Purpose: Inborn errors of metabolism (IEM) are known with poor long-term health concerns; however, the health-related quality of life (HRQoL) and the burden placed on families remain unclear. This study investigated the self- and proxy-reported HRQoL of pediatric patients with IEM with or without developmental disabilities and the burden placed on their caregivers.
Methods: Patients with IEM aged 8-15 years and their caregivers were asked to respond to the Pediatric Quality of Life Inventory (PedsQL), EuroQoL five-dimension questionnaire for younger populations (EQ-5D-Y), and Japanese version of the Zarit Caregiver Burden Interview (J-ZBI). We compared EQ-5D-Y scores with matched EQ-5D-Y population norms. Intraclass correlation coefficients (ICC) for self and proxy HRQoL scores of those without developmental disabilities were calculated. Correlation coefficients of HRQoL proxy responses with J-ZBI score were estimated.
Results: We included 66 patients with IEM (mean age, 11.5 years; males, 41.2%) in the study. The mean (± standard deviation) EQ-5D-Y scores without and with developmental disabilities were 0.957 (± 0.071) and 0.821 (± 0.175), respectively. The EQ-5D-Y scores significantly increased compared with the reference values (p < 0.01, effect size = 0.337). The ICC values were 0.331 and 0.477 for the EQ-5D-Y and PedsQL scores, respectively. HRQoL proxy scores had strong negative correlations with J-ZBI scores.
Conclusion: The HRQoL of patients with IEM without developmental disabilities in our study was similar to that of the general Japanese population. The HRQoL of patients with IEM with developmental disabilities was low and associated with a tendency towards an increased burden of care.
{"title":"Health-related quality of life and caregiver burden of pediatric patients with inborn errors of metabolism in Japan using EQ-5D-Y, PedsQL, and J-ZBI.","authors":"Keiko Konomura, Chikahiko Numakura, Akari Nakamura-Utsunomiya, Eri Hoshino, Go Tajima, Hironori Kobayashi, Kimitoshi Nakamura, Nobuyuki Shimozawa, Ryosuke Bo, Takeru Shiroiwa, Yosuke Shigematsu, Takashi Fukuda","doi":"10.1007/s11136-024-03775-0","DOIUrl":"https://doi.org/10.1007/s11136-024-03775-0","url":null,"abstract":"<p><strong>Purpose: </strong>Inborn errors of metabolism (IEM) are known with poor long-term health concerns; however, the health-related quality of life (HRQoL) and the burden placed on families remain unclear. This study investigated the self- and proxy-reported HRQoL of pediatric patients with IEM with or without developmental disabilities and the burden placed on their caregivers.</p><p><strong>Methods: </strong>Patients with IEM aged 8-15 years and their caregivers were asked to respond to the Pediatric Quality of Life Inventory (PedsQL), EuroQoL five-dimension questionnaire for younger populations (EQ-5D-Y), and Japanese version of the Zarit Caregiver Burden Interview (J-ZBI). We compared EQ-5D-Y scores with matched EQ-5D-Y population norms. Intraclass correlation coefficients (ICC) for self and proxy HRQoL scores of those without developmental disabilities were calculated. Correlation coefficients of HRQoL proxy responses with J-ZBI score were estimated.</p><p><strong>Results: </strong>We included 66 patients with IEM (mean age, 11.5 years; males, 41.2%) in the study. The mean (± standard deviation) EQ-5D-Y scores without and with developmental disabilities were 0.957 (± 0.071) and 0.821 (± 0.175), respectively. The EQ-5D-Y scores significantly increased compared with the reference values (p < 0.01, effect size = 0.337). The ICC values were 0.331 and 0.477 for the EQ-5D-Y and PedsQL scores, respectively. HRQoL proxy scores had strong negative correlations with J-ZBI scores.</p><p><strong>Conclusion: </strong>The HRQoL of patients with IEM without developmental disabilities in our study was similar to that of the general Japanese population. The HRQoL of patients with IEM with developmental disabilities was low and associated with a tendency towards an increased burden of care.</p>","PeriodicalId":20748,"journal":{"name":"Quality of Life Research","volume":null,"pages":null},"PeriodicalIF":3.3,"publicationDate":"2024-09-04","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142126514","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2024-09-04DOI: 10.1007/s11136-024-03781-2
Laura B Vater, Ali Ajrouch, Patrick O Monahan, Laura Jennewein, Yan Han, Ahmad Karkash, Nasser H Hanna
Purpose: As cancer survivorship increases, there is a need for simple tools to measure and promote healthy behaviors. We created a wellness behavioral tool (the SMILE Scale) to encourage self-monitoring of wellness behaviors. This study aimed to determine the feasibility of collecting daily self-reported SMILE Scale data and weekly quality of life data among patients with cancer. We also aimed to measure the association between SMILE Scale responses and validated health-related quality of life (HRQOL) tools (PROMIS-29 + 2 and SymTrak-8) as a pilot test of the hypothesis that increased wellness behaviors may impact quality of life.
