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Thalidomide in Gastrointestinal Disorders

  • Therapy in Practice
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Abstract

Thalidomide was originally marketed as a sedative, but was removed from the market in 1961 after it was associated with an epidemic of severe birth defects. Subsequently, it has been shown to have therapeutic efficacy in a number of the gastrointestinal tract conditions characterised by immune dysregulation. The exact mechanism of the immunosuppressive effects of thalidomide is unknown; proposed mechanisms include inhibition of tumour necrosis factor alpha release and inhibition of angiogenesis.

In chronic graft versus host disease, use of high dose thalidomide (1200 mg/day) may bring about a response in 20% of patients with refractory disease. Thalidomide 200 mg/day helps eradicate ulcers in 50% of patients with HIV-associated oral aphthous ulceration. In Behçet’s disease, thalidomide 100 to 300 mg/day can decrease the number of mucocutaneous ulcers, although full remission occurs in less than 20% of patients. In Crohn’s disease, thalidomide 50 to 300 mg/day may decrease the severity of mucosal disease and prompt closure of fistulae.

Patients to be placed on thalidomide therapy must practice either abstinence or strict birth control; women must undergo regular pregnancy testing and utilise 2 forms of contraception. Other adverse effects include sedation (present in nearly all patients), symptomatic neuropathy (present in approximately 20%), and skin rashes. Given the potential toxicity, thalidomide use should generally be limited to clinical protocols with institutional review board oversight.

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Acknowledgements

Dr Bousvaros was supported in part by a research grant from Crohn’s and Colitis Foundation of America.

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Correspondence to Athos Bousvaros.

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Bousvaros, A., Mueller, B. Thalidomide in Gastrointestinal Disorders. Drugs 61, 777–787 (2001). https://doi.org/10.2165/00003495-200161060-00006

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