糖皮质激素维持治疗阵发性睡眠性血红蛋白尿症疗效研究  

Efficacy of glucocorticoid maintenance therapy for paroxysmal nocturnal hemoglobinuria

作　　者：杨慧[1] 徐丹[2] 徐金格 宫跃敏 李悦 吴雨洁[1] 乔纯[1] 张闰 何广胜[1] 李建勇[1] YANG Hui;XU Dan;XU Jin-ge;GONG Yue-min;LI Yue;WU Yu-jie;QIAO Chun;ZHANG Run;HE Guang-sheng;LI Jianyong(Department of Hematology,the First Affiliated Hospital of Nanjing Medical University,Jiangsu Province Hospital,Nanjing 212009,China;不详)

机构地区：[1]南京医科大学附属第一医院、江苏省人民医院血液科,江苏南京212009 [2]阜宁县人民医院血液科,江苏盐城224400 [3]徐州医科大学第二附属医院血液科,江苏徐州221006

出　　处：《中国实用内科杂志》2023年第7期560-566,共7页Chinese Journal of Practical Internal Medicine

基　　金：江苏省科教能力提升工程(ZDXK202209);徐州市科技计划项目(KC21244);徐州市卫生健康委员会青年医学创新项目(XWKYHT20200028)。

摘　　要：目的评估糖皮质激素在阵发性睡眠性血红蛋白尿症(PNH)维持治疗中的作用。方法纳入2020年9月至2022年4月在江苏省人民医院等3家医院诊治的26例疾病高活动状态的PNH患者,自身对照分析予以泼尼松1.0 mg/(kg·d)治疗前后贫血、溶血、血栓形成状态、慢性肾损伤指标变化。结果治疗后1、3、6个月无患者达到溶血控制[乳酸脱氢酶(LDH)<1.5×正常上限(ULN)],血清乳酸脱氢酶[(5.47±2.55)×ULN、(5.00±3.06)×ULN、(4.84±2.24)×ULN、(5.21±2.44)×ULN,P=0.436]、间接胆红素水平[(21.95±14.30)μmol/L、(21.42±10.54)μmol/L、(21.82±15.80)μmol/L、(23.95±17.30)μmol/L,P=0.881]、外周血网织红细胞比例[(7.28±4.47)%、(6.23±4.08)%、(6.62±3.92)%、(7.11±4.73)%,P=0.457]、D-二聚体水平[(1.07±2.02)mg/L、(0.61±0.93)mg/L、(0.59±0.85)mg/L、(0.45±0.40)mg/L,P=0.263]治疗前与治疗后1、3、6个月比较未见改善。与治疗前相比,治疗后1个月血红蛋白得到提高[(69.12±14.83)g/L比(77.50±18.11)g/L,P<0.05],而3、6个月未见差异[(69.12±14.83)g/L比(74.15±18.23)g/L,P=0.15;(69.12±14.83)g/L比(75.54±19.91)g/L,P=0.09]。治疗前后肾小球滤过率无差别[(114.15±42.98)mL/(min·1.73 m^(2))、(117.79±37.69)mL/(min·1.73 m^(2))、(120.85±44.05)mL/(min·1.73 m^(2))、(110.30±36.58)mL/(min·1.73 m^(2))](P=0.42,P=0.43,P=0.41)。结论泼尼松在维持治疗PNH过程中不能控制溶血,不能改善贫血、血栓情况及肾功能,不建议使用糖皮质激素维持治疗PNH。Objective To evaluate the role of glucocorticoid in maintenance treatment of paroxysmal nocturnal hemoglobinuria(PNH).Methods 26 patients with PNH who were diagnosed and treated in Jiangsu Province Hospital and other 3 hospitals from September 2020 to April 2022 were included.The changes of anemia,hemolysis,thrombosis and chronic kidney injury indexes before and after treatment with prednisone 1.0 mg/(kg·d)were analyzed by self-control.Results No patients achieved remission of hemolysis(lactate dehydrogenase(LDH)<1.5×ULN)at 1,3,and 6 months;the level of LDH[(5.47±2.55)×ULN、(5.00±3.06)×ULN、(4.84±2.24)×ULN、(5.21±2.44)×ULN,P=0.436],indirect bilirubin(IBIL)[(21.95±14.30)μmol/L、(21.42±10.54)μmol/L、(21.82±15.80)μmol/L、(23.95±17.30)μmol/L,P=0.881],reticulocyte percentage[(7.28±4.47)%、(6.23±4.08)%、(6.62±3.92)%、(7.11±4.73)%,P=0.457]and D-dimer[(1.07±2.02)mg/L、(0.61±0.93)mg/L、(0.59±0.85)mg/L、(0.45±0.40)mg/L,P=0.263]showed no changes.After therapy,hemoglobin was increased at 1st month,but no difference was observed at 3rd and 6th months[(69.12±14.83)vs.(77.50±18.11),P<0.05;(69.12±14.83)vs.(74.15±18.23),P=0.15;(69.12±14.83)vs.(75.54±19.91),P=0.09].Glomerular filtration rate(GFR)at 1,3,and 6 months showed no difference compared to GFR before treatment[(114.15±42.98)mL/(min·1.73 m^(2))、(117.79±37.69)mL/(min·1.73 m^(2))、(120.85±44.05)mL/(min·1.73 m^(2))、(110.30±36.58)mL/(min·1.73 m^(2))](P=0.42,P=0.43,P=0.41).Conclusion Prednisone can not control hemolysis,improve anemia,thrombosis and renal function during maintenance treatment of PNH,so glucocorticoid maintenance treatment of PNH is not recommended.

关 键 词：糖皮质激素 阵发性睡眠性血红蛋白尿症 疾病高活动状态 溶血 贫血 血栓 肾功能 

分 类 号：R552[医药卫生—血液循环系统疾病]