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Medical Therapy for Craniopharyngiomas. 颅咽管瘤的药物治疗
Pub Date : 2021-11-01 Epub Date: 2021-11-08 DOI: 10.17925/EE.2021.17.2.121
Krystallenia I Alexandraki, Paraskevi Xekouki

Craniopharyngiomas are rare benign neoplasms presenting in two different types, adamantinomatous (ACP) or papillary (PCP), which are molecularly and clinically distinct. Traditional treatment includes surgical resection and radiotherapy, which are accompanied by a number of debilitating complications because of the tumours' proximity to important brain structures. Recent advances in the understanding of molecular pathogenesis of craniopharyngiomas have opened horizons to medical therapeutic options. ACPs are mainly characterized by mutations of β-catenin, which activate Wingless/Int (Wnt), and alter the mitogen extracellular kinase (MEK)/extracellular signal-regulated kinase (ERK) pathway, as well as inflammatory, cellular senescence, programmed cell death and sonic hedgehog (SHH) pathways. PCPs are mainly characterized by Ras/Raf/MEK/ERK pathway activation secondary to BRAF-V600E mutations. MEK inhibitors, such as binimetinib, or anti-inflammatory mediators, such as tocilizumab or interferon, have been administered to patients with ACP and the efficacy is mostly favourable. On the other hand, BRAF inhibitors, such as dabrafenib or vemurafenib, either alone or in combination with the MEK inhibitors trametinib and cobimetinib, have been administered to patients with PCP resulting in favourable responses. A number of ongoing trials will shed light on schemes, doses, combined treatments and safety issues of the new molecular-targeted treatments, changing the management of patients with craniopharyngiomas by launching the era of personalized medicine in these rare neoplasms. We conducted a systematic review to identify case series or case reports with patients currently treated with systemic medical therapy.

颅咽管瘤是一种罕见的良性肿瘤,表现为两种不同的类型,金刚腺瘤(ACP)和乳头状瘤(PCP),它们在分子和临床上是不同的。传统的治疗方法包括手术切除和放射治疗,由于肿瘤靠近重要的大脑结构,这伴随着许多使人衰弱的并发症。颅咽管瘤分子发病机制的最新进展为医学治疗选择开辟了视野。acp的主要特征是β-catenin突变,其激活无翼/Int (Wnt),改变丝裂原细胞外激酶(MEK)/细胞外信号调节激酶(ERK)通路,以及炎症、细胞衰老、细胞程序性死亡和sonic hedgehog (SHH)通路。pcp主要以BRAF-V600E突变继发的Ras/Raf/MEK/ERK通路激活为特征。MEK抑制剂(如binimetinib)或抗炎介质(如tocilizumab或干扰素)已被用于ACP患者,且大多数疗效良好。另一方面,BRAF抑制剂,如dabrafenib或vemurafenib,单独使用或与MEK抑制剂trametinib和cobimetinib联合使用,已被用于PCP患者,并产生良好的反应。一些正在进行的试验将揭示新的分子靶向治疗的方案、剂量、联合治疗和安全性问题,通过启动这些罕见肿瘤的个性化医疗时代,改变颅咽管瘤患者的管理。我们进行了一项系统综述,以确定目前接受全身药物治疗的患者的病例系列或病例报告。
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引用次数: 5
Changing the Concept: From the Traditional Glucose-centric to the New Cardiorenal-metabolic Approach for the Treatment of Type 2 Diabetes. 改变观念:从传统的以血糖为中心到新的心肾代谢法治疗 2 型糖尿病。
Pub Date : 2021-11-01 Epub Date: 2021-11-17 DOI: 10.17925/EE.2021.17.2.92
Dimitrios G Chatzis, Konstantinos Kolokathis, Kalliopi Magounaki, Stefanos Chatzidakis, Konstantinos Avramidis, Marianna Leopoulou, Theodoros P Angelopoulos, John Doupis

Type 2 diabetes mellitus (T2DM) is a chronic disease with a constantly increasing prevalence worldwide. It is well established that T2DM affects both the macro- and microvasculature, and its presence is associated with a high risk of acute and chronic cardiovascular events. Traditionally, the management of T2DM has been mainly focused on the optimization of blood glucose levels with the use of antidiabetic medications. During recent years, however, an impressive accumulation of evidence has arisen from studies designed to explore the plausible effects of new antidiabetic drugs on cardiovascular outcomes in patients with diabetes. This review article aims to emphasize the findings of these studies and to highlight the substantial role of the newer classes of antidiabetic drugs in treating T2DM in a holistic, cardiorenal-metabolic approach, thus shifting the paradigm from the traditional, simplistic, glucose-lowering approach.

