Health and Quality of Life Outcomes最新文献

{"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":null,"pages":null},"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":null,"pages":null},"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":null,"pages":null},"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}

{"title":"Association between sensory processing sensitivity and quality of life among cancer patients: a mediation and moderation of resilience and social determinants.","authors":"Qinghuan Yang, Jiao Yang, Xudong Xiang, Yanqiu Zhao, Xiaomin Sun, Yu Xing, Ni Jiang, Yuanxiao Wang, Hailiang Ran, Qiubo Huang","doi":"10.1186/s12955-024-02283-6","DOIUrl":"10.1186/s12955-024-02283-6","url":null,"abstract":"<p><strong>Background: </strong>Individuals with sensory processing sensitivity (SPS) tend to be overreactive in response to negative environmental stimuli. More is known about the positive relationship between SPS and quality of life (QoL); nevertheless, less is known regarding the roles of resilience and social determinants in this association. This research aimed to investigate the potential mediation effect of resilience and the moderation effect of social determinants on the relationship between SPS and QoL in a large sample of Chinese cancer patients.</p><p><strong>Methods: </strong>We used the most recent datasets from an ongoing project conducted in southwest China. A two-stage random sampling strategy with a probability proportionate to sample size (PPS) design was adopted. The associations between resilience, SPS, and QoL were evaluated using a linear regression model. Path analysis was adopted to examine the mediation of resilience.</p><p><strong>Results: </strong>Resilience was positively associated with quality of life, while increased sensory processing sensitivity was negatively associated with quality of life. The restricted cubic spline analysis revealed that as resilience increased, the coefficients of quality of life rapidly increased across all domains. Conversely, the coefficients for quality of life gradually decreased with the escalation of sensory processing sensitivity. Resilience was a significant mediator, accounting for 21.88% of the total SPS-QoL association. The mediation effect of resilience varied across ethnicity and sex.</p><p><strong>Conclusion: </strong>Sensory processing sensitivity was significantly associated with quality of life in cancer patients, and promoting resilience could mitigate this negative impact. However, the effect of resilience varies across sex and ethnicity. Therefore, targeted resilience promotion interventions, especially those integrating social characteristics, should be considered for implementation.</p>","PeriodicalId":12980,"journal":{"name":"Health and Quality of Life Outcomes","volume":null,"pages":null},"PeriodicalIF":3.2,"publicationDate":"2024-08-23","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11342669/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142046612","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":"Active social engagement and health among older adults: assessing differences by cancer survivorship status.","authors":"Jennifer L Moss, Veronica Bernacchi, Erin Kitt-Lewis","doi":"10.1186/s12955-024-02281-8","DOIUrl":"10.1186/s12955-024-02281-8","url":null,"abstract":"<p><strong>Introduction: </strong>The number of older adults who are cancer survivors is rapidly growing. Evidence is needed to inform interventions to support successful aging among older adults (including older adult cancer survivors). Active engagement with life, that is, spending time with family and/or close friends, may be related to health outcomes, but this concept remains understudied.</p><p><strong>Methods: </strong>We used survey data to assess active engagement among older adults (ages 50 + years) from seven mid-Atlantic US states (n = 2,914), and geocoded their residence to collect collected measures of community availability of social interaction. Outcomes were physical and mental health-related quality of life (HRQoL), assessed with the SF-12. We used multivariable, multilevel linear regression to evaluate relationships between social interactions (i.e., \"active engagement with life,\" or visiting with family and/or friends at least once per week and having at least three close friends, and community-level availability, measured with census tract-level park land and walkability and with county-level availability of social associations) and HRQoL. Finally, we explored differences in these relationships by recent cancer survivorship.</p><p><strong>Results: </strong>Overall, 1,518 (52.3%) participants were actively engaged. Active engagement was associated with higher physical HRQoL (estimate = 0.94, standard error [SE] = 0.46, p = .04) and mental HRQoL (estimate = 2.10, SE = 0.46, p < .001). The relationship between active engagement and physical HRQoL was stronger for recent cancer survivors (estimate = 4.95, SE = 1.84, p < .01) than for the general population (estimate = 1.10, SE = 0.43, p = .01). Community-level availability of social interaction was not associated with HRQoL.