摘要
目的 观察和评价硼替佐米治疗复发难治纯红细胞再生障碍性贫血(PRCA)的疗效和安全性。方法 回顾性分析6例复发难治PRCA患者的临床特征。患者于2019年1月至2021年6月在本院接受硼替佐米(1.3 mg·m^(-2),皮下注射,每周1次)单药治疗或硼替佐米(给药方式同前)联合利妥昔单抗(500 mg,硼替佐米第1剂用药前1日静脉输注)治疗,均4周为一个疗程。根据患者血红蛋白水平变化和输血量来评估治疗后3个月的疗效,观察不良反应及至随访期末患者转归情况。结果 6例患者中4例为特发性PRCA,1例为细小病毒B19继发PRCA,1例为骨髓增生异常综合征(MDS)表现为PRCA。PRCA起病到硼替佐米方案治疗的中位时间为2.5年(3个月~6年),患者在接受硼替佐米治疗之前已采用2~7种药物治疗。6例患者共完成9个疗程硼替佐米治疗,其中单药治疗5个疗程,联合治疗4个疗程。6例患者中1例特发性PRCA患者部分缓解且维持1年,其余5例患者中3例疾病稳定,2例无效。所有患者均耐受良好,无3级及以上不良反应发生。至随访期末,1例死于肺部感染,2例输血依赖,1例改用西罗莫司治疗有效,1例修正诊断MDS患者采用来那度胺治疗,1例B19继发患者停免疫抑制剂后血红蛋白恢复正常。结论 复发难治PRCA可以尝试硼替佐米单药或联合利妥昔单抗治疗,患者耐受性好,但有效率较低。
AIM To observe and evaluate the efficacy and safety of bortezomib in the treatment of recurrent refractory pure red cell aplasia(PRCA). METHODS The clinical features of six patients with relapsed and refractory PRCA were retrospectively analyzed. The patients were treated with bortezomib(1.3 mg·m^(-2), sc, once a week) or bortezomib(the same as before) combined with rituximab(500 mg,intravenously infused one day before the first dose of bortezomib) in our hospital from January 2019 to June 2021, and all of which were treated for four weeks as a course of treatment. The curative effect of three months after treatment was evaluated according to the change of hemoglobin level and blood transfusion, and the adverse reactions and the prognosis of patients were observed at the end of the follow-up period.RESULTS Among the 6 patients, 4 cases were idiopathic PRCA, one case was PRCA secondary to parvovirus B19, and one case was finally diagnosed as myelodysplastic syndrome(MDS). The median time interval from the onset of PRCA to bortezomib treatment was 2.5 years(3 months to 6 years) and the patients had been treated with 2-7 drugs before receiving bortezomib. Six patients received 9 courses of bortezomib treatment, including 5 courses of bortezomib and 4 courses of bortezomib combined with rituximab. Among the 6 patients, one patient with idiopathic PRCA was partial remission and responded for one year, while the remaining 5 patients were stable in 3 cases and ineffective in 2 cases. All patients were well tolerated and no grade 3 or above adverse reactions occurred. By the end of follow-up, one patient died of pulmonary infection, 2 patients were dependent on blood transfusion. One patient was effectively treated with sirolimus, one patient with modified diagnosis of MDS was treated with lenalidomide and one case of B19 secondary patient recovered to normal after stopping immunosuppressive agents. CONCLUSION For relapsed and refractory PRCA, bortezomib alone or combined with rituximab can be tried, and the patients with good tolerated, but the efficiency is low.
作者
陈苗
陈泽松
韩冰
CHEN Miao;CHEN Ze-song;HAN Bing(Department of Hematology,Peking Union Medical College Hospital,Peking Union Medical College&Chinese Academy of Medical Sciences,BEIJING 100730,China)
出处
《中国新药与临床杂志》
CAS
CSCD
北大核心
2022年第11期675-679,共5页
Chinese Journal of New Drugs and Clinical Remedies
基金
国家自然科学基金(81970106)
中国医学科学院医学与健康科技创新工程重大协同创新项目中国罕见病的精准诊疗研究(2021-I2M-1-003)。
