Journal of Patient-Reported Outcomes最新文献

{"title":"Identifying social outcomes of importance for childhood cancer survivors: an e-Delphi study.","authors":"Sarah H Milner, R G Feltbower, K L Absolom, A W Glaser","doi":"10.1186/s41687-023-00676-7","DOIUrl":"10.1186/s41687-023-00676-7","url":null,"abstract":"<p><strong>Purpose: </strong>Childhood cancer survivors (CCS) are at risk of deficits in their social outcomes, a key aspect of overall health and quality of life. Social outcomes of import are ill-defined leading to potential gaps in research and service provision. In this study, we undertook a preliminary consensus seeking exercise to support the development of a framework of the important social outcomes for CCS.</p><p><strong>Methods: </strong>A modified e-Delphi study was conducted with four groups: CCS, health professionals, social workers and teachers. Round 1, developed from a literature review, included 34 questions rated for importance on a 7-point Likert scale. Rounds 2 and 3 presented items not achieving consensus, additionally proposed items and in round 3, a ranking question.</p><p><strong>Results: </strong>Survey 1 was completed by 38 participants, 31 (82%) completed survey 2 and 28 (76%) completed survey 3. A total of 36 items were prioritised across 6 domains (education, independence, work, relationships, community, lifestyle), together forming the final list of social outcomes. Of these, 22 items met consensus for importance. Items rated most important were \"having autonomy\" and \"avoiding social isolation\". Quantitative and qualitative results reflected that social outcomes for survivors and general public should be the same.</p><p><strong>Conclusion: </strong>We have generated initial consensus on important social outcomes for CCS, highlighting the need for these to be matched to those of the general population. It suggests strategies are required to ensure autonomy and appropriate support for independence and relationships are provided through long-term aftercare and beyond. Further work is needed to validate and develop these findings into a framework to support appropriate social aftercare for CCS.</p>","PeriodicalId":36660,"journal":{"name":"Journal of Patient-Reported Outcomes","volume":null,"pages":null},"PeriodicalIF":2.7,"publicationDate":"2024-02-05","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10844160/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"139693115","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Health-related quality of life and quality-adjusted progression free survival for carfilzomib and dexamethasone maintenance following salvage autologous stem-cell transplantation in patients with multiple myeloma: a randomized phase 2 trial by the Nordic Myeloma Study Group.","authors":"Lene Kongsgaard Nielsen, Fredrik Schjesvold, Sören Möller, Nina Guldbrandsen, Markus Hansson, Kari Remes, Valdas Peceliunas, Niels Abildgaard, Henrik Gregersen, Madeleine T King","doi":"10.1186/s41687-024-00691-2","DOIUrl":"10.1186/s41687-024-00691-2","url":null,"abstract":"<p><strong>Background: </strong>Decisions regarding maintenance therapy in patients with multiple myeloma should be based on both treatment efficacy and health-related quality of life (HRQL) consequences. In the CARFI trial, patients with first relapse of multiple myeloma underwent salvage autologous stem cell transplantation (salvage ASCT) before randomization to carfilzomib-dexamethasone maintenance therapy (Kd) or observation. The primary clinical endpoint was time to progression, which was extended by 8 months by Kd. The aim of this paper is to present the all HRQL endpoints of the CARFI trial including the HRQL effect of Kd maintenance therapy relative to observation. The primary HRQL endpoint was assessed by EORTC QLQ-C30 Summary score (QLQ-C30-sum) at 8 months follow-up. A key secondary HRQL endpoint was quality-adjusted progression-free-survival (QAPFS).</p><p><strong>Methods: </strong>HRQL was assessed with EORTC QLQ-C30, EORTC QLQ-MY20 and FACT/GOG-Ntx at randomization and every second month during follow-up. HRQL data were analyzed with linear mixed effect models until 8 months follow-up. QAPFS per individual was calculated by multiplying progression-free survival (PFS) by two quality-adjustment metrics, the QLQ-C30-sum and EORTC Quality of Life Utility Measure-Core 10 dimensions (QLU-C10D). The QAPFS per treatment group was estimated with the Kaplan-Meier method. P < 0.05 was used for statistical significance, and a between-group minimal important difference of 10 points was interpreted as clinically relevant for the QLQ-C30-sum.