芦可替尼联合TCP方案治疗骨髓纤维化患者的真实世界研究

肖超, 宋陆茜, 许峰, 等. 芦可替尼联合TCP方案治疗骨髓纤维化患者的真实世界研究[J]. 临床血液学杂志, 2020, 33(11): 753-758. doi: 10.13201/j.issn.1004-2806.2020.11.005
引用本文: 肖超, 宋陆茜, 许峰, 等. 芦可替尼联合TCP方案治疗骨髓纤维化患者的真实世界研究[J]. 临床血液学杂志, 2020, 33(11): 753-758. doi: 10.13201/j.issn.1004-2806.2020.11.005
XIAO Chao, SONG Lu-qian, XU Feng, et al. Ruxolitinib combined with TCP therapy for treatment of myelofibrosis:A real world study[J]. J Clin Hematol, 2020, 33(11): 753-758. doi: 10.13201/j.issn.1004-2806.2020.11.005
Citation: XIAO Chao, SONG Lu-qian, XU Feng, et al. Ruxolitinib combined with TCP therapy for treatment of myelofibrosis:A real world study[J]. J Clin Hematol, 2020, 33(11): 753-758. doi: 10.13201/j.issn.1004-2806.2020.11.005

芦可替尼联合TCP方案治疗骨髓纤维化患者的真实世界研究

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    通讯作者: 常春康,E-mail:changchunkang@sjtu.edu.cn
  • 中图分类号: R551.3

Ruxolitinib combined with TCP therapy for treatment of myelofibrosis:A real world study

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  • 目的:为了减轻芦可替尼对骨髓纤维化(MF)患者的血液学毒副反应,我们开展了一项小剂量芦可替尼联合TCP方案的探索性治疗,并呈现该研究方案的真实数据。方法:于2019-02-01—2020-03-31对就诊于我院血液科的19例MF患者,给予小剂量芦可替尼联合TCP方案治疗(芦可替尼5 mg Bid;沙利度胺50 mg QN,骨化三醇25 mg Bid,泼尼松10 mg QD)。治疗过程中每个月复查血常规和肝肾功能,每3个月复查肝脾超声和MF症状评估表(MPN-SAF-TSS)。结果:共19例患者纳入研究,男10例,女9例;中位年龄63(31~82)岁,<60岁和60~74岁患者各7例(各36.84%),≥75岁5例(26.32%);有1例高白细胞(WBC≥25×109/L),5例贫血(Hb<100 g/L),3例血小板减少(PLT<100×109/L);PMF患者13例(68.42%),post-ET MF和post-PV MF患者各3例(各15.79%)。DIPSS预后分组:中危-1组9例(47.37%),中危-2组10例(52.63%);MIPSS70积分:中危组13例(68.42%),高危组6例(31.58%);染色体核型:极高危核型1例(5.26%),有利核型16例(84.21%),不良核型2例(10.53%);二代基因检测:JAK2V617F突变阳性14例(73.68%),CARL突变阳性2例(10.53%),JAK2、CALR、MPL三阴性3例(15.79%),5例(26.32%)患者有1个高危突变,1例(5.26%)患者有2个高危突变。治疗前MPN-SAF-TSS评分33(11~60)分;15例(78.95%)患者B超证实有脾肿大。中位治疗时间6(3~13)个月。19例患者经治疗后,高白细胞、贫血、血小板减少患者各减少1例。19例患者经治疗后最低MPN-SAF-TSS评分17(8~35)分,评分下降幅度13(3~45)分;有4例(21.05%)患者症状评分降低≥50%(最大75%)。15例脾肿大患者治疗后13例(86.67%)有不同程度的脾脏缩小,缩小比例38.67%(4.36%~53.26%),缩小比例≥35%者有8例(53.33%)。接受该治疗方案的19例患者目前均未停药,有1例在用药1个月时出现血红蛋白和血小板计数下降(治疗2个月后恢复正常),另1例用药2个月时贫血加重(治疗3个月后血红蛋白回升),其余17例患者均未见血液学不良反应;未出现Ⅲ/Ⅳ级非血液学不良反应;Ⅰ/Ⅱ级非血液学不良反应包括便秘1例、腹胀2例,经胃肠道调节治疗后好转。没有患者因不良反应中断治疗或者用药剂量调整。结论:小剂量芦可替尼联合TCP方案的治疗方案,对于MF患者安全有效,不仅能减少常规剂量芦可替尼引发的贫血和血小板减少的发生率,而且对于老年MF患者,甚至是>75岁的超高龄患者,也可以作为一种治疗选择。然而,该方案也存在脾脏缩小不敏感的患者,有待于调整芦可替尼剂量。
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收稿日期:  2020-08-04

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