PeDRA

Pediatric Dermatology Research Alliance

Active Studies

Post-Market Surveillance of the Safety of Systemic JAK Inhibitors for Dermatologic Diseases in Children

By

Principal Investigator:

Jeff Yu, MD
Massachusetts General Hospital

Overview:

Oral Janus Kinase inhibitors (JAKi) are novel therapeutic options for patients with various inflammatory skin conditions, such as alopecia areata, atopic dermatitis, and psoriasis. Currently, most of the data that exists on the long-term side effects of JAKis comes from the rheumatology literature, where JAKis are used to treat highly inflammatory systemic diseases (i.e. rheumatoid arthritis). There is scant literature on the side effect profile or recommended laboratory monitoring during the use of JAKis in dermatologic conditions. Thus, the purpose of this database will be to provide pediatric dermatologists with accurate safety information and appropriate lab monitoring during the use of JAKis in the treatment of cutaneous inflammatory diseases, such as those listed above.

Status:

This project is new and needs additional sites. If you are interested in joining this study, please contact Hadley Johnson.

Neonatal Cutaneous Microbiome Predictors of Infantile Eczema

By

Principal Investigator:

Jennifer Schoch, MD
University of Florida

Overview:

Though the cause of eczema is yet unknown, animal studies suggest importance of the early interaction between bacteria present on the skin and the immune system. If bacteria are present on the skin of mice shortly after birth, then the immune system learns to “tolerate” the bacteria, and doesn’t create inflammation (i.e. eczema) when later exposed to the bacteria. To explore the possibility of a similar human mechanism, we will collect skin microbiome samples (including bacterial genetic material) from infants weekly for the first 4 weeks of life. The bacteria found on the skin in infants who later develop infantile eczema will be compared to infants who do not develop eczema. We hypothesize that specific bacteria (e.g. Staphylococcus) will be more abundant in the early skin microbiome of infants who do not develop eczema, compared to infants who do develop eczema. Successful completion will guide strategies to prevent eczema.

Status:

This study was funded through the 2021 Childhood Eczema Challenge Grant.

Characterization of Lipoprotein Composition and Function in Pediatric Psoriasis Before and After Treatment

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Principal Investigator:

Amy Paller, MD
Northwestern University

Overview:

Psoriasis is a chronic inflammatory skin disease associated with early onset cardiovascular disease. Despite studies demonstrating high cholesterol, increased diabetes and higher rate of obesity in psoriasis, these traditional risk factors only explain a fraction of total cardiovascular risk in psoriasis. One-third of the psoriasis patients start developing psoriatic plaques during childhood and studies have shown that the children with psoriasis have similar or higher risk for obesity and future problems with high cholesterol and diabetes. In fact, children have been shown to have abnormal cholesterol as early as age 13 years in psoriasis. However, little is known about how pediatric psoriasis severity affects cholesterol function, including its relationship with proteins involved in inflammation. Importantly, whether treatment of pediatric psoriasis early in the life leads to improvement in cholesterol function and overall lipoprotein composition is unknown. Therefore, the goal of this proposal is to understand the effect of disease activity and psoriasis treatment on cholesterol and metabolic markers in pediatric psoriasis.

Status:

This project was funded as a 2018 PeDRA and NPF Pediatric Psoriasis Challenge Grant and is currently underway.

Understanding Racial/Ethnic Disparities in Health Care Utilization for Childhood Atopic Dermatitis

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Principal Investigator:

Junko Takeshita, MD, PhD, MSCE
University of Pennsylvania

Overview:

Atopic dermatitis (AD) is a common skin disease that disproportionately affects minority children and is associated with a large financial burden in the U.S. Racial/ethnic disparities in health care use for AD also exist. For example, minority children are more likely than white children to go to the emergency room (ER) for their AD. In order to reduce differences in health care use for AD between white and minority children as well as minimize potentially avoidable and costly ER use, we propose to evaluate racial/ethnic differences in the barriers to routine health care use and reasons for specific health care use patterns among children with AD. We will interview caregivers of white, black, and Hispanic children with AD and identify the factors that affect health care use for AD by race/ethnicity. Our findings will inform future interventions to optimize and reduce racial/ethnic disparities in health care use for childhood AD.

Status:

This study was funded by PeDRA in partnership with the National Eczema Association (NEA) through the 2020 Childhood Eczema Challenge Grant and is currently underway.

COVID Acral Ischemia/Perniosis in children

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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.

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).

Genomic and Non genomic Causes of Segmental Pigmentation Disorder

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Principal Investigator:

Marcia Hogeling, MD
University of California, Los Angeles

Overview:

Segmental Pigmentation Disorder (SegPD) is an uncommon skin condition affecting young children. Segmental Pigmentation Disorder consists of white and brown pigmented birthmarks that appear as large patches on the body. The underlying cause of Segmental Pigmentation Disorder is not known, but it suspected to be due to genetic changes in these different areas of skin color, in the way that the skin cells produce pigment. The purpose of this study is to search for the cause of SegPD, by performing genetic testing of skin samples. Knowing the underlying cause of SegPD will allow us to better explain this skin problem to parents, and make informed recommendations on screening, monitoring and possibly treatments. Many birthmarks in children have the same mutations seen in cancer, and we think our study also might contribute to cancer research if SegPD has these mutations.

Status:

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

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.