Yang W R, Han B, Chang H, Wu B Y, Meng F K, Ji D X, Li Y M, Zheng Z J, Fei Y, Shen J P, Hu P, Ding X Q, Zhang P, Wang Y Q, Zhang F K
Department of Anemia Therapeutic Center, State Key Laboratory of Experimental Hematology, National Clinical Research Center for Blood Diseases, Institute of Hematology & Blood Diseases Hospital, Chinese Academy of Medical Sciences & Peking Union Medical, Tianjin 300020, China.
Peking Union Medical College Hospital, Chinese Academy of Medicine Sciences, Beijing 100730, China.
Zhonghua Xue Ye Xue Za Zhi. 2020 Nov 14;41(11):890-895. doi: 10.3760/cma.j.issn.0253-2727.2020.11.002.
To evaluate the safety and efficacy of eltrombopag combined with immunosuppressive therapy in patients with aplastic anemia (AA) in China. 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. 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. 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.
评估在中国再生障碍性贫血（AA）患者中，艾曲泊帕联合免疫抑制疗法的安全性和有效性。我们调查并分析了来自14个血液治疗中心的AA患者的临床数据，这些患者接受口服艾曲泊帕治疗至少3个月。我们纳入了56例AA患者，包括19例初治患者和37例免疫抑制治疗难治性患者。艾曲泊帕的中位给药期为7（3 - 31）个月，中位最大稳定剂量为75mg/d（50 - 150mg/d）。在10例接受一线艾曲泊帕和标准免疫抑制治疗（抗胸腺细胞球蛋白+环孢素）的重型再生障碍性贫血（SAA）患者中，3个月血液学缓解（HR）率为60%，完全缓解（CR）率为30%。9例接受艾曲泊帕和环孢素±雄激素一线治疗的SAA患者中有8例有反应（8/9，89%），4例（44%）达到CR。到随访期末，这19例患者的总体HR和CR率分别为79%和52.6%。在19例对环孢素±雄激素难治的AA患者中，11例在3个月时达到HR（57.9%），在标准免疫抑制治疗（抗胸腺细胞球蛋白+环孢素）难治的18例患者中，接受艾曲泊帕治疗后最佳HR率为44%。51%的患者经历了轻度或中度不良事件，胃肠道不适是研究对象报告的最常见不良反应。在一线免疫抑制治疗中加用艾曲泊帕可加速AA患者血液学反应的获得并改善反应质量。具有相对较多残留造血细胞的AA患者可能用艾曲泊帕和非抗胸腺细胞球蛋白免疫抑制治疗效果良好。艾曲泊帕可作为环孢素±雄激素难治性患者的挽救治疗。在中国，艾曲泊帕对AA患者总体安全且耐受性良好。
