艾曲波帕治疗儿童慢性免疫性血小板减少症3例并文献复习

Eltrombopag for the treatment of chronic immune thrombocytopenia in children: a report of 3 cases and literature review

目的探讨艾曲波帕治疗儿童慢性免疫性血小板减少症(cITP)的疗效及安全性。方法收集3例应用艾曲波帕治疗cITP患儿的临床资料并复习相关文献。结果 3例cITP患儿,男2例,女1例,年龄8~10岁,反复皮肤瘀点瘀斑,血小板计数(PLT)低于20×109/L,类固醇激素依赖或无效,艾曲波帕治疗前病程均超过12个月;口服艾曲波帕25mg qd[约1mg/(kg·d)],1~2周后均出现治疗反应(PLT> 50×109/L),瘀点瘀斑消失。由于经济原因,1例于治疗2个月后停药,停药2周后PLT下降至7×109/L;1例治疗5个月后停药,继续随访1年PLT稳定于50×109/L以上。1例艾曲波帕加量[约2mg/(kg·d)]后,血小板计数恢复正常。3例在口服艾曲波帕期间均未出现不良反应。结论艾曲波帕口服方便且较为安全,能短期内提升cITP患儿的血小板水平,改善出血症状,疗效可能与疗程和剂量相关,但费用较高,在国内长期治疗受到限制。

Objective To assess the efficacy and safety of Eltrombopag for the treatment of chronic immune thrombocytopenia( cITP) in children. Methods Clinical data of 3 cases with cITP who were treated with Eltrombopagwere was collected and literature reviewed. Results 2 male patient and 1 female patient with cITP,( 8 ~ 10 years old) were corticosteroid-dependent or no response to corticosteroids,and the course lasted longer than 12 months. All three patients received oral Eltrombopag 25 mg qd [about1 mg/( kg·d) ],and all had a treatment response after 1 to 2 weeks( platelet count > 50 × 109/L). Due to insufficient expenses,one patient discontinued Eltrombopag after 2 months of treatment,and the platelet count decreased to 7 × 109/L after 2 weeks of discontinuation;while another patient discontinued Eltrombopag after 2 months of treatment,and the platelet count remained stable at 50 × 109/L after 1 year of follow-up. Platelet count reached a normal value after increasing the dose of Eltrombopag [about 2 mg/( kg·d) ]. There were no adverse effects in these 3 patients with Eltrombopag. Conclusions Eltrombopag is an oral preparation and has high safety. It can increase the platelet level of children with cITP in a short time and reduce the risk of bleeding. Sufficient treatment course and dose are related to the efficacy. However the costs limit the longterm treatment in China.