Pediatric acute lymphoblastic leukemia.
Pediatric acute lymphoblastic leukemia.
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DOI：
10.3324/haematol.2020.247031
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发表时间：
2020-11-01
期刊：
影响因子：
10.1
通讯作者：
Mullighan CG
中科院分区：
文献类型：
--
作者：
Inaba H;Mullighan CG
在过去的十年中，我们对儿童急性淋巴细胞白血病（ALL）的遗传和生物学基础的理解取得了巨大进展，实验模型的发展探索了机制和评估了新的治疗方法，并开发了更有效的治疗分层。基因组分析彻底改变了我们对ALL分子分类的理解，这些进步推动了在ALL临床管理中实施基因组和转录组表征，以促进更准确的风险分层，并在某些情况下进行靶向治疗。虽然突变或途径导向的靶向治疗（例如，使用酪氨酸激酶抑制剂治疗费城染色体[Ph]阳性和Phlike b细胞ALL）目前仅适用于少数ALL患儿，但许多新发现的分子改变已导致探索针对失调细胞途径的方法。随着嵌合抗原受体t细胞疗法和双特异性参与blinatumumab治疗晚期疾病的成功，细胞或体液免疫疗法的疗效已得到证实。这篇综述描述了我们对ALL生物学的理解以及风险分层和治疗的最佳方法的关键进展，并提出了基础和临床研究的关键领域。
The last decade has witnessed great advances in our understanding of the genetic and biological basis of childhood acute lymphoblastic leukemia (ALL), the development of experimental models to probe mechanisms and evaluate new therapies, and the development of more efficacious treatment stratification. Genomic analyses have revolutionized our understanding of the molecular taxonomy of ALL, and these advances have led the push to implement genome and transcriptome characterization in the clinical management of ALL to facilitate more accurate risk-stratification and, in some cases, targeted therapy. Although mutation- or pathway-directed targeted therapy (e.g., using tyrosine kinase inhibitors to treat Philadelphia chromosome [Ph]-positive and Phlike B-cell-ALL) is currently available for only a minority of children with ALL, many of the newly identified molecular alterations have led to the exploration of approaches targeting deregulated cell pathways. The efficacy of cellular or humoral immunotherapy has been demonstrated with the success of chimeric antigen receptor T-cell therapy and the bispecific engager blinatumomab in treating advanced disease. This review describes key advances in our understanding of the biology of ALL and optimal approaches to risk-stratification and therapy, and it suggests key areas for basic and clinical research.
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影响因子：
51.1
作者：
Den Boer, Monique L.;van Slegtenhorst, Marjon;De Menezes, Renee X.;Cheok, Meyling H.;Buijs-Gladdines, Jessica G. C. A. M.;Peters, Susan T. C. J. M.;Van Zutven, Laura C. M.;Beverloo, H. Berna;Van der Spek, Peter J.;Escherich, Gaby;Horstmann, Martin A.;Janka-Schoub, Gritta E.;Kamps, Willem A.;Evans, William E.;Pieters, Rob
通讯作者：
Pieters, Rob
影响因子：
11.4
作者：
Bhojwani D;Sposto R;Shah NN;Rodriguez V;Yuan C;Stetler-Stevenson M;O'Brien MM;McNeer JL;Quereshi A;Cabannes A;Schlegel P;Rossig C;Dalla-Pozza L;August K;Alexander S;Bourquin JP;Zwaan M;Raetz EA;Loh ML;Rheingold SR
通讯作者：
Rheingold SR
影响因子：
82.9
作者：
通讯作者：
--
影响因子：
1.9
作者：
Carroll, Andrew J.;Shago, Mary;Heerema, Nyla A.
通讯作者：
Heerema, Nyla A.
影响因子：
45.3
作者：
Albertsen, Birgitte Klug;Grell, Kathrine;Schmiegelow, Kjeld
通讯作者：
Schmiegelow, Kjeld