儿童克罗恩病先进疗法的未来

IF 3.4 3区 医学 Q1 PEDIATRICS
Pediatric Drugs Pub Date : 2023-11-01 Epub Date: 2023-08-23 DOI:10.1007/s40272-023-00590-x
Julie Gallagher, Joel R Rosh, Benjamin Sahn
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引用次数: 0

摘要

儿童克罗恩病通常表现为中度至重度肠道炎症,如果不能缓解,并发症的风险更大。抗肿瘤坏死因子疗法提供了深度和持久缓解的可能性;然而,随着时间的推移,许多孩子没有反应或不再有反应。此外,一些儿童不需要更广泛的全身免疫抑制来获得缓解,并且可以通过替代治疗策略获得更好的服务。自抗肿瘤坏死因子药物问世以来,成人患者可以使用先进的生物和小分子疗法,正确利用这些疗法对于更严格地控制大部分儿童的疾病至关重要。新的先进疗法,如抗整合素和抗白细胞介素生物制剂,以及几种小分子制剂,通过更窄的免疫靶点和降低免疫原性,利用了各种机制。鉴于这些药物用于儿童克罗恩病的监管批准有限,临床医生继续依赖从成年患者临床试验、稀疏的儿科研究以及日益增长的治疗选择和优化的现实经验中推断出的数据。在这篇文章中,我们讨论了目前可用的治疗方案、管道药物和相关数据,因为它们与治疗儿童克罗恩病面临的一些最紧迫的临床挑战有关。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
The Future of Advanced Therapies for Pediatric Crohn's Disease.

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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来源期刊
Pediatric Drugs
Pediatric Drugs PEDIATRICS-PHARMACOLOGY & PHARMACY
CiteScore
7.20
自引率
0.00%
发文量
54
审稿时长
>12 weeks
期刊介绍: Pediatric Drugs promotes the optimization and advancement of all aspects of pharmacotherapy for healthcare professionals interested in pediatric drug therapy (including vaccines). The program of review and original research articles provides healthcare decision makers with clinically applicable knowledge on issues relevant to drug therapy in all areas of neonatology and the care of children and adolescents. The Journal includes: -overviews of contentious or emerging issues. -comprehensive narrative reviews of topics relating to the effective and safe management of drug therapy through all stages of pediatric development. -practical reviews covering optimum drug management of specific clinical situations. -systematic reviews that collate empirical evidence to answer a specific research question, using explicit, systematic methods as outlined by the PRISMA statement. -Adis Drug Reviews of the properties and place in therapy of both newer and established drugs in the pediatric population. -original research articles reporting the results of well-designed studies with a strong link to clinical practice, such as clinical pharmacodynamic and pharmacokinetic studies, clinical trials, meta-analyses, outcomes research, and pharmacoeconomic and pharmacoepidemiological studies. Additional digital features (including animated abstracts, video abstracts, slide decks, audio slides, instructional videos, infographics, podcasts and animations) can be published with articles; these are designed to increase the visibility, readership and educational value of the journal’s content. In addition, articles published in Pediatric Drugs may be accompanied by plain language summaries to assist readers who have some knowledge of, but not in-depth expertise in, the area to understand important medical advances.
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