索拉非尼在急性髓系白血病中的应用

张钰, 姚足容, 余国攀. 索拉非尼在急性髓系白血病中的应用[J]. 临床血液学杂志, 2024, 37(5): 306-311. doi: 10.13201/j.issn.1004-2806.2024.05.005
引用本文: 张钰, 姚足容, 余国攀. 索拉非尼在急性髓系白血病中的应用[J]. 临床血液学杂志, 2024, 37(5): 306-311. doi: 10.13201/j.issn.1004-2806.2024.05.005
ZHANG Yu, YAO Zurong, YU Guopan. Therapeutic application of sorafenib in acute myeloid leukemia[J]. J Clin Hematol, 2024, 37(5): 306-311. doi: 10.13201/j.issn.1004-2806.2024.05.005
Citation: ZHANG Yu, YAO Zurong, YU Guopan. Therapeutic application of sorafenib in acute myeloid leukemia[J]. J Clin Hematol, 2024, 37(5): 306-311. doi: 10.13201/j.issn.1004-2806.2024.05.005

索拉非尼在急性髓系白血病中的应用

  • 基金项目:
    国家重点研发计划项目(No: 2021YFC2500300、2021YFC2500302)
详细信息
    作者简介:

    专家简介:张钰,2001年毕业于第一军医大学七年制,内科血液病专业,硕士学位,副主任医师。现任南方医科大学南方医院血液内科急性髓系白血病(AML)亚专科主任。率先建立并实施AML规范化诊疗路径及基于分子遗传学与微小残留病动态危险分层的AML个体化治疗体系,获得国内国际同行认可。目前担任中华医学会血液学分会白血病淋巴瘤学组委员,中国老年医学会血液学分会常务委员,海峡两岸医药卫生交流协会血液学专委会委员,中国抗癌协会肿瘤血液专委会委员,中国抗癌协会CACA指南编委及认证医师,广东省老年保健协会血液病专业委员会副主任委员,广东省预防医学会血液肿瘤专业委员会常务委员,广东省抗癌协会血液肿瘤专业委员会委员,广东省医学会内科学分会委员,广东省医学会血液病学分会白血病学组委员。Blood杂志中文版青年编委。以主要研究者(PI)身份承担AML国际国内及研究者发起的多中心临床研究23项。在Clin Cancer Res、Blood Cancer JJAMA Netw OpenJ Intern MedSignal Transduct Target Ther等杂志发表SCI文章17篇。2019年被评为广州市实力中青年医师,2020年被评为羊城好医生

    通讯作者: 张钰, E-mail: scidzy@163.com
  • 中图分类号: R733.71

Therapeutic application of sorafenib in acute myeloid leukemia

More Information
  • 随着多种靶向药物的问世,急性髓系白血病(acute myeloid leukemia,AML)逐渐进入靶向治疗时代。其中索拉非尼作为一种以靶向FLT3-ITD为主的多激酶抑制剂,在AML治疗中得到广泛应用,主要用于FLT3-ITD+AML的治疗,特别是移植后的维持治疗。另外,鉴于索拉非尼的多激酶抑制特征,也有不少研究尝试将其用于FLT3野生组AML的治疗。本文综述了索拉非尼在AML中的治疗应用及相关研究进展。
  • 加载中
  • [1]

    Döhner H, Wei AH, Appelbaum FR, et al. Diagnosis and management of AML in adults: 2022 recommendations from an international expert panel on behalf of the ELN[J]. Blood, 2022, 140(12): 1345-1377. doi: 10.1182/blood.2022016867

    [2]

    National Comprehensive Cancer Network. Acute Myeloid Leukemia(version 3)[EB/OL]. https://www.nccn.org/professionals/physician_gls/pdf/aml.pdf(2023).

    [3]

    中华医学会血液学分会白血病淋巴瘤学组. 成人急性髓系白血病(非急性早幼粒细胞白血病)中国诊疗指南(2023年版)[J]. 中华血液学杂志, 2023, 44(9): 705-712.

