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. 2024 Jun 14;45(6):549-555.
doi: 10.3760/cma.j.cn121090-20240127-00041.

[Analysis and summary of clinical characteristics of 289 patients with paroxysmal nocturnal hemoglobinuria in Zhejiang Province]

[Article in Chinese]
G X Xu  1 , W M Jin  2 , B D Ye  3 , S F Jiang  4 , C Hu  1 , X Huang  1 , B S Xie  5 , H F Jiang  6 , L L Chen  7 , R X Yao  8 , Y Lu  9 , L J Li  10 , J Zhang  11 , G F Ouyang  12 , Y W Hong  13 , H W Kong  14 , Z J Qiu  15 , W J Luo  16 , B B Chu  17 , H Q Zhang  18 , H Zeng  19 , X J Zhou  20 , P F Shi  21 , Y Xu  1 , J Jin  1 , H Y Tong  1
Affiliations

[Analysis and summary of clinical characteristics of 289 patients with paroxysmal nocturnal hemoglobinuria in Zhejiang Province]

[Article in Chinese]
G X Xu et al. Zhonghua Xue Ye Xue Za Zhi. .

Abstract
in English, Chinese

Objective: To further improve the understanding of paroxysmal nocturnal hemoglobinuria (PNH), we retrospectively analyzed and summarized the clinical characteristics, treatment status, and survival status of patients with PNH in Zhejiang Province. Methods: This study included 289 patients with PNH who visited 20 hospitals in Zhejiang Province. Their clinical characteristics, comorbidity, laboratory test results, and medications were analyzed and summarized. Results: Among the 289 patients with PNH, 148 males and 141 females, with a median onset age of 45 (16-87) years and a peak onset age of 20-49 years (57.8% ). The median lactic dehydrogenase (LDH) level was 1 142 (604-1 925) U/L. Classified by type, 70.9% (166/234) were classical, 24.4% (57/234) were PNH/bone marrow failure (BMF), and 4.7% (11/234) were subclinical. The main clinical manifestations included fatigue or weakness (80.8%, 235/289), dizziness (73.4%, 212/289), darkened urine color (66.2%, 179/272), and jaundice (46.2%, 126/270). Common comorbidities were hemoglobinuria (58.7% ), renal dysfunction (17.6% ), and thrombosis (15.0% ). Moreover, 82.3% of the patients received glucocorticoid therapy, 70.9% required blood transfusion, 30.7% used immunosuppressive agents, 13.8% received anticoagulant therapy, and 6.3% received allogeneic hematopoietic stem cell transplantation. The 10-year overall survival (OS) rate was 84.4% (95% CI 78.0% -91.3% ) . Conclusion: Patients with PNH are more common in young and middle-aged people, with a similar incidence rate between men and women. Common clinical manifestations include fatigue, hemoglobinuria, jaundice, renal dysfunction, and recurrent thrombosis. The 10-year OS of this group is similar to reports from other centers in China.

目的： 对浙江省阵发性睡眠性血红蛋白尿症（PNH）患者的临床特征、治疗现状及其生存情况进行多中心回顾性分析，以提高对该疾病的认识和规范化诊治水平。 方法： 纳入了2005年9月至2023年5月就诊于浙江省20家医院的289例PNH患者，随访并收集数据，对其临床特点、诊治现状及生存情况进行分析。 结果： 289例PNH患者中男148例，女141例，中位发病年龄为45（16～87）岁，发病高峰年龄为20～49岁（57.8%）。中位血清乳酸脱氢酶（LDH）水平为1 142（604～1 925）U/L 。其中经典型PNH占70.9%，合并骨髓衰竭性疾病（PNH/BMF）型占24.4%，亚临床型PNH占4.7%。病程中主要临床表现包括疲劳或无力（80.8%，235/289）、头晕（73.4%，212/289）、尿色加深（66.2%，179/272）和黄疸（46.2%，126/270）。常见的合并症为血红蛋白尿（58.7%）、肾功能损害（17.6%）、血栓（15.0%）。有82.3%患者接受糖皮质激素治疗，70.9%需要输血，30.7%使用免疫抑制剂，13.8%接受抗凝治疗，6.3%接受了异基因造血干细胞移植。10年总生存（OS）率为84.4%（95%CI 78.0%～91.3%）。 结论： PNH患者好发于中青年，男女比例相当，临床常见表现为疲劳、头晕、血红蛋白尿、黄疸、肾功能损害、反复血栓等。本组患者的10年OS率和国内其他中心报道相当。.

Keywords: Lactic dehydrogenase; PNH clone; Paroxysmal nocturnal hemoglobinuria; Thrombus.

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Conflict of interest statement

利益冲突 所有作者声明无利益冲突

Figures

图1
图1. 阵发性睡眠性血红蛋白尿症（PNH）患者纳入及分类流程图
注 PNH/BMF：骨髓衰竭疾病相关型PNH
图2
图2. 浙江省289例阵发性睡眠性血红蛋白尿症患者年龄分布
图3
图3. 浙江省阵发性睡眠性血红蛋白尿症患者生存分析 A 246例有生存数据患者的总生存情况；B 明确有血栓形成和明确无血栓形成患者生存分析比较；C 明确有肾功能损害和明确无肾功能损害患者生存分析比较
See this image and copyright information in PMC

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References

    1. 中华医学会血液学分会红细胞疾病(贫血)学组. 阵发性睡眠性血红蛋白尿症诊断与治疗中国专家共识[J] 中华血液学杂志. 2013;34(3):276–279. doi: 10.3760/cma.j.issn.0253-2727.2013.03.024. - DOI
    2. Chinese Society of Hematology, Chinese Medical Association. Expert consensus of diagnosis and treatment of paroxysmal nocturnal hemoglobinuria[J] Chin J Hematol. 2013;34(3):276–279. doi: 10.3760/cma.j.issn.0253-2727.2013.03.024. - DOI - PubMed
    1. Pu JJ, Brodsky RA. Paroxysmal nocturnal hemoglobinuria from bench to bedside[J] Clin Transl Sci. 2011;4(3):219–224. doi: 10.1111/j.1752-8062.2011.00262.x. - DOI - PMC - PubMed
    1. Brodsky RA. Paroxysmal nocturnal hemoglobinuria[J] Blood. 2014;124(18):2804–2811. doi: 10.1182/blood-2014-02-522128. - DOI - PMC - PubMed
    1. Hill A, DeZern AE, Kinoshita T, et al. Paroxysmal nocturnal haemoglobinuria[J] Nat Rev Dis Primers. 2017;3:17028. doi: 10.1038/nrdp.2017.28. - DOI - PMC - PubMed
    1. 中华医学会血液学分会红细胞疾病(贫血)学组. 再生障碍性贫血诊断与治疗中国指南(2022年版)[J] 中华血液学杂志. 2022;43(11):881–888. doi: 10.3760/cma.j.issn.0253-2727.2022.11.001. - DOI
    2. Red Blood Cell Disease (Anemia) Group, Chinese Society of Hematology, Chinese Medical Association. Guidelines for the diagnosis and management of aplastic anemia in China (2022) [J] Chin J Hematol. 2022;43(11):881–888. doi: 10.3760/cma.j.issn.0253-2727.2022.11.001. - DOI - PMC - PubMed