Division of Pediatric Hematology/Oncology/Stem Cell Transplant, Department of Pediatrics, Columbia University Irving Medical Center, New York, NY, 10069, USA.
Myeloproliferative Neoplasms Program, Memorial Sloan Kettering Cancer Center, New York, NY, 10065, USA.
Curr Hematol Malig Rep. 2022 Oct;17(5):121-126. doi: 10.1007/s11899-022-00673-5. Epub 2022 Aug 3.
Due to lack of pediatric-specific data, the management of chronic myeloid leukemia (CML) in pediatric, adolescents, and young adults is guided by adult CML evidence-based recommendations. Pediatric CML presents differently than adult CML and is often a more aggressive disease with different biological and host factors, yet there is sparse literature on how to address those differences.
Over the past two decades, tyrosine kinase inhibitors (TKIs) have changed the way CML is treated. There are currently three FDA-approved TKIs (imatinib, dasatinib, and nilotinib) for pediatric patients. When choosing which TKI to begin treatment with, there are many factors that should be considered on a case-to-case basis to obtain optimal outcomes. The safety profiles for long-term TKI use in pediatrics require further study. Unlike adults, children are still actively growing during TKI use, and the effect on development can be detrimental. TKI therapy is not recommended during pregnancy with variable but significant risk of fetal abnormalities and miscarriage, warranting counseling for young female patients prior to beginning TKIs. Attempts for treatment-free remission (TFR) by planned TKI cessation in eligible adult patients in deep and sustained molecular remission are now done as a standard of practice. However, data is sparse in the pediatric population. There is currently an ongoing Children's Oncology Group (COG) study to determine the feasibility of TFR as a treatment goal. Further research and additional pediatric trials are needed to characterize the unique aspects of CML in children and adolescents and optimize outcomes.
由于缺乏儿科特定数据，儿童、青少年和年轻成人慢性髓性白血病（CML）的治疗以成人 CML 的循证建议为指导。儿科 CML 的表现与成人 CML 不同，通常是一种更具侵袭性的疾病，具有不同的生物学和宿主因素，但关于如何解决这些差异的文献很少。
在过去的二十年中，酪氨酸激酶抑制剂（TKI）改变了 CML 的治疗方式。目前有三种 FDA 批准的 TKI（伊马替尼、达沙替尼和尼洛替尼）用于儿科患者。在选择开始治疗的 TKI 时，应根据具体情况考虑许多因素，以获得最佳疗效。儿科患者长期使用 TKI 的安全性概况需要进一步研究。与成人不同，儿童在使用 TKI 期间仍在积极生长，对发育的影响可能有害。不建议在怀孕期间使用 TKI，因为胎儿畸形和流产的风险存在差异且显著，需要在开始使用 TKI 之前为年轻女性患者提供咨询。在深度和持续分子缓解的合格成年患者中，通过计划 TKI 停药实现无治疗缓解（TFR）已成为一种标准治疗方法。然而，儿科人群的数据很少。目前，儿童肿瘤学组（COG）正在进行一项研究，以确定 TFR 作为治疗目标的可行性。需要进一步研究和额外的儿科试验来描述儿童和青少年 CML 的独特方面，并优化疗效。
