Placebo-controlled trial of thalidomide in the treatment of severe apthous ulceration in AIDS

OBJECTIVE: To evaluate the safety and efficacy of thalidomide in the treatment of severe aphthous ulceration of the mouth or oesophagus in AIDS patients.

METHODS: Ten males entered a double blind, placebo controlled crossover trial of thalidomide l00mg daily plus Corlan (topical steroid) pellets qid versus placebo plus Corlan pellets. If no improvement at 7 or 14 days, the code was broken and the placebo treated patient given thalidomide. All patients had either oral aphthous ulceration of at least 7 days’ duration, or endoscopic documentation of oesophageal ulceration (not due to HSV, CMV, Candida sp. or MAC).

RESULTS: Six patients with oral ulcers for 10 days-3mths, 3 patients with oesophageal ulceration of 1-6mths duration and 1 patient with both oral and oesophageal ulceration present for 2mths entered the study. Of 5 who received placebo topical steroid initially, 3 had no improvement and crossed over to receive thalidomide, 1 had slow healing of mouth ulcers over 4 weeks and 1 patient with CD4 cells of 350/ul had complete resolution (CR) in 3 days. Of 8 who received thalidomide (5 initially and 3 crossover) 4 had CR of oral ulcers in 6-9 days, 4 had CR of symptoms from oesophageal ulcers in 14-28 days and 1 had no improvement in 8 days and stopped drug due to side effects. Another patient developed a rash after resolution of symptoms. Eight of the 10 patients had CD4 cells <50/ul. Five of 7 patients who responded to thalidomide gained 2.4 to 4kg in weight.

CONCLUSION: Thalidomide l00mg daily is effective in the treatment of severe aphthous ulceration of oral or oesophageal mucosa in patients with advanced AIDS.