Multicenter Study of Long-Term Outcomes and Quality of Life in PHACE Syndrome after Age 10.

OBJECTIVE: To characterize long-term outcomes of PHACE syndrome. STUDY DESIGN: Multicenter study with cross-sectional interviews and chart review of individuals with definite PHACE syndrome ≥10 years of age. Data from charts were collected across multiple PHACE-related topics. Data not available in charts were collected from patients directly. Likert scales were used to assess the impact of specific findings. Patient-Reported Outcomes Measurement Information System (PROMIS) scales were used to assess quality of life domains. RESULTS: A total of 104/153 (68%) individuals contacted participated in the study at a median of 14 years of age (range 10-77 years). There were infantile hemangioma (IH) residua in 94.1%. Approximately one-half had received laser treatment for residual IH, and the majority (89.5%) of participants were satisfied or very satisfied with the appearance. Neurocognitive manifestations were common including headaches/migraines (72.1%), participant-reported learning differences (45.1%), and need for individualized education plans (39.4%). Cerebrovascular arteriopathy was present in 91.3%, with progression identified in 20/68 (29.4%) of those with available follow-up imaging reports. Among these, 6/68 (8.8%) developed moyamoya vasculopathy or progressive stenoocclusion, leading to isolated circulation at or above the level of the circle of Willis. Despite the prevalence of cerebrovascular arteriopathy, the proportion of those with ischemic stroke was low (2/104; 1.9%). PROMIS global health scores were lower than population norms by at least 1 SD. CONCLUSIONS: PHACE syndrome is associated with long-term, mild to severe morbidities including IH residua, headaches, learning differences, and progressive arteriopathy. Primary and specialty follow-up care is critical for PHACE patients into adulthood.

A core domain set for pyoderma gangrenosum trial outcomes: an international eDelphi and consensus study from the UPGRADE initiative.

BACKGROUND: Pyoderma gangrenosum (PG) is a rare ulcerative skin condition with no current standardized outcomes or outcome measures. With a rich investigational therapeutic pipeline, standardization of outcomes and improvement of data quality and interpretability will promote the appropriate and consistent evaluation of potential new therapies. Core outcome sets (COS) are agreed, standardized sets of outcomes that represent the minimum that should be measured and reported in all clinical trials of a specific condition. OBJECTIVES: To identify and reach a consensus on which domains (what to be measured) should be included in the Understanding Pyoderma Gangrenosum: Review and Analysis of Disease Effects (UPGRADE) core domain set for clinical trials in PG. METHODS: Collaborative discussions between patients and PG experts, and a systematic review of the literature identified items and prospective domains. A three-round international eDelphi exercise was performed to prioritize the domains and refine the provisional items (consensus: ≥ 70% of participants rating a domain as 'extremely important' and < 15% of participants voting 'not important'), followed by an international meeting to reach consensus on the core domain set (consensus: < 30% disagreement). Item-generation discussions and consensus meetings were hosted via online videoconferences. The eDelphi exercise and consensus voting were performed using Qualtrics survey software. Participants were adults with PG, healthcare professionals, researchers and industry representatives. RESULTS: Collaborative discussions and systematic reviews yielded 115 items, which were distilled into 15 prospective domains. The eDelphi exercise removed the three lowest-priority domains ('laboratory tests', 'treatment costs' and 'disease impact on family') and ranked 'pain', 'quality of life' and 'physical symptoms' as the highest-priority prospective domains. Consensus was reached on the domains of 'pain', 'quality of life' and 'clinical signs'. The domain of 'disease course/disease progression' narrowly failed to reach consensus for inclusion in the core set (32% of participants voted 'no'). Refinement of this domain definition will be required and presented for consideration at future consensus meetings. CONCLUSIONS: The UPGRADE core domain set for clinical trials in PG has been agreed by international multistakeholder consensus. Future work will develop and/or select outcome measurement instruments for these domains to establish a COS.

The Eczema Area and Severity Index-A Practical Guide.

ABSTRACT: Atopic dermatitis is a chronic inflammatory skin condition that affects approximately 18 million people in the United States. Assessing the extent and severity of atopic dermatitis is critical for determining baseline disease burden and treatment effectiveness for both investigators and clinicians. Considerable efforts over the past several decades have been made in developing a highly validated instrument called the Eczema Area and Severity Index (EASI). Although several guides exist for the EASI, questions continue to arise regarding its use and interpretation. This review was developed to serve as the definitive guide for the EASI and to address commonly asked questions.

Shared Decision-making in Dermatology: A Scoping Review.