2 型糖尿病(T2DM)是一种慢性疾病,在全球的发病率不断上升。T2DM 对大血管和微血管都有影响,而且与发生急性和慢性心血管事件的高风险相关,这一点已得到公认。传统上,T2DM 的治疗主要侧重于通过使用抗糖尿病药物来优化血糖水平。然而,近年来,旨在探索新型抗糖尿病药物对糖尿病患者心血管预后的合理影响的研究积累了大量证据,令人印象深刻。这篇综述文章旨在强调这些研究的结果,并突出新型抗糖尿病药物在以心肾代谢综合方法治疗 T2DM 方面的重要作用,从而改变传统、简单的降糖方法。
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引用次数: 0
Non-alcoholic Steatohepatitis: From Pathophysiology to Clinical Practice. 非酒精性脂肪性肝炎:从病理生理学到临床实践。
Pub Date : 2021-11-01 Epub Date: 2021-09-14 DOI: 10.17925/EE.2021.17.2.112
Sherwyn Schwartz, Jean Lucas, Mark H DeLegge

Non-alcoholic steatohepatitis (NASH) is becoming a global disease with significant associated comorbidities. To date, there are no commercialized drugs to treat NASH, outside of India; however, there is an abundance of new molecular entities which are in clinical development, some in phase III trials. Many of these trials have created an especially heavy demand for USA-based subjects. Hepatologists currently play a major role in the diagnosis, treatment and clinical-trial enrolment of patients with NASH. However, NASH has a strong metabolic component, with patients often carrying comorbid diseases, such as type 2 diabetes mellitus, obesity, hyperlipidaemia, hypothyroidism and sex steroid disorders. The primary care physician, internist and endocrinologist stand at a pivotal position in the NASH healthcare delivery system, as many of the diseases they commonly encounter are associated with a higher risk of developing NASH. Specialty society practice guidelines are evolving regarding the identification and care of patients with NASH. This review of the literature, and assessment of IQVIA's proprietary patient claims database of diagnosis codes, patient encounters and treatments, substantiates the importance of the primary care provider and endocrinologist in the clinical care and clinical research of patients with NASH.

非酒精性脂肪性肝炎(NASH)正在成为一种具有显著相关合并症的全球性疾病。到目前为止,在印度以外,还没有商业化的治疗NASH的药物;然而,有大量新的分子实体正在临床开发中,其中一些正在进行III期试验。其中许多试验对美国受试者产生了特别大的需求。肝病学家目前在NASH患者的诊断、治疗和临床试验登记方面发挥着重要作用。然而,NASH具有很强的代谢成分,患者经常携带合并症,如2型糖尿病、肥胖、高脂血症、甲状腺功能减退和性类固醇疾病。初级保健医生、内科医生和内分泌学家在NASH医疗保健系统中处于关键地位,因为他们经常遇到的许多疾病都与患NASH的风险较高有关。关于NASH患者的识别和护理,专业协会的实践指南正在不断发展。这篇文献综述,以及对IQVIA专有的诊断代码、患者遭遇和治疗患者索赔数据库的评估,证实了初级保健提供者和内分泌学家在NASH患者的临床护理和临床研究中的重要性。
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引用次数: 0
Controversies in Gestational Diabetes. 妊娠期糖尿病的争议。
Pub Date : 2021-11-01 Epub Date: 2021-08-04 DOI: 10.17925/EE.2021.17.2.102
Chloe A Zera, Ellen W Seely

Gestational diabetes mellitus (GDM) complicates approximately 7% of pregnancies in the USA. Despite recognition of the benefits of diagnosing and treating GDM, there are several areas of controversy that remain unresolved. There is debate as to whether to screen for GDM with the one-step versus the two-step approach. While the former identifies more pregnancies with potential adverse outcomes, data are lacking as to whether treatment of these pregnancies will improve outcomes, while increasing costs by diagnosing more women. Though it is well established that the diagnosis of even mild GDM, and treatment with lifestyle recommendations and insulin, improves pregnancy outcomes, it is controversial as to which type and regimen of insulin is optimal, and whether oral agents can be used safely and effectively to control glucose levels. Finally, it is recommended that women with GDM get tested for type 2 diabetes within several months of delivery; however, many women do not undergo this testing and alternative approaches are needed. These controversies are discussed with data from both sides of the debate to enable clinicians to make patient-centered decisions until more definitive data are available.

