developing new measures, which need to be better informed by the preferences of people living with diabetes.</p>","PeriodicalId":12980,"journal":{"name":"Health and Quality of Life Outcomes","volume":"22 1","pages":"70"},"PeriodicalIF":3.2,"publicationDate":"2024-09-02","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11367869/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142106915","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Psychometrics of the modified family-centered care assessment short version for childhood obesity.","authors":"Meg Simione, Paola Ferreira, Man Luo, Clarissa Hoover, Meghan Perkins, Lauren Fiechtner, Elsie M Taveras","doi":"10.1186/s12955-024-02284-5","DOIUrl":"10.1186/s12955-024-02284-5","url":null,"abstract":"<p><strong>Background: </strong>Incorporating principles of family-centered care into pediatric weight management interventions can improve the effectiveness and quality of treatment and reduce attrition rates. To assess the family-centeredness of interventions, reliable, valid, and easy-to-administer scales are needed. The purpose of the study was to develop a shortened version of the modified Family Centered Care Assessment (mFCCA) and assess its psychometric properties.</p><p><strong>Methods: </strong>The mFCCA, a scale to assess the family-centeredness of interventions for childhood obesity, was administered to families following the Connect for Health randomized control trial evaluating the effectiveness of a primary care-based pediatric weight management intervention. We iteratively removed items from the mFCCA and used Rasch modeling to examine the reliability and validity of the shortened scale.</p><p><strong>Results: </strong>We included data from 318 parents and the exploratory factor analysis showed the presence of a single factor. The results of the Rasch modeling demonstrated acceptable internal consistency of the scale (0.7) and strong validity as evidenced by the overall model fit and range of item difficulty. Following the psychometric analyses, we reduced the number of items from 24 to 8 items.</p><p><strong>Conclusion: </strong>The mFCCA short version demonstrates good psychometrics and can be used to evaluate the family-centeredness of childhood obesity interventions with reduced participant burden, thereby improving outcomes for children with obesity.</p><p><strong>Trial registration: </strong>Clinicaltrials.gov NCT02124460 registered on April 24, 2014.</p>","PeriodicalId":12980,"journal":{"name":"Health and Quality of Life Outcomes","volume":"22 1","pages":"71"},"PeriodicalIF":3.2,"publicationDate":"2024-09-02","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11367898/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142106916","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Cataloging health state utility estimates for Duchenne muscular dystrophy and related conditions.","authors":"Lauren A Do, Lauren E Sedita, Alexa C Klimchak, Rachel Salazar, David D Kim","doi":"10.1186/s12955-024-02287-2","DOIUrl":"10.1186/s12955-024-02287-2","url":null,"abstract":"<p><strong>Background: </strong>Duchenne muscular dystrophy (DMD) is a genetic disease resulting in progressive muscle weakness, loss of ambulation, and cardiorespiratory complications. Direct estimation of health-related quality of life for patients with DMD is challenging, highlighting the need for proxy measures. This study aims to catalog and compare existing published health state utility estimates for DMD and related conditions.</p><p><strong>Methods: </strong>Using two search strategies, relevant utilities were extracted from the Tufts Cost-Effectiveness Analysis Registry, including health states, utility estimates, and study and patient characteristics. Analysis One identified health states with comparable utility estimates to a set of published US patient population utility estimates for DMD. A minimal clinically important difference of ± 0.03 was applied to each DMD utility estimate to establish a range, and the registry was searched to identify other health states with associated utilities that fell within each range. Analysis Two used pre-defined search terms to identify health states clinically similar to DMD. Mapping was based on the degree of clinical similarity.</p><p><strong>Results: </strong>Analysis One identified 4,308 unique utilities across 2,322 cost-effectiveness publications. The health states captured a wide range of acute and chronic conditions; 34% of utility records were extrapolated for US populations (n = 1,451); 1% were related to pediatric populations (n = 61). Analysis Two identified 153 utilities with health states clinically similar to DMD. The median utility estimates varied among identified health states. Health states similar to the early non-ambulatory DMD phase exhibited the greatest difference between the median estimate of the sample (0.39) and the existing estimate from published literature (0.21).</p><p><strong>Conclusions: </strong>When available estimates are limited, using novel search strategies to identify utilities of clinically similar conditions could be an approach for overcoming the information gap. However, it requires careful evaluation of the utility instruments, tariffs, and raters (proxy or self).</p>","PeriodicalId":12980,"journal":{"name":"Health and Quality of Life Outcomes","volume":"22 1","pages":"72"},"PeriodicalIF":3.2,"publicationDate":"2024-09-02","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11367812/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142106903","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Development of a novel patient reported outcome measure for health-related quality of life in amyotrophic lateral sclerosis (PROQuALS): study protocol.","authors":"Jill Carlton, Philip Powell, Donna Rowen, Claire Williams, Alys Wyn Griffiths, Esther Hobson, Christopher McDermott","doi":"10.1186/s12955-024-02286-3","DOIUrl":"https://doi.org/10.1186/s12955-024-02286-3","url":null,"abstract":"<p><strong>Background: </strong>Patient reported outcome measures (PROMs) can be used to assess the impact of health conditions upon an individual's health-related quality of life (HRQoL). Whilst PROMs have been used to quantify the HRQoL impact of amyotrophic lateral sclerosis (ALS), existing instruments may not fully capture what matters to people living with ALS (plwALS) or be appropriate to be used directly to inform the cost-effectiveness of new treatments. This highlights a need for a new condition-specific PROM that can both capture what's important to plwALS and be used in economic evaluation. This study has two key aims: 1) to produce a novel PROM for measuring HRQoL in plwALS (PROQuALS). 2) to value a set of items from the novel PROM to generate an associated preference-weighted measure (PWM) that will enable utility values to be generated.</p><p><strong>Methods: </strong>A mixed-methods study design will be conducted across three stages. Stage 1 involves concept elicitation and the generation of draft PROM content from a robust and comprehensive systematic review of HRQoL in ALS, with input from plwALS. Stage 2 consists of cognitive debriefing of the draft PROM content to ascertain its content validity (Stage 2a), followed by a psychometric survey (Stage 2b) to assess statistical performance. Evidence from Stage 2 will be used to make decisions on the final content and format of the novel PROM. Stage 3 will involve valuation and econometric modeling using health economics methods to generate preference weights, so a PWM derived from the novel PROM can be used in the cost-effectiveness analyses of treatments. Patient and clinical advisory groups will have critical, collaborative input throughout the project.</p><p><strong>Discussion: </strong>The novel PROM will be designed to comprehensively assess important aspects of HRQoL to plwALS and to quantify HRQoL in terms of subjective impact. The PROQuALS measure will be available for use in research and healthcare settings. The associated PWM component will extend and enable the use of PROQuALS in cost-effective analyses of new treatments for ALS.</p><p><strong>Trial registration: </strong>Not applicable.</p>","PeriodicalId":12980,"journal":{"name":"Health and Quality of Life Outcomes","volume":"22 1","pages":"69"},"PeriodicalIF":3.2,"publicationDate":"2024-08-30","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11365122/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142106914","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}