Methods: We surveyed 100 patients with cancer at the Indiana University Simon Comprehensive Cancer Center. Participants were asked to complete daily SMILE Scale assessments over a two-week period, as well as weekly PROMIS-29 + 2 and SymTrak-8 surveys. The primary endpoint was the SMILE Scale completion rate. Secondary endpoints in this single-arm pilot study included correlations between the SMILE Scale and other HRQOL tools.
Results: Daily completion rate of the SMILE Scale ranged from 57% to 65% of participants over a 14-day period. Among the 61% of participants who completed SMILE on day 1, 87% completed SMILE on 10 of 14 days. By end of study, participants who self-reported more wellness behaviors (i.e., higher daily SMILE scores) demonstrated significantly higher PROMIS physical health (p = 0.003), higher PROMIS mental health (p = 0.008), and lower (better) SymTrak total symptom burden (p = 0.006). Further, among those who completed at least 1 of 14 daily SMILE assessments, quality of life significantly improved over the two-week period for PROMIS mental health (p = 0.018) and SymTrak total symptom burden (p = 0.014).
Conclusion: The SMILE Scale completion rate did not satisfy our pre-planned ≥70% threshold for feasibility; however, the rate for completing SMILE at least once during the 14 days (77%) met this threshold. Participants with higher average daily SMILE scores had significantly better scores across other validated HRQOL tools. While these results may be correlative and not causative, this suggests a potential physical and mental health benefit for delivering the SMILE Scale in clinical practice to help encourage healthy behaviors and warrants testing the SMILE Scale's impact in future studies.
{"title":"The SMILE scale: a wellness behavioral tool for patients with cancer.","authors":"Laura B Vater, Ali Ajrouch, Patrick O Monahan, Laura Jennewein, Yan Han, Ahmad Karkash, Nasser H Hanna","doi":"10.1007/s11136-024-03781-2","DOIUrl":"https://doi.org/10.1007/s11136-024-03781-2","url":null,"abstract":"<p><strong>Purpose: </strong>As cancer survivorship increases, there is a need for simple tools to measure and promote healthy behaviors. We created a wellness behavioral tool (the SMILE Scale) to encourage self-monitoring of wellness behaviors. This study aimed to determine the feasibility of collecting daily self-reported SMILE Scale data and weekly quality of life data among patients with cancer. We also aimed to measure the association between SMILE Scale responses and validated health-related quality of life (HRQOL) tools (PROMIS-29 + 2 and SymTrak-8) as a pilot test of the hypothesis that increased wellness behaviors may impact quality of life.</p><p><strong>Methods: </strong>We surveyed 100 patients with cancer at the Indiana University Simon Comprehensive Cancer Center. Participants were asked to complete daily SMILE Scale assessments over a two-week period, as well as weekly PROMIS-29 + 2 and SymTrak-8 surveys. The primary endpoint was the SMILE Scale completion rate. Secondary endpoints in this single-arm pilot study included correlations between the SMILE Scale and other HRQOL tools.</p><p><strong>Results: </strong>Daily completion rate of the SMILE Scale ranged from 57% to 65% of participants over a 14-day period. Among the 61% of participants who completed SMILE on day 1, 87% completed SMILE on 10 of 14 days. By end of study, participants who self-reported more wellness behaviors (i.e., higher daily SMILE scores) demonstrated significantly higher PROMIS physical health (p = 0.003), higher PROMIS mental health (p = 0.008), and lower (better) SymTrak total symptom burden (p = 0.006). Further, among those who completed at least 1 of 14 daily SMILE assessments, quality of life significantly improved over the two-week period for PROMIS mental health (p = 0.018) and SymTrak total symptom burden (p = 0.014).</p><p><strong>Conclusion: </strong>The SMILE Scale completion rate did not satisfy our pre-planned ≥70% threshold for feasibility; however, the rate for completing SMILE at least once during the 14 days (77%) met this threshold. Participants with higher average daily SMILE scores had significantly better scores across other validated HRQOL tools. While these results may be correlative and not causative, this suggests a potential physical and mental health benefit for delivering the SMILE Scale in clinical practice to help encourage healthy behaviors and warrants testing the SMILE Scale's impact in future studies.</p>","PeriodicalId":20748,"journal":{"name":"Quality of Life Research","volume":null,"pages":null},"PeriodicalIF":3.3,"publicationDate":"2024-09-04","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142126515","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2024-09-03DOI: 10.1007/s11136-024-03733-w
Ruth C Jarvis, Philip Pallmann, Collett Clements, Hrishikesh Joshi
Purpose: Patients with urinary calculi undergo resource-intensive follow-up. Application of a PROM, Urinary Stones and Intervention Quality of Life (USIQoL), can potentially optimise current practices if it matches the outcomes of traditional follow-up. Our objective was to develop, and conduct, a preliminary validation of the USIQoL based prediction model to aid triage.
