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The Future of Advanced Therapies for Pediatric Crohn's Disease
Julie Gallagher1, Joel R Rosh1, Benjamin Sahn2,3
1Division of Pediatric Gastroenterology, Liver Diseases, and Nutrition, Steven and Alexandra Cohen Children's Medical Center, Northwell Health, 1991 Marcus Ave, Suite M100, New Hyde Park, NY, 11042, USA.
Insights
Pediatric Crohn's disease requires effective treatments for intestinal inflammation. Advanced therapies offer new options, but careful selection is crucial for achieving remission in children.
Area of Science:
- Pediatric Gastroenterology
- Immunology
- Pharmacology
Background:
- Pediatric Crohn's disease often involves severe intestinal inflammation, risking complications if remission is not achieved.
- Anti-tumor necrosis factor (TNF) therapies can induce remission, but many children lack response or lose it over time.
- Some children may benefit from alternative strategies avoiding broad immunosuppression.
Purpose of the Study:
- To review current and emerging advanced therapies for pediatric Crohn's disease.
- To discuss the challenges and data guiding treatment selection in children.
- To highlight the importance of optimizing disease control in pediatric Crohn's disease.
Main Methods:
- Review of current literature on advanced biologic and small-molecule therapies.
- Analysis of data extrapolated from adult trials and pediatric studies.
- Discussion of real-world evidence for treatment optimization.
Main Results:
- Newer advanced therapies (anti-integrin, anti-interleukin, small molecules) offer targeted mechanisms with reduced immunogenicity.
- Limited regulatory approvals for these agents in children necessitate reliance on adult data and real-world experience.
- Effective utilization of these advanced therapies is key for improved disease control.
Conclusions:
- Advanced therapies are crucial for managing pediatric Crohn's disease, especially for those unresponsive to anti-TNF agents.
- Careful consideration of efficacy, safety, and available data is paramount for treatment selection in children.
- Ongoing research and real-world data are vital for optimizing the use of novel therapies in pediatric Crohn's disease.
Abstract:
Pediatric Crohn's disease commonly presents with moderate-to-severe intestinal inflammation with a greater risk of complications if remission is not achieved. Anti-tumor necrosis factor therapies have offered the possibility of deep and durable remission; however, many children do not respond or no longer respond over time. Further, some children do not require broader systemic immunosuppression to achieve remission and are better served by an alternative treatment strategy. Proper utilization of advanced biologic and small-molecule therapies, which have become available for adult patients since anti-tumor necrosis factor medications, is paramount for tighter disease control for a large proportion of children. Newer advanced therapies such as anti-integrin and anti-interleukin biologics, and several small-molecule agents capitalize on various mechanisms through narrower immunologic targets and reduced immunogenicity. Given limited regulatory approvals of these agents for use in children with Crohn's disease, clinicians continue to rely on data extrapolated from clinical trials in adult patients, sparse pediatric studies, and a growing real-world experience for treatment selection and optimization. In this article, we discuss currently available treatment options, pipeline drugs, and relevant data as they pertain to some of the most pressing clinical challenges faced in treating pediatric Crohn's disease.
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