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Phase I trial of sargramostim in pediatric Crohn's disease

Judith R Kelsen1, Joel Rosh, Mel Heyman

  • 1Department of Pediatrics, Children's Hospital of Philadelphia, Philadelphia, Pennsylvania, USA.

Inflammatory Bowel Diseases
|January 7, 2010
PubMed

Insights

Sargramostim (SRG) treatment in children with Crohn's disease (CD) was safe and well-tolerated at both tested doses. Encouraging remission and response rates were observed, warranting further investigation into SRG's efficacy for pediatric CD.

Area of Science:

  • Pediatric gastroenterology
  • Immunomodulatory therapies
  • Clinical pharmacology

Background:

  • Crohn's disease (CD) is a chronic inflammatory bowel disease affecting children.
  • Improving granulocyte function is a potential therapeutic strategy for CD.
  • Sargramostim (SRG), a granulocyte-macrophage colony-stimulating factor, was investigated in pediatric CD.

Purpose of the Study:

  • To evaluate the safety and tolerability of sargramostim (SRG) in children with moderate to severely active Crohn's disease (CD).
  • To determine the pharmacokinetic profile of SRG in pediatric patients.
  • To assess the preliminary efficacy of SRG in inducing remission or response in pediatric CD.

Main Methods:

  • A multicenter, open-label Phase I-2 trial involving 22 pediatric patients (6-16 years) with moderate to severe CD.
  • Patients received sargramostim (SRG) at 4 or 6 microg/kg subcutaneously daily for 8 weeks, with or without corticosteroids.
  • Safety, tolerability, pharmacokinetics (PK), and efficacy (Pediatric CD Activity Index - PCDAI) were assessed.

Main Results:

  • Both 4 and 6 microg/kg doses of sargramostim (SRG) were safe and well-tolerated, with mild injection-site reactions being the most common adverse event.
  • Pharmacokinetic analysis indicated dose proportionality, with no significant impact from corticosteroid co-administration.
  • A high rate of clinical response or remission (88%) was observed, defined by a significant decrease in PCDAI scores.

Conclusions:

  • Sargramostim (SRG) is a safe and tolerable treatment option for pediatric patients with Crohn's disease (CD) at doses of 4 and 6 microg/kg.
  • The observed pharmacokinetic profile supports dose proportionality.
  • While preliminary efficacy data are encouraging, further clinical trials are necessary to confirm the therapeutic benefit of SRG in pediatric CD.
Abstract

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