Journal of Patient-Reported Outcomes最新文献

{"title":"Discrepancies in perceptions of well-being: comparing parental and pediatric PROMIS-patient-reported outcomes in Crohn's disease.","authors":"Sara Azevedo, Maria Miguel Oliveira, Paulo Jorge Nogueira, Ana Isabel Lopes","doi":"10.1186/s41687-025-00870-9","DOIUrl":"10.1186/s41687-025-00870-9","url":null,"abstract":"<p><strong>Background: </strong>This study aims to evaluate and compare the perspectives of pediatric Crohn's disease (CD) patients and their parents/caregivers concerning global physical, emotional, and social health as well as health-related quality of life (HRQQL), using both the Patient-Reported Outcomes Measurement Information System (PROMIS) and the IMPACT III questionnaire.</p><p><strong>Methods: </strong>In a cross-sectional study, 31 dyads of pediatric CD patients (aged 8-17 years) and their parents/caregivers were recruited from an outpatient Pediatric Gastroenterology Center. Participants completed PROMIS (Global Health, Depressive Symptoms, Anxiety, Meaning and Purpose Pain Interference Life Satisfaction, Peer Relationships, Physical Activity and Fatigue) and IMPACT III measures. Comparative analyses using t-tests and multivariate analyses assessed the impact of demographic factors on score differences. Cohen's Kappa analysis evaluated the alignment between parent and child perceptions of disease status.</p><p><strong>Results: </strong>The sample comprised 58% females with a mean age of 15.2 (± 2) years and a mean disease duration of 2.7 (± 2.7) years. Most patients were in disease remission (83.9%) and perceived their disease as better or unchanged in the past 6 months. Concerning PROMIS scores, parents reported significantly lower global health scores (p < 0.001) and higher meaning and purpose scores (p = 0.029) compared to their children. Parental education and professional status significantly influenced PROMIS score differences. Specifically, mothers with specialized professions showed smaller differences in PROMIS depression and pain interference, although greater differences in PROMIS meaning and purpose, as compared to their respective children's scores. Fathers with specialized professions demonstrated greater differences in PROMIS anxiety scores but smaller differences in PROMIS life satisfaction scores. A significant misalignment between parent and child subjective perceptions of disease status was observed (p = 0.004), suggesting that parents may overestimate symptom severity or underestimate improvements compared to their children's experiences.</p><p><strong>Conclusion: </strong>This study highlights the importance of integrating patient and parental perspectives in the clinical management of pediatric CD. The observed discrepancies in disease-related perceptions, influenced by parental educational and professional background, underscore the need for comprehensive assessments to ensure accurate, patient-centered care. For broader generalization, further research should explore these dynamics in newly diagnosed and hospitalized patients.</p>","PeriodicalId":36660,"journal":{"name":"Journal of Patient-Reported Outcomes","volume":"9 1","pages":"37"},"PeriodicalIF":2.4,"publicationDate":"2025-03-28","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11953492/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143744283","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":"Development and psychometric evaluation of a patient-reported symptom index for patients with non-muscle invasive bladder cancer: the NMIBC-SI.","authors":"Claudia Rutherford, Margaret-Ann Tait, Daniel S J Costa, Madeleine T King, David P Smith, Shomik Sengupta, Joseph Ischia, Andrew Mitterdorfer, Dickon Hayne, Roger Watson, Paul Anderson, Mark Frydenberg, Peter Gilling, Nicholas Buchan, Euan Green, Noel Clarke, Stephen A Boorjian, Badrinath Konety, Jeffrey M Holzbeierlein, Peter C Black, Venu Chalasani, Jörg Henseler, Manish I Patel","doi":"10.1186/s41687-025-00864-7","DOIUrl":"10.1186/s41687-025-00864-7","url":null,"abstract":"<p><strong>Background and objective: </strong>Non-muscle invasive bladder cancer (NMIBC) is a chronic condition requiring frequent follow-up with endoscopic examinations, tumour resections and intravesical treatments. In this clinical context, patient-reported outcomes (PROs) have enormous potential to inform treatment assessment and recommendations for NMIBC. We aimed to develop and evaluate a patient-reported NMIBC Symptom Index (NMIBC-SI) to facilitate clinical research and enhance care.</p><p><strong>Methods: </strong>NMIBC-SI items were developed based on existing literature and qualitative interviews with patients and clinicians, and evaluated in two field tests: item reduction, using NMIBC-SI data from 220 patients on active treatment from nine Australian centres; reliability and validity evaluation of item-reduced version using NMIBC-SI data from 232 patients from five countries.