PeDRA

Pediatric Dermatology Research Alliance

Skin Tumors and Reactions to Cancer Therapies

A Multi-Center Retrospective Cohort Study on the Utility of Ancillary Studies in the Risk Assessment and Management of Spitz Nevi, Atypical Spitzoid Tumors, and Spitzoid Melanoma in the Pediatric Population

By



Principal Investigator:

Kristen Berrebi, MD
University of Iowa Health Care

Overview:

Spitz tumors are melanocytic proliferations seen in children and are divided into three categories along a spectrum: (1) benign Spitz nevus, (2) atypical Spitz tumor, and (3) Spitzoid melanoma. The current gold standard for diagnosis is biopsy. Identification of Spitzoid melanoma is important because diagnosis and appropriate treatment can prevent morbidity and mortality, but the differentiation between this spectrum of lesions remains a challenge.

Additional immunohistochemical and molecular/genetic studies have been developed for further characterization of these lesions, however their utility is not well-defined.. Clinical uncertainty arises when benign histopathologic features do not align with results of these studies.

This study aims to pool data for patients with Spitzoid neoplasms in an effort to evaluate how these aforementioned ancillary studies affect clinical plan and outcome. We hope to identify the value and cost effectiveness of these studies with a future goal of determining treatment guidelines of Spitzoid neoplasms.

Status:

This project was funded by a 2020 PeDRA Research Grant.

Beyond the phenotype – A targeted sequencing study to identify the spectrum of genetic mutations in pyogenic granulomas

By


Principal Investigator:

Carrie Coughlin, MD
Washington University School of Medicine in St. Louis

Overview:

Pyogenic granulomas are benign growths of blood vessels which can grow quickly and bleed. It can be difficult to stop the bleeding, which can make them an urgent problem. Their primary treatment is surgical, which carries the risks of infection, scarring, and recurrence. Therefore, understanding the changes that occur in the DNA of these growths may lead to better medicines to treat them and avoid the potential side effects of surgery. In this study, we propose examining the DNA of 60 previously removed pyogenic granulomas to investigate for the presence of spontaneous mutations in 177 different genes which may have caused the formation of these benign growths. These 60 samples will be representative of diversity in age, sex, and location on the body to provide the best profile of DNA mutations to inform the future development of targeted medicines.

Status:

This project was funded by a 2020 PeDRA Research Grant.

Keratinocyte Carcinoma in Pediatric Solid Organ Transplant Patients

By

Fellow

Muskaan Sachdeva, BHSc
University of Toronto

Mentor

Cathryn Sibbald, MScPhm, MD, FRCPC, MSc
University of Toronto
The Hospital for Sick Children

Overview

Keratinocyte carcinomas (KC), which include basal cell and squamous cell carcinomas, are the most common malignancy in organ transplant recipients. Specifically, the incidence of basal cell carcinoma is 10 times higher, and squamous cell carcinoma is 100 times more frequent in adult transplant recipients than the general population. Although the success of transplants has vastly improved for children, limited research exists on the risk of post-transplant KC in pediatric populations, with a recent study excluding these cancers. Pediatric transplant patients are immunocompromised for longer durations than patients transplanted in adulthood, and therefore the onset of KC in pediatric transplant recipients may occur at an earlier age. Analysis of specific pediatric data is important to fully quantify and understand the risk of KC in this population.

Status

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

The impact of dilute bleach compresses on the incidence and severity of radiation dermatitis

By

Fellow

Danny Linggonegoro
Harvard Medical School

Mentor

Jennifer Huang, MD
Harvard Medical School

Overview

Acute radiation skin toxicity is one of the most common side effects encountered in those undergoing radiation therapy, affecting nearly all patients; however, no standard criterion currently exists for the prevention of acute radiation induced skin toxicity.
This study aims to:
1. Determine if dilute bleach compresses can attenuate the incidence and severity of radiation dermatitis.
2. Evaluate and describe patient-reported outcomes.

