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Management of Multiple Myeloma in Older Patients 老年多发性骨髓瘤患者的治疗
Pub Date : 2021-07-27 DOI: 10.33590/emjhematol/20-00241
Jian Li, Henry Chan
Multiple myeloma is a condition that affects predominantly the older population. There are now various approved chemotherapy regimens as a result of advances in treatment. Choosing the optimal regimen for older patients with myeloma remains a challenge because of frailty and a lack of head-to-head comparisons between backbone regimens. The purpose of this literature review is to summarise the recent literature on frailty assessment, disease biology, and treatment efficacy in the frontline and relapsed settings to aid the decision-making process.
多发性骨髓瘤是一种主要影响老年人的疾病。由于治疗技术的进步,现在有各种各样的化疗方案得到批准。为老年骨髓瘤患者选择最佳治疗方案仍然是一个挑战,因为骨髓瘤患者身体虚弱,而且脊柱治疗方案之间缺乏正面比较。本文献综述的目的是总结最近在一线和复发情况下的衰弱评估、疾病生物学和治疗效果方面的文献,以帮助决策过程。
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引用次数: 1
Glanzmann Thrombasthenia: A Case Report of a Rare Inherited Coagulation Disorder Presenting with Traumatic Head Injury 格兰兹曼血栓减少症:一例罕见的遗传性凝血障碍表现为颅脑外伤
Pub Date : 2021-07-27 DOI: 10.33590/emjhematol/21-00008
Vishal Chakati, Durga Prasad Bukka, Srinivas Rao Erigaisi, S. Anchuri
This case study deals with a 32-year-old Indian male patient who presented with a traumatic head injury in the hospital, experienced uncontrolled bleeding after conducting surgery, and was eventually diagnosed with Glanzmann thrombasthenia. Glanzmann thrombasthenia is a rare hereditary blood clotting disorder characterised by a lack of platelet aggregation due to the absence of platelet glycoprotein IIb/IIIa. This occurrence is generally triggered by consanguineous marriages and is apparent in approximately one in one million people. Education and raising awareness about consanguinity in communities may help to reduce challenging, unusual genetic diseases.
本病例研究涉及一名32岁的印度男性患者,他在医院表现为外伤性头部损伤,在手术后出现无法控制的出血,最终被诊断为Glanzmann血栓减少症。格兰兹曼凝血症是一种罕见的遗传性凝血障碍,其特征是由于缺乏血小板糖蛋白IIb/IIIa而导致血小板聚集不足。这种情况通常是由近亲婚姻引起的,大约百万分之一的人会出现这种情况。教育和提高社区对血缘关系的认识可能有助于减少具有挑战性的、不寻常的遗传疾病。
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引用次数: 0
Isatuximab and Belantamab Mafodotin: A Primer to an Evolving Multiple Myeloma Landscape Isatuximab和Belantamab matodotin:一个不断发展的多发性骨髓瘤景观的引物
Pub Date : 2021-04-05 DOI: 10.33590/EMJHEMATOL/20-00231
R. Gonzalez, Hanna Bailey, Omar Castaneda Puglianini
Multiple myeloma (MM) continues to be an incurable disease impacting mainly an ageing population. Comorbidities, disease characteristics, and drug toxicity profiles heavily influence treatment selections. Despite single agent activity of many anti-MM agents, opportunities to maintain responses most often include combination therapy with immunomodulator and/or proteasome inhibitor therapies. Monoclonal antibodies (moAb) have become an additional backbone to both newly diagnosed and relapsed or refractory transplant eligible and ineligible patients. Tolerability of these agents offers an additional benefit particularly to an ageing population. Two newly approved moAb targeting CD38 and B-cell maturation antigen have been added to the anti-MM arsenal. Isatuximab, a chimeric anti-CD38 moAb, is the second U.S. Food and Drug Administration (FDA)-approved CD38 targeted therapy offering unique mechanisms of action owing to differences in epitope binding and favourable side effect profiles. Belantamab mafodotin, a B-cell maturation antigen drug-antibody conjugate, is a first-in-class humanised moAb containing a distinct microtubule-disrupting agent: monomethyl auristatin-F. Its distinctive anti-MM activity includes antibody-dependent cellular cytotoxicity and phagocytosis, as well as direct cytotoxicity caused by internalisation of monomethyl auristatin-F. This review focusses primarily on the mechanisms of action, resistance patterns, and clinical utility of two recently FDA approved agents; isatuximab in combination with pomalidomide and dexamethasone for relapsed or refractory MM exposed to at least two or more lines of therapy, and belantamab mafodotin monotherapy in relapsed or refractory MM exposed to four or more lines of therapy.
