Heterogeneity of reported outcomes in epidermolysis bullosa clinical research: a scoping review as a first step towards outcome harmonization.

BACKGROUND: Epidermolysis bullosa (EB) is a rare, genetically and clinically heterogeneous group of skin fragility disorders. No cure is currently available, but many novel and repurposed treatments are upcoming. For adequate evaluation and comparison of clinical studies in EB, well-defined and consistent consensus-endorsed outcomes and outcome measurement instruments are necessary. OBJECTIVES: To identify previously reported outcomes in EB clinical research, group these outcomes by outcome domains and areas and summarize respective outcome measurement instruments. METHODS: A systematic literature search was performed in the databases MEDLINE, Embase, Scopus, Cochrane CENTRAL, CINAHL, PsycINFO and trial registries covering the period between January 1991 and September 2021. Studies were included if they evaluated a treatment in a minimum of three patients with EB. Two reviewers independently performed the study selection and data extraction. All identified outcomes and their respective instruments were mapped onto overarching outcome domains. The outcome domains were stratified according to subgroups of EB type, age group, intervention, decade and phase of clinical trial. RESULTS: The included studies (n = 207) covered a range of study designs and geographical settings. A total of 1280 outcomes were extracted verbatim and inductively mapped onto 80 outcome domains and 14 outcome areas. We found a steady increase in the number of published clinical trials and outcomes reported over the past 30 years. The included studies mainly focused on recessive dystrophic EB (43%). Wound healing was reported most frequently across all studies and referred to as a primary outcome in 31% of trials. Great heterogeneity of reported outcomes was observed within all stratified subgroups. Moreover, a diverse range of outcome measurement instruments (n = 200) was identified. CONCLUSIONS: We show substantial heterogeneity in reported outcomes and outcome measurement instruments in EB clinical research over the past 30 years. This review is the first step towards harmonization of outcomes in EB, which is necessary to expedite the clinical translation of novel treatments for patients with EB.

Risk stratification in adult and pediatric pulmonary arterial hypertension: A systematic review.

Introduction: Currently, risk stratification is the cornerstone of determining treatment strategy for patients with pulmonary arterial hypertension (PAH). Since the 2015 European Society of Cardiology/European Respiratory Society (ESC/ERS) guidelines for the diagnosis and treatment of pulmonary hypertension recommended risk assessment, the number of studies reporting risk stratification has considerably increased. This systematic review aims to report and compare the variables and prognostic value of the various risk stratification models for outcome prediction in adult and pediatric PAH. Methods: A systematic search with terms related to PAH, pediatric pulmonary hypertension, and risk stratification was performed through databases PubMed, EMBASE, and Web of Science up to June 8, 2022. Observational studies and clinical trials on risk stratification in adult and pediatric PAH were included, excluding case reports/series, guidelines, and reviews. Risk of bias was assessed using the Prediction model Risk Of Bias Assessment Tool. Data on the variables used in the models and the predictive strength of the models given by c-statistic were extracted from eligible studies. Results: A total of 74 studies were eligible for inclusion, with this review focusing on model development (n = 21), model validation (n = 13), and model enhancement (n = 9). The variables used most often in current risk stratification models were the non-invasive WHO functional class, 6-minute walk distance and BNP/NT-proBNP, and the invasive mean right atrial pressure, cardiac index and mixed venous oxygen saturation. C-statistics of current risk stratification models range from 0.56 to 0.83 in adults and from 0.69 to 0.78 in children (only two studies available). Risk stratification models focusing solely on echocardiographic parameters or biomarkers have also been reported. Conclusion: Studies reporting risk stratification in pediatric PAH are scarce. This systematic review provides an overview of current data on risk stratification models and its value for guiding treatment strategies in PAH. Systematic review registration: [https://www.crd.york.ac.uk/prospero/display_record.php?ID=CRD42022316885], identifier [CRD42022316885].

Benefits and drawbacks of videoconferencing for collaborating multidisciplinary teams in regional oncology networks: a scoping review.

INTRODUCTION: Various forms of videoconferenced collaborations exist in oncology care. In regional oncology networks, multidisciplinary teams (MDTs) are essential in coordinating care in their region. There is no recent overview of the benefits and drawbacks of videoconferenced collaborations in oncology care networks. This scoping review presents an overview of videoconferencing (VC) in oncology care and summarises its benefits and drawbacks regarding decision-making and care coordination. DESIGN: We searched MEDLINE, Embase, CINAHL (nursing and allied health) and the Cochrane Library from inception to October 2020 for studies that included VC use in discussing treatment plans and coordinating care in oncology networks between teams at different sites. Two reviewers performed data extraction and thematic analyses. RESULTS: Fifty studies were included. Six types of collaboration between teams using VC in oncology care were distinguished, ranging from MDTs collaborating with similar teams or with national or international experts to interactions between palliative care nurses and experts in that field. Patient benefits were less travel for diagnosis, better coordination of care, better access to scarce facilities and treatment in their own community. Benefits for healthcare professionals were optimised treatment plans through multidisciplinary discussion of complex cases, an ability to inform all healthcare professionals simultaneously, enhanced care coordination, less travel and continued medical education. VC added to the regular workload in preparing for discussions and increased administrative preparation. DISCUSSION: Benefits and drawbacks for collaborating teams were tied to general VC use. VC enabled better use of staff time and reduced the time spent travelling. VC equipment costs and lack of reimbursement were implementation barriers. CONCLUSION: VC is highly useful for various types of collaboration in oncology networks and improves decision-making over treatment plans and care coordination, with substantial benefits for patients and specialists. Drawbacks are additional time related to administrative preparation.