Methods: We performed a two phase prospective cohort study. The 1st phase included development of the USIQoL-based decision model using multicentre data. The 2nd phase involved prospective single-blind external validation for the outpatient application. The aim was to evaluate correlations between the USIQoL scores and key predictors; clinical outcomes and global health ratings (EuroQoL EQ-5D). We used statistical analysis to validate USIQoL cut-off scores to aid triage and the decision to intervene.
Results: Of 503 patients invited, 91% (n = 455, Development [305] and Validation [150]; M = 308, F = 147) participated. The relationship between USIQoL domain scores and clinical outcomes was consistently significant (estimated odds: PPH 1.24, p < 0.001, 95% CI 1.13-1.36; PSH 1.22, p < 0.001, 95% CI 1.12-1.33). The ROC values for the model were ≥ 0.75. The optimum domain cut-off scores were derived with rising scores implying increased need to intervene. The model demonstrated satisfactory sensitivity (0.81-0.89) and specificity (0.36-0.47).
Conclusions: The study demonstrates satisfactory correlation between the USIQoL and clinical outcomes making this model a valid aid for triage and optimising outpatient management with the cut-off scores able to identify high risk patients who need active treatment.
{"title":"Development and preliminary validation of a diagnostic prediction model to optimise outpatient management of patients with urolithiasis using urinary stones and intervention quality of life (USIQoL) measure.","authors":"Ruth C Jarvis, Philip Pallmann, Collett Clements, Hrishikesh Joshi","doi":"10.1007/s11136-024-03733-w","DOIUrl":"https://doi.org/10.1007/s11136-024-03733-w","url":null,"abstract":"<p><strong>Purpose: </strong>Patients with urinary calculi undergo resource-intensive follow-up. Application of a PROM, Urinary Stones and Intervention Quality of Life (USIQoL), can potentially optimise current practices if it matches the outcomes of traditional follow-up. Our objective was to develop, and conduct, a preliminary validation of the USIQoL based prediction model to aid triage.</p><p><strong>Methods: </strong>We performed a two phase prospective cohort study. The 1st phase included development of the USIQoL-based decision model using multicentre data. The 2nd phase involved prospective single-blind external validation for the outpatient application. The aim was to evaluate correlations between the USIQoL scores and key predictors; clinical outcomes and global health ratings (EuroQoL EQ-5D). We used statistical analysis to validate USIQoL cut-off scores to aid triage and the decision to intervene.</p><p><strong>Results: </strong>Of 503 patients invited, 91% (n = 455, Development [305] and Validation [150]; M = 308, F = 147) participated. The relationship between USIQoL domain scores and clinical outcomes was consistently significant (estimated odds: PPH 1.24, p < 0.001, 95% CI 1.13-1.36; PSH 1.22, p < 0.001, 95% CI 1.12-1.33). The ROC values for the model were ≥ 0.75. The optimum domain cut-off scores were derived with rising scores implying increased need to intervene. The model demonstrated satisfactory sensitivity (0.81-0.89) and specificity (0.36-0.47).</p><p><strong>Conclusions: </strong>The study demonstrates satisfactory correlation between the USIQoL and clinical outcomes making this model a valid aid for triage and optimising outpatient management with the cut-off scores able to identify high risk patients who need active treatment.</p>","PeriodicalId":20748,"journal":{"name":"Quality of Life Research","volume":null,"pages":null},"PeriodicalIF":3.3,"publicationDate":"2024-09-03","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142120397","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2024-09-03DOI: 10.1007/s11136-024-03774-1
Yiyin Cao, Huan Zhang, Nan Luo, Haofei Li, Ling Jie Cheng, Weidong Huang
Objective: To examine the test-retest reliability of the Functional Assessment of Cancer Therapy - 8 Dimension (FACT-8D) for the first time, and to conduct a head-to-head comparison of the distribution properties and validity between the FACT-8D and EQ-5D-5L in Colorectal Cancer (CRC) Patients.
Methods: We conducted a longitudinal study on Chinese CRC patients, employing Functional Assessment of Cancer Therapy-General (FACT-G) and EQ-5D-5L at baseline, and FACT-G during follow-up (2-7 days from baseline). Utility scores for FACT-8D were derived from all available value sets (Australia, Canada and USA), while EQ-5D-5L scores were obtained from corresponding value sets for various countries. We assessed convergent validity using pairwise polychoric correlations between the FACT-8D and EQ-5D-5L; known-groups validity by discriminating participants' clinical characteristics, and effect size (ES) was tested; test-retest reliability for FACT-8D using kappa and weighted Kappa for choice consistency, and intraclass correlation coefficient (ICC) and Bland-Altman method for utility consistency.