</p><p><strong>Results: </strong>168 patients were randomized. HRQL questionnaire compliance was 93%. For the QLQ-C30-sum, the difference of 4.62 points (95% confidence interval (CI) -8.9: -0.4, p = 0.032) was not clinically relevant. PFS was 19.3 months for the Kd maintenance group and 16.8 months for the observation group; difference = 2.5 months (95% CI 0.5; 4.5). QAPFS based on the QLQ-C30-sum for the Kd maintenance group was 18.0 months (95% CI 16.4; 19.6) and for the observation group 15.0 months (95% CI 13.5; 16.5); difference = 3.0 months (95% CI 0.8-5.3). QAPFS based on the QLU-C10D for the Kd maintenance group was 17.5 months (95% CI 15.9; 19.2) and 14.0 months (95% CI 12.4; 15.5) for the observation group; difference = 3.5 months (95% CI 1.1-5.9).</p><p><strong>Conclusions: </strong>Kd maintenance therapy after salvage ASCT did not adversely affect overall HRQL, but adjustment for HRQL reduced the PFS compared to unadjusted PFS. PFS of maintenance therapy should be quality-adjusted to balance the benefits and HRQL impact.</p>","PeriodicalId":36660,"journal":{"name":"Journal of Patient-Reported Outcomes","volume":null,"pages":null},"PeriodicalIF":2.7,"publicationDate":"2024-02-05","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10844184/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"139693114","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"A qualitative study of daily activities that inform a process-based model of well-being among Singaporean adults.","authors":"Jumana Hashim, Huso Yi, Pin Sym Foong, E Shyong Tai, Robert P Nolan","doi":"10.1186/s41687-024-00690-3","DOIUrl":"10.1186/s41687-024-00690-3","url":null,"abstract":"<p><strong>Background: </strong>Individual appraisals of personal well-being consider a spectrum of constructs including the reflections on the degree to which one's life is ultimately secure, meaningful, or valued in the context of dynamic changes in their bio-psycho-social environments. Standardized questionnaires for well-being evaluate an ideal state of health which is operationally defined by abstract constructs. Patient reports describe well-being as a more dynamic construct that relates to how they adapt to successive changes in their life situations. While response shift theory addresses this dynamic nature, little is known about how personal well-being is pursued as personal aspirations evolve. In this study, we identify regularly practiced goal-directed activities reported to contribute to the pursuit of personal well-being. We then propose a taxonomy of these reported activities to inform a process-based model for well-being.</p><p><strong>Methods: </strong>Purposive sampling was conducted with individuals, 30 to 60 years of age, with diverse ethnicities reflecting the Singaporean population. Individual semi-structured interviews were administered with the primary question: \"What are the things or activities you have done in the last two weeks that made you feel like you had a good day?\" Probes explored the personally salient meaning of each activity. A thematic approach was used for data analysis followed by a framework analysis to categorize the activities into major themes.</p><p><strong>Results: </strong>We interviewed 40 individuals comprised of 60% female participants. Thematic analysis identified eight types of goal-directed activities, which were categorized under three major themes: (i) Self-Affirming Care through individual self-care and maintaining interpersonal relationships, (ii) Achievement-Focused Tasks as indicated by work-related accomplishments and developing a skill or hobby, (iii) Affiliative Growth and Support as reflected through religious practices and community services. Planned physical activity contributed to self-affirming care and achievement-focused tasks. Social affirming roles were associated with both achievement-focused tasks and affiliative growth and support.