Pub Date : 2024-05-06DOI: 10.1136/bmjebm-2023-112492
Sitanshu Sekhar Kar, Parthibane Sivanantham, Vanessa Ravel, Abha Mehndiratta, Kirti Tyagi, Daniel A Ollendorf
Objective To assess the cost-effectiveness of emicizumab prophylaxis for patients having haemophilia A with inhibitors in the Indian context using an adaptive health technology assessment (aHTA) methodology. Design Economic evaluation using multiple approaches aimed at adjusting previously generated cost-effectiveness results based on (1) price differences only (‘simple’) and (2) differences in cost and expected treatment duration (‘moderate’) and differences in cost, inflation and life expectancy (‘complex’). Setting Typical haemophilia care in India. Participants Patients with haemophilia A and inhibitors. Intervention Emicizumab prophylaxis using two vial strengths (30 or 150 mg/mL) in comparison to no prophylaxis. Main outcome measures Adjusted incremental cost-effectiveness ratio (ICERa), incremental costs and incremental quality-adjusted life years associated with emicizumab prophylaxis from both the health system and societal perspectives. Results Using the simple ICER adjustment method, emicizumab prophylaxis resulted in potential cost savings from the payers’ perspective for both vial strengths in patients aged ≥12 and <12 years. However, from a societal perspective, emicizumab prophylaxis was not cost-effective. Using the moderate adjustment method, emicizumab prophylaxis showed potential cost saving from the health system perspective. The complex adjustment method also revealed cost savings for emicizumab prophylaxis from the health system and societal perspectives across different age groups. Conclusion We found that implementing emicizumab prophylaxis for patients with haemophilia A and inhibitors in India has the potential to result in cost savings. This study highlights the feasibility of using the expanded aHTA methodology for rapid evidence generation in the Indian context. However, it is crucial to address certain research gaps, including data limitations, challenges in translating international evidence to Indian context and associated uncertainties. Additionally, conducting a comprehensive budget impact analysis is necessary. These findings hold significant implications for decision-making regarding the potential provision of emicizumab prophylaxis through federal or/and state government-funded programmes and institutions in India. Data are available upon reasonable request. Data are available upon reasonable request to the corresponding author.
目的 采用适应性卫生技术评估 (aHTA) 方法，评估在印度对使用抑制剂的 A 型血友病患者进行埃米珠单抗预防治疗的成本效益。设计 采用多种方法进行经济评估，旨在根据（1）仅价格差异（"简单"）和（2）成本和预期治疗时间差异（"中等"）以及成本、通货膨胀和预期寿命差异（"复杂"）调整之前得出的成本效益结果。研究背景 印度典型的血友病治疗。参与者 A 型血友病患者和抑制剂患者。干预措施 埃米珠单抗两种剂量（30 或 150 毫克/毫升）的预防性治疗与无预防性治疗的比较。主要结果指标 从卫生系统和社会角度看与埃米珠单抗预防治疗相关的调整后增量成本效益比（ICERa）、增量成本和增量质量调整生命年。结果 采用简单的ICER调整方法，从支付方的角度来看，在年龄≥12岁和＜12岁的患者中，两种剂量的埃米珠单抗预防性治疗都有可能节约成本。然而，从社会角度来看，埃米珠单抗预防性治疗并不具有成本效益。采用中度调整法，从卫生系统的角度来看，埃米珠单抗预防性治疗有可能节约成本。复杂调整法也显示，从卫生系统和社会角度来看，不同年龄组的埃米珠单抗预防性治疗均可节约成本。结论 我们发现，在印度为血友病 A 和抑制剂患者实施埃米珠单抗预防治疗有可能节约成本。这项研究强调了在印度使用扩大的 aHTA 方法快速生成证据的可行性。然而，解决某些研究缺口至关重要，包括数据限制、将国际证据转化为印度国情所面临的挑战以及相关的不确定性。此外，有必要进行全面的预算影响分析。这些研究结果对印度通过联邦政府或/和邦政府资助的项目和机构提供埃米珠单抗预防性治疗的决策具有重要意义。如有合理要求，可提供数据。数据可向通讯作者索取。
{"title":"Cost-effectiveness of emicizumab prophylaxis for haemophilia A with inhibitors: an adaptive health technology assessment for the Indian setting","authors":"Sitanshu Sekhar Kar, Parthibane Sivanantham, Vanessa Ravel, Abha Mehndiratta, Kirti Tyagi, Daniel A Ollendorf","doi":"10.1136/bmjebm-2023-112492","DOIUrl":"https://doi.org/10.1136/bmjebm-2023-112492","url":null,"abstract":"Objective To assess the cost-effectiveness of emicizumab prophylaxis for patients having haemophilia A with inhibitors in the Indian context using an adaptive health technology assessment (aHTA) methodology. Design Economic evaluation using multiple approaches aimed at adjusting previously generated cost-effectiveness results based on (1) price differences only (‘simple’) and (2) differences in cost and expected treatment duration (‘moderate’) and differences in cost, inflation and life expectancy (‘complex’). Setting Typical haemophilia care in India. Participants Patients with haemophilia A and inhibitors. Intervention Emicizumab prophylaxis using two vial strengths (30 or 150 mg/mL) in comparison to no prophylaxis. Main outcome measures Adjusted incremental cost-effectiveness ratio (ICERa), incremental costs and incremental quality-adjusted life years associated with emicizumab prophylaxis from both the health system and societal perspectives. Results Using the simple ICER adjustment method, emicizumab