Results: Among the 287 patients with CRC at baseline, 131 were included in the retest analysis. The utility scores of FACT-8D were highly positively correlated with EQ-5D-5L across various country value sets (r = 0.65-0.77), and most of the dimensions of FACT-8D and EQ-5D-5L were positively correlated. EQ-5D-5L failed to discriminate known-groups in cancer stage across all value sets, whereas both were significant in FACT-8D (ES = 0.35-0.48, ES = 0.38-0.52). FACT-8D showed good test-retest reliability (Cohen's weighted Kappa = 0.494-0.722, ICC = 0.748-0.786).
Conclusion: The FACT-8D can be used as a valid and reliable instrument for clinical evaluation of patients with CRC, outperforming EQ-5D-5L in differentiating clinical subgroups and showing promise for cancer practice and research.
{"title":"Assessing the reliability of a novel cancer-specific multi-attribute utility instrument (FACT-8D) and comparing its validity to EQ-5D-5L in colorectal cancer patients.","authors":"Yiyin Cao, Huan Zhang, Nan Luo, Haofei Li, Ling Jie Cheng, Weidong Huang","doi":"10.1007/s11136-024-03774-1","DOIUrl":"https://doi.org/10.1007/s11136-024-03774-1","url":null,"abstract":"<p><strong>Objective: </strong>To examine the test-retest reliability of the Functional Assessment of Cancer Therapy - 8 Dimension (FACT-8D) for the first time, and to conduct a head-to-head comparison of the distribution properties and validity between the FACT-8D and EQ-5D-5L in Colorectal Cancer (CRC) Patients.</p><p><strong>Methods: </strong>We conducted a longitudinal study on Chinese CRC patients, employing Functional Assessment of Cancer Therapy-General (FACT-G) and EQ-5D-5L at baseline, and FACT-G during follow-up (2-7 days from baseline). Utility scores for FACT-8D were derived from all available value sets (Australia, Canada and USA), while EQ-5D-5L scores were obtained from corresponding value sets for various countries. We assessed convergent validity using pairwise polychoric correlations between the FACT-8D and EQ-5D-5L; known-groups validity by discriminating participants' clinical characteristics, and effect size (ES) was tested; test-retest reliability for FACT-8D using kappa and weighted Kappa for choice consistency, and intraclass correlation coefficient (ICC) and Bland-Altman method for utility consistency.</p><p><strong>Results: </strong>Among the 287 patients with CRC at baseline, 131 were included in the retest analysis. The utility scores of FACT-8D were highly positively correlated with EQ-5D-5L across various country value sets (r = 0.65-0.77), and most of the dimensions of FACT-8D and EQ-5D-5L were positively correlated. EQ-5D-5L failed to discriminate known-groups in cancer stage across all value sets, whereas both were significant in FACT-8D (ES = 0.35-0.48, ES = 0.38-0.52). FACT-8D showed good test-retest reliability (Cohen's weighted Kappa = 0.494-0.722, ICC = 0.748-0.786).</p><p><strong>Conclusion: </strong>The FACT-8D can be used as a valid and reliable instrument for clinical evaluation of patients with CRC, outperforming EQ-5D-5L in differentiating clinical subgroups and showing promise for cancer practice and research.</p>","PeriodicalId":20748,"journal":{"name":"Quality of Life Research","volume":null,"pages":null},"PeriodicalIF":3.3,"publicationDate":"2024-09-03","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142120396","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2024-08-31DOI: 10.1007/s11136-024-03768-z
Christos Mousoulis, Andrew D Firth, Alanna Marson, Joel J Gagnier
Purpose: To extensively review Patient Reported Outcome Measures (PROMs) used to assess outcomes in persons with hand fractures in terms of their psychometric properties.
Methods: A COnsensus-based Standards for the selection of health Measurement INstruments (COSMIN) methodological review was conducted. Six electronic databases were searched (Medline, Embase, Scopus, Web of Science, PsycINFO, CINAHL) for studies evaluating the psychometric properties of PROMs assessing recovery from hand fracture. Titles and abstracts, full text review, quality assessment and data extraction were performed by two reviewers. Any disagreements were resolved after review by a third, expert reviewer. Quality assessment of included studies was performed using the COSMIN checklist.
Results: This COSMIN review found that there were only 4 studies that fulfilled the inclusion criteria in terms of assessing measurement properties of PROMs in hand fractures. Only the construct validity of the Disabilities of the Arm, Shoulder, and Hand (DASH), the Quick DASH (QDASH) and the Duruoz Hand Index (DHI), and the responsiveness of the DASH, the Patient Specific Functional Scale (PSFS), the Patient-Rated Wrist and Hand Evaluation (PRWHE) and the Michigan Hand Outcomes Questionnaire (MHQ) were assessed in these studies. The overall rating of the studies was assessed as insufficient or indeterminate and quality of evidence was assessed as moderate, low, or very low by our team.
Conclusions: This study identified that there is a lack of evidence in the medical literature with regards to the measurement properties of PROMs in patients with hand fractures. The 4 included studies do not provide good quality data to support the use of these PROMS in patients with hand fractures. There is a need for more studies for more PROMs. This has important consequences for how outcomes will be measured in clinical studies in hand research and in clinical practice.
