DUPILUMAB EFFICACY IN CHILDREN WITH EOSINOPHILIC ESOPHAGITIS AND PRIOR USE OF OR PRIOR INADEQUATE RESPONSE, INTOLERANCE, OR CONTRAINDICATION TO SWALLOWED TOPICAL CORTICOSTEROIDS: RESULTS FROM THE PHASE 3 EOE KIDS STUDY

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Introduction

EoE is a debilitating disease with physical, behavioral, and psychosocial impacts, resulting in substantial impairment in quality of life for patients and caregivers. Children with EoE can present with nonspecific symptoms such as food refusal or feeding difficulties, gastroesophageal reflux disease (GERD), nausea, and vomiting. Dupilumab is approved in the USA and EU for patients with EoE aged ≥1 year, weighing ≥15 kg. In the phase 3 randomized placebo-controlled EoE KIDS study (NCT04394351), in children aged 1 to 11 years, dupilumab significantly improved symptoms of EoE vs placebo, as measured by the Pediatric Eosinophilic Esophagitis Symptom Score version 2.0 (caregiver version; PEESSv2.0) after 16 weeks of treatment.

Aims & Methods

In this post-hoc analysis of the EoE KIDS study, we evaluated the effects of long-term dupilumab treatment on PEESSv2.0 scores during the Part C open-label extension. PEESSv2.0 is a validated observer-reported outcome measure that assesses the frequency and severity of EoE symptoms and adaptive behaviors, divided into four domains: GERD, dysphagia, pain, and nausea; each is scored 0–100, with higher scores indicating a greater symptom domain severity. Patients who completed Week 52 in Part B of EoE KIDS were eligible for Part C, where all patients received open-label dupilumab, using the weight-tiered regimen later approved by the FDA (similar to the Part A and B higher-exposure regimen). In patients who received placebo or dupilumab higher exposure during Parts A and B, changes from baseline of the EoE KIDS study (Part A baseline) in the PEESSv2.0 total, frequency, severity, and domain scores were assessed at Week 52 (Part C baseline), Week 76, and Week 100.

Results

At Week 52, the mean change from baseline (95% confidence interval) in PEESSv2.0 total score was –23.8 (–32.1, –15.6; Table), with further reductions observed at Week 100 (–31.5 [–41.5, –21.4]). Mean changes from baseline in PEESSv2.0 frequency and severity scores were further reduced from Week 52 (–25.1 [–34.2, –16.1] and –22.2 [–30.3, –14.1], respectively), to Week 100 (–33.8 [–44.9, –22.7] and –28.6 [–38.3, –19.0]). Similar patterns of reduction were observed for individual primary domain scores, namely, GERD, dysphagia, pain, and nausea. During the Part C open-label extension, dupilumab safety was consistent with the known dupilumab safety profile.

Conclusion

Children with EoE, treated with long-term dupilumab, experienced sustained improvements in the frequency and severity of symptoms of EoE up to Week 100, assessed by the PEESSv2.0. Reductions in GERD, dysphagia, pain, and nausea domain scores were also maintained or in some cases further improved, although interpretation of Part C symptom data is limited due to the open-label study design. Future studies should consider the correlation of symptoms with trends in both endoscopic and histologic outcomes.

Table. Mean change from Part A baseline in PEESSv2.0 total, frequency, and severity scores during the Part C open-label extension of EoE KIDS
^aTotal, frequency, and severity scores are all scored 0–100. ^bPEESSv2.0 was not measured as a clinical endpoint in Part B of the EoE KIDS study; Week 52 data represent PEESSv2.0 scores at Part C baseline. CI, confidence interval; EoE, eosinophilic esophagitis; GERD, gastroesophageal reflux disease; n, number of patients; PEESSv2.0, Pediatric Eosinophilic Esophagitis Symptom Score version 2.0 (caregiver version).
PEESv2.0 element, mean change (95% CI)^a	Week 52^b(n = 32)	Week 76 (n = 30)	Week 100 (n = 23)
Total	–23.83 (–32.05, –15.60)	–31.08 (–39.38, –22.78)	–31.47 (–41.49, –21.44)
Frequency	–25.14 (–34.17, –16.11)	–34.17 (–43.07, –25.26)	–33.79 (–44.88, –22.71)
Severity	–22.22 (–30.31, –14.13)	–27.31 (–35.97, –18.66)	–28.62 (–38.29, –18.96)

