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Follicle-stimulating hormone sweetness: How carbohydrate structures impact on the biological function of the hormone 促卵泡激素的甜味:碳水化合物结构如何影响激素的生物功能。
IF 4.7 3区 医学 Q1 MEDICINE, RESEARCH & EXPERIMENTAL Pub Date : 2024-10-05 DOI: 10.1016/j.arcmed.2024.103091
Alfredo Ulloa-Aguirre , Regina Llamosas , James A. Dias
Follicle-stimulating hormone (FSH), or follitropin, exists in multiple molecular forms due largely to its protein-carbohydrate composition and the complexity of the glycans attached to the protein core. The heterogeneity of gonadotropins exists in two forms, macroheterogeneity, which results from the absence of one or two oligosaccharide chains in the ß-subunit, and microheterogeneity which results from variation in the structures and complexity of the glycans attached to the hormone. In the clinical arena, FSH compounds are widely used by fertility specialists to promote ovarian follicle growth and maturation to a preovulatory follicle containing a fertilization-competent oocyte. Several genetically engineered recombinant human FSH preparations have been added to the arsenal of follitropin preparations in several countries for the treatment of infertility, particularly in women attending assisted reproduction clinics. Although the primary structure of these recombinant proteins is identical to that of naturally occurring FSH, the cell context and variations in the production and purification processes may impact the glycosidic profile of the recombinant FSH macro- and micro-heterogeneity, which may potentially influence the pharmacokinetics and pharmacodynamics of the compound. This review concentrates on the structure-function correlates of follitropin, with emphasis on the physiological and biological importance of the carbohydrates attached to its protein core, including its pharmacokinetics and biological activity. Emphasis is placed on pituitary FSH, and the available data on the various recombinant FSH preparations employed in therapeutics are also discussed, considering that this gonadotropin represents the cornerstone for the treatment of infertility in modern assisted reproduction.
促卵泡激素(FSH)或称促性腺激素(follitropin)具有多种分子形式,这主要是由于其蛋白质-碳水化合物组成以及附着在蛋白质核心上的聚糖的复杂性。促性腺激素的异质性有两种形式,一种是大异质性,这是因为ß-亚基中缺少一个或两个寡糖链;另一种是微异质性,这是因为附着在激素上的聚糖的结构和复杂性不同。在临床上,生育专家广泛使用 FSH 复合物来促进卵泡生长和成熟,使其成为含有受精能力卵母细胞的排卵前卵泡。一些国家已将几种基因工程重组人 FSH 制剂添加到卵泡刺激素制剂库中,用于治疗不孕症,尤其是辅助生殖诊所的妇女。虽然这些重组蛋白的主要结构与天然存在的 FSH 相同,但细胞环境以及生产和纯化过程中的变化可能会影响重组 FSH 的糖苷谱宏观和微观异构体,从而可能影响化合物的药代动力学和药效学。这篇综述集中探讨了促绒毛膜促性腺激素的结构-功能相关性,重点是附着在其蛋白质核心上的碳水化合物在生理和生物学方面的重要性,包括其药代动力学和生物活性。考虑到垂体促性腺激素是现代辅助生殖中治疗不孕症的基石,本综述将重点放在垂体促性腺激素上,并讨论了治疗中使用的各种重组促性腺激素制剂的现有数据。
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引用次数: 0
Comment on “Serendipitous adrenal hyperplasia in patients admitted to the emergency department for suspected SARS-CoV-2 infection is linked to increased mortality” 就 "急诊科收治的疑似 SARS-CoV-2 感染者偶然出现的肾上腺增生与死亡率升高有关 "发表评论
IF 4.7 3区 医学 Q1 MEDICINE, RESEARCH & EXPERIMENTAL Pub Date : 2024-10-05 DOI: 10.1016/j.arcmed.2024.103088
Hinpetch Daungsupawong , Viroj Wiwanitkit
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引用次数: 0
Design and implementation of a stakeholder consultation process for rapid health technology assessments in Argentina 阿根廷快速卫生技术评估利益相关者磋商程序的设计与实施。
IF 4.7 3区 医学 Q1 MEDICINE, RESEARCH & EXPERIMENTAL Pub Date : 2024-10-05 DOI: 10.1016/j.arcmed.2024.103093
Andrea Alcaraz, Emiliano Navarro, Verónica Alfie, Lucas Perelli, Sebastián García Martí, Agustín Ciapponi, Ariel Bardach, Andrés Pichon-Riviere, Federico Augustovski

