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Clinical Trial
. 2025 Jul;42(7):3186-3206.
doi: 10.1007/s12325-025-03150-6. Epub 2025 May 8.

Dupilumab Efficacy in Children with Atopic Dermatitis with Different Phenotypes and Endotypes: A Case Series

Ana B Rossi  1 , Adriana M Mello  2 , Joseph Zahn  3
Affiliations
Clinical Trial

Dupilumab Efficacy in Children with Atopic Dermatitis with Different Phenotypes and Endotypes: A Case Series

Ana B Rossi et al. Adv Ther. 2025 Jul.

Abstract

Introduction: Atopic dermatitis (AD) is a prevalent disease in infants and young children worldwide. Dupilumab has been shown to rapidly and significantly improve AD signs, symptoms, and quality of life in pediatric patients with moderate-to-severe AD.

Methods: In LIBERTY AD PRESCHOOL, a randomized, double-blind, placebo-controlled, phase 3 clinical trial, patients aged 6 months to 5 years with moderate-to-severe AD received subcutaneous dupilumab or matched placebo every 4 weeks for 16 weeks. All patients received concomitant low-potency topical corticosteroids. Here, we present 12 photographic cases of patients with different phenotypes and endotypes in the dupilumab group, representative of the study population, before and after treatment. Each case is presented with clinical outcome measures of AD severity and quality of life, as well as relevant biomarkers, with percent improvement from baseline.

Results: Treatment with dupilumab led to visible improvements in signs of lichenification, erythema, excoriations, skin dryness, and oozing/crusting. Clinically meaningful improvements in the measured outcomes were observed in most of the patients, including AD extent and severity, clinical lesions, itch, sleep loss, frequency of symptoms, and quality of life. These improvements were associated with substantial reductions in AD-related biomarkers.

Conclusion: Treatment with dupilumab improved signs, symptoms, and quality of life, and reduced AD-related serum biomarkers in young children with moderate-to-severe AD with different phenotypes and endotypes.

Trial registration: The trial was registered with ClinicalTrials.gov with ID number NCT03346434 on November 17, 2017. Video abstract available for this article. Video abstract (MP4 1,02,609 KB).

Keywords: Atopic dermatitis; Dupilumab; Endotypes; Pediatric dermatology; Phenotypes.

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Conflict of interest statement

Declarations. Conflict of Interest: Ana B. Rossi and Adriana M. Mello are employees of and may hold stock and/or stock options in Sanofi. Joseph Zahn is an employee and shareholder of Regeneron Pharmaceuticals Inc. Ethical Approval: The study was conducted in accordance with the Declaration of Helsinki, the International Conference on Harmonization Good Clinical Practice guidelines, and applicable regulatory requirements. An independent data and safety monitoring committee conducted blinded monitoring of patient safety data. The local Institutional Review Board or Ethics Committee at each study center oversaw trial conduct and documentation. The study was approved by the Copernicus Group ethics committee. All parents/guardians provided written informed consent before participating in the trial, including for the publication of clinical photos taken in selected centers. Pediatric patients provided assent according to the Ethics Committee (Institutional Review Board/Independent Ethics Committee)-approved standard practice for pediatric patients at each participating center.

