Ponatinib is a tyrosine kinase inhibitor used in children with chronic myeloid leukemia. We report a case of neutrophilic panniculitis following ponatinib administration in a child. With the expanded use of immunomodulatory medications in the pediatric population, this report aims to expand knowledge regarding potential drug-related adverse events and the inclusion of ponatinib-related adverse events in the differential diagnosis of neutrophilic panniculitis.

Epidermolysis bullosa (EB) is a group of rare, fragile skin disorders that poses unique challenges. To support clinicians who lack familiarity with caring for patients with EB, we utilized a tool called an "OurPractice Advisory" (OPA) within Epic, our institution's electronic health record (EHR) platform. The OPA displays crucial resources for providers to assist with the management of patients with EB during an emergency department visit and/or hospitalization. The OPA includes practical guidance regarding wound care, supply procurement, consult recommendations, and complication prevention strategies. By leveraging an existing EHR tool, this initiative aims to provide immediate assistance to providers, enhance patient outcomes, and serve as a model for managing various complex conditions.

Epidermolysis bullosa (EB) is a chronic blistering skin disease with a propensity for bacterial colonization, critical colonization and/or infection, namely with Staphylococcus aureus and Pseudomonas aeruginosa. While P. aeruginosa cutaneous and systemic infection are associated with significant morbidity in the EB population, critical colonization poses its own risks, given its impairment of wound healing and the hypothesized association of flagellated bacteria with wound-induced skin cancer. There is a paucity of published literature describing topical management of P. aeruginosa colonization/critical colonization in patients with EB. The purpose of this review is to describe current and emerging treatment modalities for the topical management of P. aeruginosa colonization/critical colonization in patients with EB, extrapolating from current evidence-based practices in other skin conditions that may be relevant to patients with EB.

Dermatomyofibroma (DMF), rare in the pediatric population, is a benign dermal tumor composed of myofibroblasts and fibroblasts. We identified 21 histologically confirmed cases of pediatric DMF from our institution's dermatopathology database. Most cases occurred in male children, were located on the neck, and were difficult to distinguish clinically from lesions such as dermatofibromas and cysts. Following chart review of seven patients who received dermatologic care at our institution, we report unusual presentations of DMF and describe post-biopsy follow-up evaluations.

Staphylococcal scalded skin syndrome (SSSS) requires empiric anti-staphylococcal antibiotic management, selected based on regional rates of resistance. We report a retrospective chart review of the 58 patients diagnosed with SSSS through dermatology consult in our pediatric emergency department between 2015 and 2024. Only one patient had methicillin-resistant bacteria isolated on superficial bacterial culture, despite antibiogram data from our pediatric emergency department demonstrating methicillin resistance in 39.5%-46.8% of overall isolates growing Staphylococcus aureus. When selecting empiric antibiotic management for SSSS, providers should consider that institutional rates of MRSA in SSSS may differ from overall MRSA rates.

Children with medical complexity are at a higher risk of skin injury and wound development in the perioperative period. This article highlights the risk factors that predispose children with medical complexity to skin injury and outlines strategies for prevention of severe skin breakdown, infection and poor outcomes. Approaches to protect compromised skin and promote healing are explored, with emphasis on the unique challenges faced by pediatric patients with medical complexity.

Pediatric dermatologists serve as key members of multidisciplinary clinics (MDCs), during which providers from at least two specialties engage in a visit with a patient. Limited studies have explored the broader landscape of pediatric dermatology MDCs. A total of 39 pediatric dermatology division leaders (59% response rate) from the Society of Pediatric Dermatology completed a survey characterizing the structure, benefits, and challenges of these clinics. Survey results suggested that MDCs provide significant value in inter-specialty learning and complex patient management, but face challenges related to time, space, and financial burden.