prophylaxis resulted in potential cost savings from the payers’ perspective for both vial strengths in patients aged ≥12 and <12 years. However, from a societal perspective, emicizumab prophylaxis was not cost-effective. Using the moderate adjustment method, emicizumab prophylaxis showed potential cost saving from the health system perspective. The complex adjustment method also revealed cost savings for emicizumab prophylaxis from the health system and societal perspectives across different age groups. Conclusion We found that implementing emicizumab prophylaxis for patients with haemophilia A and inhibitors in India has the potential to result in cost savings. This study highlights the feasibility of using the expanded aHTA methodology for rapid evidence generation in the Indian context. However, it is crucial to address certain research gaps, including data limitations, challenges in translating international evidence to Indian context and associated uncertainties. Additionally, conducting a comprehensive budget impact analysis is necessary. These findings hold significant implications for decision-making regarding the potential provision of emicizumab prophylaxis through federal or/and state government-funded programmes and institutions in India. Data are available upon reasonable request. Data are available upon reasonable request to the corresponding author.","PeriodicalId":9059,"journal":{"name":"BMJ Evidence-Based Medicine","volume":"62 1","pages":""},"PeriodicalIF":5.8,"publicationDate":"2024-05-06","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140883738","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 : 2024-05-06DOI: 10.1136/bmjebm-2023-112518
Celeste McCracken, Zahra Raisi-Estabragh, Liliana Szabo, Michele Veldsman, Betty Raman, Anya Topiwala, Adriana Roca-Fernández, Masud Husain, Steffen E Petersen, Stefan Neubauer, Thomas E Nichols
Objectives Despite rising rates of multimorbidity, existing risk assessment tools are mostly limited to a single outcome of interest. This study tests the feasibility of producing multiple disease risk estimates with at least 70% discrimination (area under the receiver operating curve, AUROC) within the time and information constraints of the existing primary care health check framework. Design Observational prospective cohort study Setting UK Biobank. Participants 228 240 adults from the UK population. Interventions None. Main outcome measures Myocardial infarction, atrial fibrillation, heart failure, stroke, all-cause dementia, chronic kidney disease, fatty liver disease, alcoholic liver disease, liver cirrhosis and liver failure. Results Using a set of predictors easily gathered at the standard primary care health check (such as the National Health Service Health Check), we demonstrate that it is feasible to simultaneously produce risk estimates for multiple disease outcomes with AUROC of 70% or greater. These predictors can be entered once into a single form and produce risk scores for stroke (AUROC 0.727, 95% CI 0.713 to 0.740), all-cause dementia (0.823, 95% CI 0.810 to 0.836), myocardial infarction (0.785, 95% CI 0.775 to 0.795), atrial fibrillation (0.777, 95% CI 0.768 to 0.785), heart failure (0.828, 95% CI 0.818 to 0.838), chronic kidney disease (0.774, 95% CI 0.765 to 0.783), fatty liver disease (0.766, 95% CI 0.753 to 0.779), alcoholic liver disease (0.864, 95% CI 0.835 to 0.894), liver cirrhosis (0.763, 95% CI 0.734 to 0.793) and liver failure (0.746, 95% CI 0.695 to 0.796). Conclusions Easily collected diagnostics can be used to assess 10-year risk across multiple disease outcomes, without the need for specialist computing or invasive biomarkers. Such an approach could increase the utility of existing data and place multiorgan risk information at the fingertips of primary care providers, thus creating opportunities for longer-term multimorbidity prevention. Additional work is needed to validate whether these findings would hold in a larger, more representative cohort outside the UK Biobank. Data may be obtained from a third party and are not publicly available. This analysis was produced under UK Biobank Access Application 59867. The data in this study are owned by the UK Biobank (www. ukbiobank.ac.uk) and legal constraints do not permit public sharing of the data. The UK Biobank, however, is open to all bona fide researchers anywhere in the world. Thus, the data used in this communication can be easily and directly accessed by applying through the UK Biobank Access Management System (www.ukbiobank.ac.uk/ register-apply). Results from this study will be returned to UK Biobank according to their published returns policy.