{"title":"Psychometric properties of patient-reported outcome measures assessing recovery from hand fractures: a systematic review.","authors":"Christos Mousoulis, Andrew D Firth, Alanna Marson, Joel J Gagnier","doi":"10.1007/s11136-024-03768-z","DOIUrl":"https://doi.org/10.1007/s11136-024-03768-z","url":null,"abstract":"<p><strong>Purpose: </strong>To extensively review Patient Reported Outcome Measures (PROMs) used to assess outcomes in persons with hand fractures in terms of their psychometric properties.</p><p><strong>Methods: </strong>A COnsensus-based Standards for the selection of health Measurement INstruments (COSMIN) methodological review was conducted. Six electronic databases were searched (Medline, Embase, Scopus, Web of Science, PsycINFO, CINAHL) for studies evaluating the psychometric properties of PROMs assessing recovery from hand fracture. Titles and abstracts, full text review, quality assessment and data extraction were performed by two reviewers. Any disagreements were resolved after review by a third, expert reviewer. Quality assessment of included studies was performed using the COSMIN checklist.</p><p><strong>Results: </strong>This COSMIN review found that there were only 4 studies that fulfilled the inclusion criteria in terms of assessing measurement properties of PROMs in hand fractures. Only the construct validity of the Disabilities of the Arm, Shoulder, and Hand (DASH), the Quick DASH (QDASH) and the Duruoz Hand Index (DHI), and the responsiveness of the DASH, the Patient Specific Functional Scale (PSFS), the Patient-Rated Wrist and Hand Evaluation (PRWHE) and the Michigan Hand Outcomes Questionnaire (MHQ) were assessed in these studies. The overall rating of the studies was assessed as insufficient or indeterminate and quality of evidence was assessed as moderate, low, or very low by our team.</p><p><strong>Conclusions: </strong>This study identified that there is a lack of evidence in the medical literature with regards to the measurement properties of PROMs in patients with hand fractures. The 4 included studies do not provide good quality data to support the use of these PROMS in patients with hand fractures. There is a need for more studies for more PROMs. This has important consequences for how outcomes will be measured in clinical studies in hand research and in clinical practice.</p>","PeriodicalId":20748,"journal":{"name":"Quality of Life Research","volume":null,"pages":null},"PeriodicalIF":3.3,"publicationDate":"2024-08-31","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142111347","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2024-08-31DOI: 10.1007/s11136-024-03764-3
Marina Zeldovich, Leonie Krol, Inga K Koerte, Katrin Cunitz, Matthias Kieslich, Marlene Henrich, Knut Brockmann, Anna Buchheim, Michael Lendt, Christian Auer, Axel Neu, Joenna Driemeyer, Ulrike Wartemann, Claudius Thomé, Daniel Pinggera, Steffen Berweck, Michaela V Bonfert, Joachim Suss, Holger Muehlan, Nicole von Steinbuechel
Purpose: The impact of pediatric traumatic brain injury (pTBI) on health-related quality of life (HRQoL) in children and adolescents remains understudied. Short scales have some advantages in terms of economy and administration over longer scales, especially in younger children. The aim of the present study is to psychometrically evaluate the six-item German version of the QOLIBRI-OS-KID/ADO scale for children and adolescents. In addition, reference values from a general German pediatric population are obtained to assist clinicians and researchers in the interpretation of HRQoL after pTBI.
Methods: A total of 297 individuals after TBI and 1997 from a general population sample completed the questionnaire. Reliability, validity, and comparability of the assessed construct were examined.
Results: The questionnaire showed satisfactory reliability (α = 0.75 and ω = 0.81 and α = 0.85 and ω = 0.86 for the TBI and general population samples, respectively). The QOLIBRI-OS-KID/ADO was highly correlated with its long version (R2 = 67%) and showed an overlap with disease-specific HRQoL (R2 = 55%) in the TBI sample. The one-dimensional factorial structure could be replicated and tested for measurement invariance between samples, indicating a comparable HRQoL construct assessment. Therefore, reference values and cut-offs indicating clinically relevant impairment could be provided using percentiles stratified by factors significantly associated with the total score in the regression analyses (i.e., age group and gender).
Conclusion: In combination with the cut-offs, the QOLIBRI-OS-KID/ADO provides a cost-effective screening tool, complemented by interpretation guidelines, which may help to draw clinical conclusions and indications such as further administration of a longer version of the instrument to gain more detailed insight into impaired HRQoL domains or omission of further steps in the absence of an indication.
