A small percentage of patients diagnosed with CML are children and young adults. CML represents about 3 percent of newly diagnosed childhood leukemias.
Because CML is rare in children, there have only been a few pediatric studies evaluating the use of TKI therapy in children. Consequently, the treatment of children with CML is not standardized. It often follows guidelines developed for adults, even though there are differences between CML in children and adults in terms of disease presentation and progression. Children with CML should be treated by doctors who specialize in treating children with blood cancers.
Although there are not a great number of studies focused on the treatment of pediatric patients with CML, there is evidence that imatinib may slow growth, particularly for children who are treated before they reach puberty. Other rare side effects of imatinib seen in adults such as cardiotoxicity and thyroid dysfunction appear to be very rare in children. Since children with CML may receive TKI therapy for much longer than adults and during periods of active growth, follow-up care is very important. In addition to testing their pediatric patients’ responses to therapy, doctors should also monitor children’s
- Height and weight—Doctors should consider a bone scan and a bone density scan if there is evidence of abnormal growth.
- Puberty—Doctors should refer patients to an endocrinologist if there is a delay in puberty.
- Thyroid function
- Heart—Patients should have an annual echocardiogram.
Poor adherence to therapy, particularly in adolescents and young adults, is an additional concern. With oral TKIs, it is important to follow the doctor’s directions and keep taking the medication for as long as prescribed. Nonadherence to TKI treatment has the potential of suboptimal response or even treatment failure.
Taking into account the potential concerns of lifelong TKI treatment, researchers are studying stopping TKIs after a period of deep molecular response. At this time, there is limited information on the long-term outcomes of patients with CML after they have stopped taking imatinib. Intermittent TKI dosing is another potential approach to reducing long-term side effects in pediatric CML patients, but more studies are needed to evaluate this approach.
Allogeneic stem cell transplantation is another treatment option. Because there have been no randomized controlled trials comparing stem cell transplantation and imatinib in children due to the small number of pediatric patients, the decision on how to treat CML has been individualized. Stem cell transplantation should be evaluated against the complications associated with lifelong TKI use.
Talk to your child’s doctor about the best treatment for your child 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 booklets Choosing a Blood Cancer Specialist or Treatment Center and Coping With Childhood Leukemia and Lymphoma for more information.
- Download or order The Leukemia & Lymphoma Society's free booklet, Chronic Myeloid Leukemia