PeDRA

Pediatric Dermatology Research Alliance

Cross-cutting Research

N-Acetylcysteine for Habit – Based Cutaneous Conditions: A Review of Real-World Use in the Pediatric Population

By

Fellow

Diana Zarowin
Albert Einstein College of Medicine

Mentor

James Treat, MD
Children’s Hospital of Philadelphia

Overview

The aim of this study is to (1) establish the breadth of habit-driven skin disorder diagnosis treated at CHOP with oral Nacetylcysteine, (2) determine dosing range and length of benefit in these patient populations, and (3) determine any potential side effects of oral N-acetylcysteine in real-world use. By doing this, we will be able to provide better recommendations on the use of oral N-acetylcysteine to treat many dermatologic disorders in pediatric populations – an area of treatment that has not yet been fully explored. The Children’s Hospital of Philadelphia (CHOP) database will be the source of patient data (100s of eligible patients expected), and the project will have personnel support from CHOP pediatric dermatologists and the Penn Department of Dermatology.

Status

This project was funded through a 2022 PeDRA Research Fellowship Grant.

Effects of IBAT inhibitors on itch in pediatric liver disease patients

By


Fellow

Siri Choragudi
University of Miami Miller School of Medicine

Mentor

Gil Yosipovitch, MD
University of Miami Miller School of Medicine

Overview

A common and often unbearable side effect of cholestatic genetic liver disease is that patients experience uncontrollable itch due to increased levels of bile acids in the bloodstream. This side effect is especially difficult for children to deal with, as they may have limited understanding of their conditions and little experience coping with significant physical discomfort. IBAT inhibitors, drugs that can reduce bile acids and therefore itchiness, were recently approved by the FDA for use in children. However, little is known about the efficacy of these drugs in this population. This research study aims to fill this gap in knowledge and improve treatment by using an in-depth Itch Severity, Quality of Life and Well Being surveys to assess 30-40 pediatric liver transplant patients’ experiences before and after IBAT inhibitor use.

Status

This project was funded through a 2022 PeDRA Research Fellowship Grant.

Investigating the role of Staphylococci in Pediatric Skin Disease

By


Principal Investigator:

Emily Gurnee, MD
University of Colorado Denver

Overview:

Staphylococcus aureus is a type of bacteria that commonly causes skin infections. Infections with this type of bacteria may worsen both common and rare skin diseases seen in children. There are several different types of Staphylococci and some may be more likely to cause severe disease than others. We plan to learn more about the relationship between skin disease and the type of bacteria by collecting skin swabs from children with atopic dermatitis, and epidermolysis bullosa, two very different diseases cared for by pediatric dermatologists. We plan to compare how severe these skin diseases are when Staphylococcal bacteria are present, and to investigate how the Staphylococci we find might be causing worsening disease. The purpose of this project is to better understand the relationship between Staphylococcus and pediatric skin disease with the long term goal of better understanding how to treat skin diseases.

Status:

This project was funded by a 2020 Weston Career Development Award.

COVID Acral Ischemia/Perniosis in children

By

Please help us collect important information. This is a survey for all health care professionals taking care of children who have developed acral ischemia/pernio or pernio-like changes in the setting of COVID-19 exposure. We are aware that the AAD is also collecting cases of dermatological changes in the setting of COVID and encourage you to fill out the AAD survey as well.

This survey will collect data only on acral changes noted in the pediatric population. The case report form should take 10 minutes to complete and there is a space for all comments at the end of the survey. There are no required elements so please complete as much as you can. No patient identifiers will be collected. We will collect your name and email address (if you volunteer this information) so we may contact you if we have any questions. All data will be de-identified and be kept confidential and stored on a secure REDCap server at the Children’s Hospital of Philadelphia. Researchers involved in data analysis will be allowed access to this data. This study tool/ survey was reviewed by the Institutional Review Board at Children’s Hospital of Philadelphia (IRB 20-017553) and an exemption was granted.

The registry can be completed by health care professionals from all over the United States and all countries. Patients should not enter their own cases.

Case REport Form

Please contact Leslie Castelo-Soccio, MD, PhD at castelosocciol@email.chop.edu with any questions.

An International Study to Develop A Patient-Reported Outcome Measure for Scars (The SCAR-Q), and to Determine the Psychosocial Impact of Scarring

By

Principal Investigator:

Joel Fish, MD
The Hospital for Sick Children

Overview:

Beyond being just a permanent skin mark, a scar can influence quality of life, and alter a person’s view of themselves. However, healthcare providers often only assess a scar’s physical features. In children particularly, without probing, the true impact of scars may therefore go unrecognized. This project’s purpose is two-fold – firstly to complete validation of the SCAR-Q (a patientfocused scar-specific questionnaire), and secondly to evaluate the psychosocial impact of scars in children. Consented children will complete the SCAR-Q before and after scar treatment. The scores will be used to determine the SCAR-Q’s responsiveness (its ability to measure change), and to quantify the psychosocial burden of scars. Ultimately, the SCAR-Q will allow better appreciation of patient feelings about their scars, while purposefully characterizing the psychosocial impact of scarring will inform clinical practice. Specifically, by understanding the mental, emotional, and social weight of scars, healthcare providers may
deliver improved patient-centered care.

Status:

This project was funded by a 2019 PeDRA Research Grant.

An Examination of the Dermatologic Needs of Sexual/Gender Minority Youth

By

Principal Investigator:

Markus Boos, MD, PhD
University of Washington and Seattle Children’s Hospital

Overview:

Relatively little is known about the dermatologic needs of sexual and gender minority (SGM) persons, and no research has been performed specifically evaluating the needs of SGM children and youth. This is an observational, cross-sectional, survey-based study that will assess the dermatologic needs and concerns of sexual and gender minority (SGM) youth (lesbian, gay, bisexual, transgender, questioning/queer) relative to their cis-gender/heterosexual peers.  This project will directly address knowledge gaps in our understanding of the skin health of a historically vulnerable population, and will lay the groundwork for developing future studies addressing those dermatologic discrepancies identified in SGM youth.

Status:

Funded as a 2018 PeDRA Pilot Grant. Currently enrolling patients.

The Burden of Pediatric Skin Disease — the “Big Study”

By

 

What is the study about?

We hope to better understand the burden of skin disorders in school-age children, and how these skin disorders affect a child’s personal and social perceptions. We want to study the relationship between the burden and how severe or visible the skin problem is.

Who is this study for?

This study will enroll children ages 8 to 17 years of age with chronic and highly visible skin conditions, as determined by the physician.

Both the child and participating parent must speak English and be able to complete questionnaires about the child’s skin condition, social experiences, and quality of life.

Status:

Data collection is underway with the target of 3500 enrolled patients and parents anticipated in early 2020.

Sites:

What will you do if you participate?

If you qualify and decide to participate, you will be asked to:

  • Complete one in-person visit at a local recruiting site
  • Complete questionnaires for both the parent (~15-20 minutes to complete) and the child (~10-20 minutes)
  • Have a brief physician-conducted clinical evaluation
  • You will receive $10 compensation

We hope that parents and children will consider completing some questionnaires to help provide these answers. Participation is entirely voluntary.

What should you do if you are interested in participating?

If you would like to learn more about this study, you can:

trial page
study Newsletters