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Parents are advised to

  • Seek treatment from a hematologist/oncologist who is experienced in treating JMML, or from a hematologist/oncologist who is in consultation with a cancer center.
  • Speak with their child’s doctor about the most appropriate treatment. 

Without treatment, JMML progresses rapidly. There are two widely used JMML treatment protocols: stem cell transplantation and drug therapy.

Stem Cell Transplantation

Allogeneic stem cell transplantation has been widely used in the treatment of JMML patients, and it remains the only known cure for JMML. Although this treatment has been noted to achieve long-term remission in up to 50 percent of patients, relapses occur in up to 30 to 40 percent of patients after transplantation, often within the first year. While the rates of relapse are high, patients may achieve a cure with a second stem cell transplant.

Drug Therapy

Without treatment, JMML progresses rapidly. Using chemotherapy alone will almost always lead to the disease coming back after the treatment is over. The use of standard chemotherapy before a stem cell transplant is often undertaken to control the disease during the 1 to 2-month period that is required to arrange a stem cell transplant. Since JMML is difficult to treat with currently available drug therapy, participating in a clinical trial investigating new drugs may be an option for some children, even right after diagnosis.

Clinical Trials

Taking part in a clinical trial may be a good treatment choice for your child. Clinical trials are under way to help extend survival and increase the quality of life for juvenile myelomonocytic leukemia (JMML) patients. Today's standard treatments for cancer are based on earlier clinical trials. The Leukemia & Lymphoma Society continues to invest funds in JMML research.

Clinical trials can involve new drugs, new combinations of drugs, or previously approved drugs being studied in new ways such as new drug doses or new schedules to administer the drugs. Clinical trials are conducted worldwide under rigorous guidelines to help doctors find out whether new cancer treatments are safe and effective or better than the standard treatment.

Receive one-on-one navigation from an LLS Clinical Trial Specialist who will personally assist you throughout the entire clinical-trial process: Click Here

Current JMML Research and Clinical Trials

Below are some examples of therapies currently under study to achieve longer-lasting remissions for JMML patients:

  • Hypomethylating agents (HMAs). These drugs stop cells from making DNA, which inhibits cell division and makes cancer cells more likely to die.
  • Azacitidine (Vidaza®) is a medication approved for treating chronic myelomonocytic leukemia (CMML). It is currently being studied as a single agent and in combination with other drugs to treat JMML patients.
  • MEK inhibitor. The majority of JMML patients harbor a mutation in the RAS pathway. Inhibition of this pathway is one therapeutic strategy currently under investigation. The efficacy of the MEK inhibitor trametinib is being tested in a clinical trial sponsored by the Children’s Oncology Group (COG), for treating relapsed and refractory JMML patients.
  • Second stem cell transplantation. The effectiveness of second allogeneic stem cell transplantation in JMML patients who have relapsed after a first transplant is being studied in clinical trials. 

Finding the Best Treatment Approach

As you develop a treatment plan with your child's doctor, be sure to discuss:

  • The results you can expect from treatment
  • Potential side effects, including late-term effects and long-term effects
  • The possibility of participating in a clinical trial, where your child will have access to advanced medical treatment that may be more beneficial than standard treatment

You may find it helpful to bring a loved one with you to your doctor's visits for support and to take notes and ask follow-up questions. It's a good idea to prepare questions you'd like to ask when you visit your doctor. You can also record your conversations with your doctor and listen more closely when you get home.

To download lists of suggested questions to ask your healthcare providers, click here.

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