Introduction

Health technology assessment (HTA) is a standardized methodology that allows the assessment technologies’ value. By incorporating the perspective of stakeholders in a public consultation process, transparency and quality of decisions can be improved.

Objective

To describe the active stakeholder consultation process for rapid HTAs in an Argentinean, independent, academic, non-profit HTA agency, assessing its initial five years.

Methods

Since 2017, we have been conducting an active public consultation process for rapid HTA documents, inviting producers, healthcare professionals, and patient organizations to provide comments, and their input may lead to changes in the HTA documents. Changes were classified as major (changes to the coverage recommendation), intermediate (changes in efficacy, net benefit, or cost-effectiveness that did not change coverage recommendations), and minor modifications (other changes).

Results

From May 2017–August 2022, 308 rapid HTA (rHTA) reports were published, and 3,438 invitations were sent. 140 rHTA (45.5%) received a total of 228 comments. Comments came from producers in 53% (n = 112) and healthcare professional organizations in 31.2% (n = 66). Technologies evaluated were drugs in 37% (n = 114), procedures in 35.5% (n = 109), diagnostic methods in 15.3% (n = 47), and devices in 12.2% (n = 38). Out of 308 rHTA documents, 120 (39%) were modified—mostly minor adjustments (n = 100; 80%), followed by major (n = 12; 10%) and intermediate modifications (n = 8; 6.4%).

Conclusion

Implementing an active stakeholder involvement process in HTA is feasible in a low- to middle-income country context and strengthens and improves the HTA process.
导言:卫生技术评估(HTA)是一种可以评估技术价值的标准化方法。通过将利益相关者的观点纳入公共咨询过程,可以提高决策的透明度和质量:描述阿根廷一家独立、学术性、非营利性 HTA 机构为快速 HTA 积极开展利益相关者咨询的过程,评估其最初五年的情况:自 2017 年以来,我们一直在对快速 HTA 文件进行积极的公众咨询,邀请生产者、医疗保健专业人士和患者组织提供意见,他们的意见可能会导致 HTA 文件的更改。修改分为重大修改(覆盖建议的修改)、中级修改(疗效、净效益或成本效益的修改,但不改变覆盖建议)和轻微修改(其他修改):从 2017 年 5 月至 2022 年 8 月,共发布了 308 份快速 HTA(rHTA)报告,发出了 3438 份邀请函。140份rHTA(45.5%)共收到228条意见。53%(n = 112)的评论来自生产商,31.2%(n = 66)的评论来自医疗保健专业组织。接受评估的技术中,药物占 37%(114 人),程序占 35.5%(109 人),诊断方法占 15.3%(47 人),设备占 12.2%(38 人)。在 308 份 rHTA 文件中,有 120 份(39%)进行了修改--主要是小幅调整(n = 100;80%),其次是大幅修改(n = 12;10%)和中级修改(n = 8;6.4%):结论:在中低收入国家的情况下,在 HTA 中实施积极的利益相关者参与流程是可行的,并能加强和改进 HTA 流程。
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引用次数: 0
Bypassing sources of care by level and coverage: Access to essential services in Peru and Uruguay in the post-pandemic era 按级别和覆盖范围划分的绕过医疗来源:后流行病时代秘鲁和乌拉圭的基本服务获取情况。
IF 4.7 3区 医学 Q1 MEDICINE, RESEARCH & EXPERIMENTAL Pub Date : 2024-10-05 DOI: 10.1016/j.arcmed.2024.103087
Jesús Medina-Ranilla , Hannah H. Leslie , Javier Roberti , Laura Espinoza-Pajuelo , Marina Guglielmino , Agustina Mazzoni , Ezequiel García-Elorrio , Patricia J. García

Background and aims

Healthcare provision to distinct social groups in Latin America contributes to inequities. Individuals make active choices by bypassing their coverage and intended healthcare source. After the pandemic, we sought to characterize bypassing behaviors and quantify their effects on access to essential services.

