Pub Date : 2025-01-23DOI: 10.1007/s11136-025-03901-6
Kieran Sweeney, Kristine Bissenbakker, Volkert Siersma, Alexandra Jønsson, Eddie Donaghy, David Henderson, Stewart W Mercer, John Brandt Brodersen
Purpose: MMQ1 is a Danish-language patient-reported outcome measure (PROM) for quality of life (QOL) in people with multiple long-term conditions (MLTC). It measures needs-based QOL across six scales: Physical ability, Concerns and worries, Limitations in daily life, Social life, Personal finances and Self-image. There is currently no such measure available in English. This study aimed to translate and validate MMQ1 for use in the United Kingdom.
Methods: Translation used a two-panel method (expert panel: n = 5; and lay panel: n = 6). Content validity was assessed via cognitive interviews (n = 6). A postal survey of 2,753 patients with MLTC recruited through eight GP practices in Scotland included EQ-5D-5L and ICE-CAP as comparator measures alongside MMQ1. Classical test theory psychometric analysis of survey responses followed the International Society for Quality of Life Research minimum reporting standards.
Results: Translation resulted in an English-language MMQ1 with good face validity. Cognitive interviews established good content validity. 597 survey responses were received (response rate 22%). Good internal consistency reliability and concurrent validity were demonstrated. Confirmatory factor analysis showed acceptable fit to the six-scales of MMQ1. Five of the six scales also demonstrated good discriminative ability for detecting clinically meaningful differences in QOL. However, inter-item and inter-scale correlations suggested item redundancy and scale overlap.
Conclusion: The English-language MMQ1 demonstrated adequate psychometric properties using classical test theory. Further validation using Rasch analysis is planned, and may help optimise and abbreviate the measure. This PROM has the potential to improve the measurement of QOL in MLTC research, including trials.
{"title":"The Multimorbidity Questionnaire (MMQ1): English translation and validation of a Danish patient reported outcome measure for quality of life in people with multiple long-term conditions in a cross-sectional survey.","authors":"Kieran Sweeney, Kristine Bissenbakker, Volkert Siersma, Alexandra Jønsson, Eddie Donaghy, David Henderson, Stewart W Mercer, John Brandt Brodersen","doi":"10.1007/s11136-025-03901-6","DOIUrl":"https://doi.org/10.1007/s11136-025-03901-6","url":null,"abstract":"<p><strong>Purpose: </strong>MMQ1 is a Danish-language patient-reported outcome measure (PROM) for quality of life (QOL) in people with multiple long-term conditions (MLTC). It measures needs-based QOL across six scales: Physical ability, Concerns and worries, Limitations in daily life, Social life, Personal finances and Self-image. There is currently no such measure available in English. This study aimed to translate and validate MMQ1 for use in the United Kingdom.</p><p><strong>Methods: </strong>Translation used a two-panel method (expert panel: n = 5; and lay panel: n = 6). Content validity was assessed via cognitive interviews (n = 6). A postal survey of 2,753 patients with MLTC recruited through eight GP practices in Scotland included EQ-5D-5L and ICE-CAP as comparator measures alongside MMQ1. Classical test theory psychometric analysis of survey responses followed the International Society for Quality of Life Research minimum reporting standards.</p><p><strong>Results: </strong>Translation resulted in an English-language MMQ1 with good face validity. Cognitive interviews established good content validity. 597 survey responses were received (response rate 22%). Good internal consistency reliability and concurrent validity were demonstrated. Confirmatory factor analysis showed acceptable fit to the six-scales of MMQ1. Five of the six scales also demonstrated good discriminative ability for detecting clinically meaningful differences in QOL. However, inter-item and inter-scale correlations suggested item redundancy and scale overlap.</p><p><strong>Conclusion: </strong>The English-language MMQ1 demonstrated adequate psychometric properties using classical test theory. Further validation using Rasch analysis is planned, and may help optimise and abbreviate the measure. This PROM has the potential to improve the measurement of QOL in MLTC research, including trials.</p>","PeriodicalId":20748,"journal":{"name":"Quality of Life Research","volume":" ","pages":""},"PeriodicalIF":3.3,"publicationDate":"2025-01-23","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143024354","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 : 2025-01-22DOI: 10.1007/s11136-025-03890-6
Kate H Marshall, Yves d'Udekem, David S Winlaw, Diana Zannino, David S Celermajer, Karen Eagleson, Ajay J Iyengar, Dominica Zentner, Rachael Cordina, Gary F Sholler, Susan R Woolfenden, Nadine A Kasparian
Purpose: To examine global and health-related quality of life (QOL) among parents of individuals with Fontan physiology and determine associations with sociodemographic, parent and child-related health, psychological, and relational factors.