在美国,妊娠期糖尿病(GDM)并发症约占妊娠的7%。尽管认识到诊断和治疗GDM的好处,但仍有几个争议领域尚未解决。关于是用一步法还是两步法筛查GDM存在争议。虽然前者确定了更多有潜在不良后果的怀孕,但缺乏数据表明,对这些怀孕的治疗是否会改善结果,同时因诊断出更多的妇女而增加了成本。虽然已经确定,即使是轻度GDM的诊断,以及生活方式建议和胰岛素治疗,都可以改善妊娠结局,但哪种类型和方案的胰岛素是最佳的,以及口服药物是否可以安全有效地控制血糖水平,这些都是有争议的。最后,建议患有GDM的妇女在分娩后几个月内进行2型糖尿病检测;然而,许多妇女不接受这种检查,需要其他方法。这些争议讨论与数据从辩论双方,使临床医生做出以患者为中心的决定,直到更明确的数据可用。
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引用次数: 0
Contemporary Non-hormonal Therapies for the Management of Vasomotor Symptoms Associated with Menopause: A Literature Review. 当代非激素疗法治疗与更年期相关的血管舒缩症状:文献综述
Pub Date : 2021-11-01 Epub Date: 2021-10-13 DOI: 10.17925/EE.2021.17.2.133
Sabrina Sahni, Angie Lobo-Romero, Taryn Smith
Nearly 75% of all menopausal women experience bothersome vasomotor symptoms including hot flushes and night sweats. Yet vasomotor symptoms continue to be an undertreated and underdiagnosed symptom of menopause which can negatively affect a woman's overall quality of life. While hormone therapy has been widely utilized to ameliorate hot flushes, not all women are candidates for use, especially those with increased risk of cardiovascular disease, thromboembolic disease, and/or women at an increased risk of certain hormone-dependent cancers. The current literature provides strong evidence for non-hormonal therapies in women who experience vasomotor symptoms. This article reviews the evidence for the use of non-hormonal pharmacologic therapies for the treatment of menopausal symptoms including antidepressants, gabapentinoids, clonidine and anticholinergics. We also review data on emerging therapies including the latest evidence on neurokinin-1 and -3 antagonists. These therapies should be considered when hormonal options are contraindicated and/or not preferred by the patient. While there are many options available, clinicians should individualize therapy based on the patient's needs and goals while mitigating bothersome side effects.
近75%的绝经期妇女经历了令人烦恼的血管舒缩症状,包括潮热和盗汗。然而,血管舒缩症状仍然是一种未得到充分治疗和诊断的更年期症状,它会对女性的整体生活质量产生负面影响。虽然激素疗法已被广泛用于改善潮热,但并非所有妇女都适合使用,特别是那些心血管疾病、血栓栓塞性疾病风险增加的妇女,和/或某些激素依赖性癌症风险增加的妇女。目前的文献为经历血管舒缩症状的女性提供了非激素治疗的有力证据。本文综述了使用非激素药物治疗更年期症状的证据,包括抗抑郁药、加巴喷丁类药物、可乐定和抗胆碱能药物。我们还回顾了新兴疗法的数据,包括神经激肽-1和-3拮抗剂的最新证据。当患者有激素治疗禁忌和/或不喜欢激素治疗时,应考虑使用这些治疗方法。虽然有很多选择,临床医生应该根据病人的需要和目标来个性化治疗,同时减轻恼人的副作用。
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引用次数: 5
Finerenone: A Potential Treatment for Patients with Chronic Kidney Disease and Type 2 Diabetes Mellitus. 非格列酮慢性肾病和 2 型糖尿病患者的潜在治疗方法。
Pub Date : 2021-11-01 Epub Date: 2021-11-10 DOI: 10.17925/EE.2021.17.2.84
Luis D'Marco, María Jesús Puchades, Lorena Gandía, Claudia Forquet, Elena Giménez-Civera, Nayara Panizo, Javier Reque, Isabel Juan-García, Valmore Bermúdez, José Luis Gorriz

