Frequently Asked Questions

LCH in Childhood

The questions below are regarding LCH in Childhood specifically. Please click on a question to view the answer.

  1. What causes LCH?
  2. Is there a cure for LCH?
  3. What is considered to be remission?
  4. Where did LCH get its name?
  5. Is LCH fatal?
  6. What are the different therapies/treatments commonly used to treat LCH?
  7. Can an infant be tested at birth for LCH?
  8. Is LCH hereditary?
  9. Does LCH spread?
  10. Is there a blood test to diagnose LCH?
  11. Is it true that LCH is mostly a childhood disease?
  12. With the diagnosis and treatment of LCH, is my child more likely to develop cancer?
  13. What are permanent consequences of LCH?
  14. What are the chances my child will develop permanent consequences?
  15. What is Neurodegeneration?
  16. Can neurodegeneration be prevented/reversed/treated?
  17. What is “PLCH?”
  18. What kind of physician should we use?
  19. What should I look for in a doctor?
  20. Will my child grow normally?
  21. What is the treatment for stunted growth related to LCH?
  22. Is growth hormone treatment safe?
  23. Are immunizations safe?
  24. What type of LCH involvement puts my child at higher risk for developing Diabetes Insipidus?
  25. What are the chances my child will develop diabetes insipidus?
  26. What are risk organs?
  27. What happens if my child has a recurrence or does not respond to treatment?
  28. What is reduced-intensity conditioning (RIC)?

Possible Side Effects of Treatment

  1. What are the side effects of vinblastine?
  2. What are the side effects of prednisone?
  3. What are the side effects of methotrexate?
  4. What are the possible side effects of 6-MP (mercaptopurine)?
  5. What are the possible side effects of 2-CdA (cladribine/leustatin)?

Histiocytic Disorders and Orphan Diseases

The questions below are regarding the Histiocytic Disorders in general, as well as Orphan Diseases. Please click on the question to view the answer.

  1. What are histiocytic disorders and how are they classified?
  2. Why are all of these disease with different names considered to be related to each other?
  3. Where can I find reliable information about histiocytosis?
  4. How can I explain histiocytosis to family and friends?
  5. What is an orphan disease?
  6. How many orphan diseases are there?
  7. Where can I learn more about rare diseases in general?

LCH in Childhood

  1. What causes LCH?
    To date, the cause has not been determined. Multiple potential causes have been explored, including viruses, molds, infections, genetics, geographic location, racial clustering, seasonal changes, and environmental exposure. Results of these studies are still insufficient to provide a definitive answer. Further studies are needed.

  2. Is there a cure for LCH?
    While some patients go into remission and may live normal lives with or without treatment, we usually don’t use the term “cure” with this disease. No specific amount of time without active disease has yet been established for adults to determine when a patient is considered to be cured.

  3. What is considered to be remission?
    Complete remission means that there is no evidence of disease, whereas partial remission means that most of the signs and symptoms of LCH are gone, but some still remain. Doctors use the term response and “non-active” to describe patients who are free of symptoms and signs of LCH. Usually a cure is linked to being in remission for a certain period of time. There is no established period of “non-active” disease before LCH is considered cured, but the chance for recurrence is low after five years from end of treatment.

  4. Where did LCH get its name?
    In 1868, the German pathologist Paul Langerhans discovered a type of white blood cell which eventually came to bear his name. The various manifestations of LCH were previously known by a number of different names (histiocytosis-X, eosinophilic granuloma, Letterer-Siwe disease, Hand-Schüller-Christian syndrome, etc.). In 1983, it was suggested that this disorder be named “Langerhans cell histiocytosis,” to recognize the key role of the Langerhans cell in all of the different manifestations. This name was later approved by the scientists comprising the Histiocyte Society.

  5. Is LCH fatal?
    It can be. A small percentage of patients, most often those with multisystem risk-organ involvement that is unresponsive to treatment, may not survive.

  6. What are the different therapies/treatments commonly used to treat LCH?
    Treatment is based upon the organ(s) involved, extension of disease, and in some cases, age of the patient. In some cases, no treatment is necessary. Others may respond to surgical removal, steroids, or anti-inflammatory drugs (NSAIDs). Low-dose radiation is helpful in some situations, but should be carefully used in children. There are patients who require chemotherapy such as vinblastine, vincristine, etoposide (VP-16), methotrexate, cytosine-arabinoside (Ara-C), and/or 6-MP. In patients with severe disease that does not respond to initial treatment, stronger chemotherapy combinations may be used. Ultraviolet light (PUVA) may be helpful for limited skin disease. In very rare instances, a transplant of the liver, lung, or bone marrow may be necessary.

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  7. Can an infant be tested at birth for LCH?
    A biopsy of the affected tissue, rather than a blood test, is required for diagnosis and would therefore not be appropriate as a routine test unless this disease is suspected.

  8. Is LCH hereditary?
    Although there are rare families (less than 2% of all cases) documented with more than one member diagnosed with LCH, at this point, there is no clear evidence that this disease is inherited.

