Pub Date : 2024-09-16DOI: 10.1186/s12874-024-02332-0
Phil Edwards, Chloe Perkins
A decade ago paper questionnaires were more common in epidemiology than those administered online, but increasing Internet access may have changed this. Researchers planning to use a self-administered questionnaire should know whether response rates to questionnaires administered electronically differ to those of questionnaires administered by post. We analysed trials included in a recently updated Cochrane Review to answer this question. We exported data of randomised controlled trials included in three comparisons in the Cochrane Review that had evaluated hypotheses relevant to our research objective and imported them into Stata for a series of meta-analyses not conducted in the Cochrane review. We pooled odds ratios for response using random effects meta-analyses. We explored causes of heterogeneity among study results using subgroups. We assessed evidence for reporting bias using Harbord’s modified test for small-study effects. Twenty-seven trials (66,118 participants) evaluated the effect on response of an electronic questionnaire compared with postal. Results were heterogeneous (I-squared = 98%). There was evidence for biased (greater) effect estimates in studies at high risk of bias; A synthesis of studies at low risk of bias indicates that response was increased (OR = 1.43; 95% CI 1.08–1.89) using postal questionnaires. Ten trials (39,523 participants) evaluated the effect of providing a choice of mode (postal or electronic) compared to an electronic questionnaire only. Response was increased with a choice of mode (OR = 1.63; 95% CI 1.18–2.26). Eight trials (20,909 participants) evaluated the effect of a choice of mode (electronic or postal) compared to a postal questionnaire only. There was no evidence for an effect on response of a choice of mode compared with postal only (OR = 0.94; 95% CI 0.86–1.02). Postal questionnaires should be used in preference to, or offered in addition to, electronic modes.
十年前，纸质问卷在流行病学中比在线问卷更常见，但互联网接入的不断增加可能已经改变了这一状况。计划使用自填式问卷的研究人员应该了解电子问卷的回复率与邮寄问卷的回复率是否存在差异。为了回答这个问题，我们分析了最近更新的 Cochrane 综述中所包含的试验。我们导出了 Cochrane 综述中三项比较试验中包含的随机对照试验数据，这些试验评估了与我们的研究目标相关的假设，我们将这些数据导入到 Stata 中，进行了一系列 Cochrane 综述中未进行的荟萃分析。我们使用随机效应荟萃分析对反应的几率比进行了汇总。我们通过分组探讨了研究结果之间存在异质性的原因。我们使用哈伯德修正的小规模研究效应检验法评估了报告偏倚的证据。27项试验（66118名参与者）评估了电子问卷与邮寄问卷相比对回复率的影响。结果不尽相同（I 方 = 98%）。有证据表明，在偏倚风险较高的研究中，效果估计值存在偏倚（较大）；对偏倚风险较低的研究进行的综合分析表明，使用邮寄问卷的应答率有所提高（OR = 1.43; 95% CI 1.08-1.89）。十项试验（39,523 名参与者）评估了提供方式选择（邮寄或电子）与仅提供电子问卷的效果。选择调查方式可提高回复率（OR = 1.63；95% CI 1.18-2.26）。八项试验（20,909 名参与者）评估了与仅使用邮寄问卷相比，选择电子问卷或邮寄问卷的效果。没有证据表明，与仅采用邮寄方式相比，选择电子或邮寄方式会对回复率产生影响（OR = 0.94; 95% CI 0.86-1.02）。邮寄问卷应优先于电子问卷，或作为电子问卷的补充。
{"title":"Response is increased using postal rather than electronic questionnaires – new results from an updated Cochrane Systematic Review","authors":"Phil Edwards, Chloe Perkins","doi":"10.1186/s12874-024-02332-0","DOIUrl":"https://doi.org/10.1186/s12874-024-02332-0","url":null,"abstract":"A decade ago paper questionnaires were more common in epidemiology than those administered online, but increasing Internet access may have changed this. Researchers planning to use a self-administered questionnaire should know whether response rates to questionnaires administered electronically differ to those of questionnaires administered by post. We analysed trials included in a recently updated Cochrane Review to answer this question. We exported data of randomised controlled trials included in three comparisons in the Cochrane Review that had evaluated hypotheses relevant to our research objective and imported them into Stata for a series of meta-analyses not conducted in the Cochrane review. We pooled odds ratios for response using random effects meta-analyses. We explored causes of heterogeneity among study results using subgroups. We assessed evidence for reporting bias using Harbord’s modified test for small-study effects. Twenty-seven trials (66,118 participants) evaluated the effect on response of an electronic questionnaire compared with postal. Results were heterogeneous (I-squared = 98%). There was evidence for biased (greater) effect estimates in studies at high risk of bias; A synthesis of studies at low risk of bias indicates that response was increased (OR = 1.43; 95% CI 1.08–1.89) using postal questionnaires. Ten trials (39,523 participants) evaluated the effect of providing a choice of mode (postal or electronic) compared to an electronic questionnaire only. Response was increased with a choice of mode (OR = 1.63; 95% CI 1.18–2.26). Eight trials (20,909 participants) evaluated the effect of a choice of mode (electronic or postal) compared to a postal questionnaire only. There was no evidence for an effect on response of a choice of mode compared with postal only (OR = 0.94; 95% CI 0.86–1.02). Postal questionnaires should be used in preference to, or offered in addition to, electronic modes.","PeriodicalId":9114,"journal":{"name":"BMC Medical Research Methodology","volume":null,"pages":null},"PeriodicalIF":4.0,"publicationDate":"2024-09-16","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142248518","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 : 2024-09-16DOI: 10.1186/s12874-024-02326-y
Samuel I. Watson, Eleni Gkini, Jon Bishop, Katie Scandrett, Indra Napit, Richard J. Lilford
Experimental studies of wound healing often use survival analysis and time to event outcomes or differences in wound area at a specific time point. However, these methods do not use a potentially large number of observations made over the course of a trial and may be inefficient. A model-based approach can leverage all trial data, but there is little guidance on appropriate models and functional forms to describe wound healing. We derive a general statistical model and review a wide range of plausible mathematical models to describe wound healing. We identify a range of possible derived estimands and their derivation from the models. Using data from a trial of an intervention to promote ulcer healing in patients affected by leprosy that included three measurement methods repeated across the course of the study, we compare the goodness-of-fit of the models using a range of methods and estimate treatment effects and healing rate functions with the best-fitting models. Overall, we included 5,581 ulcer measurements of 1,578 unique images from 130 patients. We examined the performance of a range of models. The square root, log square root, and log quadratic models were the best fitting models across all outcome measurement methods. The estimated treatment effects magnitude and sign varied by time post-randomisation, model type, and outcome type, but across all models there was little evidence of effectiveness. The estimated effects were significantly more precise than non-parametric alternatives. For example, estimated differences from the three outcome measurements at 42-days post-randomisation were − 0.01 cm2 (-0.77, 0.74), -0.44 cm2 (-1.64, 0.76), and 0.11 cm2 (-0.87, 1.08) using a non-parametric method versus − 0.03 cm2 (-0.14, 0.06), 0.06 cm2 (-0.05, 0.17), and 0.03 cm2 (-0.07, 0.17) using a square-root model. Model-based analyses can dramatically improve the precision of estimates but care must be taken to carefully compare and select the best fitting models. The (log) square-root model is strongly recommended reflecting advice from a century ago.
