Langerhans Cell Histiocytosis Treatment
- Langerhans cell histiocytosis is a type of cancer that can damage tissue or cause lesions to form in one or more places in the body.
- Family history or having a parent who was exposed to certain chemicals may increase the risk of LCH.
- The signs and symptoms of LCH depend on where it is in the body.
- Skin and nails
- Lymph nodes and thymus
- Endocrine system
- Central nervous system (CNS)
- Liver and spleen
- Bone marrow
- Tests that examine the organs and body systems where LCH may occur are used to detect (find) and diagnose LCH.
- Certain factors affect prognosis (chance of recovery) and treatment options.
Langerhans cell histiocytosis is a type of cancer that can damage tissue or cause lesions to form in one or more places in the body.
Langerhans cell histiocytosis (LCH) is a rare cancer that begins in LCH cells (a type of dendritic cell which fights infection). Sometimes there are mutations (changes) in LCH cells as they form. These include mutations of the BRAF gene. These changes may make the LCH cells grow and multiply quickly. This causes LCH cells to build up in certain parts of the body, where they can damage tissue or form lesions.
LCH is not a disease of the Langerhans cells that normally occur in the skin.
LCH may occur at any age, but is most common in young children. Treatment of LCH in children is different from treatment of LCH in adults. The treatments for LCH in children and adults are described in separate sections of this summary.
Check the list of NCI-supported cancer clinical trials that are now accepting patients with childhood Langerhans cell histiocytosis. For more specific results, refine the search by using other search features, such as the location of the trial, the type of treatment, or the name of the drug. Talk with your child’s doctor about clinical trials that may be right for your child. General information about clinical trials is available from the NCI website.
Family history or having a parent who was exposed to certain chemicals may increase the risk of LCH.
Anything that increases your risk of getting a disease is called a risk factor. Having a risk factor does not mean that you will get cancer; not having risk factors doesn’t mean that you will not get cancer. Talk with your doctor if you think you may be at risk. Risk factors for LCH include the following:
- Having a parent who was exposed to certain chemicals such as benzene.
- Having a parent who was exposed to metal, granite, or wood dust in the workplace.
- A family history of cancer, including LCH.
- Having infections as a newborn.
- Having a personal history or family history of thyroid disease.
- Smoking, especially in young adults.
- Being Hispanic.
Screening and Detection
Tests that examine the organs and body systems where LCH may occur are used to detect (find) and diagnose LCH.
The following tests and procedures may be used to detect (find) and diagnose LCH or conditions caused by LCH:
- Physical exam and history: An exam of the body to check general signs of health, including checking for signs of disease, such as lumps or anything else that seems unusual. A history of the patient’s health habits and past illnesses and treatments will also be taken.
- Neurological exam: A series of questions and tests to check the brain, spinal cord, and nerve function. The exam checks a person’s mental status, coordination, and ability to walk normally, and how well the muscles, senses, and reflexes work. This may also be called a neuro exam or a neurologic exam.
- Complete blood count (CBC) with differential: A procedure in which a sample of blood is drawn and checked for the following:
- The amount of hemoglobin (the protein that carries oxygen) in the red blood cells.
- The portion of the blood sample made up of red blood cells.
- The number and type of white blood cells.
- The number of red blood cells and platelets.
- Blood chemistry studies: A procedure in which a blood sample is checked to measure the amounts of certain substances released into the body by organs and tissues in the body. An unusual (higher or lower than normal) amount of a substance can be a sign of disease.
- Liver function test: A blood test to measure the blood levels of certain substances released by the liver. A high or low level of these substances can be a sign of disease in the liver.
- BRAF gene testing: A laboratory test in which a sample of blood or tissue is tested for the BRAF gene.
- Urinalysis: A test to check the color of urine and its contents, such as sugar, protein, red blood cells, and white blood cells.
- Water deprivation test: A test to check how much urine is made and whether it becomes concentrated when little or no water is given. This test is used to diagnose diabetes insipidus, which may be caused by LCH.
