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Efficacy and safety of thalidomide in children and young adults with intractable inflammatory bowel disease: long‐term results
Author(s) -
LAZZERINI M.,
MARTELOSSI S.,
MARCHETTI F.,
SCABAR A.,
BRADASCHIA F.,
RONFANI L.,
VENTURA A.
Publication year - 2007
Publication title -
alimentary pharmacology and therapeutics
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 3.308
H-Index - 177
eISSN - 1365-2036
pISSN - 0269-2813
DOI - 10.1111/j.1365-2036.2006.03211.x
Subject(s) - medicine , thalidomide , ulcerative colitis , inflammatory bowel disease , gastroenterology , adverse effect , refractory (planetary science) , surgery , crohn's disease , disease , multiple myeloma , physics , astrobiology
Summary Background 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. Aim To investigate the long‐term efficacy and safety of thalidomide in a group of children and young adults with refractory inflammatory bowel disease. Methods 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. Results 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). Conclusions 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.