Positioning Biologic Therapies in the Management of Pediatric Inflammatory Bowel Disease
Jessica Breton1, Arthur Kastl1, Maire A Conrad1
1Division of Gastroenterology, Hepatology, and Nutrition, Children's Hospital of Philadelphia; Department of Pediatrics, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, Pennsylvania.
Insights
Pediatric inflammatory bowel disease (IBD) is increasing globally, especially in young children. Biologic therapies, including anti-tumor necrosis factor (TNF) agents, are vital for treating pediatric IBD, but challenges remain.
Area of Science:
- Pediatric Gastroenterology
- Immunology
- Pharmacology
Background:
- Pediatric inflammatory bowel disease (IBD) incidence and prevalence are rising globally, with a notable increase in children under five.
- Pediatric IBD often presents with a more severe phenotype, including growth impairment, compared to adult IBD.
- Current treatment aims include intestinal healing, optimal growth, and improved quality of life while minimizing drug toxicity.
Purpose of the Study:
- To summarize evidence for biologic agents in pediatric IBD treatment.
- To discuss challenges and barriers in pediatric drug development for IBD.
Main Methods:
- Review of current literature on biologic therapies for pediatric IBD.
- Analysis of treatment goals and outcomes in pediatric IBD patients.
- Discussion of challenges in pediatric drug development and approval.
Main Results:
- Anti-tumor necrosis factor (TNF) α agents have improved treatment potential for pediatric IBD over the last two decades.
- Approximately one-third of patients experience nonresponse or loss of response to anti-TNFα therapies.
- Novel biologic therapies offer alternatives, but their pediatric use is limited by delayed approvals.
Conclusions:
- Biologic agents, particularly anti-TNFα therapies, are crucial in managing pediatric IBD.
- Addressing nonresponse and delayed approvals for novel biologics is essential for improving pediatric IBD care.
- Further research and streamlined development pathways are needed for pediatric-specific IBD treatments.
Abstract:
The incidence and prevalence of pediatric inflammatory bowel disease (IBD) are rising worldwide, with a steep increase in children under 5 years of age. Compared to adult IBD, pediatric IBD presents with a more severe, aggressive phenotype and unique complications, notably growth impairment. Treatment goals include achieving intestinal healing, reaching growth potential, and optimizing quality of life, all while limiting drug toxicities. In the last 2 decades, the advent of anti-tumor necrosis factor (TNF) α agents has significantly increased the potential to reach these goals. However, nonresponse or loss of response to anti- TNFα agents is still encountered in approximately one-third of patients. Although the development of novel biologic therapies has offered new alternatives in recent years, the use of these therapies in the pediatric setting has been limited due to delayed approval. This article summarizes the key evidence for biologic agents currently used in the treatment of pediatric IBD and discusses challenges and barriers unique to pediatric drug development.
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