- Bone marrow aspiration and biopsy: The removal of bone marrow and a small piece of bone by inserting a hollow needle into the hipbone. A pathologist views the bone marrow and bone under a microscope to look for signs of LCH.
The following tests may be done on the tissue that was removed:
- Immunohistochemistry: A test that uses antibodies to check for certain antigens in a sample of tissue. The antibody is usually linked to a radioactive substance or a dye that causes the tissue to light up under a microscope. This type of test may be used to tell the difference between different types of cancer.
- Flow cytometry: A laboratory test that measures the number of cells in a sample, how many cells are live, and the size of the cells. It also shows the shapes of the cells and whether there are tumor markers on the surface of the cells. The cells are stained with a light-sensitive dye, placed in a fluid, and passed in a stream before a laser or other type of light. The measurements are based on how the light-sensitive dye reacts to the light.
- Bone scan: A procedure to check if there are rapidly dividing cells in the bone. A very small amount of radioactive material is injected into a vein and travels through the bloodstream. The radioactive material collects in the bones with cancer and is detected by a scanner.
- X-ray: An x-ray of the organs and bones inside the body. 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. Sometimes a skeletal survey is done. This is a procedure to x-ray all of the bones in the body.
- CT scan (CAT scan): A procedure that makes a series of detailed pictures of areas inside the body, taken from different angles. The pictures are made by a computer linked to an x-ray machine. A dye may be injected into a vein or swallowed to help the organs or tissues show up more clearly. This procedure is also called computed tomography, computerized tomography, or computerized axial tomography.
- MRI (magnetic resonance imaging): A procedure that uses a magnet, radio waves, and a computer to make a series of detailed pictures of areas inside the body. A substance called gadolinium may be injected into a vein. The gadolinium collects around the LCH cells so that they show up brighter in the picture. This procedure is also called nuclear magnetic resonance imaging (NMRI).
- PET scan (positron emission tomography scan): A procedure to find tumor cells in the body. A small amount of radioactive glucose (sugar) is injected into a vein. The PET scanner rotates around the body and makes a picture of where glucose is being used in the body. Tumor cells show up brighter in the picture because they are more active and take up more glucose than normal cells do.
- Ultrasound exam: A procedure in which high-energy sound waves (ultrasound) are bounced off internal tissues or organs and make echoes. The echoes form a picture of body tissues called a sonogram. The picture can be printed to be looked at later.
- Bronchoscopy: A procedure to look inside the trachea and large airways in the lung for abnormal areas. A bronchoscope is inserted through the nose or mouth into the trachea and lungs. A bronchoscope is a thin, tube-like instrument with a light and a lens for viewing. It may also have a tool to remove tissue samples, which are checked under a microscope for signs of cancer.
- Endoscopy: A procedure to look at organs and tissues inside the body to check for abnormal areas in the gastrointestinal tract or lungs. An endoscope is inserted through an incision (cut) in the skin or opening in the body, such as the mouth. An endoscope is a thin, tube-like instrument with a light and a lens for viewing. It may also have a tool to remove tissue or lymph node samples, which are checked under a microscope for signs of disease.
- Biopsy: The removal of cells or tissues so they can be viewed under a microscope by a pathologist to check for LCH cells. To diagnose LCH, a biopsy of bone lesions, skin, lymph nodes, or the liver may be done.
Signs and Symptoms
The signs and symptoms of LCH depend on where it is in the body.
These and other signs and symptoms may be caused by LCH or by other conditions. Check with your doctor if you or your child have any of the following:
Skin and nails
LCH in infants may affect the skin only. In some cases, skin-only LCH may get worse over weeks or months and become a form called high-risk multisystem LCH.
In infants, signs or symptoms of LCH that affects the skin may include:
- Flaking of the scalp that may look like “cradle cap”.
- Raised, brown or purple skin rash anywhere on the body.