    [4]

    Antar AI, Otrock ZK, Jabbour E, et al. FLT3 inhibitors in acute myeloid leukemia: ten frequently asked questions[J]. Leukemia, 2020, 34: 682-696. doi: 10.1038/s41375-019-0694-3

    [5]

    Zhang W, Konopleva M, Shi YX, et al. Mutant FLT3: a direct target of sorafenib in acute myelogenous leukemia[J]. J Natl Cancer Inst, 2008, 100(3): 184-198. doi: 10.1093/jnci/djm328

    [6]

    Borthakur G, Kantarjian H, Ravandi F, et al. Phase Ⅰ study of sorafenib in patients with refractory or relapsed acute leukemias[J]. Haematologica, 2011, 96(1): 62-68. doi: 10.3324/haematol.2010.030452

    [7]

    Frohling S, Schlenk RF, Breitruck J, et al. Acute myeloid leukemia. Prognostic significance of activating FLT3 mutations in younger adults(16 to 60 years)with acute myeloid leukemia and normal cytogenetics: a study of the AML Study Group[J]. Blood, 2002, 100: 4372-4380. doi: 10.1182/blood-2002-05-1440

    [8]

    Stone RM, Mandrekar SJ, Sanford BL, et al. Midostaurin plus Chemotherapy for Acute Myeloid Leukemia with a FLT3 Mutation[J]. N Engl J Med, 2017, 377(5): 454-464. doi: 10.1056/NEJMoa1614359

    [9]

    Erba HP, Montesinos P, Kim HJ, et al. Quizartinib plus chemotherapy in newly diagnosed patients with FLT3-internal-tandem-duplication-positive acute myeloid leukaemia(QuANTUM-First): a randomised, double-blind, placebo-controlled, phase 3 trial[J]. Lancet, 2023, 401(10388): 1571-1583. doi: 10.1016/S0140-6736(23)00464-6

    [10]

    Perl AE, Martinelli G, Cortes JE, et al. Gilteritinib or Chemotherapy for Relapsed or Refractory FLT3-Mutated AML[J]. N Engl J Med, 2019, 381(18): 1728-1740. doi: 10.1056/NEJMoa1902688

    [11]

    Cortes JE, Khaled S, Martinelli G, et al. Quizartinib versus salvage chemotherapy in relapsed or refractory FLT3-ITD acute myeloid leukaemia(QuANTUM-R): a multicentre, randomised, controlled, open-label, phase 3 trial[J]. Lancet Oncol, 2019, 20(7): 984-997. doi: 10.1016/S1470-2045(19)30150-0

    [12]

    Chen YB, Li S, Lane AA, et al. Phase Ⅰ trial of maintenance sorafenib after allogeneic hematopoietic stem cell transplantation for fms-like tyrosine kinase 3 internal tandem duplication acute myeloid leukemia[J]. Biol Blood Marrow Transplant, 2014, 20(12): 2042-2048. doi: 10.1016/j.bbmt.2014.09.007

    [13]

    Xuan L, Wang Y, Chen J, et al. Sorafenib Therapy Is Associated with Improved Outcomes for FMS-like Tyrosine Kinase 3 Internal Tandem Duplication Acute Myeloid Leukemia Relapsing after Allogeneic Hematopoietic Stem Cell Transplantation[J]. Biol Blood Marrow Transplant, 2019, 25(8): 1674-1681. doi: 10.1016/j.bbmt.2019.04.018

    [14]

    Burchert A, Bug G, Fritz LV, et al. Sorafenib Maintenance After Allogeneic Hematopoietic Stem Cell Transplantation for Acute Myeloid Leukemia With FLT3-Internal Tandem Duplication Mutation(SORMAIN)[J]. J Clin Oncol, 2020, 38(26): 2993-3002. doi: 10.1200/JCO.19.03345

    [15]

