How is LCH treated?

LCH is different to almost all cancers because in some cases it may ‘burn itself out’ without any treatment. We don’t know how or why this happens. In other cases, treatment is needed to get the disease under control and prevent too much damage. Treatment may involve an operation (surgery) and/or medication. The treatment will be tailored to the individual patient and will depend on the extent and sites of their disease.

Main treatment options:

• Smoking
Patients with pulmonary LCH should stop smoking.

• Surgery
The diagnosis of LCH is made by taking a small sample of tissue from an affected part of the body (biopsy). If there is only a single bony lesion, the biopsy is sometimes combined with curettage (scraping out of the abnormal tissue in the lesion). These procedures may initiate a process of healing in the LCH lesion and sometimes this is all that is required.

• Medication
Steroids – corticosteroids are medicines used to reduce inflammation and stop the body’s immune system acting in the wrong way. You may be given prednisolone (a type of steroid) which is usually taken by mouth. Steroids can also be injected into an LCH lesion.

Chemotherapy – this is used to destroy LCH cells and is generally given intravenously. This means that the chemotherapy medicine is diluted in fluid and given straight into a vein (into the bloodstream). Some chemotherapy medicines may be taken by mouth.

Kinase inhibitors – recently drugs have been developed that target mutated molecules such as BRAF V600E. These have been used in some cases of LCH and can cause LCH lesions to shrink or go away. However, when treatment is stopped the disease usually returns.

Your oncologist or haematologist will decide on a treatment plan for you once all the relevant tests are completed. A protocol is a special plan that will detail your treatment if medication is required. There are international treatment protocols, developed over many years through large clinical trials.

The protocol will describe the frequency, timing and length of the different elements of treatment. Your treatment plan may change since it is dependent on how you respond to the treatment. You will be given a copy of your flow sheet so that you can follow progress throughout the treatment. If you need chemotherapy, you will probably need a central line. This is a tube that is inserted into a large blood vessel and tunnelled under the skin to where it can be easily accessed. It allows blood samples to be taken and treatment, including chemotherapy, to be given easily and painlessly. The central line is put in under general anaesthesia and can stay in place until the end of your treatment. Your hospital will provide more information about the types of central lines they offer.

If you require very intensive treatment you will need to be in hospital. Most patients however, receive all their intravenous treatment by visiting their hospital’s out-patient or day-care facility. This may only be available at your treating oncologist/haematologist’s hospital or it may be available at your local hospital under a shared care arrangement with a local doctor. Some treatment, like that taken by mouth, can be given at home.

While on chemotherapy and for up to 6 months afterwards, you will be susceptible to infections. This is known as being immunocompromised. If you develop a fever or become unwell you need to contact your treating team immediately for advice. You may need immediate hospital admission and treatment with antibiotics.

What are the side-effects of the medication?
The main drugs used to treat LCH and their common side effects are described below.

Different drugs cause different side effects. Everyone is different and will react to treatment in a different way. Some patients may have very few side effects while others will have a lot. Almost all side effects are only short-term and will gradually disappear once the treatment has stopped.

• Steroids – Prednisolone/Prednisone – this is a type of corticosteroid (steroid) that can reduce inflammation and suppress the immune system. It is usually given by mouth, in the form of pills to swallow, pills that dissolve in water, or as a liquid medicine.

Possible side-effects include: irritation of the stomach lining (indigestion/ discomfort/pain), increased appetite, weight gain, changes in behaviour (mood swings/difficulty in sleeping/anxiety/irritability), increase in blood sugar level (like someone with diabetes), high blood pressure, increased risk of infection due to suppression of the immune system, impaired wound healing, irregular or absent periods, and inflammation of the pancreas.

• Chemotherapy – Drugs used to treat cancer. These drugs work by killing cells that are in the process of multiplying (by dividing and forming new cells). The main areas of the body that may be affected by chemotherapy are those where cells rapidly divide and grow. Examples include the lining of the mouth causing a sore mouth, the digestive system causing diarrhoea, skin and hair, causing hair loss, and the bone marrow (spongy material that fills the bones and produces new blood cells), causing low blood counts.

There are three main types of blood cells:
• Red blood cells which carry oxygen around the body
• White blood cells which fight infections
• Platelets which help the blood to clot to prevent bleeding and bruising

Chemotherapy reduces the production, and therefore, the number of blood cells in the body. Too few red blood cells cause anaemia, and the person becomes tired and pale. If there are too few white blood cells, particularly ones called neutrophils (neutropenia), the person is at increased risk of infection. Too few platelets means the person is at increased risk of excessive bleeding when injured, having nose-bleeds or bruising easily. If the counts get really low, you may need a red cell transfusion or platelet transfusion.