The characteristics of epidermolysis bullosa (EB) demand higher than average provider support for transition from pediatric to adult care. We administered an online Qualtrics survey to members of the Epidermolysis Bullosa Clinical Research Consortium (EBCRC), a group of providers who care for patients with EB, in order to examine their practices and perspectives on transition of care (TOC) and identify barriers to successful implementation. Sixteen of eighteen medical centers completed the survey. Eighty-eight percent of center representatives expressed concerns about their patients transitioning/transferring from the pediatric to adult-centered care. Thirty-eight percent of providers reported having a formal TOC program in place. Our findings support the desire for formal TOC programs, the need for a team-based approach and, in particular, identification of adult providers to participate in the transition to improve this often challenging time.

Pediatric dermatologists are frequently consulted to evaluate children for cutaneous signs of systemic disorders. Numerical thresholds of significance have been described in the dermatologic literature for various skin findings where the likelihood of an associated extracutaneous abnormality or known genetic syndrome increases significantly. Knowledge of these numerical thresholds facilitates diagnosis and management, which improves clinical outcomes and avoids severe complications. This review highlights the clinical presentation, complications, evaluation, and numerical significance, when applicable, for the following skin findings: infantile hemangiomas, capillary malformations, café-au-lait macules, hypopigmented macules, juvenile xanthogranulomas, pilomatricomas, and angiofibromas.

BACKGROUND:Epidermolysis bullosa (EB), characterized by skin fragility and blistering, often requires hospitalization. Training for inpatient management of EB is limited, with no unified recommendations available in North America. OBJECTIVE:To develop consensus-derived best practices for hands-on inpatient management of EB in both the neonatal and postneonatal period. METHODS:A modified Delphi method (expert-based input via 2 surveys and a final review) was implemented. Available guidelines from EB Clinical Research Consortium centers were analyzed to determine areas of focus and formulate statements to be voted on by EB Clinical Research Consortium members, experienced EB nurses, and select family members. Study participants evaluated statements using a Likert scale: statements with at least 70% agreement were accepted; statements with 30% or more disagreement were rejected. RESULTS:Ten areas of focus were identified. Delphi participants included 15 dermatologists, 8 nurses, and 6 nonhealth care caregivers. Consensus was established on 103/119 neonatal statements and 105/122 postneonatal statements; no statements were rejected. Most recommendations applied to both age groups. LIMITATIONS:Recommendations may require adjustment based on individual patient's clinical context. CONCLUSION:Using the Delphi method, a consensus-derived resource for hospital-based health care professionals who manage patients with EB has been developed to improve the quality of inpatient care.

Central line dressings (CLDs) may be associated with adverse skin reactions in hospitalized children. Currently, standardized protocols to guide the management of cutaneous CLD reactions are unavailable at our children's hospital and in the pediatric literature. We surveyed dermatologists at multiple institutions who routinely perform pediatric consults to assess their management practices and/or the use of standardized protocols for addressing adverse cutaneous reactions to CLDs. All (n = 35) respondents reported receiving CLD-related consults, often involving interdisciplinary teams, yet most (66%) did not have standardized management protocols. When available, reported protocols for the management of CLD-associated skin reactions differed, including variable inclusion of chlorhexidine gluconate within polyurethane dressings and the use of patch testing for allergies or irritant reactions to applied products. Our findings support the need to further clarify patient and agent-specific factors predisposing to CLD-associated skin reactions and to develop and validate a multicenter protocol to optimize the management of CLD-associated skin reactions.

The most common bacteria isolated from wound cultures in patients recorded in the Epidermolysis Bullosa Clinical Characterization and Outcomes Database (EBCCOD) are Staphylococcus aureus and Pseudomonas aeruginosa. Given the prevalence of P. aeruginosa in this patient population and prior research implicating P. aeruginosa's potential role in carcinogenesis, we sought to further analyze patients with recorded wound cultures positive for Pseudomonas aeruginosa in the EBCCOD. We provide a descriptive analysis of this subset of patients and highlight potential avenues for future longitudinal studies that may have significant implications in our wound care management for patients with epidermolysis bullosa.

