Chronic myeloid leukemia (CML) is composed of 3% of pediatric
leukemias, making evidence-based recommendations difficult.
Imatinib has revolutionized the treatment for adult CML by eliminating allogeneic
stem cell transplantation for almost all patients in chronic phase. Shown effective in pediatric CML,
imatinib and successive
tyrosine kinase inhibitors (TKI) have provided more therapeutic options. Because
stem cell transplantation has been better tolerated in children and adolescents, the decision to treat by either TKI or
transplantation is controversial. We present a recent case of a 12-month-old boy diagnosed with BCR-ABL(+) CML to highlight the controversies in treatment recommendations. We review the pediatric
stem cell transplantation outcomes as well as the pediatric experience with
imatinib and other TKIs. Finally, we compare the side effects as well as costs associated with allogeneic
stem cell transplantation versus TKI
therapy. We recommend that frontline
therapy for pediatric CML in chronic phase is TKI
therapy without
transplantation. Patients in accelerated or
blast crisis or who fail to reach landmarks on TKIs either because of intolerance or resistance should pursue
stem cell transplantation. Although we recommend adopting adult clinical experience to guide therapeutic decision making, the issues of infant CML,
drug formulation, pharmacokinetics, and adolescent compliance merit clinical investigation.