Department of Oncology and.
Department of Pediatrics, School of Medicine, Johns Hopkins University, Baltimore, MD.
Blood. 2019 Jan 17;133(3):205-214. doi: 10.1182/blood-2018-04-785980. Epub 2018 Nov 20.
Leukemia in infants is rare but generates tremendous interest due to its aggressive clinical presentation in a uniquely vulnerable host, its poor response to current therapies, and its fascinating biology. Increasingly, these biological insights are pointing the way toward novel therapeutic approaches. Using representative clinical case presentations, we review the key clinical, pathologic, and epidemiologic features of infant leukemia, including the high frequency of gene rearrangements. We describe the current approach to risk-stratified treatment of infant leukemia in the major international cooperative groups. We highlight recent discoveries that elucidate the molecular biology of infant leukemia and suggest novel targeted therapeutic strategies, including modulation of aberrant epigenetic programs, inhibition of signaling pathways, and immunotherapeutics. Finally, we underscore the need for increased global collaboration to translate these discoveries into improved outcomes.
婴儿白血病较为罕见，但因其在极其脆弱的宿主中表现出侵袭性的临床表现、对现有治疗方法的反应较差，以及其引人入胜的生物学特性而备受关注。越来越多的生物学研究结果为新型治疗方法提供了线索。我们通过具有代表性的临床病例介绍，复习了婴儿白血病的关键临床、病理和流行病学特征，包括高频基因重排。我们还描述了国际主要合作组目前针对婴儿白血病进行风险分层治疗的方法。我们强调了最近的一些发现，这些发现阐明了婴儿白血病的分子生物学，并提出了新的靶向治疗策略，包括调节异常的表观遗传程序、抑制信号通路和免疫治疗。最后，我们强调需要加强全球合作，将这些发现转化为更好的治疗效果。
