What is thalidomide?
Thalidomide was first used in the late 1950's all over the world as a wonder drug that would help stop nausea and insomnia in pregnant women. Soon after, it was discovered to be the cause of severe birth defects in the offspring of women who had taken the drug. Thalidomide was immediately withdrawn worldwide but investigators have continued to see its potential in the treatment of other conditions.
Thalidomide is proving to be useful in the treatment of some severe dermatological conditions and is currently approved by the US Food and Drug Administration (FDA) for the treatment of cutaneous manifestations of moderate to severe erythema nodosum leprosum in leprosy. However, its use is strictly monitored and many precautions are taken to ensure its safe and correct use by both prescriber and patient. In New Zealand thalidomide has not been approved for use for any condition, however special application to the Ministry of Health may allow its use in exceptional circumstances.
How does thalidomide work?
The mechanism of action of thalidomide in the treatment of dermatological conditions is not yet completely understood. It appears that thalidomide prevents the immune system from overreacting to disease and harming the body by reducing the levels of cytokine tumour necrosis factor-alpha (TNF-alpha), a protein in the body that causes inflammatory reactions.
What is thalidomide used for?
Thalidomide is currently being used or investigated as a treatment for the following dermatological conditions:
- Erythema nodosum leprosum
- Prurigo nodularis
- Actinic prurigo
- Cutaneous lupus erythematosus
- Pyoderma gangrenosum
- Behcet disease
- Actinic prurigo
- Erythema multiforme
- Bullous pemphigoid
- Cutaneous sarcoidosis
- Jessner lymphocytic infiltration
- Paraneoplastic dermatomyositis
- IgG4-related skin disease
- Systemic sclerosis
- Severe urticaria
Thalidomide is usually only considered as a treatment in patients with severe disabling conditions where all other treatments have failed.
Thalidomide is used for some types of multiple myeloma, severe beta thalasaemia major and acute lymphoblastic leukaemia.
Side effects of thalidomide
The most well known side effect of thalidomide is its ability to cross the placenta of pregnant women and cause severe birth defects. With this in mind any use of thalidomide is under strict supervision.
Other side effects include drowsiness, rash and constipation. Peripheral neuropathy that may be irreversible has been reported. Early symptoms of nerve damage include numbness, tingling or pain in the hands, arms, legs and feet. Patients should stop treatment and report these symptoms to their doctor immediately.
Important advice to patients
- Pregnant women must not take thalidomide.
- Women of childbearing potential must ensure simultaneous use of 2 reliable forms of contraception for more than one month before, during, and for one month after stopping, therapy.
- Women must have pregnancy tests around and throughout the treatment period.
- A female patient must immediately stop taking thalidomide if she:
- becomes pregnant
- thinks she may be pregnant
- has a late or irregular period
- stops practising abstinence
- stops using birth control.
- Men must abstain from sexual intercourse or use a condom during intercourse while, and for one month after, taking thalidomide (thalidomide is present in semen).
- Men must not donate sperm during treatment.
- All patients must not donate blood during treatment.
- Seek medical advice if numbness, tingling, pain or burning sensation occurs in hands, arms, feet or legs.