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CANDOR - A Patient Safety, Medical Liability, and Healthcare Professional Wellness Program 坦率-病人安全,医疗责任和医疗保健专业健康计划
Pub Date : 2023-01-01 DOI: 10.18103/mra.v11i2.3434
F. LeCraw
The Communication and Optimal Resolution (CANDOR) program, a patient safety, medicolegal, and healthcare professional wellness program, has been implemented or in the process of being implemented in over 800 US hospitals. The program was designed to help patients suffering from unexpected adverse outcomes and healthcare professionals whose patients experience injury while under their care. The CANDOR program’s basic premise emphasizes honesty in medical error situations. It aims to prevent the recurrence of medical errors. Studies have found that, compared to the common US practice known as “Deny, Delay, and Defend,” using CANDOR not only benefits current and future patients but can improve the well-being of healthcare professionals. This paper therefore aimed to describe CANDOR and evidence regarding its effects on patients and the healthcare professional’s well-being at hospitals that implemented it in comparison to their previous practice of “Deny, Delay, and Defend.” This paper describes methods used by CANDOR teams, which include physicians, attorneys, patient advocates, and health policy leaders, in their successful endeavor to persuade hospital leaders to implement it, in essence to change the hospital’s culture from a culture of opaqueness regarding medical errors and their consequences to a culture embracing transparency. This paper also describes obstacles the teams faced in their endeavor to implement CANDOR at their institution and how they surmounted the obstacles.
沟通和最佳解决方案(CANDOR)计划是一项患者安全、医学法律和医疗保健专业健康计划,已在美国800多家医院实施或正在实施。该计划旨在帮助患有意想不到的不良后果的患者和医疗保健专业人员,他们的患者在他们的护理下遭受伤害。CANDOR项目的基本前提是强调在医疗差错情况下的诚实。它的目的是防止医疗事故再次发生。研究发现,与美国常见的“否认、拖延和辩护”做法相比,使用坦率不仅有利于当前和未来的患者,还可以改善医疗保健专业人员的福祉。因此,本文旨在描述坦诚和证据,关于其对患者和医疗保健专业人员的健康的影响,在医院实施它,与他们以前的做法“否认,延迟,和辩护。”本文描述了CANDOR团队使用的方法,其中包括医生、律师、患者倡导者和卫生政策领导者,他们成功地说服医院领导实施它,从本质上改变医院文化,从医疗错误及其后果的不透明文化转变为拥抱透明的文化。本文还描述了团队在其机构中努力实现坦率所面临的障碍,以及他们如何克服这些障碍。
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引用次数: 0
Relevance of Preventive Measures against COVID-19 and Other Communicable Diseases for University Employees and Students Returning to Face-to-Face Teaching 新冠肺炎等传染病预防措施与高校职工和学生重返面授教学的相关性
Pub Date : 2023-01-01 DOI: 10.18103/mra.v11i8.4114
Emilyn Munar, Wilfrido Simbul, Aurora Dionisio, Josefa Turingan
This study links health protocol awareness and appreciation to communicable disease prevention in higher education institution in Santiago City, Isabela, Philippines. If employees and students understand the health regulations and a majority of respondents approve and are more likely to follow, COVID-19 and other infectious diseases can be prevented. This descriptive-correlational cross-sectional study had 368 participants. The researchers prepared a more extensive questionnaire to analyze employees and students returning to face-to-face schooling in 2021-2022's trust and reliance in Covid-19 and other communicable disease prevention. Five of the higher education institution's ten leading causes of morbidity are also identified and ranked in the province. Acute respiratory, urinary, lower respiratory, and skin infections are contagious. Hypertension ranks fifth. The higher education institution's employee and student population has reached the desired herd immunity level, which is much higher than the Regional level; Group I's health problems have been alleviated by the anti-Covid preventive measles vaccination; and all five diseases are preventable, making them good targets for preventive medicine. The findings showed that "There is no significant difference between health data obtained from a general population of a region of the country and that of a school population in terms of the leading causes of morbidity and the current Covid-19 vaccination rate of the vulnerable population" and that "There is no significant difference between the level of appreciation of the relevance of health protocols between a segment of the population in a higher education institution grouped as those with health.”
