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重型再生障碍性贫血的治疗与造血干细胞移植

李燕娟, 张连生, 李莉娟. 重型再生障碍性贫血的治疗与造血干细胞移植[J]. 器官移植, 2023, 14(3): 442-448. doi: 10.3969/j.issn.1674-7445.2023.03.017
引用本文: 李燕娟, 张连生, 李莉娟. 重型再生障碍性贫血的治疗与造血干细胞移植[J]. 器官移植, 2023, 14(3): 442-448. doi: 10.3969/j.issn.1674-7445.2023.03.017
Li Yanjuan, Zhang Liansheng, Li Lijuan. Treatment of severe aplastic anemia and hematopoietic stem cell transplantation[J]. ORGAN TRANSPLANTATION, 2023, 14(3): 442-448. doi: 10.3969/j.issn.1674-7445.2023.03.017
Citation: Li Yanjuan, Zhang Liansheng, Li Lijuan. Treatment of severe aplastic anemia and hematopoietic stem cell transplantation[J]. ORGAN TRANSPLANTATION, 2023, 14(3): 442-448. doi: 10.3969/j.issn.1674-7445.2023.03.017

重型再生障碍性贫血的治疗与造血干细胞移植

doi: 10.3969/j.issn.1674-7445.2023.03.017
基金项目: 

国家血液系统疾病临床医学研究中心委托课题 2021WWA01

甘肃省科技计划项目 21JR11RA104

兰州市科技计划项目 2020-ZD-99

兰州大学第二医院萃英科技创新项目 CY2017-ZD04

兰州大学第二医院萃英科技创新项目 CY2019-MS14

详细信息
    作者简介:
    通讯作者:

    张连生(ORCID:0000-0001-6444-6421),教授,主任医师,研究方向为血液疾病及各类肿瘤的免疫发病机制及精准干预、细胞工程及细胞免疫治疗研究,Email:zhanglsh@lzu.edu.cn

    李莉娟(ORCID:0000-0001-6456-0152),教授,主任医师,研究方向为血液系统疾病的免疫发病机制、精准干预、造血干细胞移植及pDC疫苗技术的研发,Email:doctorjuan@sina.com

  • 中图分类号: R617, R551, R556.5

Treatment of severe aplastic anemia and hematopoietic stem cell transplantation

More Information
  • 摘要: 重型再生障碍性贫血(SAA)是由多种病因导致的重度骨髓造血功能衰竭综合征,临床以严重的贫血、感染、出血为主要特征。SAA发病机制复杂,至今尚未完全明了。SAA起病急,病情重,病情进展快,目前随着对SAA的深入研究以及诊疗水平的提高,对于其治疗策略也发生了改变。从经典的免疫抑制治疗,即主要以抗胸腺细胞球蛋白和环孢素为基础的治疗方案,到血小板生成素受体激动剂的应用及以异基因造血干细胞移植等为基础的联合治疗方案,均不同程度促进SAA患者的造血功能重建,极大改善其生存及预后,成为当下SAA治疗的研究热点。本文结合国内外文献对SAA治疗的新进展进行综述。

     

  • [1] LIU Q, DONG H, LI Y, et al. Apolipoprotein-A is a potential prognostic biomarker for severe aplastic anemia patients treated with ATG-based immunosuppressive therapy: a single-center retrospective study[J]. Lipids Health Dis, 2022, 21(1): 93. DOI: 10.1186/s12944-022-01703-0.
    [2] ZHU XF, HE HL, WANG SQ, et al. Current treatment patterns of aplastic anemia in China: a prospective cohort registry study[J]. Acta Haematol, 2019, 142(3): 162-170. DOI: 10.1159/000499065.
    [3] MOORE CA, KRISHNAN K. Aplastic anemia[M]. Treasure Island (FL): StatPearls Publishing, 2022.
    [4] 刘春燕, 邵宗鸿. 再生障碍性贫血发病机制研究进展[J]. 中国实用内科杂志, 2016, 36(5): 345-349. DOI: 10.7504/nk2016040101.