Disclosure

Oliva S: Celgene/Receptos/Bristol Myers Squibb, Medtronic, Ocean Pharma, Sanofi/Regeneron Pharmaceuticals Inc – advisory board/consulting fees. Medtronic, Sanofi/Regeneron Pharmaceuticals Inc. – speaker fees.
Gold B: Diasorin Molecular, IQVIA/AstraZeneca, Ironwood Pharmaceuticals, Johnson&Johnson, Mead Johnson/Reckitt, Nutricia, Pfizer Inc, Sanofi/Regeneron Pharmaceuticals – advisory board/consultancy fees.
Ashok D: Alexion Pharma, LaunchitDTx, Mirum Pharma – advisory board/consulting fees. Sanofi – speaker fees.
Parrish C: Sanofi/Regeneron, Takeda – advisory board/consulting. Regeneron Pharmaceuticals Inc., Sanofi – speaker fees. DBV technologies, NIH/NIAID – research support.
Xia C, Raphael BP, Radwan A, Thomas RB: Regeneron Pharmaceuticals Inc. – employees and may hold stock or shares.
Cazeau C, Almansa C, Tilton ST: Sanofi – employees and may hold stock or shares.

DUPILUMAB IS EFFECTIVE IN MAINTAINING LONG-TERM SYMPTOMATIC IMPROVEMENT IN CHILDREN WITH EOSINOPHILIC ESOPHAGITIS (EOE), AS REPORTED BY THEIR CAREGIVERS: 100-WEEK RESULTS FROM THE OPEN-LABEL EXTENSION OF THE EOE KIDS STUDY

Salvatore Oliva 1, Benjamin Gold 2, Dhandapani Ashok 3, Christopher Parrish 4, Changming Xia 5, Christine Cazeau 6, Bram Raphael 5, Cristina Almansa 6, Amr Radwan 5, Sarette Tilton 6, Ryan Thomas 5

1 University Hospital Umberto, Sapienza University of Rome, Rome, Italy

2 Children’s Healthcare of Atlanta, Atlanta, United States

3 Children’s Hospital, London Health Sciences Centre, Western University, London, Canada

4 UT Southwestern Medical Center, Dallas, United States

5 Regeneron Pharmaceuticals Inc., Tarrytown, United States

6 Sanofi, Cambridge, United States

Conference

UEG Week Berlin 2025

Topics

Oesophagus

Submission format

Abstract

Session

Treatment for EoE (Posters)

Citation

United European Gastroenterology Journal 2025; 13 (Supplement 8)