This study aims to examine transition of care (TOC) practices of multidisciplinary vascular anomalies centers (VACs). Thirty-seven of 71 VAC leaders to whom the survey was sent completed the questionnaire. TOC and transfer practices varied with only 16% of VACs having TOC programs. The most frequently cited barriers to developing a TOC program were lack of resources and difficulty finding expert adult providers.

BACKGROUND:Accurate diagnosis of epidermolysis bullosa (EB) has significant implications for prognosis, management, and genetic counseling.OBJECTIVE:To describe diagnostic testing patterns and assess diagnostic concordance of transmission electron microscopy (TEM), immunofluorescence mapping (IFM), and genetic analysis for EB.METHODS:A retrospective cohort included patients enrolled in the Epidermolysis Bullosa Clinical Characterization and Outcomes Database from January 1, 2004, to July 8, 2019. Tests concluding the same EB type (EB simplex, junctional EB, dominant dystrophic EB, and recessive dystrophic EB) were considered concordant; those concluding different EB types were considered discordant; and those with nonspecific/nondefinitive results were equivocal.RESULTS:A total of 970 diagnostic tests were conducted from 1984 to 2018 in 771 patients. Genetic analyses were performed chronologically later than IFM or TEM (P < .001). The likelihood of undergoing genetic analysis was greater for junctional EB and recessive dystrophic EB, and the same for dominant dystrophic EB as compared with EB simplex. TEM results in 163 patients were equivocal (55%), concordant (42%), and discordant (3%). IFM results in 185 patients were equivocal (54%), concordant (42%), and discordant (4%).LIMITATIONS:Retrospective design.CONCLUSIONS:Diagnostic testing has shifted in favor of genetic analysis. TEM and IFM frequently offer equivocal findings when compared to the specificity afforded by genetic analysis.

The evaluation of pediatric patients with subcutaneous nodules remains a diagnostic challenge. Pediatric dermatologists are regularly confronted with patients who have a nonspecific nodule. Though most masses that require evaluation are ultimately benign, the possibility of a more aggressive process, including borderline or malignant neoplasms, underscores the pivotal role of the pediatric dermatologist in recognizing these lesions. The aim of this review is to provide an overview of lumps and bumps that are important to recognize to prevent delay in diagnosis or treatment of a serious underlying condition. Clinical clues that may lead the pediatric dermatologist to have a higher index of suspicion for more aggressive lesions are reviewed. Suggestions for evaluation and workup, as well as tips for the difficult to discern lesion, are proposed.

We treated 6 patients (13-30 yrs of age) with confirmed RDEB in a multicenter, open-label Phase 2 study. PTR-01 3.0 mg/kg IV was given weekly x4, then every other week x7 with follow up assessments 1 and 3 months after treatment. Response was defined as improvement of ≥2 points on a 7-point wound impression scale (WIS) in a majority of lesions. Multiple secondary outcomes and safety were assessed. Five patients completed the study, 4 of whom were considered responders on the WIS. Over 75% of lesions (both chronic and recurrent) were closed by 50% or more at the end of treatment and remained so for at least 1 month after treatment. Wound surface area over time indexed to baseline (AUCi) showed a median decline of 46% at Day 43 of treatment in comparison to 25% in a control group and declined further to 70% at the end of treatment. Surveys of EB symptoms, impact, and patient and investigator global impressions, which comprised both cutaneous and systemic manifestations, showed rapid and persistent improvements during treatment. NC2 staining of skin biopsies showed extensive basement membrane incorporation of PTR-01 though no increase in anchoring fibrils by immunoelectron microscopy. Elevated skin fibrosis markers were markedly reduced. There were no serious or unexpected adverse events. One patient withdrew at Day 50 for lack of efficacy in association with high anti-C7 antibodies. Treatment with PTR-01 resulted in durable effects on both cutaneous and systemic manifestations of RDEB and reduced fibrosis markers in a majority of patients.