Different types of treatments are available for patients with LCH. Some treatments are standard (the currently used treatment), and some are being tested in clinical trials. A treatment clinical trial is a research study meant to help improve current treatments or obtain information on new treatments for patients. When clinical trials show that a new treatment is better than the standard treatment, the new treatment may become the standard treatment. Whenever possible, patients should take part in a clinical trial in order to receive new types of treatment for LCH.
Clinical trials are taking place in many parts of the country. Information about ongoing clinical trials is available from the
Treatment will be overseen by a pediatric oncologist, a doctor who specializes in treating children with cancer. The pediatric oncologist works with other pediatric healthcare providers who are experts in treating children with LCH and who specialize in certain areas of medicine. These may include the following specialists:
Some late effects may be treated or controlled. It is important to talk with your child's doctors about the possible late effects caused by some treatments.
LCH is usually treated with anticancer treatments. These treatments stop the LCH cells from growing and dividing.
Radiation therapy is a treatment that uses high-energy x-rays or other types of radiation to kill cells or keep them from growing. External radiation therapy uses a machine outside the body to send radiation toward the LCH lesion.
Photodynamic therapy is a treatment that uses a drug and a certain type of laser light to kill cells. A drug that is not active until it is exposed to light is injected into a vein. The drug collects more in LCH cells than in normal cells. For LCH, laser light is shined onto the skin and the drug becomes active and kills the LCH cells. Photodynamic therapy causes little damage to healthy tissue. Patients who have photodynamic therapy should not spend too much time in the sun.
Chemotherapy is a treatment that uses drugs to stop the growth of cells, either by killing the cells or by stopping them from dividing. When chemotherapy is taken by mouth or injected into a vein or muscle, the drugs enter the bloodstream and can reach cells throughout the body (systemic chemotherapy). When chemotherapy is placed directly onto the skin or into the cerebrospinal fluid, an organ, or a body cavity such as the abdomen, the drugs mainly affect cells in those areas (regional chemotherapy).
Nitrogen mustard is a drug that is put directly on the skin to treat small LCH lesions.
Biologic therapy is a treatment that uses the patient’s immune system to fight disease. Substances made by the body or made in a laboratory are used to boost, direct, or restore the body’s natural defenses against disease. This type of cancer treatment is also called biotherapy or immunotherapy.
Interferon is a type of biologic therapy used to treat LCH of the skin.
Targeted therapy is a type of treatment that uses drugs or other substances to find and attack LCH cells without harming normal cells. Imatinib mesylate is a type of targeted therapy called a tyrosine kinase inhibitor. It stops blood stem cells from turning into dendritic cells.
Other types of drugs used to treat LCH include the following:
Stem cell transplant is a method of giving chemotherapy and replacing blood-forming cells destroyed by the LCH treatment. Stem cells (immature blood cells) are removed from the blood or bone marrow of the patient or a donor and are frozen and stored. After the chemotherapy is completed, the stored stem cells are thawed and given back to the patient through an infusion. These reinfused stem cells grow into (and restore) the body's blood cells.
For some patients, taking part in a clinical trial may be the best treatment choice. Clinical trials are part of the medical research process. Clinical trials are done to find out if new treatments are safe and effective or better than the standard treatment.
Many of today's standard treatments for disease are based on earlier clinical trials. Patients who take part in a clinical trial may receive the standard treatment or be among the first to receive a new treatment.
Patients who take part in clinical trials also help improve the way diseases will be treated in the future. Even when clinical trials do not lead to effective new treatments, they often answer important questions and help move research forward.
Some clinical trials only include patients who have not yet received treatment. Other trials test treatments for patients whose disease has not gotten better. There are also clinical trials that test new ways to stop a disease from recurring (coming back) or reduce the side effects of treatment.
Clinical trials are taking place in many parts of the country. See the
Many patients with LCH get better with treatment. However, when treatment stops, new lesions may appear or old lesions may come back. This is called reactivation (recurrence) and may occur within one year after stopping treatment. Patients with multisystem disease are more likely to have a reactivation. More common sites of reactivation are bone, ears, or skin, and diabetes insipidus also may develop. Less common sites of reactivation include lymph nodes, bone marrow, spleen, liver, or lung. Some patients may have more than one reactivation over a number of years.
Some of the tests that were done to diagnose the disease may be repeated. This is to see how well the treatment is working and if there are any new lesions. These tests may include:
Other tests that may be needed include:
Decisions about whether to continue, change, or stop treatment may be based on the results of these tests.
Some of the tests will continue to be done from time to time after treatment has ended. The results of these tests can show if your condition has changed or if the disease has recurred (come back). These tests are sometimes called follow-up tests or check-ups.