Status

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

Preventative Skin Care for Children Undergoing Targeted CNS Tumor Therapy

By

Principal Investigator:

Jennifer Huang, MD
Boston Children’s Hospital

Overview:

Cancer treatments targeting MEK and BRAF proteins are used to treat childhood brain tumors. Treatments can often cause cutaneous problems, sometimes leading to discontinuation of life-saving treatments. Our study aims to develop an effective skin care routine to decrease incidence and severity of these reactions. In this prospective single-arm trial, all participants will receive a preventative skin care regimen (sun protection, dry skin care) as well as every-other-day dilute bleach baths for twelve weeks. We will examine both groups at baseline, 6 weeks, and 12 weeks. We will compare this cohort to a historical cohort that did not receive any preventative skin care regimen or dilute bleach baths. We predict that preventative skin care and dilute bleach baths will be more effective than no preventative skin care regimen or dilute bleach baths in decreasing the incidence and severity of these cutaneous reactions.

Status:

This project was funded by a 2019 PeDRA Research Grant.

What does a pediatric BAP1 inactivated melanocytic tumor (BIMT) look like?

By

Principal Investigator:

Elena Hawryluk, MD, PhD
Massachusetts General Hospital

Overview:

Spitz nevi are a type of skin growth that occur most commonly in children. This study seeks to better understand Spitz nevi in children by determining possible overlap with a different entity known as BAP1 inactivated melanocytic tumors (BIMTs). BIMTs have many microscopic features that overlap with Spitz nevi, but are not well studied in children. Providers encountering BIMTs in children are not in agreement for the most appropriate management for these patients given the lack of data on these tumors.
We propose to study these tumors by performing special stains (BAP1, BRAF) on previously collected tissue for 30 identified cases of combined Spitz and dermal nevi. By better understanding these skin growths, we will be able to better assess if this subset of Spitz nevi should be re-characterized as BIMTs, and help shed light on the understanding and management of these poorlycharacterized skin growths in the pediatric population.

Status:

This project was funded by a 2019 PeDRA Research Grant.

Consensus-based recommendations for management of pediatric Mycosis Fungoides: the ARMFUL (AppRoach Mycosis FUngoides in chiLdren) study

By

Co-Principal Investigators:

Alex Zvulunov, MD, MHA
Ben-Gurion University


Elena Pope, MD, MsC
University of Toronto

Overview:

Mycosis fungoides is a rare type of skin cancer, increasingly diagnosed in pediatric populations. To date there is little knowledge on how to manage this condition in children, most guidance comes from adult data. We propose to form an international research group, ARMFUL (AppRoach Mycosis FUngoides in chiLdren) and come up with guidelines of care using a consensus approach. We believe that by including a large representation from all key specialists involved in the care of these patients as well as patients and their families, we would be able to have these guidelines widely adopted.

Status:

This was funded through a 2019 PeDRA Consensus Guidelines Grant.

Dilute bleach compresses to reduce the severity of radiation dermatitis in children with cancer

By

Principal Investigator

Amy Paller, MD
Northwestern University

Overview

Many years ago, we were able to show that dilute bleach baths reduce the severity of pediatric atopic dermatitis. This simple and inexpensive technique is now standard-of-care and has benefitted children worldwide. T. Leung showed that this same concentration of bleach has a direct anti-inflammatory effect on keratinocytes and reduces radiation dermatitis severity in a mouse model. Radiation dermatitis remains an issue for children (and adults) treated with irradiation for cancer. We propose a double-blind study of bleach compresses vs. water to reduce the severity in cancer patients. This study will be a two-site study at Northwestern/Lurie Children’s (Paller, Vivar, Yun, etc) and Harvard/Boston’s Children’s (Huang). We envision starting the compressing 1-week before and continuing daily during the danger period (several weeks). We have joined forces with radiation oncologists at both sites already and hope to recruit approximately equal numbers of patients at both sites. Parallel studies in children and adults will be performed.

Status

Protocol development is underway.

This project received a 2019 PeDRA Study Support Grant to develop a power analysis and the statistical plan for analysis at the end of the study utilizing an in-house statistician at Northwestern University.