多发性骨髓瘤(MM)仍然是一种无法治愈的疾病,主要影响人口老龄化。合并症、疾病特征和药物毒性特征严重影响治疗选择。尽管许多抗mm药物具有单药活性,但维持反应的机会通常包括与免疫调节剂和/或蛋白酶体抑制剂治疗的联合治疗。单克隆抗体(moAb)已成为新诊断和复发或难治性移植合格和不合格患者的额外支柱。这些药物的耐受性提供了额外的好处,特别是对老龄化人口。两种新批准的靶向CD38和b细胞成熟抗原的moAb已被添加到抗mm库中。Isatuximab是一种嵌合抗CD38 moAb,是美国食品和药物管理局(FDA)批准的第二种CD38靶向治疗药物,由于表位结合的差异和有利的副作用,它提供了独特的作用机制。Belantamab mafodotin是一种b细胞成熟抗原药物-抗体偶联物,是一种一流的人源化moAb,含有一种独特的微管破坏剂:单甲基auristatin-F。其独特的抗mm活性包括抗体依赖的细胞毒性和吞噬作用,以及由内化单甲耳丁- f引起的直接细胞毒性。这篇综述主要集中在最近FDA批准的两种药物的作用机制、耐药模式和临床应用;isatuximab联合泊马度胺和地塞米松治疗复发性或难治性MM,而贝兰他单马弗多汀单药治疗复发性或难治性MM,接受至少2条或以上治疗线。
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引用次数: 1
Pneumoperitoneum, Pneumothorax, and Pneumoretroperitoneum Post Colonoscopy: A Case report and Review of Literature 结肠镜检查后的气腹、气胸、腹膜气:1例报告及文献回顾
Pub Date : 2020-12-01 DOI: 10.33590/emjgastroenterol/20-00131
Melissa Kyriakos Saad, Toufic Saber, G. Cortas, E. Saikaly
Colonic perforation post colonoscopy is rarely seen; however, when coupled with massive pneumoperitoneum in haemodynamically stable patients, a real dilemma for surgeons is created. The decision between watchful waiting versus surgical intervention is the real challenge and while most surgeons will urge for surgical intervention, conservative management on the other hand can be safely applied in selected haemodynamically stable patients.
结肠镜检查后结肠穿孔少见;然而,当血流动力学稳定的患者伴有大量气腹时,就会给外科医生带来真正的困境。在观察等待和手术干预之间的决定是真正的挑战,虽然大多数外科医生会敦促手术干预,但另一方面,保守管理可以安全地应用于选定的血流动力学稳定的患者。
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引用次数: 0
Is Allogeneic Stem Cell Transplantation a Good Option for Paroxysmal Nocturnal Haemoglobinuria? 同种异体干细胞移植是治疗阵发性夜间血红蛋白尿的好选择吗?
Pub Date : 2020-11-13 DOI: 10.33590/emjhematol/20-00023
F. Karadag, G. Saydam, F. Şahin
Paroxysmal nocturnal haemoglobinuria (PNH) is a rare, nonmalignant, haematopoietic clonal disorder that manifests with haemolytic anaemia, thrombosis, and peripheral blood cytopenias. The diagnosis is based on laboratory findings of intravascular haemolysis and flow cytometry. Clinical findings in PNH include haemolytic anaemia, thrombosis in atypical sites, or nonspecific symptoms attributable to the consequences of haemolysis. Thrombosis is the leading cause of death in PNH. Terminal complement pathway inhibition with eculizumab controls most of the symptoms of haemolysis and the life-threatening complications of PNH. However, there is still no consensus about haematopoietic stem cell transplantation (HSCT) in the management of PNH; it is the only potentially curative therapy for PNH. There are limited data and few case series about both the long-term outcomes of HSCT for PNH and the impacts of conditioning regimens on PNH clones. The authors have reviewed the findings of these studies which report on HSCT for the treatment of PNH.