{"title":"A short scale to measure health-related quality of life after traumatic brain injury in children and adolescents (QOLIBRI-OS-KID/ADO): psychometric properties and German reference values.","authors":"Marina Zeldovich, Leonie Krol, Inga K Koerte, Katrin Cunitz, Matthias Kieslich, Marlene Henrich, Knut Brockmann, Anna Buchheim, Michael Lendt, Christian Auer, Axel Neu, Joenna Driemeyer, Ulrike Wartemann, Claudius Thomé, Daniel Pinggera, Steffen Berweck, Michaela V Bonfert, Joachim Suss, Holger Muehlan, Nicole von Steinbuechel","doi":"10.1007/s11136-024-03764-3","DOIUrl":"https://doi.org/10.1007/s11136-024-03764-3","url":null,"abstract":"<p><strong>Purpose: </strong>The impact of pediatric traumatic brain injury (pTBI) on health-related quality of life (HRQoL) in children and adolescents remains understudied. Short scales have some advantages in terms of economy and administration over longer scales, especially in younger children. The aim of the present study is to psychometrically evaluate the six-item German version of the QOLIBRI-OS-KID/ADO scale for children and adolescents. In addition, reference values from a general German pediatric population are obtained to assist clinicians and researchers in the interpretation of HRQoL after pTBI.</p><p><strong>Methods: </strong>A total of 297 individuals after TBI and 1997 from a general population sample completed the questionnaire. Reliability, validity, and comparability of the assessed construct were examined.</p><p><strong>Results: </strong>The questionnaire showed satisfactory reliability (α = 0.75 and ω = 0.81 and α = 0.85 and ω = 0.86 for the TBI and general population samples, respectively). The QOLIBRI-OS-KID/ADO was highly correlated with its long version (R<sup>2</sup> = 67%) and showed an overlap with disease-specific HRQoL (R<sup>2</sup> = 55%) in the TBI sample. The one-dimensional factorial structure could be replicated and tested for measurement invariance between samples, indicating a comparable HRQoL construct assessment. Therefore, reference values and cut-offs indicating clinically relevant impairment could be provided using percentiles stratified by factors significantly associated with the total score in the regression analyses (i.e., age group and gender).</p><p><strong>Conclusion: </strong>In combination with the cut-offs, the QOLIBRI-OS-KID/ADO provides a cost-effective screening tool, complemented by interpretation guidelines, which may help to draw clinical conclusions and indications such as further administration of a longer version of the instrument to gain more detailed insight into impaired HRQoL domains or omission of further steps in the absence of an indication.</p>","PeriodicalId":20748,"journal":{"name":"Quality of Life Research","volume":null,"pages":null},"PeriodicalIF":3.3,"publicationDate":"2024-08-31","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142111345","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2024-08-29DOI: 10.1007/s11136-024-03765-2
Desiree Scott, Sarah Derrett, Valentina Prevolnik Rupel, Jennifer Jelsma, Gagan Gurung, Georgina Yaa Oduro, Cassie Withey-Rila
Purpose: To ensure the recognition and participation of all potential respondents in health research, surveys and care, including LGBTQIA + broadly, and trans people, specifically, the use of inclusive language should be considered. This scoping review aimed to identify and describe strategies considered for gender inclusivity in development and use of health questionnaires and Patient Reported Outcomes Measures (PROMs).
Methods: A systematic search of peer reviewed publications between January 2000 and September 2022 was conducted in Scopus, ProQuest Central, Ovid Medline (PubMed and EBSCO). Two reviewers independently screened identified publications titles and abstracts, followed by full text screening and data extraction from eligible articles.
Results: The search of over 5000 publications, retained 18; most acknowledged gaps in representation and advocated for gender-inclusive language. Eight articles discussed exclusion from health care and health research for gender minority groups due to the use of gendered language. Improved reliability, validity and response rates were associated with the use of gender-neutral language in seven articles. Only one article reported finding irritation among cisgender males when non-binary gender response options were used. One paper, focussing on instruments for Rheumatoid Arthritis, discussed gaps in representation if diverse gender identities were not considered when developing PROMs.
Conclusion: This scoping review points to the importance of adopting gender-inclusive language in health questionnaires and surveys to reduce the risk of excluding gender minority groups. Despite finding very few specific examples of how others have used gender-inclusive language in health questionnaires, many strengths of gender-inclusive language usage were identified.
