Claßen Merle, de Laffolie Jan, Claßen Martin, Schnell Alexander, Sohrabi Keywan, Hoerning André
Clinic for Children and Adolescent Medicine, Friedrich Alexander University Erlangen-Nuremberg, Erlangen, Germany.
Abteilung für Allgemeine Pädiatrie und Neonatologie, Justus Liebig University Gießen, Gießen, Germany.
Front Pediatr. 2022 Jul 19;10:903677. doi: 10.3389/fped.2022.903677. eCollection 2022.
In recent years, biological agents, such as anti-TNF-α blockers, have been introduced and have shown efficacy in pediatric patients with inflammatory bowel disease (IBD). Here, the prescription mode differentiated into a first/second line application, and efficacy and side effects are evaluated beginning from 2004 until today.
Statistical analyses of the prospective and ongoing CEDATA multicenter registry data from the Society of Pediatric Gastroenterology and Nutrition (GPGE) were performed for patients receiving a biological agent at least once during the period from June 2004 until November 2020 ( = 487). The analyzed parameters were patient demographics, disease extent and behavior, prior or concurrent therapies, duration and outcome of biological therapy, disease-associated complications, drug-related complications, laboratory parameters and treatment response as determined by the Physician's Global Assessment.
Crohn's disease (CD) was present in 71.5% of patients, and 52% were boys. Patients showed high disease activity when receiving a first-line TNF-α blocker. After 2016, patients who failed to respond to anti-TNF-α induction therapy were treated with off-label biologics (vedolizumab 4.3% and ustekinumab 2.1%). Propensity score matching indicated that patients with CD and higher disease activity benefitted significantly more from early anti-TNF-α therapy. This assessment was based on a clinical evaluation and lab parameters related to inflammation compared to delayed second-line treatment. Additionally, first-line treatment resulted in less treatment failure and fewer extraintestinal manifestations during TNF-α blockade.
First-line treatment with anti-TNF-α drugs is effective and safe. An earlier start significantly reduces the risk of treatment failure and is associated with fewer extraintestinal manifestations during longitudinal follow-up.
近年来，生物制剂，如抗TNF-α阻滞剂，已被引入并在儿童炎症性肠病（IBD）患者中显示出疗效。在此，将处方模式分为一线/二线应用，并对2004年至今的疗效和副作用进行评估。
对2004年6月至2020年11月期间至少接受过一次生物制剂治疗的患者（n = 487）的前瞻性且仍在进行的CEDATA多中心注册数据进行统计分析，该数据来自儿科胃肠病学和营养学会（GPGE）。分析的参数包括患者人口统计学、疾病范围和行为、先前或同时进行的治疗、生物治疗的持续时间和结果、疾病相关并发症、药物相关并发症、实验室参数以及由医生整体评估确定的治疗反应。
71.5%的患者患有克罗恩病（CD），52%为男孩。患者在接受一线TNF-α阻滞剂治疗时疾病活动度较高。2016年后，对抗TNF-α诱导治疗无反应的患者接受了未按标签用药的生物制剂治疗（维多珠单抗4.3%，优特克单抗2.1%）。倾向评分匹配表明，CD患者和疾病活动度较高的患者从早期抗TNF-α治疗中获益显著更多。与延迟的二线治疗相比，该评估基于临床评估和与炎症相关的实验室参数。此外，一线治疗在TNF-α阻断期间导致的治疗失败更少，肠外表现也更少。
抗TNF-α药物一线治疗有效且安全。更早开始治疗可显著降低治疗失败的风险，并且在长期随访中与更少的肠外表现相关。