目标 尽管多病发病率不断上升，但现有的风险评估工具大多局限于单一的相关结果。本研究测试了在现有初级保健健康检查框架的时间和信息限制条件下，生成至少具有 70% 识别率（接收器工作曲线下面积，AUROC）的多种疾病风险估计值的可行性。设计 观察性前瞻性队列研究 设置英国生物数据库。参与者 228 240 名来自英国的成年人。干预措施 无。主要结果指标 心肌梗死、心房颤动、心力衰竭、中风、全因痴呆、慢性肾病、脂肪肝、酒精肝、肝硬化和肝功能衰竭。结果 我们利用在标准初级保健健康检查（如国民健康服务健康检查）中很容易收集到的一组预测因子，证明了同时生成 AUROC 为 70% 或更高的多种疾病结果的风险估计是可行的。这些预测因子只需输入一份表格，就能得出中风（AUROC 为 0.727，95% CI 为 0.713 至 0.740）、全因痴呆（0.823，95% CI 为 0.810 至 0.836）、心肌梗塞（0.785，95% CI 为 0.775 至 0.795）、心房颤动（0.777，95% CI 为 0.768 至 0.785）、心力衰竭（0.828，95% CI 0.818 至 0.838）、慢性肾病（0.774，95% CI 0.765 至 0.783）、脂肪肝（0.766，95% CI 0.753 至 0.779）、酒精性肝病（0.864，95% CI 0.835 至 0.894）、肝硬化（0.763，95% CI 0.734 至 0.793）和肝衰竭（0.746，95% CI 0.695 至 0.796）。结论 容易收集到的诊断结果可用于评估多种疾病的 10 年风险，而无需专业计算或侵入性生物标记物。这种方法可以提高现有数据的效用，让初级保健提供者触手可及多器官风险信息，从而为长期多病预防创造机会。我们还需要做更多的工作，以验证这些发现是否能在英国生物库以外的更大规模、更具代表性的人群中成立。数据可能来自第三方，不对外公开。本分析由英国生物库访问申请 59867 生成。本研究中的数据归英国生物库（www. ukbiobank.ac.uk）所有，法律限制不允许公开共享数据。不过，英国生物库向世界上任何地方的所有善意研究人员开放。因此，只要通过英国生物库访问管理系统（www.ukbiobank.ac.uk/ register-apply）提出申请，就可以方便、直接地访问本通讯中使用的数据。本研究的结果将根据英国生物库公布的退还政策退还给英国生物库。
{"title":"Feasibility of multiorgan risk prediction with routinely collected diagnostics: a prospective cohort study in the UK Biobank","authors":"Celeste McCracken, Zahra Raisi-Estabragh, Liliana Szabo, Michele Veldsman, Betty Raman, Anya Topiwala, Adriana Roca-Fernández, Masud Husain, Steffen E Petersen, Stefan Neubauer, Thomas E Nichols","doi":"10.1136/bmjebm-2023-112518","DOIUrl":"https://doi.org/10.1136/bmjebm-2023-112518","url":null,"abstract":"Objectives Despite rising rates of multimorbidity, existing risk assessment tools are mostly limited to a single outcome of interest. This study tests the feasibility of producing multiple disease risk estimates with at least 70% discrimination (area under the receiver operating curve, AUROC) within the time and information constraints of the existing primary care health check framework. Design Observational prospective cohort study Setting UK Biobank. Participants 228 240 adults from the UK population. Interventions None. Main outcome measures Myocardial infarction, atrial fibrillation, heart failure, stroke, all-cause dementia, chronic kidney disease, fatty liver disease, alcoholic liver disease, liver cirrhosis and liver failure. Results Using a set of predictors easily gathered at the standard primary care health check (such as the National Health Service Health Check), we demonstrate that it is feasible to simultaneously produce risk estimates for multiple disease outcomes with AUROC of 70% or greater. These predictors can be entered once into a single form and produce risk scores for stroke (AUROC 0.727, 95% CI 0.713 to 0.740), all-cause dementia (0.823, 95% CI 0.810 to 0.836), myocardial infarction (0.785, 95% CI 0.775 to 0.795), atrial fibrillation (0.777, 95% CI 0.768 to 0.785), heart failure (0.828, 95% CI 0.818 to 0.838), chronic kidney disease (0.774, 95% CI 0.765 to 0.783), fatty liver disease (0.766, 95% CI 0.753 to 0.779), alcoholic liver disease (0.864, 95% CI 0.835 to 0.894), liver cirrhosis (0.763, 95% CI 0.734 to 0.793) and liver failure (0.746, 95% CI 0.695 to 0.796). Conclusions Easily collected diagnostics can be used to assess 10-year risk across multiple disease outcomes, without the need for specialist computing or invasive biomarkers. Such an approach could increase the utility of existing data and place multiorgan risk information at the fingertips of primary care providers, thus creating opportunities for longer-term multimorbidity prevention. Additional work is needed to validate whether these findings would hold in a larger, more representative cohort outside the UK Biobank. Data may be obtained from a third party and are not publicly available. This analysis was produced under UK Biobank Access Application 59867. The data in this study are owned by the UK Biobank (www. ukbiobank.ac.uk) and legal constraints do not permit public sharing of the data. The UK Biobank, however, is open to all bona fide researchers anywhere in the world. Thus, the data used in this communication can be easily and directly accessed by applying through the UK Biobank Access Management System (www.ukbiobank.ac.uk/ register-apply). Results from this study will be returned to UK Biobank according to their published returns policy.","PeriodicalId":9059,"journal":{"name":"BMJ Evidence-Based Medicine","volume":"21 1","pages":""},"PeriodicalIF":5.8,"publicationDate":"2024-05-06","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140883781","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 : 2024-05-02DOI: 10.1136/bmjebm-2023-112740
Edward Robert St John, Connor James Stewart Moore, Raghu Ram Pillarisetti, Erica Sarah Spatz
Shared decision-making (SDM) is increasingly recognised as fundamental to patient-centred care and enabling patients to make voluntary, informed decisions about their health.1 SDM is the process whereby patients and clinicians come together to share their expertise. The patient acts as an expert of themselves, understanding their own preferences and their attitudes to risk. The clinician is an expert on the medical knowledge and scientific evidence. Together, treatment options should be explored, arriving at a treatment decision that is right for the patient and supported by the clinician. When dealing with invasive or high-risk procedures (eg, operations, chemotherapy, radiotherapy, immunotherapy), once the treatment decision has been made, the conversation turns to informed consent. This is the process of communicating and agreeing to the potential risks and benefits of the procedure, while acknowledging that there are alternative treatment options that have not been chosen. Though informed consent should be the culmination of SDM, alone it does not encapsulate the entire process. There is a distinction between decision-making and consent and this should ideally be accompanied by a period for reflection. Despite advances in SDM, the subsequent informed consent process has remained stagnant, often failing to meet ethical or legal standards of supporting meaningful patient autonomy.2 In reality, rapid surgical decisions may be required (e.g. emergency or cancer pathways), where time is a precious commodity to deliver optimal patient care. In these scenarios, it is common for discussions to move quickly from diagnosis to treatment options, to consent. However, scarcity of clinician time should not be an excuse for inadequate consent. Therefore, reimagining the consent process in the digital age by ensuring the benefits, risks and alternative treatment options are clearly and correctly presented as early as possible, has the ability of transforming this step from a ritualised gesture into a …