Figures

Fig. 1
Fig. 1
Overall study population: a mean of imputed value at baseline (red circles) and week 16 (blue squares), b LS mean % improvement from baseline to week 16. BSA body surface area, CCL17 C-C motif chemokine ligand 17, CDLQI Children’s Dermatology Life Quality Index, CGID Caregiver Global Impression of Disease, DFI Dermatitis Family Impact, EASI Eczema Area and Severity Index, IDQOL Infants’ Dermatitis Quality of Life Index, IGA Investigator’s Global Assessment, IgE immunoglobulin E, LS least squares, POEM Patient-Oriented Eczema Measure, SCORAD SCORing Atopic Dermatitis, VAS visual analog scale
Fig. 2
Fig. 2
Case 1 at baseline vs. week 16 of treatment: a patient case at baseline (left) and after 16 weeks of treatment with dupilumab (right), b baseline (red) and end of treatment (blue) values, c percent improvement from baseline. Weekly average worst itch score not available at week 16. BSA body surface area, CCL17 C-C motif chemokine ligand 17, CDLQI Children’s Dermatology Life Quality Index, CGID Caregiver Global Impression of Disease, DFI Dermatitis Family Impact, EASI Eczema Area and Severity Index, IGA Investigator’s Global Assessment, IgE immunoglobulin E, POEM Patient-Oriented Eczema Measure, SCORAD SCORing Atopic Dermatitis, VAS visual analog scale
Fig. 3
Fig. 3
Case 2 at baseline vs. week 16 of treatment: a patient case at baseline (left) and after 16 weeks of treatment with dupilumab (right), b baseline (red) and end of treatment (blue) values, c percent improvement from baseline. Weekly average worst itch score not available at week 16. BSA body surface area, CDLQI Children’s Dermatology Life Quality Index, CGID Caregiver Global Impression of Disease, DFI Dermatitis Family Impact, EASI Eczema Area and Severity Index, IGA Investigator’s Global Assessment, IgE immunoglobulin E, POEM Patient-Oriented Eczema Measure, SCORAD SCORing Atopic Dermatitis, VAS visual analog scale
Fig. 4
Fig. 4
Case 3 at baseline vs. week 16 of treatment: a patient case at baseline (left) and after 16 weeks of treatment with dupilumab (right), b baseline (red) and end of treatment (blue) values, c percent improvement from baseline. Weekly average worst itch score not available at week 16. BSA body surface area, CCL17 C-C motif chemokine ligand 17, CDLQI Children’s Dermatology Life Quality Index, CGID Caregiver Global Impression of Disease, DFI Dermatitis Family Impact, EASI Eczema Area and Severity Index, IGA Investigator’s Global Assessment, IgE immunoglobulin E, POEM Patient-Oriented Eczema Measure, SCORAD SCORing Atopic Dermatitis, VAS visual analog scale
Fig. 5
Fig. 5
Case 4 at baseline vs. week 16 of treatment: a patient case at baseline (left, top right) and after 16 weeks of treatment with dupilumab (middle, bottom right), b baseline (red) and end of treatment (blue) values, c percent improvement from baseline. Weekly average worst itch score not available at week 16. BSA body surface area, CCL17 C-C motif chemokine ligand 17, CGID Caregiver Global Impression of Disease, DFI Dermatitis Family Impact, EASI Eczema Area and Severity Index, IDQOL Infants’ Dermatitis Quality of Life Index, IGA Investigator’s Global Assessment, IgE immunoglobulin E, POEM Patient-Oriented Eczema Measure, SCORAD SCORing Atopic Dermatitis, VAS visual analog scale
Fig. 6
Fig. 6
Case 5 at baseline vs. week 16 of treatment: a patient case at baseline (left) and after 16 weeks of treatment with dupilumab (right), b baseline (red hexagons) and end of treatment (blue diamonds) values, c percent improvement from baseline. BSA body surface area, CCL17 C-C motif chemokine ligand 17, CDLQI Children’s Dermatology Life Quality Index, CGID Caregiver Global Impression of Disease, DFI Dermatitis Family Impact, EASI Eczema Area and Severity Index, IGA Investigator’s Global Assessment, IgE immunoglobulin E, POEM Patient-Oriented Eczema Measure, SCORAD SCORing Atopic Dermatitis, VAS visual analog scale
Fig. 7
Fig. 7
Case 6 at baseline vs. week 16 of treatment: a patient case at baseline (left) and after 16 weeks of treatment with dupilumab (right), b baseline (red) and end of treatment (blue) values, c percent improvement from baseline. BSA body surface area, CCL17 C-C motif chemokine ligand 17, CDLQI Children’s Dermatology Life Quality Index, CGID Caregiver Global Impression of Disease, DFI Dermatitis Family Impact, EASI Eczema Area and Severity Index, IGA Investigator’s Global Assessment, IgE immunoglobulin E, POEM Patient-Oriented Eczema Measure, SCORAD SCORing Atopic Dermatitis, VAS visual analog scale
