【作者】 马莉；

【导师】 王艳；

【作者基本信息】 河北医科大学 ， 内科学（专业学位）， 2023， 硕士

【摘要】 目的:评估艾曲泊帕治疗在治疗原发免疫性血小板减少症(primary immune thrombocytopenia,ITP)的疗效和不良反应。方法:1.回顾性分析2015年12月至2022年6月于河北医科大学第二医院确诊为ITP(诊断符合成人原发免疫性血小板减少症诊断与治疗中国指南(2020年版))且应用艾曲泊帕作为二线及以上治疗方案的124例患者的临床资料,应用Wilcoxon符号秩检验比较艾曲泊帕治疗后不同时间的血小板计数水平,应用卡方检验分析不同起始剂量组别的疗效比较。2.回顾性分析患者一般资料、疗效判断及安全性。结果:1.一般资料:本次研究共纳入124例患者,男性51例,女性73例,中位年龄46(35-58)岁。确诊ITP患者病程3个月-10年。2.疗效判断:(1)124例患者治疗前中位血小板计数(PLT)值为9(4-17)×10~9/L,治疗4周后PLT中位值为45(25-100.75)×10~9/L,艾曲泊帕治疗总体有效率70.16%。(2)其中有76例治疗有效且持续用药≥12周的患者,在12周时中位血小板计数为84.5(23-123.75)×10~9/L,艾曲泊帕起始剂量25mg/d(32例),此治疗剂量的总有效率(PLT≥30×10~9/L且较基础值增加2倍以上)43.75%(14/32),最快起效时间4.3天,平均起效时间7.8天;起始剂量50mg/d组,此治疗剂量的有效率显著提升至81.81%(36/44),最快起效时间3.2天,平均起效时间6.4天,这表明持续有效率随起始剂量的增加而增高。3.不良反应:肝转氨酶及胆红素的升高及头痛等是常见的不良反应,但经过积极治疗均可正常或回落至基线水平,在艾曲泊帕治疗过程中出现1例血小板计数高导致的深静脉血栓1例。结论:1.ITP女性发病率高于男性。2.艾曲泊帕在二线及以上的持续性/慢性ITP治疗的总体有效率为70.16%3.长期使用艾曲泊帕时起始剂量为50mg/d比起始剂量为25mg/d能够更快速提升血小板至有效水平,50mg/d剂量有效率更高。4.艾曲泊帕在治疗持续性/慢性ITP时疗效显著且安全性较高。更多还原

【Abstract】 Objective:to observe the clinical efficacy of eltrombopag therapy in the primary immune thrombocytopenia(ITP).Methods:1.A retrospective analysis was conducted of the clinical data of 124patients with ITP diagnosed in the Second Hospital of Hebei Medical University from December 2015 to June 2022(diagnosis in accordance with the Chinese Guidelines for the Diagnosis and Treatment of Adult Primary Immune Thrombocytopenia(Version 2020))and who were treated with eltrombopag as a second-line or higher treatment regimen.Wilcoxon signed rank test was used to compare the platelet count levels at different times after treatment with i eltrombopag,Chi-square test was used to analyze the efficacy of different initial dose groups.2.The general data,efficacy judgment and safety of the patients were retrospectively analyzed.Results:1.General data:Among 124 patients,51 were male and 73 were female,with a median age of 46(35-58)years.The course of diagnosis of ITP patients is 3 months to 10 years.2.Efficacy evaluation:(1)The median platelet count(PLT)of 124patients before treatment was 9(4-17)×10~9/L,the median PLT after 4 weeks of treatment is 45(25-100.75)×10~9/L,the overall effective rate of eltrombopag treatment is 70.16%.(2)At 12 week,76 patients with effective treatment and continuous medication≥12 weeks had a median platelet count of 84.5(23-123.75)×10~9/L,the initial dose of eltrombopag is 25mg/d(32 cases),the total effective rate of this treatment dose(PLT≥30×10~9/L and more than twice the base value)43.75%(14/32),the fastest onset time is 4.3 days,and the average onset time is 7.8 days;In the initial dose 50mg/d group,the effective rate of this treatment dose was significantly increased to 81.81%(36/44),the fastest onset time was 3.2 days,and the average onset time was 6.4 days,indicating that the continuous effective rate increased with the increase of the initial dose.3.Adverse reactions:The increase of liver transaminase and bilirubin and headache are common adverse reactions,but after active treatment,they can be normal or fall back to the baseline level.During the treatment of eltrombopag,1 case of deep venous thrombosis caused by high platelet count occurred in 1case.Conclusions:1.The incidence rate of ITP in women is higher than that in men。2.The overall effective rate of eltrombopag in the treatment of persistent/chronic ITP at the second line and above is 70.16%.3.The initial dose of 50 mg/d for long-term use of eltrombopag can promote platelets faster than the initial dose of 25 mg/d,and the effective rate of 50 mg/d is higher.4.Eltrombopag has significant efficacy and high safety in the treatment of persistent/chronic ITP.更多还原