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Supplemental material, sj-docx-1-ctj-10.1177_17407745231182417 for Data monitoring committees in pediatric randomized controlled trials registered in ClinicalTrials.gov by Tiago Machado, Beatrice Mainoli, Daniel Caldeira, Joaquim J Ferreira and Ricardo M Fernandes in Clinical Trials

Objectives The objective of this study was to develop a core outcome set (COS) for use in future clinical trials in bronchiolitis. We wanted to find out which outcomes are important to healthcare professionals (HCPs) and to parents and which outcomes should be prioritised for use in future clinical trials.Design and setting The study used a systematic review, workshops and interviews, a Delphi survey and a final consensus workshop.Results Thirteen parents and 45 HCPs took part in 5 workshops; 15 other parents were also separately interviewed. Fifty-six items were identified from the systematic review, workshops and interviews. Rounds one and two of the Delphi survey involved 299 and 194 participants, respectively. Sixteen outcomes met the criteria for inclusion within the COS. The consensus meeting was attended by 10 participants, with representation from all three stakeholder groups. Nine outcomes were added, totalling 25 outcomes to be included in the COS.Conclusion We have developed the first parent and HCP consensus on a COS for bronchiolitis in a hospital setting. The use of this COS will ensure outcomes in future bronchiolitis trials are important and relevant, and will enable the trial results to be compared and combined.

Objective We surveyed the dissemination and use of point-of-care ultrasound (POCUS), physician training levels, and barriers and limitations to use of POCUS among pediatricians and pediatric emergency medicine (PEM) physicians across Europe and Israel. Methods A questionnaire was distributed through the PEM section of the European Society for Emergency Medicine and the Research in European Pediatric Emergency Medicine Network. Results A total of 581 physicians from 22 countries fully completed the questionnaire. Participants were primarily pediatric attending physicians (34.9% [203 of 581]) and PEM attending physicians (28.6% [166 of 581]). Most of the respondents, 58.5% (340 of 581), reported using POCUS in their practice, and 61.9% (359/581) had undergone POCUS training. Point-of-care ultrasound courses represented the most common method of becoming proficient in POCUS. Overall, the Focused Assessment with Sonography in Trauma scan was the mostly taught application, with 76.3% (274 of 359). Resuscitative, diagnostic, and procedural POCUS were rated as very useful or useful by the most of respondents. The lack of qualified personnel to train (76.9% [447 of 581]), and the insufficient time for physicians to learn, POCUS (63.7% [370 of 581]) were identified as the main limitations to POCUS implementation. Conclusions The dissemination of pediatric POCUS in the European and Israeli centers we surveyed is limited, and its applications are largely restricted to the Focused Assessment with Sonography in Trauma examination. This is likely related to lack of training programs. In contrast, the potential value of use of POCUS in PEM practice is recognized by the majority of respondents.

Madeleine Gray-Ffrench,1 Ricardo M Fernandes,2,3 Ian P Sinha,4,5 Elissa M Abrams6,7 1University of Birmingham, Faculty of Medicine, Birmingham, UK; 2Clinical Pharmacology Unit, Faculty of Medicine and Instituto de Medicina Molecular, Universidade de Lisboa, Lisbon, Portugal; 3Department of Pediatrics, Hospital de Santa Maria, Centro Hospitalar Universitário de Lisboa Norte, Lisbon, Portugal; 4Alder Hey Children’s Hospital, Liverpool, UK; 5Department of Women’s and Children’s Health, Faculty of Health and Life Sciences, University of Liverpool, Liverpool, UK; 6Department of Pediatrics, Section of Allergy and Clinical Immunology, University of Manitoba, Winnipeg, Canada; 7Department of Pediatrics, Division of Allergy and Immunology, University of British Columbia, Vancouver, British Columbia, CanadaCorrespondence: Elissa M Abrams, Department of Pediatrics, Section of Allergy and Clinical Immunology, University of Manitoba, FE125-685 William Avenue, Winnipeg, MB, R2A 5L9, Canada, Tel +1 204-255-7650, Fax +1 204-254-0730, Email elissa.abrams@gmail.comAbstract: Children exposed to various indoor and outdoor allergens are placed at an increased risk of developing asthma in later life, with sensitization in these individuals being a strong predictor of disease morbidity. In addition, aeroallergen exposure influences asthma outcomes through an interplay with adverse determinants of health. The goal of this review is to provide an introductory overview of factors related to aeroallergen exposure in type 2-high childhood asthma. These include the relevance of exposure in asthma exacerbations and severity, and the evidence-base for avoidance and treatment for sensitization to these allergens. This review will focus on both indoor aeroallergens (house dust mite, pet, cockroach, mold, and rodent) and outdoor aeroallergens (pollens and molds). Treatment of aeroallergen sensitization in children with asthma includes avoidance and removal measures, although there is limited evidence of clinical benefit especially with single-strategy approaches. We will also address the interplay of aeroallergens and climate change, adverse social determinants, and the current COVID-19 pandemic, when we have seen a dramatic reduction in asthma exacerbations and emergency department visits among children. While there are many factors that are hypothesized to contribute to this reduction, among them is a reduced exposure to outdoor seasonal aeroallergens.Keywords: asthma, aeroallergens, social determinants of health, COVID-19

Additional file 2. PRISMA Checklist-Systematic reviews and meta-analyses published in indexed Portuguese Medical journals: time trends and critical appraisal.

