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A systematic review to identify and collate factors influencing patient journeys through clinical trials. 通过系统性审查,确定并整理影响患者临床试验之旅的因素。
Pub Date : 2023-06-07 eCollection Date: 2023-06-01 DOI: 10.1177/20542704231166621
Rebecca Dobra, Gemma Wilson, Jessie Matthews, Marco Boeri, Stuart Elborn, Frank Kee, Jane C Davies, Susan Madge

Patient-centred trial design and delivery; improves recruitment and retention; increases participant satisfaction; encourages participation by a more representative cohort; and allows researchers to better meet participants' needs. Research in this area mostly focusses on narrow facets of trial participation. We aimed to systematically identify the breadth of patient-centred factors influencing participation and engagement in trials, and collate them into a framework. Through this we hoped to assist researchers to identify factors that could improve patient-centred trial design and delivery. Robust qualitative and mixed methods systematic reviews are becoming increasingly common in health research. The protocol for this review was prospectively registered on PROSPERO, CRD42020184886. We used the SPIDER (Sample, Phenomenon of Interest, Design, Evaluation, Research Type) framework as a standardised systematic search strategy tool. 3 databases were searched as well as references checking, and thematic synthesis was conducted. Screening agreement was performed and code and theme checking were conducted by 2 independent researchers. Data were drawn from 285 peer-reviewed articles. 300 discrete factors were identified, and sorted into 13 themes and subthemes. The full catalogue of factors is included in the Supplementary Material. A summary framework is included in the body of the article. This paper focusses on outlining common ground that themes share, highlighting critical features, and exploring interesting points from the data. Through this, we hope researchers from multiple specialities may be better able to meet patients' needs, protect patients' psychosocial wellbeing, and optimise trial recruitment and retention, with direct positive impact on research time and cost efficiency.

以患者为中心的试验设计和实施;改善招募和保留情况;提高参与者的满意度;鼓励更具代表性的群体参与;并使研究人员能够更好地满足参与者的需求。该领域的研究大多集中在试验参与的狭义方面。我们的目标是系统地识别影响参与试验的以患者为中心的广泛因素,并将其整理成一个框架。我们希望借此帮助研究人员找出可以改善以患者为中心的试验设计和实施的因素。在健康研究中,稳健的定性和混合方法系统性综述越来越常见。本综述的方案已在 PROSPERO 上进行了前瞻性注册,注册号为 CRD42020184886。我们使用 SPIDER(样本、感兴趣现象、设计、评价、研究类型)框架作为标准化的系统性检索策略工具。我们检索了 3 个数据库并查阅了参考文献,然后进行了专题综合。由两名独立研究人员进行筛选,并进行代码和主题检查。数据来自 285 篇经同行评审的文章。确定了 300 个离散因素,并将其分类为 13 个主题和次主题。全部因素目录见补充材料。文章正文中包含了一个总结框架。本文重点概述了各主题的共同点,突出了关键特征,并探讨了数据中的有趣之处。我们希望来自多个专业的研究人员能借此更好地满足患者的需求,保护患者的社会心理健康,优化试验招募和保留,从而对研究时间和成本效率产生直接的积极影响。
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引用次数: 0
Diabetic ketoacidosis in a patient known with Gitelman syndrome. 糖尿病酮症酸中毒1例已知的吉特尔曼综合征。
Pub Date : 2023-06-01 DOI: 10.1177/20542704231183266
Ida Ringsing Nielsen, Patricia Almine Skat-Rørdam, Ivan Werner Jensen

Gitelman syndrome is a rare hereditary nephropathy, which causes chronic metabolic alkalosis with low potassium and magnesium levels. There is no known coherence between Gitelman syndrome and Type-1 diabetes but patients with both diseases that develop diabetic ketoacidosis might present with normal acid status and receive incorrect treatment. In our case report the patient was known with both diseases and quickly diagnosed and treated but the condition is rare and previously only described in two other case reports.

Gitelman综合征是一种罕见的遗传性肾病,引起慢性代谢性碱中毒,钾和镁水平低。Gitelman综合征和1型糖尿病之间没有已知的一致性,但两种疾病的患者发生糖尿病酮症酸中毒可能表现为正常的酸状态,并接受了错误的治疗。在我们的病例报告中,患者已知患有这两种疾病,并迅速得到诊断和治疗,但这种情况很罕见,以前只在另外两例报告中描述过。
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引用次数: 0
Prediction of therapeutic value of new drugs approved by health Canada from 2011-2020: A cross-sectional study. 2011-2020年加拿大卫生部批准的新药治疗价值预测:一项横断面研究
Pub Date : 2023-05-01 DOI: 10.1177/20542704231166620
Joel Lexchin

Objectives: To examine whether a combination of three characteristics of new drugs - review type, outcome of premarket trials (surrogate or clinical) and first-in-class is associated with significant therapeutic value.