Importance: Shared decision-making (SDM) can improve the quality of care for patients. The extent to which this tool has been used and the evidence supporting its use in dermatology have not been systematically examined. Objective: To perform a scoping review of the literature regarding SDM in dermatology. Evidence Review: Searches of Ovid MEDLINE, PsycINFO, PsycARTICLES, Sciverse Scopus, and EBM Reviews were conduced on July 11, 2019, and March 6, 2020. There were no limits on date, type of article, language, or subject for the initial search. A total of 1673 titles and abstracts were screened by 2 independent reviewers in the Covidence mixed-methods platform. Forty-one full-text studies were assessed for eligibility. For inclusion, articles needed to include a dermatologic diagnosis as well as discussion of SDM or patient decision aids. Two independent reviewers screened 29 full-text articles for inclusion and extracted qualitative data using a set of 26 predefined codes. Qualitative coding was applied to excerpts to categorize the article, define and describe advantages and disadvantages of SDM, understand patient and physician requests for SDM, and discuss methods of implementation. Findings: Despite a small number of articles on SDM (n = 29) in dermatology, the selected literature provided consistent messages regarding the importance of SDM for dermatology and a number of strategies and tools for implementation. Medical dermatology was the most common subspecialty studied, with melanoma, psoriasis, and connective tissue diseases most examined. Only 5 publications introduced SDM tools specifically for dermatologic conditions; of these, only 2 tools were validated. Barriers to implementation that were cited included time and a lack of training for clinicians, although the literature also provided potential solutions to these issues. All articles emphasized the value of SDM for both patients and physicians. Conclusions and Relevance: The literature regarding SDM in dermatology consistently suggests that it is a useful tool for providing patient-centered care. Established tools have been proposed since 2012. More research is needed to implement better practices, especially in dermatologic subspecialties. However, there are substantial suggestions from the literature for strategies and tools with which to begin a shared decision-making practice.

Early Outcomes in an Emerging Facial Nerve Center: The Oregon Health and Science University (OHSU) Experience.

OBJECTIVES: Nerve transfer (NT) and free gracilis muscle transfer (FGMT) are procedures for reanimation of the paralyzed face. Assessing the surgical outcomes of these procedures is imperative when evaluating the effectiveness of these interventions, especially when establishing a new center focused on the treatment of patients with facial paralysis. We desired to discuss the factors to consider when implementing a facial nerve center and the means by which the specialist can assess and analyze outcomes. METHODS: Patients with facial palsy secondary to multiple etiologies, including cerebellopontine angle tumors, head and neck carcinoma, and trauma, who underwent NT or FGMT between 2014 and 2019 were included. Primary outcomes were facial symmetry and smile excursion, calculated using FACE-gram and Emotrics software. Subjective quality of life outcomes, including the Facial Clinimetric Evaluation (FaCE) Scale and Synkinesis Assessment Questionnaire (SAQ), were also assessed. RESULTS: 14/22 NT and 6/6 FGMT patients met inclusion criteria having both pre-and postoperative photo documentation. NT increased oral commissure excursion from 0.4 mm (SD 5.3) to 2.9 mm (SD 6.8) (P = 0.05), and improved symmetry of excursion (P < 0.001) and angle (P < 0.001). FGMT increased oral commissure excursion from -1.4 mm (SD 3.9) to 2.1 mm (SD 3.7), (P = 0.02), and improved symmetry of excursion (P < 0.001). FaCE scores improved in NT patients postoperatively (P < 0.001). CONCLUSIONS: Measuring outcomes, critical analyses, and a multidisciplinary approach are necessary components when building a facial nerve center. At our emerging facial nerve center, we found NT and FGMT procedures improved smile excursion and symmetry, and improved QOL following NT in patients with facial palsy secondary to multiple etiologies.

A Therapeutic Renaissance - Emerging Treatments for Atopic Dermatitis.

Atopic dermatitis (AD) is a chronic, inflammatory cutaneous disease that is characterized by complex immune dysregulation and skin barrier dysfunction with a wide variety of clinical phenotypes. Until recently, conventional therapeutic modalities for AD remained rather non-specific despite AD's complex etiology. Failing to take into account the underlying inflammatory pathways led to treatments with inadequate efficacy or unacceptable long-term toxicities. We are currently in the midst of a therapeutic renaissance in AD. Recent progress in molecular medicine provides us a better understanding of the AD pathogenesis, suggesting a dominant helper T cell (Th) 2/Th22 response with a varying degree of Th1/Th17 overexpression. Targeted therapeutic agents including biologics and small molecule inhibitors in development hold promises for more effective and safer therapeutic approaches for AD. A better understanding of individual differences amongst AD patients will allow for a more tailored approach in the future. This review aims to cover the most promising emerging therapies in the field of atopic dermatitis utilizing recently published manuscripts and up-to-date conference abstracts and presentations.

New and Emerging Biologics for Atopic Dermatitis.

Atopic dermatitis (AD) is a chronic inflammatory skin disease that is characterized by complex pathophysiology involving both skin barrier dysfunction and aberrant type 2 inflammation/immune responses. AD can be a debilitating condition that drastically impairs quality of life, especially in patients with moderate-to-severe disease. Currently, topical therapies such as corticosteroids and non-steroidal immunomodulatory therapy provide limited efficacy for patients with moderate-to-severe AD; limitations include inadequate response, cutaneous toxicity from overuse, and poor tolerance due to stinging and burning. Historically, the development of targeted therapies has been challenging due to the complex and multifaceted etiology of AD. Recent progress in understanding the immunopathology of AD reinforces the development of newly targeted therapeutics. The successful launch of dupilumab, a monoclonal antibody targeting the interleukin (IL)-4α receptor subunit, for AD in 2017 spurred the development of a number of biologics targeting novel cytokine and receptor targets that are now in phase II and III of development. This review aims to explore the rationale behind these novel biological therapies and to summarize current clinical studies of these agents.