</p><p><strong>Results: </strong>NMIBC-SI assesses disease and treatment-related symptom burden and two treatment-specific side-effects (cystoscopy, intravesical BCG/Chemotherapy). Composite analysis supported a single composite model including core symptom and cystoscopy index items (Intravesical index items were not tested due to small sample). Test-retest reliability was strong (range 0.894-0.91). As expected, the NMIBC-SI was able to discriminate between no treatment and any treatment groups, and no treatment and chemo/BCG groups, providing evidence towards validity.</p><p><strong>Conclusions and clinical implications: </strong>NMIBC-SI assesses patients' self-reported symptom burden and can be used to evaluate NMIBC treatments from the perspective of patients. The NMIBC-SI is acceptable to patients and has evidence for reliability and validity. Future validation work with patients with greater symptom burden is warranted.</p>","PeriodicalId":36660,"journal":{"name":"Journal of Patient-Reported Outcomes","volume":"9 1","pages":"36"},"PeriodicalIF":2.4,"publicationDate":"2025-03-27","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11950540/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143721709","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":"Patients' acceptability of a patient-reported outcome measure in cardiac rehabilitation (the PRO-Heart-DK)-a mixed methods study using the Theoretical Framework of Acceptability.","authors":"Emma Dedic, Heidi Sønderby Vistisen, Ann-Dorthe Zwisler, Bente Faurby Pedersen, Karin Lundsby Kappel, Helle Kanstrup, Rikke Elmose Mols, Cecilie Lindström Egholm","doi":"10.1186/s41687-024-00831-8","DOIUrl":"10.1186/s41687-024-00831-8","url":null,"abstract":"<p><strong>Background: </strong>The integration of Patient Reported Outcome Measures (PROM) in cardiac rehabilitation practice has potential to enhance patient involvement and management. User acceptance is crucial for successful implementation of healthcare interventions, but limited literature addresses PROM acceptability among cardiovascular patients. This study explored the acceptability of a new national PROM in cardiac rehabilitation clinical practice for patients with ischemic heart disease (IHD) in Denmark.</p><p><strong>Methods: </strong>Patients who responded to the PROM were invited to complete two brief surveys evaluating perceived relevance, usefulness, and satisfaction. A purposefully selected subsample participated in semi structured interviews to gather in-depth experiences. A parallel convergent mixed-methods design was used with the Theoretical Framework of Acceptability applied to structure and interpret findings.</p><p><strong>Results: </strong>105 and 119 patients respectively responded to the two evaluation surveys (response-rates 56.5% and 53.4% respectively) and 25 patients were interviewed. The study showed a strong willingness to engage with PROMs, indicating a high overall acceptability. Most patients perceived the PROM helpful for preparation and enhancing communication during consultations. A minority of patients reported emotional reactions and experienced issues with questionnaire comprehensiveness, structure, and relevance.</p><p><strong>Conclusion: </strong>The findings indicate that most IHD patients find PROM relevant and useful in cardiac rehabilitation. To enhance acceptability and, hence, future implementation, improvements are needed in clinical settings by providing adequate patient information, effectively using PRO results, and addressing patients' emotional reactions. Additionally, PROM development should focus on ensuring the questionnaire's relevance, comprehensiveness, and structure.</p>","PeriodicalId":36660,"journal":{"name":"Journal of Patient-Reported Outcomes","volume":"9 1","pages":"35"},"PeriodicalIF":2.4,"publicationDate":"2025-03-25","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11937467/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143711558","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 new generation of patient-reported outcome measures with large language models.","authors":"Jan Henrik Terheyden, Maren Pielka, Tobias Schneider, Frank G Holz, Rafet Sifa","doi":"10.1186/s41687-025-00867-4","DOIUrl":"10.1186/s41687-025-00867-4","url":null,"abstract":"<p><strong>Background: </strong>Patient-reported outcome measures (PROMs) are cornerstones of patient-centered clinical medicine and reflect patients' abilities, difficulties, perceptions and behaviors. The highly structured questionnaire format of PROMs currently limits their real-world validity and acceptability to patients, which becomes increasingly relevant with the high clinical interest in PROM data. In this short commentary, we aim to demonstrate the potential use of large language models (LLMs) in the context of PROM data collection and interpretation.</p><p><strong>Main body: </strong>The popularization of LLMs enables the development of a new generation of PROMs generated and administered through digital technology that interact with patients and score their responses in real time based on artificial intelligence. LLM-PROMs will need to be developed with multi-stakeholder input and careful validation against established PROMs. LLM-PROMs could complement traditional PROMs particularly in real-world clinical applications.