Methods: Parents participating in the Australian and New Zealand Fontan Registry (ANZFR) QOL Study (N = 151, Parent Mean age = 47.9 ± 10.2 years, age range: 31.6-79.6 years, 66% women; child Mean age = 16.3 ± 8.8, age range: 6.9-48.7 years, 40% female) completed a series of validated measures. Health-related QOL was assessed using the PedsQL 4.0 Core Generic Scales for adults and global QOL was assessed using a visual analogue scale (0-10).
Results: Most parents (81%) reported good global QOL (≥ 6), consistent with broader population trends. Nearly one-third of parents (28%) reported at-risk health-related QOL (based on total PedsQL scores) with physical functioning most affected (44%). Psychological factors, including psychological stress and sense of coherence, emerged as the strongest correlates of global and health-related QOL, explaining an additional 16 to 30% of the variance (using marginal R2). Final models explained 35 and 57% and of the variance in global and health-related QOL, respectively (marginal R2). Relational factors, including perceived social support and family functioning contributed minimally when analyzed alongside psychological variables.
Conclusion: While parents of individuals with Fontan physiology report good global QOL, challenges in health-related QOL exist. We identified key psychological, sociodemographic, and health-related factors associated with parental QOL outcomes. These data may aid early identification of physical and psychosocial difficulties and guide targeted health resource allocation for this population.
{"title":"Wellbeing and quality of life among parents of individuals with Fontan physiology.","authors":"Kate H Marshall, Yves d'Udekem, David S Winlaw, Diana Zannino, David S Celermajer, Karen Eagleson, Ajay J Iyengar, Dominica Zentner, Rachael Cordina, Gary F Sholler, Susan R Woolfenden, Nadine A Kasparian","doi":"10.1007/s11136-025-03890-6","DOIUrl":"https://doi.org/10.1007/s11136-025-03890-6","url":null,"abstract":"<p><strong>Purpose: </strong>To examine global and health-related quality of life (QOL) among parents of individuals with Fontan physiology and determine associations with sociodemographic, parent and child-related health, psychological, and relational factors.</p><p><strong>Methods: </strong>Parents participating in the Australian and New Zealand Fontan Registry (ANZFR) QOL Study (N = 151, Parent Mean age = 47.9 ± 10.2 years, age range: 31.6-79.6 years, 66% women; child Mean age = 16.3 ± 8.8, age range: 6.9-48.7 years, 40% female) completed a series of validated measures. Health-related QOL was assessed using the PedsQL 4.0 Core Generic Scales for adults and global QOL was assessed using a visual analogue scale (0-10).</p><p><strong>Results: </strong>Most parents (81%) reported good global QOL (≥ 6), consistent with broader population trends. Nearly one-third of parents (28%) reported at-risk health-related QOL (based on total PedsQL scores) with physical functioning most affected (44%). Psychological factors, including psychological stress and sense of coherence, emerged as the strongest correlates of global and health-related QOL, explaining an additional 16 to 30% of the variance (using marginal R<sup>2</sup>). Final models explained 35 and 57% and of the variance in global and health-related QOL, respectively (marginal R<sup>2</sup>). Relational factors, including perceived social support and family functioning contributed minimally when analyzed alongside psychological variables.</p><p><strong>Conclusion: </strong>While parents of individuals with Fontan physiology report good global QOL, challenges in health-related QOL exist. We identified key psychological, sociodemographic, and health-related factors associated with parental QOL outcomes. These data may aid early identification of physical and psychosocial difficulties and guide targeted health resource allocation for this population.</p>","PeriodicalId":20748,"journal":{"name":"Quality of Life Research","volume":" ","pages":""},"PeriodicalIF":3.3,"publicationDate":"2025-01-22","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143009954","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}
Aim: To identify instruments used to measure patient-reported outcomes after LT, and critically evaluate their measurement properties.
Methods: Five online databases were searched to find English-language LT-specific PROMs from their inception to October 2024. Studies describing the development or validation of PROMs were included. Two reviewers extracted and synthesized information about the characteristics of the studies and instruments. The methodological quality of the included studies was evaluated using the Consensus-Based Standards for the Selection of Health Measurement Instrument (COSMIN). Two reviewers independently evaluated the methodological quality of the included instruments.
