How I treat chronic myeloid leukemia in children and adolescents.
Nobuko HijiyaMeinolf SuttorpPublished in: Blood (2019)
Evidence-based recommendations have been established for treatment of chronic myeloid leukemia (CML) in adults treated with tyrosine kinase inhibitors (TKIs), but the rarity of this leukemia in children and adolescents makes it challenging to develop similar recommendations in pediatrics. In addition to imatinib, which was approved for pediatric CML in 2003, the second-generation TKIs dasatinib and nilotinib were recently approved for use in children, expanding the therapeutic options and pushing allogeneic stem cell transplantation to a third-line treatment of most pediatric cases. Yet, without sufficient data on efficacy and safety specific to pediatric patients, the selection of a TKI continues to rely on clinical experience in adults. Here, we present 4 case scenarios highlighting common yet challenging issues encountered in the treatment of pediatric CML (suboptimal response, poor treatment adherence, growth retardation, and presentation in advanced phases). Limited experience with very young children, the transition of teenagers to adult medicine, and the goal of achieving treatment-free remission for this rare leukemia are additional significant obstacles that require further clinical investigation through international collaboration.
Keyphrases
- chronic myeloid leukemia
- stem cell transplantation
- low dose
- acute myeloid leukemia
- type diabetes
- rheumatoid arthritis
- artificial intelligence
- adipose tissue
- deep learning
- metabolic syndrome
- skeletal muscle
- weight loss
- tyrosine kinase
- epidermal growth factor receptor
- childhood cancer
- glycemic control
- ulcerative colitis