Design: Cross-sectional analysis of new drugs approved by Health Canada from January 1, 2011 to December 31, 2020.

Setting: Canada.

Participants: New drugs approved by Health Canada for which therapeutic evaluations, trial outcomes and first-in-class status was available.

Main outcome measures: Distribution of therapeutic value (major, moderate, little to no) depending on how many of the three characteristics were present for each drug.

Results: Health Canada approved 340 drugs of which 243 had data available for analysis. If all three characteristics were present 10 out of the 20 drugs had a major therapeutic rating. Conversely if none were present only 2 drugs out of 37 had a major therapeutic rating.

Conclusion: This study introduces a new evaluation method for determining whether new drugs will have major therapeutic value that appears to be more successful than relying only on the type of review that drugs receive.

目的:研究新药的审评类型、上市前试验结果(替代或临床)和首次上市这三个特征的组合是否与显著的治疗价值相关。设计:对2011年1月1日至2020年12月31日加拿大卫生部批准的新药进行横断面分析。设置:加拿大。参与者:加拿大卫生部批准的新药,可获得治疗评价、试验结果和一流地位。主要结果测量:治疗价值的分布(主要、中等、很少或没有)取决于每种药物存在多少三个特征。结果:加拿大卫生部批准了340种药物,其中243种有可供分析的数据。如果所有三个特征都存在,20种药物中有10种具有主要治疗评级。相反,如果没有药物存在,37种药物中只有2种具有主要治疗评级。结论:本研究引入了一种新的评估方法来确定新药是否具有主要的治疗价值,这种评估方法似乎比仅仅依靠药物接受的审查类型更成功。
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引用次数: 1
A cross-sectional survey of English NHS Trusts on their uptake and provision of active bystander training including to address sexual harassment. 英国国民健康保险制度信托的横断面调查他们的吸收和提供积极的旁观者培训,包括解决性骚扰。
Pub Date : 2023-04-01 DOI: 10.1177/20542704231166619
Ava Robertson, Sarah Steele

Objective: Reports identify that sexual harassment is troublingly pervasive in the NHS. Active bystander training (ABT) has been promoted to address sexual harassment, alongside other forms of poor behaviour, discrimination, and harassment. We explore ABT across all English NHS Trusts and determine whether the programmes address sexual misconduct in the training content.

Design: Freedom of Information requests asking whether Trusts offer ABT, and if so, about the programme content and delivery, and to NHS England on centrally commissioned ABT.

Setting: 213 NHS Trusts in England, and NHS England.

Participants: Not applicable.

Main outcome measure: Provision of ABT, and presence of sexual harassment content in the training.

Results: 199 Trusts responded by August 2022. Of these, 35 Trusts provide ABT, the majority of which deliver content that is not specific to sexual misconduct, are in London, outsource training using private providers, and only provide workshops on an opt-in basis. One Trust offers a standalone ABT module on sexual harassment. Private providers prohibit Trusts from sharing training material, inhibiting content analysis and evaluation. Among the 163 Trusts without ABT programmes, only 23 (13%) have plans to implement training.

Conclusions: ABT is underutilised in the NHS, despite being identified as an effective intervention in other settings like the military, higher education, and government workplaces. Studies should explore whether wider NHS adoption is warranted. Robust monitoring and evaluation processes are critical to strengthening the available literature regarding the effectiveness of ABT in the healthcare context and engaging in global knowledge sharing across health systems.