{"title":"Global considerations for informed consent with shared decision-making in the digital age","authors":"Edward Robert St John, Connor James Stewart Moore, Raghu Ram Pillarisetti, Erica Sarah Spatz","doi":"10.1136/bmjebm-2023-112740","DOIUrl":"https://doi.org/10.1136/bmjebm-2023-112740","url":null,"abstract":"Shared decision-making (SDM) is increasingly recognised as fundamental to patient-centred care and enabling patients to make voluntary, informed decisions about their health.1 SDM is the process whereby patients and clinicians come together to share their expertise. The patient acts as an expert of themselves, understanding their own preferences and their attitudes to risk. The clinician is an expert on the medical knowledge and scientific evidence. Together, treatment options should be explored, arriving at a treatment decision that is right for the patient and supported by the clinician. When dealing with invasive or high-risk procedures (eg, operations, chemotherapy, radiotherapy, immunotherapy), once the treatment decision has been made, the conversation turns to informed consent. This is the process of communicating and agreeing to the potential risks and benefits of the procedure, while acknowledging that there are alternative treatment options that have not been chosen. Though informed consent should be the culmination of SDM, alone it does not encapsulate the entire process. There is a distinction between decision-making and consent and this should ideally be accompanied by a period for reflection. Despite advances in SDM, the subsequent informed consent process has remained stagnant, often failing to meet ethical or legal standards of supporting meaningful patient autonomy.2 In reality, rapid surgical decisions may be required (e.g. emergency or cancer pathways), where time is a precious commodity to deliver optimal patient care. In these scenarios, it is common for discussions to move quickly from diagnosis to treatment options, to consent. However, scarcity of clinician time should not be an excuse for inadequate consent. Therefore, reimagining the consent process in the digital age by ensuring the benefits, risks and alternative treatment options are clearly and correctly presented as early as possible, has the ability of transforming this step from a ritualised gesture into a …","PeriodicalId":9059,"journal":{"name":"BMJ Evidence-Based Medicine","volume":"19 1","pages":""},"PeriodicalIF":5.8,"publicationDate":"2024-05-02","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140841217","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 : 2024-04-29DOI: 10.1136/bmjebm-2023-112583
Zilong Bian, Lijuan Wang, Rong Fan, Jing Sun, Lili Yu, Meihong Xu, Paul R H J Timmers, Xia Shen, James F Wilson, Evropi Theodoratou, Xifeng Wu, Xue Li
Objective To investigate the associations across genetic and lifestyle factors with lifespan. Design A longitudinal cohort study. Setting UK Biobank. Participants 353 742 adults of European ancestry, who were recruited from 2006 to 2010 and were followed up until 2021. Exposures A polygenic risk score for lifespan with long (highest quintile) risk categories and a weighted healthy lifestyle score, including no current smoking, moderate alcohol consumption, regular physical activity, healthy body shape, adequate sleep duration, and a healthy diet, categorised into favourable, intermediate, and unfavourable lifestyles. Main outcome measures Lifespan defined as the date of death or the censor date minus the date of birth. Results Of the included 353 742 participants of European ancestry with a median follow-up of 12.86 years, 24 239 death cases were identified. Participants were grouped into three genetically determined lifespan categories including long (20.1%), intermediate (60.1%), and short (19.8%), and into three lifestyle score categories including favourable (23.1%), intermediate (55.6%), and unfavourable (21.3%). The hazard ratio (HR) of death for individuals with a genetic predisposition to a short lifespan was 1.21 (95% CI 1.16 to 1.26) compared to those with a genetic predisposition to a long lifespan. The HR of death for individuals in the unfavourable lifestyle category was 1.78 (95% CI 1.71 to 1.85), compared with those in the favourable lifestyle category. Participants with a genetic predisposition to a short lifespan and an unfavourable lifestyle had 2.04 times (95% CI 1.87 to 2.22) higher rates of death compared with those with a genetic predisposition to a long lifespan and a favourable lifestyle. No multiplicative interaction was detected between the polygenic risk score of lifespan and the weighted healthy lifestyle score (p=0.10). The optimal combination of healthy lifestyles, including never smoking, regular physical activity, adequate sleep duration, and a healthy diet, was derived to decrease risk of premature death (death before 75 years). Conclusion Genetic and lifestyle factors were independently associated with lifespan. Adherence to healthy lifestyles could largely attenuate the genetic risk of a shorter lifespan or premature death. The optimal combination of healthy lifestyles could convey better benefits for a longer lifespan, regardless of genetic background. Data are available in a public, open access repository. NHANES data are available at . UK Biobank study was under Application Number 66354. The UK Biobank is an open access resource and bona fide researchers can apply to use the UK Biobank dataset by registering and applying at . Further information is available from the corresponding author upon request.
