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 survival in up to 50 percent of patients, relapses occur in up to 30 to 40 percent of patients after transplantation. While the rates of relapse are high, patients may achieve a cure with a second stem cell transplant.
Standard chemotherapy, regardless of the intensity, has proven effective in only a small number of patients. Treatment with 13-cis-retinoic acid (Accutane®) has shown some responses leading to disease stabilization and partial remission (rather than complete remission). Farnesyl transferase inhibitors are a class of drugs that are currently undergoing testing as treatment for JMML.
Since JMML is difficult to treat with current chemotherapy, participating in a clinical trial investigating new drugs may be an option for children who cannot have a stem cell transplant.
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.
Current JMML Research and Clinical Trials
Below are some examples of therapies currently under study to achieve longer-lasting remissions for JMML patients:
- Etanercept (Enbrel®) blocks the hormone called the “tumor necrosis factor” (TNF), which has been shown to play a role in helping the growth of JMML cells. This drug has been approved for the treatment of rheumatoid arthritis and juvenile rheumatoid arthritis. Studies are trying to determine its effectiveness in the treatment of relapsed JMML patients.
- Tipifarnib (Zarnestra®) is a type of drug called a “farnesyl transferase inhibitor” that may stop the growth of JMML cells by blocking the enzymes necessary for cancer cell growth. This drug has demonstrated significant clinical effectiveness according to a clinical trial conducted by the Children’s Oncology Group.
- Second allogeneic 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.
Azacitidine (Vidaza®) is approved for CMML, and it is also being investigated as a treatment for JMML. It works by changing gene expression patterns in cancer cells and increasing their susceptibility to death.
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.
Click here to download lists of suggested questions to ask your healthcare providers.