</p><p><strong>Conclusions: </strong>In the dimensions of goal-directed activities identified, there is close alignment between Self-Affirming Care and attachment theory; Achievement-Focused Tasks and self-determination theory; and Affiliative Growth and Support and social mattering. These findings can contribute to a comprehensive process-based model of well-being which is more closely aligned to patient-report appraisals of personal well-being.</p>","PeriodicalId":36660,"journal":{"name":"Journal of Patient-Reported Outcomes","volume":null,"pages":null},"PeriodicalIF":2.7,"publicationDate":"2024-01-31","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10831003/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"139643084","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Validation of the Patient-Reported Outcomes Measurement Information System (PROMIS^®) physical function questionnaire in late-onset Pompe disease using PROPEL phase 3 data.","authors":"Priya S Kishnani, Simon Shohet, Syed Raza, Noemi Hummel, Jeffrey P Castelli, Sheela Sitaraman Das, Heng Jiang, Agnieszka Kopiec, Ian Keyzor, Andreas Hahn","doi":"10.1186/s41687-024-00686-z","DOIUrl":"10.1186/s41687-024-00686-z","url":null,"abstract":"<p><strong>Background: </strong>The construct validity and interpretation of the Patient-Reported Outcome Measurement Information System (PROMIS^®) Physical Function short form 20a (PF20a) questionnaire were evaluated for patients with late-onset Pompe disease (LOPD), a rare, autosomal recessive, progressive neuromuscular disorder treatable by enzyme replacement therapy (ERT).</p><p><strong>Methods: </strong>In the phase 3 PROPEL study, adults with LOPD underwent testing of physical functioning and had PRO measurements at baseline and at weeks 12, 26, 38, and 52 while receiving experimental or standard-of-care ERT. All patients were pooled for analyses, without comparisons between treatment groups. Associations and correlations between PROMIS PF20a scores and the 6-minute walk distance (6MWD), % predicted forced vital capacity (FVC), manual muscle test (MMT) of the lower extremities, Gait, Stairs, Gowers' maneuver, Chair (GSGC) score, and Rasch-built Pompe-specific Activity (R-PAct) scale were evaluated by calculating regression coefficients in linear regression models and Pearson correlation coefficients (R); patients' age, sex, race, ERT prior to study, body mass index, and study treatment were included as covariables. The minimal clinically important difference (MCID) of PROMIS PF20a was determined using distribution- and anchor-based methods.</p><p><strong>Results: </strong>123 patients received at least 1 dose of ERT. In multivariable analyses, PROMIS PF20a scores had strong correlations with R-PAct scores (R = 0.83 at baseline and R = 0.67 when evaluating changes between baseline and 52 weeks) and moderate correlations with the 6MWD (R = 0.57 at baseline and R = 0.48 when evaluating changes between baseline and 52 weeks). Moderate correlations were also observed between PROMIS PF20a and MMT (R = 0.54), GSGC (R=-0.51), and FVC (R = 0.48) at baseline. In multivariable linear regression models, associations were significant between PROMIS PF20a and 6MWD (P = 0.0006), MMT (P = 0.0034), GSGC (P = 0.0278), and R-PAct (P < 0.0001) at baseline, between PROMIS PF20a and 6MWD (P < 0.0001), FVC (P = 0.0490), and R-PAct (P < 0.0001) when combining all measurements, and between PF20a and 6MWD (P = 0.0016) and R-PAct (P = 0.0001) when evaluating changes in scores between baseline and 52 weeks. The anchor-based and distribution-based MCID for a clinically important improvement for PROMIS PF20a were 2.4 and 4.2, respectively.</p><p><strong>Conclusions: </strong>PROMIS PF20a has validity as an instrument both to measure and to longitudinally follow physical function in patients with LOPD.</p><p><strong>Trial registration: </strong>ClinicalTrials.gov, NCT03729362. Registered 2 November 2018, https://www.</p><p><strong>Clinicaltrials: </strong>gov/search?term=NCT03729362 .