{"title":"Genetic predisposition, modifiable lifestyles, and their joint effects on human lifespan: evidence from multiple cohort studies","authors":"Zilong Bian, Lijuan Wang, Rong Fan, Jing Sun, Lili Yu, Meihong Xu, Paul R H J Timmers, Xia Shen, James F Wilson, Evropi Theodoratou, Xifeng Wu, Xue Li","doi":"10.1136/bmjebm-2023-112583","DOIUrl":"https://doi.org/10.1136/bmjebm-2023-112583","url":null,"abstract":"Objective To investigate the associations across genetic and lifestyle factors with lifespan. Design A longitudinal cohort study. Setting UK Biobank. Participants 353 742 adults of European ancestry, who were recruited from 2006 to 2010 and were followed up until 2021. Exposures A polygenic risk score for lifespan with long (<lowest quintile), intermediate (quintiles 2 to 4), and short (>highest quintile) risk categories and a weighted healthy lifestyle score, including no current smoking, moderate alcohol consumption, regular physical activity, healthy body shape, adequate sleep duration, and a healthy diet, categorised into favourable, intermediate, and unfavourable lifestyles. Main outcome measures Lifespan defined as the date of death or the censor date minus the date of birth. Results Of the included 353 742 participants of European ancestry with a median follow-up of 12.86 years, 24 239 death cases were identified. Participants were grouped into three genetically determined lifespan categories including long (20.1%), intermediate (60.1%), and short (19.8%), and into three lifestyle score categories including favourable (23.1%), intermediate (55.6%), and unfavourable (21.3%). The hazard ratio (HR) of death for individuals with a genetic predisposition to a short lifespan was 1.21 (95% CI 1.16 to 1.26) compared to those with a genetic predisposition to a long lifespan. The HR of death for individuals in the unfavourable lifestyle category was 1.78 (95% CI 1.71 to 1.85), compared with those in the favourable lifestyle category. Participants with a genetic predisposition to a short lifespan and an unfavourable lifestyle had 2.04 times (95% CI 1.87 to 2.22) higher rates of death compared with those with a genetic predisposition to a long lifespan and a favourable lifestyle. No multiplicative interaction was detected between the polygenic risk score of lifespan and the weighted healthy lifestyle score (p=0.10). The optimal combination of healthy lifestyles, including never smoking, regular physical activity, adequate sleep duration, and a healthy diet, was derived to decrease risk of premature death (death before 75 years). Conclusion Genetic and lifestyle factors were independently associated with lifespan. Adherence to healthy lifestyles could largely attenuate the genetic risk of a shorter lifespan or premature death. The optimal combination of healthy lifestyles could convey better benefits for a longer lifespan, regardless of genetic background. Data are available in a public, open access repository. NHANES data are available at <http://www.cdc.gov/nchs/nhis/index.htm>. UK Biobank study was under Application Number 66354. The UK Biobank is an open access resource and bona fide researchers can apply to use the UK Biobank dataset by registering and applying at <http://ukbiobank.ac.uk/register-apply/>. Further information is available from the corresponding author upon request.","PeriodicalId":9059,"journal":{"name":"BMJ Evidence-Based Medicine","volume":"31 1","pages":""},"PeriodicalIF":5.8,"publicationDate":"2024-04-29","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140841182","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 : 2024-04-17DOI: 10.1136/bmjebm-2023-112814
Camilla Alderighi, Raffaele Rasoini, Rebecca De Fiore, Fabio Ambrosino, Steven Woloshin
Medical research gets plenty of media attention. Unfortunately, the attention is often problematic, frequently failing to provide readers with information needed to understand findings or decide whether to believe them.1 Unless journalists highlight study cautions and limitations, avoid spin2 and overinterpretation of findings, the public may draw erroneous conclusions about the reliability and actionability of the research. Coverage of observational research may be especially challenging given inherent difficulty in inferring causation, a limitation that is rarely mentioned in medical journals articles or corresponding news.3 We used news coverage of a retrospective cohort study, published in Nature Medicine in 2022,4 as a case study to assess news reporting quality. The index study used national data from US Department of Veteran Affairs to characterise the post-acute cardiovascular manifestations of COVID-19. We chose this study because of its potential public health impact (ie, reporting increased cardiovascular diseases after even mild COVID-19 infection) and its enormous media attention: one of the highest Altmetric scores ever (>20 k, coverage in over 600 news outlets and 40 000 tweets). Our study supplements a previous analysis limited to Italian news.5 To assess news quality, we derived a coding scheme (online supplemental appendix 1) from published quality measures developed to capture proper reporting of observational research6 7: the need to refrain from inappropriate causal inferences and unsupported …