Results: A total of 17 studies involving 14 PROMs met the inclusion criteria and were included in this review. The included PROMs assessed a range of outcomes, including health-related quality of life, therapeutic adherence, symptom experience, social participation, feeling for the donor, satisfaction and informational needs. None of the 14 scales reported the ten boxes of measurement properties outlined by the COSMIN. Among all the measurement properties, content validity from expert (64.3%, 9/14) and internal consistency (57.1%, 8/14) were the two most frequently measured attributes. Less than half of the studies evaluated the content validity from the patients' perspective. Four studies evaluated structural validity using exploratory factor analysis, but no study conducted confirmatory factor analysis or used IRT method to measure the structural validity.
Conclusion: This systematic review provided an overall picture and detailed analysis of LT-specific PROMs, and highlighted the paucity of well-developed and validated instruments. Further studies are urgently needed, both in terms of exploring patients' health concerns after LT and validating the instruments.
Reporting method: This study is reported according to the PRISMA Statement.
{"title":"Patient-reported outcome measures (PROMs) used among liver transplant recipients: a systematic review and methodological quality appraisal.","authors":"Qi Zhang, Xiao Chen, YiChen Kang, JingXian Yu, YuXia Zhang","doi":"10.1007/s11136-025-03893-3","DOIUrl":"https://doi.org/10.1007/s11136-025-03893-3","url":null,"abstract":"<p><strong>Aim: </strong>To identify instruments used to measure patient-reported outcomes after LT, and critically evaluate their measurement properties.</p><p><strong>Methods: </strong>Five online databases were searched to find English-language LT-specific PROMs from their inception to October 2024. Studies describing the development or validation of PROMs were included. Two reviewers extracted and synthesized information about the characteristics of the studies and instruments. The methodological quality of the included studies was evaluated using the Consensus-Based Standards for the Selection of Health Measurement Instrument (COSMIN). Two reviewers independently evaluated the methodological quality of the included instruments.</p><p><strong>Results: </strong>A total of 17 studies involving 14 PROMs met the inclusion criteria and were included in this review. The included PROMs assessed a range of outcomes, including health-related quality of life, therapeutic adherence, symptom experience, social participation, feeling for the donor, satisfaction and informational needs. None of the 14 scales reported the ten boxes of measurement properties outlined by the COSMIN. Among all the measurement properties, content validity from expert (64.3%, 9/14) and internal consistency (57.1%, 8/14) were the two most frequently measured attributes. Less than half of the studies evaluated the content validity from the patients' perspective. Four studies evaluated structural validity using exploratory factor analysis, but no study conducted confirmatory factor analysis or used IRT method to measure the structural validity.</p><p><strong>Conclusion: </strong>This systematic review provided an overall picture and detailed analysis of LT-specific PROMs, and highlighted the paucity of well-developed and validated instruments. Further studies are urgently needed, both in terms of exploring patients' health concerns after LT and validating the instruments.</p><p><strong>Reporting method: </strong>This study is reported according to the PRISMA Statement.</p>","PeriodicalId":20748,"journal":{"name":"Quality of Life Research","volume":" ","pages":""},"PeriodicalIF":3.3,"publicationDate":"2025-01-20","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143009847","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 : 2025-01-20DOI: 10.1007/s11136-025-03896-0
Aksel Paulsen, Ane Djuv, Ingvild Dalen
Purpose: Clinical cut-offs like minimum clinically important improvement (MCII) and patient acceptable symptom state (PASS) increase the interpretability of patient reported outcome measures (PROMs), but cut-off estimates vary considerably, clouding a clear definition of a successful surgical outcome. We report estimates of MCII and PASS following hip- and knee arthroplasty using multiple methods and compare the different estimation methods.
Methods: Elective hip or knee arthroplasty patients who underwent the regular pre- and postoperative assessments 2014-2018 were included. The generic EQ-5D-5L and either the Hip or Knee disability/injury and Osteoarthritis Outcome Score (HOOS/KOOS) were used. MCII and PASS were estimated based on multiple estimation techniques.
Results: Distributions were skewed, with up to 95% being acceptable according to anchor questions. MCII estimates for HOOS/KOOS Pain ranged 21-60/10-47, with fewest in-sample misclassifications for the lowest cut-offs, provided by the 75th percentile approach. PASS estimates for HOOS/KOOS Pain ranged 84-93/78-91, for EQ-5D Index/EQ-VAS 0.87-0.92/66-79 (for hip), and 0.79-0.88/66-76 (for knee), with fewest misclassifications for the 75th percentile approach (hip) and Pythagoras approach (knee). The 75th percentile approach was the approach most often giving MCII estimates below the minimal detectable change (MDC).
Conclusions: We report new one-year estimates of MCII and PASS of HOOS, KOOS and EQ-5D subscales following hip- and knee arthroplasty. Estimates varied considerably when using different anchors and estimation techniques. Overall, the 75th percentile approach had fewest misclassifications, and had the lowest thresholds for the MCII estimations, but which were often below the MDC.
