Thalidomide

Thalidomide, sold under the brand name Thalomid, among others, is an immunomodulatory drug and the prototype of the thalidomide class of drugs. Today, thalidomide is used mainly as a treatment of certain cancers (multiple myeloma) and of a complication of leprosy. Thalidomide, sold under the brand name Thalomid, among others, is an immunomodulatory drug and the prototype of the thalidomide class of drugs. Today, thalidomide is used mainly as a treatment of certain cancers (multiple myeloma) and of a complication of leprosy. Thalidomide was first marketed in 1957 in West Germany under the trade name Contergan. The German drug company Chemie Grünenthal developed and sold the drug. Primarily prescribed as a sedative or hypnotic, thalidomide also claimed to cure 'anxiety, insomnia, gastritis, and tension'. Afterwards, it was used to treat nausea and to alleviate morning sickness in pregnant women. Thalidomide became an over-the-counter drug in West Germany on October 1, 1957. Shortly after the drug was sold in West Germany, between 5,000 and 7,000 infants were born with phocomelia (malformation of the limbs). Only 40% of these children survived. Throughout the world, about 10,000 cases were reported of infants with phocomelia due to thalidomide; only 50% of the 10,000 survived. Those exposed to thalidomide in utero developed limb deformities where either limbs were not developed or presented themselves as stumps. Other defects included malformed eyes and hearts, deformed gastrointestinal and urinary tracts, blindness and deafness. The negative effects of thalidomide led to the development of more structured drug regulations and control over drug use and development. Thalidomide is used as a first-line treatment in multiple myeloma in combination with dexamethasone or with melphalan and prednisone, to treat acute episodes of erythema nodosum leprosum, and for maintenance therapy. Thalidomide is used off-label in several conditions. The bacterium that causes tuberculosis (TB) is related to leprosy. Thalidomide may be helpful in some cases where standard TB drugs and corticosteroids are not sufficient to resolve severe inflammation in the brain. It is used as a second-line treatment to manage graft versus host disease and aphthous stomatitis in children and has been prescribed for other conditions in children including actinic prurigo and epidermolysis bullosa; the evidence for these uses is weak. It is recommended only as a third line treatment in graft versus host disease in adults, based on lack of efficacy and side effects observed in clinical trials. Thalidomide should not be used by people who are breastfeeding or pregnant, trying or able to conceive a child, or cannot or will not follow the risk management program to prevent pregnancies. The prescribing doctor is required to ensure that contraception is being used, and regular pregnancy tests are taken. Some people are allergic to thalidomide and should not take it. It should be used with caution in people with chronic infections like HIV or hepatitis B. Thalidomide causes birth defects. The U.S. Food and Drug Administration (FDA) and other regulatory agencies have approved marketing of the drug only with an auditable risk evaluation and mitigation strategy that ensures that people using the drug are aware of the risks and avoid pregnancy; this applies to both men and women, as the drug can be transmitted in semen. There is a high risk that thalidomide can cause excessive blood clots. There is also a high risk that thalidomide can interfere with formation of various kinds of new blood cells, creating a risk of infection via neutropenia, leukopenia, and lymphopenia, and risks that blood will not clot via thrombocytopenia. There is also a risk of anemia via lack of red blood cells. The drug can also damage nerves, causing peripheral neuropathy that may be irreversible.