Fig. 8
Fig. 8
Case 7 at baseline vs. week 16 of treatment: a patient case at baseline (left, top right) and after 16 weeks of treatment with dupilumab (middle, bottom right), b baseline (red hexagons) and end of treatment (blue diamonds) values, c percent improvement from baseline. BSA body surface area, CCL17 C-C motif chemokine ligand 17, CGID Caregiver Global Impression of Disease, DFI Dermatitis Family Impact, EASI Eczema Area and Severity Index, IDQOL Infants’ Dermatitis Quality of Life Index, IGA Investigator’s Global Assessment, IgE immunoglobulin E, POEM Patient-Oriented Eczema Measure, SCORAD SCORing Atopic Dermatitis, VAS visual analog scale
Fig. 9
Fig. 9
Case 8 at baseline vs. week 16 of treatment: a patient case at baseline (left) and after 16 weeks of treatment with dupilumab (right), b baseline (red hexagons) and end of treatment (blue diamonds) values, c percent improvement from baseline. Weekly average worst itch score not available at week 16. BSA body surface area, CCL17 C-C motif chemokine ligand 17, CGID Caregiver Global Impression of Disease, DFI Dermatitis Family Impact, EASI Eczema Area and Severity Index, IDQOL Infants’ Dermatitis Quality of Life Index, IGA Investigator’s Global Assessment, IgE immunoglobulin E, POEM Patient-Oriented Eczema Measure, SCORAD SCORing Atopic Dermatitis, VAS visual analog scale
Fig. 10
Fig. 10
Case 9 at baseline vs. week 16 of treatment: a patient case at baseline (left, top right) and after 16 weeks of treatment with dupilumab (middle, bottom right), b baseline (red) and end of treatment (blue) values, c percent improvement from baseline*. BSA body surface area, CCL17 C-C motif chemokine ligand 17, CDLQI Children’s Dermatology Life Quality Index, CGID Caregiver Global Impression of Disease, DFI Dermatitis Family Impact, EASI Eczema Area and Severity Index, IGA Investigator’s Global Assessment, IgE immunoglobulin E, POEM Patient-Oriented Eczema Measure, SCORAD SCORing Atopic Dermatitis, VAS visual analog scale. *Negative improvement is shown as 0
Fig. 11
Fig. 11
Case 10 at baseline vs. week 16 of treatment: a patient case at baseline (left) and after 16 weeks of treatment with dupilumab (right), b baseline (red hexagons) and end of treatment (blue diamonds) values, c percent improvement from baseline. BSA body surface area, CCL17 C-C motif chemokine ligand 17, CDLQI Children’s Dermatology Life Quality Index, CGID Caregiver Global Impression of Disease, DFI Dermatitis Family Impact, EASI Eczema Area and Severity Index, IGA Investigator’s Global Assessment, IgE immunoglobulin E, POEM Patient-Oriented Eczema Measure, SCORAD SCORing Atopic Dermatitis, VAS visual analog scale
Fig. 12
Fig. 12
Case 11 at baseline vs. week 16 of treatment: a patient case at baseline (left) and after 16 weeks of treatment with dupilumab (right), b baseline (red hexagons) and end of treatment (blue diamonds) values, c percent improvement from baseline. BSA body surface area, CCL17 C-C motif chemokine ligand 17, CDLQI Children’s Dermatology Life Quality Index, CGID Caregiver Global Impression of Disease, DFI Dermatitis Family Impact, EASI Eczema Area and Severity Index, IGA Investigator’s Global Assessment, IgE immunoglobulin E, POEM Patient-Oriented Eczema Measure, SCORAD SCORing Atopic Dermatitis, VAS visual analog scale
Fig. 13
Fig. 13
Case 12 at baseline vs. week 16 of treatment: a patient case at baseline (left) and after 16 weeks of treatment with dupilumab (right), b baseline (red hexagons) and end of treatment (blue diamonds) values, c percent improvement from baseline. BSA body surface area, CCL17 C-C motif chemokine ligand 17, CGID Caregiver Global Impression of Disease, DFI Dermatitis Family Impact, EASI Eczema Area and Severity Index, IDQOL Infants’ Dermatitis Quality of Life Index, IGA Investigator’s Global Assessment, IgE immunoglobulin E, POEM Patient-Oriented Eczema Measure, SCORAD SCORing Atopic Dermatitis, VAS visual analog scale
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