{"title":"Modelling wound area in studies of wound healing interventions","authors":"Samuel I. Watson, Eleni Gkini, Jon Bishop, Katie Scandrett, Indra Napit, Richard J. Lilford","doi":"10.1186/s12874-024-02326-y","DOIUrl":"https://doi.org/10.1186/s12874-024-02326-y","url":null,"abstract":"Experimental studies of wound healing often use survival analysis and time to event outcomes or differences in wound area at a specific time point. However, these methods do not use a potentially large number of observations made over the course of a trial and may be inefficient. A model-based approach can leverage all trial data, but there is little guidance on appropriate models and functional forms to describe wound healing. We derive a general statistical model and review a wide range of plausible mathematical models to describe wound healing. We identify a range of possible derived estimands and their derivation from the models. Using data from a trial of an intervention to promote ulcer healing in patients affected by leprosy that included three measurement methods repeated across the course of the study, we compare the goodness-of-fit of the models using a range of methods and estimate treatment effects and healing rate functions with the best-fitting models. Overall, we included 5,581 ulcer measurements of 1,578 unique images from 130 patients. We examined the performance of a range of models. The square root, log square root, and log quadratic models were the best fitting models across all outcome measurement methods. The estimated treatment effects magnitude and sign varied by time post-randomisation, model type, and outcome type, but across all models there was little evidence of effectiveness. The estimated effects were significantly more precise than non-parametric alternatives. For example, estimated differences from the three outcome measurements at 42-days post-randomisation were − 0.01 cm2 (-0.77, 0.74), -0.44 cm2 (-1.64, 0.76), and 0.11 cm2 (-0.87, 1.08) using a non-parametric method versus − 0.03 cm2 (-0.14, 0.06), 0.06 cm2 (-0.05, 0.17), and 0.03 cm2 (-0.07, 0.17) using a square-root model. Model-based analyses can dramatically improve the precision of estimates but care must be taken to carefully compare and select the best fitting models. The (log) square-root model is strongly recommended reflecting advice from a century ago.","PeriodicalId":9114,"journal":{"name":"BMC Medical Research Methodology","volume":null,"pages":null},"PeriodicalIF":4.0,"publicationDate":"2024-09-16","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142248521","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 : 2024-09-13DOI: 10.1186/s12874-024-02313-3
Wei-Chun Hsu, Aaron Crowley, Craig S. Parzynski
Evaluating outcome reliability is critical in real-world evidence studies. Overall survival is a common outcome in these studies; however, its capture in real-world data (RWD) sources is often incomplete and supplemented with linked mortality information from external sources. Conflicting recommendations exist for censoring overall survival in real-world evidence studies. This simulation study aimed to understand the impact of different censoring methods on estimating median survival and log hazard ratios when external mortality information is partially captured. We used Monte Carlo simulation to emulate a non-randomized comparative effectiveness study of two treatments with RWD from electronic health records and linked external mortality data. We simulated the time to death, the time to last database activity, and the time to data cutoff. Death events after the last database activity were attributed to linked external mortality data and randomly set to missing to reflect the sensitivity of contemporary real-world data sources. Two censoring schemes were evaluated: (1) censoring at the last activity date and (2) censoring at the end of data availability (data cutoff) without an observed death. We assessed the performance of each method in estimating median survival and log hazard ratios using bias, coverage, variance, and rejection rate under varying amounts of incomplete mortality information and varying treatment effects, length of follow-up, and sample size. When mortality information was fully captured, median survival estimates were unbiased when censoring at data cutoff and underestimated when censoring at the last activity. When linked mortality information was missing, censoring at the last activity date underestimated the median survival, while censoring at the data cutoff overestimated it. As missing linked mortality information increased, bias decreased when censoring at the last activity date and increased when censoring at data cutoff. Researchers should consider the completeness of linked external mortality information when choosing how to censor the analysis of overall survival using RWD. Substantial bias in median survival estimates can occur if an inappropriate censoring scheme is selected. We advocate for RWD providers to perform validation studies of their mortality data and publish their findings to inform methodological decisions better.