Methods

Cross-sectional data from a population-based telephone survey in Peru and Uruguay were analyzed. Participants were selected by random digit dialing. Outcomes were defined as access to preventive screenings and satisfaction of emerging health needs. Bypassing by level was defined as when participants went around primary care for the usual source of care or last preventive visit; bypassing by coverage when care was sought outside of public coverage or social security. Sociodemographic characteristics were included, and the adjusted average treatment effect was calculated.

Results

Data from 1,255 participants in Peru and 1,237 participants in Uruguay were analyzed. Bypassing behaviors by level (32% Peru; 60% Uruguay) and coverage (29% Peru; 21% Uruguay) were more prevalent in more privileged groups, especially in Peru. System competence was low overall and varied by bypassing mode, especially in Peru. In the adjusted analysis, statistically significant differences were found in bypassing by coverage in Peru (–8% difference in unmet health needs) and by level in Uruguay (5% more unmet needs).

Conclusion

Provision of essential preventive services was insufficient in both countries. In Peru, bypassing could serve as a proxy measure of inequities. Reminders of preventive services could be offered to bypassers of primary care. Profound health system reforms are needed to ensure equitable access to essential services.
背景和目的:拉丁美洲为不同社会群体提供的医疗保健服务造成了不平等。个人会主动选择绕过他们的医保和预定的医疗机构。大流行后,我们试图描述绕过行为的特点,并量化其对获得基本服务的影响:方法:我们对秘鲁和乌拉圭人口电话调查的横截面数据进行了分析。调查对象通过随机数字拨号选出。结果被定义为获得预防性筛查和满足新出现的健康需求。按级别划分的绕过是指参与者绕过初级保健机构进行通常的保健或最后一次预防性就诊;按覆盖范围划分的绕过是指在公共覆盖范围或社会保障范围之外就诊。研究还包括社会人口学特征,并计算了调整后的平均治疗效果:分析了秘鲁 1,255 名参与者和乌拉圭 1,237 名参与者的数据。按级别(秘鲁 32%;乌拉圭 60%)和覆盖范围(秘鲁 29%;乌拉圭 21%)划分,绕过行为在特权群体中更为普遍,尤其是在秘鲁。系统能力总体较低,并因绕过方式的不同而不同,尤其是在秘鲁。在调整后的分析中,发现秘鲁的绕过覆盖率(未满足的保健需求相差 8%)和乌拉圭的绕过水平(未满足的需求增加 5%)在统计上有显著差异:结论:两国提供的基本预防服务都不足。在秘鲁,"绕道 "可以作为衡量不公平的替代指标。可以向绕过初级保健者提供预防服务提醒。需要对卫生系统进行深入改革,以确保公平获得基本服务。
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引用次数: 0
Genetics in reproductive medicine 生殖医学遗传学。
IF 4.7 3区 医学 Q1 MEDICINE, RESEARCH & EXPERIMENTAL Pub Date : 2024-09-28 DOI: 10.1016/j.arcmed.2024.103092
Isabel Ochando , Antonio Urbano , Joaquín Rueda
Thanks to advances in technology, genetic testing is now available to explore the causes of infertility and to assess the risk of a given couple passing on a genetic disorder to their offspring. This allows at-risk couples to make an informed decision when opting for assisted reproduction and allows professionals to offer pre-implantation diagnosis when appropriate. Genetic screening of an infertile couple has thus become standard practice for an appropriate diagnosis, treatment, and prognostic assessment. This review aims to highlight the conditions under which genetic screening plays a role in improving reproductive outcomes for infertile couples.
由于技术的进步,现在可以通过基因检测来探究不孕不育的原因,并评估特定夫妇将遗传疾病遗传给后代的风险。这样,有风险的夫妇在选择辅助生殖时就能做出明智的决定,专业人员也能在适当的时候提供植入前诊断。因此,对不孕夫妇进行基因筛查已成为适当诊断、治疗和预后评估的标准做法。本综述旨在强调基因筛查在改善不孕不育夫妇生育结果方面发挥作用的条件。
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引用次数: 0
Response to: Serendipitous adrenal hyperplasia in patients admitted to the emergency department is linked to increased mortality 回应:急诊科入院患者偶然出现的肾上腺增生与死亡率增加有关。
IF 4.7 3区 医学 Q1 MEDICINE, RESEARCH & EXPERIMENTAL Pub Date : 2024-09-28 DOI: 10.1016/j.arcmed.2024.103089
Giuseppe Reimondo, Soraya Puglisi, Massimo Terzolo
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引用次数: 0
Evaluation of TALK© training for interprofessional clinical debriefing in Latin America 对拉丁美洲跨专业临床汇报 TALK© 培训的评估
IF 4.7 3区 医学 Q1 MEDICINE, RESEARCH & EXPERIMENTAL Pub Date : 2024-09-26 DOI: 10.1016/j.arcmed.2024.103060
Cristina Diaz-Navarro , Soledad Armijo-Rivera , Carla Prudencio-Palomino , José Gamaliel Velazco-González , Pedro Castro , Esther León-Castelao