In children and adults, signs or symptoms of LCH that affects the skin and nails may include:
- Flaking of the scalp that may look like dandruff.
- Raised, red or brown, crusted rash in the groin area, abdomen, back, or chest, that may be itchy.
- Bumps or ulcers on the scalp.
- Ulcers behind the ears, under the breasts, or in the groin area.
- Fingernails that fall off or have discolored grooves that run the length of the nail.
Signs or symptoms of LCH that affects the mouth may include:
- Swollen gums.
- Sores on the roof of the mouth, inside the cheeks, or on the tongue or lips.
- Teeth that become uneven.
- Tooth loss.
Signs or symptoms of LCH that affects the bone may include:
- Swelling or a lump over a bone, such as the skull, ribs, spine, thigh bone, upper arm bone, elbow, eye socket, or bones around the ear.
- Pain where there is swelling or a lump over a bone.
- Children with LCH lesions in bones around the ears or eyes have a high risk for diabetes insipidus and other central nervous system disease.
Lymph nodes and thymus
Signs or symptoms of LCH that affects the lymph nodes or thymus may include:
- Swollen lymph nodes.
- Trouble breathing.
- Superior vena cava syndrome. This can cause coughing, trouble breathing, and swelling of the face, neck, and upper arms.
Signs or symptoms of LCH that affects the pituitary gland may include:
- Diabetes insipidus. This can cause a strong thirst and frequent urination.
- Slow growth.
- Early or late puberty.
- Being very overweight.
Signs or symptoms of LCH that affects the thyroid may include:
- Swollen thyroid gland.
- Hypothyroidism. This can cause tiredness, lack of energy, being sensitive to cold, constipation, dry skin, thinning hair, memory problems, trouble concentrating, and depression. In infants, this can also cause a loss of appetite and choking on food. In children and adolescents, this can also cause behavior problems, weight gain, slow growth, and late puberty.
- Trouble breathing.
Central nervous system (CNS)
Signs or symptoms of LCH that affects the CNS (brain and spinal cord) may include:
- Loss of balance, uncoordinated body movements, and trouble walking.
- Trouble speaking.
- Trouble seeing.
- Changes in behavior or personality.
- Memory problems.
- These signs and symptoms may be caused by lesions in the CNS or by CNS neurodegenerative syndrome.
Liver and spleen
Signs or symptoms of LCH that affects the liver or spleen may include:
- Swelling in the abdomen caused by a buildup of extra fluid.
- Trouble breathing.
- Yellowing of the skin and whites of the eyes.
- Easy bruising or bleeding.
- Feeling very tired.
Signs or symptoms of LCH that affects the lung may include:
- Collapsed lung. This condition can cause chest pain or tightness, trouble breathing, feeling tired, and a bluish color to the skin.
- Trouble breathing, especially in adults who smoke.
- Dry cough.
- Chest pain.
Signs or symptoms of LCH that affects the bone marrow may include:
- Easy bruising or bleeding.
- Frequent infections.
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 with cancer. 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. Some clinical trials are open only to patients who have not started treatment.
Clinical trials are taking place in many parts of the country. Information about ongoing clinical trials is available from the NCI website. 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 childhood cancer.
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:
- Primary care physician.
- Pediatric surgeon.
- Pediatric hematologist.
- Radiation oncologist.
- Pediatric nurse specialist.
- Rehabilitation specialist.
- Social worker.
Some cancer treatments cause side effects months or years after treatment for childhood cancer has ended.
Side effects from cancer treatment that begin during or after treatment and continue for months or years are called late effects. Late effects of cancer treatment may include the following:
- Slow growth and development.
- Hearing loss.
- Bone, tooth, liver, and lung problems.
- Changes in mood, feeling, learning, thinking, or memory.
- Second cancers, such as leukemia, retinoblastoma, Ewing sarcoma, brain or liver cancer.
