Children and adolescents with CML are treated at Dana-Farber/Boston Children's through our Hematologic Malignancy Center's Leukemia Treatment Program. Dana-Farber/Boston Children's has played a key role in refining treatment for childhood leukemia, and we continue to be a world leader in leukemia clinical trials designed to increase cure rates, decrease treatment-related side effects, and improve care for long-term survivors.
CML treatment depends on your child's individual situation, including the specific phase of the disease.
Treatment for Chronic Phase CML
Children diagnosed with chronic phase CML are most often treated with tyrosine kinase inhibitors (TKIs). Children with chronic phase CML do not receive chemotherapy and only rarely undergo a stem cell transplant.
Tyrosine-Kinase Inhibitors (TKI)
Tyrosine-Kinase Inhibitors (TKI) are drugs that target a specific gene alteration that is known to cause CML. These drug types include Imatinib (Gleevec), Dasatinib, Nilotinib, Bosutinib, and Ponatinib. Most children with CML will take Imatinib. However, if your child has specific genetic alterations that don't respond to Imatinib, your child's doctor may recommend Dasatinib or Nilotinib. Bosutinib and Ponatinib are very rarely used for children.
Stem Cell Transplant
Stem cell transplant was once the only treatment option for children in chronic phase CML but is now largely reserved for patients who are resistant to or intolerant of TKI treatment. Stem cell transplant involves replacing your child's stem cells — a specific type of cells from which blood cells develop — with healthy stem cells from another person. When your child's stem cells are replaced with those of a healthy and compatible donor, it is called allogeneic transplantation.
Treatment may begin with high-dose chemotherapy (sometimes combined with radiation therapy) to destroy all bone marrow, stem cells, and cancerous cells in your child's body. Then, healthy stem cells from another person (whose tissue must be the same, or almost the same as your child's) are used to help restore your child's normal blood production.
Treatment for Accelerated Phase and Blast Crisis CML
Children diagnosed with accelerated phase CML or blast crisis will need initial treatment with TKIs (see description above) and chemotherapy.
Chemotherapy is a drug treatment that works by interfering with the cancer cell's ability to grow or reproduce. Different groups of chemotherapy drugs work in different ways. Your child may receive chemotherapy orally, as a pill to swallow; subcutaneously, as an injection below the skin; intravenously, as a direct injection into the bloodstream, or IV; or intrathecally, as a direct injection into the spinal column through a needle.
If your child's disease continues to progress, however, the next stage of treatment will be stem cell transplant.
Other Treatments for Childhood CML
- Blood transfusions are often given to replace or supplement your child's red blood cells and platelets.
- Antibiotics may be given to your child to prevent or treat infections.
- Other medications may be given to prevent or treat damage to other systems of your child's body caused by CML treatment, or for nausea and side effects of treatment.
Your child's team of doctors will help determine the best approach for your child's unique situation, based on factors including:
- Your child's age, overall health, and medical history
- The extent of the disease
- Your child's tolerance for certain medications, procedures or therapies
- How your child's doctors expect the disease to progress
- Your opinion and preferences
Treatment for CML may cause various side effects. Supportive care will be provided to keep your child as healthy and strong as possible throughout treatment.