摘要
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％CI78.0％～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.
Abstract
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.
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