</p><p><strong>Conclusion: </strong>LLM-PROMs could allow quantifying patient-relevant dimensions based on less structured contents and foster the use of patient-reported data in digital, clinical applications of PROMs.</p>","PeriodicalId":36660,"journal":{"name":"Journal of Patient-Reported Outcomes","volume":"9 1","pages":"34"},"PeriodicalIF":2.4,"publicationDate":"2025-03-24","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11933620/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143701730","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":"Interpretation of clinically meaningful change in cancer palliative care patients' quality of life: minimally important difference for EORTC QLQ-C15-PAL.","authors":"Kikuko Miyazaki, Yoshimi Suzukamo, Masayuki Ikenaga, Shozo Ohsumi, Mari Saito, Eriko Satomi, Kojiro Shimozuma, Takeo Nakayama","doi":"10.1186/s41687-025-00858-5","DOIUrl":"10.1186/s41687-025-00858-5","url":null,"abstract":"<p><strong>Background: </strong>Palliative care for cancer helps improve and maintain patients' quality of life (QOL). Clinically meaningful changes in QOL measures are helpful when considering how a patient would want to spend the final days of their life. This study aimed to estimate the minimally important differences (MIDs) for within-person change for the European Organisation for Research and Treatment of Cancer QOL Questionnaire Core 15 Palliative Care (EORTC QLQ-C15-PAL) domains in advanced cancer patients in palliative care.</p><p><strong>Method: </strong>Participants in this multicenter observational study comprised patients with advanced cancer receiving palliative care in the last year before death. The EORTC QLQ-C15-PAL was administered at two-week intervals. During the second assessment, patients also completed the Global Rating of Change (GRC) scale to collect their subjective assessments of changes in their condition since the first assessment. MID for QOL score with a correlation of 0.3 or more with GRC score changes were estimated using anchor- and distribution-based methods.</p><p><strong>Results: </strong>Among the 257 screened patients at 13 facilities, we analyzed 181 (92 male; mean age: 67). The mean survival time was 131 days. Notably, the number of patients who responded \"no change\" for the GRC items was large (63-128). Anchor-based MIDs differed depending on the change direction (improvement vs. deterioration). The MIDs for meaningful within-person change may be used in clinical practice.</p><p><strong>Conclusion: </strong>We estimated the MIDs in EORTC QLQ-C15-PAL in patients with advanced cancer with a life expectancy of less than one year, both anchor- and distribution-based.</p>","PeriodicalId":36660,"journal":{"name":"Journal of Patient-Reported Outcomes","volume":"9 1","pages":"33"},"PeriodicalIF":2.4,"publicationDate":"2025-03-19","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11923346/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143664049","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":"Psychometric validation of the Chronic Ocular Pain Questionnaire (COP-Q).","authors":"Amy Findley, Brigitte J Sloesen, Nicola Hodson, Agkreta Leventi, Ben Pascoe, Rob Arbuckle, Paul O'Brien, Christel Naujoks, Michela Montecchi-Palmer, Diana Plaza, Paul M Karpecki, Pedram Hamrah","doi":"10.1186/s41687-025-00862-9","DOIUrl":"10.1186/s41687-025-00862-9","url":null,"abstract":"<p><strong>Background: </strong>The Chronic Ocular Pain Questionnaire (COP-Q) is a newly developed patient-reported outcome (PRO) measure intended to assess symptoms and impacts associated with Chronic Ocular Surface Pain (COSP). This study assessed the psychometric properties of the COP-Q to determine the adequacy of the COP-Q as a 'fit-for-purpose' instrument to derive trial endpoints for future clinical studies in COSP.</p><p><strong>Methods: </strong>Patients with COSP completed the COP-Q daily for four weeks on an electronic, touch-screen, tablet device as part of a longitudinal, observational study in the United States (N = 124). Analyses were conducted to assess item properties, dimensionality and scoring, reliability and validity, and interpretation of scores. In addition, 4-hour and 24-hour recall period versions of the COP-Q Symptom Module were compared.</p><p><strong>Results: </strong>Item responses were distributed across the full response scale for most COP-Q items. Inter-item correlations did not identify any redundant items (r > 0.90) and all items correlated at > 0.40 in their respective module. Confirmatory factor analysis (CFA) provided acceptable support for the unidimensional structure of the multi-item scales in the COP-Q and calculation of a total score for each module. However, CFA and Rasch analysis outlined potential redundant items for the COP-Q Visual Tasking Module (VTM), which were removed, resulting in a six-item VTM. The multi-item COP-Q modules had excellent internal consistency (α range = 0.91-0.96) and suggested fair to excellent test-retest reliability (ICC/Kappa range = 0.651-0.940) for all COP-Q modules. Construct validity for all COP-Q modules was supported by a logical pattern of correlations with concurrent measures and evidence of ability to distinguish between known-groups, with statistically significant differences between COSP severity groups. Paired t-tests, coefficient of determination (CoD) and concordance correlation coefficients (CCC) showed statistically significant differences between the two recall period versions of the Symptom Module, although the magnitude of the difference was small, and each version shares a high level of reproducibility in scores.