How I treat chronic myeloid leukemia in children and adolescents

Abstract

Chronic myeloid leukemia (CML) is rare in children and adolescents. Although outcomes have dramatically improved owing to tyrosine kinase inhibitors (TKIs) in the last 2 decades, there are still many challenges related to the management of pediatric CML, including the impact of TKIs on growth deceleration and unknown long-term adverse effects as well as defining the role of treatment-free remission. Unlike adult CML, which is driven by evidence-based guidelines, management of pediatric CML is often extrapolated from adult guidelines. However, pediatric CML differs from adult CML in many ways, presenting with different biological; molecular; and, most importantly, host factors that make it necessary for a different treatment approach. After the initial approval of first-generation imatinib for pediatric CML in 2003, 3 TKIs, all second-generation TKIs, have been approved, including dasatinib, nilotinib, and bosutinib, which have greatly expanded therapeutic options but also added complexity to treatment determination. The expanded treatment options also call into question the treatment choice for pediatric CML, long-term efficacy, and safety profiles of these TKIs. We present 3 cases commonly encountered in pediatric CML, their challenges and relevant issues, as well as recommended managements.