Thalidomide in luminal and fistulizing Crohn's disease resistant to standard therapies


Dr M. A. Kamm, St Mark's Hospital, Watford Road, Harrow HA1 3UJ, UK.


Background  Thalidomide has been shown to be an effective treatment in Crohn's disease.

Aim  To assess the efficacy and tolerability of thalidomide in refractory Crohn's disease patients.

Methods  Twenty-five patients were treated. Retrospective ‘estimated’ Crohn's Disease Activity Indices were assessed at baseline and at the end of follow-up. Clinical response was defined as symptomatic improvement and a reduction in the ‘estimated’ Crohn's Disease Activity Index of >100 points, ≥50% reduction in draining fistulas or clinical improvement in perianal ulcers. Clinical remission was defined as symptom resolution and an ‘estimated’ Crohn's Disease Activity Index <150, complete fistula closure or complete ulcer healing.

Results  Six of eight patients treated for luminal disease responded to thalidomide at a median follow-up of 12 months (three clinical responses, three clinical remissions). The median reduction in ‘estimated’ Crohn's Disease Activity Index was 212 points (P = 0.005). Nine of 11 patients with active fistulizing disease responded to thalidomide (six responses; three remissions). The four patients treated for both luminal and fistulizing disease had fistula response. Three of them had a response in luminal disease activity. One of two patients with ulcerating perianal disease responded. Twelve patients discontinued treatment because of adverse effects (three sedation; two abdominal pain; one leucopoenia; six neuropathy).

Conclusion  Thalidomide is an effective short- to medium-term treatment in selected patients with refractory luminal and fistulizing Crohn's disease. Its long-term use is limited by toxicity.