</p><p><strong>Conclusions: </strong>Findings provide evidence that the COP-Q is a valid and reliable measure of patient-reported COSP symptoms and impacts for use in future clinical trials in COSP. Both 4-hour and 24-hour Symptom Module recall period versions are likely to yield consistent results and are equally robust.</p>","PeriodicalId":36660,"journal":{"name":"Journal of Patient-Reported Outcomes","volume":"9 1","pages":"32"},"PeriodicalIF":2.4,"publicationDate":"2025-03-12","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11903982/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143617459","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":"Establishing content validity of the Dimensional Anhedonia Rating Scale.","authors":"Stephanie Bean, Rahul Dhanda, Christina A Graham, Deborah Hoffman, Mariam Rodriguez-Lee, Adrian Ionescu, Stella Karantzoulis, Sidney H Kennedy, Sakina J Rizvi","doi":"10.1186/s41687-025-00860-x","DOIUrl":"10.1186/s41687-025-00860-x","url":null,"abstract":"<p><strong>Background: </strong>This study was designed to evaluate content validity of the Dimensional Anhedonia Rating Scale (DARS), a patient-reported outcome measure, in adults with anhedonia in the context of major depressive disorder (MDD). To accomplish this, a conceptual model including the symptoms and impacts of anhedonia in the context of MDD was developed and refined through a targeted literature review, clinician interviews (N = 6), and participant interviews (N = 20).</p><p><strong>Results: </strong>Using the final conceptual model, an item mapping exercise was conducted for the DARS, demonstrating that it provided suitable concept coverage in this population. Cognitive debriefing of the DARS with participants demonstrated that it was generally well understood and clear.</p><p><strong>Conclusions: </strong>Overall, the study established that the DARS demonstrates content validity in adults with anhedonia in the context of MDD. Other measurement properties of the DARS will be evaluated in planned psychometric analyses.</p>","PeriodicalId":36660,"journal":{"name":"Journal of Patient-Reported Outcomes","volume":"9 1","pages":"31"},"PeriodicalIF":2.4,"publicationDate":"2025-03-11","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11896914/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143606703","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":"Psychometric evaluation of the unmet needs instrument for carers of people with dementia (UNI-C).","authors":"Elise Mansfield, Emilie Cameron, Matthew Clapham, Alix Hall, Allison Boyes","doi":"10.1186/s41687-025-00856-7","DOIUrl":"10.1186/s41687-025-00856-7","url":null,"abstract":"<p><strong>Background: </strong>Carers play an important role in providing practical and emotional support to people with dementia. There is a need to ensure carers are supported in this role to maximise their ability to provide care while maintaining their own wellbeing. This study aimed to develop a psychometrically rigorous self-report instrument to assess the unmet needs of carers of people with dementia.</p><p><strong>Methods: </strong>The Unmet Needs Instrument for Carers of People with Dementia (UNI-C) was developed using a multi-methods approach including a comprehensive literature review, in-depth interviews and focus groups, expert opinion, cognitive interviews and pilot testing. A cross-sectional survey of 169 carers was used to examine the psychometric characteristics of the instrument. Specifically, internal reliability, and structural and convergent validity were assessed and the number of items reduced.</p><p><strong>Results: </strong>The original 80 item instrument was reduced to 46 items across four domains based on prevalence, item-total and pairwise correlations, factor loadings and clinical relevance. The resulting instrument, named the UNI-C46, showed acceptable evidence of structural and convergent validity, and good internal reliability (α = 0.94) and acceptability. The four domains measure 'Your own wellbeing' (16 items), 'Finding information' (12 items), 'Managing practical needs' (10 items), and 'Managing dementia symptoms' (8 items).</p><p><strong>Conclusions: </strong>This study provides the first demonstration of reliability and validity of the UNI-C46. Further testing of these properties with a larger and more representative sample of carers is recommended.