Background

Healthcare systems must adapt iteratively in response to external and local challenges while keeping patients and staff safe. Clinical debriefing is a cost-effective contributor to safety culture, facilitating learning and team adaptations that lead to improved processes, patient outcomes, and staff resilience. In the aftermath of the COVID-19 pandemic, an interest has emerged in adopting TALK© to guide clinical debriefing to promote safety, mutual support, and cultural change within healthcare teams in Latin American contexts.

Aims

To evaluate the quality and applicability of TALK© debriefing training in Latin American settings and the willingness to debrief after an educational intervention.

Methods

Retrospective and descriptive study, examining anonymous data collected over 18 months after completing a “TALK© Debriefing Course for Healthcare Professionals” face-to-face or online. Data collected included participant characteristics, course details, quality and applicability of the intervention, and willingness to debrief.

Results

Five hundred and forty-five participants were enrolled, most from Argentina and Mexico. The overall quality of the intervention scored 19.62/20 points, obtaining 4.86/5 points for applicability. There were no significant differences between virtual and face-to-face sessions.
After the intervention, ≥93.76% of participants felt able to engage in clinical debriefing, and 97.06% reported willingness to debrief.

Conclusions

Dissemination of multi-professional clinical debriefing training in Latin America is feasible and easily scalable. The quality of the educational intervention was rated excellent in both virtual and face-to-face settings, supporting the value of remote educational diffusion. Most participants in this study intervention felt prepared and willing to debrief following the intervention.
背景医疗系统必须在保证患者和员工安全的前提下,不断调整以应对外部和本地的挑战。临床汇报是促进安全文化的一种具有成本效益的方式,它能促进学习和团队调整,从而改善流程、患者疗效和员工适应能力。Aims To evaluate the quality and applicability of TALK© debriefing training in Latin American settings and the willingness to debrief after an educational intervention.MethodsRetrospective and descriptive study, examining anonymous data collected after 18 months completing a "TALK© Debriefing Course for Healthcare Professionals" face-to-face or online.收集的数据包括参与者的特征、课程详情、干预的质量和适用性以及汇报的意愿。干预的总体质量得分为 19.62/20,适用性得分为 4.86/5。干预结束后,≥93.76%的参与者认为能够进行临床汇报,97.06%的参与者表示愿意进行汇报。在虚拟和面对面的环境中,教育干预的质量都被评为优秀,这支持了远程教育传播的价值。这项干预研究的大多数参与者都认为自己做好了准备,并愿意在干预结束后进行汇报。
{"title":"Evaluation of TALK© training for interprofessional clinical debriefing in Latin America","authors":"Cristina Diaz-Navarro ,&nbsp;Soledad Armijo-Rivera ,&nbsp;Carla Prudencio-Palomino ,&nbsp;José Gamaliel Velazco-González ,&nbsp;Pedro Castro ,&nbsp;Esther León-Castelao","doi":"10.1016/j.arcmed.2024.103060","DOIUrl":"10.1016/j.arcmed.2024.103060","url":null,"abstract":"<div><h3>Background</h3><div>Healthcare systems must adapt iteratively in response to external and local challenges while keeping patients and staff safe. Clinical debriefing is a cost-effective contributor to safety culture, facilitating learning and team adaptations that lead to improved processes, patient outcomes, and staff resilience. In the aftermath of the COVID-19 pandemic, an interest has emerged in adopting TALK© to guide clinical debriefing to promote safety, mutual support, and cultural change within healthcare teams in Latin American contexts.</div></div><div><h3>Aims</h3><div>To evaluate the quality and applicability of TALK© debriefing training in Latin American settings and the willingness to debrief after an educational intervention.</div></div><div><h3>Methods</h3><div>Retrospective and descriptive study, examining anonymous data collected over 18 months after completing a “TALK© Debriefing Course for Healthcare Professionals” face-to-face or online. Data collected included participant characteristics, course details, quality and applicability of the intervention, and willingness to debrief.