{"title":"The impact of different censoring methods for analyzing survival using real-world data with linked mortality information: a simulation study","authors":"Wei-Chun Hsu, Aaron Crowley, Craig S. Parzynski","doi":"10.1186/s12874-024-02313-3","DOIUrl":"https://doi.org/10.1186/s12874-024-02313-3","url":null,"abstract":"Evaluating outcome reliability is critical in real-world evidence studies. Overall survival is a common outcome in these studies; however, its capture in real-world data (RWD) sources is often incomplete and supplemented with linked mortality information from external sources. Conflicting recommendations exist for censoring overall survival in real-world evidence studies. This simulation study aimed to understand the impact of different censoring methods on estimating median survival and log hazard ratios when external mortality information is partially captured. We used Monte Carlo simulation to emulate a non-randomized comparative effectiveness study of two treatments with RWD from electronic health records and linked external mortality data. We simulated the time to death, the time to last database activity, and the time to data cutoff. Death events after the last database activity were attributed to linked external mortality data and randomly set to missing to reflect the sensitivity of contemporary real-world data sources. Two censoring schemes were evaluated: (1) censoring at the last activity date and (2) censoring at the end of data availability (data cutoff) without an observed death. We assessed the performance of each method in estimating median survival and log hazard ratios using bias, coverage, variance, and rejection rate under varying amounts of incomplete mortality information and varying treatment effects, length of follow-up, and sample size. When mortality information was fully captured, median survival estimates were unbiased when censoring at data cutoff and underestimated when censoring at the last activity. When linked mortality information was missing, censoring at the last activity date underestimated the median survival, while censoring at the data cutoff overestimated it. As missing linked mortality information increased, bias decreased when censoring at the last activity date and increased when censoring at data cutoff. Researchers should consider the completeness of linked external mortality information when choosing how to censor the analysis of overall survival using RWD. Substantial bias in median survival estimates can occur if an inappropriate censoring scheme is selected. We advocate for RWD providers to perform validation studies of their mortality data and publish their findings to inform methodological decisions better.","PeriodicalId":9114,"journal":{"name":"BMC Medical Research Methodology","volume":null,"pages":null},"PeriodicalIF":4.0,"publicationDate":"2024-09-13","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142203380","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 : 2024-09-13DOI: 10.1186/s12874-024-02325-z
Nan Wang, Weiyi Zhuang, Zhen Ran, Pinxi Wan, Jian Fu
The aim of this study is to analyze the trend of acute onset of chronic cor pulmonale at Chenggong Hospital of Kunming Yan’an Hospital between January 2018 and December 2022.Additionally, the study will compare the application of the ARIMA model and Holt-Winters model in predicting the number of chronic cor pulmonale cases. The data on chronic cor pulmonale cases from 2018 to 2022 were collected from the electronic medical records system of Chenggong Hospital of Kunming Yan’an Hospital. The ARIMA and Holt-Winters models were constructed using monthly case numbers from January 2018 to December 2022 as training data. The performance of the model was tested using the monthly number of cases from January 2023 to December 2023 as the test set. The number of acute onset of chronic cor pulmonale in Chenggong Hospital of Kunming Yan’an Hospital exhibited a downward trend overall from 2018 to 2022. There were more cases in winter and spring, with peaks observed in November to December and January of the following year. The optimal ARIMA model was determined to be ARIMA (0,1,1) (0,1,1)12, while for the Holt-Winters model, the optimal choice was the Holt-Winters multiplicative model. It was found that the Holt-Winters multiplicative model yielded the lowest error. The Holt-Winters multiplicative model predicts better accuracy. The diagnosis of acute onset of chronic cor pulmonale is related to many risk factors, therefore, when using temporal models to fit and predict the data, we must consider such factors’ influence and try to incorporate them into the models.
{"title":"Prediction of acute onset of chronic cor pulmonale: comparative analysis of Holt-Winters exponential smoothing and ARIMA model","authors":"Nan Wang, Weiyi Zhuang, Zhen Ran, Pinxi Wan, Jian Fu","doi":"10.1186/s12874-024-02325-z","DOIUrl":"https://doi.org/10.1186/s12874-024-02325-z","url":null,"abstract":"The aim of this study is to analyze the trend of acute onset of chronic cor pulmonale at Chenggong Hospital of Kunming Yan’an Hospital between January 2018 and December 2022.Additionally, the study will compare the application of the ARIMA model and Holt-Winters model in predicting the number of chronic cor pulmonale cases. The data on chronic cor pulmonale cases from 2018 to 2022 were collected from the electronic medical records system of Chenggong Hospital of Kunming Yan’an Hospital. The ARIMA and Holt-Winters models were constructed using monthly case numbers from January 2018 to December 2022 as training data. The performance of the model was tested using the monthly number of cases from January 2023 to December 2023 as the test set. The number of acute onset of chronic cor pulmonale in Chenggong Hospital of Kunming Yan’an Hospital exhibited a downward trend overall from 2018 to 2022. There were more cases in winter and spring, with peaks observed in November to December and January of the following year. The optimal ARIMA model was determined to be ARIMA (0,1,1) (0,1,1)12, while for the Holt-Winters model, the optimal choice was the Holt-Winters multiplicative model. It was found that the Holt-Winters multiplicative model yielded the lowest error. The Holt-Winters multiplicative model predicts better accuracy. The diagnosis of acute onset of chronic cor pulmonale is related to many risk factors, therefore, when using temporal models to fit and predict the data, we must consider such factors’ influence and try to incorporate them into the models.","PeriodicalId":9114,"journal":{"name":"BMC Medical Research Methodology","volume":null,"pages":null},"PeriodicalIF":4.0,"publicationDate":"2024-09-13","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142203379","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 : 2024-09-13DOI: 10.1186/s12874-024-02323-1
Imke Schilling, Ansgar Gerhardus
