Suchergebnisse

Funding: KG was supported by an MRC Methodology Research Fellowship (MR/L01193X/1). The views and opinions expressed therein are those of the authors and do not necessarily reflect those of the MRC. PRW was funded by the MRC North West Hub for Trials Methodology Research (MR/K025635/1) and the MRC/NIHR Trials Methodology Research Partnership (MR/S014357/1). The Health Services Research Unit is core-funded by the Chief Scientist Office of the Scottish Government Health and Social Care Directorates (CZU/3/3). Acknowledgements: The ELICIT Study team would like to thank Cynthia Fraser for help with developing and running the search strategies for the literature review, the DelphiManager team for all their support and guidance on the use of the Delphi platform, Health Services Research Unit Patient Involvement Group critical review of the Delphi questionnaire before dissemination, Heather Bagley for further comments on the Delphi questionnaire and also for dissemination of the survey information and link to a range of patient facing organisations, and to Beverley Smith for her assistance in organising the consensus meeting. We would also like to thank all participants of the study (including interview participants, Delphi respondents, and consensus meeting members) and organisations who disseminated the survey. Data Sharing: Data from the systematic review and Delphi survey phases of work are available from the corresponding author on request. ; Peer reviewed ; Publisher PDF

© The Author(s). ; [Background]: Acute myeloid leukemia (AML) is the most common acute leukemia in adults and has an unacceptably low cure rate. In recent years, a number of new treatment strategies and compounds were developed for the treatment of AML. There were several randomized controlled clinical trials with the objective to improve patients' management and patients' outcome in AML. Unfortunately, these trials are not always directly comparable since they do not measure the same outcomes, and currently there are no core outcome sets that can be used to guide outcome selection and harmonization in this disease area. The HARMONY (Healthcare Alliance for Resourceful Medicine Offensive against Neoplasms in Hematology) Alliance is a public-private European network established in 2017 and currently includes 53 partners and 32 associated members from 22 countries. Amongst many other goals of the HARMONY Alliance, Work Package 2 focuses on defining outcomes that are relevant to each hematological malignancy. Accordingly, this pilot study will be performed to define a core outcome set in AML. ; [Methods]: The pilot study will use a three-round Delphi survey and a final consensus meeting to define a core outcome set. Participants will be recruited from different stakeholder groups, including patients, clinicians, regulators and members of the European Federation of Pharmaceutical Industries and Associations. At the pre-Delphi stage, a literature research was conducted followed by several semi-structured interviews of clinical public and private key opinion leaders. Subsequently, the preliminary outcome list was discussed in several multi-stakeholder face-to-face meetings. The Delphi survey will reduce the preliminary outcome list to essential core outcomes. After completion of the last Delphi round, a final face-to-face meeting is planned to achieve consensus about the core outcome set in AML. ; [Discussion]: As part of the HARMONY Alliance, the pilot Delphi aims to define a core outcome set in AML on the basis of a multi-stakeholder consensus. Such a core outcome set will help to allow consistent comparison of future clinical trials and real-world evidence research and ensures that appropriate outcomes valued by a range of stakeholders are measured within future trials. ; This work was funded through the Innovative Medicines Initiative 2 joint undertaking and listed under grant agreement 116026. This joint undertaking receives support from the European Union's Horizon 2020 research and innovation program and the EFPIA. An additional cash grant was obtained from Bayer AG for conducting the Delphi survey development

Acknowledgements: We are thankful to all the patients and health care professionals who took part in our interview studies and to the patient representatives and advocates who provided insight during our various consensus meetings. We are grateful to Dr. Susie Dodd for her advice in applying the 38-item outcome taxonomy which she and her COMET colleagues developed, and we accept responsibility for any (mis)application. PIONEER is funded through the IMI2 Joint Undertaking and is listed under grant agreement No. 777492. This joint under- taking receives support from the European Union's Horizon 2020 research and innovation programme and EFPIA. ; Peer reviewed ; Publisher PDF

Objectives: The outcomes reported in trials in coronavirus disease 2019 are extremely heterogeneous and of uncertain patient relevance, limiting their applicability for clinical decision-making. The aim of this workshop was to establish a core outcomes set for trials in people with suspected or confirmed coronavirus disease 2019. Design: Four international online multistakeholder consensus workshops were convened to discuss proposed core outcomes for trials in people with suspected or confirmed coronavirus disease 2019, informed by a survey involving 9,289 respondents from 111 countries. The transcripts were analyzed thematically. The workshop recommendations were used to finalize the core outcomes set. Setting: International. Subjects: Adults 18 years old and over with confirmed or suspected coronavirus disease 2019, their family members, members of the general public and health professionals (including clinicians, policy makers, regulators, funders, researchers). Interventions: None. Measurements: None. Main Results: Six themes were identified. "Responding to the critical and acute health crisis" reflected the immediate focus on saving lives and preventing life-threatening complications that underpinned the high prioritization of mortality, respiratory failure, and multiple organ failure. "Capturing different settings of care" highlighted the need to minimize the burden on hospitals and to acknowledge outcomes in community settings. "Encompassing the full trajectory and severity of disease" was addressing longer term impacts and the full spectrum of illness (e.g. shortness of breath and recovery). "Distinguishing overlap, correlation and collinearity" meant recognizing that symptoms such as shortness of breath had distinct value and minimizing overlap (e.g. lung function and pneumonia were on the continuum toward respiratory failure). "Recognizing adverse events" refers to the potential harms of new and evolving interventions. "Being cognizant of family and psychosocial wellbeing" reflected the pervasive impacts of coronavirus disease 2019. Conclusions: Mortality, respiratory failure, multiple organ failure, shortness of breath, and recovery are critically important outcomes to be consistently reported in coronavirus disease 2019 trials. ; Flinders University National Coronavirus Disease 2019 Clinical Evidence Taskforce Australian Government CGIAR Victorian Department of Health and Human Services Ian Potter Foundation Walter Cottman Endowment Fund Lord Mayor's Charitable Foundation

