Objective: To evaluate the safety and efficacy of eltrombopag combined with immunosuppressive therapy in patients with aplastic anemia (AA) in China. Methods: We investigated and analyzed the clinical data of AA patients from 14 hematological treatment centers who were treated with oral eltrombopag for at least 3 mon. Results: We enrolled 56 AA patients, including 19 treatment-naïve patients and 37 IST-refractory patients. The median administration period for eltrombopag was 7 (3-31) months, and the median maximum stable dosage was 75 mg/d (50-150 mg/d) . The 3-month hematological response (HR) rate was 60%, and the complete response (CR) rate was 30% in 10 SAA patients who were treated with first-line eltrombopag and standard IST (ATG+CsA) . Eight of 9 eltrombopag and CsA ± androgen first-line treated SAA patients responded (8/9, 89%) and 4 (44%) gave CR. The overall HR and CR rates were 79% and 52.6%, respectively, among these 19 patients by the end of the follow-up period. Of the 19 AA patients who were refractory to CsA ± androgen, 11 achieved HR (57.9%) at 3 mon, and the best HR rate was 44% in standard IST (ATG+CsA) refractory 18 patients after eltrombopag treatment. Fifty-one percent of the patients experienced mild or moderate adverse events, and gastrointestinal discomfort was the most common adverse effect reported by the study subjects. Conclusion: Adding Eltrombopag in first-line IST can accelerate the acquisition and improve the quality of hematological responses in AA patients. AA with relatively more residual hematopoietic cells may be well treated with eltrombopag and non-ATG IST. Eltrombopag can be used as salvage therapy for CsA±androgen refractory patients. Eltrombopag was generally safe and well tolerated by AA patients in China.
目的： 评估艾曲泊帕联合免疫抑制治疗（IST）在中国再生障碍性贫血（AA）患者中的疗效及安全性。 方法： 调查分析2015年4月至2019年5月来自中国14个血液病治疗中心、连续应用艾曲泊帕治疗至少3个月的输血依赖非重型和重型AA患者临床资料。 结果： 纳入19例IST初治及37例IST难治性AA患者，中位艾曲泊帕治疗时间为7（3~31）个月，中位艾曲泊帕最大稳定使用剂量为75（50~150）mg/d。10例应用艾曲泊帕联合标准IST一线治疗的SAA患者3个月血液学反应（HR）率为60%，完全HR（CR）率为30%；9例艾曲泊帕联合环孢素A（CsA）一线治疗SAA患者后3个月HR率为89%，CR率为44%。19例患者应用艾曲泊帕联合IST一线治疗，至调查随访时HR率为79%，CR率为52.6%。19例CsA±雄激素治疗无效的AA患者，艾曲泊帕开始治疗后3个月HR率为57.9%（11/19），最佳HR率为68.4%；标准IST治疗无效的难治性AA，艾曲泊帕治疗最佳HR率为44%。51%患者应用艾曲泊帕发生不同严重程度的不良反应，以胃肠道不适最为常见。 结论： 艾曲泊帕治疗可加快HR的获得，改善HR质量；残存造血细胞较多的AA患者或可采用非抗胸腺细胞球蛋白免疫抑制联合艾曲泊帕治疗；艾曲泊帕可作为CsA±雄激素治疗无效AA的补救治疗；艾曲泊帕不良反应轻微，多可耐受。.
Keywords: Aplastic anemia; Eltrombopag.