There has been a growing push to involve patients in clinical research, shifting from conducting research on, about, or for them to conducting it with them. Two arguments advocate for this approach, known as Patient and Public Involvement (PPI): to improve research quality, appropriateness, relevance, and credibility by including patients’ diverse perspectives, and to use PPI to empower patients and democratize research for more equity in research and healthcare. However, while empowerment is a core objective, it is often not clear what is meant by empowerment in the context of PPI in clinical research. This vacancy can lead to insecurities for both patients and researchers and a disconnect between the rhetoric of empowerment in PPI and the reality of its practice in clinical trials. Thus, clarifying the understanding of empowerment within PPI in clinical research is essential to ensure that involvement does not become tokenistic and depletes patients’ capacity to advocate for their rights and needs. We explored the historical roots of empowerment, primarily emerging from mid-20th century social movements like feminism and civil rights and reflected the conceptual roots of empowerment from diverse fields to better understand the (potential) role of empowerment in PPI in clinical research including its possibilities and limitations. Common themes of empowerment in PPI and other fields are participation, challenging power structures, valuing diverse perspectives, and promoting collaboration. On the other hand, themes such as contextual differences in the empowerment objectives, the relationship between empowerment and scientific demands, research expertise, and power asymmetries mark a clear distinction from empowerment in other fields. PPI offers potential for patient empowerment in clinical trials, even when its primary goal may be research quality. Elements like participation, sharing opinions, and active engagement can contribute to patient empowerment. Nonetheless, some expectations tied to empowerment might not be met within the constraints of clinical research. To empower patients, stakeholders must be explicit about what empowerment means in their research, engage in transparent communication about its realistic scope, and continuously reflect on how empowerment can be fostered and sustained within the research process.
让患者参与临床研究的呼声日益高涨，研究工作已从针对患者、关于患者或为患者进行的研究转变为与患者共同进行的研究。这种被称为 "患者与公众参与"（Patient and Public Involvement，PPI）的方法有两个主张：一是通过纳入患者的不同观点来提高研究质量、适当性、相关性和可信度；二是利用 PPI 增强患者的能力，实现研究民主化，从而提高研究和医疗保健的公平性。然而，虽然赋权是一项核心目标，但在临床研究中的患者参与计划中，赋权的含义往往并不明确。这种空缺可能会导致患者和研究人员的不安全感，并使 PPI 中的授权说辞与临床试验中的实际做法脱节。因此，厘清临床研究中 PPI 对授权的理解至关重要，这样才能确保参与不会成为象征性的，也不会削弱患者维护自身权利和需求的能力。我们探讨了赋权的历史渊源，主要源于 20 世纪中叶的社会运动，如女权主义和民权运动，并从不同领域反映了赋权的概念渊源，从而更好地理解赋权在临床研究 PPI 中的（潜在）作用，包括其可能性和局限性。PPI 和其他领域赋权的共同主题是参与、挑战权力结构、重视不同观点和促进合作。另一方面，赋权目标的背景差异、赋权与科学需求之间的关系、研究专长和权力不对称等主题也标志着与其他领域赋权的明显区别。PPI 为临床试验中的患者赋权提供了潜力，即使其主要目标可能是提高研究质量。参与、分享意见和积极参与等要素都有助于增强患者的能力。然而，在临床研究的限制条件下，一些与赋权相关的期望可能无法实现。为了增强患者的能力，利益相关者必须明确增强能力在研究中的意义，就其现实范围进行透明的沟通，并不断反思如何在研究过程中促进和维持增强能力。
{"title":"Is this really Empowerment? Enhancing our understanding of empowerment in patient and public involvement within clinical research","authors":"Imke Schilling, Ansgar Gerhardus","doi":"10.1186/s12874-024-02323-1","DOIUrl":"https://doi.org/10.1186/s12874-024-02323-1","url":null,"abstract":"There has been a growing push to involve patients in clinical research, shifting from conducting research on, about, or for them to conducting it with them. Two arguments advocate for this approach, known as Patient and Public Involvement (PPI): to improve research quality, appropriateness, relevance, and credibility by including patients’ diverse perspectives, and to use PPI to empower patients and democratize research for more equity in research and healthcare. However, while empowerment is a core objective, it is often not clear what is meant by empowerment in the context of PPI in clinical research. This vacancy can lead to insecurities for both patients and researchers and a disconnect between the rhetoric of empowerment in PPI and the reality of its practice in clinical trials. Thus, clarifying the understanding of empowerment within PPI in clinical research is essential to ensure that involvement does not become tokenistic and depletes patients’ capacity to advocate for their rights and needs. We explored the historical roots of empowerment, primarily emerging from mid-20th century social movements like feminism and civil rights and reflected the conceptual roots of empowerment from diverse fields to better understand the (potential) role of empowerment in PPI in clinical research including its possibilities and limitations. Common themes of empowerment in PPI and other fields are participation, challenging power structures, valuing diverse perspectives, and promoting collaboration. On the other hand, themes such as contextual differences in the empowerment objectives, the relationship between empowerment and scientific demands, research expertise, and power asymmetries mark a clear distinction from empowerment in other fields. PPI offers potential for patient empowerment in clinical trials, even when its primary goal may be research quality. Elements like participation, sharing opinions, and active engagement can contribute to patient empowerment. Nonetheless, some expectations tied to empowerment might not be met within the constraints of clinical research. To empower patients, stakeholders must be explicit about what empowerment means in their research, engage in transparent communication about its realistic scope, and continuously reflect on how empowerment can be fostered and sustained within the research process.","PeriodicalId":9114,"journal":{"name":"BMC Medical Research Methodology","volume":null,"pages":null},"PeriodicalIF":4.0,"publicationDate":"2024-09-13","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142203349","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}
Clinical trials play a crucial role in biomedical research, and it is important to register them in public registries to ensure transparency and prevent research waste. In this study, we wished to determine what steps need to be taken to identify every clinical trial run in India that has been registered in any of the (non-Indian) World Health Organization-recognised primary registries. Of the 16 registries, we studied all except that of the European Union, which will be studied separately. Two methodologies were employed for each registry, except for four that did not facilitate one or the other method. Methodology A involved downloading all the records in a registry and querying them. Methodology B involved conducting a search via the registry website. Only four registries provided consistent results with both methodologies. Seven registries had different results from the two methodologies. Of these, in four cases, in Methodology A one field indicated that the study ran in India, while another indicated otherwise. The above-mentioned ambiguities should be addressed by the concerned registries. Overall, this study reinforces the need for improved data accuracy and transparency in clinical trial registries and emphasizes the importance of resolving complications faced by users while navigating the registries. Ensuring accurate and comprehensive registration of clinical trials is essential for meta-research and the use of such data by a variety of stakeholders.