    LIU CY, SHAO ZH. Advances in studies on the pathogenesis of aplastic anemia[J]. Chin J Pract Intern Med, 2016, 36(5): 345-349. DOI: 10.7504/nk2016040101.
    [5] 杨洁茹, 王化泉, 邵宗鸿. 再生障碍性贫血发病机制的研究进展[J]. 中华血液学杂志, 2019, 40(9): 796-800. DOI: 10.3760/cma.j.issn.0253-2727.2019.09.022.

    YANG JR, WANG HQ, SHAO ZH. Advances in the pathogenesis of aplastic anaemia[J]. Chin J Hematol, 2019, 40(9): 796-800. DOI: 10.3760/cma.j.issn.0253-2727.2019.09.022.
    [6] 梁俊娟. 免疫抑制疗法治疗再生障碍性贫血研究进展[J]. 中国小儿血液与肿瘤杂志, 2013, 18(1): 39-42. DOI: 10.3969/j.issn.1673-5323.2013.01.011.

    LIANG JJ. Advances in immunosuppressive therapy for aplastic anemia[J]. J Chin Pediatr Blood Cancer, 2013, 18(1): 39-42. DOI: 10.3969/j.issn.1673-5323.2013.01.011.
    [7] IFTIKHAR R, CHAUDHRY QUN, ANWER F, et al. Allogeneic hematopoietic stem cell transplantation in aplastic anemia: current indications and transplant strategies[J]. Blood Rev, 2021, 47: 100772. DOI: 10.1016/j.blre.2020.100772.
    [8] YANG N, CHEN J, ZHANG H, et al. Horse versus rabbit antithymocyte globulin in immunosuppressive therapy of treatment-naïve aplastic anemia: a systematic review and meta-analysis[J]. Ann Hematol, 2017, 96(12): 2031-2043. DOI: 10.1007/s00277-017-3136-1.
    [9] KANDA Y, MORI T, NARITA A, et al. Efficacy and safety of equine anti-thymocyte immunoglobulin (eATG) in three Japanese patients with moderate to very severe aplastic anemia: a case series[J]. Int J Hematol, 2023, 117(1): 37-43. DOI: 10.1007/s12185-022-03496-5.
    [10] HU J, ZHANG L, ZHAO X, et al. First-line immunosuppressive therapy with rATG and CsA for severe aplastic anemia: 15 years' experience[J]. Ann Hematol, 2022, 101(11): 2405-2412. DOI: 10.1007/s00277-022-04952-2.
    [11] NARITA A, MURAMATSU H, ICHIKAWA D, et al. Relationship between plasma rabbit anti-thymocyte globulin concentration and immunosuppressive therapy response in patients with severe aplastic anemia[J]. Eur J Haematol, 2021, 107(2): 255-264. DOI: 10.1111/ejh.13644.
    [12] PATEL BA, TOWNSLEY DM, SCHEINBERG P. Immunosuppressive therapy in severe aplastic anemia[J]. Semin Hematol, 2022, 59(1): 21-29. DOI: 10.1053/j.seminhematol.2022.01.002.
    [13] LI R, ZHOU J, LIU Z, et al. Predicting response of severe aplastic anemia to rabbit-antithymocyte immunoglobulin based immunosuppressive therapy combined with eltrombopag[J]. Front Immunol, 2022, 13: 884312. DOI: 10.3389/fimmu.2022.884312.
    [14] PEFFAULT DE LATOUR R, KULASEKARARAJ A, IACOBELLI S, et al. Eltrombopag added to immunosuppression in severe aplastic anemia[J]. N Engl J Med, 2022, 386(1): 11-23. DOI: 10.1056/NEJMoa2109965.
    [15] GORONKOVA O, NOVICHKOVA G, SALIMOVA T, et al. Efficacy of combined immunosuppression with or without eltrombopag in children with newly diagnosed aplastic anemia[J]. Blood Adv, 2023 7(6): 953-962. DOI: 10.1182/bloodadvances.2021006716.
    [16] YU W, WANG Q, GE M, et al. Natural killer cells in peripheral blood at diagnosis predict response to immunosuppressive therapy in severe aplastic anemia[J]. Clin Exp Med, 2022, DOI: 10.1007/s10238-022-00909-x[Epubaheadofprint].
    [17] DREXLER B, PASSWEG J. Current evidence and the emerging role of eltrombopag in severe aplastic anemia[J]. Ther Adv Hematol, 2021, 12: 2040620721998126. DOI: 10.1177/2040620721998126.
    [18] 付蓉. 再生障碍性贫血诊断与治疗中国专家共识(2017年版)[J]. 中华血液学杂志, 2017, 38(1): 1-5. DOI: 10.3760/cma.j.issn.0253-2727.2017.01.001.