Additional file 1: Search strategy, study selection and risk of bias.

© The Author(s) 2022. Open Access This article is licensed under a Creative Commons Attribution-NonCommercial 4.0 International License, which permits any non-commercial use, sharing, adaptation, distribution and reproduction in any medium or format, as long as you give appropriate credit to the original author(s) and the source, provide a link to the Creative Commons licence, and indicate if changes were made. The images or other third party material in this article are included in the article's Creative Commons licence, unless indicated otherwise in a credit line to the material. If material is not included in the article's Creative Commons licence and your intended use is not permitted by statutory regulation or exceeds the permitted use, you will need to obtain permission directly from the copyright holder. To view a copy of this licence, visit http://creativecommons.org/licenses/by-nc/4.0/., Cancer diagnosis and therapeutics have been traditionally based on pathologic classification at the organ of origin. The availability of an unprecedented amount of clinical and biologic data provides a unique window of opportunity for the development of new drugs. What was once treated as a homogeneous disease with a one-size-fits-all approach was shown to be a rather heterogeneous condition, with multiple targetable mutations that can vary during the course of the disease. Clinical trial designs have had to adapt to the exponential growth of targetable mechanisms and new agents, with ensuing challenges that are closer to those experienced with rare diseases and orphan medicines. To face these problems, precision/enrichment and other novel trial designs have been developed, and the concept of histology-agnostic targeted therapeutic agents has emerged. Patients are selected for a specific agent based on specific genomic or molecular alterations, with the same compound used to potentially treat a multiplicity of cancers, granted that the actionable driver alteration is present. There are currently approved drugs for such indications, but this approach has raised issues on multiple levels. This review aims to address the challenges of this new concept and provide insights into possible solutions and frameworks on how to tackle them.

Additional file 2: Analyses of exercise time (measured as mean difference, and subgroup analyses), CCS class change and anginal episodes.

Additional file 4: Sensitivity analyses and meta-regressions.

Additional file 3: Publication bias.

© The Author(s) 2022. Open Access This article is licensed under a Creative Commons Attribution 4.0 International License, which permits use, sharing, adaptation, distribution and reproduction in any medium or format, as long as you give appropriate credit to the original author(s) and the source, provide a link to the Creative Commons licence, and indicate if changes were made. The images or other third party material in this article are included in the article’s Creative Commons licence, unless indicated otherwise in a credit line to the material. If material is not included in the article’s Creative Commons licence and your intended use is not permitted by statutory regulation or exceeds the permitted use, you will need to obtain permission directly from the copyright holder. To view a copy of this licence, visit http://creativecommons.org/licenses/by/4.0/. The Creative Commons Public Domain Dedication waiver (http://creativeco mmons.org/publicdomain/zero/1.0/) applies to the data made available in this article, unless otherwise stated in a credit line to the data., Introduction: Over the last years, the number of systematic reviews published is steadily increasing due to the global interest in this type of evidence synthesis. However, little is known about the characteristics of this research published in Portuguese medical journals. This study aims to evaluate the publication trends and overall quality of these systematic reviews. Material and methods: This was a methodological study. We aimed the most visible Portuguese medical journals indexed in MEDLINE. Systematic reviews were identified through an electronic search (through PUBMED). We included systematic reviews published up to August 2020. Systematic reviews selection and data extraction were done independently by three authors. The overall quality critical appraisal using the A MeaSurement Tool to Assess systematic Reviews (AMSTAR-2) was independently assessed by three authors. Disagreements were solved by consensus. Results: Sixty-six systematic reviews published in 5 Portuguese medical journals were included. Most (n = 53; 80.3%) were systematic reviews without meta-analysis. Up to 2010 there was a steady increase in the number of systematic reviews published, followed by a period of great variability of publication, ranging from 1 to 10 in a given year. According to the systematic reviews' typology, most have been predominantly conducted to assess the effectiveness/efficacy of health interventions (n = 27; 40.9%). General and Internal Medicine (n = 20; 30.3%) was the most addressed field. Most systematic reviews (n = 46; 69.7%) were rated as being of "critically low-quality". Conclusions: There were consistent flaws in the methodological quality report of the systematic reviews included, particularly in establishing a prior protocol and not assessing the potential impact of the risk of bias on the results. Through the years, the number of systematic reviews published increased, yet their quality is suboptimal. There is a need to improve the reporting of systematic reviews in Portuguese medical journals, which can be achieved by better adherence to quality checklists/tools.