临床试验在生物医学研究中发挥着至关重要的作用，在公共登记处登记临床试验对于确保透明度和防止研究浪费非常重要。在这项研究中，我们希望确定需要采取哪些措施来识别在印度进行的、已在世界卫生组织认可的任何（非印度）主要登记处登记的每项临床试验。在这 16 个登记处中，我们研究了除欧盟登记处以外的所有登记处，欧盟登记处将单独研究。每个登记处都采用了两种方法，只有四个登记处没有采用其中一种方法。方法 A 包括下载登记册中的所有记录并进行查询。方法 B 涉及通过登记处网站进行查询。只有四个登记处使用两种方法提供了一致的结果。有 7 个登记册在使用两种方法时结果不同。其中，有四个登记册在 "方法 A "中的一个字段表示研究在印度进行，而另一个字段则表示不在印度进行。相关登记册应解决上述模糊问题。总之，本研究强化了提高临床试验注册数据准确性和透明度的必要性，并强调了解决用户在注册过程中遇到的复杂问题的重要性。确保临床试验登记的准确性和全面性对元研究和各利益相关方使用这些数据至关重要。
{"title":"Identifying trials run in India that are registered in other clinical trial registries: a cross-sectional study","authors":"Rishima Borah, Anwesha Dhal Samanta, Jaishree Mendiratta, Manish Mishra, Gayatri Saberwal","doi":"10.1186/s12874-024-02336-w","DOIUrl":"https://doi.org/10.1186/s12874-024-02336-w","url":null,"abstract":"Clinical trials play a crucial role in biomedical research, and it is important to register them in public registries to ensure transparency and prevent research waste. In this study, we wished to determine what steps need to be taken to identify every clinical trial run in India that has been registered in any of the (non-Indian) World Health Organization-recognised primary registries. Of the 16 registries, we studied all except that of the European Union, which will be studied separately. Two methodologies were employed for each registry, except for four that did not facilitate one or the other method. Methodology A involved downloading all the records in a registry and querying them. Methodology B involved conducting a search via the registry website. Only four registries provided consistent results with both methodologies. Seven registries had different results from the two methodologies. Of these, in four cases, in Methodology A one field indicated that the study ran in India, while another indicated otherwise. The above-mentioned ambiguities should be addressed by the concerned registries. Overall, this study reinforces the need for improved data accuracy and transparency in clinical trial registries and emphasizes the importance of resolving complications faced by users while navigating the registries. Ensuring accurate and comprehensive registration of clinical trials is essential for meta-research and the use of such data by a variety of stakeholders.","PeriodicalId":9114,"journal":{"name":"BMC Medical Research Methodology","volume":null,"pages":null},"PeriodicalIF":4.0,"publicationDate":"2024-09-12","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142203381","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 : 2024-09-12DOI: 10.1186/s12874-024-02300-8
Myriam Dagher, Sawsan Abdulrahim, Berthe Abi Zeid, Maia Sieverding
Sexual and reproductive empowerment (SRE) is an important determinant of women’s and girls’ health yet measuring it is complex due to cultural and domain-specific variations. This study describes the process of adapting an SRE scale consisting of four domains (self-efficacy; future orientation; social support; and safety) and testing its psychometric properties among Arabic speaking adolescent girls in Lebanon. An SRE scale developed in a Western context was adapted in four steps: (1) reviewing the scale and selecting culturally appropriate domains for translation to standard Arabic; (2) conducting cognitive interviews with 30 11-17-year-old adolescent girls in Lebanon; (3) administering the scale to 339 refugee adolescent girls who participated in an early marriage intervention; and (4) conducting confirmatory factor analysis (CFA) on the data to assess the scale’s psychometric properties. The original model for the 13-item, four-domain adapted scale demonstrated poor fit in CFA. After iteratively removing two items, scale properties were improved, albeit were not optimal. The validity and reliability results for the self-efficacy domain were acceptable. Cognitive interview data revealed that Arab adolescent girls understood self-efficacy in relational terms, recognizing that autonomous decision-making is not necessarily favored but is influenced by parents and family. This study presents an effort to customize an SRE scale for use in studies on the health of adolescent girls in an Arab cultural context. Findings from cognitive interviews highlight the importance of taking into consideration relationality in adolescent sexual and reproductive decision-making. The self-efficacy domain in the adapted scale demonstrates acceptable psychometric properties and is recommended for use in health studies to capture SRE.