  9. Does LCH spread?
    The exact mechanism that causes lesions to appear in other locations in the body is not yet known. However, some researchers believe that abnormal LCH cells travel through the blood like tumor cells and “seed” in different locations, creating new lesions. Others believe that there are different manifestations of the same disease.

  10. Is there a blood test to diagnose LCH?
    LCH is diagnosed with a biopsy of the affected tissue. Blood tests may be done to help determine the extent and/or severity of involvement, but blood tests are not diagnostic of the disease.

  11. Is it true that LCH is mostly a childhood disease?
    Not necessarily. Although we do know the incidence of childhood LCH, there is not enough data to determine how many adults are affected by this disease.

  12. With the diagnosis and treatment of LCH, is my child more likely to develop cancer?
    Although this occurs rarely, LCH is associated with cancer more often than would be expected by chance. This can occur before, during, or after the diagnosis of LCH. Some cancers following the LCH diagnosis might be related to the treatments given. When cancer occurs before LCH, the histiocytosis might represent a “reaction” to the cancer itself.

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  13. What are permanent consequences of LCH?
    Permanent consequences are also known as late effects of LCH, although they can occur early on. They are believed to be mostly related to the disease rather than treatment and include:
    • Diabetes Insipidus
    • Stunted growth
    • Bone abnormalities
    • Hearing loss
    • Neurological problems, including poor coordination, unsteadiness, difficulty with handwriting, abnormal eye movements, problems with speech, learning disabilities/decreased school performance, memory loss, and behavior difficulties.
    • Loss of teethLoss of spinal height
    • Delayed puberty
    • Bulging eyesScarring of lungs
    • Scarring of liver/cirrhosis
    • Secondary cancers
    Read more about permanent consequences of LCH.

  14. What are the chances my child will develop permanent consequences?
    One or more permanent consequences are reported in an estimated 50% of overall LCH patients, making long-term follow-up a necessity. Severity and type depends on the affected organs, number of lesions, and the treatment administered. Read more about permanent consequences of LCH.

  15. What is Neurodegeneration?
    Neurodegeneration is progressive loss of brain function. It occurs as a permanent consequence in some cases of LCH.

  16. Can neurodegeneration be prevented/reversed/treated?
    It is not currently known whether neurodegeneration can be prevented. It is believed that neurodegeneration cannot be reversed, and there is controversy whether patients with neurodegeneration can be successfully treated. There have been some promising results with Ara-C but more extensive scientific studies are required.

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  17. What is “PLCH?”
    PLCH (pulmonary Langerhans cell histiocytosis) is LCH of the lung. It affects mostly adults who smoke and often occurs without other LCH involvement.

  18. What kind of physician should we use?
    A pediatric oncologist most often provides primary treatment and coordinates a team of health professionals, which may include, but are not limited to, the primary care physician, pediatric surgeon, radiologist, pediatric nurses, and social workers. Find a Physician in your area today.
  19. What should I look for in a doctor?
    LCH is most often followed and treated by an oncologist, who specializes in cancer-type illnesses. The level of experience with LCH can vary widely among physicians. If he/she is not knowledgeable about this disease, a willingness to learn more and consult with the experts can go a long way. Other qualities to look for are accessibility and good communication skills with you, as well as other physicians.

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  20. Will my child grow normally?
    Most children with LCH do grow normally; it is believed that growth hormone deficiency affects approximately 10% of children with this disease.

  21. What is the treatment for stunted growth related to LCH?
    If stunted growth is due to growth hormone deficiency, which occurs in approximately 10% of LCH patients, it can be treated with daily injections of growth hormone under the supervision of an endocrinologist. The treatment is usually prescribed as long as the child is growing.

  22. Is growth hormone treatment safe?
    Growth hormone replacement appears to be safe and effective in LCH patients and is not associated with an increased risk of disease reactivation.

  23. Are immunizations safe?
    This is a controversial topic. Most researchers believe that children should wait 3-6 months after chemotherapy and/or steroids to take regular vaccinations, especially live-virus vaccinations such as influenza, MMR, and polio. There is no proof that vaccinations trigger LCH. It is important to consult with your physician regarding your child’s particular case.

  24. What type of LCH involvement puts my child at higher risk for developing Diabetes Insipidus?
    CNS risk lesions have been identified as lesions affecting the facial bones or the front or side(s) of the skull. These include the temporal (around the temples), sphenoidal (behind the sinuses), ethmoidal (between the eyes), zygomatic (cheekbone), and orbital (eye socket) bone with tumor extension into the brain. Involvement of these bones increases the risk of developing Diabetes Insipidus, which is the hallmark of CNS disease.

  25. What are the chances my child will develop diabetes insipidus?
    DI occurs in as many as 25% of all patients and as many as 50% of patients with multisystem LCH.

  26. What are risk organs?
    Risk organs include bone marrow, spleen, and liver and are more difficult to treat than other sites of involvement with LCH.

