Spergel Jonathan M, Chehade Mirna, Dellon Evan S, Bredenoord Albert J, Sun Xian, Glotfelty Lila, Shabbir Arsalan, Tilton Sarette T, McCann Eilish
Division of Allergy and Immunology, Children's Hospital of Philadelphia, and Department of Pediatrics, Perelman School of Medicine at University of Pennsylvania School of Medicine, Philadelphia, Pennsylvania, USA.
Mount Sinai Center for Eosinophilic Disorders, Icahn School of Medicine at Mount Sinai, New York, New York, USA.
Am J Gastroenterol. 2024 Dec 1;119(12):2398-2407. doi: 10.14309/ajg.0000000000002924. Epub 2024 Jun 28.
Improvements in symptomatic experience and health-related quality of life (HRQoL) are among the most important treatment benefits in patients with eosinophilic esophagitis (EoE). We assessed the impact of dupilumab treatment on HRQoL, patients' impression of dysphagia, and symptoms beyond dysphagia in adults/adolescents (≥12 years) with EoE in parts A and B of the LIBERTY EoE TREET (NCT03633617) study.
The EoE Symptom Questionnaire (EoE-SQ; frequency and severity of nondysphagia symptoms), EoE Impact Questionnaire (impact of EoE on HRQoL), and Patient Global Impression of Severity and Patient Global Impression of Change of dysphagia were used to assess the efficacy of weekly dupilumab 300 mg vs placebo.
At week 24, dupilumab reduced EoE-SQ Frequency (least squares mean difference vs placebo [95% confidence interval] part A -1.7 [-2.9, -0.5], part B -1.4 [-2.3, -0.5]; both P < 0.01) and EoE-SQ Severity (part A -2.0 [-3.9, 0.0], P < 0.05, part B -1.5 [-3.0, 0.1], P = 0.07) overall scores, and improved scores across all individual items. Improvement in the dupilumab group was clinically meaningful to patients. Dupilumab also meaningfully improved EoE Impact Questionnaire average scores and improved individual item scores at week 24, particularly emotional and sleep disturbance. More dupilumab-treated patients reported improvement in the Patient Global Impression of Change of dysphagia vs placebo or reported having no symptoms per the Patient Global Impression of Severity of dysphagia at week 24.
Dupilumab reduced the impact of EoE on multiple aspects of HRQoL, patients' impression of dysphagia, and frequency and severity of symptoms beyond dysphagia in adults/adolescents with EoE.
症状体验和健康相关生活质量（HRQoL）的改善是嗜酸性粒细胞性食管炎（EoE）患者最重要的治疗益处之一。在LIBERTY EoE TREET（NCT03633617）研究的A部分和B部分中，我们评估了度普利尤单抗治疗对≥12岁成人/青少年EoE患者HRQoL、患者对吞咽困难的印象以及吞咽困难以外症状的影响。
使用EoE症状问卷（EoE-SQ；非吞咽困难症状的频率和严重程度）、EoE影响问卷（EoE对HRQoL的影响）以及患者对吞咽困难严重程度的整体印象和患者对吞咽困难变化的整体印象，来评估每周300mg度普利尤单抗与安慰剂的疗效。
在第24周时，度普利尤单抗降低了EoE-SQ频率（与安慰剂相比的最小二乘均值差异[95%置信区间]，A部分-1.7[-2.9, -0.5]，B部分-1.4[-2.3, -0.5]；均P<0.01）和EoE-SQ严重程度（A部分-2.0[-3.9, 0.0]，P<0.05，B部分-1.5[-3.0, 0.1]，P = 0.07）的总体得分，并且所有单项得分均有所改善。度普利尤单抗组的改善对患者具有临床意义。度普利尤单抗在第24周时还显著改善了EoE影响问卷的平均得分，并改善了单项得分，尤其是情绪和睡眠障碍方面。与安慰剂相比，更多接受度普利尤单抗治疗的患者在第24周时报告了吞咽困难变化的患者整体印象有所改善，或者根据患者对吞咽困难严重程度的整体印象报告无症状。
度普利尤单抗降低了EoE对成人/青少年EoE患者HRQoL的多个方面、患者对吞咽困难的印象以及吞咽困难以外症状的频率和严重程度的影响。