本研究将菲律宾伊莎贝拉市圣地亚哥高等教育机构的卫生协议意识和赞赏与传染病预防联系起来。如果员工和学生了解卫生法规,并且大多数受访者认可并更有可能遵守这些法规,就可以预防COVID-19和其他传染病。这项描述性相关横断面研究有368名参与者。研究人员准备了一份更广泛的调查问卷,以分析2021-2022年重返面对面学校的员工和学生对Covid-19和其他传染病预防的信任和依赖。在高等教育机构的十大主要致病原因中,有五个也被确定并在该省排名。急性呼吸道、泌尿系统、下呼吸道和皮肤感染具有传染性。高血压排名第五。高校职工和学生群体免疫水平达到预期水平,远高于地区水平;第一组人群的健康问题通过抗疫预防麻疹疫苗得到缓解;这五种疾病都是可以预防的,这使它们成为预防医学的良好目标。研究结果显示,“在发病的主要原因和当前易感人群的Covid-19疫苗接种率方面,从该国一个地区的一般人群中获得的健康数据与从学校人群中获得的健康数据之间没有显著差异”,“高等教育机构中一部分人群对健康方案相关性的认识水平之间没有显著差异。健康。”
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引用次数: 0
Microscopic Manifestations of Maternal Diabetes in Placental Structure 母体糖尿病在胎盘结构中的显微表现
Pub Date : 2023-01-01 DOI: 10.18103/mra.v11i7.2.4141
M. Jirkovská
The human placenta is crucial for prenatal development and good pregnancy outcome. Maternal diabetic disorder influences normal intrauterine development of individual and expresses itself in the placenta. Due to the progress in diagnostic methods and methods of metabolic compensation the macroscopic signs associated with maternal diabetes, e.g., placentomegaly, occur nowadays rarely. In the microscopic picture of placenta there are no structural differences specific for maternal diabetes. However, the application of quantitative morphological methods has revealed some differences of normal and diabetic placenta. Stereological studies have shown significantly higher total volume of peripheral villi that distorts shapes and dimensions of pores in the intervillous space, and larger surface areas of peripheral villi and villous capillaries. Methods of confocal microscopy and 3D reconstruction gave the evidence that the fetus reacts on the hypoxia in maternal diabetic environment by enhanced angiogenesis and branching of villous capillaries. The enhanced tissue volumes are conditioned by higher mitotic activity. However, it decreases the proliferative potential of cells taking part in the enlargement of key structures for maternofetal transport at the end of pregnancy. Syncytiotrophoblast produces many factors playing important roles in maternal and fetal regulation and in the placenta itself. Quantitative methods of catalytic histochemistry and immunocytochemistry pointed at the role of maternal diabetes in decreased synthesis of some of those factors (e.g., alkaline phosphatase, SP1 glycoprotein). The presented data show that the quantitative morphological methods contribute to better understanding of the influence of maternal diabetes on fetoplacental unit.