Objectives: There are over 4,000 trials conducted in people with coronavirus disease 2019. However, the variability of outcomes and the omission of patient-centered outcomes may diminish the impact of these trials on decision-making. The aim of this study was to generate a consensus-based, prioritized list of outcomes for coronavirus disease 2019 trials. Design: In an online survey conducted in English, Chinese, Italian, Portuguese, and Spanish languages, adults with coronavirus disease 2019, their family members, health professionals, and the general public rated the importance of outcomes using a 9-point Likert scale (7-9, critical importance) and completed a Best-Worst Scale to estimate relative importance. Participant comments were analyzed thematically. Setting: International. Subjects: Adults 18 years old and over with confirmed or suspected coronavirus disease 2019, their family members, members of the general public, and health professionals (including clinicians, policy makers, regulators, funders, and researchers). Interventions: None. Measurements: None. Main Results: In total, 9,289 participants from 111 countries (776 people with coronavirus disease 2019 or family members, 4,882 health professionals, and 3,631 members of the public) completed the survey. The four outcomes of highest priority for all three groups were: mortality, respiratory failure, pneumonia, and organ failure. Lung function, lung scarring, sepsis, shortness of breath, and oxygen level in the blood were common to the top 10 outcomes across all three groups (mean > 7.5, median >= 8, and > 70% of respondents rated the outcome as critically important). Patients/family members rated fatigue, anxiety, chest pain, muscle pain, gastrointestinal problems, and cardiovascular disease higher than health professionals. Four themes underpinned prioritization: fear of life-threatening, debilitating, and permanent consequences; addressing knowledge gaps; enabling preparedness and planning; and tolerable or infrequent outcomes. Conclusions: Life-threatening respiratory and other organ outcomes were consistently highly prioritized by all stakeholder groups. Patients/family members gave higher priority to many patient-reported outcomes compared with health professionals. ; Flinders University National Coronavirus Disease 2019 Clinical Evidence Taskforce Australian Government CGIAR Victorian Department of Health and Human Services Ian Potter Foundation Walter Cottman Endowment Fund Lord Mayor's Charitable Foundation

ABSTRACT Objective: There are over 4000 trials conducted in people with COVID-19. However, the variability of outcomes and the omission of patient-centered outcomes may diminish the impact of these trials on decision-making. The aim of this study was to generate a consensus-based, prioritized list of outcomes for COVID-19 trials. Design: In an online survey conducted in English, Chinese, Italian, Portuguese and Spanish languages, adults with COVID-19, their family members, health professionals and the general public rated the importance of outcomes using a 9-point Likert scale (7-9, critical importance) and completed a Best-Worst Scale to estimate relative importance. Participant comments were analysed thematically. Setting: International Participants: Adults aged 18 years and over with confirmed or suspected COVID-19, their family members, members of the general public and health professionals (including clinicians, policy makers, regulators, funders, researchers). Main Results: In total, 9289 participants from 111 countries (776 people with COVID-19 or family members, 4882 health professionals, and 3631 members of the public) completed the survey. The four outcomes of highest priority for all three groups were: mortality, respiratory failure, pneumonia and organ failure. Lung function, lung scarring, sepsis, shortness of breath, and oxygen level in the blood were common to the top 10 outcomes across all three groups (mean >7.5, median ≥ 8, and >70% of respondents rated the outcome as critically important). Patients/family members rated fatigue, anxiety, chest pain, muscle pain, gastrointestinal problems and cardiovascular disease higher than health professionals. Four themes underpinned prioritization: fear of life-threatening, debilitating and permanent consequences; addressing knowledge gaps; enabling preparedness and planning; and tolerable or infrequent outcomes. Conclusions: Life-threatening respiratory and other organ outcomes were consistently highly prioritized by all stakeholder groups. Patients/family members gave higher priority to many patient-reported outcomes compared with health professionals. ; The project is funded by the Flinders University and the National COVID-19 Clinical Evidence Taskforce, convened by the Australian Living Evidence Consortium, hosted by Cochrane Australia, School of Public Health and Preventive Medicine, Monash University supported by the Australian Government, Victorian Department of Health and Human Services, Ian Potter Foundation, Walter Cottman Endowment Fund (managed by Equity Trustees) and the Lord Mayor's Charitable Foundation). AT is supported by The University of Sydney Robinson Fellowship. ACM is supported by a Clinical Research Career Development Fellowship from the Wellcome Trust (WT 2055214/Z/16/Z)