{"title":"He/She/They - gender inclusivity in developing and using health-related questionnaires: a scoping review.","authors":"Desiree Scott, Sarah Derrett, Valentina Prevolnik Rupel, Jennifer Jelsma, Gagan Gurung, Georgina Yaa Oduro, Cassie Withey-Rila","doi":"10.1007/s11136-024-03765-2","DOIUrl":"https://doi.org/10.1007/s11136-024-03765-2","url":null,"abstract":"<p><strong>Purpose: </strong>To ensure the recognition and participation of all potential respondents in health research, surveys and care, including LGBTQIA + broadly, and trans people, specifically, the use of inclusive language should be considered. This scoping review aimed to identify and describe strategies considered for gender inclusivity in development and use of health questionnaires and Patient Reported Outcomes Measures (PROMs).</p><p><strong>Methods: </strong>A systematic search of peer reviewed publications between January 2000 and September 2022 was conducted in Scopus, ProQuest Central, Ovid Medline (PubMed and EBSCO). Two reviewers independently screened identified publications titles and abstracts, followed by full text screening and data extraction from eligible articles.</p><p><strong>Results: </strong>The search of over 5000 publications, retained 18; most acknowledged gaps in representation and advocated for gender-inclusive language. Eight articles discussed exclusion from health care and health research for gender minority groups due to the use of gendered language. Improved reliability, validity and response rates were associated with the use of gender-neutral language in seven articles. Only one article reported finding irritation among cisgender males when non-binary gender response options were used. One paper, focussing on instruments for Rheumatoid Arthritis, discussed gaps in representation if diverse gender identities were not considered when developing PROMs.</p><p><strong>Conclusion: </strong>This scoping review points to the importance of adopting gender-inclusive language in health questionnaires and surveys to reduce the risk of excluding gender minority groups. Despite finding very few specific examples of how others have used gender-inclusive language in health questionnaires, many strengths of gender-inclusive language usage were identified.</p>","PeriodicalId":20748,"journal":{"name":"Quality of Life Research","volume":null,"pages":null},"PeriodicalIF":3.3,"publicationDate":"2024-08-29","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142111346","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2024-08-29DOI: 10.1007/s11136-024-03773-2
Meaghan O'Connor, Lynne Broderick, Miranda Lauher-Charest, Laura Tesler Waldman, Kristi Jackson, Mark Kosinski, Michelle Carty
Purpose: Mapping or matching the items in a clinical outcome assessment (COA) to concepts that define a condition is a common method for evaluating a COA's concept coverage. The purpose of this research was to address the lack of formal guidance for conducting this task by developing a framework for best practices in COA concept mapping and applying it to a case study.
Methods: To develop the framework, we examined the literature and created a draft set of best practices which was then reviewed by experienced researchers through focus groups before being finalized. To conduct the case study, we extracted data from a systematic review of knee osteoarthritis (KO) symptoms and impacts and used the framework to map relevant concepts to items in the SF-36v2® Health Survey (SF-36v2).
Results: The framework guides researchers in defining the purpose of and data sources for the mapping, establishing guiding principles and decision-making thresholds, and conducting the mapping exercise. The results of the case study demonstrate the usefulness of the framework in identifying 27/36 items (75%) in the SF-36v2 that addressed concepts that define KO.
Conclusion: This case study illustrates how the framework for best practices in COA concept mapping may be used, highlighting how establishing clear concept definitions and guiding principles and following a structured process throughout can help produce consistent, reliable, and reproducible results. The results from this rigorous approach can provide valuable evidence to support decisions about the appropriateness of a COA for the intended patient population.
目的:将临床结果评估(COA)中的项目与定义病情的概念进行映射或匹配,是评估 COA 概念覆盖范围的常用方法。本研究的目的是通过制定 COA 概念映射的最佳实践框架并将其应用于案例研究,解决在开展这项工作时缺乏正式指导的问题:为了制定该框架,我们研究了相关文献,并创建了一套最佳实践草案,然后由经验丰富的研究人员通过焦点小组对该草案进行审查,最后定稿。为了进行案例研究,我们从膝关节骨性关节炎(KO)症状和影响的系统综述中提取数据,并使用该框架将相关概念映射到 SF-36v2® 健康调查(SF-36v2)的项目中:结果:该框架指导研究人员确定映射的目的和数据来源,建立指导原则和决策阈值,并开展映射工作。案例研究的结果表明,该框架在确定 SF-36v2 中涉及界定 KO 概念的 27/36 个项目(75%)方面非常有用:本案例研究说明了如何使用 COA 概念映射最佳实践框架,强调了建立明确的概念定义和指导原则,以及在整个过程中遵循结构化流程如何有助于产生一致、可靠和可重复的结果。这种严谨方法得出的结果可以提供有价值的证据,支持有关 COA 是否适合目标患者群体的决策。
{"title":"A framework for best practices in clinical outcome assessment (COA) concept mapping: a case study.","authors":"Meaghan O'Connor, Lynne Broderick, Miranda Lauher-Charest, Laura Tesler Waldman, Kristi Jackson, Mark Kosinski, Michelle Carty","doi":"10.1007/s11136-024-03773-2","DOIUrl":"https://doi.org/10.1007/s11136-024-03773-2","url":null,"abstract":"<p><strong>Purpose: </strong>Mapping or matching the items in a clinical outcome assessment (COA) to concepts that define a condition is a common method for evaluating a COA's concept coverage. The purpose of this research was to address the lack of formal guidance for conducting this task by developing a framework for best practices in COA concept mapping and applying it to a case study.