    Xuan L, Wang Y, Huang F, et al. Sorafenib maintenance in patients with FLT3-ITD acute myeloid leukaemia undergoing allogeneic haematopoietic stem-cell transplantation: an open-label, multicentre, randomised phase 3 trial[J]. Lancet Oncol, 2020, 21(9): 1201-1212. doi: 10.1016/S1470-2045(20)30455-1

    [16]

    Xuan L, Wang Y, Yang K, et al. Sorafenib maintenance after allogeneic haemopoietic stem-cell transplantation in patients with FLT3-ITD acute myeloid leukaemia: long-term follow-up of an open-label, multicentre, randomised, phase 3 trial[J]. Lancet Haematol, 2023, 10(8): e600-e611. doi: 10.1016/S2352-3026(23)00117-5

    [17]

    Pratz KW, Cho E, Levis MJ, et al. A pharmacodynamic study of sorafenib in patients with relapsed and refractory acute leukemias[J]. Leukemia, 2010, 24(8): 1437-1444. doi: 10.1038/leu.2010.132

    [18]

    Ravandi F, Cortes JE, Jones D, et al. Phase Ⅰ/Ⅱ study of combination therapy with sorafenib, idarubicin, and cytarabine in younger patients with acute myeloid leukemia[J]. J Clin Oncol, 2010, 28: 1856-1862. doi: 10.1200/JCO.2009.25.4888

    [19]

    Sasaki K, Kantarjian HM, Kadia T, et al. Sorafenib plus intensive chemotherapy improves survival in patients with newly diagnosed, FLT3-internal tandem duplication mutation-positive acute myeloid leukemia[J]. Cancer, 2019, 125(21): 3755-3766. doi: 10.1002/cncr.32387

    [20]

    Chen F, Sun J, Yin C, et al. Impact of FLT3-ITD allele ratio and ITD length on therapeutic outcome in cytogenetically normal AML patients without NPM1 mutation[J]. Bone Marrow Transplant, 2020, 55(4): 740-748. doi: 10.1038/s41409-019-0721-z

    [21]

    Xuan L, Wang Y, Chen J, et al. Sorafenib Therapy Is Associated with Improved Outcomes for FMS-like Tyrosine Kinase 3 Internal Tandem Duplication Acute Myeloid Leukemia Relapsing after Allogeneic Hematopoietic Stem Cell Transplantation[J]. Biol Blood Marrow Transplant, 2019, 25(8): 1674-1681. doi: 10.1016/j.bbmt.2019.04.018

    [22]

    Xuan L, Wang Y, Huang F, et al. Effect of sorafenib on the outcomes of patients with FLT3-ITD acute myeloid leukemia undergoing allogeneic hematopoietic stem cell transplantation[J]. Cancer, 2018, 124(9): 1954-1963. doi: 10.1002/cncr.31295

    [23]

    Rollig C, Serve H, Huttmann A, et al. Addition of sorafenib versus placebo to standardtherapy in patients aged 60 years or younger with newly diag-nosed acute myeloid leukaemia(SORAML): a multicentre, phase 2, randomised controlled trial[J]. Lancet Oncol, 2015, 16: 1691-1699. doi: 10.1016/S1470-2045(15)00362-9

    [24]

    Röllig C, Serve H, Noppeney R, et al. Sorafenib or placebo in patients with newly diagnosed acute myeloid leukaemia: long-term follow-up of the randomized controlled SORAML trial[J]. Leukemia, 2021, 35(9): 2517-2525. doi: 10.1038/s41375-021-01148-x

    [25]

    Pollard JA, Alonzo TA, Gerbing R, et al. Sorafenib in Combination With Standard Chemotherapy for Children With High Allelic Ratio FLT3/ITD+Acute Myeloid Leukemia: A Report From the Children's Oncology Group Protocol AAML1031[J]. J Clin Oncol, 2022, 40(18): 2023-2035. doi: 10.1200/JCO.21.01612

    [26]

