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芦可替尼治疗骨髓纤维化的疗效与安全性
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王菀菀 ,叶 隽 ,程 海 ,姚 伟 ,刘桂玲 (1.蚌埠市第三人民医院血液内科,安徽 蚌埠 233000;2.徐州
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医科大学附属医院血液内科,江苏 徐州 221000)
中图分类号 R559;R969.3 文献标志码 A 文章编号 1001-0408(2025)14-1781-05
DOI 10.6039/j.issn.1001-0408.2025.14.16
摘 要 目的 探究芦可替尼治疗骨髓纤维化(MF)的疗效和安全性。方法 回顾性收集2018年9月至2024年4月于蚌埠市第三
人民医院就诊且规范使用芦可替尼超过6个月的42例MF患者资料。分析治疗前后患者的临床症状评分、脾脏缩小情况和MF分
级情况,并记录关联性评价结果为“肯定”“很可能”“可能有关”的不良反应的发生情况,随访患者的生存情况。结果 治疗6个月
后,患者的各单项临床症状评分及总分均较治疗前显著降低(P<0.05),脾脏长径、厚径均较治疗前显著缩短(P<0.05)。5例患者
的MF分级较基线下降1级,1例由MF-2级下降至MF-0级,14例MF分级未发生变化。不良反应以贫血(26例次)、血小板减少(14
例次)、感染(11例次)、胃肠道不适(9例次)为主。39例患者存活,生存率为92.86%。结论 芦可替尼能有效改善MF患者的临床
症状,缩小脾脏,稳定甚至改善MF分级,有望为MF患者带来长期生存获益;不良反应主要表现为贫血、血小板减少、感染及胃肠
道不适。
关键词 芦可替尼;骨髓纤维化;疗效;安全性
Efficacy and safety of ruxolitinib in the treatment of myelofibrosis
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WANG Wanwan ,YE Jun ,CHENG Hai ,YAO Wei ,LIU Guiling(1. Dept. of Hematology, the Third People’s
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Hospital of Bengbu, Anhui Bengbu 233000, China;2. Dept. of Hematology, the Affiliated Hospital of Xuzhou
Medical University, Jiangsu Xuzhou 221000, China)
ABSTRACT OBJECTIVE To explore the efficacy and safety of ruxolitinib in the treatment of myelofibrosis (MF). METHODS
A retrospective collection of data was conducted on 42 MF patients who were treated with ruxolitinib in a standardized manner for
more than 6 months in the Third People’s Hospital of Bengbu from September 2018 to April 2024. The clinical symptom scores,
spleen size reduction, and MF grading of the patients before and after treatment were analyzed. Additionally, the occurrence of
adverse reactions with a causality assessment result of “definite”“probable” or “possible” was recorded. The patients’ survival
status was followed up. RESULTS After 6 months of treatment, both clinical symptom scores and the total score were significantly
decreased than before treatment (P<0.05). The length and thickness of the spleen were significantly shorter than before treatment
(P<0.05). MF classification in 5 patients decreased by 1 level compared with baseline, 1 case was level 2 and dropped to level 0,
14 patients remained stable. The main adverse reactions were anemia (26 cases), thrombocytopenia (14 cases), infection (11
cases), and gastrointestinal discomfort (9 cases). Thirty-nine patients survived, with a survival rate of 92.86%. CONCLUSIONS
Ruxolitinib can effectively improve the clinical symptoms of patients with MF, shrink the spleen, stabilize and even improve MF
grading, and holds promise for bringing long-term survival benefits to MF patients. Adverse reactions are mainly anemia,
thrombocytopenia, infection and gastrointestinal discomfort.
KEYWORDS ruxolitinib; myelofibrosis; efficacy; safety
经 典 型 骨 髓 增 殖 性 肿 瘤(myeloproliferative neo‐ 髓纤维化(myelofibrosis,MF),即继发性 MF ;同时,继
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plasms,MPNs)是一组由造血干细胞克隆性增殖引起的 发于ET和PV的MF被分别称为ET后骨髓纤维化(post-
疾病,包括原发性骨髓纤维化(primary myelofibrosis, essential thrombocythemia myelofibrosis,PET-MF)和 PV
PMF)、原发性血小板增多症(essential thrombocythemia, 后 骨 髓 纤 维 化(post-polycythemia vera myelofibrosis,
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ET)和真性红细胞增多症(polycythemia vera,PV) 。随 PPV-MF)。MF 的临床表现包括血细胞进行性下降、肝
着疾病的进展,部分ET和PV患者亦可在后期进展为骨
脾肿大和全身体质性症状(如发热、乏力、盗汗、体重减
Δ 基金项目 国家自然科学基金项目(No.82070127);蚌埠医学院 轻等),且转化为白血病的风险极高,严重影响患者的生
科技项目(自然科学类)(No.2021byzd228) 存质量和生存期 。
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*第一作者 主治医师,硕士。研究方向:良恶性血液系统疾病的
有研究报道,Janus 激酶/信号转导及转录活化因子
诊断及治疗。E-mail:957620015@qq.com
(Janus kinase/signal transducer and activator of transcrip‐
# 通信作者 主任医师,硕士生导师。研究方向:良恶性血液系统
疾病的诊断及治疗。E-mail:Liubbsy@163.com tion,JAK/STAT)信号通路与MPNs发病及纤维化进展紧
中国药房 2025年第36卷第14期 China Pharmacy 2025 Vol. 36 No. 14 · 1781 ·