Only a small number of LCH patients require transfusions, but in some more severe cases, transfusions play an important part in supporting patients. Transfusions are also needed to support those patients who undergo intensive chemotherapy.

As the chemotherapy affects your immune system you may need antibiotics to help fight infections. This means, if you have a temperature of 38°C or more or becomes unwell (even with a normal temperature), you should immediately contact your medical team. You may need tests, antibiotics and admission to hospital for a few days. You may also be asked to avoid using paracetamol and ibuprofen since these drugs lower high temperatures and may mask an infection. In addition, if you are in contact with someone who has chickenpox, shingles or measles, then you should let your hospital know as these are potentially dangerous infections for a patient with a low (suppressed) immune system. Both you and your family should ideally have the annual flu jab during the autumn/winter.

Chemotherapy can cause nausea and vomiting. Anti-sickness medicines, known as anti-emetic drugs, are given to control this side effect.

Hair loss can occur, but this does not always happen with the chemotherapy used to treat LCH. Fortunately, all hair loss from chemotherapy is temporary and hair growth returns once treatment has stopped.

The chemotherapy normally used to treat LCH does not affect fertility, but you should discuss your particular treatment with your oncologist/haematologist for more information.
Chemotherapy can cause increased skin sensitivity to sunlight, therefore avoid intense sunlight and use sun block during treatment.

The two most commonly used chemotherapy drugs for LCH are Vinblastine and 6-Mercaptopurine.

Vinblastine
– is a chemotherapy drug that is injected into your blood stream through the central line. This drug is administered by a ‘push’ rather than a slow drip through.
In addition to the side-effects previously described, Vinblastine can sometimes, though rarely, cause tingling sensations in the hands and feet, hoarseness, constipation, muscle weakness and bone pain. It may also cause damage to the skin if it is injected directly into a vein (rather than through a central line) and then leaks into the skin.

Mercaptopurine
– is a chemotherapy drug taken either as tablets or in a liquid form. It is usually taken at home.
In addition to the other side effects already mentioned, 6-mercaptopurine may affect the liver and this will be monitored by your doctor.
Sometimes other drugs are used to treat LCH, for instance when the above mentioned drugs are not successful or when the disease returns. Should this become necessary, your doctor will discuss the different options with you.

Kinase inhibitors – These have been used in some cases of LCH and can cause LCH lesions to shrink or go away. Treatment with kinase inhibitors has shown promise in patients with neuro-degeneration. However, when treatment is stopped the disease usually returns. 

It is not yet known how long treatment with kinase inhibitors can be safely continued. Side effects include, skin irritation, fatigue, joint pain, raised blood pressure, diarrhoea, nausea and patchy hair loss. In adults skin cancers are common. Kinase inhibitors can be very useful in cases that are difficult to treat.

What happens to patients after they had LCH?
It is important to know that the vast majority of patients will recover completely from LCH. Some patients however, are left with persistent/recurring problems and for a very small number of patients with multi-system LCH, it can be a life-threatening condition. After successfully completing treatment, most patients will have follow-up clinic appointments. LCH sometimes comes back (‘reactivates’) and may need treatment again. If this happens, treatments for LCH that have worked for them before may be effective again. The same or different treatment may then be required. Patients are also monitored for possible permanent consequences of the disease (e.g. a low production of certain hormones) and may need treatment for these late effects.

What are clinical trials?

Clinical trials are an essential step in the development of treatment for various diseases and are necessary for improving the outcome for patients. Clinical trials are the only way to find out if a new treatment approach to a disease is better than the standard treatments currently used. Patients volunteering to take part in a trial are randomly assigned to either receiving the standard treatment or a different or experimental treatment. This is known as a randomised trial and is the most common way in which treatments are compared. This process (randomisation) ensures that the groups are similar in every way other than the fact that they receive different treatments. Any difference in outcome between the groups is then likely to be caused by the difference in the treatment and not by other differences between the groups. If an experimental treatment proves to be better than the standard treatment and does not cause unacceptable side-effects, it is recommended as the new standard treatment for future patients. This is how new treatments or approaches to treatment are introduced to patients.

The conduct of clinical trials is carefully regulated to ensure patients’ well-being is protected. Patients will only be able to participate in a trial if there is an open trial at that time, if they meet the eligibility criteria, and if their treating hospital is taking part. Patients, must understand the implications of taking part in a clinical trial and give written consent (agreement) to allow them to be enrolled on a trial. If a trial is available to you the doctor will explain it in more detail and you will be given the necessary information to allow you to decide whether to take part or not. While on a clinical trial you will be very closely monitored.

Please be advised that all the information you read here is not a replacement for the advice you will get from your consultant and their team.

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