原发免疫性血小板减少症患者真实世界治疗现状与疾病负担研究  

Real-world treatment status and disease burden in patients with primary immune thrombocytopenia

作　　者：王蕾 贺小宁[1,2] 李正翔[3] 杨建华 刘芳[5] 于鲁海[6] 张鹏[7] 王亚峰[8] 张弋[9] 吴晶[1,2] WANG Lei;HE Xiaoning;LI Zhengxiang;YANG Jianhua;LIU Fang;YU Luhai;ZHANG Peng;WANG Yafeng;ZHANG Yi;WU Jing(School of Pharmaceutical Science and Technology,Tianjin University,Tianjin 300072,China;Center for Social Science Survey and Data,Tianjin University,Tianjin 300072,China;Department of Pharmacy,Tianjin Medical University General Hospital,Tianjin 300052,China;Department of Pharmacy,The First Affiliated Hospital of Xinjiang Medical University,Xinjiang Urumqi 830011,China;Department of Pharmacy,First Teaching Hospital of Tianjin University of TCM,Tianjin 300380,China;Department of Pharmacy,People's Hospital of Xinjiang Uygur Autonomous Region,Xinjiang Urumqi 830001,China;Department of Pharmacy,Shaanxi Provincial People's Hospital,Shaanxi Xi'an 710068,China;Department of Pharmacy,Qinghai Provincial People's Hospital,Qinghai Xining 810600,China;Department of Pharmacy,Tianjin First Central Hospital,Tianjin 300192,China)

机构地区：[1]天津大学药物科学与技术学院,天津300072 [2]天津大学社会科学调查与数据中心,天津300072 [3]天津医科大学总医院药剂科,天津300052 [4]新疆医科大学第一附属医院药学部,新疆乌鲁木齐830011 [5]天津中医药大学第一附属医院药学部,天津300380 [6]新疆维吾尔自治区人民医院药学部,新疆乌鲁木齐830001 [7]陕西省人民医院药学部,陕西西安710068 [8]青海省人民医院药学部,青海西宁810600 [9]天津市第一中心医院药学部南开大学医学院,天津300192

出　　处：《中国医院药学杂志》2023年第14期1602-1607,共6页Chinese Journal of Hospital Pharmacy

摘　　要：目的:评估真实世界下原发免疫性血小板减少症(ITP)患者的治疗现状与疾病负担,并对比艾曲泊帕与重组人血小板生成素(rhTPO)的治疗情况与费用。方法:收集2021年7月1日—12月31日4个城市6家三甲医院中正在接受二线治疗的成人慢性ITP患者的病历资料,进行回顾性分析。结果:ITP患者(n=55)随访期6个月内二线治疗的用药时间为(48.1±52.8)d,使用二线治疗后患者血小板较基线显著提升,总反应率达90.9%。36.4%的患者发生过出血,人均出血次数(1.0±1.9)次。94.5%的患者有过ITP特异住院,仅52.7%的患者有过ITP门诊就诊。人均随访ITP特异总直接医疗费用为(46169.5±38253.4)元,其中住院费用占比92.2%。艾曲泊帕组(n=28)的用药依从性显著高于rhTPO组(n=21)[(0.4±0.3)vs.(0.1±0.1),P<0.01)],同时也带来了更高的人均药品费用[(32253.0±28350.8)元vs.(11046.0±9265.3)元,P<0.01]。结论:中国ITP患者的总体治疗现状不容乐观,患者用药依从性较差,门诊就诊率较低,直接医疗费用较高。与rhTPO相比,使用艾曲泊帕的患者用药依从性更好,费用更高。建议各地积极探索进一步提升ITP门诊医保报销待遇,提高门诊就诊率,改善患者预后,提升ITP疾病管理水平。OBJECTIVE To evaluate real-world treatment status and disease burden in patients with primary immune thrombocytopenia(ITP),and to compare the treatment and costs between eltrombopag and recombinant human thrombopoietin(rhTPO).METHODS Adult patients with chronic ITP receiving second-line therapies were enrolled in six tertiary grade A hospitals in four cities in China,and their medical records between July 1 and December 31,2021 were retrospectively analyzed.RESULTS During the six months of follow-up period,the average days covered by second-line therapy of ITP patients(n=55)was(48.1±52.8)days.After receiving second-line therapies,patients’platelet was significantly increased,with the overall response rate of 90.9%.36.4%patients occurred bleeding events,with the mean number of(1.0±1.9)times.94.5%patients had ITP-specific hospitalization,and only 52.7%had ITP-specific outpatient visits.ITP-specific cost was￥(46169.5±38253.4),of which hospitalization cost accounted for 92.2%.The adherence to eltrombopag(n=28)was significantly higher than that of rhTPO(n=21)[(0.4±0.3)vs.(0.1±0.1),P<0.01)].Meanwhile,the medication costs per patient[￥(32253.0±28350.8)vs.￥(11046.0±9265.3),P<0.01]in group eltrombopag were significantly higher than those in group rhTPO.CONCLUSION The overall diagnosis and treatment status of patients with ITP in China is not optimistic.The medication adherence was poor,the outpatient visiting rate was low,and patients had high direct medical costs.Compared with rhTPO,eltrombopag had better adherence and higher costs.It is suggested that all localities actively explore ways to improve ITP outpatient medical insurance reimbursement,to further improve the outpatient visiting rate,the clinical prognosis,and the disease management of ITP.

关 键 词：原发免疫性血小板减少症 二线治疗 治疗现状 经济负担 真实世界研究 

分 类 号：R956[医药卫生—药学]