Clinical analysis of eltrombopag in the treatment of primary immune thrombocytopenia
-
摘要: 目的:观察艾曲泊帕治疗成人原发免疫性血小板减少症(ITP)的疗效及安全性。方法:回顾性分析在我院住院应用艾曲泊帕治疗的75例ITP患者的临床资料。结果:①75例患者中男25例、女50例,中位年龄44(19~66)岁,治疗前血小板计数(PLT)中位值13(1~66)×109/L,治疗后第2周、第4周、第3个月及第6个月PLT中位值分别为29(1~286)×109/L、57(3~616)×109/L、67(3~300)×109/L、92(10~397)×109/L,明显高于治疗前PLT水平(均P=0.00);治疗总有效率81.33%(61/75例);PLT≥30×109/L的中位时间为14(3~42) d。②不同艾曲泊帕剂量组间的体重或BMI水平比较发现均不影响药物的起效剂量,P>0.05。36例患者既往接受重组人血小板生成素(rhTPO)治疗(11例为rhTPO治疗有效),结果显示rhTPO治疗是否有效不影响艾曲泊帕的疗效,P>0.05;另外,艾曲泊帕的疗效在不论血小板抗体阳性与否以及巨核细胞数目增多或正常组间比较,均差异无统计学意义,总有效率分别为81.82% vs 75.00%、87.88% vs 76.19%,均P>0.05。③肝脏转氨酶及胆红素升高为最常见的艾曲泊帕治疗相关不良反应,经治疗后均恢复正常或回至基线水平;期间发生血小板升高相关脑梗死1例。结论:艾曲泊帕对于ITP患者有效率高,为快速提升PLT水平、降低出血风险,推荐成人患者可选50 mg剂量起始,同时联合预防性保肝治疗。但艾曲泊帕停药后维持作用时间短暂、费用较昂贵。Abstract: Objective:To investigate the effect and safety of eltrombopag for the treatment of primary immune thrombocytopenia (ITP) in adults.Method:We retrospectively analyzed the clinical data of 75 cases with ITP inpatient treated with eltrombopag.Result:①The 75 patients included 25 males and 50 females,with a median age of 44 years (from 19 to 66 years).The median platelet count before treatment was 13(1 to 66)×109/L,the median platelet of patients at 2 weeks,4 weeks,3 months and 6 months after treatment were 29(1 to 286)×109/L,57(3 to 616)×109/L,67(3 to 300)×109/L,and 92(10 to 397)×109/L,and the platelet level after treatment was significantly higher than that before treatment (P=0.00).The total response rate was 81.33% (61/75 cases).The median time to platelet recovery ≥30×109/L after treatment was 14 days (3 to 42 days).②As to the possible related factors affecting drug efficacy,the results showed that there was no significant relationship between body weight or BMI level with the effective dose (P>0.05).Thirty-six of the 75 patients received rhTPO treatment (11 cases were effective in rhTPO treatment),the results showed that there was no statistically significant difference in the efficacy of eltrombopag with or without the effect of previous treatment of rhTPO (P>0.05);There was no significant difference in the response rate of patients with specific autoantibodies against platelet glycoprotein positive contrast with the negative group (81.82% vs 75.00%,P>0.05).The number of megakaryocytes in all patients was normal or increased,and there was no statistically significant difference in the therapeutic efficiency between the increased group and the normal group (87.88% vs 76.19%,P>0.05).③The adverse events which occurred more frequently due to eltrombopag included increased transaminase and blood bilirubin.All liver adverse events returned to normal or baseline after treatment.A case of cerebral infarction was associated with thrombocytopenia.Conclusion:Eltrombopag is an effective and safe treatment option for ITP,in order to rapidly improve platelet level and minimize the hemorrhage risk associated with decreased platelet counts,the recommended initial dose is 50 mg per day for adult patients,and combined with hepatoprotective therapy.But there is no curative effect soon after the drug is discontinued.
-
Key words:
- eltrombopag /
- thrombocytopenia /
- outcome /
- adverse events
-
-
[1] Kuter DJ.New thrombopoietic growth factors[J].Blood, 2007, 109:4607-4616.
[2] 李早荣, 胡锡林, 周巧慧, 等.骨髓巨核细胞和有核细胞计数的实验报告[J].现代检验医学杂志, 2003, 18 (4):20-21.
[3] Cheng G, Saleh MN, Marcher C, et al.Eltrombopag for management of chronic immune thrombocytopenia (RAISE):a 6-month, randomised, phase 3study[J].Lancet, 2011, 377:393-402.
[4] Yang R, Li J, Jin J, et al.Multicentre, randomised phase III study of the efficacy and safety of eltrombopag in Chinese patients with chronic immune thrombocytopenia[J].Br J Haematol, 2017, 176:101-110.
[5] Saleh MN, Bussel JB, Cheng G, et al.Safety and efficacy of eltrombopag for treatment of chronic immune thrombocytopenia:results of the long-term, open-label EXTEND study[J].Blood, 2013, 121:537-545.
[6] Mahévas M, Fain O, Ebbo M, et al.The temporary use of thrombopoietin-receptor agonists may induce a prolonged remission in adult chronic immune thrombocytopenia.Results of a French observational study[J].Br J Haematol, 2014, 165:865-869.
[7] Gibiansky E, Zhang J, Williams D, et al.Population pharmacokinetics of eltrombopag in healthy subjects and patients with chronic idiopathic thrombocytopenic purpura[J].J Clin Pharmacol, 2011, 51:842-856.
[8] Hayes S, Ouellet D, Zhang J, et al.Population PK/PD modeling of eltrombopag in healthy volunteers and patients with immune thrombocytopenic purpura and optimization of response-guided dosing[J].J Clin Pharmacol, 2011, 51:1403-1417.
[9] Zhou B, Zhao H, Yang RC, et al.Multi-dysfunctional pathophysiology in ITP[J].Crit Rev Oncol Hematol, 2005, 54:107-116.
[10] Kim TO, Despotovic J, Lambert MP.Eltrombopag for use in children with immune thrombocytopenia[J].Blood Adv, 2018, 2:454-461.
-
计量
- 文章访问数: 303
- PDF下载数: 90
- 施引文献: 0
下载: