In the 1960s the drug thalidomide was used by some pregnant women in Europe and Canada to treat morning sickness. Women who took the drug in early pregnancy gave birth to children with severe birth defects such as missing or shortened limbs. More than 10,000 children around the world were born with major malformations, many missing arms and legs, because their mothers had taken the drug during early pregnancy. Mothers who had taken the drug when arms and legs were beginning to form had babies with a widely varying but recognizable pattern of limb deformities.
The most well-known pattern, absence of most of the arm with the hands extending flipper-like from the shoulders, is called phocomelia. Another frequent arm malformation called radial aplasia was absence of the thumb and the adjoining bone in the lower arm. Similar limb malformations occurred in the lower extremities. The affected babies almost always had both sides affected and often had both the arms and the legs malformed. In addition to the limbs, the drug caused malformations of the eyes and ears, heart, genitals, kidneys, digestive tract, and nervous system.
Thalidomide was recognized as a powerful human teratogen (a drug or other agent that causes abnormal development in the embryo or fetus). Taking even a single dose of thalidomide during early pregnancy may cause major birth defects. At that time, it was not approved in the United States because Dr. Frances Kelsey from the Food and Drug Administration (FDA) stated there was insufficient proof of the drug’s safety in humans. Shortly after the birth defects were observed, thalidomide was banned worldwide In 1998 the Food and Drug Administration (FDA) approved thalidomide for use in treating leprosy symptoms.
Studies are also being conducted to determine the effectiveness of thalidomide in treating symptoms associated with AIDS, Behchet disease, lupus, Sjogren syndrome, rheumatoid arthritis, inflammatory bowel disease, macular degeneration, and some cancers To prevent fetal exposure to thalidomide, the drug’s manufacturer has developed the System for Thalidomide Education and Prescribing Safety (STEPS) program. Only physicians who are registered may prescribe thalidomide to patients and those patients both male and female must comply with mandatory contraceptive measures, patient registration and patient surveys.
Thalidomide may be dispensed only by licensed pharmacists who are registered in the S. T. E. P. S. program and have been educated to understand the risk of severe birth defects if thalidomide is used during pregnancy. In addition, female patients’ prescriptions will not be filled without a physician’s written report of a negative pregnancy test that has been conducted within 24 hours of starting thalidomide therapy. Pregnancy testing will continue to be required weekly during the first month of use, then monthly thereafter in women with regular cycles, or every two weeks if cycles are irregular. Prescriptions are only for one month’s supply.
A female patient must abstain from sexual intercourse or use two highly effective methods of birth control at the same time for at least one month before receiving thalidomide and continue their use until one month after the last thalidomide dose. All patients must participate in a mandatory registry that will provide follow-up to detect any adverse effects of using thalidomide and will hopefully identify areas in which safeguards need to be improved, if problems occur. The FDA reminds female patients that no method of birth control is completely reliable, except for having no sexual intercourse at all.
Women being treated with thalidomide should refrain from any other activities that could result in pregnancy, such as assisted fertilization methods. Breast feeding a baby and donating blood are also prohibited. Male patients must abstain from sexual intercourse or use a condom during intercourse while taking thalidomide and for one month after, because it is not known if thalidomide in semen is a hazard. Donating sperm or blood is also prohibited. It is vital that each patient understands that thalidomide is prescribed for him or her alone and must not be shared with, or given to, others.