Thalomid (thalidomide) is a treatment approved by the U.S. Food and Drug Administration (FDA) and usually used in combination with other medicines to treat multiple myeloma, a cancer of the white blood cells, and alone or in combination to treat a form of leprosy.

Thalidomide may be used to treat the symptoms of sarcoidosis, an inflammatory disease in which the immune system responds to a threat such as an infection by forming clumps of immune cells called granulomas throughout the body. These granulomas can interfere with the function of tissues and organs, and over time, lead to scarring. Sarcoidosis can affect the skin and the lungs, as well as other tissues and organs.

How Thalomid works

Thalomid works by inhibiting a pro-inflammatory (or immune system activating) protein called tumor necrosis factor-alpha (TNF-alpha). Cells affected or damaged by an infection make TNF-alpha to send a signal to the immune system to attack the invader.

In sarcoidosis, the immune system can be activated too strongly, or activated by the wrong things. By suppressing TNF-alpha, Thalomid may reduce the immune response, though the exact mechanism by which it inhibits TNF-alpha is not known.

Thalomid in clinical trials

Several small studies have examined the efficacy of Thalomid in treating cutaneous or pulmonary sarcoidosis. Some of these studies produced positive results while others showed no benefit associated with Thalomid treatment in cutaneous or pulmonary sarcoidosis.

One study, published in the scientific journal Chest treated 15 patients with chronic cutaneous sarcoidosis, who were unresponsive to previous therapies, with increasing doses of thalidomide until there was an improvement in their skin lesions, as assessed by the treating physician and a blinded panel.

Five patients improved after one month of receiving 50 mg of thalidomide per day, and seven patients after an additional month on 100 mg of thalidomide daily.  Two patients needed a third month of daily thalidomide at 200 mg. One patient dropped out of the study for “probably unrelated” reasons. Side effects reported were tiredness seen in nine patients, dizziness (two patients), constipation (five patients), rash and and shortness of breath (both reported in one patient).

A study published in the Journal of the American Academy of Dermatology retrospectively evaluated the effectiveness of 100 or 200 mg of thalidomide each day in 12 patients with cutaneous sarcoidosis. Results showed that the patients’ lesions became smaller within five months of treatment, with an average improvement time of two to three months in 10 patients. Four patients achieved a complete response where their lesions healed, and five showed a partial response with their lesions reducing in size or severity but still being present. Two patients showed no response to treatment and one patient deep vein thrombosis.

A Phase 3 randomized, double-blind, parallel, placebo-controlled, multi-center clinical trial (NCT00305552) was conducted in 39 patients with cutaneous sarcoidosis. During the first three months of the trial, patients received daily either one dose of 100 mg of thalidomide or placebo. From months three to six, all patients were given either 100 or 200 mg of thalidomide each day. The size of the patients’ lesions was assessed at the beginning, middle, and end of the study.

Results published in the journal Chest in 2014 showed that 20 percent of patients on thalidomide and 21 percent of patients on placebo saw partial improvements in lesions, but no patients saw a complete improvement. Four patients dropped out of the study in the first three months, and five out in the second three months because of side effects.

A small study in 10 patients with pulmonary sarcoidosis was published in the journal Sarcoidosis, Vasculitis, and Diffuse Lung Diseases in 2006. In this study, patients were treated with 200 mg per day of thalidomide for 24 weeks in combination with corticosteroids. No improvement in difficulty breathing was observed, as measured by a patient questionnaire. In addition, nine out of ten patients needed to reduce the amount of thalidomide they were taking due to side effects like tiredness, constipation, and peripheral neuropathy, or damage to the nerves in hands and feet. “Thalidomide does not significantly improve pulmonary function or quality of life in patients with corticosteroid-dependent sarcoidosis,”  the study concluded.

Additional information

Thalomid has several side effects, including drowsiness, dizziness, rash, and slowed heartbeat. Some patients may also develop peripheral neuropathy.

Thalomid causes severe birth defects, so women who are pregnant or trying to become pregnant or who are nursing should not use this treatment.

Like other immunosuppressant treatments, thalidomide can make patients more susceptible to infections.

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