</p>","PeriodicalId":36660,"journal":{"name":"Journal of Patient-Reported Outcomes","volume":null,"pages":null},"PeriodicalIF":2.7,"publicationDate":"2024-01-31","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10830974/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"139643085","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Enhancing quality of life and medication adherence for people living with HIV: the impact of an information system.","authors":"Maria Panayi, Georgios K Charalambous, Eleni Jelastopulu","doi":"10.1186/s41687-023-00680-x","DOIUrl":"10.1186/s41687-023-00680-x","url":null,"abstract":"<p><strong>Background: </strong>The widespread availability of antiretroviral therapy has led to improvements in life expectancy and thus an increase in the number of people living with HIV/AIDS (PLWHA) worldwide. However, a similar increase in the number of newly-diagnosed patients in Cyprus suggests the need for solutions designed to improve monitoring, planning, and patient communication. In this study, we aimed to determine whether the use of an information system to manage PLWHA might contribute to improved quality of life and critical adherence to prescribed drug regimens and ongoing medical care.</p><p><strong>Methods: </strong>A randomized controlled trial study was conducted in Cyprus based on information that we collected using the highly valid and reliable Greek translation of the World Health Organization (WHO) Quality of Life (QOL) HIV-BREF questionnaire to assess sociodemographic variables and patient compliance. We distributed 200 questionnaires before implementing a Health Medical Care (HMC) information system at our clinic. Six months after implementing this system, 68 of the completed questionnaires were selected, including two groups of 34 participants who had been assigned at random to the intervention or the control group. Participants included PLWHA aged ≥ 18 years who had been receiving antiretroviral therapy for more than 12 months between July 15, 2020, and July 15, 2022.</p><p><strong>Results: </strong>The changes in baseline to six-month scores reported for the intervention group were significantly higher than in the control group in all six subscales assessed with the WHOQOL-HIV-BREF questionnaire, as well as in the assessment of compliance. Furthermore, compliance with treatment was associated with higher scores in the questionnaire subscales, including physical health, psychological health, degree of autonomy, social relationships, life circumstances, and spirituality/religious/personal beliefs. We also identified specific demographic factors and behaviors that were associated with better compliance with scheduled medical care and the prescribed drug regimen. Specifically, men exhibited better compliance than women and younger PLWHA exhibited better compliance than the elderly as did individuals who reported a higher level of educational attainment. Additionally, individuals who did not use addictive substances, consumed less alcohol, and were managed using the monitoring information system all exhibited better compliance compared to those in the control group.</p><p><strong>Conclusion: </strong>The results of this study suggest that management of PLWHA via the use of an information system can contribute to improved QOL and drug compliance.</p>","PeriodicalId":36660,"journal":{"name":"Journal of Patient-Reported Outcomes","volume":null,"pages":null},"PeriodicalIF":2.7,"publicationDate":"2024-01-23","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10805742/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"139521984","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Differential associations of sex and age with changes in HRQoL during outpatient cardiac rehabilitation.","authors":"Lorenza L S Lanini, Sebastian Euler, Claudia Zuccarella-Hackl, Rubén Fuentes Artiles, David Niederseer, Bianca Auschra, Roland von Känel, Lena Jellestad","doi":"10.1186/s41687-024-00688-x","DOIUrl":"10.1186/s41687-024-00688-x","url":null,"abstract":"<p><strong>Introduction: </strong>Cardiovascular diseases (CVD) represent the world's leading cause of death. Health-related quality of life (HRQoL) is a widely applied concept of patients' perceived health and is directly linked to CVD morbidity, mortality, and re-hospitalization rates. Cardiac rehabilitation (CR) improves both cardiovascular outcomes and HRQoL. Regrettably, CR is still underutilized, especially in subgroups like women and elderly patients. The aim of our study was to investigate the predictive potential of sex and age on change of HRQoL throughout outpatient CR.