Some late effects may be treated or controlled. It is important to talk with your child’s doctors about the effects cancer treatment can have on your child. (See the PDQ summary on Late Effects of Treatment for Childhood Cancer for more information.)
Many patients with multisystem LCH have late effects caused by treatment or by the disease itself. These patients often have long-term health problems that affect their quality of life.
Nine types of standard treatment are used:
Chemotherapy is a cancer treatment that uses drugs to stop the growth of cancer 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 cancer 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 cancer cells in those areas (regional chemotherapy).
Chemotherapy agents given by injection or by mouth are used to treat LCH. Chemotherapy agents include vinblastine, cytarabine, cladribine, and methotrexate. Nitrogen mustard is a drug that is put directly on the skin to treat small LCH lesions.
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.
When there is severe liver or lung damage, the entire organ may be removed and replaced with a healthy liver or lung from a donor.
Radiation therapy is a cancer treatment that uses high-energy x-rays or other types of radiation to kill cancer cells or keep them from growing. External radiation therapy uses a machine outside the body to send radiation toward the cancer. In LCH, a special lamp may be used to send ultraviolet B (UVB) radiation toward LCH skin lesions.
Photodynamic therapy is a cancer treatment that uses a drug and a certain type of laser light to kill cancer cells. A drug that is not active until it is exposed to light is injected into a vein. The drug collects more in cancer cells than in normal cells. For LCH, laser light is aimed at the skin and the drug becomes active and kills the cancer 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 (PUVA) therapy, the patient receives a drug called psoralen and then ultraviolet A radiation is directed to the skin.
Biologic therapy is a treatment that uses the patient’s immune system to fight cancer. Substances made by the body or made in a laboratory are used to boost, direct, or restore the body’s natural defenses against cancer. 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. Immunomodulators are also a type of biologic therapy. Thalidomide is an immunomodulator used to treat LCH.
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 that may become cancer cells. Other types of kinase inhibitors that affect cells with the BRAF gene, such as dabrafenib and vemurafenib, are being studied in clinical trials for LCH.
A family of genes, called ras genes, may cause cancer when they are mutated (changed). Ras genes make proteins that are involved in cell signaling pathways, cell growth, and cell death. Ras pathway inhibitors are a type of targeted therapy being studied in clinical trials. They block the actions of a mutated ras gene or its protein and may stop the growth of cancer.
Other drug therapy
Other drugs used to treat LCH include the following:
- Steroid therapy, such as prednisone, is used to treat LCH lesions.
- Bisphosphonate therapy (such as pamidronate, zoledronate, or alendronate) is used to treat LCH lesions of the bone and to lessen bone pain.
- Nonsteroidal anti-inflammatory drugs (NSAIDs) are drugs (such as aspirin and ibuprofen) that are commonly used to decrease fever, swelling, pain, and redness. Sometimes an NSAID called indomethacin is used to treat LCH.
- Retinoids, such as isotretinoin, are drugs related to vitamin A that can slow the growth of LCH cells in the skin. The retinoids are taken by mouth.
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.
Observation is closely monitoring a patient’s condition without giving any treatment until signs or symptoms appear or change.
New types of treatment are being tested in clinical trials.
Information about clinical trials is available from the NCI website.
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 cancer research process. Clinical trials are done to find out if new cancer treatments are safe and effective or better than the standard treatment.
Many of today’s standard treatments for cancer 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 cancer 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 cancer has not gotten better. There are also clinical trials that test new ways to stop cancer from recurring (coming back) or reduce the side effects of cancer treatment.
Clinical trials are taking place in many parts of the country. See the Treatment Options for Childhood LCH and the Treatment Options for Adult LCH 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. 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 LCH may be repeated. This is to see how well the treatment is working and if there are any new lesions. These tests may include:
- Physical exam.
- Neurological exam.
- Ultrasound exam.
- CT scan.
- PET scan.
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 cancer has recurred (come back). These tests are sometimes called follow-up tests or check-ups.