</p>","PeriodicalId":36660,"journal":{"name":"Journal of Patient-Reported Outcomes","volume":"9 1","pages":"28"},"PeriodicalIF":2.4,"publicationDate":"2025-03-11","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11896954/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143606705","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":"Depression drives perceived quality of life following minor stroke.","authors":"Martina Gjyzari, Elisabeth Breese Marsh","doi":"10.1186/s41687-025-00861-w","DOIUrl":"10.1186/s41687-025-00861-w","url":null,"abstract":"<p><strong>Background: </strong>Stroke outcomes are typically assessed using objective scales focused on severity and functional ability that may overlook subtle cognitive changes and fail to account for patients' perceptions of recovery and quality of life. This study aimed to compare patient-reported outcomes (PROs) to objective recovery metrics in patients with minor stroke and identify factors associated with perceived recovery and quality of life.</p><p><strong>Methodology: </strong>Data from 134 patients with minor stroke were prospectively collected at 1-, 6-, and 12-months post-infarct. Objective assessments measured stroke severity, functional outcomes, activities of daily living, and global cognitive function. PROs included assessments of function, depression, fatigue, symptomatic improvement, and quality of life. Regression models were used to evaluate the relationship between subjective PROs and physician-obtained measures.</p><p><strong>Results: </strong>Analyses revealed an important role for mental health factors in subjective measures of recovery, though cognitive dysfunction was not significantly associated with either subjective improvement or quality of life despite being commonly endorsed. Depression and fatigue were inversely associated with both satisfaction and quality of life, along with stroke severity and overall functional impairment during both short- and long-term recovery periods. The impact of depression on quality of life increased over time, while stroke severity and functional status were associated with perceived symptomatic improvement at all time points.</p><p><strong>Conclusions: </strong>For patients with minor stroke, depression is negatively associated with perception of symptomatic recovery and quality of life, particularly at later time points. Addressing post-stroke depression may improve patient-reported outcomes, though further research is needed to determine its impact on broader measures of post-stroke morbidity.</p>","PeriodicalId":36660,"journal":{"name":"Journal of Patient-Reported Outcomes","volume":"9 1","pages":"29"},"PeriodicalIF":2.4,"publicationDate":"2025-03-11","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11896911/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143606700","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":"Sarcoma Assessment Measure-Paediatric Version (SAM-Paeds): development of a disease-specific patient reported outcome measure for children with sarcoma.","authors":"Rachel M Taylor, Sophie-Anne Purnell, Sian Hocking, Rachael Windsor, Craig Gerrand, Meriel Jenney, Madeleine Adams","doi":"10.1186/s41687-025-00857-6","DOIUrl":"10.1186/s41687-025-00857-6","url":null,"abstract":"<p><strong>Purpose: </strong>Sarcomas account for approximately 10-15% of all cancer in children aged ≤ 16. Poorer health-related quality of life (HRQoL) is recorded in comparison to other cancers; however, these studies are limited by generic HRQoL measures not being specific to patients with sarcoma. The aim of this study was to develop paediatric version of the Sarcoma Assessment Measure (SAM).</p><p><strong>Methods: </strong>This mixed methods study comprised three stages: item generation, item reduction and establishing content validity. Children aged 8-16 years and parents of children aged 0-16 years with a diagnosis of sarcoma and within 5 years of completion of treatment were invited to participate.</p><p><strong>Results: </strong>A total of 29 children and 38 parents from three sites participated in the study. Content analysis of the interview transcripts identified 277 post-diagnosis experience statements of which 128 'items' were included in an Item Reduction Questionnaire, grouped into six domains; physical, disability and inclusion; impact of diagnosis; emotional, impact on family, education. Items with a mean score < 5 and a content validity index of < 0.75 were removed. The final version of SAM-Paeds comprises 33 items (parent version) and 21 items (child version).</p><p><strong>Conclusion: </strong>This study has developed the first disease-specific HRQoL measure for paediatric sarcoma patients. SAM-Paeds is planned for inclusion within international sarcoma clinical trials and will be validated alongside current generic measures. Developed with the same methodology as the adult SAM questionnaire will facilitate the assessment of QoL longitudinally to assess the long-term impact of the diagnosis and treatment of sarcoma in childhood.</p>","PeriodicalId":36660,"journal":{"name":"Journal of Patient-Reported Outcomes","volume":"9 1","pages":"30"},"PeriodicalIF":2.4,"publicationDate":"2025-03-11","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11896951/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143606707","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}