    Loo S, Roberts AW, Anstee NS, et al. Sorafenib plus intensive chemotherapy in newly diagnosed FLT3-ITD AML: a randomized, placebo-controlled study by the ALLG[J]. Blood, 2023, 142(23): 1960-1971. doi: 10.1182/blood.2023020301

    [27]

    Serve H, Krug U, Wagner R, et al. Sorafenib in combination with intensive chemotherapy in elderly patients with acute myeloid leukemia: results from a randomized, placebo-controlled trial[J]. J Clin Oncol, 2013;31: 3110-3118. doi: 10.1200/JCO.2012.46.4990

    [28]

    Daver N, Perl AE, Maly J, et al. Venetoclax Plus Gilteritinib for FLT3-Mutated Relapsed/Refractory Acute Myeloid Leukemia[J]. J Clin Oncol, 2022, 40(35): 4048-4059. doi: 10.1200/JCO.22.00602

    [29]

    Short NJ, Daver N, Dinardo CD, et al. Azacitidine, Venetoclax, and Gilteritinib in Newly Diagnosed and Relapsed or Refractory FLT3-Mutated AML[J]. J Clin Oncol, 2024, 26: JCO2301911.

    [30]

    Ravandi F, Alattar ML, Grunwald MR, et al. Phase 2 study of azacytidine plus sorafenib in patients with acute myeloid leukemia and FLT-3 internal tandem duplication mutation[J]. Blood, 2013, 121: 4655-4662. doi: 10.1182/blood-2013-01-480228

    [31]

    Zhang C, Lam SSY, Leung GMK, et al. Sorafenib and omacetaxine mepesuccinate as a safe and effective treatment for acute myeloid leukemia carrying internal tandem duplication of Fms-like tyrosine kinase 3[J]. Cancer, 2020, 126(2): 344-353. doi: 10.1002/cncr.32534

    [32]

    张钰, 宣丽, 范志平, 等. 索拉非尼作为挽救性治疗在难治复发性FLT3突变阳性急性髓系白血病中的临床应用研究[J]. 中华血液学杂志, 2016, 37(4): 292-296.

    [33]

    Yu S, Zhang Y, Yu G, et al. Sorafenib plus triplet therapy with venetoclax, azacitidine and homoharringtonine for refractory/relapsed acute myeloid leukemia with FLT3-ITD: A multicenter phase 2 study[J]. J Intern Med, 2024, 295(2): 216-228. doi: 10.1111/joim.13738

    [34]

    Shao R, Zhang Y, He J, et al. Impact of genetic patterns on sorafenib efficacy in patients with FLT3-ITD acute myeloid leukemia undergoing allogeneic hematopoietic stem cell transplantation: a multi-center, cohort study[J]. Signal Transduct Target Ther, 2023, 8(1): 348. doi: 10.1038/s41392-023-01614-1

    [35]

    Mathew NR, Baumgartner F, Braun L, et al. Sorafenib promotes graft-versus-leukemia activity in mice and humans through IL-15 production in FLT3-ITD-mutant leukemia cells[J]. Nat Med, 2018, 24: 282-291. doi: 10.1038/nm.4484

    [36]

    Romero D. Sorafenib prevents AML relapse after allo-HSCT[J]. Nat Rev Clin Oncol, 2020, 17(10): 591. doi: 10.1038/s41571-020-00431-7

    [37]

    Bazarbachi A, Labopin M, Battipaglia G, et al. Sorafenib improves survival of FLT3-mutated acute myeloid leukemia in relapse after allogeneic stem cell transplantation: a report of EBMT acute leukemia Working Party[J]. Haematologica, 2019, 104: e398-e401. doi: 10.3324/haematol.2018.211615

    [38]

    Bazarbachi A, Bug G, Baron F, et al. Clinical practice recommendation on hematopoietic stem cell transplantation for acute myeloid leukemia patients with FLT3-internal tandem duplication: a position statement from the Acute Leukemia Working Party of the European Society for Blood and Marrow Transplantation[J]. Haematologica, 2020, 105(6): 1507-1516. doi: 10.3324/haematol.2019.243410

