艾曲波帕联合标准剂量强化免疫抑制治疗老年重型再生障碍性贫血

Intensive immunosuppression combined with eltrombopag in the treatment of elderly patients with severe aplastic anemia

目的:探讨艾曲波帕联合强化免疫抑制剂治疗老年重型再生障碍性贫血(SAA)的疗效。方法:对12例老年SAA患者采用艾曲波帕(75 mg/d维持6个月),抗人胸腺细胞免疫球蛋白(ATG):兔ATG 3.5 mg/(kg·d)×5 d或猪ATG 30 mg/(kg·d)×5 d联合环孢菌素A(CsA),谷浓度维持150~200 ng/ml,评估患者的疗效及耐受性。结果:治疗后1、3、6、9个月的有效率分别为18.2%、40.0%、55.6%、66.7%。治疗有效者血小板计数和网织红细胞计数均显著改善(P<0.05)。治疗后Treg水平较治疗前显著升高(P=0.007)。治疗期间监测肝肾功能均维持在正常范围。高龄(≥70岁)和高查尔森指数(≥5分)患者的疗效不佳(100%vs 25%,P=0.048;100%vs 0%,P=0.012)。结论:老年SAA患者使用艾曲波帕联合用药ATG+CsA疗效确切,患者耐受良好,值得进一步探索。

Objective:To investigate the efficacy of eltrombopag combined with intensive immunosuppressive therapy in the treatment of patients with elderly severe aplastic anemia(SAA).Method:A total of 12 patients with elderly SAA were treated with eltrombopag(75 mg/d for 6 months)combined with antithymocyte immunoglobulin(ATG):rabbit ATG 3.5 mg/(kg·d)×5 days or porcine ATG 30 mg/(kg·d)×5 days and cyclosporine A(CsA),with a maintained serum concentration range of 150 to 200 ng/ml.The efficacy and tolerability were evaluated.Result:The effective rates in the 1 st month,the 3 rd month,the 6 th month,the 9 th month after treatment were 18.2%,40.0%,55.6%and 66.7%,respectively.The platelet count and the reticulocyte count were significantly improved(P<0.05).The percentage of Treg also increased significantly(P=0.007).Liver and kidney function were monitored and maintained in the normal range during the treatment.Elderly patients(≥70 years old)and high Charlesson index(≥5 points)had poor efficacy(100%vs 25%,P=0.048;100%vs 0%,P=0.012).Conclusion:The use of eltrombopag in combination with ATG+CsA in elderly patients with SAA may be effective,and the patients tolerate it well.It is worthy of further exploration of this regimen.