Thalidomide in severe and resistant orogenital ulcers in Behçet Disease – a group of case reports

Abstract

Thalidomide was commercialized in 1956 to be used as a sedative
and anti-emetic drug, but was rapidly removed from the market after
it had been linked to cases of severe birth defects. Nowadays the only
approved FDA indications for the use of Thalidomide are in Multiple
Myeloma and Erithema Nodosum Leprosum. The anti-inflammatory,
anti-angiogenic and anti-TNF alpha effect of the drug has led to
continued interest and research about Thalidomide’s immunomodulatory activities. There are several clinical studies and many case reports of Thalidomide treatment for Behçet Disease (BD), with rapid
healing of the orogenital ulcers while patients are on Thalidomide
but with relapse after its discontinuation. Despite possible benefits,
Thalidomide is difficult to obtain, available as an investigational agent
only (in most countries), carries a significant risk of neuropathy, and
is generally reserved for resistant disease in patients who will not
become pregnant. So, the use of Thalidomide in the management
of BD is restricted to experienced physicians.
We report a group of seven patients with BD with painful oral
aphthae who experienced rapid (2 to 4 weeks) complete and sustained improvement with Thalidomide after a prolonged but ineffective treatment with steroids and/or colchicine. No major side-effect were
experienced (somnolence in two). Three patients who relapsed after
withdrawal of Thalidomide, needed maintenance therapy at a lower
dose, to prevent relapse.
In our group of 7 patients we confirm the benefit of Thalidomide
in recurrent mucocutaneous aphthae and BD resistant to classic
drugs, without major side-effects. The effectiveness of Thalidomide
is present even with low dosages, but treatment must be continuous
because new lesions usually appear a few weeks after the end of treatment.