人胎盘对产前发育和妊娠结局至关重要。母体糖尿病紊乱影响个体正常的宫内发育,并在胎盘中表达。由于诊断方法和代谢代偿方法的进步,目前与母体糖尿病相关的宏观体征,如胎盘肿大,很少发生。在显微镜下的胎盘图片中,没有母体糖尿病特有的结构差异。然而,定量形态学方法的应用揭示了正常胎盘与糖尿病胎盘的一些差异。体视学研究表明,外周绒毛的总体积显著增加,从而扭曲了绒毛间隙中孔隙的形状和尺寸,外周绒毛和绒毛毛细血管的表面积也显著增加。共聚焦显微镜和三维重建的方法证明,胎儿对缺氧的母体糖尿病环境的反应是通过增强血管生成和绒毛毛细血管分支。组织体积的增加是由更高的有丝分裂活性决定的。然而,它降低了在妊娠末期参与母胎运输关键结构扩大的细胞的增殖潜力。合体滋养细胞产生许多在母胎调节和胎盘本身中起重要作用的因子。催化组织化学和免疫细胞化学的定量方法指出了母亲糖尿病在这些因子(如碱性磷酸酶,SP1糖蛋白)合成减少中的作用。这些数据表明,定量形态学方法有助于更好地了解母体糖尿病对胎儿胎盘单位的影响。
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引用次数: 0
Stakeholder Engagement to Drive Iterative Software Development of the ART-Access Web-Based Application for Community Pharmacy Dispensing of Anti-Retroviral Therapy 利益相关方参与推动ART-Access基于web的社区药房分配抗逆转录病毒治疗应用程序的迭代软件开发
Pub Date : 2023-01-01 DOI: 10.18103/mra.v11i1.3378
E. Kyomugisha, M. Balaba, Eva Nakibuuka, R. King, R. Parkes-Ratanshi
Introduction: Significant advances in combination anti-retroviral therapy have been instrumental in improving the quality of lives and life expectancy for people living with HIV. However, in the absence of a cure, sustained investment and innovation is required to improve adherence and quality of life for people living with HIV. We developed ARTAccess, a web-based application that links patient information on anti-retroviral therapy and viral load to an algorithm that guides a private community pharmacist on anti-retroviral therapy refills without the need for an additional nurse in the pharmacy. The aim of this paper is to describe the present the development process of the ARTAccess application and the exploration of the perceptions about its use by end users. Methods: Between October–December 2018, we conducted a qualitative observational study to document the processes of the ART-Access™ application development. Using theoretical frameworks of participatory action research and human-centred design, we undertook structured and unstructured observations of the application development review meetings. We observed and had interactions in 12 stakeholder meetings. Three observers attended each development meeting and independently drafted a reflective narration of the transcript and separately conducted their own analyses. ARTAccess was launched in January 2019 and in March 2019, three in-depth interviews were conducted with the nurse dispensers running the refill program at the three pharmacies where ARTAccess was piloted. Results: The ARTAccess application development meetings generated emerging themes. Introduction of a mHealth application for efficiency introduced job insecurity fears of health workers which needed to be addressed, to allow for increased engagement by health worker stakeholders. Stakeholder meetings provided important perceived gaps and needs for improvement at each stage of the ARTAccess application development. The user-centred design process led to five application versions; three more than the two originally planned; the feedback on the ARTAccess application became more positive as later versions were presented to stakeholders. Conclusions: The study provides evidence that participatory action research in a human-centred design approach enhanced the application development process of a new technology for health. In resource limited settings, where digital technologies may be used to support overstretched health systems, health workers need re-assurance that digital tools being developed will not threaten their employment.