{"title":"Bad news: how the media reported on an observational study about cardiovascular outcomes of COVID-19","authors":"Camilla Alderighi, Raffaele Rasoini, Rebecca De Fiore, Fabio Ambrosino, Steven Woloshin","doi":"10.1136/bmjebm-2023-112814","DOIUrl":"https://doi.org/10.1136/bmjebm-2023-112814","url":null,"abstract":"Medical research gets plenty of media attention. Unfortunately, the attention is often problematic, frequently failing to provide readers with information needed to understand findings or decide whether to believe them.1 Unless journalists highlight study cautions and limitations, avoid spin2 and overinterpretation of findings, the public may draw erroneous conclusions about the reliability and actionability of the research. Coverage of observational research may be especially challenging given inherent difficulty in inferring causation, a limitation that is rarely mentioned in medical journals articles or corresponding news.3 We used news coverage of a retrospective cohort study, published in Nature Medicine in 2022,4 as a case study to assess news reporting quality. The index study used national data from US Department of Veteran Affairs to characterise the post-acute cardiovascular manifestations of COVID-19. We chose this study because of its potential public health impact (ie, reporting increased cardiovascular diseases after even mild COVID-19 infection) and its enormous media attention: one of the highest Altmetric scores ever (>20 k, coverage in over 600 news outlets and 40 000 tweets). Our study supplements a previous analysis limited to Italian news.5 To assess news quality, we derived a coding scheme (online supplemental appendix 1) from published quality measures developed to capture proper reporting of observational research6 7: the need to refrain from inappropriate causal inferences and unsupported …","PeriodicalId":9059,"journal":{"name":"BMJ Evidence-Based Medicine","volume":"33 1","pages":""},"PeriodicalIF":5.8,"publicationDate":"2024-04-17","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140611957","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 : 2024-04-17DOI: 10.1136/bmjebm-2023-112540
Till Bruckner, Aminul Schuster, Belén Chavarría, Carolina Cruz, Fabiola Karely Lizárraga Illán, Ronak Borana, Tungamirai Ishe Bvute, Daniel Sánchez
Worldwide, a significant proportion of clinical trials end up as costly research waste because their results are never made public.1–3 The resulting gaps in the medical evidence base harm patients and undermine public health.4 The Declaration of Helsinki and the WHO both call for all clinical trial results to be made public.5 6 In the wake of a 2018 UK parliamentary enquiry, non-commercial clinical trial sponsors in the UK substantially improved outcome reporting for drug trials (Clinical Trials of Investigative Medicinal Products, CTIMPs) by uploading the summary results of many CTIMPs onto the European Union Clinical Trials Register (EUCTR) which exclusively lists drug trials. However, these efforts typically did not extend to other types of trials, which are listed on other trial registries. This study assesses the current publication status of 145 clinical trials that are not CTIMPs that were sponsored by ten major UK non-commercial sponsors and were completed or terminated in 2017, allowing a 5-year follow-up period for publication. The lead researcher (TB) identified the 10 most prolific non-commercial sponsors of clinical trials in the UK by accessing the EU Trials Tracker on 27 October 2022, employing the …
{"title":"Major UK non-commercial sponsors’ efforts to reduce research waste: a mixed-methods study","authors":"Till Bruckner, Aminul Schuster, Belén Chavarría, Carolina Cruz, Fabiola Karely Lizárraga Illán, Ronak Borana, Tungamirai Ishe Bvute, Daniel Sánchez","doi":"10.1136/bmjebm-2023-112540","DOIUrl":"https://doi.org/10.1136/bmjebm-2023-112540","url":null,"abstract":"Worldwide, a significant proportion of clinical trials end up as costly research waste because their results are never made public.1–3 The resulting gaps in the medical evidence base harm patients and undermine public health.4 The Declaration of Helsinki and the WHO both call for all clinical trial results to be made public.5 6 In the wake of a 2018 UK parliamentary enquiry, non-commercial clinical trial sponsors in the UK substantially improved outcome reporting for drug trials (Clinical Trials of Investigative Medicinal Products, CTIMPs) by uploading the summary results of many CTIMPs onto the European Union Clinical Trials Register (EUCTR) which exclusively lists drug trials. However, these efforts typically did not extend to other types of trials, which are listed on other trial registries. This study assesses the current publication status of 145 clinical trials that are not CTIMPs that were sponsored by ten major UK non-commercial sponsors and were completed or terminated in 2017, allowing a 5-year follow-up period for publication. The lead researcher (TB) identified the 10 most prolific non-commercial sponsors of clinical trials in the UK by accessing the EU Trials Tracker on 27 October 2022, employing the …","PeriodicalId":9059,"journal":{"name":"BMJ Evidence-Based Medicine","volume":"166 1-2 1","pages":""},"PeriodicalIF":5.8,"publicationDate":"2024-04-17","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140612327","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 : 2024-04-10DOI: 10.1136/bmjebm-2023-112767
Areti Angeliki Veroniki, Sai Surabi Thirugnanasampanthar, Menelaos Konstantinidis, Jasmeen Dourka, Marco Ghassemi, Dipika Neupane, Paul Khan, Vera Nincic, Margarita Corry, Reid Robson, Amanda Parker, Charlene Soobiah, Angela Sinilaite, Pamela Doyon-Plourde, Anabel Gil, Winnie Siu, Nasheed Moqueet, Adrienne Stevens, Kelly English, Ivan D Florez, Juan J Yepes-Nuñez, Brian Hutton, Matthew Muller, Lorenzo Moja, Sharon Straus, Andrea C Tricco
