Soudant Juliette, Lejeune Stéphanie, Aumar Madeleine, Caron Nicolas, Lengline Hélène, Hoarau Cyrille, Kalach Nicolas, Enaud Raphaël, Bonneton Marjorie, Dupont-Lucas Claire, Fabre Alexandre, Butori Mathilde, Lemoine Anaïs, Bridoux-Henno Laure, Rebeuh Julie, Dimitrov Georges, Caillau Émeline, Gottrand Frédéric, Tran Léa Chantal
Univ.Lille, CHU Lille, Pediatric Gastroenterology Hepatology and Nutrition Department, Jeanne de Flandre Hospital, CHU Lille, F-59000, Lille, France.
Univ. Lille, CHU Lille, Pediatric Pulmonology and Allergy Department, Jeanne de Flandre Hospital, CHU Lille, F-59000, Lille, France.
Pediatr Res. 2025 Jun 7. doi: 10.1038/s41390-025-04011-2.
No biological treatment has been approved for pediatric eosinophilic esophagitis (EoE) in France. For patients refractory to conventional treatments, although compassionate use of monoclonal antibodies has developed, experience remains limited.
We conducted a national multicenter study across French pediatric tertiary care centers where children (younger than 18 years) who presented with EoE were treated with biological therapies between January 2015 and December 2023. The main objective was to characterize this patient population, and the indications for prescribing biologics. The secondary goals were to assess these patients' clinical, endoscopic, and histologic finding, as well as patient tolerance, and to compare our cohort at baseline with pediatric patients from two European registers of EoE treated with conventional therapies.
Thirty-six patients were prescribed 37 biologics (omalizumab, n = 1; mepolizumab, n = 6; dupilumab, n = 30). At diagnosis, the mean patient age was 7.4 (±4.4) years, and most patients had at least one atopic comorbidity (91.7%, n = 33). Failure of first-line treatments was the main reason for starting biological therapy (75.7%, n = 28), prescribed as compassionate use (54.1%, n = 20). Dupilumab showed significant clinical (48%, p < 0.01) and histological (82.6%, p < 0.01) improvement. Compared with children treated with conventional therapies, patients in our cohort at baseline presented significantly more asthma, food allergies, and atopic dermatitis, as well as more fibrostenotic phenotype and digestive symptoms. No severe side effect was reported within a 6-12-month follow-up.
Dupilumab is the most frequently prescribed, and appears to be the most effective biotherapy, regarding clinical and histologic remission. All biologics were well-tolerated.
Pending marketing authorization, biological therapies for pediatric eosinophilic esophagitis are mainly prescribed after failure of first-line treatments and on a compassionate basis in France. Dupilumab is the biotherapy most frequently used, is associated with clinical and histological efficacy and is well-tolerated. Children and adolescents requiring biologics appear to be younger and more severe at diagnosis than naive pediatric patients in Europeans registries.
在法国，尚无生物疗法被批准用于治疗儿童嗜酸性粒细胞性食管炎（EoE）。对于传统治疗无效的患者，尽管已开展了单克隆抗体的同情用药，但经验仍然有限。
我们在法国多家儿科三级护理中心开展了一项全国性多中心研究，纳入2015年1月至2023年12月期间接受生物疗法治疗的EoE患儿（年龄小于18岁）。主要目的是描述该患者群体特征以及生物制剂的处方指征。次要目标是评估这些患者的临床、内镜和组织学检查结果以及患者耐受性，并将我们队列的基线情况与两个接受传统疗法治疗的欧洲EoE患儿登记处的患儿进行比较。
36例患者使用了37种生物制剂（奥马珠单抗，n = 1；美泊利单抗，n = 6；度普利尤单抗，n = 30）。诊断时，患者平均年龄为7.4（±4.4）岁，大多数患者至少有一种特应性合并症（91.7%，n = 33）。一线治疗失败是开始生物治疗的主要原因（75.7%，n = 28），以同情用药方式处方（54.1%，n = 20）。度普利尤单抗显示出显著的临床改善（48%，p < 0.01）和组织学改善（82.6%，p < 0.01）。与接受传统疗法治疗的儿童相比，我们队列中的患者在基线时哮喘、食物过敏和特应性皮炎更多，纤维狭窄表型和消化症状也更多。在6至12个月的随访中未报告严重副作用。
就临床和组织学缓解而言，度普利尤单抗是最常处方的生物疗法，且似乎是最有效的生物疗法。所有生物制剂耐受性良好。
在法国，等待上市许可期间，儿童嗜酸性粒细胞性食管炎的生物疗法主要在一线治疗失败后且基于同情用药的基础上处方。度普利尤单抗是最常用的生物疗法，具有临床和组织学疗效且耐受性良好。与欧洲登记处的初治儿科患者相比，需要生物制剂治疗的儿童和青少年在诊断时似乎更年轻且病情更严重。
