Division of Hematology/Oncology, Department of Pediatrics, Stanford University School of Medicine, CCSR-1215C, 269 Campus Drive, Stanford, CA, USA.
Division of Pediatric Hematology, Oncology and Stem Cell Transplant, Department of Pediatrics, Columbia University Irving Medical Center, New York, NY, USA.
Curr Oncol Rep. 2021 Mar 14;23(4):40. doi: 10.1007/s11912-021-01025-x.
Chronic myelogenous leukemia (CML) is rare in children, requiring extrapolation from treatment of adults. In this review, we explore similarities and differences between adult and pediatric CML with a focus on therapeutic advances and emerging clinical questions.
Pediatric CML is effectively treated with long-term targeted therapy using tyrosine kinase inhibitors (TKIs). Newly diagnosed pediatric patients in chronic phase can now be treated with imatinib, dasatinib, or nilotinib without allogeneic hematopoietic stem cell transplantation. While treatment-free remission is possible in adults in chronic phase with optimal response to therapy, data are currently insufficient to support stopping TKI in pediatrics outside of a clinical trial. Knowledge gaps remain regarding long-term and late effects of TKIs in pediatric CML. Targeted therapy has markedly improved outcomes for pediatric CML, while raising a number of clinical questions, including the possibility of treatment-free remission and long-term health implications of prolonged TKI exposure at a young age.
慢性髓细胞白血病（CML）在儿童中罕见，需要从成人的治疗中推断。在这篇综述中，我们探讨了成人和儿科 CML 之间的相似点和不同点，重点是治疗进展和新出现的临床问题。
儿科 CML 通过长期使用酪氨酸激酶抑制剂（TKI）进行靶向治疗可有效治疗。现在，新诊断的慢性期儿科患者可以用伊马替尼、达沙替尼或尼洛替尼治疗，而无需进行异基因造血干细胞移植。虽然成人慢性期对治疗有最佳反应可能会出现无治疗缓解，但目前的数据不足以支持在临床试验之外停止儿科 TKI 治疗。儿科 CML 中 TKI 的长期和迟发性影响仍存在知识空白。靶向治疗显著改善了儿科 CML 的预后，同时提出了许多临床问题，包括无治疗缓解的可能性以及在年幼时长期接触 TKI 对健康的长期影响。
