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The use of ciclosporin in paediatric inflammatory bowel disease: an Italian experience
A Barabino1, F Torrente, E Castellano
1Pediatric Gastroenterology, G Gaslini Institute, Genoa, Italy. abarab@tin.it
Insights
Ciclosporin showed disappointing long-term efficacy for pediatric inflammatory bowel disease (IBD). However, it helped avoid urgent surgery in severe colitis cases, despite significant side effects like neurotoxicity.
Area of Science:
- Pediatric Gastroenterology
- Immunosuppressive Therapy
Background:
- Inflammatory bowel disease (IBD) presents significant challenges in pediatric patients.
- Refractory IBD cases often require novel therapeutic approaches.
Purpose of the Study:
- To evaluate the efficacy and safety of ciclosporin in children with IBD.
- To assess ciclosporin's role in managing severe, unresponsive, or fistulizing pediatric IBD.
Main Methods:
- Retrospective study of 23 Italian children with IBD treated with ciclosporin.
- Analysis of treatment duration, follow-up, and reasons for drug discontinuation.
- Indications included severe colitis, chronic active colitis, and fistulizing Crohn's disease.
Main Results:
- Ciclosporin was ineffective long-term for 39% of patients requiring surgery and 13% with partial response.
- Clinical remission was achieved in 35% of patients, maintained in 17% after withdrawal.
- Urgent colectomy was avoided in 85% of 14 patients with severe colitis.
- Side effects occurred in 26% of patients, with neurotoxicity leading to discontinuation in 13%.
Conclusions:
- Ciclosporin demonstrates limited long-term effectiveness for refractory pediatric IBD.
- It plays a crucial role in preventing emergent surgery for severe, unresponsive colitis.
- Significant adverse events, including neurotoxicity, necessitate careful monitoring.
Aim:
To asses the efficacy and safety of ciclosporin in a paediatric population with inflammatory bowel disease.
Patients And Methods:
Twenty-three Italian children treated with ciclosporin were studied retrospectively. The indications for treatment were severe unresponsive colitis, chronic active colitis or severe fistulizing Crohn's disease. The treatment duration, follow-up and causes of drug discontinuation were assessed.
Results:
Sixteen patients were treated intravenously for a mean time of 10 +/- 7 days (1-24 days) and 19 orally for a mean time of 133 days (17-660 days). The mean follow-up of all patients was 13.2 months. Ciclosporin was totally ineffective, being discontinued for surgery, in nine of 23 patients (39%); it was discontinued for partial response in three patients (13%). During treatment, clinical remission was achieved in eight children (35%) and maintained after drug withdrawal in four (17%). In severe unresponsive colitis, urgent colectomy was avoided in 12 (85%) of 14 patients who tolerated the drug. Side-effects appeared in six of 23 patients (26%), and three (13%) required ciclosporin to be discontinued due to neurotoxicity.
Conclusions:
Ciclosporin shows disappointing long-term results in the treatment of refractory inflammatory bowel disease, but can play an important role in preventing urgent surgery in unresponsive severe colitis. Severe side-effects can occur.
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