    FU R. Chinese expert consensus on the diagnosis and treatment of aplastic anemia (2017)[J]. Chin J Hematol, 2017, 38(1): 1-5. DOI: 10.3760/cma.j.issn.0253-2727.2017.01.001.
    [19] DING S, LIANG X, ZHANG T, et al. The effectiveness of rapamycin combined with eltrombopag in murine models of immune-mediated bone marrow failure[J]. J Immunol Res, 2020: 1798795. DOI: 10.1155/2020/1798795.
    [20] FÜREDER W, CERNY-REITERER S, SPERR WR, et al. Evaluation of efficacy of alemtuzumab in 5 patients with aplastic anemia and/or myelodysplastic neoplasm[J]. Wien Klin Wochenschr, 2017, 129(11/12): 404-410. DOI: 10.1007/s00508-016-1091-9.
    [21] ZHU C, LIAN Y, WANG C, et al. Single-cell transcriptomics dissects hematopoietic cell destruction and T-cell engagement in aplastic anemia[J]. Blood, 2021, 138(1): 23-33. DOI: 10.1182/blood.2020008966.
    [22] VAN ANTWERP E, KOENIG ZA, MCCARTHY R. Modern medical miracle: matched unrelated donor hematopoietic stem cell transplant after aplastic anemia[J]. Cureus, 2021, 13(2): e13050. DOI: 10.7759/cureus.13050.
    [23] 闫宇辰, 李振宇. 重型再生障碍性贫血的临床诊治进展[J]. 医学研究杂志, 2022, 51(4): 18-20. DOI: 10.11969/j.issn.1673-548X.2022.04.005.

    YAN YC, LI ZY. Progress in clinical diagnosis and treatment of severe aplastic anemia[J]. J Med Res, 2022, 51(4): 18-20. DOI: 10.11969/j.issn.1673-548X.2022.04.005.
    [24] 王书春, 邹尧, 刘晓明, 等. 减低剂量环磷酰胺联合环孢菌素A治疗重型再生障碍性贫血患儿的疗效分析[J]. 中国实验血液学杂志, 2020, 28(1): 225-229. DOI: 10.19746/j.cnki.issn1009-2137.2020.01.038.

    WANG SC, ZOU Y, LIU XM, et al. The outcomes of severe aplastic anemia children treated with reduced dose of cyclophosphamide combined with cyclosporine A[J]. J Exp Hematol, 2020, 28(1): 225-229. DOI: 10.19746/j.cnki.issn1009-2137.2020.01.038.
    [25] 何平, 王畅, 谭业辉. 不同类型供者造血干细胞移植治疗儿童再生障碍性贫血的进展[J]. 中国免疫学杂志, 2017, 33(3): 477-480. DOI: 10.3969/j.issn.1000-484X.2017.03.035.

    HE P, WANG C, TAN YH. Progress of different types of donor hematopoietic stem cell transplantation in the treatment of children with aplastic anemia[J]. Chin J Immunol, 2017, 33(3): 477-480. DOI: 10.3969/j.issn.1000-484X.2017.03.035.
    [26] YOSHIDA N, KOBAYASHI R, YABE H, et al. First-line treatment for severe aplastic anemia in children: bone marrow transplantation from a matched family donor versus immunosuppressive therapy[J]. Haematologica, 2014, 99(12): 1784-1791. DOI: 10.3324/haematol.2014.109355.
    [27] LEI M, ZHANG Y, JIAO W, et al. Comparison of haploidentical hematopoietic stem cell transplant with or without unrelated cord blood infusion in severe aplastic anemia: outcomes of a multicenter study[J]. Front Immunol, 2022, 13: 912917. DOI: 10.3389/fimmu.2022.912917.
    [28] 王丽, 吴亚妹, 曹永彬, 等. 单倍体相合造血干细胞移植治疗重型再生障碍性贫血进展[J]. 解放军医学院学报, 2017, 38(3): 274-276. DOI: 10.3969/j.issn.2095-5227.2017.03.024.