导言:联合抗逆转录病毒疗法的重大进展有助于改善艾滋病毒感染者的生活质量和预期寿命。然而,在没有治愈方法的情况下,需要持续的投资和创新,以改善艾滋病毒感染者的依从性和生活质量。我们开发了ARTAccess,这是一个基于网络的应用程序,它将患者关于抗逆转录病毒治疗和病毒载量的信息与一种算法联系起来,该算法可以指导私人社区药剂师在不需要额外护士的情况下进行抗逆转录病毒治疗。本文的目的是描述目前ARTAccess应用程序的开发过程,并探讨最终用户对其使用的看法。方法:在2018年10月至12月期间,我们进行了一项定性观察研究,以记录ART-Access™应用程序开发的过程。使用参与式行动研究和以人为本设计的理论框架,我们对应用程序开发审查会议进行了结构化和非结构化的观察。我们在12次利益相关者会议上进行了观察和互动。三名观察员出席了每次发展会议,并独立起草了一份对会议记录的反思叙述,并分别进行了他们自己的分析。ARTAccess于2019年1月启动,2019年3月,我们对在ARTAccess试点的三家药店负责补充项目的护士配药员进行了三次深入采访。结果:ARTAccess应用程序开发会议产生了新兴主题。为提高效率而推出的移动医疗应用程序引发了卫生工作者对工作不安全的担忧,这一担忧需要得到解决,从而使卫生工作者利益攸关方能够更多地参与进来。涉众会议在ARTAccess应用程序开发的每个阶段提供了重要的可感知的差距和改进需求。以用户为中心的设计过程产生了五个应用程序版本;比原计划的两个增加了三个;随着后期版本呈现给涉众,对ARTAccess应用程序的反馈变得更加积极。结论:本研究提供证据表明,以人为本设计方法的参与性行动研究促进了卫生新技术的应用开发过程。在资源有限的情况下,数字技术可用于支持超负荷运转的卫生系统,卫生工作者需要再次得到保证,即正在开发的数字工具不会威胁到他们的就业。
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引用次数: 0
Bidirectional Regulation of NAD(P)H Quinone Dehydrogenase 1 Expression in Mouse Hepatic Stellate Cells- Acute versus Long-Term Ethanol Exposure 小鼠肝星状细胞NAD(P)H醌脱氢酶1表达的双向调控——急性与长期乙醇暴露
Pub Date : 2023-01-01 DOI: 10.18103/mra.v11i2.3586
H. Yang, Ningya Zhang, Zhongmao Guo
Conclusions: Our findings suggest that ethanol upregulates NQO1 expression by activating the AhR-Nrf2 pathway. Long-term ethanol exposure sustains low level of AhR protein and diminishes the inducibility of its target genes Nrf2 and NQO1 by BaP. These findings will contribute to understanding the synergistic toxicity of ethanol and polycyclic aromatic hydrocarbon compounds, such as BaP.
结论:我们的研究结果表明,乙醇通过激活AhR-Nrf2通路上调NQO1的表达。长期乙醇暴露使AhR蛋白维持在低水平,并降低其靶基因Nrf2和NQO1被BaP诱导的能力。这些发现将有助于理解乙醇和多环芳烃化合物(如BaP)的协同毒性。
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引用次数: 0
Asthma in Children with Sickle Cell Disease 镰状细胞病儿童哮喘
Pub Date : 2023-01-01 DOI: 10.18103/mra.v11i5.3567
O. Sikdar, A. Greenough
Sickle Cell Disease is a life-threatening hereditary blood disorder which affects millions of people worldwide. Pulmonary complications are important causes of morbidity and mortality in patients with sickle cell disease. Asthma is a recognised comorbidity of sickle cell disease and may occur in between 15 and 28% of children with sickle cell disease. It has been associated with increased episodes of acute chest syndrome and all cause mortality. Obstructive lung disease, however, is common in children with sickle cell disease, independent of an asthma diagnosis. This review explores the pathophysiology, diagnosis and therapeutic opportunities for asthma in sickle cell disease patients. The diagnostic challenges and inconsistencies in current clinical approaches are highlighted. Convergence of inflammatory pathways in sickle cell disease and asthma occurs, but there is also a heightened level of inflammation unique to sickle cell disease. Thus, wheezing may not be due to asthma but be a manifestation of sickle cell disease per se and the result of the increased pulmonary vascular volume. As a consequence, anti-asthma therapy may not be appropriate for all wheezy children with sickle cell disease and commencing treatment on the basis of a physician’s diagnosis alone is inappropriate. Data from paediatric cohorts suggest use of spirometry, aeroallergen sensitisation tests, impulse oscillometry and dedicated interdisciplinary pulmonary clinics could improve diagnosis accuracy. Corticosteroids and bronchodilators are well-established treatments for asthma; observational studies suggest they may provide benefit for some children with sickle cell disease, but therapies such as hydroxyurea may improve respiratory outcomes in others. It is, therefore, essential children are thoroughly investigated and followed-up and a personalised approach taken to their care. Prospective randomised studies are required to establish the effectiveness of asthma therapies in children with sickle cell disease.