{"title":"Clinical cut-offs for hip- and knee arthroplasty outcome - minimal clinically important improvement (MCII) and patient acceptable symptom state (PASS) of patient-reported outcome measures (PROM).","authors":"Aksel Paulsen, Ane Djuv, Ingvild Dalen","doi":"10.1007/s11136-025-03896-0","DOIUrl":"https://doi.org/10.1007/s11136-025-03896-0","url":null,"abstract":"<p><strong>Purpose: </strong>Clinical cut-offs like minimum clinically important improvement (MCII) and patient acceptable symptom state (PASS) increase the interpretability of patient reported outcome measures (PROMs), but cut-off estimates vary considerably, clouding a clear definition of a successful surgical outcome. We report estimates of MCII and PASS following hip- and knee arthroplasty using multiple methods and compare the different estimation methods.</p><p><strong>Methods: </strong>Elective hip or knee arthroplasty patients who underwent the regular pre- and postoperative assessments 2014-2018 were included. The generic EQ-5D-5L and either the Hip or Knee disability/injury and Osteoarthritis Outcome Score (HOOS/KOOS) were used. MCII and PASS were estimated based on multiple estimation techniques.</p><p><strong>Results: </strong>Distributions were skewed, with up to 95% being acceptable according to anchor questions. MCII estimates for HOOS/KOOS Pain ranged 21-60/10-47, with fewest in-sample misclassifications for the lowest cut-offs, provided by the 75th percentile approach. PASS estimates for HOOS/KOOS Pain ranged 84-93/78-91, for EQ-5D Index/EQ-VAS 0.87-0.92/66-79 (for hip), and 0.79-0.88/66-76 (for knee), with fewest misclassifications for the 75th percentile approach (hip) and Pythagoras approach (knee). The 75th percentile approach was the approach most often giving MCII estimates below the minimal detectable change (MDC).</p><p><strong>Conclusions: </strong>We report new one-year estimates of MCII and PASS of HOOS, KOOS and EQ-5D subscales following hip- and knee arthroplasty. Estimates varied considerably when using different anchors and estimation techniques. Overall, the 75th percentile approach had fewest misclassifications, and had the lowest thresholds for the MCII estimations, but which were often below the MDC.</p>","PeriodicalId":20748,"journal":{"name":"Quality of Life Research","volume":" ","pages":""},"PeriodicalIF":3.3,"publicationDate":"2025-01-20","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143010569","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 : 2025-01-17DOI: 10.1007/s11136-025-03891-5
Kirsten A Nyrop, Allison M Deal, Coral H Aman, Hyman B Muss, Bryce B Reeve
Background: As patient-reported symptoms are increasingly incorporated into routine clinical practice and captured in electronic medical records these data can be used to conduct health-related quality of life research studies. This study compares symptom reports from the Patient-Reported Outcomes version of the Common Terminology Criteria for Adverse Events (PRO-CTCAE) and its precursor patient reported symptom monitoring (PRSM) (hereafter PRSM/PRO-CTCAE) with multi-item patient-reported outcome (PRO) scales for fatigue (Functional Assessment of Chronic Illness Therapy/FACIT-Fatigue) and depression and anxiety (Mental Health Index/MHI).
Methods: This is a secondary analysis of data collected from women with early breast cancer (Stage I-III) scheduled for chemotherapy who completed PRSM/PRO-CTCAE, FACIT-Fatigue, and MHI scales pre- and post-chemotherapy. Spearman correlation coefficients estimated the magnitude and direction of correlations between measures (convergent validity). For each symptom, patients were then categorized based on who improved, stayed the same, or worsened on the composite PRSM/PRO-CTCAE score, and changes in scores on the PRO scales were compared.
Results: In a sample of 374 women, mean age was 57 years (SD 12.6) with 76% White. PRSM/PRO-CTCAE fatigue measures were strongly correlated with FACIT-Fatigue total scale and had mixed moderate to strong correlation for individual items within the FACIT-Fatigue scale. PRSM/PRO-CTCAE Sad and Anxiety measures were strongly correlated with MHI-Depression and MHI-Anxiety total scales, respectively, and had mixed moderate to strong correlation with individual items within the MHI subscales. PRSM/PRO-CTCAE pre-post changes in symptom scores mirrored pre-post changes in FACIT-Fatigue and MHI subscales.