Type 2 diabetes mellitus (T2DM) affects an estimated 463 million people worldwide, equivalent to 1 in 11 adults. Moreover, the rapid growth of this disease has resulted in a high incidence of diabetic kidney disease (DKD), which, together with hypertension, is the main cause of chronic kidney disease (CKD). Hyperglycaemia, low-grade inflammation, altered lipid metabolism and hyperactivation of the renin-angiotensin-aldosterone system (RAAS) seem to be interrelated mechanisms contributing to both T2DM and microvascular complications. The introduction of drugs such as sodium-glucose cotransporter 2 inhibitors and glucagon-like peptide 1 receptor agonists has improved the ability to slow the progression of DKD, and has also demonstrated benefits in cardiovascular disease. Beyond the effects of these novel antidiabetic drugs, a body of evidence suggests that the overactivation of the mineralocorticoid receptor also contributes to CKD progression. Moreover, new and ongoing trials have demonstrated that the selective nonsteroidal mineralocorticoid receptor antagonist (MRA) finerenone improves the risk of CKD progression and cardiovascular events in patients with CKD and T2DM and optimized RAAS blockade. We review the rationale for the development and use of MRA drugs to slow CKD progression in patients with DKD, as well as other pleiotropic effects, and highlight the warnings associated with these agents.

全世界约有 4.63 亿人患有 2 型糖尿病(T2DM),相当于每 11 个成年人中就有 1 人。此外,这种疾病的快速增长导致糖尿病肾病(DKD)的高发病率,而糖尿病肾病与高血压一起,是慢性肾病(CKD)的主要病因。高血糖、低度炎症、脂质代谢改变和肾素-血管紧张素-醛固酮系统(RAAS)亢进似乎是导致 T2DM 和微血管并发症的相互关联的机制。钠-葡萄糖共转运体 2 抑制剂和胰高血糖素样肽 1 受体激动剂等药物的问世,提高了延缓 DKD 病程进展的能力,也显示出对心血管疾病的益处。除了这些新型抗糖尿病药物的作用外,大量证据表明,矿物质皮质激素受体的过度激活也是导致慢性肾脏病进展的原因之一。此外,新的和正在进行的试验表明,选择性非甾体类矿化皮质激素受体拮抗剂(MRA)非那西酮可改善慢性肾功能衰竭和 T2DM 患者的慢性肾功能衰竭进展风险和心血管事件,并优化 RAAS 阻断。我们回顾了开发和使用 MRA 药物以延缓 DKD 患者 CKD 病程进展的理论依据以及其他多生物效应,并强调了与这些药物相关的警告。
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引用次数: 0
Congenital Adrenal Hyperplasia Due to 17-α-hydroxylase Deficiency: A Case Report. 17-α-羟化酶缺乏所致先天性肾上腺增生1例。
Pub Date : 2021-11-01 Epub Date: 2021-09-08 DOI: 10.17925/EE.2021.17.2.138
Lucas Ribeiro Dos Santos, Erico Paulo Heilbrun, Charles Simões Félix, Márcio Luis Duarte

Congenital adrenal hyperplasia, an innate error in adrenal steroid biosynthesis, triggers a wide range of consequences based on the level of enzyme blockade. Due to the various forms of enzyme deficiency and degree of penetration, the clinical features are very variable. In this case report, we present a form of congenital adrenal hyperplasia due to an enzymatic defect of CYP17A1, with a late diagnosis. The recognition of this pathology should occur as early as possible to avoid sequelae, both metabolic and psychological.

先天性肾上腺增生是肾上腺类固醇生物合成的先天错误,根据酶阻断的水平会引发广泛的后果。由于各种形式的酶缺乏和渗透程度,临床特征是非常不同的。在这个病例报告中,我们提出了一种由于CYP17A1酶缺陷导致的先天性肾上腺增生,诊断较晚。对这种病理的认识应尽早发生,以避免代谢和心理上的后遗症。
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引用次数: 0
Diagnosis and Clinical Management of Long-chain Fatty-acid Oxidation Disorders: A Review. 长链脂肪酸氧化障碍的诊断和临床治疗综述。
Pub Date : 2021-11-01 Epub Date: 2021-09-10 DOI: 10.17925/EE.2021.17.2.108
Joshua J Baker, Barbara K Burton

Long-chain fatty-acid oxidation disorders (LC-FAODs) are autosomal recessive inherited metabolic conditions that occur due to a disruption in the body's ability to perform mitochondrial beta oxidation. Expanded newborn screening is widening phenotypic understanding of these disorders, as well improving our knowledge of disease incidence. Management of these disorders is focused on avoidance of fasting, dietary changes and supplementation with energy sources that bypass the metabolic block. Recent US Food and Drug Administration approval of triheptanoin has improved the outcome for affected individuals. New research into dietary modifications and novel pharmacologic therapies continues for these disorders. In this article, we review the major LC-FAODs and their clinical presentation.

