CML in Children and Young Adults

Most cases of CML occur in adults. From 2012 to 2016, approximately 2 percent of all cases of CML occurred in children, adolescents and young adults less than age 20.

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. Some studies indicate that children and young adult patients have lower rates of complete cytogenetic and major molecular responses compared with older adults. Children and young adults might have a slightly higher risk of transformation to accelerated and blast phase. Children with CML should be treated by pediatric hematologist-oncologists (doctors who specialize in treating pediatric patients with blood cancer).

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 in 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. The following medications are used in the treatment of children with CML:

  • Imatinib (Gleevec®)
  • Dasatinib (Sprycel®)
  • Nilotinib (Tasigna®)
  • Bosutinib (Bosulif®)

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 is known to increase the risk of lower response or possibly treatment failure. 

Taking into account the potential concerns of lifelong TKI treatment, researchers are studying stopping TKIs after a period of deep molecular response. Treatment-free remission is now considered a goal of treatment for selected patients and is a focus of study in various ongoing clinical trials. Intermittent TKI dosing is another potential method to reduce 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.

Caring for Kids and Adolescents with Blood Cancer: A Workbook for Families

Download or order a copy of the workbook which provides information, resources and worksheets to use throughout your child's cancer journey. Call an Information Specialist at 800.955.4572 to order a copy. 

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