Dipasquale Valeria, Romano Claudio
Pediatric Gastroenterology and Cystic Fibrosis Unit, Department of Human Pathology in Adulthood and Childhood "G. Barresi", University Hospital "G. Martino", 98122 Messina, Italy.
Healthcare (Basel). 2025 Apr 17;13(8):923. doi: 10.3390/healthcare13080923.
Pediatric gastroenterology is entering a pivotal phase marked by significant challenges and emerging opportunities in treating conditions like celiac disease (CeD), eosinophilic esophagitis (EoE), inflammatory bowel disease (IBD), and autoimmune hepatitis (AIH) pose significant clinical hurdles, but new therapeutic avenues are emerging. Advances in precision medicine, particularly proteomics, are reshaping care by tailoring treatments to individual patient characteristics. For CeD, therapies like gluten-degrading enzymes (latiglutenase, Kuma030) and zonulin inhibitors (larazotide acetate) show promise, though clinical outcomes are inconsistent. Immunotherapy and microbiota modulation, including probiotics and fecal microbiota transplantation (FMT), are also under exploration, with potential benefits in symptom management. Transglutaminase 2 inhibitors like ZED-1227 could help prevent gluten-induced damage. Monoclonal antibodies targeting immune pathways, such as AMG 714 and larazotide acetate, require further validation in pediatric populations. In EoE, biologics like dupilumab, cendakimab, dectrekumab (IL-13 inhibitors), and mepolizumab, reslizumab, and benralizumab (IL-5/IL-5R inhibitors) show varying efficacy, while thymic stromal lymphopoietin (TSLP) inhibitors like tezepelumab are also being investigated. These therapies require more pediatric-specific research to optimize their use. For IBD, biologics like vedolizumab, ustekinumab, and risankizumab, as well as small molecules like tofacitinib, etrasimod, and upadacitinib, are emerging treatments. New medications for individuals with refractory or steroid-dependent AIH have been explored. Personalized therapy, integrating precision medicine, therapeutic drug monitoring, and lifestyle changes, is increasingly guiding pediatric IBD management. This narrative review explores recent breakthroughs in treating CeD, EoE, IBD, and AIH, with a focus on pediatric studies when available, and discusses the growing role of proteomics in advancing personalized gastroenterological care.
儿科胃肠病学正进入一个关键阶段，在治疗乳糜泻（CeD）、嗜酸性食管炎（EoE）、炎症性肠病（IBD）和自身免疫性肝炎（AIH）等疾病方面面临重大挑战，但也出现了新的机遇。这些疾病带来了重大的临床障碍，但新的治疗途径正在涌现。精准医学的进展，尤其是蛋白质组学，正在通过根据个体患者特征定制治疗方案来重塑医疗护理。对于CeD，谷蛋白降解酶（拉替谷蛋白酶、Kuma030）和zonulin抑制剂（醋酸兰瑞肽）等疗法显示出前景，尽管临床结果并不一致。免疫疗法和微生物群调节，包括益生菌和粪便微生物群移植（FMT），也在探索中，在症状管理方面具有潜在益处。ZED - 1227等转谷氨酰胺酶2抑制剂有助于预防谷蛋白诱导的损伤。靶向免疫途径的单克隆抗体，如AMG 714和醋酸兰瑞肽，需要在儿科人群中进一步验证。在EoE中，度普利尤单抗、森达基单抗、德雷克单抗（IL - 13抑制剂）以及美泊利单抗、瑞斯拉单抗和贝那利珠单抗（IL - 5/IL - 5R抑制剂）等生物制剂显示出不同的疗效，而tezepelumab等胸腺基质淋巴细胞生成素（TSLP）抑制剂也在研究中。这些疗法需要更多针对儿科的研究以优化其使用。对于IBD，维多珠单抗、乌司奴单抗和瑞莎珠单抗等生物制剂以及托法替布、艾曲莫德和乌帕替尼等小分子药物正在成为新的治疗方法。已经探索了针对难治性或依赖类固醇的AIH患者的新药物。整合精准医学、治疗药物监测和生活方式改变的个性化治疗越来越多地指导儿科IBD的管理。本叙述性综述探讨了在治疗CeD、EoE、IBD和AIH方面的最新突破，如有可用则重点关注儿科研究，并讨论了蛋白质组学在推进个性化胃肠病护理中日益重要的作用。
