How We Treat Childhood Chronic Myeloid Leukemia (CML)

Expert Care and Treatment for Childhood Blood Cancers

The Hematologic Malignancy Center at Dana-Farber/Boston Children's Cancer and Blood Disorders Center is one of the top pediatric leukemia and lymphoma treatment centers in the world. In addition to treating blood cancers, our Center also treats histiocytosis, a condition that shares some of the characteristics of cancer.

Childhood Hematologic Malignancy Center

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

Supportive Care

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.

Childhood Cancer Clinical Trials

Find answers to common questions about clinical trials for childhood cancer, including whether or not a clinical trial may be the right choice for your child. Contact us and we can help you navigate your options.

Clinical Trials for Pediatric Patients

Clinical Trials for Childhood Chronic Myeloid Leukemia (CML)

Dana-Farber/Boston Children's is actively engaged in leukemia clinical trials, leading to new therapies and aiming for eventual cures. Our clinical team works closely with our leukemia researchers to quickly develop new treatments based on the latest scientific discoveries.

Clinical trials at Dana-Farber/Boston Children's are designed to investigate new therapies for refractory or relapsed leukemia, novel combinations of chemotherapy drugs, and promising new treatment agents. Our researchers are conducting randomized clinical studies to reduce treatment intensity to lessen side effects and to test more intense and newer therapies to improve cure rates.

Learn more about clinical trials for pediatric cancer.

Long-Term Outlook for Children Treated for CML

Your child's prognosis greatly depends on the extent of the disease, the cancer's response to treatment and the leukemia's specific genetic abnormalities. Your child's age, overall health, and tolerance for specific medications, procedures, or therapies will also play a role.

As with any cancer, prognosis and long-term survival can vary greatly. Prompt medical attention and aggressive therapy are important for the best prognosis, and continuous follow-up care is essential. New methods to improve treatment and decrease side effects of CML treatment are continually being discovered.

Relapse can occur during any stage of treatment, even with aggressive therapy, or may occur months or years after treatment has ended. If CML recurs, the treatment plan often includes targeted therapy (drugs, such as TKIs, that specifically attack a molecular pathway required to keep a tumor growing), chemotherapy and immunotherapy (treatment that stimulates the immune system to reject and destroy a tumor).

Long-Term Follow-Up

Continuous follow-up care is essential for any child diagnosed with chronic myeloid leukemia (CML). However, the specific type of care needed will depend on the type of treatment your child received or is receiving.

Children who are able to manage their disease through TKIs will need to follow up regularly with their clinical care team to monitor their disease and to assess potential side effects.

Children who are cured of CML through stem cell transplant or other therapies will need ongoing cancer survivorship care. Side effects of chemotherapy, as well as second malignancies, can occur in survivors of CML. Our pediatric cancer survivorship programs, which set the national standard for childhood cancer survivorship care, offer ongoing care to manage late effects caused by your child's cancer or by the treatment they received.