Purpose of Review
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.
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Conflict of Interest
Stephanie M. Smith and Kathleen M. Sakamoto declare no conflict of interest. Nobuko Hijiya has served on data and safety monitoring boards (DSMB) for Novartis and Incyte Corporation.
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Smith, S.M., Hijiya, N. & Sakamoto, K.M. Chronic Myelogenous Leukemia in Childhood. Curr Oncol Rep 23, 40 (2021). https://doi.org/10.1007/s11912-021-01025-x
- Chronic myelogenous leukemia
- Chronic myeloid leukemia
- Targeted biologic therapy
- Tyrosine kinase inhibitor
- Pediatric CML
- Childhood CML