Efficacy and safety of thalidomide in children and young adults with intractable inflammatory bowel disease: long-term results
Article first published online: 31 JAN 2007
Alimentary Pharmacology & Therapeutics
Volume 25, Issue 4, pages 419–427, February 2007
How to Cite
LAZZERINI, M., MARTELOSSI, S., MARCHETTI, F., SCABAR, A., BRADASCHIA, F., RONFANI, L. and VENTURA, A. (2007), Efficacy and safety of thalidomide in children and young adults with intractable inflammatory bowel disease: long-term results. Alimentary Pharmacology & Therapeutics, 25: 419–427. doi: 10.1111/j.1365-2036.2006.03211.x
- Issue published online: 31 JAN 2007
- Article first published online: 31 JAN 2007
- Publication data Submitted 24 May 2006 First decision 13 June 2006 Resubmitted 30 October 2006 Resubmitted 22 November 2006 Accepted 23 November 2006
Anti-tumour necrosis factor-α antibodies are useful for the treatment of refractory Crohn's disease and ulcerative colitis. Thalidomide is another agent with tumour necrosis factor-α suppressive properties.
To investigate the long-term efficacy and safety of thalidomide in a group of children and young adults with refractory inflammatory bowel disease.
Twenty-eight patients with refractory moderate-severe inflammatory bowel disease (19 Crohn's disease, 9 ulcerative colitis) received thalidomide 1.5–2.5 mg/kg/day. Patients were assessed at baseline, at weeks 2, 4, 8 and 12, and then every 12 weeks by patient's diary, physical examinations, laboratory analyses and scoring on activity indexes. Primary outcomes were: (i) efficacy in inducing remission; and (ii) efficacy in maintaining remission.
Remission was achieved with thalidomide in 21 of 28 (75%) patients (17 with Crohn's disease, 4 with ulcerative colitis). Mean duration of remission was 34.5 months. Sixteen of 20 (80%) patients suspended steroids. Reversible neuropathy occurred in seven of 28 (25%) patients, but only with cumulative doses over 28 g. Other side effects requiring thalidomide suspension were vertigo/somnolence (one of 28), and agitation/hallucinations (one of 28).
Thalidomide seems to be effective in inducing long-term remission in children and adolescents with intractable inflammatory bowel disease. Neuropathy is the main adverse effect, but appears to be cumulative dose-dependent, thus allowing long-term remission before drug suspension.