目的:报告指出,性骚扰是令人不安的普遍在NHS。积极旁观者培训(ABT)已得到推广,以解决性骚扰以及其他形式的不良行为、歧视和骚扰问题。我们在所有英国NHS信托基金中探索ABT,并确定该计划是否在培训内容中解决性行为不端的问题。设计:信息自由请求,询问信托机构是否提供ABT,如果提供,关于项目内容和交付,并向NHS英格兰进行集中委托的ABT。设置:213个英格兰NHS信托机构和NHS英格兰。参与者:不适用。主要结果测量:提供ABT,以及在培训中存在性骚扰内容。结果:到2022年8月,199家信托公司做出了回应。其中,有35家信托机构提供ABT,其中大多数提供的内容并非针对性行为不端,它们位于伦敦,使用私人提供商外包培训,并且只在选择加入的基础上提供讲习班。One Trust提供了一个关于性骚扰的独立ABT模块。私人供应商禁止信托基金分享培训材料,阻碍了内容分析和评估。在163个没有ABT项目的信托基金中,只有23个(13%)有实施培训的计划。结论:尽管在军事、高等教育和政府工作场所等其他环境中被认为是一种有效的干预措施,但ABT在NHS中的利用不足。研究应该探讨是否有必要更广泛地采用NHS。强有力的监测和评估过程对于加强有关ABT在卫生保健方面有效性的现有文献和参与卫生系统之间的全球知识共享至关重要。
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引用次数: 4
Secondary haemophagocytic lymphohistiocytosis vs methotrexate toxicity - a diagnostic conundrum. 继发性噬血细胞淋巴组织细胞增多症与甲氨蝶呤毒性-一个诊断难题。
Pub Date : 2023-04-01 DOI: 10.1177/20542704231155815
Leah Hawkins, Jack Gibbs, Connor MacMillan

A 74-year-old female with a background of rheumatoid arthritis, managed with weekly methotrexate, was admitted with; oedema, dyspnoea, cachexia and jaundice. Bloods revealed pancytopenia, hyperferritinaemia, hyperbilirubinaemia, hypoalbuminaemia and hypofolataemia. Imaging showed a large right-sided pleural effusion, requiring therapeutic aspiration, and splenomegaly. Bone marrow aspirate revealed haemophagocytosis. Differential diagnoses included methotrexate toxicity (MTXT) and haemophagocytic lymphohistiocytosis (HLH). Management was initiated for MTXT whilst ongoing investigation for possible HLH continued.

74岁女性,类风湿关节炎背景,每周接受甲氨蝶呤治疗。水肿、呼吸困难、恶病质、黄疸。血液显示全血细胞减少症、高铁血症、高胆红素血症、低白蛋白血症和低叶酸血症。影像显示右侧大量胸腔积液,需要治疗性抽吸和脾肿大。骨髓抽吸显示噬血细胞症。鉴别诊断包括甲氨蝶呤毒性(MTXT)和噬血细胞淋巴组织细胞增多症(HLH)。已开始对MTXT进行管理,同时仍在继续对可能的HLH进行调查。
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引用次数: 1
Area postrema syndrome: Intractable hiccups and vomiting as a result of neuromyelitis Optica Spectrum disorder. 区域后遗症综合征:顽固性呃逆和呕吐的结果神经脊髓炎视谱障碍。
Pub Date : 2023-04-01 DOI: 10.1177/20542704231159601
Kiran Prabhu, Marc Woodman

A 31 year old woman was referred to the medical team for further investigation of intractable hiccups and vomiting. Initial investigations including blood tests, endoscopy and CT imaging did not identify any cause of symptoms. Following multidisciplinary team review, serial MRI Head imaging was arranged, which revealed progressive posterior fossa signal abnormality with involvement of the area postrema. In combination with a positive serum Aquaporin-4 antibody result, this helped establish a diagnosis of Neuromyelitis Optica Spectrum Disorder (NMOSD). Treatment included high dose steroids, plasma exchange and immunomodulatory therapy, and led to a marked improvement in symptoms. This case highlights the importance of utilising specialty team input and broadening lines of investigation, when managing patients with intractable hiccups and vomiting in whom an initial workup has not established a clear diagnosis. While NMOSD is rare, early identification can inform treatment strategies that may lead to a significant improvement in clinical outcome.

一名31岁妇女因顽固性呃逆和呕吐被转诊至医疗小组作进一步调查。包括血液检查、内窥镜检查和CT成像在内的初步调查没有发现任何症状的原因。在多学科团队复查后,安排了一系列MRI头部成像,发现后颅窝信号进行性异常并累及后脑区。结合血清水通道蛋白-4抗体阳性结果,这有助于建立神经脊髓炎视谱障碍(NMOSD)的诊断。治疗包括高剂量类固醇、血浆交换和免疫调节治疗,症状明显改善。本病例强调了利用专业团队的投入和扩大调查线的重要性,当管理顽固性打嗝和呕吐的患者,在最初的检查没有建立一个明确的诊断。虽然NMOSD很少见,但早期识别可以为治疗策略提供信息,从而显著改善临床结果。
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引用次数: 1
Collision metastasis from prostate adenocarcinoma and pancreatic ductal adenocarcinoma to a lung nodule. 前列腺腺癌和胰腺导管腺癌碰撞转移至肺部结节。
Pub Date : 2023-03-07 eCollection Date: 2023-03-01 DOI: 10.1177/20542704231153562
Ahmed Mohamed, Mark Yao, Sonia Saw, Amanda Clark, Sanjeev Madaan

Introduction: Prostate cancer and pancreatic cancer are often complex pathologies that affect millions of patients worldwide. However, the incidence of a distant collision metastasis of both malignancies remains a rare and often poorly documented incidence.

