• 抗人T细胞猪免疫球蛋白联合海曲泊帕治疗重型再生障碍性贫血的临床疗效
  • 陈小玉.抗人T细胞猪免疫球蛋白联合海曲泊帕治疗重型再生障碍性贫血的临床疗效[J].内科急危重症杂志,2025,31(2):126-129
    DOI:10.11768/nkjwzzzz20250206
    中文关键词:  再生障碍性贫血  抗人T细胞猪免疫球蛋白  海曲泊帕
    英文关键词:
    基金项目:国家自然科学基金青年基金(81900109);江苏省自然科学基金青年项目(BK20230734);南京市江宁区科技惠民计划项目(2023081S)
    作者单位E-mail
    陈小玉 南京医科大学第一附属医院 江苏省人民医院 yx021021@sina.com 
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    中文摘要:
          摘要 目的:研究以抗人T细胞猪免疫球蛋白(p-ATG)为基础的强化免疫抑制治疗(IST)联合海曲泊帕在初发重型再生障碍性贫血中的疗效及安全性。方法:前瞻性纳入31例初发重型再生障碍性贫血(SAA)患者,均接受p-ATG为基础的IST联合海曲泊帕治疗。结果:治疗后6个月的完全反应率和总体反应率分别为26.2%、84.3%。中位随访时间9(0~28)个月,总生存率84.2%,中位无事件生存期为7(0~ 28)个月。治疗1个月内感染发生率为71.0%(22/31),3例发生≥3级肝功能损害。结论:以p-ATG为基础的IST联合海曲泊帕治疗SAA可获得较为满意的疗效,耐受性和安全性良好。
    英文摘要:
          Abstract Objective: To evaluate the efficacy and safety of anti-human T-cell porcine immunoglobin (p-ATG) based intensive immunosuppressive therapy (IST) combined with hetrombopag in patients with severe aplastic anemia (SAA). Methods: In total, 31 patients newly diagnosed with SAA were prospectively enrolled. All patients received IST, based on p-ATG, combined with hetrombopag. Results: The complete response rate and overall response rate at 6 months after treatment were 26.2% and 84.3%, respectively. The median follow-up time was 9 (0-28) months, the overall survival rate was 84.2%, and the median event-free survival (EFS) time was 7 (0-28) months. The incidence of infection within 1 month after treatment was 71.0% (22/31), and 3 patients developed ≥ grade 3 liver function damage. Conclusions: IST based on p-ATG combined with hetrombopag is effective and safe in the treatment of SAA.