</p><p><strong>Conclusion: </strong>Our analysis demonstrated promising associations between active engagement with life and HRQoL among older adults, with large benefits for older cancer survivors. Additional research is needed on how active engagement is associated with better HRQoL, which can inform future policies and programs to optimize the aging process in the US.</p>","PeriodicalId":12980,"journal":{"name":"Health and Quality of Life Outcomes","volume":null,"pages":null},"PeriodicalIF":3.2,"publicationDate":"2024-08-20","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11337903/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142008746","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":"Validation and psychometric evaluation of the Short Warwick-Edinburgh Mental Well-Being Scale (SWEMWBS) among Czech adolescents using Item Response Theory.","authors":"Radka Hanzlová, Aleš Kudrnáč","doi":"10.1186/s12955-024-02280-9","DOIUrl":"10.1186/s12955-024-02280-9","url":null,"abstract":"<p><strong>Background: </strong>The topic of adolescent mental health is currently a subject of much debate due to the increasing prevalence of mental health problems among this age group. Therefore, it is crucial to have high-quality and validated mental well-being measurement tools. While such tools do exist, they are often not tailored specifically to adolescents and are not available in Czech language. The aim of this study is to validate and test the Czech version of the Short Warwick-Edinburgh Mental Well-Being Scale (SWEMWBS) on a large sample of Czech adolescents aged 15 to 18 years.</p><p><strong>Methods: </strong>The analysis is based on data from the first wave of the Czech Education Panel Survey (CZEPS) and was mainly conducted using Item Response Theory (IRT), which is the most appropriate method for this type of analysis. Specifically, the Graded Response Model (GRM) was applied to the data. This comprehensive validation study also included reliability and three types of validity (construct, convergent and criterion) testing.</p><p><strong>Results: </strong>The study found that the Czech version of the SWEMWBS for adolescents aged 15 to 18 years (N = 22,498) has good quality and psychometric properties. The data was analysed using the GRM model as it met the assumptions for the use of IRT. The estimated parameter values by GRM demonstrated good discriminant and informative power for all items, except for item 7, which showed poorer results compared to the others. However, excluding it from the scale would not enhance the overall quality of the scale. The five-category response scale functions effectively. Additionally, the results demonstrated high reliability, and all types of validity tested were also confirmed.</p><p><strong>Conclusions: </strong>The Czech version of the SWEMWBS for adolescents has been validated as a psychometrically sound, reliable and valid instrument for measuring mental well-being. It can therefore be used with confidence in future studies.</p>","PeriodicalId":12980,"journal":{"name":"Health and Quality of Life Outcomes","volume":null,"pages":null},"PeriodicalIF":3.2,"publicationDate":"2024-08-19","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11331616/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142004112","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":"Heterogeneity of patient-reported outcome measures in clinical research.","authors":"Jan Henrik Terheyden, Lisa Gittel, Julie Jungblut, Deanna J Taylor, Frank G Holz, David P Crabb, Robert P Finger","doi":"10.1186/s12955-024-02282-7","DOIUrl":"10.1186/s12955-024-02282-7","url":null,"abstract":"<p><strong>Background: </strong>The use of patient-reported outcome measures (PROMs) in clinical research increases and use of heterogeneous instruments reflects how well diverse traits are captured by a medical specialty. In order to reflect the heterogeneity of current PROM use in ophthalmology, we reviewed the available literature.</p><p><strong>Methods: </strong>The medical literature database Web of Science was searched for the most cited articles in clinical ophthalmology. Titles, abstracts and full text articles were reviewed for the use of PROMs and a list of the 100 most cited articles using PROMs was obtained and stratified by year of publication.</p><p><strong>Results: </strong>A total of 1,996 articles were screened. Seventy-seven out of the 100 articles identified included one PROM, and the average number of instruments was 1.5 ± 1.1. The most widely used PROMs were the National Eye Institute Visual Function Questionnaire (33%), the Ocular Surface Disease Index (14%) and the Medical Outcomes Study Short Form (13%). A simulation analysis suggested that the distribution of PROM use in ophthalmology study did not significantly differ from a power law distribution. Twenty-two percent and fifteen percent of articles did not reference and did not specify the PROM used, respectively. This rate decreased in the more recently published articles (p = 0.041).