Case presentation: A 75-year-old male patient with past history of prostate cancer on maximal androgen blockade was recently diagnosed with pancreatic cancer for which he underwent radical surgical resection of pancreas with curative intent. A metastatic lung nodule was noted on surveillance CT imaging and, subsequently, biopsied. A diagnosis of collision metastasis from two distinct primary malignancies was histopathologically confirmed following immunohistochemical evaluation.

Conclusion: We report the first case of collision metastasis of prostate and pancreatic adenocarcinomata to a lung nodule to the best of our knowledge.

导言:前列腺癌和胰腺癌通常是一种复杂的病症,影响着全球数百万患者。然而,这两种恶性肿瘤的远处碰撞转移的发生率仍然很罕见,而且往往记录不全:一名 75 岁的男性患者既往有前列腺癌病史,接受过最大程度的雄激素阻断治疗,最近被诊断出患有胰腺癌,并接受了根治性胰腺手术切除。他在监测 CT 成像时发现了一个转移性肺结节,随后进行了活检。经免疫组化评估后,组织病理学确诊为来自两种不同原发恶性肿瘤的碰撞转移:据我们所知,我们报告了首例前列腺癌和胰腺癌碰撞转移至肺部结节的病例。
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引用次数: 0
The impact of "freedom day" on COVID-19 health protective behaviour in England: An observational study of hand hygiene, face covering use and physical distancing in public spaces pre and post the relaxing of restrictions. 自由日 "对英格兰 COVID-19 健康保护行为的影响:对放宽限制前后公共场所手部卫生、面部覆盖物的使用和身体距离的观察研究。
Pub Date : 2023-03-06 eCollection Date: 2023-03-01 DOI: 10.1177/20542704231153563
Rachel Davies, Alex F Martin, Louise E Smith, Fiona Mowbray, Lisa Woodland, Richard Amlôt, G James Rubin

Objectives: To study the prevalence of COVID-19 health protective behaviours before and after rules eased in England on the 19th July 2021.

Design: Observational study pre (12th-18th July) and post (26th July-1st August) 19th July, and a cross-sectional online survey (26th to 27th July).

Setting: Observations occurred in supermarkets (n = 10), train stations (n = 10), bus stops (n = 10), a coach station (n = 1) and a London Underground station (n = 1). The survey recruited a nationally representative sample.

Participants: All adults entering the observed locations during a one-hour period (n = 3819 pre- and n = 2948 post-19th July). In the online survey, 1472 respondents reported having been shopping for groceries/visited a pharmacy and 566 reported having used public transport or having been in a taxi/minicab in the last week.

Main outcome measures: We observed whether people wore a face covering, maintained distance from others and cleaned their hands. We investigated self-reports of wearing a face covering while in shops or using public transport.

Results: In most locations observed, the proportion of people wearing face coverings, cleaning the hands and maintaining physical distance declined post 19th July. Pre 19th July, 70.2% (95% CI 68.7 to 71.7%) of people were observed to be wearing a face covering versus 55.8% (54.2 to 57.9%) post 19th July. Equivalent rates for physical distancing were 40.9% (39.0 to 42.8%) versus 29.5% (27.4 to 31.7%), and for hand hygiene were 4.4% (3.8 to 5.1%) versus 3.9% (3.2 to 4.6%). Self-reports of "always" wearing face coverings were broadly similar to observed rates.

Conclusions: Adherence to protective behaviours was sub-optimal and declined during the relaxation of restrictions, despite appeals to exercise caution. Self-reports of "always" wearing a face covering in specific locations appear valid.

