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Novel gene therapies for sickle cell disease, Duchenne muscular dystrophy, and hemophilia A. 镰状细胞病、杜氏肌营养不良症和 A 型血友病的新型基因疗法。
IF 0.8 4区 医学 Q3 MEDICINE, GENERAL & INTERNAL Pub Date : 2024-11-01 Epub Date: 2024-10-29 DOI: 10.1097/01.JAA.0000000000000142
Laura Solano

Abstract: This article discusses novel genetic therapies for sickle cell disease, Duchenne muscular dystrophy, and hemophilia A. Gene therapies have the potential to deliver more targeted and effective approaches to treatment, especially for rare diseases for which the availability of approved therapies is limited. This article describes the first FDA-approved CRISPR/Cas9 treatment and the treatment protocols, indications, warnings, precautions, cost, and contraindications of four novel genetic therapies.

摘要:本文讨论了镰状细胞病、杜氏肌营养不良症和 A 型血友病的新型基因疗法。基因疗法有可能提供更有针对性、更有效的治疗方法,尤其是对于那些获批疗法有限的罕见疾病。本文介绍了首个获得 FDA 批准的 CRISPR/Cas9 疗法,以及四种新型基因疗法的治疗方案、适应症、警告、注意事项、成本和禁忌症。
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引用次数: 0
Filling the void: Expanding the role of PAs in palliative care. 填补空白:扩大 PA 在姑息关怀中的作用。
IF 0.8 4区 医学 Q3 MEDICINE, GENERAL & INTERNAL Pub Date : 2024-11-01 Epub Date: 2024-10-29 DOI: 10.1097/01.JAA.0000000000000138
Rebecca B Jones, Natalie F Mohammad

Abstract: Palliative care, which focuses on symptom management, quality of life, and communication, is appropriate for patients at any age and any stage in the disease process. Because of increased patient demand, the United States faces a shortage of palliative care specialists. Expansion of palliative care curriculum in physician associate/assistant (PA) programs, on-the-job training, and improvement of regulatory practice guidelines can help PAs alleviate the shortage of palliative care professionals.

摘要:姑息关怀侧重于症状管理、生活质量和沟通,适合任何年龄段和疾病过程中任何阶段的患者。由于患者需求增加,美国面临姑息关怀专家短缺的问题。在助理医师(PA)项目中扩展姑息关怀课程、开展在职培训以及改进监管实践指南,可帮助助理医师缓解姑息关怀专业人员短缺的问题。
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引用次数: 0
Polymyalgia rheumatica. 多发性风湿痛
IF 0.8 4区 医学 Q3 MEDICINE, GENERAL & INTERNAL Pub Date : 2024-11-01 Epub Date: 2024-10-29 DOI: 10.1097/01.JAA.0000000000000144
Trevor Young
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引用次数: 0
Deprescribing strategies for proton pump inhibitors. 质子泵抑制剂的停药策略。
IF 0.8 4区 医学 Q3 MEDICINE, GENERAL & INTERNAL Pub Date : 2024-11-01 Epub Date: 2024-10-29 DOI: 10.1097/01.JAA.0000000000000130
Julia Young, Jordan A Fuller, Corey M Guidry

Abstract: Proton pump inhibitors (PPIs) are widely used for a number of gastric-acid related diseases. Although these agents demonstrate enhanced efficacy and tolerability compared with other acid-suppressive therapies, long-term use is associated with a number of adverse reactions. This article summarizes appropriate indications for short- and long-term use of PPIs and describes deprescribing strategies that clinicians can use to reduce unnecessary PPI use in their patients.

摘要:质子泵抑制剂(PPIs)被广泛用于治疗多种胃酸相关疾病。虽然与其他抑酸疗法相比,这些药物具有更强的疗效和耐受性,但长期使用会产生一系列不良反应。本文总结了短期和长期使用 PPIs 的适当适应症,并介绍了临床医生可用于减少患者不必要使用 PPI 的处方策略。
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引用次数: 0
The long scar. 长长的疤痕
IF 0.8 4区 医学 Q3 MEDICINE, GENERAL & INTERNAL Pub Date : 2024-11-01 Epub Date: 2024-10-29 DOI: 10.1097/01.JAA.0000000000000147
Ethan Stonerook
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引用次数: 0
Stardust. 星尘
IF 0.8 4区 医学 Q3 MEDICINE, GENERAL & INTERNAL Pub Date : 2024-11-01 Epub Date: 2024-10-29 DOI: 10.1097/01.JAA.0000000000000145
Carol Mackenzie Jackson
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引用次数: 0
A rare case of peritoneal coccidioidomycosis. 一例罕见的腹膜球孢子菌病。
IF 0.8 4区 医学 Q3 MEDICINE, GENERAL & INTERNAL Pub Date : 2024-11-01 Epub Date: 2024-10-29 DOI: 10.1097/01.JAA.0000000000000123
Kaegan G Williams, Rachel S Robeck

Abstract: Coccidioidomycosis, also known as valley fever, is a fungal infection that can develop after inhalation of arthroconidia from soil. Coccidioidomycosis infections are most common in the southwestern United States and typically involve the lungs. Risk factors for extrapulmonary spread, which can be life-threatening, include older age, diabetes, HIV and AIDS, late-term pregnancy, immunosuppressant drug use and African or Filipino ancestry. This article describes incidental identification of coccidioidomycosis of the peritoneum, an extremely rare site for dissemination, during bariatric surgery in a patient without known risk factors.

