National Cancer Institute


Posted Date: Apr 27, 2014

Expert-reviewed information summary about the treatment of childhood Langerhans Cell Histiocytosis.

Langerhans Cell Histiocytosis Treatment

General Information About Langerhans Cell Histiocytosis (LCH)

Key Points for this Section

  • Langerhans cell histiocytosis is a disease that can damage tissue or cause lesions to form in one or more places in the body.
  • Family history and having a parent who was exposed to certain chemicals may increase the risk of LCH.
  • The cause of LCH is not known.
  • The signs and symptoms of LCH depend on where it occurs in the body.
    • Skin and nails
    • Mouth
    • Bone
    • Lymph nodes and thymus
    • Endocrine system (including the pituitary gland)
    • Thyroid
    • Central nervous system
    • Liver and spleen
    • Lung
    • 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 disease that can damage tissue or cause lesions to form in one or more places in the body.

Langerhans cell histiocytosis (LCH) is a rare disease that begins in LCH cells (a type of dendritic cell which fights infection). Sometimes there are changes in LCH cells as they form. These changes may cause the LCH cells to grow and multiply quickly. This causes the 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 Langerhans cells that normally form in skin.

Scientists do not agree on whether LCH is a type of cancer or is a condition caused by a change in the immune system. LCH is mainly treated with anticancer drugs. Some of these drugs are also used to treat immune system conditions.

LCH may occur at any age, but is most common in young children. Treatment of LCH in childhood 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 for U.S. clinical trials from NCI's list of 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 Web site.

Family history and 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 the disease; not having risk factors doesn't mean that you will not get the disease. Talk with your doctor if you think you may be at risk. Risk factors for LCH may include the following:

The cause of LCH is not known.

The signs and symptoms of LCH depend on where it occurs 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

In infants, signs or symptoms of LCH may include:

  • Flaking of the scalp that may look like “cradle cap”.
  • Raised, brown or purple spots anywhere on the body.

In children and adults, signs or symptoms of LCH 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 with discolored grooves that run the length of the nail or lost nails.

Mouth

Signs or symptoms of LCH may include:

  • Swollen gums.
  • Sores on the roof of the mouth, inside the cheeks, or on the tongue or lips.
  • Having teeth that become uneven.
  • Tooth loss.

Bone

Signs or symptoms of LCH may include:

  • Swelling or a lump over a bone, such as the skull, ribs, spine, thigh bone, upper arm bone, elbow, or eye socket.
  • Pain where there is swelling or a lump over a bone.

Lymph nodes and thymus

Signs or symptoms of LCH may include:

Endocrine system (including the pituitary gland)

Signs or symptoms of LCH may include:

Thyroid

Signs or symptoms of LCH 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 teens, this can also cause behavior problems, weight gain, slow growth, and late puberty.
  • Trouble breathing.

Central nervous system

Signs or symptoms of LCH may include:

  • Loss of balance, uncoordinated body motions, and trouble walking.
  • Trouble speaking.
  • Changes in behavior.
  • Memory problems.

Liver and spleen

Signs or symptoms of LCH may include:

  • Swelling in the abdomen caused by a build up of extra fluid.
  • Trouble breathing.
  • Yellowing of the skin and whites of the eyes.
  • Itching.
  • Easy bruising or bleeding.
  • Feeling very tired.

Lung

Signs or symptoms of LCH may include:

  • Spontaneous pneumothorax (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.

Bone marrow

Signs or symptoms of LCH may include:

  • Easy bruising or bleeding.
  • Fever.
  • Frequent infections.

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.
    • 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 in the organ or tissue that makes it.
  • 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.
  • 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, blood, and a small piece of bone by inserting a hollow needle into the hipbone. A pathologist views the bone marrow, blood, and bone under a microscope to look for signs of LCH. Bone marrow aspiration and biopsy. After a small area of skin is numbed, a Jamshidi needle (a long, hollow needle) is inserted into the patient’s hip bone. Samples of blood, bone, and bone marrow are removed for examination under a microscope.
  • 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 and is detected by a scanner.Bone scan. A small amount of radioactive material is injected into the patient's bloodstream and collects in abnormal cells in the bones. As the patient lies on a table that slides under the scanner, the radioactive material is detected and images are made on a computer screen or film.
  • 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.Computed tomography (CT) scan of the abdomen. The patient lies on a table that slides through the CT machine, which takes x-ray pictures of the inside of the body.
  • 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).Magnetic resonance imaging (MRI) of the abdomen. The patient lies on a table that slides into the MRI machine, which takes pictures of the inside of the body. The pad on the patient’s abdomen helps make the pictures clearer.
  • 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.PET (positron emission tomography) scan. The patient lies on a table that slides through the PET machine. The head rest and white strap help the patient lie still. A small amount of radioactive glucose (sugar) is injected into the patient's vein, and a scanner makes a picture of where the glucose is being used in the body. Cancer cells show up brighter in the picture because they 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.
  • Endoscopy: A procedure to look at organs and tissues inside the body to check for abnormal areas. 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.

Certain factors affect prognosis (chance of recovery) and treatment options.