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  27. What happens if my child has a recurrence or does not respond to treatment?
    If disease recurs after treatment, repeat of the same chemotherapy is often used but will depend on which organs are involved and the length of time since previous treatment. If LCH recurs immediately after therapy or does not improve with therapy, alternative treatment such as 2CdA, Ara-C, vincristine, methotrexate, or bisphosphonates may be used. Rare cases of risk-organ disease that is progressive and not responsive to treatment may require a RIC stem-cell transplant. Always consult a specialist!

  28. What is reduced-intensity conditioning (RIC)?
    Reduced-intensity conditioning is a less toxic pre-transplant therapy with the goal of suppressing the patient’s immune system enough so that it will accept donor stem cells while reducing the side effects of high dose chemotherapy The RIC may be used in some HLH patients, as well as some LCH patients with severe, resistant disease.

Possible Side Effects of Treatment


  1. What are the side effects of vinblastine?
    Side effects include:
    • Low blood counts (with higher risk of infection).Mild nausea/vomiting/constipation
    • Easily sunburned
    • Skin irritation at site of injection
    • Thin or brittle hair
    • Fatigue
    • Bone pain
    • Hoarseness
    • Seizures
    • Shortness of breath
    • Nerve damage (especially in adults) with tingling, numbness and/or pain of the hands and feet
  2. What are the side effects of prednisone?
    Side effects include:
    • Increase in blood sugar
    • Increase in appetite
    • Heartburn
    • Bloating/fluid retention/weight gain
    • Difficulty sleeping
    • Mood/behavior/personality changes
    • Higher risk of infection
    • Slow wound healing
    • Muscle weakness
    • Loss of bone calcium
    • Increased hair growth
    More unusual side effects may include:
    • Problems with vision/eye pain
    • Seizures
    • Confusion

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  3. What are the side effects of methotrexate?
    Side effects include:
    • Mouth sores/swollen, tender gums
    • Nausea/vomiting/diarrhea/decreased appetite
    • Low blood counts
    • Dizziness/drowsiness
    • Headache
    More unusual side effects may include:
    • Blurred vision or loss of vision
    • Seizures
    • Confusion
    • Weakness/difficulty moving one or both sides of the body
    • Loss of consciousness
    • Lung damage
    • Allergic reactions
  4. What are the possible side effects of 6-MP (mercaptopurine)?
    More common signs/symptoms include:
    • Low blood counts (red cells, white cells, and clotting cells)
    • Nausea/vomiting/decreased appetite
    • Headache
    • Weakness/fatigue/achiness
    • Rash/darkening of the skin
  5. What are the possible side effects of 2-CdA (cladribine/leustatin)?
    More common signs/symptoms include:
    • Flu-like symptoms (Fever, chills, headache, fatigue, nausea/vomiting)
    • Decreased appetite
    • Constipation
    • Low blood counts (red cells, white cells, and clotting cells)
    • Skin rash/redness/itching

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Histiocytic Disorders and Orphan Diseases

 

  1. What are histiocytic disorders and how are they classified?
    Histiocytic disorders are a diverse group of diseases caused by over-production of white blood cells known as histiocytes, which can lead to organ damage and tumor formation. They include a wide variety of conditions that can affect both children and adults.

    The disorders are classified into three groups based on the types of histiocyte cells involved.
    • The first group is called a dendritic cell disorder, and the most common disease in this group is Langerhans cell histiocytosis. Also included in this group are more rare diseases, Juvenile Xanthogranuloma (JXG) and Erdheim Chester.
    • The second group is called a macrophage cell disorder, and includes primarily hemophagocytic lymphohistiocytosis (HLH) and Rosai-Dorfman.
    • The third group is called malignant histiocytosis and includes certain kinds of leukemia and tumors. 
  2. Why are all of these diseases with different names considered to be related to each other?
    All of the diseases are caused by the over-production of white blood cells called histiocytes. Their different classifications depend on the type of histiocyte involved.

  3. Where can I find reliable information about histiocytosis?
    The Histiocytosis Association’s online community provides a number of informational documents and articles, as well as links to medical articles about the histiocytic disorders. While the Internet does provide a significant volume of information about histiocytic disorders, some of this information is not accurate. It is important to look for documents that are current, are free of grammatical and spelling errors, appear to be objective, are free of advertisements, and clearly state their sources.

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  4. How can I explain histiocytosis to family and friends?
    Histiocytosis is a rare disease that is caused by the over-production of a type of white cell that can lead to organ damage and the formation of tumors. The Histiocytosis Association's Disease Fact Sheets are also a great way to help explain these complicated diseases to family and friends

  5. What is an orphan disease?
    According to the Rare Disease Act of 2002, an orphan disease, also known as a rare disease, affects less than 200,000 persons in the U.S., or less than 1 in 1500 people. The criteria may vary in other countries. For example in Europe, an orphan disease is defined to occur in less than 1 in 2000.

  6. How many orphan diseases are there?
    According to the National Institutes of Health there are approximately 6800 such diseases. Combined, they affect nearly 30 million Americans.

  7. Where can I learn more about rare diseases in general?

    Help ensure that we can continue to bring you this vital educational material, make a donation today.

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