</p><p><strong>Conclusions: </strong>Our data suggest that the heterogeneity of PROMs applied in ophthalmology studies is low. The selection of PROMs for clinical studies should be done carefully, depending on the research goal.</p>","PeriodicalId":12980,"journal":{"name":"Health and Quality of Life Outcomes","volume":null,"pages":null},"PeriodicalIF":3.2,"publicationDate":"2024-08-17","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11330056/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141995653","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":"Converting PROMIS^®-29 v2.0 profile data to SF-36 physical and mental component summary scores in patients with cardiovascular disorders.","authors":"Gregor Liegl, Felix H Fischer, Carl N Martin, Maria Rönnefarth, Annelie Blumrich, Michael Ahmadi, Leif-Hendrik Boldt, Kai-Uwe Eckardt, Matthias Endres, Frank Edelmann, Holger Gerhardt, Ulrike Grittner, Arash Haghikia, Norbert Hübner, Ulf Landmesser, David Leistner, Knut Mai, Jil Kollmus-Heege, Dominik N Müller, Christian H Nolte, Sophie K Piper, Kai M Schmidt-Ott, Tobias Pischon, Simrit Rattan, Ira Rohrpasser-Napierkowski, Katharina Schönrath, Jeanette Schulz-Menger, Oliver Schweizerhof, Joachim Spranger, Joachim E Weber, Martin Witzenrath, Sein Schmidt, Matthias Rose","doi":"10.1186/s12955-024-02277-4","DOIUrl":"10.1186/s12955-024-02277-4","url":null,"abstract":"<p><strong>Background: </strong>Health-related quality of life (HRQL) has become an important outcome parameter in cardiology. The MOS 36-ltem Short-Form Health Survey (SF-36) and the PROMIS-29 are two widely used generic measures providing composite HRQL scores. The domains of the SF-36, a well-established instrument utilized for several decades, can be aggregated to physical (PCS) and mental (MCS) component summary scores. Alternative scoring algorithms for correlated component scores (PCS_c and MCS_c) have also been suggested. The PROMIS-29 is a newer but increasingly used HRQL measure. Analogous to the SF-36, physical and mental health summary scores can be derived from PROMIS-29 domain scores, based on a correlated factor solution. So far, scores from the PROMIS-29 are not directly comparable to SF-36 results, complicating the aggregation of research findings. Thus, our aim was to provide algorithms to convert PROMIS-29 data to well-established SF-36 component summary scores.</p><p><strong>Methods: </strong>Data from n = 662 participants of the Berlin Long-term Observation of Vascular Events (BeLOVE) study were used to estimate linear regression models with either PROMIS-29 domain scores or aggregated PROMIS-29 physical/mental health summary scores as predictors and SF-36 physical/mental component summary scores as outcomes. Data from a subsequent assessment point (n = 259) were used to evaluate the agreement between empirical and predicted SF-36 scores.</p><p><strong>Results: </strong>PROMIS-29 domain scores as well as PROMIS-29 health summary scores showed high predictive value for PCS, PCS_c, and MCS_c (R² ≥ 70%), and moderate predictive value for MCS (R² = 57% and R² = 40%, respectively). After applying the regression coefficients to new data, empirical and predicted SF-36 component summary scores were highly correlated (r > 0.8) for most models. Mean differences between empirical and predicted scores were negligible (|SMD|<0.1).</p><p><strong>Conclusions: </strong>This study provides easy-to-apply algorithms to convert PROMIS-29 data to well-established SF-36 physical and mental component summary scores in a cardiovascular population. Applied to new data, the agreement between empirical and predicted SF-36 scores was high. However, for SF-36 mental component summary scores, considerably better predictions were found under the correlated (MCS_c) than under the original factor model (MCS). Additionally, as a pertinent byproduct, our study confirmed construct validity of the relatively new PROMIS-29 health summary scores in cardiology patients.</p>","PeriodicalId":12980,"journal":{"name":"Health and Quality of Life Outcomes","volume":null,"pages":null},"PeriodicalIF":3.2,"publicationDate":"2024-08-15","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11328444/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141987876","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":"A scoping review of the use of minimally important difference of EQ-5D utility index and EQ-VAS scores in health technology assessment.","authors":"Caroline Shaw, Louise Longworth, Bryan Bennett, Louise McEntee-Richardson, James W Shaw","doi":"10.1186/s12955-024-02272-9","DOIUrl":"10.1186/s12955-024-02272-9","url":null,"abstract":"<p><strong>Objectives: </strong>Estimates of minimally important differences (MID) can assist interpretation of data collected using patient-reported outcomes (PRO), but variability exists in the emphasis placed on MIDs in health technology assessment (HTA) guidelines. This study aimed to identify to what extent information on the MID of a commonly used PRO, the EQ-5D, is required and utilised by selected HTA agencies.