目标:研究 2021 年 7 月 19 日英格兰放宽规则前后 COVID-19 健康保护行为的流行情况:研究 2021 年 7 月 19 日英格兰放宽规定前后 COVID-19 健康保护行为的流行情况:观察研究:7 月 19 日之前(7 月 12 日至 18 日)和之后(7 月 26 日至 8 月 1 日),以及横断面在线调查(7 月 26 日至 27 日):观察地点:超市(10 个)、火车站(10 个)、公共汽车站(10 个)、长途汽车站(1 个)和伦敦地铁站(1 个)。调查招募了具有全国代表性的样本:所有在一小时内进入观察地点的成年人(7 月 19 日前为 3819 人,7 月 19 日后为 2948 人)。在在线调查中,有 1472 名受访者表示在上周曾去买过日用品/逛过药店,566 名受访者表示在上周曾使用过公共交通工具或乘坐过出租车/小巴:我们观察了人们是否佩戴面罩、是否与他人保持距离以及是否清洁双手。我们还调查了在商店或乘坐公共交通工具时戴面具的自我报告:在观察到的大多数地点,7 月 19 日后戴面罩、清洁双手和保持身体距离的人数比例都有所下降。7 月 19 日之前,70.2%(95% CI 68.7 至 71.7%)的人戴面罩,而 7 月 19 日之后为 55.8%(54.2 至 57.9%)。身體保持距離的比率為 40.9%(39.0 至 42.8%)對比 29.5%(27.4 至 31.7%),手部衞生的比率為 4.4%(3.8 至 5.1%)對比 3.9%(3.2 至 4.6%)。关于 "总是 "佩戴面部覆盖物的自我报告与观察到的比率大致相似:结论:尽管呼吁人们谨慎行事,但保护行为的坚持率并不理想,而且在放宽限制期间有所下降。在特定地点 "总是 "佩戴面罩的自我报告似乎是有效的。
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引用次数: 0
Acute subcutaneous emphysema: A rare clinical presentation of large bowel perforation. 急性皮下气肿:大肠穿孔的罕见临床表现。
Pub Date : 2023-02-08 eCollection Date: 2023-02-01 DOI: 10.1177/20542704231153529
Matthew Dunckley, Frank Smedley, Brian Andrews

These atypical presentations of colonic perforation illustrate the importance of considering acute intra-abdominal pathology when subcutaneous emphysema is identified.

这些结肠穿孔的非典型表现说明,当发现皮下气肿时,考虑急性腹腔内病变的重要性。
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引用次数: 0
Long recurrence-free survival of localized rectal melanoma after abdominoperineal resection in comparison to partial excision and highlighting the place of immunotherapy: A case report. 与部分切除术相比,腹腔镜切除术后局部直肠黑色素瘤的无复发生存期更长,并突出了免疫疗法的地位:病例报告。
Pub Date : 2023-02-06 eCollection Date: 2023-02-01 DOI: 10.1177/20542704221148059
Othmane Bourouail, Noureddine Njoumi, Youssef Elmahdaouy, Mohamed Fahssi, Mbarek Yaka, Abderrahmane Hejjouji, Abdelmounaim Ait Ali

Rectal malignant melanomas (RMM) are remarkably uncommon, the rectal location represents less than one percent of all rectal cancer. Because of its low global survival rate, the surgical strategy is a subject of controversy for attaining a r0 resection. the palliative treatment is also debateable, but recently; radiotherapy and immunotherapy became the preferred treatment and offer the best outcome. to ensure r0 resection, abdominoperineal resection (apr) is considered the main surgical option, but because of its morbidity and functional limitations, local excision techniques such as endoscopic mucosal resection (emr) combined with chemoradiotherapy are being increasingly performed to preserve the quality of life and reduce local recurrence rate. In this report, we evaluate the place that apr still keeps as surgical procedure in comparison to partial excision. we report a case of a 72 years old patient, who presented rectal syndrome with rectorrhagia for 2 months, the diagnosis of localized rectal melanoma was confirmed by endoscopy, magnetic resonance imaging, histological analysis tissue with immunohistochemistry. the procedure strategy was a surgical treatment with apr. After a long recurrence-free survival period, the patient develops local recurrence and immunotherapy-resistant metastasis.

直肠恶性黑色素瘤(RMM)非常罕见,直肠部位的恶性黑色素瘤占所有直肠癌的比例不到百分之一。由于其全球生存率较低,手术策略在实现 r0 切除方面存在争议。姑息治疗也存在争议,但最近,放射治疗和免疫治疗成为首选治疗方法,并取得了最佳疗效。为确保 r0 切除,腹会阴部切除术(abdominoperineal resection,apr)被认为是主要的手术选择,但由于其发病率和功能限制,越来越多的人开始采用内镜粘膜切除术(endoscopic mucosal resection,emr)结合放化疗等局部切除技术,以保持患者的生活质量并降低局部复发率。我们报告了一例 72 岁患者的病例,该患者出现直肠综合征并伴有 2 个月的排便困难,经内镜检查、磁共振成像、免疫组化组织学分析确诊为局部直肠黑色素瘤。经过较长的无复发生存期后,患者出现局部复发和免疫治疗耐药转移。
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引用次数: 0
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