</p><p><strong>Methods: </strong>497 patients of outpatient CR were retrospectively assessed from August 2015 to September 2019 at the University Hospital Zurich. A final sample of 153 individuals with full HRQoL data both at CR entry and discharge was analyzed. HRQoL was measured using the 36-Item Short Form Survey (SF-36) with its physical (PCS) and mental (MCS) component scale. In two-factorial analyses of variance, we analyzed sex- and age-specific changes in HRQoL scores throughout CR, adjusting for psychosocial and clinical characteristics. Age was grouped into participants over and under the age of 65.</p><p><strong>Results: </strong>In both sexes, mean scores of physical HRQoL improved significantly during CR (p <.001), while mean scores of mental HRQoL improved significantly in men only (p =.003). Women under the age of 65 had significantly greater physical HRQoL improvements throughout CR, compared with men under 65 (p =.043) and women over 65 years of age (p =.014). Sex and age did not predict changes in mental HRQoL throughout CR.</p><p><strong>Conclusions: </strong>Younger women in particular benefit from CR with regard to their physical HRQoL. Among older participants, women report equal improvements of physical HRQoL than men. Our results indicate that sex- and age-related aspects of HRQoL outcomes should be considered in CR.</p>","PeriodicalId":36660,"journal":{"name":"Journal of Patient-Reported Outcomes","volume":null,"pages":null},"PeriodicalIF":2.7,"publicationDate":"2024-01-23","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10805744/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"139521981","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Mixed-methods research to support the use of new lymphoma-specific patient-reported symptom measures derived from the EORTC item library.","authors":"Jessica T Markowitz, Flora Mazerolle, Teya Lovell, Lisa M Hess, Paolo B Abada, Antoine Regnault, Nalin Payakachat","doi":"10.1186/s41687-024-00683-2","DOIUrl":"10.1186/s41687-024-00683-2","url":null,"abstract":"<p><strong>Background: </strong>No specific measures exist to assess patient-reported symptoms experienced by individuals with chronic lymphocytic leukemia (CLL)/small lymphocytic lymphoma (SLL) or mantle cell lymphoma (MCL). This study was conducted to elicit patient-reported CLL/SLL- and MCL-related symptoms and their impact on patients' lives. The study qualitatively and quantitatively evaluated sets of conceptually-selected EORTC Item Library items for assessing CLL/SLL- and MCL-related symptoms.</p><p><strong>Methods: </strong>The qualitative component of the research included a literature review, clinician consultations, and patient interviews. Concepts important to patients were identified and coded; cognitive debriefing of the selected library items was completed with patients. CLL/SLL and MCL-related symptoms and impacts were organized in a structured conceptual model, which was mapped to item sets from the Item Library. The quantitative component comprised exploratory macro-level Rasch measurement theory (RMT) analysis conducted to provide supportive quantitative insight on the item sets.</p><p><strong>Results: </strong>41 patients (21-MCL; 20-CLL/SLL) and 5 clinicians participated in the qualitative study; 57 unique patients (30-MCL; 27-CLL/SLL) completed the EORTC items. The conceptual models generated from the qualitative work included symptoms and functional impacts of CLL/SLL and MCL. Symptom domains included swollen lymph nodes, B symptoms, abdominal issues, pain, fatigue, subjective cognitive impairment, anemia-related symptoms, bleeding, infection, and other issues (appetite loss, temperature fluctuation, rash, weight gain, sleep problems, cough). Impacts included physical function, role function, and other functions (psychological, social). Cognitive debriefing demonstrated that the separate item sets for CLL/SLL and MCL-related symptoms were well understood and aligned with patients' experiences. All selected items were included in the conceptual models. The exploratory RMT analysis showed that the item sets provided adequate coverage of the continuum of CLL/SLL- and MCL-related symptom severity.