    WANG L, WU YM, CAO YB, et al. Advances in haploidentical hematopoietic stem cell transplantation for severe aplastic anemia[J]. Acad J Chin Pla Med Sch, 2017, 38(3): 274-276. DOI: 10.3969/j.issn.2095-5227.2017.03.024.
    [29] 季艳萍, 孙自敏. 重型再生障碍性贫血治疗进展[J]. 器官移植, 2020, 11(2): 293-297, 310. DOI: 10.3969/j.issn.1674-7445.2020.02.018.

    JI YP, SUN ZM. Treatment progress of severe aplastic anemia[J]. Organ Transplant, 2020, 11(2): 293-297, 310. DOI: 10.3969/j.issn.1674-7445.2020.02.018.
    [30] DEZERN AE, ZAHURAK ML, SYMONS HJ, et al. Haploidentical BMT for severe aplastic anemia with intensive GVHD prophylaxis including posttransplant cyclophosphamide[J]. Blood Adv, 2020, 4(8): 1770-1779. DOI: 10.1182/bloodadvances.2020001729.
    [31] 左书凝, 许兰平. 单倍体相合造血干细胞移植治疗重型再生障碍性贫血的现状与进展[J]. 中华血液学杂志, 2015, 36(8): 707-710. DOI: 10.3760/cma.j.issn.0253-2727.2015.08.019.

    ZUO SN, XU LP. The current status and development of haploidentical allogeneic hematopoietic stem cell transplantation for severe aplastic anemia[J]. Chin J Hematol, 2015, 36(8): 707-710. DOI: 10.3760/cma.j.issn.0253-2727.2015.08.019.
    [32] YANG S, YUAN X, MA R, et al. Comparison of outcomes of frontline immunosuppressive therapy and frontline haploidentical hematopoietic stem cell transplantation for children with severe aplastic anemia who lack an HLA-matched sibling donor[J]. Biol Blood Marrow Transplant, 2019, 25(5): 975-980. DOI: 10.1016/j.bbmt.2019.01.017.
    [33] DE MOLLA VC, BARBOSA MCR, JUNIOR AM, et al. Natural killer cells 56bright16- have higher counts in the umbilical cord blood than in the adult peripheral blood[J]. Hematol Transfus Cell Ther, 2022, DOI: 10.1016/j.htct.2022.07.001[Epubaheadofprint].
    [34] LIU L, ZHANG Y, JIAO W, et al. Comparison of efficacy and health-related quality of life of first-line haploidentical hematopoietic stem cell transplantation with unrelated cord blood infusion and first-line immunosuppressive therapy for acquired severe aplastic anemia[J]. Leukemia, 2020, 34(12): 3359-3369. DOI: 10.1038/s41375-020-0933-7.
    [35] ZHOU F, ZHANG F, ZHANG L, et al. A multicentre trial of intensive immunosuppressive therapy combined with umbilical cord blood for the treatment of severe aplastic anaemia[J]. Ann Hematol, 2022, 101(8): 1785-1794. DOI: 10.1007/s00277-022-04864-1.
    [36] LIU L, LEI M, FU R, et al. Matched related transplantation versus immunosuppressive therapy plus eltrombopag for first-line treatment of severe aplastic anemia: a multicenter, prospective study[J]. J Hematol Oncol, 2022, 15(1): 105. DOI: 10.1186/s13045-022-01324-1.
    [37] 李华, 李晓帆, 李乃农. 间充质干细胞促进脐带血干细胞体外扩增的研究进展[J/CD]. 中华细胞与干细胞杂志(电子版), 2022, 12(1): 34-38. DOI: 10.3877/cma.j.issn.2095-1221.2022.01.006.

    LI H, LI XF, LI NN. Research advances ofumbilical cord blood stem cells proliferation promoted by mesenchymal stem cells in vitro[J/CD]. Chin J Cell Stem Cell (Electr Edit), 2022, 12(1): 34-38. DOI: 10.3877/cma.j.issn.2095-1221.2022.01.006.
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出版历程
  • 收稿日期:  2023-01-18
  • 刊出日期:  2023-05-15

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