</p><p><strong>Methods: </strong>To develop the framework, we examined the literature and created a draft set of best practices which was then reviewed by experienced researchers through focus groups before being finalized. To conduct the case study, we extracted data from a systematic review of knee osteoarthritis (KO) symptoms and impacts and used the framework to map relevant concepts to items in the SF-36v2<sup>®</sup> Health Survey (SF-36v2).</p><p><strong>Results: </strong>The framework guides researchers in defining the purpose of and data sources for the mapping, establishing guiding principles and decision-making thresholds, and conducting the mapping exercise. The results of the case study demonstrate the usefulness of the framework in identifying 27/36 items (75%) in the SF-36v2 that addressed concepts that define KO.</p><p><strong>Conclusion: </strong>This case study illustrates how the framework for best practices in COA concept mapping may be used, highlighting how establishing clear concept definitions and guiding principles and following a structured process throughout can help produce consistent, reliable, and reproducible results. The results from this rigorous approach can provide valuable evidence to support decisions about the appropriateness of a COA for the intended patient population.</p>","PeriodicalId":20748,"journal":{"name":"Quality of Life Research","volume":null,"pages":null},"PeriodicalIF":3.3,"publicationDate":"2024-08-29","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142111344","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2024-08-28DOI: 10.1007/s11136-024-03757-2
C Gibbons, G Brown, S C Lu, A Elrick, Y Tang, M Kaufman, M Williams, C Xu, C Harrison, C Swisher
{"title":"Correction: Introduction and validation of the open symptom framework: a public domain modular framework for patient-reported measurement of symptoms related to cancer and its treatment.","authors":"C Gibbons, G Brown, S C Lu, A Elrick, Y Tang, M Kaufman, M Williams, C Xu, C Harrison, C Swisher","doi":"10.1007/s11136-024-03757-2","DOIUrl":"https://doi.org/10.1007/s11136-024-03757-2","url":null,"abstract":"","PeriodicalId":20748,"journal":{"name":"Quality of Life Research","volume":null,"pages":null},"PeriodicalIF":3.3,"publicationDate":"2024-08-28","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142081378","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2024-08-28DOI: 10.1007/s11136-024-03761-6
Lidwine B Mokkink, Ellen B M Elsman, Caroline B Terwee
Purpose: Systematic reviews of patient-reported outcome measures (PROMs) are important tools to select the most suitable PROM for a study or clinical application. Conducting these reviews is challenging, and the quality of these reviews needs to be improved. We updated the COSMIN guideline for systematic reviews of PROMs, including the COSMIN Risk of Bias checklist, and the COSMIN criteria for good measurement properties.
Methods: Adaptations to the methodology were based on our experience with applying the COSMIN guideline, through discussions among the authors, and results from two related Delphi studies.
Results: The updated guideline places more emphasis on key aspects that are often missing or sub optimally conducted in published systematic reviews of PROMs, such as formulating a well-defined research question and developing a comprehensive search strategy, assessing risk of bias, applying criteria for good measurement properties, summarizing results, and grading the quality of the evidence. We also stress the importance of evaluating the measurement properties of each subscale of a PROM separately and evaluating content validity of all included PROMs.
Conclusion: The quality of systematic reviews of PROMs can be improved by using this updated version of the COSMIN guideline for systematic reviews of PROMs. Improved quality will lead to better PROM selection and increased standardization of PROM use.
{"title":"COSMIN guideline for systematic reviews of patient-reported outcome measures version 2.0.","authors":"Lidwine B Mokkink, Ellen B M Elsman, Caroline B Terwee","doi":"10.1007/s11136-024-03761-6","DOIUrl":"https://doi.org/10.1007/s11136-024-03761-6","url":null,"abstract":"<p><strong>Purpose: </strong>Systematic reviews of patient-reported outcome measures (PROMs) are important tools to select the most suitable PROM for a study or clinical application. Conducting these reviews is challenging, and the quality of these reviews needs to be improved. We updated the COSMIN guideline for systematic reviews of PROMs, including the COSMIN Risk of Bias checklist, and the COSMIN criteria for good measurement properties.</p><p><strong>Methods: </strong>Adaptations to the methodology were based on our experience with applying the COSMIN guideline, through discussions among the authors, and results from two related Delphi studies.</p><p><strong>Results: </strong>The updated guideline places more emphasis on key aspects that are often missing or sub optimally conducted in published systematic reviews of PROMs, such as formulating a well-defined research question and developing a comprehensive search strategy, assessing risk of bias, applying criteria for good measurement properties, summarizing results, and grading the quality of the evidence. We also stress the importance of evaluating the measurement properties of each subscale of a PROM separately and evaluating content validity of all included PROMs.</p><p><strong>Conclusion: </strong>The quality of systematic reviews of PROMs can be improved by using this updated version of the COSMIN guideline for systematic reviews of PROMs. Improved quality will lead to better PROM selection and increased standardization of PROM use.</p>","PeriodicalId":20748,"journal":{"name":"Quality of Life Research","volume":null,"pages":null},"PeriodicalIF":3.3,"publicationDate":"2024-08-28","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142093693","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}