The return of thalidomide - Can birth defects be prevented?

Citation
Jm. Lary et al., The return of thalidomide - Can birth defects be prevented?, DRUG SAFETY, 21(3), 1999, pp. 161-169
Citations number
23
Categorie Soggetti
Pharmacology
Journal title
DRUG SAFETY
ISSN journal
01145916 → ACNP
Volume
21
Issue
3
Year of publication
1999
Pages
161 - 169
Database
ISI
SICI code
0114-5916(199909)21:3<161:TROT-C>2.0.ZU;2-9
Abstract
Thalidomide, the drug that caused a worldwide epidemic of serious birth def ects in the late 1950s and early 1960s, was recently approved by the U-S Fo od and Drug Administration (FDA) for use in treating the skin disease eryth ema nodosum leprosum, a complication of leprosy. The drug has also shown pr omise in the treatment of other serious diseases. If thalidomide is eventua lly approved for use in the US and other countries for treatment of disease s more prevalent than erythema nodosum leprosum, or if use of the drug for non-approved indications becomes widespread, hundreds of thousands of women with childbearing ability could be treated. If this should happen, can we prevent another epidemic of birth defects? In an effort to prevent fetal exposures to thalidomide, the FDA mandated a comprehensive programme to regulate prescription, dispensing and use of the drug. The programme is designed to require registration of all participati ng pre scribers, pharmacies and patients. It also requires use of effective methods of contraception and periodic pregnancy testing of all patients wi th childbearing ability during treatment. Prescribers are directed to couns el both female and male patients on the risks, benefits and proper use of t he drug, as well as on the proper use of contraceptives during treatment. T he patient is required to sign an informed consent form before beginning tr eatment. Prescription and dispensing of thalidomide will be tightly control led. A thalidomide registry will monitor prescription, dispensing and use o f the drug, and will investigate all reported fetal exposures. This mandatory, but untested, programme promises to be effective at prevent ing fetal exposures to thalidomide, provided that patients, prescribers and pharmacists comply with all of its provisions. However, even if the progra mme proves to be successful in the US, there is concern that thalidomide ma y eventually be widely used in countries that may not require such stringen t controls. In Brazil, where thalidomide is commercially available for trea tment of leprosy patients, 33 cases of thalidomide embryopathy have already been reported in the literature. Even in countries that may tightly regula te the distribution and use of thalidomide, some patients may obtain the dr ug through black market sources. Should these events occur, many cases of t halidomide-induced birth defects could appear. There fore, there is a need to develop nonteratogenic analogues of thalidomide that can provide effecti ve treatment for erythema nodosum leprosum and other serious conditions wit hout increasing the potential for another epidemic of thalidomide-related b irth defects.