{"title":"Comparison of patient-reported symptoms with multi-item patient-reported outcome measures of fatigue, anxiety, and depression in the clinical care of women undergoing chemotherapy for early breast cancer.","authors":"Kirsten A Nyrop, Allison M Deal, Coral H Aman, Hyman B Muss, Bryce B Reeve","doi":"10.1007/s11136-025-03891-5","DOIUrl":"https://doi.org/10.1007/s11136-025-03891-5","url":null,"abstract":"<p><strong>Background: </strong>As patient-reported symptoms are increasingly incorporated into routine clinical practice and captured in electronic medical records these data can be used to conduct health-related quality of life research studies. This study compares symptom reports from the Patient-Reported Outcomes version of the Common Terminology Criteria for Adverse Events (PRO-CTCAE) and its precursor patient reported symptom monitoring (PRSM) (hereafter PRSM/PRO-CTCAE) with multi-item patient-reported outcome (PRO) scales for fatigue (Functional Assessment of Chronic Illness Therapy/FACIT-Fatigue) and depression and anxiety (Mental Health Index/MHI).</p><p><strong>Methods: </strong>This is a secondary analysis of data collected from women with early breast cancer (Stage I-III) scheduled for chemotherapy who completed PRSM/PRO-CTCAE, FACIT-Fatigue, and MHI scales pre- and post-chemotherapy. Spearman correlation coefficients estimated the magnitude and direction of correlations between measures (convergent validity). For each symptom, patients were then categorized based on who improved, stayed the same, or worsened on the composite PRSM/PRO-CTCAE score, and changes in scores on the PRO scales were compared.</p><p><strong>Results: </strong>In a sample of 374 women, mean age was 57 years (SD 12.6) with 76% White. PRSM/PRO-CTCAE fatigue measures were strongly correlated with FACIT-Fatigue total scale and had mixed moderate to strong correlation for individual items within the FACIT-Fatigue scale. PRSM/PRO-CTCAE Sad and Anxiety measures were strongly correlated with MHI-Depression and MHI-Anxiety total scales, respectively, and had mixed moderate to strong correlation with individual items within the MHI subscales. PRSM/PRO-CTCAE pre-post changes in symptom scores mirrored pre-post changes in FACIT-Fatigue and MHI subscales.</p>","PeriodicalId":20748,"journal":{"name":"Quality of Life Research","volume":" ","pages":""},"PeriodicalIF":3.3,"publicationDate":"2025-01-17","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143010571","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 : 2025-01-15DOI: 10.1007/s11136-024-03886-8
Ludvig Daae Bjørndal, Ragnhild Bang Nes, Ziada Ayorech, Olav Vassend, Espen Røysamb
{"title":"Correction: Multiple social factors are associated with wellbeing when accounting for shared genetic and environmental confounding.","authors":"Ludvig Daae Bjørndal, Ragnhild Bang Nes, Ziada Ayorech, Olav Vassend, Espen Røysamb","doi":"10.1007/s11136-024-03886-8","DOIUrl":"10.1007/s11136-024-03886-8","url":null,"abstract":"","PeriodicalId":20748,"journal":{"name":"Quality of Life Research","volume":" ","pages":""},"PeriodicalIF":3.3,"publicationDate":"2025-01-15","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142984611","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}
Purpose: To investigate whether surgery is more effective than follow-up in reducing psychological distress for patients with observable indeterminate pulmonary nodules (IPNs) and to assess if psychological distress can serve as a potential surgical indication for IPNs.
Methods: This prospective observational study included 341 patients with abnormal psychometric results, as measured by the Hospital Anxiety and Depression Scale (HADS). Of these, 262 patients opted for follow-up and 79 chose surgery. Initial psychological assessments (HADS1) were conducted at enrollment following nodule detection, with a second assessment (HADS2) one year later. A comparative analysis of dynamic psychological changes (ΔHADS: HADS2-HADS1) between the follow-up and surgical groups was performed.
Results: Both groups showed reductions in HADS-A [- 3 (IQR, - 7 to - 1) for follow-up and - 3 (IQR, - 6 to - 1) for surgery] and HADS-D scores [- 2 (IQR, - 4 to 0) for follow-up and - 3 (IQR, - 7 to 0) for surgery]. Univariate analysis revealed that the surgical group had a significantly greater reduction in HADS-D scores compared to the follow-up group (Z = - 2.08, P = 0.037), but there were no significant differences in the changes in HADS-A scores between the groups (Z = - 1.04, P = 0.300). However, in multivariable analysis, surgery did not significantly improve the alleviation of depressive symptoms compared to follow-up (β = - 0.72, 95% CI: - 1.57 to 0.14, P = 0.101). Within the surgical group, female patients reported less relief from anxiety than male patients (Z = - 2.32, P = 0.021), and symptomatic patients experienced less relief from both anxiety (Z = - 2.14, P = 0.032) and depression (Z = - 3.01, P = 0.003).
