A small percentage of patients diagnosed with CML are children and young adults. CML represents about 3 percent of newly diagnosed childhood leukemias.
While CML appears to have the same disease course in children as it does in adults, there are no specific guidelines for CML treatment in children yet. The features of disease at diagnosis and the response to therapy in children seem to be similar to that in adults.
Imatinib mesylate (Gleevec®) is the primary treatment used for children diagnosed with CML. More than 80 percent of children with chronic phase disease treated with Gleevec achieve complete cytogenetic remission.
Although there are not a great number of studies focused on the treatment of pediatric patients with CML, there is evidence that TKI drugs, such as Gleevec, may slow growth, particularly for those treated before they reach puberty. Stopping treatment with Gleevec can result in catch-up skeletal growth (when the body begins to grow again after a period of slowed development). Careful monitoring of the child’s height and overall growth during and after treatment is recommended.
Other treatment options for children who have CML and do not respond well to Gleevec can be treatment with other tyrosine kinase inhibitors (TKIs), and, possibly stem cell transplantation. Currently other TKIs, which include such drugs as dasatinib (Sprycel®) or nilotinib (Tasigna®), are used. When resistance to a TKI is observed, then analysis for specific resistance causing mutations is commonly performed. Complications of a transplant remain challenging, so treatment with Gleevec continues to be the first choice for younger patients in chronic phase despite the potential side effects associated with its use.
With oral medications, it is important to follow the doctor’s directions and keep taking the medication for as long as prescribed. This can be overwhelming for parents of children and young adults because remembering to take the drug can be hard.
Talk to your child’s doctor about the best treatment for him or her and discuss any concerns regarding the risks associated with your child’s therapy. It is important for your child to be seen by a doctor who specializes in pediatric leukemia. See the free LLS publications Choosing a Blood Cancer Specialist or Treatment Center and Coping With Childhood Leukemia and Lymphoma for more information.