# Systemic Treatments and Outcomes for Pediatric Atopic Dermatitis: Setting the Stage for Comparative Effectiveness Research

> **NIH NIH R03** · JOHNS HOPKINS UNIVERSITY · 2024 · $170,490

## Abstract

PROJECT SUMMARY / ABSTRACT
Atopic dermatitis, or eczema, affects up to 20% of children and is associated with significant physical,
emotional, and social morbidity. Up to one-third of children with atopic dermatitis have moderate-to-severe
disease and require systemic medications to achieve adequate disease control. While systemic therapies for
pediatric atopic dermatitis were historically limited and consisted of broadly immunosuppressive medications
such as methotrexate, highly efficacious and targeted systemic treatments, such as dupilumab and oral Janus
kinase inhibitors, have recently emerged. However, the optimal treatment for any individual child remains
uncertain, as real-world data on the long-term effectiveness and safety of both novel and traditional systemic
medications for pediatric atopic dermatitis remain sparse. Thus, comparative effectiveness research (CER) is
critically needed to fill these current knowledge gaps. Since new treatments for atopic dermatitis continue to
rapidly emerge and traditional randomized trials are often lengthy and expensive, pragmatic trials and
observational studies may be more efficient for CER in pediatric populations and nimbler in adapting to the
expanding treatment landscape for atopic dermatitis. The growth of large clinical research networks, such as
the national Patient-Centered Outcomes Research Network (PCORnet), also provides opportunities to
leverage real-world data to conduct CER in pediatric atopic dermatitis. However, before such efforts can be
undertaken, tools to easily and accurately identify children with atopic dermatitis within clinical research
networks must be developed and methods to utilize observational data for CER in a way that ensures valid
inferences must be employed. To meet these needs, this proposal has two specific aims: (1) develop and
validate computable phenotypes for pediatric atopic dermatitis, and (2) apply a target trial emulation framework
to compare the effectiveness and safety of dupilumab and methotrexate for atopic dermatitis treatment.
Utilizing routinely-collected electronic health data held in the PCORnet common data model, this project will
apply machine learning approaches to develop validated computable phenotypes for pediatric atopic
dermatitis, including those distinguishing moderate-to-severe disease from mild disease, using structured data
elements. In the target trial emulation comparing new pediatric users of dupilumab or methotrexate for atopic
dermatitis, this project will test the hypothesis that dupilumab is associated with fewer topical medication
prescriptions, lower rates of systemic treatment switching or intensification, and lower rates of adverse effects
compared to methotrexate. This research will enable the efficient conduct of future pragmatic trials in pediatric
atopic dermatitis and provide preliminary data for large, multi-center observational CER studies that will be the
focus of future R01 applications. Ultimately, the findings f...

## Key facts

- **NIH application ID:** 10954351
- **Project number:** 1R03AR084585-01
- **Recipient organization:** JOHNS HOPKINS UNIVERSITY
- **Principal Investigator:** Joy Wan
- **Activity code:** R03 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 2024
- **Award amount:** $170,490
- **Award type:** 1
- **Project period:** 2024-07-19 → 2026-06-30

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/10954351

## Citation

> US National Institutes of Health, RePORTER application 10954351, Systemic Treatments and Outcomes for Pediatric Atopic Dermatitis: Setting the Stage for Comparative Effectiveness Research (1R03AR084585-01). Retrieved via AI Analytics 2026-05-26 from https://api.ai-analytics.org/grant/nih/10954351. Licensed CC0.

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