{"title":"Adaptation and psychometric assessment of a sexual and reproductive empowerment scale in Arabic among refugee and non-refugee adolescent girls","authors":"Myriam Dagher, Sawsan Abdulrahim, Berthe Abi Zeid, Maia Sieverding","doi":"10.1186/s12874-024-02300-8","DOIUrl":"https://doi.org/10.1186/s12874-024-02300-8","url":null,"abstract":"Sexual and reproductive empowerment (SRE) is an important determinant of women’s and girls’ health yet measuring it is complex due to cultural and domain-specific variations. This study describes the process of adapting an SRE scale consisting of four domains (self-efficacy; future orientation; social support; and safety) and testing its psychometric properties among Arabic speaking adolescent girls in Lebanon. An SRE scale developed in a Western context was adapted in four steps: (1) reviewing the scale and selecting culturally appropriate domains for translation to standard Arabic; (2) conducting cognitive interviews with 30 11-17-year-old adolescent girls in Lebanon; (3) administering the scale to 339 refugee adolescent girls who participated in an early marriage intervention; and (4) conducting confirmatory factor analysis (CFA) on the data to assess the scale’s psychometric properties. The original model for the 13-item, four-domain adapted scale demonstrated poor fit in CFA. After iteratively removing two items, scale properties were improved, albeit were not optimal. The validity and reliability results for the self-efficacy domain were acceptable. Cognitive interview data revealed that Arab adolescent girls understood self-efficacy in relational terms, recognizing that autonomous decision-making is not necessarily favored but is influenced by parents and family. This study presents an effort to customize an SRE scale for use in studies on the health of adolescent girls in an Arab cultural context. Findings from cognitive interviews highlight the importance of taking into consideration relationality in adolescent sexual and reproductive decision-making. The self-efficacy domain in the adapted scale demonstrates acceptable psychometric properties and is recommended for use in health studies to capture SRE.","PeriodicalId":9114,"journal":{"name":"BMC Medical Research Methodology","volume":null,"pages":null},"PeriodicalIF":4.0,"publicationDate":"2024-09-12","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142203383","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 : 2024-09-12DOI: 10.1186/s12874-024-02328-w
Carmen Koschollek, Beate Gaertner, Julia Geerlings, Ronny Kuhnert, Elvira Mauz, Claudia Hövener
Germany is the second most common country of immigration after the US. However, people with own or familial history of migration are not represented proportionately to the population within public health monitoring and reporting. To bridge this data gap and enable differentiated analyses on migration and health, we conducted the health interview survey GEDA Fokus among adults with Croatian, Italian, Polish, Syrian, or Turkish citizenship living throughout Germany. The aim of this paper is to evaluate the effects of recruitment efforts regarding participation and sample composition. Data collection for this cross-sectional and multilingual survey took place between 11/2021 and 5/2022 utilizing a sequential mixed-mode design, including self-administered web- and paper-based questionnaires as well as face-to-face and telephone interviews. The gross sample (n = 33436; age range 18–79 years) was randomly drawn from the residents’ registers in 120 primary sampling units based on citizenship. Outcome rates according to the American Association for Public Opinion Research, the sample composition throughout the multistage recruitment process, utilization of survey modes, and questionnaire languages are presented. Overall, 6038 persons participated, which corresponded to a response rate of 18.4% (range: 13.8% for Turkish citizenship to 23.9% for Syrian citizenship). Home visits accounted for the largest single increase in response. During recruitment, more female, older, as well as participants with lower levels of education and income took part in the survey. People with physical health problems and less favourable health behaviour more often took part in the survey at a later stage, while participants with symptoms of depression or anxiety more often participated early. Utilization of survey modes and questionnaire languages differed by sociodemographic and migration-related characteristics, e.g. participants aged 50 years and above more often used paper- than web-based questionnaires and those with a shorter duration of residence more often used a translated questionnaire. Multiple contact attempts, including home visits and different survey languages, as well as offering different modes of survey administration, increased response rates and most likely reduced non-response bias. In order to adequately represent and include the diversifying population in public health monitoring, national public health institutes should tailor survey designs to meet the needs of different population groups considered hard to survey to enable their survey participation.
