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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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                          1
          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
                                                                                                   [2]
          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,
                                                    [1]
          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 。
                                                                           [1]
             *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 ·
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