Leukeran (chlorambucil)  is a chemotherapy agent used to treat certain forms of leukemia. It is used selectively to treat sarcoidosis patients who do not respond to corticosteroids.

How Leukeran works

Sarcoidosis is a chronic disease in which immune cells surround threats, but instead of destroying the threat and dissipating, they remain in clumps called granulomas. Over time, scar tissue can form around these granulomas and interfere with the working of tissues and organs. Sarcoidosis can affect many tissues and organs, including the lungs, nerves, heart, and skin.

When cells duplicate, they make a complete copy of their entire genome (all of the genetic information in the cell). Interfering with the process of copying the genome affects all rapidly duplicating cells, such as immune cells and cancer cells.

Leukeran is a cytotoxic, or cell killing, agent. It works in three ways to reduce cell growth. It chemically modifies the DNA, causing it to be broken down by cellular enzymes as they try to fix the damage. It also chemically connects DNA strands to each other, so they cannot be separated for duplication. Finally, the chemical modifications caused by Leukeran are likely to introduce mutations in the DNA when it is duplicated, as the chemical modification masks the true genetic sequence, leading to cell death.

The combination of these effects results in the slower immune cell growth, which in sarcoidosis patients may reduce the formation of granulomas.

Leukeran in studies of sarcoidosis

While no large-scale clinical trials have evaluated Leukeran in people with sarcoidosis, small numbers of sarcoidosis patients have been treated with the agent with mixed levels of success.

One study, published in the scientific journal Chest in 1980, assessed the effects of treatment in 10 people with progressive pulmonary sarcoidosis. Patients were treated with chlorambucil alone or in combination with corticosteroids. Eight showed some degree of improvement within three months of treatment. Bone marrow suppression, a side effect of the treatment, was observed but not detectable six months after treatment.

Another study, published in the journal Sarcoidosis in 1991, analyzed 31 patients with severe sarcoidosis who did not respond to corticosteroids. A marked improvement was observed in 15 of these patients, while a moderate improvement was recorded in 13 others. However, researchers noted that a six-month course of chlorambucil was often followed by a relapse.

Other information

Although Leukeran is used to treat certain cancers, it can — in rare cases — also cause cancer. Side effects of the medicine include nausea, vomiting, infertility, liver toxicity, and skin rash.

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