Langerhans Cell Histiocytosis Treatment - Treatment Option Overview for LCH

There are different types of treatment for patients with Langerhans cell histiocytosis (LCH).

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 NCI Web site. Choosing the most appropriate treatment is a decision that ideally involves the patient, family, and health care team.

Children with LCH should have their treatment planned by a team of health care providers who are experts in treating this disease in children.

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 treatments for LCH cause side effects months or years after treatment has ended.

Some treatments cause side effects that continue or appear months or years after treatment has ended. These are called late effects. Late effects of treatment for LCH may include the following:
  • Slow growth and development.
  • Hearing loss.
  • Bone, tooth, liver, and lung problems.
  • Changes in mood, feeling, learning, thinking, or memory.
  • Risk of cancer related to treatment.
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. Many patients with multisystem LCH have late effects caused by treatment or the disease itself. These patients often have long-term health problems that affect their quality of life.

Nine types of standard treatment are used:

LCH is usually treated with anticancer treatments. These treatments stop the LCH cells from growing and dividing.

Watchful waiting

Watchful waiting is closely monitoring a patient's condition without giving any treatment until signs or symptoms appear or change.


Surgery may be used to remove LCH lesions and a small amount of nearby healthy tissue. Curettage is a type of surgery that uses a curette (a sharp, spoon-shaped tool) to scrape LCH cells from bone.

Radiation therapy

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

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. In one type of photodynamic therapy, called psoralen and ultraviolet A therapy (PUVA), the patient receives a drug called psoralen and then ultraviolet radiation is directed to the skin.


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

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 treatment is also called biotherapy or immunotherapy. Interferon is a type of biologic therapy used to treat LCH of the skin.

Targeted therapy

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 drug therapy

Other drugs used to treat LCH include the following:

Stem cell transplant

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.

New types of treatment are being tested in clinical trials.

Information about clinical trials is available from the NCI Web site.

Patients may want to think about taking part in a clinical trial.

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.

Patients can enter clinical trials before, during, or after starting their treatment.

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 and the sections for links to current treatment clinical trials. These have been retrieved from NCI's clinical trials database.

When treatment of LCH stops, new lesions may appear or old lesions may come back.

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.

Follow-up tests may be needed.

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:
  • Brain stem auditory evoked response (BAER) test: A test that measures the brain's response to clicking sounds or certain tones.
  • Pulmonary function test (PFT): A test to see how well the lungs are working. It measures how much air the lungs can hold and how quickly air moves into and out of the lungs. It also measures how much oxygen is used and how much carbon dioxide is given off during breathing. This is also called a lung function test.
  • Chest x-ray: An x-ray of the organs and bones inside the chest. An x-ray is a type of energy beam that can go through the body and onto film, making a picture of areas inside the body.
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.