</div></div><div><h3>Results</h3><div>Five hundred and forty-five participants were enrolled, most from Argentina and Mexico. The overall quality of the intervention scored 19.62/20 points, obtaining 4.86/5 points for applicability. There were no significant differences between virtual and face-to-face sessions.</div><div>After the intervention, ≥93.76% of participants felt able to engage in clinical debriefing, and 97.06% reported willingness to debrief.</div></div><div><h3>Conclusions</h3><div>Dissemination of multi-professional clinical debriefing training in Latin America is feasible and easily scalable. The quality of the educational intervention was rated excellent in both virtual and face-to-face settings, supporting the value of remote educational diffusion. Most participants in this study intervention felt prepared and willing to debrief following the intervention.</div></div>","PeriodicalId":8318,"journal":{"name":"Archives of Medical Research","volume":"55 7","pages":"Article 103060"},"PeriodicalIF":4.7,"publicationDate":"2024-09-26","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142323088","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
New Drugs and Promising Drug Combinations in the Treatment of Chagas Disease in Brazil: A Systematic Review and Meta-Analysis 治疗巴西南美锥虫病的新药和有前途的药物组合:系统回顾与元分析
IF 4.7 3区 医学 Q1 MEDICINE, RESEARCH & EXPERIMENTAL Pub Date : 2024-09-26 DOI: 10.1016/j.arcmed.2024.103084
Elainne Silva Freire , Letícia Pinto da Silva , Aline do Carmo Silva , Pedro Alves Soares Vaz de Castro , Giovanna Rotondo de Araújo , Dayane Andriotti Otta , Danilo Cavalcante Braz , Juliana Maria Trindade Bezerra
Chagas disease (CD) is a parasitic infection caused by the protozoan Trypanosoma cruzi (Kinetoplastida, Trypanosomatidae). Benznidazole (Bz) has a limited ability to interfere with the pathogenicity of the parasite, which manages to overcome host defenses. This study aimed to conduct a systematic literature review and meta-analysis to understand and describe the drugs and their combinations, as well as new promising compounds used in the treatment of CD in Brazil. This study was registered in the Open Science Framework (OSF) and the International Prospective Register of Systematic Reviews, following the Preferred Reporting Items for Systematic Reviews and Meta-Analyses (PRISMA) guidelines. Searches were performed in the electronic scientific databases PubMed, LILACS, SciELO, and BVS. Searches were conducted using descriptors cataloged in the Health Sciences Descriptors (DeCS) and Medical Subject Headings (MeSH), in Portuguese, English, and Spanish. Of the 26 articles included in this systematic review and meta-analysis, 16 were related to drug combinations, and nine described new inhibitors of parasitic molecules. Despite high heterogeneity (I² = 92%), studies that evaluated the combination of Bz with other treatments for CD had an overall grouped cure rate of 74% (95% CI 54–94%). Only one study presented drug repositioning by monotherapy. Thus, drug combinations offer quick and accessible solutions for CD treatment, acting against resistant strains of T. cruzi. Certainly, the introduction of these promising compounds into the pharmaceutical market is distant, and the adoption of prophylactic measures is recommended as a barrier to the increasing number of CD cases.
南美锥虫病(CD)是由原生动物克鲁斯锥虫(Kinetoplastida,Trypanosomatidae)引起的寄生虫感染。苯并咪唑(Bz)干扰寄生虫致病性的能力有限,而寄生虫却能克服宿主的防御能力。本研究旨在进行系统的文献综述和荟萃分析,以了解和描述巴西用于治疗 CD 的药物及其组合,以及有前景的新化合物。本研究遵循系统综述和荟萃分析首选报告项目(PRISMA)指南,在开放科学框架(OSF)和国际系统综述前瞻性登记处进行了登记。在电子科学数据库 PubMed、LILACS、SciELO 和 BVS 中进行了检索。检索时使用了葡萄牙语、英语和西班牙语的健康科学描述符(DeCS)和医学主题词表(MeSH)中的描述符。在纳入本系统综述和荟萃分析的 26 篇文章中,16 篇与药物组合有关,9 篇描述了寄生虫分子的新抑制剂。尽管异质性很高(I² = 92%),但对 Bz 与其他 CD 治疗方法联合使用进行评估的研究显示,总体分组治愈率为 74%(95% CI 54-94%)。只有一项研究介绍了通过单一疗法重新定位药物的情况。因此,联合用药为 CD 的治疗提供了快速、便捷的解决方案,并能对抗耐药的 T. cruzi 菌株。当然,将这些前景广阔的化合物引入医药市场还很遥远,建议采取预防措施,以阻止 CD 病例的增加。
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引用次数: 0
Transcranial Direct Current Stimulation for Cognitive Impairment Rehabilitation: A Bibliometric Analysis 经颅直流电刺激用于认知障碍康复:文献计量分析
IF 4.7 3区 医学 Q1 MEDICINE, RESEARCH & EXPERIMENTAL Pub Date : 2024-09-25 DOI: 10.1016/j.arcmed.2024.103086
Ying Xu , Haoyu Huang , Mengyuan Wu , Zesen Zhuang , Hong Liu , Meijin Hou , Cong Chen