阵发性夜间血红蛋白尿(PNH)是一种罕见的、非恶性的造血克隆性疾病,表现为溶血性贫血、血栓形成和外周血细胞减少。诊断基于血管内溶血和流式细胞术的实验室结果。PNH的临床表现包括溶血性贫血、非典型部位血栓形成或溶血后果引起的非特异性症状。血栓形成是PNH患者死亡的主要原因。eculizumab的终末补体途径抑制可控制大部分溶血症状和危及生命的PNH并发症。然而,关于造血干细胞移植(HSCT)在PNH治疗中的应用仍未达成共识;这是唯一可能治愈PNH的疗法。关于HSCT治疗PNH的长期结果和调理方案对PNH克隆的影响的数据和病例序列有限。作者回顾了这些关于HSCT治疗PNH的研究结果。
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引用次数: 0
Adult-Onset Still’s Disease Complicated with Haemophagocytic Lymphohistiocytosis (HLH): A Case Report 成人发病的斯蒂尔氏病合并嗜血球性淋巴组织细胞增多症1例报告
Pub Date : 2020-07-30 DOI: 10.33590/emjhematol/20-00048
M. Asghar, Abubakar Tauseef, Warda Fatmi, Narmin Khan, Maryam Zafar, U. Rasheed, Nimra Naveed Shaikh, M. Akram, Basmah Fayaz, Z. Iqbal
Haemophagocytic lymphohistiocytosis (HLH) is a rare but potentially aggressive and life-threatening syndrome of overactive histiocytes and lymphocytes that commonly affects infants; it is also observed in children and adults of all ages. The disease is differentiated into either primary or secondary causes. Primary HLH tends to be of genetic origin, while secondary HLH results from either infection, autoimmune disorders, or malignancies. Secondary HLH is most commonly associated with viral infections in immunocompromised patients. This paper presents a case of HLH in a tertiary care hospital, associated with adult-onset Still’s disease, diagnosed on both biochemical criteria and histopathologic examination of bone marrow smear.
嗜血球性淋巴组织细胞增多症(HLH)是一种罕见但具有潜在侵袭性和危及生命的综合征,由组织细胞和淋巴细胞过度活跃引起,常见于婴儿;在所有年龄段的儿童和成人中也可以观察到。这种疾病可分为原发性和继发性病因。原发性HLH往往是遗传的,而继发性HLH是由感染、自身免疫性疾病或恶性肿瘤引起的。继发性HLH最常与免疫功能低下患者的病毒感染相关。本文提出一个病例HLH在三级护理医院,与成人发病斯蒂尔氏病,诊断的生化标准和骨髓涂片组织病理学检查。
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引用次数: 0
Pancytopenia Secondary to Adult Osteopetrosis 成人骨质疏松症继发的全血细胞减少症
Pub Date : 2020-07-30 DOI: 10.33590/emjhematol/20-00012
Abdullah Y. Alkhowaiter, Anwer S. Alenazi, Ali G. Alghamdi
Osteopetrosis (OP) is a rare genetically metabolic bone disorder caused by severe impairment of osteoclast-mediated bone resorption. It is characterised by extensive sclerosis of the skeleton, fragility fracture, haematopoietic insufficiency, nerve entrapment syndromes, and growth impairment. It is clinically classified into two major types: infantile (autosomal recessive, malignant) and adult (autosomal dominant, benign) OP. The infantile type is usually diagnosed early in life, while adult type is diagnosed in late adolescence or adulthood. Approximately one-half of patients are asymptomatic and the diagnosis is made incidentally. However, some patients might present with one or more complications of OP, and the diagnosis is made during the work-up and evaluation. Here, the authors describe an unusual case of adult type OP presented with pancytopenia.
骨质疏松症(OP)是一种罕见的遗传性代谢性骨疾病,由破骨细胞介导的骨吸收严重受损引起。其特点是骨骼广泛硬化、脆性骨折、造血功能不全、神经卡压综合征和生长障碍。临床上分为两种主要类型:婴儿型(常染色体隐性,恶性)和成人型(常染色体显性,良性)。婴儿型通常在生命早期诊断,而成人型则在青春期晚期或成年期诊断。大约一半的患者是无症状的,诊断是偶然的。然而,一些患者可能会出现一种或多种OP并发症,在检查和评估中做出诊断。在这里,作者描述了一个不寻常的成人型OP表现为全血细胞减少症。
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引用次数: 0
Expanding the Role of CAR-T Cell Therapy to Systemic Lupus Erythematosus 扩大CAR-T细胞治疗系统性红斑狼疮的作用
Pub Date : 2020-07-30 DOI: 10.33590/emjhematol/20-00079
Shreya Patel, Kelly J. Brassil, Paiboon Jungsuwadee
Systemic lupus erythematosus (SLE) is a chronic inflammatory autoimmune disorder resulting from autoantibodies produced by B-cell derived plasma cells. Clinical presentation ranges from mild skin rash to multiorgan failure. Regardless of the clinical presentation or severity of the disease, patients with SLE often require life-long treatment. Current treatment recommendations for SLE include hydroxychloroquine, glucocorticoids, immunomodulatory agents, cyclophosphamide, and biologic agents. Despite availability of these agents, the condition of some patients with SLE progressively worsens. With limited treatment options, new and novel therapeutic approaches are needed. Given the active role of B cells in the pathophysiology of SLE, they present an attractive target for therapies evolving in the oncology field. Amongst these, immune effector cell therapies, including chimeric antigen receptor (CAR)-T cell therapy, have proven beneficial in targeting B cells. The eradication of B cells, along with the potential for T cell persistence, has resulted in prolonged remission or stable disease. This review provides an overview of the pathophysiology of SLE; current treatment options, including monoclonal antibodies targeting cluster of differentiation-20 (CD20), CD22, and B cell-activating factor (BAFF); and explores why and how immune effector cell therapies may prove a promising therapeutic option for this patient population, particularly for individuals with refractory disease. Clinical implications from currently approved U.S. Food and Drug Administration (FDA) agents for haematologic malignancies are discussed and provide insight into considerations for applying this therapy to the patient population with SLE in the context of clinical trials.