{"title":"Recruiting people with selected citizenships for the health interview survey GEDA Fokus throughout Germany: evaluation of recruitment efforts and recommendations for future research","authors":"Carmen Koschollek, Beate Gaertner, Julia Geerlings, Ronny Kuhnert, Elvira Mauz, Claudia Hövener","doi":"10.1186/s12874-024-02328-w","DOIUrl":"https://doi.org/10.1186/s12874-024-02328-w","url":null,"abstract":"Germany is the second most common country of immigration after the US. However, people with own or familial history of migration are not represented proportionately to the population within public health monitoring and reporting. To bridge this data gap and enable differentiated analyses on migration and health, we conducted the health interview survey GEDA Fokus among adults with Croatian, Italian, Polish, Syrian, or Turkish citizenship living throughout Germany. The aim of this paper is to evaluate the effects of recruitment efforts regarding participation and sample composition. Data collection for this cross-sectional and multilingual survey took place between 11/2021 and 5/2022 utilizing a sequential mixed-mode design, including self-administered web- and paper-based questionnaires as well as face-to-face and telephone interviews. The gross sample (n = 33436; age range 18–79 years) was randomly drawn from the residents’ registers in 120 primary sampling units based on citizenship. Outcome rates according to the American Association for Public Opinion Research, the sample composition throughout the multistage recruitment process, utilization of survey modes, and questionnaire languages are presented. Overall, 6038 persons participated, which corresponded to a response rate of 18.4% (range: 13.8% for Turkish citizenship to 23.9% for Syrian citizenship). Home visits accounted for the largest single increase in response. During recruitment, more female, older, as well as participants with lower levels of education and income took part in the survey. People with physical health problems and less favourable health behaviour more often took part in the survey at a later stage, while participants with symptoms of depression or anxiety more often participated early. Utilization of survey modes and questionnaire languages differed by sociodemographic and migration-related characteristics, e.g. participants aged 50 years and above more often used paper- than web-based questionnaires and those with a shorter duration of residence more often used a translated questionnaire. Multiple contact attempts, including home visits and different survey languages, as well as offering different modes of survey administration, increased response rates and most likely reduced non-response bias. In order to adequately represent and include the diversifying population in public health monitoring, national public health institutes should tailor survey designs to meet the needs of different population groups considered hard to survey to enable their survey participation.","PeriodicalId":9114,"journal":{"name":"BMC Medical Research Methodology","volume":null,"pages":null},"PeriodicalIF":4.0,"publicationDate":"2024-09-12","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142203382","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}
The prognosis, recurrence rates, and secondary prevention strategies varied significantly among different subtypes of acute ischemic stroke (AIS). Machine learning (ML) techniques can uncover intricate, non-linear relationships within medical data, enabling the identification of factors associated with etiological classification. However, there is currently a lack of research utilizing ML algorithms for predicting AIS etiology. We aimed to use interpretable ML algorithms to develop AIS etiology prediction models, identify critical factors in etiology classification, and enhance existing clinical categorization. This study involved patients with the Third China National Stroke Registry (CNSR-III). Nine models, which included Natural Gradient Boosting (NGBoost), Categorical Boosting (CatBoost), Extreme Gradient Boosting (XGBoost), Random Forest (RF), Light Gradient Boosting Machine (LGBM), Gradient Boosting Decision Tree (GBDT), Adaptive Boosting (AdaBoost), Support Vector Machine (SVM), and logistic regression (LR), were employed to predict large artery atherosclerosis (LAA), small vessel occlusion (SVO), and cardioembolism (CE) using an 80:20 randomly split training and test set. We designed an SFS-XGB with 10-fold cross-validation for feature selection. The primary evaluation metrics for the models included the area under the receiver operating characteristic curve (AUC) for discrimination and the Brier score (or calibration plots) for calibration. A total of 5,213 patients were included, comprising 2,471 (47.4%) with LAA, 2,153 (41.3%) with SVO, and 589 (11.3%) with CE. In both LAA and SVO models, the AUC values of the ML models were significantly higher than that of the LR model (P < 0.001). The optimal model for predicting SVO (AUC [RF model] = 0.932) outperformed the optimal LAA model (AUC [NGB model] = 0.917) and the optimal CE model (AUC [LGBM model] = 0.846). Each model displayed relatively satisfactory calibration. Further analysis showed that the optimal CE model could identify potential CE patients in the undetermined etiology (SUE) group, accounting for 1,900 out of 4,156 (45.7%). The ML algorithm effectively classified patients with LAA, SVO, and CE, demonstrating superior classification performance compared to the LR model. The optimal ML model can identify potential CE patients among SUE patients. These newly identified predictive factors may complement the existing etiological classification system, enabling clinicians to promptly categorize stroke patients’ etiology and initiate optimal strategies for secondary prevention.
不同亚型的急性缺血性脑卒中（AIS）在预后、复发率和二级预防策略方面差异很大。机器学习（ML）技术可以发现医疗数据中错综复杂的非线性关系，从而识别与病因分类相关的因素。然而，目前还缺乏利用 ML 算法预测 AIS 病因的研究。我们旨在利用可解释的 ML 算法开发 AIS 病因学预测模型，识别病因学分类的关键因素，并加强现有的临床分类。本研究涉及第三届中国卒中登记（CNSR-III）的患者。九种模型包括自然梯度提升（NGBoost）、分类提升（CatBoost）、极端梯度提升（XGBoost）、随机森林（RF）、轻梯度提升机（LGBM）、梯度提升决策树（GBDT）、采用自适应提升（AdaBoost）、支持向量机（SVM）和逻辑回归（LR）来预测大动脉粥样硬化（LAA）、小血管闭塞（SVO）和心肌栓塞（CE）：20 随机分配训练集和测试集。我们设计的 SFS-XGB 采用 10 倍交叉验证进行特征选择。模型的主要评估指标包括用于判别的接收者操作特征曲线下面积（AUC）和用于校准的布赖尔评分（或校准图）。共纳入了 5213 例患者，其中 LAA 患者 2471 例（47.4%），SVO 患者 2153 例（41.3%），CE 患者 589 例（11.3%）。在 LAA 和 SVO 模型中，ML 模型的 AUC 值均显著高于 LR 模型（P < 0.001）。预测 SVO 的最佳模型（AUC [RF 模型] = 0.932）优于最佳 LAA 模型（AUC [NGB 模型] = 0.917）和最佳 CE 模型（AUC [LGBM 模型] = 0.846）。每个模型都显示出相对令人满意的校准效果。进一步分析表明，最佳 CE 模型可以识别病因未定（SUE）组中潜在的 CE 患者，在 4156 例患者中占 1900 例（45.7%）。与 LR 模型相比，ML 算法能有效地对 LAA、SVO 和 CE 患者进行分类，显示出更优越的分类性能。最佳的 ML 模型可以在 SUE 患者中识别出潜在的 CE 患者。这些新发现的预测因素可以补充现有的病因分类系统，使临床医生能够及时对卒中患者的病因进行分类，并启动二级预防的最佳策略。