Conclusions: Surgery does not provide additional psychological benefits over follow-up. This study does not support using psychological distress as a criterion for surgical intervention in IPNs from a psychological perspective. Trial registry ClinicalTrials.gov (NCT04857333).
{"title":"Impact of surgery versus follow-up on psychological distress in patients with indeterminate pulmonary nodules: A prospective observational study.","authors":"Junhan Wu, Weitao Zhuang, Rixin Chen, Haijie Xu, Zijie Li, Zihua Lan, Xin Xia, Zhe He, Shaopeng Li, Cheng Deng, Wei Xu, Qiuling Shi, Yong Tang, Guibin Qiao","doi":"10.1007/s11136-024-03876-w","DOIUrl":"https://doi.org/10.1007/s11136-024-03876-w","url":null,"abstract":"<p><strong>Purpose: </strong>To investigate whether surgery is more effective than follow-up in reducing psychological distress for patients with observable indeterminate pulmonary nodules (IPNs) and to assess if psychological distress can serve as a potential surgical indication for IPNs.</p><p><strong>Methods: </strong>This prospective observational study included 341 patients with abnormal psychometric results, as measured by the Hospital Anxiety and Depression Scale (HADS). Of these, 262 patients opted for follow-up and 79 chose surgery. Initial psychological assessments (HADS1) were conducted at enrollment following nodule detection, with a second assessment (HADS2) one year later. A comparative analysis of dynamic psychological changes (ΔHADS: HADS2-HADS1) between the follow-up and surgical groups was performed.</p><p><strong>Results: </strong>Both groups showed reductions in HADS-A [- 3 (IQR, - 7 to - 1) for follow-up and - 3 (IQR, - 6 to - 1) for surgery] and HADS-D scores [- 2 (IQR, - 4 to 0) for follow-up and - 3 (IQR, - 7 to 0) for surgery]. Univariate analysis revealed that the surgical group had a significantly greater reduction in HADS-D scores compared to the follow-up group (Z = - 2.08, P = 0.037), but there were no significant differences in the changes in HADS-A scores between the groups (Z = - 1.04, P = 0.300). However, in multivariable analysis, surgery did not significantly improve the alleviation of depressive symptoms compared to follow-up (β = - 0.72, 95% CI: - 1.57 to 0.14, P = 0.101). Within the surgical group, female patients reported less relief from anxiety than male patients (Z = - 2.32, P = 0.021), and symptomatic patients experienced less relief from both anxiety (Z = - 2.14, P = 0.032) and depression (Z = - 3.01, P = 0.003).</p><p><strong>Conclusions: </strong>Surgery does not provide additional psychological benefits over follow-up. This study does not support using psychological distress as a criterion for surgical intervention in IPNs from a psychological perspective. Trial registry ClinicalTrials.gov (NCT04857333).</p>","PeriodicalId":20748,"journal":{"name":"Quality of Life Research","volume":" ","pages":""},"PeriodicalIF":3.3,"publicationDate":"2025-01-15","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142984612","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 : 2025-01-08DOI: 10.1007/s11136-024-03882-y
Marco Viola, Carlotta Sacerdote, Giovannino Ciccone, Edoardo Donarelli, Manolis Kogevinas, Assunta Rasulo, Anna Toscano, Eva Pagano, Rosalba Rosato
Purpose: Prior evidence suggests that patients' Health Related Quality of Life (HRQoL) worsens after COVID-19. This study aimed to investigate HRQoL in Italian patients post-hospitalization for COVID-19, focusing on changes in physical and mental HRQoL over time since COVID-19 diagnosis.
Methods: A cohort of patients hospitalized for COVID-19 at Molinette Hospital in Turin, Italy, was contacted post-discharge to assess HRQoL using the SF-36 questionnaire. Patients completed the questionnaire only once at a different time since diagnosis. This design allowed for the analysis of responses up to three years after diagnosis. Measured scores were compared with normative data from the Italian population using z-scores. HRQoL differences by gender, comorbidities, and self-perception of health status before and after COVID-19 were tested. The effect of time since diagnosis on physical (PCS) and mental (MCS) SF-36 scores was analysed using multiple linear models and stratified analyses.
Results: A total of 601 patients completed the questionnaire. HRQoL was significantly lower compared to the normative Italian population. Men and individuals without comorbidities had better HRQoL, and self-perceptions of health status before and after COVID-19 influenced HRQoL. Time since diagnosis was associated with improved PCS, but MCS remained unaffected. Mental health declined in patients using anxiolytics post-COVID-19, and increasing age negatively affected physical health.
