PeDRA

Pediatric Dermatology Research Alliance

Inflammatory Skin Disease

Utilization of the Topical Steroid Volume Calculator and its Effect on Atopic Dermatitis Clinical Outcomes

By

Fellow

Stephanie Lee, MD
University of California, San Diego
Rady Children’s Hospital

Mentor

Lawrence Eichenfield, MD
University of California, San Diego
Rady Children’s Hospital

Overview

Topical corticosteroids are the first-line treatment for acute flares of atopic dermatitis, the most common chronic inflammatory dermatologic condition affecting children. Despite evidence of its efficacy, topical corticosteroids are often underutilized by patients leading to subpar therapeutic results. Treatment non-adherence may be due to a variety of factors, including steroid phobia, a lack of full understanding of the regimen, prescriber/family communication issues, and non-specific recommendations about how to utilize medications for both acute and long-term care.
The proposed research project will help establish the clinical efficacy of the topical steroid volume calculator, with hopes that the utilization of the calculator will improve clinical outcomes of patients with atopic dermatitis by providing standardized volumes tailored to each patient. This will be a multi-center project, starting with two centers initially, and with the goal of three to five centers. The calculator is being incorporated into a standardized electronic medical record for ease of use by providers to give clear patient instructions (EPIC). As a result, it can help providers communicate better to their patients specifically how much topical corticosteroids to use to treat
acute flares of atopic dermatitis.

Status

This project was funded through a 2020 PeDRA Research Fellowship Grant.

Discovering the Immune Phenotype of Morphologic Subsets of Atopic Dermatitis at the Single Cell Level

By

Fellow

Carmel Aghdasi
University of California, San Francisco

Mentor

Kelly Cordoro, MD
University of California, San Francisco

Overview

The purpose of this work is to characterize the immunologic signatures and clinical characteristics of a diverse population of pediatric patients with atopic dermatitis. Understanding the unique immune pathogenesis of pediatric atopic dermatitis will drive discovery of targeted therapies appropriate to the unique needs of pediatric patients.

Status

This project was funded through a 2020 PeDRA Research Fellowship Grant.

Investigating Constitutive and Cell-Driven Fibroblast Activation in Pediatric Morphea

By

Fellow

Laila Abbas
UT Southwestern Medical Center

Mentor

Heidi Jacobe, MD, MSCS
UT Southwestern Medical Center

Overview

Morphea, or localized scleroderma, is a neglected autoimmune disease of the skin and underlying tissue that produces permanent disability due to joint contracture and soft tissue loss. Approximately half of patients have disease onset in childhood, and pediatric morphea is associated with significant morbidity, with reported complications including loss of range of motion, joint deformity, facial disfigurement, and neurologic manifestations. Morphea has remitting and relapsing activity, marked by inflammation and fibrosis, and damage which produces atrophy. The overarching hypothesis of this project is that fibroblasts are constitutively activated in morphea and that both T cells and macrophages will promote activation of fibroblasts.
Aim 1: Determine constitutive expression of pro-fibrotic and inflammatory markers in fibroblasts derived from morphea lesions as compared to matched control specimens.
Aim 2: Co-cultures will be performed with combinations of healthy and morphea fibroblasts along with healthy and morphea derived T cells and macrophages to determine if these cells can potentiate fibroblast activation.

Status

This project was funded through a 2020 PeDRA Research Fellowship Grant.

Expanding Access to Severe Acne Care for Navajo Adolescents Through Education

By

Principal Investigator:

Lucinda Kohn, MD, MHS
University of Colorado Denver

Overview:

It is a well-known fact that pediatric dermatologists are in short supply. We have many more potential patients than we do open clinical slots. We are also not spread evenly geographically, and there are several pockets of the U.S. that lack access to pediatric dermatology. What if there was a way to expand our reach to the patients who cannot easily access our expertise? What if we could safely partner with pediatricians to deliver high quality care? What kind of impact could we have on a community if we were able to offer its children treatments for their skin disease?

This pilot Project ECHO acne clinic at the Gallup Indian Medical Center will teach pediatricians how to treat acne with a medication called isotretinoin that is typically managed by dermatologists. The goal of this project is to show that with support and education, pediatricians can safely prescribe this medication.

Status:

This project was funded by a 2019 Weston Career Development Award.