镰状细胞病是一种危及生命的遗传性血液疾病,影响着全世界数百万人。肺部并发症是镰状细胞病患者发病和死亡的重要原因。哮喘是镰状细胞病公认的合并症,可能在15%至28%的镰状细胞病患儿中发生。它与急性胸综合征发作和全因死亡率增加有关。然而,阻塞性肺病在镰状细胞病患儿中很常见,独立于哮喘诊断。本文综述镰状细胞病患者哮喘的病理生理学、诊断和治疗机会。诊断的挑战和不一致,目前的临床方法强调。镰状细胞病和哮喘发生炎症途径的趋同,但镰状细胞病特有的炎症水平也较高。因此,喘息可能不是由于哮喘,而是镰状细胞病本身的表现和肺血管体积增加的结果。因此,抗哮喘治疗可能不适合所有患有镰状细胞病的喘息儿童,仅根据医生的诊断开始治疗是不合适的。来自儿科队列的数据表明,使用肺活量测定法、空气过敏原致敏试验、脉冲振荡测定法和专门的跨学科肺部诊所可以提高诊断的准确性。皮质类固醇和支气管扩张剂是公认的治疗哮喘的药物;观察性研究表明,它们可能对一些患有镰状细胞病的儿童有益,但羟基脲等疗法可能会改善其他儿童的呼吸结果。因此,必须对儿童进行彻底调查和跟踪,并对他们采取个性化的照顾方法。需要前瞻性随机研究来确定哮喘治疗对镰状细胞病儿童的有效性。
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引用次数: 0
Good Things that have Come out of the Pandemic: Between Steppingstones and Comfort Zone 大流行带来的好事:在垫脚石和舒适区之间
Pub Date : 2023-01-01 DOI: 10.18103/mra.v11i5.3553
R. Hoek
The risk scenarios supply chain managers encountered during the COVID19 pandemic were unique and provides many opportunities for learning and the development of more robust risk management approaches and strategies. In this reflection we offer five lessons that provide stepping stones for supply chain managers seeking to create more resilient supply chains. These lessons are: (1) the need to move from relational to social contracting, (2) the need to move from compliance to a force for good, (3) the need to move from planned optimization to agile resiliency, (4) the need to move from ERP and reporting the past to visibility into realtime and (5) the need to resist tendency to revert back to comfort zones. In this paper we reflect upon these lessons aiming to inspire progress in industry and research.
供应链管理人员在2019冠状病毒病大流行期间遇到的风险情景是独特的,为学习和制定更强有力的风险管理方法和战略提供了许多机会。在这个反思中,我们提供了五个经验教训,为寻求创建更有弹性的供应链的供应链管理者提供了垫脚石。这些经验教训是:(1)需要从关系契约转向社会契约,(2)需要从遵从性转向善的力量,(3)需要从计划优化转向敏捷弹性,(4)需要从ERP和报告过去转向实时可见性,以及(5)需要抵制回归舒适区的趋势。在本文中,我们对这些经验教训进行了反思,旨在激励工业和研究的进步。
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引用次数: 0
Diagnostic Significance of Eosinophilic Pleural Effusion 嗜酸性胸腔积液的诊断意义
Pub Date : 2023-01-01 DOI: 10.18103/mra.v11i4.3581
A. Casalini, P. Mori, R. Pisi, Federico Maniscalco, M. Corradi, M. Goldoni
A pleural effusion is defined as eosinophilic when eosinophils represent ≥ 10% of the total nucleated cell count, and accounts for approximately 10% of all pleural effusions. The diagnostic significance of eosinophilic pleural effusion has yet to be determined. Objective and Methods: A retrospective study was conducted on 65 patients with eosinophilic pleural effusion to evaluate the correlation between the percentage of eosinophils present in the pleural fluid and the benign or malignant nature of the effusion. An original aspect of current study was the evaluation of other variables in association with pleural eosinophilia, in particular pleural fluid lymphocytosis (≥ 50%), and the presence or absence of fever. Results: Data showed the trend towards a decrease in neoplastic incidence with increasing percentages of eosinophilic counts, although this correlation was not statistically significant. The presence of fever correlated with low incidence of neoplasms (10% of neoplastic effusions in patients with fever) and was the most significant variable (p=0.001), with a Negative Predictive Value of neoplastic disease of 90%, with sensitivity 92.6% and specificity 47.4%. When evaluated together with fever, eosinophils increased their discriminating sensitivity to the benign or malignant nature of the effusion but lost in specificity. When evaluated as absence or presence of lymphocytosis (≥50% lymphocytes), associated with eosinophilia, lymphocytes were significantly associated with the neoplastic nature of the effusion. Conclusions: the study showed that the finding of eosinophilic pleural effusion should not be considered an indicator of benignity of the effusion; the association of other parameters with eosinophilia, lymphocytosis of the pleural fluid and fever can provide more precise prognostic indications; a high percentage of eosinophils, the absence of lymphocytosis and the presence of fever would seem to be associated with a low probability of a neoplastic nature of the effusion.