长链脂肪酸氧化障碍(LC-FAODs)是一种常染色体隐性遗传代谢疾病,是由于机体线粒体氧化能力的破坏而发生的。扩大新生儿筛查扩大了对这些疾病的表型理解,也提高了我们对疾病发病率的认识。这些疾病的管理重点是避免禁食、改变饮食和补充绕过代谢障碍的能量来源。最近美国食品和药物管理局批准了三庚烷酸,改善了受影响个体的预后。对这些疾病的饮食调整和新的药物治疗的新研究仍在继续。在本文中,我们回顾了主要的LC-FAODs及其临床表现。
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引用次数: 3
Diabetes Mellitus of Pituitary Origin: A Case Report. 垂体源性糖尿病1例报告。
Pub Date : 2021-04-01 Epub Date: 2021-04-28 DOI: 10.17925/EE.2021.17.1.68
Mandeep Singla, Jaspreet Kaur Saini

We describe the case report of 36-year-old female who presented to the emergency department with diabetic ketoacidosis. On detailed clinical examination, coarse facial features in the form of large fleshy nose, thick lips, prognathism, and thickening of hands and feet were noticed, suggestive of acromegaly. Subsequently, she was diagnosed with acromegaly due to somatotropinoma. Impaired glucose tolerance and diabetes mellitus are frequently associated with acromegaly. Persistent growth hormone excess impairs insulin sensitivity, increases gluconeogenesis, reduces glucose uptake in adipose tissue and muscle, and alters pancreatic β-cell function. Rarely, diabetic ketoacidosis can be the presenting manifestation, as seen in this case.

我们描述了一例36岁女性因糖尿病酮症酸中毒到急诊科就诊的病例报告。在详细的临床检查中,发现了粗糙的面部特征,如大而多肉的鼻子、厚嘴唇、前突和手脚增厚,提示肢端肥大症。随后,她被诊断为生长激素瘤引起的肢端肥大症。糖耐量受损和糖尿病经常与肢端肥大症有关。持续过量的生长激素会损害胰岛素敏感性,增加糖异生,减少脂肪组织和肌肉中的葡萄糖摄取,并改变胰腺β细胞的功能。如本例所示,糖尿病酮症酸中毒可能是罕见的表现。
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引用次数: 1
Case Report of a Rare Adrenocortical Oncocytoma Suspected to be an Adrenal Carcinoma. 怀疑为肾上腺癌的罕见肾上腺皮质瘤1例报告。
Pub Date : 2021-04-01 Epub Date: 2021-04-28 DOI: 10.17925/EE.2021.17.1.71
Inês Isabel Ferreira Barros, Fernando Manso, Margarida Teixeira, Maria Ramires Silva Lopes Pereira

An adrenal oncocytic neoplasm is an extremely rare tumour arising from the adrenal gland and it should be considered in the differential diagnosis of an adrenal incidentaloma, since it is frequently non-functioning. The suspicion for malignancy is high when an adrenal incidentaloma is >4 cm in size; however, adrenal oncocytomas are large, measuring an average of 8 cm, are round and encapsulated, and normally have a benign behaviour. We present a case of a 55-year-old male patient with dyslipidaemia, medicated with simvastatin. Upon complaints of abdominal pain, the general physician asked for an abdominal ultrasound that revealed an adrenal lesion, further characterized with a computed tomography scan, which showed an adrenal lesion measuring 49 × 64 × 56 mm and a calcification focus. The patient was referred to the general surgery and endocrinology department. The analytical study was negative for pheochromocytoma or Cushing's syndrome, which allowed surgery to be conducted, as is recommended. The aim of this case report is to contribute to the knowledge on adrenal oncocytomas, since there is scarce information based on singular experiences.

肾上腺嗜酸细胞肿瘤是一种极为罕见的由肾上腺引起的肿瘤,在肾上腺偶发瘤的鉴别诊断中应考虑它,因为它经常不起作用。当肾上腺偶发瘤的大小>4cm时,恶性肿瘤的怀疑度很高;然而,肾上腺嗜酸细胞瘤体积大,平均8厘米,呈圆形和包膜,通常具有良性行为。我们报告一例55岁男性患者,患有血脂异常,服用辛伐他汀。在抱怨腹痛后,普通医生要求进行腹部超声检查,发现肾上腺病变,并通过计算机断层扫描进行进一步表征,显示肾上腺病变尺寸为49×64×56 mm,并有钙化灶。病人被转诊到普通外科和内分泌科。分析研究对嗜铬细胞瘤或库欣综合征呈阴性,因此可以按照建议进行手术。本病例报告的目的是增加对肾上腺嗜酸细胞瘤的了解,因为基于单一经验的信息很少。
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引用次数: 2
期刊
TouchREVIEWS in endocrinology
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