</p><p><strong>Conclusions: </strong>This study gathered qualitative and early quantitative evidence supporting use of the EORTC Item Library to assess CLL/SLL- and MCL-related symptoms and impacts. These items are promising candidates for measurement of patient-reported disease symptoms in these populations. A larger sample size will be essential to establish the psychometric properties necessary to support use in clinical trials. Patients who suffer from rare cancers of the blood, bone marrow, and lymph nodes can experience chronic and debilitating symptoms. At present, however, there are no dedicated instruments for assessing the patient's experience of symptoms of conditions like chronic lymphocytic leukemia (CLL)/small lymphocytic lymphoma (SLL) or mantle cell lymphoma (MCL), or for assessing their impact on patients' lives. This ","PeriodicalId":36660,"journal":{"name":"Journal of Patient-Reported Outcomes","volume":null,"pages":null},"PeriodicalIF":2.7,"publicationDate":"2024-01-22","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10803695/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"139511239","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Meaningful score changes for SF-36v2, FACIT-fatigue, and RASIQ in rheumatoid arthritis.","authors":"Regina Rendas-Baum, Xiaochen Lin, Mark Kosinski, Jakob Bue Bjorner, Marguerite G Bracher, Wen-Hung Chen","doi":"10.1186/s41687-024-00685-0","DOIUrl":"10.1186/s41687-024-00685-0","url":null,"abstract":"<p><strong>Background: </strong>Interpretation thresholds for patient-reported outcome (PRO) scores are of crucial importance, particularly when interpreting treatment benefit. This study was designed to determine the within-patient meaningful improvement (WPMI) thresholds for the Short-Form 36 Health Survey version 2 (SF-36v2), the Functional Assessment of Chronic Illness Therapy-Fatigue (FACIT-Fatigue), and the novel Rheumatoid Arthritis Symptoms and Impact Questionnaire (RASIQ) among patients with rheumatoid arthritis (RA).</p><p><strong>Methods: </strong>In this post-hoc analysis, anchor-based and supportive distribution-based methods were used to derive WPMI based on blinded data from all treatment arms in two Phase 2 RA trials with otilimab. Patient's Global Assessment of Disease Activity (PtGA) was the general anchor for all SF-36v2 scales. SF-36 Patient's Global Impression of Status (PGIS), PtGA, and VT03 (an SF-36v2 item) were used as anchors for FACIT-Fatigue. SF-36 PGIS, PtGA, and Patient's Assessment of Arthritis Pain (PAIN) were anchors for RASIQ. Mean change was calculated for the anchor category associated with minimal meaningful improvement from baseline to Week 24 for SF-36v2 and FACIT-Fatigue, and to Week 12 for RASIQ. Sensitivity and specificity were used to evaluate the accuracy of estimated WPMI values.</p><p><strong>Results: </strong>For the SF-36v2 physical functioning, role physical, bodily pain, general health, vitality, social functioning, role emotional, and mental health domains, anchor-based estimates of WPMI based on 0-100 scores were 24.5, 24.5, 25.4, 13.6, 21.5, 20.5, 16.9, and 14.3, respectively. Anchor-based WPMI estimates were 9.7 for the Physical Component Summary score and 7.6 for the Mental Component Summary score (using norm-based T-score metric). For FACIT-Fatigue (range 0-52), WPMI estimates ranged from 9.7 to 11.3 points. For RASIQ (range 0-100), anchor-based WPMI was determined as a change between -32.7 and -21.7 points for the Joint Pain scale, -26.7 to -23.7 for the Joint Stiffness scale, and -21.1 to -17.4 for the Impact scale.</p><p><strong>Conclusions: </strong>This study derived WPMI thresholds for SF-36v2, FACIT-Fatigue, and RASIQ among patients with RA, using multiple anchors. Derivation of WPMI thresholds for these PRO instruments will enable their broader use in evaluating and interpreting treatment benefit in future RA studies.</p>","PeriodicalId":36660,"journal":{"name":"Journal of Patient-Reported Outcomes","volume":null,"pages":null},"PeriodicalIF":2.7,"publicationDate":"2024-01-22","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10806967/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"139511131","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"The subjective and objective quality of life score (SOQOLTM) for the quantification of general health status: a preliminary study with initial normative population values.","authors":"Ralph J Mobbs, R Dineth Fonseka, Pragadesh Natarajan, Lianne Koinis, Monish Maharaj","doi":"10.1186/s41687-024-00684-1","DOIUrl":"10.1186/s41687-024-00684-1","url":null,"abstract":"<p><strong>Background: </strong>Established health-related quality of life scores do not consider both subjective and objective indices of health. We propose the subjective and objective quality of life score (SOQOL) for the comprehensive assessment of health-related quality of life and aim to provide normative population data. The SOQOL is compatible with smartphone applications, allowing widespread use on a global scale.