当嗜酸性粒细胞占有核细胞总数的10%以上,约占所有胸腔积液的10%时,定义为嗜酸性。嗜酸性胸腔积液的诊断意义尚未确定。目的和方法:对65例嗜酸性胸腔积液患者进行回顾性研究,评价胸腔积液中嗜酸性粒细胞百分比与胸腔积液良恶性的相关性。本研究的一个原始方面是评估与胸膜嗜酸性粒细胞增多症相关的其他变量,特别是胸膜液淋巴细胞增多症(≥50%),以及有无发热。结果:数据显示,随着嗜酸性粒细胞计数百分比的增加,肿瘤发病率呈下降趋势,尽管这种相关性没有统计学意义。发热与肿瘤的低发病率相关(发热患者中肿瘤积液的10%),是最显著的变量(p=0.001),肿瘤疾病的阴性预测值为90%,敏感性92.6%,特异性47.4%。当与发热一起评估时,嗜酸性粒细胞增加了对积液的良恶性的鉴别敏感性,但失去了特异性。当评估为没有或存在淋巴细胞增多(≥50%淋巴细胞),伴有嗜酸性粒细胞增多时,淋巴细胞与积液的肿瘤性质显著相关。结论:研究表明,嗜酸性胸腔积液的发现不应被视为积液良性的指标;其他参数与嗜酸性粒细胞增多、胸水淋巴细胞增多和发热的关系可以提供更精确的预后指征;嗜酸性粒细胞比例高,淋巴细胞增多症不存在和发热的存在似乎与低可能性的肿瘤性质的积液有关。
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引用次数: 0
Disease-Specific Treatment For Primary Membranous Nephropathy: The Role of Monoclonal Antibodies 原发性膜性肾病的疾病特异性治疗:单克隆抗体的作用
Pub Date : 2023-01-01 DOI: 10.18103/mra.v11i3.3601
P. Ruggenenti
Primary Membranous Nephropathy is an autoimmune disease caused by the deposition of Immunoglobulin G and complement components on the subepithelial layer of the glomerular capillary wall. It affects 5-10 patients per million population and is the second cause of nephrotic syndrome in adults after diabetic kidney disease. For decades steroids and non-specific immunosuppressive medications have been advocated as a therapeutic option for patients with membranous nephropathy at increased risk of kidney failure because of persistent nephrotic syndrome. These medications, however, have major and potentially fatal adverse effects that offset their potential benefits and should be abandoned. The discovery of nephritogenic autoantibodies against podocyte M-type phospholipase A2 receptor (PLA2R) and thrombospondin type-1 domain-containing protein 7A (THSD7A) antigens provided a clear pathophysiological rationale for interventions specifically targeting B cell lineages to prevent antibody production and subepithelial deposition. The first-in-class anti-CD20 monoclonal antibody rituximab is safe and achieves remission in approximately two-thirds of patients with nephrotic membranous nephropathy. In PLA2R-related disease, remission is invariably preceded by depletion of anti PLA2R autoantibodies and relapse by their re-emergence into the circulation. Because of its superior risk/benefit profile as compared to non-specific immunosuppressive therapy, rituximab is now first-line therapy for patients with membranous nephropathy at risk of kidney failure. Novel monoclonal antibodies targeting CD20 cells (such as ofatumumab and obinutuzumab) and their differentiation (belimumab) or targeting long-living antibody producing CD38 memory cells (daratumumab, felzartamab) along with proteasome inhibitors such as bortezomib are being evaluated for the treatment of nephrotic patients with membranous nephropathy who are resistant or intolerant to rituximab. Complement inhibitor therapy might serve to stop the glomerular inflammatory process until the benefits of these medications become effective. Thus, major advances in the understanding of the mechanisms of membranous nephropathy have led to novel treatment perspectives. The integrated evaluation of serum autoantibody titer and proteinuria, together with serum albumin levels in patients with overt nephrotic syndrome, could guide diagnosis of membranous nephropathy and individually tailored treatment protocols. The introduction of monoclonal antibodies targeting disease-specific mechanisms will pave the way for a novel therapeutic paradigm based on the principle of precision medicine and personalized therapy.