Background and aims

As global demographics shift toward an older population, cognitive impairment is becoming increasingly critical. Transcranial Direct Current Stimulation (tDCS), an innovative brain stimulation technique, has the potential to significantly improve cognitive function. Our main aim is to comprehensively analyze the existing literature, identify key aspects of tDCS research in the rehabilitation of cognitive impairment, and predict future trends in this field.

Methods

We used the Web of Science (WOS) database to search for English articles and reviews relevant to this topic. For visual analysis of the literature, we employed the WOS analysis tool, CiteSpace, along with VOSviewer software to ensure comprehensive analysis.

Results

We included 2940 articles published between 1998 and 2023. Over 25 years, annual publications and citations in this field increased steadily, peaking at 379 articles in 2021. Michael A. Nitsche was a major contributor. Most articles came from developed countries, primarily North America and Europe, and journals generally had modest impact factors. Research in this field primarily aims to treat cognitive impairment resulting from pathological aging or neuropsychiatric disorders, with a particular focus on specific brain regions. Recently, researchers have integrated various treatment modalities with tDCS techniques to actively investigate effective strategies to mitigate cognitive impairments associated with pathological aging.

Conclusion

This study presents the first bibliometric analysis of the literature on tDCS in the rehabilitation of cognitive impairment, highlighting key areas of research and emerging trends. These findings provide critical insights for future tDCS interventions targeting cognitive impairment associated with pathological aging.
背景和目的 随着全球人口结构向老年人群转变,认知障碍正变得越来越严重。经颅直流电刺激(tDCS)是一种创新的脑刺激技术,具有显著改善认知功能的潜力。我们的主要目的是全面分析现有文献,确定经颅直流电刺激(tDCS)在认知障碍康复方面的关键研究内容,并预测该领域的未来趋势。为了对文献进行可视化分析,我们使用了 WOS 分析工具 CiteSpace 和 VOSviewer 软件,以确保分析的全面性。25年间,该领域的年发表量和引用量稳步增长,到2021年达到379篇的峰值。迈克尔-尼采(Michael A. Nitsche)是该领域的主要贡献者。大多数文章来自发达国家,主要是北美和欧洲,期刊的影响因子一般不高。该领域的研究主要旨在治疗病理衰老或神经精神疾病导致的认知障碍,尤其关注特定脑区。最近,研究人员将各种治疗方式与 tDCS 技术相结合,积极探索缓解病理衰老相关认知障碍的有效策略。结论本研究首次对有关 tDCS 在认知障碍康复中的应用的文献进行了文献计量分析,突出了关键研究领域和新兴趋势。这些发现为未来针对病理衰老相关认知障碍的 tDCS 干预措施提供了重要启示。
