艾曲泊帕联合强化免疫治疗老年重型再生障碍性贫血35例分析

Eltrombopag combing with intensive immunosuppressive therapy for elderly severe aplastic anemia:clinical analysis of 35 cases

目的评估艾曲泊帕联合抗人胸腺细胞免疫球蛋白(ATG)和环孢素(CsA)强化免疫抑制治疗(IST)老年重型再生障碍性贫血(SAA)疗效。方法收集2017年11月至2020年11月来自连云港市第二人民医院、江苏省人民医院、南京市第二人民医院、浙江省中医院、吉林大学第一医院、北京大学人民医院的35例老年重型再生障碍性贫血患者的临床资料,分析治疗方案、疗效及死亡影响因素。结果强化免疫抑制治疗单用或联合艾曲泊帕治疗1个月、3个月、6个月、9个月的有效率分别为0%vs.8.6%(P=0.464),9%vs.47.8%(P=0.03),27.3%vs.72.7%(P=0.025),30%vs.79.2%(P=0.002)。高龄(≥70岁)和高查尔森指数(≥5分)患者的疗效不佳(60%vs.40%,P=0.011;73.3%vs.26.7%,P=0.033)。多因素分析发现:使用艾曲泊帕(P=0.012)、中性粒细胞计数(P=0.035)与IST疗效正相关,而年龄(P=0.004)、查尔森合并症指数(P=0.022)与疗效负相关;高查尔森合并症指数(P=0.041)和低中性粒细胞计数(P=0.035)对患者生存不利。结论对于老年严重型再生障碍性贫血,IST联合艾曲泊帕疗效肯定;高龄(≥70岁)、高查尔森合并症指数(≥5分)以及治疗前粒细胞缺乏症(<0.5×109/L)患者,疗效较差,生存不佳。

Objective To evaluate the efficacy of the combination of eltrombopag with antithymocyte immunoglobulin(ATG)and cyclosporin A(Cs A)for elderly severe aplastic anemia(SAA).Methods The clinical data of 35 elderly patients with severe aplastic anemia were collected from the second people’s Hospital of Lianyungang City,Jiangsu Provincial People’s Hospital,Nanjing Second People’s Hospital,Zhejiang hospital of traditional Chinese medicine,Jilin University First Hospital and people’s Hospital of Peking University from November 2017 to November 2020,and analyzed the treatment plan,efficacy and death influencing factors.Results The response rates of IST alone or in combination with eltrombopag in the 1 st month,the 3 rd month,the 6 th month,the 9 th month after treatment were 0%vs.8.6%(P=0.464),9%vs.47.8%(P=0.03),27.3%vs.72.7%(P=0.025),30%vs.79.2%(P=0.002),respectively.Patients with advanced age(≥70 years old)or high Charson index(≥5 points)responded to IST poorly(60%vs.40%,P=0.011;73.3%vs.26.7%,P=0.033).Multivariate analysis showed that eltrombopag(P=0.012)and neutrophil count(P=0.035)were found to be predictive of improved response,while age(P=0.004)and Charlson’s comorbidity index(P=0.022)were found to be associated with response negatively.High Charson comorbidity index(P=0.041)and low neutrophil count(P=0.035)were found to predict for survival negatively.Conclusion Intensive immunosuppressive therapy combined with eltrombopag works very well in elderly patients with SAA.Patients with advanced age(≥70 years),high Charson index(≥5 points)or agranulocytosis(<0.5×109/L)tend to have poor efficacy and survival.