    [39]

    Halpern AB, Rodríguez-Arbolí E, Othus M, et al. Phase 1/2 study of sorafenib added to cladribine, high-dose cytarabine, G-CSF, and mitoxantrone in untreated AML[J]. Blood Adv, 2023, 7(17): 4950-4961. doi: 10.1182/bloodadvances.2023010392

    [40]

    Yang N, Gu Z, Liu Z, et al. Sorafenib as a Salvage Therapy in FLT3-ITD Negative Relapse/Refractory Acute Myeloid Leukemia[J]. Anticancer Agents Med Chem, 2018, 18(10): 1489-1494.

    [41]

    Chen X, Huang J, Xu N, at al. A phase 2 study of sorafenib combined with conventional therapies in refractory central nervous system leukemia[J]. Cancer, 2022, 128(11): 2138-2147. doi: 10.1002/cncr.34182

    [42]

    Liu S, Wang Y. Diagnosis and management of adult central nervous system leukemia[J]. Blood Sci, 2023, 5(3): 141-149. doi: 10.1097/BS9.0000000000000162

    [43]

    Massard C, Zonierek J, Gross-Goupil M, et al. Incidence of brain metastases in renal cell carcinoma treated with sorafenib[J]. Ann Oncol, 2010, 21: 1027-1031. doi: 10.1093/annonc/mdp411

    [44]

    Sprinzl MF, Reisinger F, Puschnik A, et al. Sorafenib perpetuates cellular anticancer effector functions by modulating the crosstalk between macrophages and natural killer cells[J]. Hepatology, 2013, 57: 2358-2368. doi: 10.1002/hep.26328

    [45]

    Wang YY, Zhou GB, Yin T, et al. AML1-ETO and C-KIT mutation/overexpression in t(8;21) leukemia: implication in stepwise leukemogenesis and response to Gleevec[J]. Proc Natl Acad Sci U S A, 2005, 102(4): 1104-1109. doi: 10.1073/pnas.0408831102

    [46]

    Joshi SK, Nechiporuk T, Bottomly D, et al. The AML microenvironment catalyzes a stepwise evolution to gilteritinib resistance[J]. Cancer Cell, 2021, 39(7): 999-1014. doi: 10.1016/j.ccell.2021.06.003

    [47]

    Yang T, Hu M, Qi W, et al. Discovery of Potent and Orally Effective Dual Janus Kinase 2/FLT3 Inhibitors for the Treatment of Acute Myelogenous Leukemia and Myeloproliferative Neoplasms[J]. J Med Chem, 2019, 62(22): 10305-10320. doi: 10.1021/acs.jmedchem.9b01348

    [48]

    Xu D, Chen Y, Yang Y, et al. Autophagy activation mediates resistance to FLT3 inhibitors in acute myeloid leukemia with FLT3-ITD mutation[J]. J Transl Med, 2022, 20(1): 300. doi: 10.1186/s12967-022-03498-1

    [49]

    Zhang W, Yu G, Zhang H, et al. Concomitant targeting of FLT3 and BTK overcomes FLT3 inhibitor resistance in acute myeloid leukemia through the inhibition of autophagy[J]. Haematologica, 2023, 108(6): 1500-1514.

    [50]

    Xu X, Ma W, Qiu G, et al. Venetoclax Overcomes Sorafenib Resistance in Acute Myeloid Leukemia by Targeting BCL2[J]. Biology(Basel), 2023, 12(10): 1337.

    [51]

    张钰, 邵若洋, 刘启发. FLT3突变急性髓系白血病的全程管理[J]. 临床血液学杂志, 2023, 36(5): 303-308. doi: 10.13201/j.issn.1004-2806.2023.05.002

  • 加载中
计量
  • 文章访问数:  529
  • PDF下载数:  29
  • 施引文献:  0
出版历程
收稿日期:  2024-03-20
刊出日期:  2024-05-01

目录