目的:对已发表的罗普司亭治疗成人原发免疫性血小板减少症(ITP)的经济学评价进行系统综述。方法:通过检索3个中文数据库(中国知网、万方数据库、维普数据库)、3个英文数据库(PubMed、Embase、Web of Science)并进行文献筛选、资料提取...目的:对已发表的罗普司亭治疗成人原发免疫性血小板减少症(ITP)的经济学评价进行系统综述。方法:通过检索3个中文数据库(中国知网、万方数据库、维普数据库)、3个英文数据库(PubMed、Embase、Web of Science)并进行文献筛选、资料提取和信息汇总,按照卫生经济研究质量评价工具(QHES)对文献质量进行评价。结果:共纳入6篇文献,且研究质量较好。其中一半从卫生体系视角出发,一半从医保支付方视角出发。大部分研究采用Markov模型模拟的成本-效用分析(CUA)方法,也有研究基于决策树的CUA、成本-效果分析、最小成本分析、成本-结果模型等方法开展评价。模型状态设置主要由血小板反应驱动,模拟周期均为4周,关注的结局指标涉及反应率、出血、不良反应、生命年(LY)、质量调整生命年(QALY)、增量成本-效果比(ICER)。有2篇研究认为,与艾曲泊帕相比,罗普司亭治疗成人ITP具有成本-效果优势。结论:罗普司亭用于治疗成人ITP的经济性价值有待进一步探讨,罗普司亭用于治疗我国成人ITP需要高质量的本土经济学评价以提供决策依据。展开更多
Thrombocytopenia is a multifactorial disorder that is common in patients with chronic liver disease(CLD),leading to challenging perioperative planning.As thrombocytopenia in CLD is associated with thrombopoietin(TPO)d...Thrombocytopenia is a multifactorial disorder that is common in patients with chronic liver disease(CLD),leading to challenging perioperative planning.As thrombocytopenia in CLD is associated with thrombopoietin(TPO)deficiency,the use of TPO-receptor agonists(TPO-RAs)to increase platelet counts is a promising approach.This has led to the development of various TPO-RAs,including romiplostim,eltrombopag,avatrombopag,and lusutrombopag.Of these,only avatrombopag and lusutrombopag are approved by the United States Food and Drug Administration for the perioperative treatment of thrombocytopenia in patients with CLD.Platelet transfusion is commonly used for the clinical management of thrombocytopenia in patients with CLD undergoing invasive procedures.However,the limitations and possible risks of transfusion,including short duration of efficacy,development of antiplatelet antibodies,risk of infections and such complications as transfusion-related acute lung injury or circulatory overload,and possibility of refractoriness,limit its use.Moreover,there is no consensus among guidelines as to the platelet count at which transfusions are indicated.Results from studies using TPO-RAs perioperatively in patients with thrombocytopenia and CLD are promising and provide an alternative to platelet transfusions in the pre-and post-operative setting.These TPO-RAs are the subject of this review,with focus on their use in the perioperative setting in patients with thrombocytopenia,associated supporting clinical trials,efficacy and safety data,and their use with respect to platelet transfusions.展开更多
Second generation thrombopoietin agonists including eltrombopag and romiplostim act on the thrombopoietin receptor to increase the megakaryocyte production. These agents were needed as use of first generation recombin...Second generation thrombopoietin agonists including eltrombopag and romiplostim act on the thrombopoietin receptor to increase the megakaryocyte production. These agents were needed as use of first generation recombinant products was associated with formation of autoantibodies. Eltrombopag is an oral thrombopoietin agonist found effective in raising platelet counts in patients with immune thrombocytopenia. The drug has now been found to be useful in raising platelet counts in thrombocytopenia related to liver disease including cirrhosis and chronic viral hepatitis. Although the drug may help enable adequate interferon therapy in patients with HCV infection and help carry out invasive procedures in patients with cirrhosis, concerns have been raised of possible thrombotic complications including portal vein thrombosis. Randomized trials have shown that use of eltrombopag concomitant with pegylated interferon and ribavirin increased the chances of sustained virologic response while decreasing the dose reductions of interferon. The data on use of romiplostim in these clinical indications is also emerging. However, in the future, availability of interferon free regimens is likely to decrease the use of eltrombopag for enabling antiviral therapy. The review discusses the role of eltrombopag in management of liver disease related thrombocytopenia in wake of recent data as also the dosage, precautions and adverse effects associated with its use.展开更多