Conclusion: Our findings highlight the impact of time since diagnosis on HRQoL post-COVID-19 in an Italian population and suggest the need for further investigation into the pandemic's effects on HRQoL. Physicians should implement measures to improve mental HRQoL post-COVID-19.
{"title":"Long‑term physical and mental Health-Related Quality of Life in Italian patients post COVID-19 hospitalisation.","authors":"Marco Viola, Carlotta Sacerdote, Giovannino Ciccone, Edoardo Donarelli, Manolis Kogevinas, Assunta Rasulo, Anna Toscano, Eva Pagano, Rosalba Rosato","doi":"10.1007/s11136-024-03882-y","DOIUrl":"https://doi.org/10.1007/s11136-024-03882-y","url":null,"abstract":"<p><strong>Purpose: </strong>Prior evidence suggests that patients' Health Related Quality of Life (HRQoL) worsens after COVID-19. This study aimed to investigate HRQoL in Italian patients post-hospitalization for COVID-19, focusing on changes in physical and mental HRQoL over time since COVID-19 diagnosis.</p><p><strong>Methods: </strong>A cohort of patients hospitalized for COVID-19 at Molinette Hospital in Turin, Italy, was contacted post-discharge to assess HRQoL using the SF-36 questionnaire. Patients completed the questionnaire only once at a different time since diagnosis. This design allowed for the analysis of responses up to three years after diagnosis. Measured scores were compared with normative data from the Italian population using z-scores. HRQoL differences by gender, comorbidities, and self-perception of health status before and after COVID-19 were tested. The effect of time since diagnosis on physical (PCS) and mental (MCS) SF-36 scores was analysed using multiple linear models and stratified analyses.</p><p><strong>Results: </strong>A total of 601 patients completed the questionnaire. HRQoL was significantly lower compared to the normative Italian population. Men and individuals without comorbidities had better HRQoL, and self-perceptions of health status before and after COVID-19 influenced HRQoL. Time since diagnosis was associated with improved PCS, but MCS remained unaffected. Mental health declined in patients using anxiolytics post-COVID-19, and increasing age negatively affected physical health.</p><p><strong>Conclusion: </strong>Our findings highlight the impact of time since diagnosis on HRQoL post-COVID-19 in an Italian population and suggest the need for further investigation into the pandemic's effects on HRQoL. Physicians should implement measures to improve mental HRQoL post-COVID-19.</p>","PeriodicalId":20748,"journal":{"name":"Quality of Life Research","volume":" ","pages":""},"PeriodicalIF":3.3,"publicationDate":"2025-01-08","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142953982","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 : 2025-01-08DOI: 10.1007/s11136-024-03874-y
Xiaohang Mei, Joseph C Cappelleri, Jinxiang Hu
Purpose: Patient-Reported Outcomes (PROs) are widely used in clinical trials, epidemiological research, quality of life (QOL) studies, routine clinical care, and medical surveillance. The Patient Reported Outcomes Measurement Information System (PROMIS) is a system of reliable and standardized measures of PROs developed with Item Response Theory (IRT) using latent scores. Power estimation is critical to clinical trials and research designs. However, in clinical trials with PROs as endpoints, observed scores are often used to calculate power rather than latent scores.
Methods: In this paper, we conducted a series of simulations to compare the power obtained with IRT latent scores, including Bayesian IRT, Frequentist IRT, and observed scores, focusing on small sample size common in pilot studies and Phase I/II trials. Taking the PROMIS depression measures as an example, we simulated data and estimated power for two-armed clinical trials manipulating the following factors: sample size, effect size, and number of items. We also examined how misspecification of effect size affected power estimation.
Results: Our results showed that the Bayesian IRT, which incorporated prior information into latent score estimation, yielded the highest power, especially when sample size was small. The effect of misspecification diminished as sample size increased.
Conclusion: For power estimation in two-armed clinical trials with standardized PRO endpoints, if a medium effect size or larger is expected, we recommend BIRT simulation with well-grounded informative priors and a total sample size of at least 40.
