JMML is not easily treated, though several therapies offer hope for a successful outcome. A stem cell transplant is the best option; however, your child's team of doctors will help determine the best approach for your child's unique situation, based on:
- 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 Options for JMML
Stem Cell (Bone Marrow) Transplant
Stem cells are a specific type of cell from which all blood cells develop. They can grow into red blood cells to carry oxygen, white blood cells to fight disease and infection, and platelets to aid in blood clotting. Stem cells are found primarily in the bone marrow, but some also circulate in the blood stream.
When your child's stem cells are replaced with those of a healthy and compatible donor, it is called allogeneic transplantation. The goal is for healthy stem cells from another person (whose tissue must be the same, or almost the same, as your child's) to restore your child's normal blood production.
All children undergoing a stem cell transplant for JMML must receive high-dose chemotherapy to make room in their bone marrow for the new stem cells. Called a "conditioning regimen" this chemotherapy accomplishes several important things. It suppresses the body's immune system to prevent rejection of the stem cells, and it destroys cancer cells in the body. Like a blood transfusion, stem cells are given to a child through an intravenous (IV) catheter. Children are awake through this painless process.
It generally takes two to four weeks for the stem cells to multiply and make new blood cells. This is called engraftment. Side effects, such as fever, chills, and shortness of breath, can accompany the infusion of stem cells. More significant complications, including graft rejection and graft-versus-host disease (a condition in which the donor's immune cells attack the patient's body), can occur following the transplant.
Chemotherapy is a drug treatment that works by interfering with the cancer cell's ability to grow or reproduce. Standard chemotherapy alone is not adequate for curing JMML but may be used to decrease symptoms while your child is prepared for a stem cell transplant.
Different groups of chemotherapy drugs work in different ways. Your child may receive chemotherapy orally, as a pill to swallow; intramuscularly, as an injection into the muscle or fat tissue; intravenously, as a direct injection into the bloodstream or IV; or intrathecally, as a direct injection into the spinal column through a needle.
While chemotherapy can be quite effective in treating certain cancers, the drugs cannot differentiate normal healthy cells from cancer cells. As a result, there may be adverse side effects during treatment. Being able to anticipate these side effects can help the care team, child, and family prepare (and, in some cases, prevent) these complications from occurring.
Biological therapy refers to a wide range of substances that may be able to involve the body's own immune system to fight cancer or lessen harmful side effects of some treatments.