Chronic myeloid leukemia (CML) is a rare myeloproliferative disease in children. The primary cause of CML is the chimeric BCR::ABL1 gene in hematopoietic stem cells, which leads to leukocytosis, platelet proliferation, and splenomegaly. Lately, tyrosine kinase inhibitors (TKIs) have replaced hematopoietic cell transplantation, which was previously considered the only curative therapy, as the first-line treatment for chronic-phase CML. However, the clinical efficacy of TKIs, including those effective in adult CML, has not been well-investigated in pediatric CML. This review describes the recommended TKI-based management strategies for pediatric CML according to the literature and guidelines. Furthermore, we discuss the prospects for TKI discontinuation to avoid important adverse events, such as growth impairment, in children.
Keywords: Adverse event; Chronic myeloid leukemia; Pediatric; Tyrosine kinase inhibitor.
© 2022. Japanese Society of Hematology.