中东地区阵发性夜间血红蛋白尿的临床特点和治疗:叙述性回顾。

IF 3.5 4区 医学 Q2 MEDICINE, RESEARCH & EXPERIMENTAL
Mohammed Almakadi, Noura AlHashim, Murtadha Al-Khabori, Hazzaa Alzahrani, Hani Yousif Osman, Mervat Mattar, Ahmed Sabah
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引用次数: 0

摘要

阵发性夜间性 血红蛋白尿(PNH)是一种罕见的血液学疾病,由不受控制的终末补体激活引起。它与血管内溶血、血栓栓塞事件、器官损害、生活质量受损和过早死亡有关。由于没有来自中东国家的PNH登记数据,因此对该地区的管理知之甚少。本文综述了中东阿拉伯裔人群中PNH的患病率、特征、诊断和治疗方面的现有数据。对PubMed和EMBASE从成立到2025年5月31日的检索确定了15篇相关出版物:5篇来自沙特阿拉伯,4篇来自伊朗,2篇来自科威特,埃及、阿曼、黎巴嫩各1篇,1篇报道了一名在德国接受治疗的中东患者。在阿曼队列中,PNH的估计发病率为每500万人1.9例。PNH诊断的平均和中位年龄分别为38岁(伊朗回顾性研究,n = 81)和22.5岁(阿曼病例系列,n = 10)。在报告中,贫血和疲劳是常见的症状。很少有文献报道C5抑制剂的治疗,尽管现有数据表明eculizumab通常能改善患者的临床状况。ravulizumab在中东的吸收和临床使用仍未记录。由于现有数据的限制,中东地区对PNH的管理办法似乎与其他区域所报告的大体一致。然而,需要更多的数据来更深入地了解PNH的状况及其在中东人口中的管理。
本文章由计算机程序翻译,如有差异,请以英文原文为准。

Clinical characteristics and management of paroxysmal nocturnal hemoglobinuria in the Middle East: a narrative review.

Clinical characteristics and management of paroxysmal nocturnal hemoglobinuria in the Middle East: a narrative review.

Clinical characteristics and management of paroxysmal nocturnal hemoglobinuria in the Middle East: a narrative review.

Paroxysmal nocturnal hemoglobinuria (PNH) is a rare hematological disorder caused by uncontrolled terminal complement activation of blood cells. It is associated with intravascular hemolysis, thromboembolic events, organ damage, impaired quality of life and premature mortality. As there are no PNH registry data from Middle Eastern countries, little is known about its management in the region. This narrative review summarizes available data on the prevalence, characteristics, diagnosis and treatment of PNH in Middle Eastern populations of Arabic origin. A search of PubMed and EMBASE from inception to 31 May 2025 identified 15 relevant publications: five from Saudi Arabia, four from Iran, two from Kuwait, one each from Egypt, Oman, Lebanon, and one reporting a Middle Eastern patient treated in Germany. The estimated incidence rate of PNH in an Omani cohort was 1.9 per 5 million population. Mean and median ages at PNH diagnosis were 38 years (Iranian retrospective review, n = 81) and 22.5 years (Omani case series, n = 10), respectively. Where reported, anemia and fatigue were common presenting symptoms. Few publications reported on treatment with C5 inhibitors, although available data indicate that eculizumab generally improves patients' clinical condition. Uptake and clinical use of ravulizumab in the Middle East remains undocumented. Subject to limitations of the available data, the management approach to PNH in the Middle East appears to be generally consistent with that reported in other regions. However, additional data are required to gain greater insight into the status of PNH and its management in Middle Eastern populations.

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来源期刊
Clinical and Experimental Medicine
Clinical and Experimental Medicine 医学-医学:研究与实验
CiteScore
4.80
自引率
2.20%
发文量
159
审稿时长
2.5 months
期刊介绍: Clinical and Experimental Medicine (CEM) is a multidisciplinary journal that aims to be a forum of scientific excellence and information exchange in relation to the basic and clinical features of the following fields: hematology, onco-hematology, oncology, virology, immunology, and rheumatology. The journal publishes reviews and editorials, experimental and preclinical studies, translational research, prospectively designed clinical trials, and epidemiological studies. Papers containing new clinical or experimental data that are likely to contribute to changes in clinical practice or the way in which a disease is thought about will be given priority due to their immediate importance. Case reports will be accepted on an exceptional basis only, and their submission is discouraged. The major criteria for publication are clarity, scientific soundness, and advances in knowledge. In compliance with the overwhelmingly prevailing request by the international scientific community, and with respect for eco-compatibility issues, CEM is now published exclusively online.
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