文摘目的:对已发表的罗普司亭治疗成人原发免疫性血小板减少症(ITP)的经济学评价进行系统综述。方法:通过检索3个中文数据库(中国知网、万方数据库、维普数据库)、3个英文数据库(PubMed、Embase、Web of Science)并进行文献筛选、资料提取和信息汇总,按照卫生经济研究质量评价工具(QHES)对文献质量进行评价。结果:共纳入6篇文献,且研究质量较好。其中一半从卫生体系视角出发,一半从医保支付方视角出发。大部分研究采用Markov模型模拟的成本-效用分析(CUA)方法,也有研究基于决策树的CUA、成本-效果分析、最小成本分析、成本-结果模型等方法开展评价。模型状态设置主要由血小板反应驱动,模拟周期均为4周,关注的结局指标涉及反应率、出血、不良反应、生命年(LY)、质量调整生命年(QALY)、增量成本-效果比(ICER)。有2篇研究认为,与艾曲泊帕相比,罗普司亭治疗成人ITP具有成本-效果优势。结论:罗普司亭用于治疗成人ITP的经济性价值有待进一步探讨,罗普司亭用于治疗我国成人ITP需要高质量的本土经济学评价以提供决策依据。
文摘Thrombocytopenia is a multifactorial disorder that is common in patients with chronic liver disease(CLD),leading to challenging perioperative planning.As thrombocytopenia in CLD is associated with thrombopoietin(TPO)deficiency,the use of TPO-receptor agonists(TPO-RAs)to increase platelet counts is a promising approach.This has led to the development of various TPO-RAs,including romiplostim,eltrombopag,avatrombopag,and lusutrombopag.Of these,only avatrombopag and lusutrombopag are approved by the United States Food and Drug Administration for the perioperative treatment of thrombocytopenia in patients with CLD.Platelet transfusion is commonly used for the clinical management of thrombocytopenia in patients with CLD undergoing invasive procedures.However,the limitations and possible risks of transfusion,including short duration of efficacy,development of antiplatelet antibodies,risk of infections and such complications as transfusion-related acute lung injury or circulatory overload,and possibility of refractoriness,limit its use.Moreover,there is no consensus among guidelines as to the platelet count at which transfusions are indicated.Results from studies using TPO-RAs perioperatively in patients with thrombocytopenia and CLD are promising and provide an alternative to platelet transfusions in the pre-and post-operative setting.These TPO-RAs are the subject of this review,with focus on their use in the perioperative setting in patients with thrombocytopenia,associated supporting clinical trials,efficacy and safety data,and their use with respect to platelet transfusions.
文摘Second generation thrombopoietin agonists including eltrombopag and romiplostim act on the thrombopoietin receptor to increase the megakaryocyte production. These agents were needed as use of first generation recombinant products was associated with formation of autoantibodies. Eltrombopag is an oral thrombopoietin agonist found effective in raising platelet counts in patients with immune thrombocytopenia. The drug has now been found to be useful in raising platelet counts in thrombocytopenia related to liver disease including cirrhosis and chronic viral hepatitis. Although the drug may help enable adequate interferon therapy in patients with HCV infection and help carry out invasive procedures in patients with cirrhosis, concerns have been raised of possible thrombotic complications including portal vein thrombosis. Randomized trials have shown that use of eltrombopag concomitant with pegylated interferon and ribavirin increased the chances of sustained virologic response while decreasing the dose reductions of interferon. The data on use of romiplostim in these clinical indications is also emerging. However, in the future, availability of interferon free regimens is likely to decrease the use of eltrombopag for enabling antiviral therapy. The review discusses the role of eltrombopag in management of liver disease related thrombocytopenia in wake of recent data as also the dosage, precautions and adverse effects associated with its use.
