It's important that your child's doctor is experienced in treating children with juvenile myelomonocytic leukemia (JMML) or works in consultation with a JMML specialist. This type of specialist is usually called a hematologist oncologist.
Stem cell transplantation is the most common treatment for JMML. Standard chemotherapy, despite its intensity, has helped only a small number of patients. Some patients have reacted favorably to 13-cis-retinoic acid therapy (Accutane®). The drug has been able to stabilize the disease and bring it into a partial remission.
Allogeneic Stem Cell Transplantation
An allogeneic stem cell transplantation offers the only known cure for JMML and is widely used to treat the disease. Up to 50 percent of patients have achieved long-term survival, but up to 30 percent to 40 percent of those patients have suffered relapses.
Although it's not the only type of stem cell transplantation, allogeneic stem cell transplantation is the most common. Allogeneic transplantation involves transferring stem cells from a healthy person (the donor) to the patient. The procedure follows high-intensity chemotherapy, potent drugs that must be toxic enough to kill leukemic cells. Unfortunately, the drugs also take aim at normal stem cells in the bone marrow.
The main reasons for doing an allogeneic stem cell transplant are to:
- Give strong doses of chemotherapy to kill JMML cells
- Start a new supply of red cells, white cells and platelets with help from the transplanted donor stem cells
Some patients undergo a second allogeneic transplantation. A second transplantation can be effective when used with reduced immunosuppression. This method presumably helps donor immune cells better fight the disease. This attack is called the graft-versus-tumor (GVT) effect.
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 approved drugs being studied to treat patients 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 research and trials for new or improved drug therapies and other treatment under way:
- Etanercept (Enbrel®). Scientists are studying the effects of the drug etanercept on relapsed JMML patients. The drug blocks a hormone called the tumor necrosis factor, which plays a role in helping JMML cells grow. Etanercept is currently used to treat rheumatoid arthritis and juvenile rheumatoid arthritis.
- Tipifarnib (Zarnestra®). Tipifarnib is a type of drug called a farnesyl transferase inhibitor. It works by blocking enzymes needed by cancer cells to form. A clinical trial conducted by the Children's Oncology Group, a childhood cancer research organization, has shown tipifarnib to be significantly effective.
- Second allogeneic stem cell transplantation. A second transplantation can be effective for JMML patients who have relapsed after a first transplant.
Finding the Best Treatment Approach
There is no one standard treatment for JMML that's used or accepted throughout the international medical community. However, two widely used JMML treatment protocols (practice guidelines) are based on studies by two groups:
- The Children's Oncology Group (COG) JMML Study in North America. COG is the world's largest childhood cancer research organization and supported by the National Cancer Institute.
- The European Working Group of MDS (EWOG-MDS) and JMML in Childhood Study. EWOG-MDS is an international cooperative study group.
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.