儿童急性髓系白血病的研究进展：分子机制与精准治疗策略
Journal: Basic Medical Theory Research DOI: 10.12238/bmtr.v7i3.14422
Abstract
儿童急性髓系白血病(AML)是儿童血液系统恶性肿瘤的重要类型,其分子分型与预后高度相关。近年来,随着二代测序技术的应用,AML的分子机制研究不断深入,推动了靶向治疗和免疫治疗的快速发展。本文系统综述儿童AML的流行病学特征、分子分型与预后分层、新型治疗策略(包括标准化疗优化、靶向药物及CAR-T细胞疗法)以及复发/难治性AML的管理进展。尽管个体化精准治疗显著改善了患者生存,但耐药机制、移植后复发及治疗毒性仍是临床挑战。未来需结合多组学技术与新型临床试验设计,进一步优化儿童AML的全程管理。
Keywords
儿童急性髓系白血病(AML)；流行病学特点；分子分型；治疗策略；复发/难治性AML
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[5] 郭碧赟,王玥,李健,等.华南地区儿童核心结合因子相关急性髓系白血病临床特征及预后多中心研究[J].中华儿科杂志,2023,61(10):881-888.
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[7] Sinha,C.;Cunningham,L.C.;Liu,P.P.Core Binding Factor Ac ute Myeloid Leukemia: New Prognostic Categories and Therape utic Opportunities. Semin.Hematol.2015,52,215-222.
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[17] Hosono,N.Genetic abnormalities and pathophysiology of MDS. Int.J.Clin.Oncol.2019,24,885–892.
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[24] Kenderian,S.S.;Ruella,M.;Shestova,O.;etal.CD33-Specific Ch imeric Antigen Receptor T Cells Exhibit Potent Preclinical Activi ty Against Human Acute Myeloid Leukemia. Leukemia 2015,29(8),1637-1647.
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[27] Uy,G.L.;Aldoss,I.;Foster,M.C.;et al.Flotetuzumab as Salv age Immunotherapy for Refractory Acute Myeloid Leukemia.Blo od 2021,137(6),751-762.
[28] Leung,W.H.;Neale,G.;Behm,F.;et al.T Cell Therapy for Ac ute Myeloid Leukemia: Current Status and Future Directions. Front.Immunol.2020,11,574289.
[29] Lamble,A.J.;Lind,E.F.Targeting the Immune Microenvi ronment in Acute Myeloid Leukemia: A Focus on T Cell Immun ity.Front.Oncol.2018,8,213.
[30] Lamble,A.J.;Lind,E.F.Immune Checkpoint Inhibition in Acute Myeloid Leukemia: Current Status and Future Directio ns.J.Hematol. Oncol.2019,12(1),107.
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[34] Locatelli,F.;Merli,P.;Pagliara,D.;LiPira,G.;Falco,M.;Pende,D.; Rondelli, R.; Lucarelli, B.; Brescia, L.P.; Masetti, R.; et al. Outcome of children with acute myeloid leukaemia given haploidentical hematopoietic cell transplantation with posttransplantcyclophosphamide.Br.J.Haematol.2017,178,578-586.
[35] Kaspers G J L, Creutzig U. Pediatric acute myeloid leukemia:international progress and future directions[J]. Leukemia,2005,19(12):2025-2029.
[36] Kanakry CG,de Lima MJ,Luznik L.Alternative Donor Allo geneic Hematopoietic Cell Transplantation for Acute Myeloid Leukemia.Semin Hematol.2015;52(3):232-242.
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