Objectives To compare the efficacy of influenza vaccines of any valency for adults 60 years and older. Design and setting Systematic review with network meta-analysis (NMA) of randomised controlled trials (RCTs). MEDLINE, EMBASE, JBI Evidence-Based Practice (EBP) Database, PsycINFO, and Cochrane Evidence -Based Medicine database were searched from inception to 20 June 20, 2022. Two reviewers screened, abstracted, and appraised articles (Cochrane Risk of Bias (ROB) 2.0 tool) independently. We assessed certainty of findings using Confidence in Network Meta-Analysis and Grading of Recommendations, Assessment, Development and Evaluations approaches. We performed random-effects meta-analysis and network meta-analysis (NMA), and estimated odds ratios (ORs) for dichotomous outcomes and incidence rate ratios (IRRs) for count outcomes along with their corresponding 95% confidence intervals (CIs) and prediction intervals. Participants Older adults (≥60 years old) receiving an influenza vaccine licensed in Canada or the USA (vs placebo, no vaccine, or any other licensed vaccine), at any dose. Main outcome measures Laboratory-confirmed influenza (LCI) and influenza-like illness (ILI). Secondary outcomes were the number of vascular adverse events, hospitalisation for acute respiratory infection (ARI) and ILI, inpatient hospitalisation, emergency room (ER) visit for ILI, outpatient visit, and mortality, among others. Results We included 41 RCTs and 15 companion reports comprising 8 vaccine types and 206 032 participants. Vaccines may prevent LCI compared with placebo, with high-dose trivalent inactivated influenza vaccine (IIV3-HD) (NMA: 9 RCTs, 52 202 participants, OR 0.23, 95% confidence interval (CI) (0.11 to 0.51), low certainty of evidence) and recombinant influenza vaccine (RIV) (OR 0.25, 95%CI (0.08 to 0.73), low certainty of evidence) among the most efficacious vaccines. Standard dose trivalent IIV3 (IIV3-SD) may prevent ILI compared with placebo, but the result was imprecise (meta-analysis: 2 RCTs, 854 participants, OR 0.39, 95%CI (0.15 to 1.02), low certainty of evidence). Any HD was associated with prevention of ILI compared with placebo (NMA: 9 RCTs, 65 658 participants, OR 0.38, 95%CI (0.15 to 0.93)). Adjuvanted quadrivalent IIV (IIV4-Adj) may be associated with the least vascular adverse events, but the results were very uncertain (NMA: eight 8 RCTs, 57 677 participants, IRR 0.18, 95%CI (0.07 to 0.43), very low certainty of evidence). RIV on all-cause mortality may be comparable to placebo (NMA: 20 RCTs, 140 577 participants, OR 1.01, 95%CI (0.23 to 4.49), low certainty of evidence). Conclusions This systematic review demonstrated efficacy associated with IIV3-HD and RIV vaccines in protecting older persons against LCI. RIV vaccine may reduce all-cause mortality when compared with other vaccines, but the evidence is uncertain. Differences in efficacy between influenza vaccines remain uncertain with very low to moderate certainty of evidence. PROS
{"title":"Trivalent and quadrivalent seasonal influenza vaccine in adults aged 60 and older: a systematic review and network meta-analysis","authors":"Areti Angeliki Veroniki, Sai Surabi Thirugnanasampanthar, Menelaos Konstantinidis, Jasmeen Dourka, Marco Ghassemi, Dipika Neupane, Paul Khan, Vera Nincic, Margarita Corry, Reid Robson, Amanda Parker, Charlene Soobiah, Angela Sinilaite, Pamela Doyon-Plourde, Anabel Gil, Winnie Siu, Nasheed Moqueet, Adrienne Stevens, Kelly English, Ivan D Florez, Juan J Yepes-Nuñez, Brian Hutton, Matthew Muller, Lorenzo Moja, Sharon Straus, Andrea C Tricco","doi":"10.1136/bmjebm-2023-112767","DOIUrl":"https://doi.org/10.1136/bmjebm-2023-112767","url":null,"abstract":"Objectives To compare the efficacy of influenza vaccines of any valency for adults 60 years and older. Design and setting Systematic review with network meta-analysis (NMA) of randomised controlled trials (RCTs). MEDLINE, EMBASE, JBI Evidence-Based Practice (EBP) Database, PsycINFO, and Cochrane Evidence -Based Medicine database were searched from inception to 20 June 20, 2022. Two reviewers screened, abstracted, and appraised articles (Cochrane Risk of Bias (ROB) 2.0 tool) independently. We assessed certainty of findings using Confidence in Network Meta-Analysis and Grading of Recommendations, Assessment, Development and Evaluations approaches. We performed random-effects meta-analysis and network meta-analysis (NMA), and estimated odds ratios (ORs) for dichotomous outcomes and incidence rate ratios (IRRs) for count outcomes along with their corresponding 95% confidence intervals (CIs) and prediction intervals. Participants Older adults (≥60 years old) receiving an influenza vaccine licensed in Canada or the USA (vs placebo, no vaccine, or any other licensed vaccine), at any dose. Main outcome measures Laboratory-confirmed influenza (LCI) and influenza-like illness (ILI). Secondary outcomes were the number of vascular adverse events, hospitalisation for acute respiratory infection (ARI) and ILI, inpatient hospitalisation, emergency room (ER) visit for ILI, outpatient visit, and mortality, among others. Results We included 41 RCTs and 15 companion reports comprising 8 vaccine types and 206 032 participants. Vaccines may prevent LCI compared with placebo, with high-dose trivalent inactivated influenza vaccine (IIV3-HD) (NMA: 9 RCTs, 52 202 participants, OR 0.23, 95% confidence interval (CI) (0.11 to 0.51), low certainty of evidence) and recombinant influenza vaccine (RIV) (OR 0.25, 95%CI (0.08 to 0.73), low certainty of evidence) among the most efficacious vaccines. Standard dose trivalent IIV3 (IIV3-SD) may prevent ILI compared with placebo, but the result was imprecise (meta-analysis: 2 RCTs, 854 participants, OR 0.39, 95%CI (0.15 to 1.02), low certainty of evidence). Any HD was associated with prevention of ILI compared with placebo (NMA: 9 RCTs, 65 658 participants, OR 0.38, 95%CI (0.15 to 0.93)). Adjuvanted quadrivalent IIV (IIV4-Adj) may be associated with the least vascular adverse events, but the results were very uncertain (NMA: eight 8 RCTs, 57 677 participants, IRR 0.18, 95%CI (0.07 to 0.43), very low certainty of evidence). RIV on all-cause mortality may be comparable to placebo (NMA: 20 RCTs, 140 577 participants, OR 1.01, 95%CI (0.23 to 4.49), low certainty of evidence). Conclusions This systematic review demonstrated efficacy associated with IIV3-HD and RIV vaccines in protecting older persons against LCI. RIV vaccine may reduce all-cause mortality when compared with other vaccines, but the evidence is uncertain. Differences in efficacy between influenza vaccines remain uncertain with very low to moderate certainty of evidence. PROS","PeriodicalId":9059,"journal":{"name":"BMJ Evidence-Based Medicine","volume":"30 1","pages":""},"PeriodicalIF":5.8,"publicationDate":"2024-04-10","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140561447","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 : 2024-04-10DOI: 10.1136/bmjebm-2023-112641
