Development of standard clinical endpoints for use in dengue interventional trials: introduction and methodology

Autor: Liane Agulto, Thomas Jaenisch, Stephen J. Thomas, Morgan A. Marks, Walla Dempsey, Catherine A. Laughlin, Federico Narvaez, Annelies Wilder-Smith, Michael P. Fay, Maïna L’Azou, Duane J. Gubler, João Bosco Siqueira, Kay M. Tomashek, Martin Erpicum, Rémy Teyssou, Yee Sin Leo, M. Cristina Cassetti, Hasitha Tissera, Kim Hendrickx, Derek Wallace
Rok vydání: 2018
Předmět:
Delphi Technique
Epidemiology
Psychological intervention
Delphi method
VACCINE
Disease
Dengue
610 Medical sciences Medicine
0302 clinical medicine
Outcome Assessment
Health Care
Validation
Clinical endpoint
030212 general & internal medicine
Severe dengue
Clinical Trials as Topic
lcsh:R5-920
Hospitalization
Therapeutic
lcsh:Medicine (General)
Life Sciences & Biomedicine
Research Article
medicine.medical_specialty
Endpoint Determination
030231 tropical medicine
Dengue Vaccines
Intervention
Health Informatics
Pathophysiology
CLASSIFICATION
03 medical and health sciences
medicine
Humans
AMERICA
Intensive care medicine
Dengue vaccine
DELPHI
Science & Technology
business.industry
Public health
Reproducibility of Results
EFFICACY
Standardization
Health Care Sciences & Services
Clinical research
Endpoints
CONSENSUS
business
Vaccine
Zdroj: BMC Medical Research Methodology, Vol 18, Iss 1, Pp 1-10 (2018)
BMC Medical Research Methodology
ISSN: 1471-2288
DOI: 10.1186/s12874-018-0601-z
Popis: BACKGROUND: As increasing numbers of dengue vaccines and therapeutics are in clinical development, standardized consensus clinical endpoint definitions are urgently needed to assess the efficacy of different interventions with respect to disease severity. We aimed to convene dengue experts representing various sectors and dengue endemic areas to review the literature and propose clinical endpoint definitions for moderate and severe disease based on the framework provided by the WHO 2009 classification. METHODS: The endpoints were first proposed and discussed in a structured expert consultation. After that, the Delphi method was carried out to assess the usefulness, validity and feasibility of the standardized clinical disease endpoints for interventional dengue research. RESULTS: Most respondents (> 80%) agreed there is a need for both standardized clinical endpoints and operationalization of severe endpoints. Most respondents (67%) felt there is utility for moderate severity endpoints, but cited challenges in their development. Hospitalization as a moderate endpoint of disease severity or measure of public health impact was deemed to be useful by only 47% of respondents, but 89% felt it could bring about supplemental information if carefully contextualized according to data collection setting. Over half of the respondents favored alignment of the standard endpoints with the WHO guidelines (58%), but cautioned that the endpoints could have ramifications for public health practice. In terms of data granularity of the endpoints, there was a slight preference for a categorical vs numeric system (e.g. 1-10) (47% vs 34%), and 74% of respondents suggested validating the endpoints using large prospective data sets. CONCLUSION: The structured consensus-building process was successful taking into account the history of the debate around potential endpoints for severe dengue. There is clear support for the development of standardized endpoints for interventional clinical research and the need for subsequent validation with prospective data sets. Challenges include the complexity of developing moderate disease research endpoints for dengue. ispartof: BMC MEDICAL RESEARCH METHODOLOGY vol:18 issue:1 ispartof: location:England status: published
Databáze: OpenAIRE
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