Challenges in Pediatric CML

Chronic myeloid leukemia (CML) in children is a very rare disease. Differences exist between pediatric and adult CML. Children and adolescents in chronic phase (CP-CML) presented with more aggressive features including higher leukocytes counts, higher frequency of splenomegaly and larger spleen compared to adult patients. A prognostic score has not been established in the pediatric population with CML. Long term administration of imatinib, a tyrosine kinase inhibitor (TKI) of first generation, is the current standard of care for childhood CP-CML. Early response (BCR-ABL1/ABL ratio <10%) after 3 months of imatinib could identify a subset of children who require alternative treatment strategies. The possibility of stopping imatinib in the subset of children who are in deep and prolonged molecular remission is currently explored. Second generation TKIs (nilotinib, dasatinib) are currently  investigated in pediatric CML. Allogeneic hematopoietic stem cell transplantation in pediatric CML is currently restricted to de novo advanced phases, recurrent or progressive disease.