Jason N Doctor, Daniella Meeker, Craig R Fox, Stephen D Persell, Zachary Wagner, Kathryn E Bouskill, Kyle A Zanocco, Robert J Romanelli, Chad M Brummett, Allison Kirkegaard, Katherine E Watkins
Shared decision-making in medicine is widely viewed as a collaboration between the patient and the clinician. For example, Montori et al state, ‘The patient and clinician must collaborate to arrive at a useful formulation of the problem’.1 Patients are encouraged to evaluate care choices in light of the benefits and harms of each, state their preferences and identify the best course of action along with their doctor.2 Despite its broad reach, shared decision-making solely between a patient and doctor has clear limits. Over 30 years ago, Brock and Wartman cautioned that ‘[p]atients do not have an unqualified right to make even rational individual choices that risk serious harm to others’.3 Elywin et al noted that ‘limits on shared decision-making will occur when… wider interests overrule individual wishes’.4 These authors lay out problems with shared decisions for antibiotics, opioids and vaccine hesitancy. A crucial gap is how to address these problems in practice. Antibiotic-resistant bacterial infections, overdoses from diverted opioid pills and the resurgence of measles are all medical problems that affect an individual through actions others in the community have taken. Here cooperation has either failed or has not been attempted at all. Lack of cooperation occurs when individuals believe it is in their best interest to deviate from the action that they would like others to take.5 While various forms of cooperative behaviour exist in the wild (eg, a large number of individuals choose to recycle, vote, tip at restaurants and donate to charity),5 there are barriers to cooperation in medicine that require special attention. Two of the biggest barriers are a lack of awareness that cooperation is needed and the implementation of approaches to encourage cooperation. To address barriers, community members benefit from working towards a resolution on a common strategy that …
{"title":"A call for community-shared decisions","authors":"Jason N Doctor, Daniella Meeker, Craig R Fox, Stephen D Persell, Zachary Wagner, Kathryn E Bouskill, Kyle A Zanocco, Robert J Romanelli, Chad M Brummett, Allison Kirkegaard, Katherine E Watkins","doi":"10.1136/bmjebm-2023-112641","DOIUrl":"https://doi.org/10.1136/bmjebm-2023-112641","url":null,"abstract":"Shared decision-making in medicine is widely viewed as a collaboration between the patient and the clinician. For example, Montori et al state, ‘The patient and clinician must collaborate to arrive at a useful formulation of the problem’.1 Patients are encouraged to evaluate care choices in light of the benefits and harms of each, state their preferences and identify the best course of action along with their doctor.2 Despite its broad reach, shared decision-making solely between a patient and doctor has clear limits. Over 30 years ago, Brock and Wartman cautioned that ‘[p]atients do not have an unqualified right to make even rational individual choices that risk serious harm to others’.3 Elywin et al noted that ‘limits on shared decision-making will occur when… wider interests overrule individual wishes’.4 These authors lay out problems with shared decisions for antibiotics, opioids and vaccine hesitancy. A crucial gap is how to address these problems in practice. Antibiotic-resistant bacterial infections, overdoses from diverted opioid pills and the resurgence of measles are all medical problems that affect an individual through actions others in the community have taken. Here cooperation has either failed or has not been attempted at all. Lack of cooperation occurs when individuals believe it is in their best interest to deviate from the action that they would like others to take.5 While various forms of cooperative behaviour exist in the wild (eg, a large number of individuals choose to recycle, vote, tip at restaurants and donate to charity),5 there are barriers to cooperation in medicine that require special attention. Two of the biggest barriers are a lack of awareness that cooperation is needed and the implementation of approaches to encourage cooperation. To address barriers, community members benefit from working towards a resolution on a common strategy that …","PeriodicalId":9059,"journal":{"name":"BMJ Evidence-Based Medicine","volume":"12 1","pages":""},"PeriodicalIF":5.8,"publicationDate":"2024-04-10","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140561446","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 : 2024-03-22DOI: 10.1136/bmjebm-2023-112590
Martin O'Flaherty, Nikkil Sudharsanan, Chris Kypridemos
{"title":"Can the HEARTS initiative reduce the burden of cardiovascular disease?","authors":"Martin O'Flaherty, Nikkil Sudharsanan, Chris Kypridemos","doi":"10.1136/bmjebm-2023-112590","DOIUrl":"https://doi.org/10.1136/bmjebm-2023-112590","url":null,"abstract":"","PeriodicalId":9059,"journal":{"name":"BMJ Evidence-Based Medicine","volume":" ","pages":""},"PeriodicalIF":5.8,"publicationDate":"2024-03-22","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140189487","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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