系统性红斑狼疮(SLE)是一种慢性炎症性自身免疫性疾病,由b细胞源性浆细胞产生的自身抗体引起。临床表现从轻度皮疹到多器官功能衰竭。无论疾病的临床表现或严重程度如何,SLE患者通常需要终身治疗。目前推荐的SLE治疗包括羟氯喹、糖皮质激素、免疫调节剂、环磷酰胺和生物制剂。尽管有这些药物,一些SLE患者的病情仍会逐渐恶化。由于治疗选择有限,需要新的和新颖的治疗方法。鉴于B细胞在SLE病理生理中的积极作用,它们为肿瘤领域的治疗提供了一个有吸引力的靶点。其中,免疫效应细胞疗法,包括嵌合抗原受体(CAR)-T细胞疗法,已被证明对靶向B细胞有益。B细胞的根除,连同潜在的T细胞持久性,导致了长期的缓解或稳定的疾病。本文综述了SLE的病理生理学;目前的治疗方案,包括针对CD20、CD22和B细胞活化因子(BAFF)的单克隆抗体;并探讨了免疫效应细胞疗法为什么以及如何被证明是一种有希望的治疗选择,特别是对于难治性疾病的个体。本文讨论了目前美国食品和药物管理局(FDA)批准的血液系统恶性肿瘤药物的临床意义,并提供了在临床试验背景下将该疗法应用于SLE患者群体的考虑因素。
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引用次数: 2
Coagulopathy and Hyperinflammation in COVID-19 COVID-19的凝血功能障碍和高脂血症
Pub Date : 2020-07-30 DOI: 10.33590/emjhematol/20f0730
K. Colvin
August 2020 • HEMATOLOGY 25 COVID-19, a disease resulting from SARS-CoV-2 infection, generally results in mild-to-moderate illnesses in those affected. However, 15% of symptomatic patients develop severe interstitial pneumonia, with 5% going on to develop profound and life-threatening complications including acute respiratory distress syndrome, sepsis, hyperinflammatory syndromes, and multiorgan failure. Clinical complications of COVID-19 include myocarditis, acute myocardial infarction, heart failure, venous thromboembolism, and cerebrovascular events. These vascular and thromboembolic complications suggest a coagulopathic impairment requiring specialist haematological examination and input. THROMBOSIS
COVID-19是一种由SARS-CoV-2感染引起的疾病,通常会导致感染者患上轻中度疾病。然而,有症状的患者中有15%会发展为严重的间质性肺炎,其中5%会发展为严重的危及生命的并发症,包括急性呼吸窘迫综合征、败血症、高炎症综合征和多器官衰竭。COVID-19的临床并发症包括心肌炎、急性心肌梗死、心力衰竭、静脉血栓栓塞和脑血管事件。这些血管和血栓栓塞并发症提示凝血功能障碍,需要专门的血液学检查和输入。血栓形成
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引用次数: 0
Concerns Regarding the Management of β-Thalassaemia Patients in the Era of COVID-19 COVID-19时代β-地中海贫血患者管理的关注
Pub Date : 2020-07-30 DOI: 10.33590/emjhematol/20-00090
C. Aggeli, S. Delicou, D. Patsourakos, A. Xydaki, J. Koskinas, D. Tousoulis
Many cases of pneumonia clustered in the city of Wuhan, China, were reported in December 2019, and source tracing has showed Huanan Seafood Market, Wuhan, China, as the origin. In this work, the authors summarise their concerns for thalassaemia patients, a unique group with several heart, liver, and blood comorbidities.
2019年12月,中国武汉市报告了多例聚集性肺炎病例,源头追踪显示,中国武汉华南海鲜市场为源头。在这项工作中,作者总结了他们对地中海贫血患者的关注,地中海贫血患者是一个具有多种心脏、肝脏和血液合并症的独特群体。
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引用次数: 0
期刊
EMJ Hematology
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