{"title":"Predictive etiological classification of acute ischemic stroke through interpretable machine learning algorithms: a multicenter, prospective cohort study","authors":"Siding Chen, Xiaomeng Yang, Hongqiu Gu, Yanzhao Wang, Zhe Xu, Yong Jiang, Yongjun Wang","doi":"10.1186/s12874-024-02331-1","DOIUrl":"https://doi.org/10.1186/s12874-024-02331-1","url":null,"abstract":"The prognosis, recurrence rates, and secondary prevention strategies varied significantly among different subtypes of acute ischemic stroke (AIS). Machine learning (ML) techniques can uncover intricate, non-linear relationships within medical data, enabling the identification of factors associated with etiological classification. However, there is currently a lack of research utilizing ML algorithms for predicting AIS etiology. We aimed to use interpretable ML algorithms to develop AIS etiology prediction models, identify critical factors in etiology classification, and enhance existing clinical categorization. This study involved patients with the Third China National Stroke Registry (CNSR-III). Nine models, which included Natural Gradient Boosting (NGBoost), Categorical Boosting (CatBoost), Extreme Gradient Boosting (XGBoost), Random Forest (RF), Light Gradient Boosting Machine (LGBM), Gradient Boosting Decision Tree (GBDT), Adaptive Boosting (AdaBoost), Support Vector Machine (SVM), and logistic regression (LR), were employed to predict large artery atherosclerosis (LAA), small vessel occlusion (SVO), and cardioembolism (CE) using an 80:20 randomly split training and test set. We designed an SFS-XGB with 10-fold cross-validation for feature selection. The primary evaluation metrics for the models included the area under the receiver operating characteristic curve (AUC) for discrimination and the Brier score (or calibration plots) for calibration. A total of 5,213 patients were included, comprising 2,471 (47.4%) with LAA, 2,153 (41.3%) with SVO, and 589 (11.3%) with CE. In both LAA and SVO models, the AUC values of the ML models were significantly higher than that of the LR model (P < 0.001). The optimal model for predicting SVO (AUC [RF model] = 0.932) outperformed the optimal LAA model (AUC [NGB model] = 0.917) and the optimal CE model (AUC [LGBM model] = 0.846). Each model displayed relatively satisfactory calibration. Further analysis showed that the optimal CE model could identify potential CE patients in the undetermined etiology (SUE) group, accounting for 1,900 out of 4,156 (45.7%). The ML algorithm effectively classified patients with LAA, SVO, and CE, demonstrating superior classification performance compared to the LR model. The optimal ML model can identify potential CE patients among SUE patients. These newly identified predictive factors may complement the existing etiological classification system, enabling clinicians to promptly categorize stroke patients’ etiology and initiate optimal strategies for secondary prevention.","PeriodicalId":9114,"journal":{"name":"BMC Medical Research Methodology","volume":null,"pages":null},"PeriodicalIF":4.0,"publicationDate":"2024-09-10","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142203395","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 : 2024-09-09DOI: 10.1186/s12874-024-02306-2
Stephanie Wied, Martin Posch, Ralf-Dieter Hilgers
In the context of clinical research, there is an increasing need for new study designs that help to incorporate already available data. With the help of historical controls, the existing information can be utilized to support the new study design, but of course, inclusion also carries the risk of bias in the study results. To combine historical and randomized controls we investigate the Fill-it-up-design, which in the first step checks the comparability of the historical and randomized controls performing an equivalence pre-test. If equivalence is confirmed, the historical control data will be included in the new RCT. If equivalence cannot be confirmed, the historical controls will not be considered at all and the randomization of the original study will be extended. We are investigating the performance of this study design in terms of type I error rate and power. We demonstrate how many patients need to be recruited in each of the two steps in the Fill-it-up-design and show that the family wise error rate of the design is kept at 5 $$%$$ . The maximum sample size of the Fill-it-up-design is larger than that of the single-stage design without historical controls and increases as the heterogeneity between the historical controls and the concurrent controls increases. The two-stage Fill-it-up-design represents a frequentist method for including historical control data for various study designs. As the maximum sample size of the design is larger, a robust prior belief is essential for its use. The design should therefore be seen as a way out in exceptional situations where a hybrid design is considered necessary.
{"title":"Evaluation of the Fill-it-up-design to use historical control data in randomized clinical trials with two arm parallel group design","authors":"Stephanie Wied, Martin Posch, Ralf-Dieter Hilgers","doi":"10.1186/s12874-024-02306-2","DOIUrl":"https://doi.org/10.1186/s12874-024-02306-2","url":null,"abstract":"In the context of clinical research, there is an increasing need for new study designs that help to incorporate already available data. With the help of historical controls, the existing information can be utilized to support the new study design, but of course, inclusion also carries the risk of bias in the study results. To combine historical and randomized controls we investigate the Fill-it-up-design, which in the first step checks the comparability of the historical and randomized controls performing an equivalence pre-test. If equivalence is confirmed, the historical control data will be included in the new RCT. If equivalence cannot be confirmed, the historical controls will not be considered at all and the randomization of the original study will be extended. We are investigating the performance of this study design in terms of type I error rate and power. We demonstrate how many patients need to be recruited in each of the two steps in the Fill-it-up-design and show that the family wise error rate of the design is kept at 5 $$%$$ . The maximum sample size of the Fill-it-up-design is larger than that of the single-stage design without historical controls and increases as the heterogeneity between the historical controls and the concurrent controls increases. The two-stage Fill-it-up-design represents a frequentist method for including historical control data for various study designs. As the maximum sample size of the design is larger, a robust prior belief is essential for its use. The design should therefore be seen as a way out in exceptional situations where a hybrid design is considered necessary.","PeriodicalId":9114,"journal":{"name":"BMC Medical Research Methodology","volume":null,"pages":null},"PeriodicalIF":4.0,"publicationDate":"2024-09-09","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142203396","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}
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