Published

2025

^aNumber of patients who completed the Week 52 visit in Part B were n = 17 for PBO/DUP HE and n = 35 for DUP HE/DUP HE. ^bPatients with missing PEC were considered non-responders if not due to COVID-19, and were imputed by MI if due to COVID-19; patients with missing values for change from Part A baseline in EREFS total score, and HSS grade and stage scores were imputed by WOCF method if not due to COVID-19, and by MI if due to COVID-19; patients with missing values for change from Part A baseline in the proportion of days with ≥1 EoE sign measured by PESQ-C were imputed by WOCF method if due to rescue treatment/adverse events/lack of efficacy, and by MI if due to other reasons. ^cChange from Part A baseline: LS mean (SE) at W16; mean (SD) at W52. DUP, dupilumab; EoE, eosinophilic esophagitis; eos/hpf, eosinophils/high-power field; EREFS, Endoscopic Reference Score; HE, higher-exposure; HSS, Histologic Scoring System; LS, least squares; MI, multiple imputations; NA, not applicable as n=1; NI, number imputed; PBO, placebo; PEC, peak eosinophil count; PESQ-C, Pediatric EoE Sign/Symptom Questionnaire – Caregiver version; SD, standard deviation; SE, standard error; STC, swallowed topical corticosteroid; WOCF, worst observation carried forward.
		Week 16				Week 52
		PBO (N = 34)		DUP HE (N = 37)		PBO/DUP HE (N = 18)^a		DUP HE/DUP HE (N = 37)^a
		n/NI^b		n/NI^b		n		n
Patients with PEC of ≤6 eos/hpf, n (%)	With prior STC Without prior STC	27/0 4/3	0 1 (14.3)	28/0 9/0	17 (60.7) 8 (88.9)	16 1	8 (50.0) 1 (100.0)	26 9	13 (50.0) 9 (100.0)
Patients with PEC of <15 eos/hpf, n (%)	With prior STC Without prior STC	27/0 4/3	0 1 (14.3)	28/0 9/0	22 (78.6) 9 (100.0)	16 1	10 (62.5) 1 (100.0)	26 9	21 (80.8) 9 (100.0)
EREFS total score^c	With prior STC Without prior STC	22/0 5/2	0.3 (0.50) –0.7 (0.68)	24/2 8/0	–3.8 (0.46) –2.1 (0.63)	13 1	–3.08 (2.69) –11.00 (NA)	22 8	–5.14 (3.30) –3.75 (2.25)
HSS grade^c	With prior STC Without prior STC	27/0 4/3	0.07 (0.05) –0.14 (0.09)	28/0 9/0	–0.91 (0.05) –0.74 (0.08)	16 1	–0.87 (0.30) –1.07 (NA)	26 9	–1.03 (0.42) –0.77 (0.23)
HSS stage^c	With prior STC Without prior STC	27/0 4/3	0.06 (0.05) –0.01 (0.10)	28/0 9/0	–0.87 (0.05) –0.71 (0.09)	16 1	–0.85 (0.36) –1.01 (NA)	26 9	–0.93 (0.33) –0.78 (0.25)
PESQ-C^c	With prior STC Without prior STC	25/2 5/2	–0.13 (0.06) –0.27 (0.10)	23/4 8/1	–0.27 (0.06) –0.26 (0.09)	8 1	–0.50 (0.41) –0.22 (NA)	19 8	–0.28 (0.34) –0.37 (0.17)

	Placebo/Dupilumab qw			Dupilumab qw/Dupilumab qw
	Baseline Part B, mean (SD)	Absolute change from baseline Part B, mean (SD) Week 24 Week 52		Baseline Part B, mean (SD)	Absolute change from baseline Part B, mean (SD) Week 24 Week 52
EREFS
With prior STC use Without prior STC use	8.0 (3.7) 8.1 (2.8)	-1.5 (3.5) -2.1 (5.3)	-5.8 (3.7) -6.9 (3.5)	7.0 (3.0) 6.6 (3.2)	-5.0 (3.6) -3.9 (2.8)	-5.7 (2.8) -4.6 (2.9)
HSS grade score
With prior STC use	1.36 (0.34)	-0.22 (0.48)	-0.95 (0.40)	1.39 (0.40)	-1.01 (0.39)	-1.05 (0.41)
Without prior STC use	1.18 (0.34)	-0.13 (0.58)	-0.78 (0.37)	1.15 (0.36)	-0.75 (0.39)	-0.79 (0.41)
HSS stage score
With prior STC use	1.33 (0.29)	-0.21 (0.45)	-0.90 (0.36)	1.37 (0.33)	-0.98 (0.35)	-1.01 (0.35)
Without prior STC use	1.20 (0.33)	-0.14 (0.61)	-0.79 (0.31)	1.12 (0.29)	-0.68 (0.37)	-0.76 (0.38)

	Week 16^a			Week 52
	Placebo (N=34)	Dupilumab (N=37)	LS mean difference	Placebo (N=18)	Dupilumab (N=37)
EoE-HSS grade
Total score	5.0%	-64.4%	-69.4****	-63.6%	-68.5%
Inflammatory sub-score	5.7%	-77.4%	-83.1****	-79.4%	-83.7%
Architectural sub-score	1.0%	-50.5%	-51.5****	-45.3%	-55.5%
EoE-HSS stage
Total score	5.8%	-62.3%	-68.1****	-58.2%	-63.5%
Inflammatory sub-score	7.9%	-96.3%	-104.3****	-89.6%	-96.6%
Architectural sub-score	9.0%	-41.0%	-50.0****	-32.4%	-51.0%

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