阿伐曲泊帕联合他克莫司治疗老年难治性重型再生障碍性贫血1例报告

A case report of elderly patient with refractory severe aplastic anemia by treatment of avatrombopag in combination with tacrolimus

  • 摘要:
    目的  总结阿伐曲泊帕联合他克莫司治疗老年难治性重型再生障碍性贫血的临床疗效和不良反应。
    方法  报道1例阿伐曲泊帕联合他克莫司治疗老年难治性重型再生障碍性贫血患者,并针对阿伐曲泊帕、他克莫司作用机制及安全性进行文献复习。
    结果  患者经环孢素、十一酸睾酮、艾曲泊帕联合治疗6个月无效,并出现肝肾功能损伤,停药并予以阿伐曲泊帕(20 mg, 每日1次)联合他克莫司(1 mg, 每日2次)治疗9个月后,患者血小板升至54×109/L, 血红蛋白升至124 g/L, 白细胞升至8.23×109/L, 肝肾功能也均恢复到正常, 未观察到血栓事件等其他不良反应,且治疗后CD4/CD8下降, CD8+T细胞增多, Treg细胞比例增多。
    结论  阿伐曲泊帕联合他克莫司治疗老年难治性再生障碍性贫血有效且耐受性良好,今后仍需长期追踪观察及继续积累病例。

     

    Abstract:
    Objective  To summarize the clinical effect and adverse reactions of avatrombopag combined with tacrolimus in the treatment of elderly patient with refractory severe aplastic anemia.
    Methods  An elderly patient with refractory severe aplastic anemia was treated with avatrombopag and tacrolimus, and the literature review on the mechanism of action and safety of avatrombopag and tacrolimus was conducted.
    Results  The patient was treated with a combination of cyclosporine, testosterone undecanoate, and itraconazole for 6 months without success, and developed liver and kidney dysfunction. The medication was discontinued, and the patient was treated with a combination of avatrombopag (20 mg, once per day) and tacrolimus (1 mg, twice per day). After 9 months of treatment, the patient's platelet count increased to 54×109/L, the hemoglobin increased to 124 g/L, the white blood cells increased to 8.23×109/L, liver and kidney function also returned to normal level, and no other adverse reactions such as thrombotic events were observed. After treatment, CD4/CD8 decreased, CD8+ T cells increased, and the proportion of Treg cells increased as well.
    Conclusion  The combination of avatrombopag and tacrolimus is effective and well tolerated in the treatment of elderly patient with refractory aplastic anemia, but the long-term follow-up observation and continued accumulation of cases are still needed in the future.

     

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