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引用次数: 0
Preliminary efficacy and safety analysis of tafamidis in post-liver transplant patients with hereditary transthyretin cardiac amyloidosis 他法米迪对肝移植后遗传性转甲状腺素心脏淀粉样变性患者的初步疗效和安全性分析
IF 4.7 3区 医学 Q1 MEDICINE, RESEARCH & EXPERIMENTAL Pub Date : 2024-09-17 DOI: 10.1016/j.arcmed.2024.103083
Takahiro Okumura, Kenji Furusawa, Ryota Ito, Hiroaki Hiraiwa, Toyoaki Murohara

Hereditary transthyretin cardiac amyloidosis (ATTRv-CA) after liver transplantation remains challenging to treat due to residual amyloid deposits in extrahepatic organs, including the heart. Tafamidis, a transthyretin tetramer stabilizer, has shown promise in the treatment of ATTRv-CA; however, its efficacy and safety after liver transplantation are uncertain. In this preliminary retrospective review, we assessed the efficacy and safety of tafamidis (80 mg) in three ATTRv-CA cases after liver transplantation. Following one year of treatment, all patients experienced improvement in dyspnea, New York Heart Association functional class, brain natriuretic peptide levels, and cardiac troponin T levels. No significant changes in echocardiographic parameters were observed. Notably, no cardiovascular or drug-related adverse events occurred during treatment. Our findings suggest that tafamidis may benefit post-liver transplant patients with ATTRv-CA and warrant further investigation through randomized controlled trials with larger cohorts. This study highlights a potential therapeutic avenue for the management of cardiovascular involvement in this challenging patient population.

肝移植后的遗传性转甲状腺素心脏淀粉样变性(ATTRv-CA)由于在包括心脏在内的肝外器官中残留淀粉样蛋白沉积物,其治疗仍具有挑战性。Tafamidis是一种转甲状腺素四聚体稳定剂,已显示出治疗ATTRv-CA的前景;然而,其在肝移植后的疗效和安全性尚不确定。在这项初步回顾性研究中,我们评估了他法米迪(80 毫克)在三例 ATTRv-CA 肝移植后的疗效和安全性。治疗一年后,所有患者的呼吸困难、纽约心脏协会功能分级、脑钠肽水平和心肌肌钙蛋白 T 水平均有所改善。超声心动图参数未见明显变化。值得注意的是,治疗期间未发生心血管或药物相关不良事件。我们的研究结果表明,他法米迪可能对肝移植后的 ATTRv-CA 患者有益,值得通过更大规模的随机对照试验进行进一步研究。这项研究为治疗这一具有挑战性的患者群体的心血管疾病提供了一条潜在的治疗途径。
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引用次数: 0
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