including drug-induced lupus, autoimmune hepatitis, polyarteritis
nodosa, and polyarthritis.45 Tetracyclines should not be
used in pregnant women or children less than 9 years of age
because of effects on bone and cartilage.
Thalidomide
In the early 1950s thalidomide was marketed as an antiepileptic
agent and sedative prior to being rebranded as an antiemetic
for use during pregnancy. By 1962 it became clear that this substance
possessed significant teratogenic properties when used
during the first trimester, causing severe anomalies such as
phocomelia and long bone defects. General use of thalidomide
ceased in the early 1960s, by which point it had caused an estimated
12,000 birth defects, mainly in Germany. However, in
1965 thalidomide was found to be effective for erythema nodosum
leprum, and was eventually approved by the FDA for that
purpose in 1998. In 2006 it was approved for use in multiple
myeloma. The orphan drug status of thalidomide has facilitated
its innovative application in many dermatologic conditions.46,47
Thalidomide’s mechanism of action is not yet fully understood,
but it acts through diverse sedative, immunomodulatory, antiinflammatory,
and anti-angiogenic effects.46,47 Following failure
of standard therapy, thalidomide has been used experimentally in numerous dermatologic conditions, found to be very effective
in aphthous stomatitis,48 Behçet’s syndrome,49 cutaneous lupus
erythematosus,50 and prurigo nodularis.51 It is moderately effective
for actinic prurigo,52 adult Langerhans cell histiocytosis,53
cutaneous sarcoidosis,54 erythema multiforme,55 graft-versushost
disease,56 Jessner lymphocytic infiltrate of the skin,57 and
uremic pruritus.58 These are the conditions for which evidence
advocating use of thalidomide is strongest; the drug has been
piloted in many others, with less convincing results.47
Obtaining thalidomide in the United States requires participation
in the System for Thalidomide Education and Prescribing
Safety (S.T.E.P.S.) program in order to prevent fetal drug exposure.
Apart from teratogenesis, thalidomide is commonly
associated with sedation, dizziness, peripheral neuropathy,
thromboembolism, constipation, and skin reactions.47,59
Conclusion
As these examples illustrate, by utilizing off-label prescribing
dermatology has discovered innovative means of treating
the myriad disease entities that fall within its scope of practice.
Off-label prescribing simultaneously avoids the time
and expense of running clinical trials for oftentimes rare diseases
while offering patients expedient, effective therapeutic