{"title":"Bayesian item response theory to estimate power in clinical trials with patient-reported outcomes as endpoints.","authors":"Xiaohang Mei, Joseph C Cappelleri, Jinxiang Hu","doi":"10.1007/s11136-024-03874-y","DOIUrl":"https://doi.org/10.1007/s11136-024-03874-y","url":null,"abstract":"<p><strong>Purpose: </strong>Patient-Reported Outcomes (PROs) are widely used in clinical trials, epidemiological research, quality of life (QOL) studies, routine clinical care, and medical surveillance. The Patient Reported Outcomes Measurement Information System (PROMIS) is a system of reliable and standardized measures of PROs developed with Item Response Theory (IRT) using latent scores. Power estimation is critical to clinical trials and research designs. However, in clinical trials with PROs as endpoints, observed scores are often used to calculate power rather than latent scores.</p><p><strong>Methods: </strong>In this paper, we conducted a series of simulations to compare the power obtained with IRT latent scores, including Bayesian IRT, Frequentist IRT, and observed scores, focusing on small sample size common in pilot studies and Phase I/II trials. Taking the PROMIS depression measures as an example, we simulated data and estimated power for two-armed clinical trials manipulating the following factors: sample size, effect size, and number of items. We also examined how misspecification of effect size affected power estimation.</p><p><strong>Results: </strong>Our results showed that the Bayesian IRT, which incorporated prior information into latent score estimation, yielded the highest power, especially when sample size was small. The effect of misspecification diminished as sample size increased.</p><p><strong>Conclusion: </strong>For power estimation in two-armed clinical trials with standardized PRO endpoints, if a medium effect size or larger is expected, we recommend BIRT simulation with well-grounded informative priors and a total sample size of at least 40.</p>","PeriodicalId":20748,"journal":{"name":"Quality of Life Research","volume":" ","pages":""},"PeriodicalIF":3.3,"publicationDate":"2025-01-08","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142953981","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 : 2025-01-05DOI: 10.1007/s11136-024-03888-6
Bethany L Brockbank, Greg S J Dewar, Richard J D Hewitt, Colin C R Butler, Jo Wray
Background: Aortopexy is a procedure to reduce pressure on the trachea in children with severe tracheomalacia. Health-related quality of life (HRQoL) has not been studied in children who have undergone aortopexy; we aimed to explore parents' perceptions of their child's HRQoL before and after aortopexy.
Methods: Parents of children < 18 years who underwent aortopexy at a quaternary specialist centre were purposively sampled and invited to participate in a semi-structured interview. Data were analysed using reflective thematic and content analysis.
Results: Eight interviews (23-56 min) were completed with parents whose children were aged 1.9-12.1 years. HRQoL is explored through themes: a challenging diagnosis, lifechanging, and an understanding of limitations. Parents struggled to obtain a diagnosis of tracheomalacia while their child experienced serious symptoms. Aortopexy was seen as life-saving and allowed a return to family life. Airway safety, having their health and achieving a better quality of life were important outcomes of the procedure.
Conclusion: Quality of life following aortopexy was perceived to be greatly improved. Children still struggled in aspects of their life, however families worked within their child's limitations, appreciating the life-saving nature of the intervention, and accepting the complexities of their child's comorbidities. Holistic interventions are important for those at risk of poor HRQoL outcomes.
{"title":"\"I think her life got so much better\": parents' perceptions of children's quality of life following treatment of tracheomalacia with aortopexy.","authors":"Bethany L Brockbank, Greg S J Dewar, Richard J D Hewitt, Colin C R Butler, Jo Wray","doi":"10.1007/s11136-024-03888-6","DOIUrl":"https://doi.org/10.1007/s11136-024-03888-6","url":null,"abstract":"<p><strong>Background: </strong>Aortopexy is a procedure to reduce pressure on the trachea in children with severe tracheomalacia. Health-related quality of life (HRQoL) has not been studied in children who have undergone aortopexy; we aimed to explore parents' perceptions of their child's HRQoL before and after aortopexy.</p><p><strong>Methods: </strong>Parents of children < 18 years who underwent aortopexy at a quaternary specialist centre were purposively sampled and invited to participate in a semi-structured interview. Data were analysed using reflective thematic and content analysis.</p><p><strong>Results: </strong>Eight interviews (23-56 min) were completed with parents whose children were aged 1.9-12.1 years. HRQoL is explored through themes: a challenging diagnosis, lifechanging, and an understanding of limitations. Parents struggled to obtain a diagnosis of tracheomalacia while their child experienced serious symptoms. Aortopexy was seen as life-saving and allowed a return to family life. Airway safety, having their health and achieving a better quality of life were important outcomes of the procedure.</p><p><strong>Conclusion: </strong>Quality of life following aortopexy was perceived to be greatly improved. Children still struggled in aspects of their life, however families worked within their child's limitations, appreciating the life-saving nature of the intervention, and accepting the complexities of their child's comorbidities. Holistic interventions are important for those at risk of poor HRQoL outcomes.</p>","PeriodicalId":20748,"journal":{"name":"Quality of Life Research","volume":" ","pages":""},"PeriodicalIF":3.3,"publicationDate":"2025-01-05","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142932495","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}