Exploring Clinical Trialists’ Perspectives on Core Outcome Sets for Immune Thrombocytopenic Purpura (ITP) Trials: A Web-Based Survey Study

Introduction: Immune Thrombocytopenic Purpura (ITP) is a complex autoimmune disorder associated with a low platelet count, and elevated bleeding risk. Conducting clinical trials in ITP is vital for comprehending the disease and improving patient outcomes. However, diverse outcome measures across trials impede result comparison, necessitating the use of Core Outcome Sets (COS). COS are specific trial outcomes for uniform measurement and reporting, aiming to enhance research quality, evidence synthesis, and patient-centered outcomes. Despite potential benefits, COS adoption in the ITP clinical trial community remains inconsistent. This cross-sectional, web-based survey investigates clinical trialists' perceptions, knowledge, and usage patterns of COS in ITP research. Our objective is to obtain insight on the awareness, integration, barriers, and recommendations for ITP COS. This study's insights aim to inform strategies for enhancing COS adoption, advancing standardization in ITP trials, and optimizing therapeutic intervention evidence.

Methods: Using a sample of ITP trials from a previous study, eligible participants are identified from the 'Contacts and Locations' section of ClinicalTrials.gov registry profiles. The survey, conducted via REDCap, consists of 20 questions covering demographic information, COS familiarity, and practical experiences with COS. Questions vary in format, including multiple-choice, Likert scale, and open-ended, allowing for both quantitative and qualitative data collection. Informed by the COMET Initiative's core outcome set database, the survey addresses awareness, knowledge, use, and perceptions of COS in ITP trials. Data collection occurs through an eight-week email-based process, ensuring participant anonymity and confidentiality. The analysis, combining descriptive and inferential statistics, as well as thematic analysis of qualitative data, aims to provide a comprehensive understanding of clinical trialists' perspectives on COS in ITP trials. Data analysis will include descriptive and inferential statistics, as well as qualitative data obtained from open-ended questions.

Results: Data is currently in the collection phase of this study. Analysis of survey responses will include:
1. Descriptive statistics: summarizing patient demographics and responses to close-ended questions.
2. Inferential statistics: examples include chi-square tests and t-tests which may be used to identify relationships between variables or differences among subgroups.
3. Qualitative data: derived from responses to open-ended questions, which will undergo thematic analysis to discern recurring themes and patterns.

Conclusion: Upon concluding this study, our findings will provide insights into the utilization and awareness of Core Outcome Sets among clinical trialists. The knowledge acquired from this research may establish a basis for forthcoming initiatives and interventions dedicated to improving the adoption of COS within the clinical trial community. Furthermore, these outcomes have the potential to foster consistency in clinical reporting of COS, ultimately contributing to enhanced patient outcomes for ITP patients.