原发性膜性肾病是由免疫球蛋白G和补体成分沉积在肾小球毛细血管壁上皮下层引起的自身免疫性疾病。每百万人中有5-10名患者,是继糖尿病肾病之后导致成人肾病综合征的第二大原因。几十年来,类固醇和非特异性免疫抑制药物一直被提倡作为膜性肾病患者的治疗选择,这些患者由于持续性肾病综合征而导致肾衰竭的风险增加。然而,这些药物有严重和潜在致命的副作用,抵消了它们潜在的益处,应该放弃。针对足细胞m型磷脂酶A2受体(PLA2R)和血小板反应蛋白1型结构域蛋白7A (THSD7A)抗原的肾源性自身抗体的发现,为特异性靶向B细胞谱系的干预提供了明确的病理生理学依据,以防止抗体的产生和上皮下沉积。抗cd20单克隆抗体利妥昔单抗是安全的,并且在大约三分之二的肾病膜性肾病患者中获得缓解。在PLA2R相关疾病中,缓解总是发生在抗PLA2R自身抗体的消耗和它们重新出现在循环中而复发之前。由于与非特异性免疫抑制治疗相比,利妥昔单抗具有更高的风险/获益,因此目前利妥昔单抗是有肾衰竭风险的膜性肾病患者的一线治疗。针对CD20细胞(如ofatumumab和obinutuzumab)及其分化(belimumab)或靶向产生CD38记忆细胞的长效抗体(daratumumab, felzartamab)以及蛋白酶体抑制剂(如硼替佐米)的新型单克隆抗体正在被评估用于治疗对利妥昔单抗耐药或不耐受的膜性肾病患者。补体抑制剂治疗可能有助于阻止肾小球炎症过程,直到这些药物的益处变得有效。因此,对膜性肾病机制的理解取得了重大进展,带来了新的治疗前景。综合评价显性肾病综合征患者的血清自身抗体滴度、蛋白尿和血清白蛋白水平,可以指导膜性肾病的诊断和个体化治疗方案。针对疾病特异性机制的单克隆抗体的引入将为基于精准医学和个性化治疗原则的新型治疗范式铺平道路。
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引用次数: 0
SC Hemoglobinopathy: a rare case report SC血红蛋白病:罕见病例报告
Pub Date : 2023-01-01 DOI: 10.18103/mra.v11i7.1.4112
Fernanda Zottmann, Jiviane Silva, S. Khatib, Gabriela Guimarães, Julia Coneglian, Bibiana Vinholes, L. Gebrin, C. Borges
In SC hemoglobinopathy, a rare type of sickle cell disease, patients may experience vaso-occlusive phenomena, but in a milder condition than in the SS form, considered classic and the most common among them. This current study aims to present a case of a patient with this rare form of hemoglobinopathy, who received a late diagnosis, and its clinical evolution, including symptoms, treatment and life expectancy based on the literature, but mainly on how the patient is clinically found after seven years of follow-up. It is important to have epidemiological studies about hemoglobinopathies, specially the rare forms, to obtain more information regarding the incidence/prevalence of the disease and clinical manifestations.
SC血红蛋白病是一种罕见的镰状细胞病,患者可能会出现血管闭塞现象,但病情较SS形式轻微,SS形式被认为是经典的,也是最常见的。本研究的目的是介绍一例这种罕见的血红蛋白病的患者,他得到了很晚的诊断,其临床演变,包括症状,治疗和预期寿命,基于文献,但主要是关于患者是如何在7年的随访后被临床发现的。重要的是对血红蛋白病,特别是罕见的血红蛋白病进行流行病学研究,以获得有关该疾病的发病率/流行率和临床表现的更多信息。
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引用次数: 0
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