Studying Topical Adherence in Atopic Dermatitis: What We Can Learn from Positive Outliers

By

Principal Investigator:

Steven Feldman, MD, PhD
Wake Forest University Health Sciences

Overview:

Although topical medications are highly effective treatments for atopic dermatitis, poor treatment adherence can lead to poor treatment outcomes and unnecessary escalation of costly therapy. The purpose of this study is to identify new approaches to help patients use their medicine by using a positive outlier approach: identifying novel strategies that successful patients already use to take their medication correctly. The adherence strategies used by patients who successfully use their topicals may help us identify novel ways to improve other patients’ use of medication and serve as a foundation for future studies to develop and test innovative adherence interventions.

Status:

This project was funded by a 2019 PeDRA Research Grant.

The Relationship between the Cutaneous Microbiome and Skin Barrier in Infantile Atopic Dermatitis

By

Principal Investigator:

Jennifer Schoch, MD
University of Florida

Co-Investigator:

Reesa Monir, MD
University of Florida

Overview:

Infantile atopic dermatitis (i.e. eczema) is a common pediatric skin condition leading to significant complications for patients and parents. Sleep disturbances, behavioral issues, and parental exhaustion are just a few of the negative ramifications. Though advances have been made in disease treatment, preventive interventions lag far behind.

Infancy is a unique, impressionable period of time during which the physical skin barrier, the cutaneous immune system, and the skin microbiome are developing in parallel. These closely intertwined factors are critical to the development of eczema. The skin barrier becomes “leaky” in kids with eczema, making it dry and prone to infection. The skin’s immune system becomes more reactive, leading to itching and inflammation. The collection of microbes that live in and on the skin (the skin microbiome) also changes in kids with eczema. The timeline of these changes and how they are related, however, is unclear.

In this study, we will begin exploration of the relationship between skin structure, cutaneous immunity, and the cutaneous microbiome. We will examine skin pH and the cutaneous microbiome in infants with newly-onset atopic dermatitis, compared to infants without eczema. Infants age three to nine months with newly-onset atopic dermatitis and without atopic dermatitis will be compared, with the goal of identifying how and when various aspects of skin development go awry. Understanding normal and pathologic skin changes is the first step in developing preventative interventions.

Status:

This study was funded through the 2019 PeDRA Research Hot Seat – A PeDRA Shark Tank.

Quality of Life of Parents with Children with Alopecia Areata

By

Principal Investigator:

Leslie Castelo-Soccio, MD, PhD
Children’s Hospital of Philadelphia

Overview:

Alopecia areata (AA) is an autoimmune disease resulting in non-scarring hair loss of hair-bearing areas, most frequently the scalp with a lifetime incidence of 1-2%. There is no sex predominance and it occurs in all parts of the world.  In the united states it accounts for 2.4 million doctor visits per year.   The more severe variants alopecia totalis (AT) and alopecia universalis (AU) lead to more extensive hair loss .  Due to its visibility, lack of curative therapy, and chronic nature, alopecia areata can create a significant psychosocial burden on patients who suffer from the disease. Though many studies have been conducted to determine psychosocial impact from the patient perspective, caregiver-oriented research is lacking. When available, clinicians could use information from such quality of life (QoL) tools to guide management and tailor support and education for patients and their families.

This was a multi-center, prospective study examining the impact on QoL of parents with children affected by alopecia areata. The overall goal was to quantitatively evaluate impairment in QoL in parents of children with AA. Secondary objectives were to investigate the relationship between scores on QoL tools and severity of disease, child’s QoL, and time since diagnosis.

Status:

This study was funded as a 2017 PeDRA Pilot Grant and is complete, with one publication resulting in 2019 (see publications page).

Genetic Variations in Non-coding Regulatory Regions in Linear Morphea

By

Principal Investigator:

Dawn Eichenfield, MD, PhD
University of California, San Diego

Overview:

Morphea is an inflammatory skin condition leading to hardened, discolored, and ultimately, scarred skin. It has several different presentations. Linear morphea commonly appears in children as a depressed or bound-down line on the forehead or extremity. Exactly why morphea occurs is unknown; however, studies suggest it is caused by a combination of genetic and environmental factors. Our research aims to uncover the genetic causes of linear morphea. Where our study differs from prior studies is the focus on studying parts of the genome that do not code for proteins, but instead are involved in regulating when or how genes are active. It is only recently that we realized the importance and disease relevance of these “non-coding” portions and could feasibly study their effects. Our project will utilize the latest sequencing technologies to investigate genetic mutations in morphea that can lead to improved diagnosis and earlier treatment.

Status:

This study was funded as a 2018 PeDRA Pilot Grant and is currently underway.