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Thalidomide as a treatment for inflammatory bowel disease in children and adolescents: A systematic review

Tingting Qiu1,2, Huibo Li1,2, Tong Sun3

  • 1Department of Pharmacy, Peking University Third Hospital, Beijing, China.

Journal of Clinical Pharmacy and Therapeutics
|August 4, 2020
PubMed

Insights

Thalidomide shows promise for treating pediatric inflammatory bowel disease (IBD) refractory to other treatments, improving remission rates. However, peripheral neuropathy is a key risk requiring careful monitoring in children and adolescents.

Area of Science:

  • Pediatric Gastroenterology
  • Pharmacology
  • Immunology

Background:

  • Thalidomide is an off-label treatment for inflammatory bowel disease (IBD).
  • Its use in pediatric populations is not well-established, with contraindications for children due to unknown safety and efficacy.
  • This review focuses on thalidomide's role in pediatric IBD management.

Purpose of the Study:

  • To review the efficacy and safety of thalidomide for treating IBD in children and adolescents.
  • To synthesize current evidence on thalidomide's effectiveness in pediatric IBD patients.

Main Methods:

  • A comprehensive literature search was conducted in databases including PubMed, Embase, and Cochrane Library.
  • Studies included systematic reviews, meta-analyses, randomized controlled trials (RCTs), cohort studies, case-control studies, and case series up to June 2019.
  • Inclusion criteria focused on thalidomide use for IBD in pediatric populations.

Main Results:

  • Seven studies (2 RCTs, 5 case series) involving 134 pediatric patients (32 with ulcerative colitis, 102 with Crohn's disease) were analyzed.
  • Thalidomide demonstrated clinical remission rates of 44%-100% and steroid tapering rates of 50%-100% in refractory pediatric IBD.
  • Peripheral neuropathy was the most frequent serious adverse event, appearing dose-dependent.

Conclusions:

  • Thalidomide can effectively induce and maintain remission in children and adolescents with refractory IBD.
  • Peripheral neuropathy is a significant adverse reaction that necessitates monitoring and preventative strategies.
  • Informed consent and thorough communication with parents are crucial before initiating thalidomide therapy in pediatric patients.
Abstract

Keywords:
adolescentschildreninflammatory bowel diseasethalidomide

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