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Efficacy and Tolerance of Thalidomide in Patients With Very Early Onset Inflammatory Bowel Disease

Matteo Bramuzzo1, Fabiola Giudici2, Serena Arrigo3

  • 1Institute for Maternal and Child Health, IRCCS Burlo Garofolo, Trieste, Italy.

Inflammatory Bowel Diseases
|February 17, 2023
PubMed

Insights

Thalidomide shows efficacy in very early onset inflammatory bowel disease (VEOIBD) but with higher discontinuation rates due to lack of efficacy. Adverse events were less frequent in VEOIBD patients compared to pediatric-onset IBD (pIBD).

Area of Science:

  • Pediatric Gastroenterology
  • Inflammatory Bowel Disease Research
  • Pharmacological Efficacy Studies

Background:

  • Limited therapeutic options exist for very early onset inflammatory bowel disease (VEOIBD).
  • Thalidomide's efficacy and safety profile in VEOIBD remain under-investigated.
  • Comparison with pediatric-onset IBD (pIBD) is crucial for understanding disease-specific responses.

Purpose of the Study:

  • To evaluate the efficacy and tolerance of thalidomide in pediatric patients diagnosed with VEOIBD.
  • To compare treatment outcomes, including persistence, remission, mucosal healing, and adverse events, between VEOIBD and pIBD cohorts.
  • To identify reasons for drug discontinuation in both VEOIBD and pIBD patient groups.

Main Methods:

  • Retrospective cohort study utilizing propensity score 1:1 matching for patient selection.
  • Analysis of treatment persistence, drug withdrawal causes, clinical remission, and mucosal healing rates at 1, 2, and 3 years.
  • Assessment and comparison of adverse events (AEs) between VEOIBD and pIBD patients receiving thalidomide.

Main Results:

  • Treatment persistence rates at 1, 2, and 3 years were 68.2%, 57.0%, and 50.9% for VEOIBD, and 81.7%, 60.0%, and 33.0% for pIBD, respectively (P = .12).
  • VEOIBD patients showed a significantly higher rate of discontinuation due to lack of efficacy (48.2% vs 17.2%; P = .03).
  • Clinical remission and mucosal healing rates were comparable, with significantly fewer AEs observed in VEOIBD patients (35.9% vs 76.9%; P = .0005).

Conclusions:

  • Thalidomide demonstrates effectiveness and tolerability in children with VEOIBD.
  • While discontinuation due to lack of efficacy is more common in VEOIBD, adverse events are less frequent compared to pIBD.
  • Thalidomide represents a viable therapeutic option for VEOIBD, warranting further investigation into optimizing its use.
Abstract

Keywords:
childreninflammatory bowel diseasethalidomidetherapyvery early onset

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