Sarcoidosis is a rare disease characterized by clumps of inflammatory immune cells, called granulomas, forming in different tissues and organs throughout the body. These can injure the tissue, causing permanent damage, and affect how the organs work.

There is no cure for sarcoidosis, but the symptoms can be managed with various therapies.

Corticosteroids are generally used as a first-line treatment for the disease, but not everyone responds to these. Moreover, their long-term use can have severe side effects. Therefore, other medications, such as cytotoxic and cytostatic compounds, may be prescribed alone or in combination with corticosteroids to treat sarcoidosis.

What are cytotoxic and cytostatic medications?

Cytotoxic and cytostatic medications are commonly used in large doses as cancer treatments due to their ability to either kill cells (cytotoxic) or prevent them from multiplying (cytostatic), which can prevent the growth of tumors.

When used in smaller doses, they can act to suppress the immune system and reduce inflammation, for example, by preventing the activation and growth of inflammatory immune cells.

Since sarcoidosis is caused by an overactive immune system, suppressing it can prevent the cause of the inflammation and potentially prevent the symptoms of the disease.

Types of cytotoxic and cytostatic medications

Methotrexate is the most common cytotoxic medication prescribed to treat sarcoidosis. Imuran (azathioprine) or Arava (leflunomide) have been shown to be effective in sarcoidosis, but are generally only considered when patients are intolerant to methotrexate.

Results of a double-blind, randomized trial of methotrexate in sarcoidosis, published in the journal Sarcoidosis, Vasculitis and Diffuse Lung Diseases, showed that the medicine can be an effective treatment for sarcoidosis and reduce the need for corticosteroid treatment.

Results from a study on leflunomide in sarcoidosis, published in the European Respiratory Journal, demonstrated the effectiveness of the medication in treating lung involvement in sarcoidosis and reducing the dose of corticosteroid medication required to control the disease.

In rare cases where the disease cannot be controlled effectively using methotrexate or Imuran, other cytotoxic medications such as cyclophosphamide may also be considered. However, due to their severe side effects, these are normally not used unless absolutely necessary.

Side effects of cytotoxic and cytostatic medications

Because cytotoxic and cytostatic medications suppress the immune system, patients who are using them are more susceptible to serious infections. Furthermore, these medications are toxic to the liver and can lead to liver disease if the damage is not detected early. It is normally recommended that patients have blood tests to check for infection and signs of liver damage every four to eight weeks.

Common side effects of these medications include dizziness, drowsiness, headache, swollen or tender gums, decreased appetite, reddened eyes, and hair loss.

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