国产尼洛替尼治疗新诊断的慢性髓系白血病慢性期患者疗效及安全性分析  

Efficacy and safety of nilotinib produced in China in the treatment of patients with newly diagnosed chronic myeloid leukemia in chronic phase

作　　者：梁艳[1] 韩丹壘[2] 程芳 成延娟 王海燕[2] 蔡惠丽 唐元艳[1] 黄知平[1] 黎纬明[5] LIANG Yan;HAN Danlei;CHENG Fang;CHENG Yanjuan;WANG Haiyan;CAI Huili;TANG Yuanyan;HUANG Zhiping;LI Weiming(Department of Hematology,Jingzhou Hospital,Affiliated to Yangtze University,Jingzhou,Hubei,434020,China;The First College of Clinical Medical Science,China Three Gorges University&Yichang Central People's Hospital;Department of Pharmacy,Union Hospital,Tongji Medical College,Huazhong University of Science and Technology;Hubei Province Clinical ResearchCenter for Precision Medicine for Critical Illness;Department of Hematology,Union Hospital,Tongji Medical College,Huazhong University of Science and Technology)

机构地区：[1]长江大学附属荆州医院血液科,湖北荆州434020 [2]宜昌市中心人民医院血液科 [3]华中科技大学同济医学院附属协和医院药剂科 [4]湖北省重大疾病精准用药医学研究中心 [5]华中科技大学同济医学院附属协和医院血液科

出　　处：《临床血液学杂志》2025年第3期198-204,共7页Journal of Clinical Hematology

摘　　要：目的:探讨国产尼洛替尼对新诊断的慢性髓系白血病(chronic myeloid leukemia,CML)慢性期(chronic phase,CP)患者的临床疗效及安全性。方法:收集2023年9月1日—2024年10月1日于华中科技大学附属协和医院、长江大学附属荆州医院、宜昌市中心医院就诊的CML-CP患者,未经过除羟基脲以外其他任何抗CML治疗的CML-CP患者,给予国产尼洛替尼300mg/次,口服,2次/d,评估治疗3、6、12个月时患者的血液学、细胞遗传学和分子学反应及安全性。结果:治疗≥3个月的患者23例,治疗≥6个月的患者17例,治疗≥12个月的患者9例。治疗3个月时,23例患者中21例获得完全血液学反应(complete hematologic response,CHR);19例患者获得完全遗传学反应(complete cytogenetic response,CCyR);23例均进行了分子检测(实时定量PCR法检测,BCR-ABL转录水平以IS表示),BCR-ABLIS≤10%的患者21例,其中BCR-ABLIS≤0.1%7例,BCRABLIS≤0.01%2例。治疗6个月时,17例患者中16例获得CHR;17例患者获得CCyR;17例均进行了分子检测,BCR-ABLIS≤1%的患者14例,其中14例BCR-ABLIS≤0.1%,4例BCR-ABLIS≤0.01%。治疗12个月时,9例患者中全部获得CHR和CCyR;6例BCR-ABLIS≤0.1%,其中5例BCR-ABLIS≤0.01%。Ⅲ~Ⅳ级白细胞减少、中性粒细胞减少、贫血、血小板减少的发生率分别为17.4%、26.1%、17.4%、26.1%,有2例患者发生Ⅳ级血小板减少持续不能恢复而停药。主要的非血液学不良反应依次为皮肤瘙痒(21.7%)、皮疹(17.4%)、肌肉酸痛(13.0%)、恶心(13.0%)、高胆红素血症(13.0%)、腹胀(8.7%),均为Ⅰ~Ⅱ级,无患者发生Ⅲ~Ⅳ级非血液学不良反应。无药物毒性相关性死亡。结论:国产尼洛替尼治疗新诊断的CML-CP能够使患者早期、快速获得深层分子学反应和细胞遗传学反应,且安全性良好。Objective:To explore the clinical efficacy and safety of Nilotinib produced in China in patients with newly diagnosed chronic myeloid leukemia in chronic phase(CML-CP).Methods:We collected data from CML-CP patients who visited Huazhong University of Science and Technology Union Hospital,Yangtze University Affiliated Jingzhou Hospital,and Yichang Central Hospital from September 1,2023,to October 1,2024.Patients included in the study had not received any anti-CML treatment other than hydroxyurea.They were treated with Nilotinib produced in China at a dose of 300mg per dose,orally,twice daily.The hematologic,cytogenetic and molecular responses were assessed at 3,6,and 12months of treatment,and adverse effects of the drug were eval uated.Results:23patients were treated with Nilotinib produced in China for≥3months,of which 17patients were treated for≥6months and 9patients were treated for≥12months.At 3rd month of treatment,21out of 23 patients achieved complete hematologic response(CHR);19achieved complete cytogenetic response(CCyR);Among 23patients who underwent molecular testing,21patients had BCR-ABLIS≤10%,including 7patients with BCR-ABLIS≤0.1%,and 2patients with BCR-ABLIS≤0.01%.At 6th month of treatment,16out of 17patients achieved CHR;all 17achieved CCyR;Among 17patients who underwent molecular testing,14patients had BCRABLIS≤1%,including 14patients with BCR-ABLIS≤0.1%and 4patients with BCR-ABLIS≤0.01%.At 12nd month of treatment,all 9patients achieved CHR and CCyR;6patients had BCR-ABLIS≤0.1%,with 5patients having BCR-ABLIS≤0.01%.The incidence rates of gradeⅢ-Ⅳleukopenia,neutropenia,anemia,and thrombocytopenia were 17.4%,26.1%,17.4%,and 26.1%,respectively,with 2patients experiencing gradeⅣthrombocytopenia that persisted and led to discontinuation of treatment.The main non-hematological adverse reactions were skin itching(21.7%),rash(17.4%),muscle pain(13.0%),nausea(13.0%),hyperbilirubinemia(13.0%),and abdominal distension(8.7%),all of which were gradeⅠ-Ⅱ,with no patients experienc

关 键 词：国产尼洛替尼 慢性髓系白血病 分子学反应 

分 类 号：R733.7[医药卫生—肿瘤]