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儿童急性淋巴细胞白血病:通过合作取得的进展

Childhood Acute Lymphoblastic Leukemia: Progress Through Collaboration.

作者信息

Pui Ching-Hon, Yang Jun J, Hunger Stephen P, Pieters Rob, Schrappe Martin, Biondi Andrea, Vora Ajay, Baruchel André, Silverman Lewis B, Schmiegelow Kjeld, Escherich Gabriele, Horibe Keizo, Benoit Yves C M, Izraeli Shai, Yeoh Allen Eng Juh, Liang Der-Cherng, Downing James R, Evans William E, Relling Mary V, Mullighan Charles G

机构信息

Ching-Hon Pui, Jun J. Yang, James R. Downing, Williams E. Evans, Mary V. Relling, and Charles G. Mullighan, St Jude Children's Research Hospital and the University of Tennessee Health Science Center, Memphis, TN; Stephen P. Hunger, University of Colorado School of Medicine and the University of Colorado Cancer Center and Children's Hospital Colorado, Aurora, CO; Rob Pieters, Princess Máxima Center for Pediatric Oncology, Utrecht, the Netherlands; Martin Schrappe, University Medical Center Schleswig-Holstein, Christian-Albrechts-University, Kiel; Gabriele Escherich, Clinic of Pediatric Hematology and Oncology, University Medical Center Hamburg Eppendorf, Hamburg, Germany; Andrea Biondi, Clinica Pediatrica and Centro Ricerca Tettamanti, Università di Milano-Bicocca, Monza, Italy; Ajay Vora, Children's Cancer Group, School of Cancer, Manchester Academic Health Sciences Centre, University of Manchester, Manchester, United Kingdom; André Baruchel, Hôpital Robert Debré and University of Paris Diderot, Paris, France; Lewis B. Silverman, Dana-Farber Cancer Institute and Boston Children's Hospital, Boston, MA; Kjeld Schmiegelow, Institute of Clinical Medicine, University of Copenhagen and Juliane Marie Centre, the University Hospital Rigshospitalet, Copenhagen, Denmark; Keizo Horibe, Nagoya Medical Center, Clinical Research Center, Nagoya, Japan; Yves C.M. Benoit, Universiteit Gent, Gent, Belgium; Shai Izraeli, Chaim Sheba Medical Center and Sackler Medical School, Tel Aviv University, Tel Aviv, Israel; Allen Eng Juh Yeoh, Yong Loo Lin School of Medicine and Cancer Science Institute, National University of Singapore, and Viva-University Children's Cancer Centre, National University Hospital, Singapore; and Der-Cherng Liang, Mackay Memorial Hospital, Taipei, Taiwan.

出版信息

J Clin Oncol. 2015 Sep 20;33(27):2938-48. doi: 10.1200/JCO.2014.59.1636. Epub 2015 Aug 24.

Abstract

PURPOSE

To review the impact of collaborative studies on advances in the biology and treatment of acute lymphoblastic leukemia (ALL) in children and adolescents.

METHODS

A review of English literature on childhood ALL focusing on collaborative studies was performed. The resulting article was reviewed and revised by the committee chairs of the major ALL study groups.

RESULTS

With long-term survival rates for ALL approaching 90% and the advent of high-resolution genome-wide analyses, several international study groups or consortia were established to conduct collaborative research to further improve outcome. As a result, treatment strategies have been improved for several subtypes of ALL, such as infant, MLL-rearranged, Philadelphia chromosome-positive, and Philadelphia chromosome-like ALL. Many recurrent genetic abnormalities that respond to tyrosine kinase inhibitors and multiple genetic determinants of drug resistance and toxicities have been identified to help develop targeted therapy. Several genetic polymorphisms have been recognized that show susceptibility to developing ALL and that help explain the racial/ethnic differences in the incidence of ALL.

CONCLUSION

The information gained from collaborative studies has helped decipher the heterogeneity of ALL to help improve personalized treatment, which will further advance the current high cure rate and the quality of life for children and adolescents with ALL.

摘要

目的

回顾协作研究对儿童和青少年急性淋巴细胞白血病(ALL)生物学及治疗进展的影响。

方法

对聚焦于协作研究的儿童ALL英文文献进行综述。所得文章由主要ALL研究组的委员会主席进行审阅和修订。

结果

随着ALL的长期生存率接近90%以及高分辨率全基因组分析的出现,几个国际研究组或联盟成立,开展协作研究以进一步改善治疗结果。结果,ALL的几种亚型,如婴儿型、MLL重排型、费城染色体阳性型和费城染色体样ALL的治疗策略得到了改进。已确定许多对酪氨酸激酶抑制剂有反应的复发性遗传异常以及耐药性和毒性的多个遗传决定因素,以帮助开发靶向治疗。已识别出几种遗传多态性,它们显示出患ALL的易感性,并有助于解释ALL发病率的种族/民族差异。

结论

从协作研究中获得的信息有助于解读ALL的异质性,以帮助改善个性化治疗,这将进一步提高目前的高治愈率以及ALL儿童和青少年的生活质量。

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