Cyclophosphamide (brand names, Cytoxan and Neosar) is a chemotherapy approved to treat a number of cancers, and may be used to treat select sarcoidosis patients because of its ability to suppress the immune system.

Specifically, cyclophosphamide is generally reserved for people with severe disease, such as neurosarcoidosis and cardiac sarcoidosis. According to the sarcoidosis treatment guidelines of the Foundation for Sarcoidosis Research (FSR), the use of cyclophosphamide should be reserved for patients whose severe disease cannot be controlled by medications such as methotrexate or azathioprine.

Sarcoidosis is characterized by the formation of clumps of inflammatory immune cells, called granulomas, in different tissues and organs, affecting their ability to function.

How cyclophosphamide works

Cyclophosphamide belongs to a group of medications called alkylating agents. They work by binding to DNA and blocking it from being “read,” so as to prevent protein synthesis. Cells that cannot synthesize proteins die — a benefit when these cells are cancerous.

Cyclophosphamide can also suppress the activity of immune system cells, which in the case of an overactive immune system disease, such as sarcoidosis, can lead to a lowering of the levels of reactive immune cells and help reduce inflammation.

Cyclophosphamide in clinical trials for sarcoidosis

No large-scale, controlled clinical trials have investigated the potential benefits of cyclophosphamide in patients with sarcoidosis. However, a report of a patient with cardiac sarcoidosis, published in the CHEST journal in 1998, reported that cyclophosphamide treatment was highly effective in suppressing the disease over a period of six years.

Another study, published in 2003, examined a short-course, pulse-dose regimen of cyclophosphamide used to treat seven neurosarcoidosis patients who were either not responsive to corticosteroids or did not tolerate high-doses of corticosteroids. They were treated for a mean of 5.4 months.

Of the seven patients, four reported an easing of symptoms while on the therapy, and all seven patients demonstrated improvements in MRI or cerebrospinal fluid markers, leading researchers to conclude that “cyclophosphamide appears to be a reasonable, steroid-sparing treatment option for patients with corticosteroid-refractory neurosarcoidosis.” These results were also published in CHEST.

Other details

Cyclophosphamide is approved by the U.S. Food and Drug Administration (FDA) to treat several types of cancer and specific types of kidney disease in children.

The most common side effects associated with cyclophosphamide are a general weakness, stomach discomfort or pain, diarrhea, nausea or vomiting, and low white blood cell counts.

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