LCH in organs such as the skin, bones, lymph nodes, or pituitary gland usually gets better with treatment and is called "low- risk". LCH in the spleen, liver, or bone marrow is harder to treat and is called "high-risk".

The prognosis (chance of recovery) and treatment options depend on the following:

  • How old the patient is when diagnosed with LCH.
  • How many body systems the disease affects.
  • Whether the disease is found in the liver, spleen, bone marrow, or certain bones in the skull.
  • How quickly the disease responds to initial treatment.
  • Whether the disease has just been diagnosed or has come back ( recurred).

In infants up to one year of age, LCH may go away without treatment.

Stages of LCH

Key Points for this Section

  • There is no staging system for Langerhans cell histiocytosis (LCH).
  • Treatment of LCH is based on where LCH cells are found in the body and whether one or more body systems are affected.

There is no staging system for Langerhans cell histiocytosis (LCH).

The extent or spread of disease is usually described as stages. There is no staging system for LCH.

Treatment of LCH is based on where LCH cells are found in the body and whether one or more body systems are affected.

LCH is described as single-system disease or multisystem disease, depending on how many body systems are affected:

  • Single-system LCH: LCH is found in one part of an organ or body system (unifocal) or in more than one part of that organ or body system (multifocal).
  • Multisystem LCH: LCH occurs in two or more organs or body systems.

LCH may affect low-risk organs or high-risk organs, depending on where it occurs in the body:

Recurrent LCH

Recurrent Langerhans cell histiocytosis (LCH) is disease that has recurred (come back) after it has been treated. The disease may come back in the same place or in other parts of the body. It often recurs in the bone, ears, skin, or pituitary gland. LCH often recurs the year after stopping treatment. Recurrent LCH is also called reactivation.

Treatment Option Overview for LCH

Key Points for this Section

  • There are different types of treatment for patients with Langerhans cell histiocytosis (LCH).
  • Children with LCH should have their treatment planned by a team of health care providers who are experts in treating this disease in children.
  • Some treatments for LCH cause side effects months or years after treatment has ended.
  • Nine types of standard treatment are used:
    • Watchful waiting
    • Surgery
    • Radiation therapy
    • Photodynamic therapy
    • Chemotherapy
    • Biologic therapy
    • Targeted therapy
    • Other drug therapy
    • Stem cell transplant
  • New types of treatment are being tested in clinical trials.
  • Patients may want to think about taking part in a clinical trial.
  • Patients can enter clinical trials before, during, or after starting their treatment.
  • When treatment of LCH stops, new lesions may appear or old lesions may come back.
  • Follow-up tests may be needed.

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

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

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 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, 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.

Treatment Options for LCH in Children

Check for U.S. clinical trials from NCI's list of 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 Web site.

Treatment of Low-Risk Disease

Treatment of childhood Langerhans cell histiocytosis (LCH) skin lesions may include the following:

Treatment of childhood LCH bone lesions in the front, sides, or back of the skull, or in any other single bone may include the following:

Treatment of childhood LCH lesions in bones around the ears or eyes may lower the risk of diabetes insipidus and other long-term problems. Treatment may include:

Treatment of spine or hip bone lesions that have weakened the bone and may lead to a broken bone in childhood LCH may include:

  • Radiation therapy.
  • Chemotherapy for lesions that spread from the spine into nearby tissue.
  • Surgery to strengthen the weakened bone by bracing or fusing the bones together.

Treatment of two or more bone lesions may include:

Treatment of combinations of childhood LCH skin, lymph node, pituitary gland, and bone lesions may include:

  • Chemotherapy and steroid therapy.
  • Bisphosphonate therapy.

Treatment of High-Risk Disease

Treatment of childhood LCH multisystem disease lesions in the spleen, liver, or bone marrow (with or without skin, bone, lymph node, lung, or pituitary gland lesions) may include:

Treatment of childhood LCH central nervous system lesions may include various combinations of the following:

  • Chemotherapy with or without steroid therapy.
  • Steroid therapy.

Treatment Options for Recurrent, Refractory, and Progressive Childhood LCH

Recurrent LCH is disease that cannot be detected for some time after treatment and then comes back. Treatment of recurrent childhood LCH in the skin, bone, lymph nodes, gastrointestinal tract, pituitary gland, or central nervous system (low-risk organs) may include:

Refractory LCH is disease that does not get better with treatment. Treatment of refractory childhood LCH in high-risk organs and in multisystem low-risk organs may include high-dose chemotherapy. Treatment of childhood LCH in multisystem high-risk organs that did not respond to chemotherapy may include stem cell transplant.

Progressive LCH is disease that continues to grow during treatment. Treatment of progressive childhood LCH in patients with multisystem disease may include anticancer drugs that have not been given to the patient before.

Treatment Options for LCH in Adults

For more information about LCH trials for adults, see the Histiocyte Society Web site.

Check for U.S. clinical trials from NCI's list of cancer clinical trials that are now accepting patients with adult 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 Web site.

Langerhans cell histiocytosis (LCH) in adults is a lot like LCH in children and can form in the same


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