A DELPHI study priority setting the remaining challenges for the use of routinely collected data in trials: COMORANT-UK.

BACKGROUND: Researchers are increasingly seeking to use routinely collected data to support clinical trials. This approach has the potential to transform the way clinical trials are conducted in the future. The availability of routinely collected data for research, whether healthcare or administrative, has increased, and infrastructure funding has enabled much of this. However, challenges remain at all stages of a trial life cycle. This study, COMORANT-UK, aimed to systematically identify, with key stakeholders across the UK, the ongoing challenges related to trials that seek to use routinely collected data. METHODS: This three-step Delphi method consisted of two rounds of anonymous web-based surveys and a virtual consensus meeting. Stakeholders included trialists, data infrastructures, funders of trials, regulators, data providers and the public. Stakeholders identified research questions or challenges that they considered were of particular importance and then selected their top 10 in the second survey. The ranked questions were taken forward to the consensus meeting for discussion with representatives invited from the stakeholder groups. RESULTS: In the first survey, 66 respondents yielded over 260 questions or challenges. These were thematically grouped and merged into a list of 40 unique questions. Eighty-eight stakeholders then ranked their top ten from the 40 questions in the second survey. The most common 14 questions were brought to the virtual consensus meeting in which stakeholders agreed a top list of seven questions. We report these seven questions which are within the following domains: trial design, Patient and Public Involvement, trial set-up, trial open and trial data. These questions address both evidence gaps (requiring further methodological research) and implementation gaps (requiring training and/or service re-organisation). CONCLUSION: This prioritised list of seven questions should inform the direction of future research in this area and should direct efforts to ensure that the benefits in major infrastructure for routinely collected data are achieved and translated. Without this and future work to address these questions, the potential societal benefits of using routinely collected data to help answer important clinical questions will not be realised.

The importance of NOT being Other: Time to address the invisibility of nuanced gender and sexuality in clinical trials.

BACKGROUND: Representation of all members of society within research, especially those typically underserved, is needed to ensure that trial evidence applies to the relevant population, and that effective interventions are available to all. The lack of appropriate and representative options in demographic questions around sex, gender and sexuality may result in the exclusion of LGBTQIA + people from health research. MAIN BODY: Sex and gender are not the same, yet this is rarely recognised in trial data collection, with the terms sex and gender often being used interchangeably. Sex or gender is often used as a stratification factor at randomisation and/or to define sub-groups at the time of data analysis, so correct data collection is essential for producing high-quality science. Sexuality also suffers from 'othering' with identities not being acknowledged but simply provided as an alternative to the perceived main identities. When collecting sexuality information, it is important to consider the purposes of collecting this data. CONCLUSION: We call on those involved in trials to consider how sex, gender and sexuality data are collected, with an active consideration of inclusivity. Through the description of all non-straight, non-cisgender people as 'other' you may be ignoring the needs of these populations and doing science, yourself, and them a disservice. Inclusivity may require small but important changes to ensure your research findings are inclusive and develop the evidence base for often overlooked populations.

Expert consensus on the systemic treatment of atopic dermatitis in special populations.

With the increasing number of options for the treatment of moderate-to-severe atopic dermatitis, clinicians need guidance on a practical approach to selecting a systemic agent for specific patient populations. We convened an expert panel consisting of 12 members to conduct a literature review and summarize relevant data related to six scenarios of clinical interest: comorbid asthma, ocular surface disease, history of cancer, past and ongoing infections of interest (including herpes simplex virus, herpes zoster, hepatitis B, and tuberculosis), pregnancy and lactation, and the elderly. We performed a literature search and examined each clinical scenario with respect to three major categories of available systemic agents: traditional systemics (azathioprine, cyclosporine A, methotrexate, and mycophenolate mofetil), Janus kinase inhibitors (abrocitinib, baricitinib, and upadacitinib), and biologics (dupilumab, lebrikizumab, and tralokinumab). The expert panel and steering committee met virtually to review the data and discuss the drafted consensus statements. A modified Delphi process was used to arrive at a set of final consensus statements related to the systemic treatment of AD in these specific patient populations. To provide practical guidance on the choice of systemic therapy for atopic dermatitis in these six topics of clinical interest, 25 expert consensus statements and a summary of the supporting data are presented herein.

A Review of Fabry Disease.

Fabry disease (FD) is an X-linked lysosomal storage disease. A lack of alpha-galactosidase activity results in the accumulation of globotriaosylceramide in cells of various systems, leading to multi-systemic effects. The cutaneous hallmark of FD is a specific distribution of angiokeratoma. Other common symptoms include cornea verticillata, acroparesthesia, and sweating abnormalities. FD-specific symptoms, history, as well as examination of angiokeratoma can assist in the differential diagnosis. Enzyme replacement therapy is the current mainstay of treatment.

Occult cause of paraneoplastic acanthosis nigricans in a patient with known breast dcis: case and review.

Paraneoplastic acanthosis nigricans (pan) is an infrequently encountered cutaneous manifestation of internal malignancy. Here, we describe a case of pan in the setting of a known breast ductal carcinoma in situ, which, to our knowledge, had not been described in association with pan. As a result, thorough investigation was undertaken to search for another concurrent neoplasm that would better explain the development of pan. In so doing, we identified a coexisting metastatic cholangiocarcinoma. We thus conclude that when pan is observed in an uncommon association with a known malignancy, further investigation should be undertaken to explore whether a more likely occult culprit exists.