</p><p><strong>Methods: </strong>Normative SOQOL population data was sourced from pre-existing datasets on the EQ-5D-5L, daily step count, and walking speed. Normative values were calculated using weighted grand means. We trialled the SOQOL in a group of five patients presenting to a spinal neurosurgery clinic.</p><p><strong>Results: </strong>SOQOL scores decreased with age, and women had lower scores in every age group. In our case series, the spine patients with the biggest SOQOL deficit compared to age- and sex-matched population averages were found to be surgical while the rest were non-surgical.</p><p><strong>Conclusions: </strong>The SOQOL shows promise as a simple and effective scoring tool that is compatible with smartphones, potentially useful for screening in primary and specialized care settings, and for assessment following healthcare interventions. This study, however, is preliminary, and the findings are primarily suggestive. They underline the necessity for future, more comprehensive studies to validate and expand upon these initial observations. The conclusion of both this abstract and the full paper will clearly state these limitations and the preliminary nature of the study.</p>","PeriodicalId":36660,"journal":{"name":"Journal of Patient-Reported Outcomes","volume":null,"pages":null},"PeriodicalIF":2.7,"publicationDate":"2024-01-18","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10796876/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"139485688","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Valuing selected WAItE health states using the Time Trade-Off methodology: findings from an online interviewer-assisted remote survey.","authors":"Tomos Robinson, Sarah Hill, Giovany Orozco-Leal, Ashleigh Kernohan, William King, Yemi Oluboyede","doi":"10.1186/s41687-023-00674-9","DOIUrl":"10.1186/s41687-023-00674-9","url":null,"abstract":"<p><strong>Purpose: </strong>The Weight-Specific Adolescent Instrument for Economic Evaluation (WAItE) is a physical weight-specific patient reported outcome measure for use in adolescence. The purpose of this study was to use the Time Trade-Off (TTO) methodology, administered using an online interviewer-assisted remote survey, to obtain utility values for several health states from the WAItE descriptive system from a sample of the UK adult general population.</p><p><strong>Methods: </strong>The adult sample was gathered using a market research company and a sample of local residents. All participants completed the same interviewer-assisted remote survey, which included rating WAItE states of varying impairment using the TTO.</p><p><strong>Results: </strong>42 adults completed the survey. Utility values were gathered for four health states, ranging from low impairment to the most severe health from the WAItE descriptive system (the Pits state). Consistent orderings of the WAItE health states were observed; the health state with the lowest level of impairment was valued highest and the Pits state was valued lowest. Several respondents (n = 7, 17%) considered the Pits state to be worse than death; however, the mean value of this health state was 0.23.</p><p><strong>Conclusions: </strong>The utility value of the Pits state relative to death generated from this study will be used to anchor latent values for WAItE health states generated from a Discrete Choice Experiment onto the 0 = death, 1 = full health Quality Adjusted Life Year (QALY) scale as part of a valuation study for the WAItE in the UK population. This study also provides further evidence that interviewer-assisted digital studies are feasible for collecting TTO data.</p>","PeriodicalId":36660,"journal":{"name":"Journal of Patient-Reported Outcomes","volume":null,"pages":null},"PeriodicalIF":2.7,"publicationDate":"2024-01-12","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10786771/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"139425639","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}