Chronic myeloid leukemia in children and adolescents: The achilles heel of oncogenesis and tyrosine kinase inhibitors

Abstract

Chronic myeloid leukemia (CML) is a rare disease in children and adolescents. The goal of therapy in children and adolescents is normal life expectancy, without compromising normal growth and development and potential for achievement of milestones in adult life. The perspective of cure is also reflected in the goal of treatment‐free remission, with its surrogate markers, such as deep molecular response, also becoming the new endpoints of therapy efficacy in children and ad-olescents. Chronic myeloid leukemia was a fatal disease to children and adolescents in the past. Following the treatment paradigm of imatinib, it became a chronic disease with the potential of complete remission and even cure without the long‐term hazards of allogeneic hematopoietic cell transplantation. The diagnosis and treatment of CML affect a child’s trajectory through life and important physiological events like development and procreation.