实用医学杂志 ›› 2021, Vol. 37 ›› Issue (9): 1187-1192.doi: 10.3969/j.issn.1006⁃5725.2021.09.019

• 药物与临床 • 上一篇    下一篇

芦可替尼治疗骨髓纤维化阶段性疗效及安全性评估

罗洁, 谭雅娴,黄继贤, 吴婉儿, 刘靓, 周玲玲, 周璇, 阴常欣,刘晓力, 许娜   

  1. 1 南方医科大学南方医院血液科(广州 510515);2 粤北人民医院血液科(广东韶关512025)

  • 出版日期:2021-05-10 发布日期:2021-05-10
  • 通讯作者: 许娜 E⁃mail:sprenaa@163.com
  • 基金资助:

    国家自然科学基金(编号:81700104);广州市科技 计划(编号:201904010488);南方医科大学南方医院院长基金(编 号:2016A002)

Phased efficacy and safety of ruxolitinib in the treatment of myelofibrosis 

LUO Jie,TAN Yaxian,HUANG Jixian,WU Waner,LIU Liang,ZHOU Linlin,ZHOU Xuan,YIN Changxin,LIU Xiaoli,XU Na.    

  1. Department of Hematology,Nanfang Hospital,Southern Medical University,GuangZhou 510515,China 

  • Online:2021-05-10 Published:2021-05-10
  • Contact: XU Na E⁃mail:sprenaa@163.com

摘要:

目的 评价芦可替尼治疗骨髓纤维化(myelofibrosis,MF)患者的疗效及安全性。方法 回顾性分析 2017 6 月至 2019 5 月就诊于南方医院规范服用芦可替尼超过 12 个月的 60 MF 患者;采用 MPN 症状评估表(MPN⁃SAF⁃TSS)评价症状,B 超评估脾脏大小,观察骨髓纤维化及 JAK2V617F 基因突变负 荷变化和不良反应情况,同时探讨芦可替尼对患者免疫功能的影响。结果 服用芦可替尼12个月,51.67% MF患者总症状评分降低50%以上;脾脏中位长厚径分别由基线的(16.65 ± 3.42)、(5.86 ± 1.54)cm,缩短 至(14.93 ± 3.02)、(5.32 ± 1.29)cm,差异有统计学意义(P<0.001);92.86%患者骨髓纤维化稳定或改善, 45例患者合并JAK2V617F等位基因突变,基因突变负荷中位差异无统计学意义(P = 0.600)。血液学不良反 应主要为贫血(68.09%)和血小板减少(40.43%);非血液学不良反应主要观察到感染的发生(23.33%);用药后 1 年外周血 CD3+、CD4+、CD8+ T 细胞及 NK 细胞百分率均较前减少,其中 NK 细胞治疗前后的差异有统计学意义(P = 0.020)。结论 随访1年的结果显示芦可替尼用于治疗骨髓纤维化患者是安全且有效的。

关键词:

芦可替尼, 骨髓纤维化, 疗效, 安全性, 免疫

Abstract: Objective To explore the efficacy and safety of ruxolitinib in the treatment of myelofibrosis (MF). Methods This study enrolled 60 cases with MF,aged over 18 years and diagnosed with PMF,PPV,or PET in Nanfang Hospital from June 2017 and May 2019.All patients received ruxolitinib over 12 months. The symp⁃ toms were evaluated by MPN Symptom Assessment Form⁃Total symptom score(MPN⁃SAF⁃TSS)and B⁃ultrasound was used to assess the size of spleen. The improvement of bone marrow fibrosis,JAK2V617 gene mutation burden and adverse events were observed and the effect of ruxolitinib on lymphocytes was analyzed. Results The total symptom score reduced at least 50% in 51.67% of the patients. The median length and thickness of the spleen were shortened and reduced from(16.65 ± 3.42)cm and(5.86 ± 1.54)cm at baseline to(14.93 ± 3.02)cm and(5.32 ± 1.29)cm respectively(< 0.001). A total of 92.86% of the patients were with stable or improved bone marrow fibrosis;45 patients with JAK2V617F gene mutation and the median mutation burden decreased by 0.90%(= 0.600). The most common hematologic adverse events were anemia(68.09%)and thrombocytopenia(40.43%)and infection(23.33%)was the frequently⁃seen non⁃hematologic adverse event. CD3+,CD4+,CD8+,and NK cells were reduced after the treatment,and the difference in terms of NK cells before and after the treatment was statistically significant(= 0.020). Conclusion Ruxolitinib is safe and effective for the treatment of patients with myelofibrosis.

Key words:

ruxolitinib, myelofibrosis, efficacy, safety, immune