摘要:球孢子菌病又称 "山谷热",是一种真菌感染,吸入土壤中的关节孢子后即可发病。球孢子菌病感染在美国西南部最为常见,通常累及肺部。肺外传播可危及生命,其风险因素包括高龄、糖尿病、艾滋病毒和艾滋病、晚期妊娠、使用免疫抑制剂以及非洲或菲律宾血统。本文描述了一名无已知危险因素的患者在接受减肥手术时意外发现腹膜球孢子菌病,这是一种极为罕见的传播部位。
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引用次数: 0
What is the cause of this infant's ptosis? 该婴儿上睑下垂的原因是什么?
IF 0.8 4区 医学 Q3 MEDICINE, GENERAL & INTERNAL Pub Date : 2024-11-01 Epub Date: 2024-10-29 DOI: 10.1097/01.JAA.0000000000000143
Andrew Parke Chastain, Anne Geary, Mark Lutz
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引用次数: 0
The PA profession: A worldwide phenomenon revisited. PA 专业:重新审视全球现象。
IF 0.8 4区 医学 Q3 MEDICINE, GENERAL & INTERNAL Pub Date : 2024-11-01 Epub Date: 2024-10-29 DOI: 10.1097/01.JAA.0000000000000135
Kathy Pedersen, Scott Smalley, Richard Dehn
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引用次数: 0
The benefits and limitations of establishing the PA profession globally: A systematic review and mixed-methods study. 在全球范围内建立 PA 专业的益处和局限性:系统回顾与混合方法研究。
IF 0.8 4区 医学 Q3 MEDICINE, GENERAL & INTERNAL Pub Date : 2024-11-01 Epub Date: 2024-10-29 DOI: 10.1097/01.JAA.0000000000000146
Arden R Turkewitz, Jane P Sallen, Rachel M Smith, Kandi Pitchford, Kimberly Lay, Scott Smalley

Objective: Physician associates/assistants (PAs) and their equivalents offer a solution to the supply and demand crisis to alleviate global healthcare needs. This study investigated how PA and PA equivalents address global healthcare needs across different healthcare systems, revealing recommendations for their use. The study also sought to catalog the global healthcare needs that PAs and equivalents are successfully alleviating, the roles in which they function, and the barriers facing implementation.

Methods: A systematic review was conducted from October 2021 to April 2022 and rerun in June 2023 following PRISMA 2020 guidelines; additionally, supporting interviews were conducted with PAs and global health experts. Primary outcomes were geographic region, economy, healthcare needs, and healthcare systems. Secondary outcomes were PA use, license recognition, and successes or barriers when implementing PAs.

Results: The literature and interviews focused on the global use of PAs and PA equivalents in six geographic regions, 63 countries, and five US territories where PAs or PA equivalents are employed, have been employed, are volunteering, or are being considered to support global healthcare needs. Most countries have a developing economy and an out-of-pocket healthcare system. PAs and PA equivalents hold 35 different practice titles, and most work in primary care. PAs alleviate healthcare shortages and economic disparities, specifically related to inequitable healthcare access. Globally, the profession is limited by a lack of legislation, regulation, and support.

Conclusions: PAs and PA equivalents worldwide belong to an adaptable profession that has well-documented success in alleviating the global healthcare shortage and addressing healthcare needs. Countries desiring PAs or PA equivalents should identify their specific needs, train their existing workforce, employ pilot programs, and focus on seeking early legislation and regulation. Broad support for existing PA and PA equivalent international organizations is recommended for global collaboration. This study serves as a guide for those advocating for the continued or future implementation of PAs and PA equivalents in their own country and provides a comprehensive resource to aid in the globalization of this profession. We offer recommendations to address the dire healthcare needs and workforce shortage faced across the globe.

目的:医生助理/助手(PA)及其同等人员为缓解全球医疗保健需求的供需危机提供了一种解决方案。本研究调查了助理医师和助理医师同等人员如何在不同的医疗保健系统中满足全球医疗保健需求,并提出了使用这些人员的建议。该研究还试图对助理医师和等同人员成功缓解的全球医疗保健需求、他们在其中发挥的作用以及实施过程中面临的障碍进行编目:方法:2021 年 10 月至 2022 年 4 月进行了系统性综述,并于 2023 年 6 月按照 PRISMA 2020 指南重新进行了综述;此外,还对助理医师和全球健康专家进行了支持性访谈。主要结果包括地理区域、经济、医疗保健需求和医疗保健系统。次要结果是 PA 的使用、许可证认可度以及实施 PA 时的成功经验或障碍:文献和访谈的重点是在六个地理区域、63 个国家和 5 个美国领土上的助理医师和等同于助理医师的人员的全球使用情况,在这些地区,助理医师或等同于助理医师的人员被雇用、已经被雇用、正在志愿服务或正在考虑支持全球医疗保健需求。大多数国家的经济都处于发展阶段,并拥有自费医疗系统。助理医师和相当于助理医师的人员拥有 35 种不同的执业头衔,大多数从事初级保健工作。助理医师可以缓解医疗保健短缺和经济差距,特别是与不公平的医疗保健获取有关的问题。在全球范围内,这一职业因缺乏立法、监管和支持而受到限制:全世界的助理医师和同等医师都属于适应性强的职业,在缓解全球医疗短缺和满足医疗需求方面取得了有据可查的成功。希望获得助理医师或助理医师同等职位的国家应确定其具体需求,培训现有劳动力,采用试点项目,并重点寻求早期立法和监管。建议广泛支持现有的助理医师和相当于助理医师的国际组织,以促进全球合作。本研究为那些倡导在本国继续或在未来实施助理医师和助理医师同等职位的人提供了指南,并为帮助这一职业的全球化提供了全面的资源。我们提出了一些建议,以解决全球面临的迫切的医疗保健需求和劳动力短缺问题。
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引用次数: 0
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