Brown Patrick A
Department of Oncology, Johns Hopkins Kimmel Cancer Center, Baltimore, MD, USA; Department of Pediatrics, Johns Hopkins Kimmel Cancer Center, Baltimore, MD, USA.
Clin Perinatol. 2021 Mar;48(1):15-33. doi: 10.1016/j.clp.2020.11.002.
Neonates are at risk for 3 major forms of leukemia in the first year of life: acute leukemia, juvenile myelomonocytic leukemia, and transient abnormal myelopoiesis associated with Down syndrome. These disorders are rare but generate interest due to aggressive clinical presentation, suboptimal response to current therapies, and fascinating biology. Each can arise as a result of unique constitutional and acquired genetic events. Genetic insights are pointing the way toward novel therapeutic approaches. This article reviews key epidemiologic, clinical, and molecular features of neonatal leukemias, focusing on risk stratification, treatment, and strategies for developing novel molecularly targeted approaches to improve future outcomes.
急性白血病、青少年粒单核细胞白血病以及与唐氏综合征相关的一过性异常髓系造血。这些疾病很罕见，但因其侵袭性的临床表现、对当前治疗的欠佳反应以及引人入胜的生物学特性而备受关注。每一种都可能由独特的先天性和后天性遗传事件引发。遗传学见解正为新型治疗方法指明方向。本文综述了新生儿白血病的关键流行病学、临床和分子特征，重点关注风险分层、治疗以及开发新型分子靶向方法以改善未来治疗结果的策略。