</p><p><strong>Methods: </strong>Technology appraisal (TA) documents from HTA agencies in England, France, Germany, and the US between 2019 and 2021 were reviewed to identify documents which discussed MID of EQ-5D data as a clinical outcome assessment (COA) endpoint.</p><p><strong>Results: </strong>Of 151 TAs utilising EQ-5D as a COA endpoint, 58 (38%) discussed MID of EQ-5D data. Discussion of MID was most frequent in Germany, in 75% (n = 12/16) of Gemeinsamer Bundesausschuss (G-BA) and 44% (n = 34/78) of Institut für Qualität und Wirtschaftlichkeit im Gesundheitswesen, (IQWiG) TAs. MID was predominantly applied to the EQ-VAS (n = 50), most frequently using a threshold of > 7 or > 10 points (n = 13). G-BA and IQWiG frequently criticised MID analyses, particularly the sources of MID thresholds for the EQ-VAS, as they were perceived as being unsuitable for assessing the validity of MID.</p><p><strong>Conclusion: </strong>MID of the EQ-5D was not frequently discussed outside of Germany, and this did not appear to negatively impact decision-making of these HTA agencies. While MID thresholds were often applied to EQ-VAS data in German TAs, analyses were frequently rejected in benefit assessments due to concerns with their validity. Companies should pre-specify analyses of continuous data in statistical analysis plans to be considered for treatment benefit assessment in Germany.</p>","PeriodicalId":12980,"journal":{"name":"Health and Quality of Life Outcomes","volume":null,"pages":null},"PeriodicalIF":3.2,"publicationDate":"2024-08-13","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11321174/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141971003","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":"Patient-reported outcomes and daily activity assessed with a digital wearable device in patients with paroxysmal nocturnal hemoglobinuria treated with ravulizumab: REVEAL, a prospective, observational study.","authors":"Elizabeth A Griffiths, Jae S Min, Wei-Nchih Lee, Jeffrey C Yu, Yogesh Patel, Karl-Johan Myren, David Dingli","doi":"10.1186/s12955-024-02279-2","DOIUrl":"10.1186/s12955-024-02279-2","url":null,"abstract":"<p><strong>Background: </strong>Paroxysmal nocturnal hemoglobinuria (PNH) is a rare, chronic blood disorder. Symptoms such as fatigue can have a substantial impact on patients' physical activity levels, sleep, quality of life, and work productivity. Ravulizumab treatment can reduce thrombosis risk, improve survival and quality of life, and reduce fatigue in PNH, but information is limited on how it impacts sleep and physical activity. Here, data on resting heart rate, daily physical activity, and sleep in ravulizumab-treated patients with PNH were passively collected via a digital wearable activity-tracking device and patient-reported outcome (PRO) data were collected via weekly surveys in the same cohort.</p><p><strong>Methods: </strong>REVEAL was a 32-week prospective observational cohort study in individuals with PNH receiving ravulizumab in the USA. A wrist-worn Fitbit™ collected data on resting heart rate, daily step count, and sleep duration from eligible patients. Patients also completed the following electronic weekly surveys: Functional Assessment of Chronic Illness Therapy (FACIT) - Fatigue, Patient-Reported Outcomes Measurement Information System (PROMIS) Global Physical Health, PROMIS Global Mental Health, PROMIS Sleep-Related Impairment and Sleep Disturbance, and Work Productivity and Activity Impairment Questionnaire - Specific Health Problem (WPAI-SHP). Data collected from the activity trackers and surveys were compared against US general population values reported in the literature.</p><p><strong>Results: </strong>Twenty-eight ravulizumab-treated patients were included (median age: 34 years; 54% female). PRO scores were within US general population normative values, including FACIT-Fatigue (40.0), PROMIS Global Physical Health (51.0), Global Mental Health (51.0), Sleep-Related Impairment (50.0), and Sleep Disturbance (49.0). Similarly, mean resting heart rate (67 bpm), daily step count (7476), and sleep duration (7.7 h) were within the range of US general population values. Daily step count was positively correlated with PROMIS Global Physical and Mental Health scores.</p><p><strong>Conclusions: </strong>This was the first study to use digital monitoring technology to collect data on physical activity and sleep in patients with PNH. The findings indicate that ravulizumab treatment enables patients with PNH to achieve activity levels (heart rate, sleep duration, step count) and quality of life that are comparable to those of the US general population. A weak positive correlation was identified between patient-reported physical and mental health and daily physical activity levels.</p>","PeriodicalId":12980,"journal":{"name":"Health and Quality of Life Outcomes","volume":null,"pages":null},"PeriodicalIF":3.2,"publicationDate":"2024-08-09","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11313122/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141912391","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}