Sickle cell disease (SCD) is the most common inherited red blood cell disorder in the United States, affecting 70 000 to 100 000 Americans and causing a range of serious medical complications. Although the cause of SCD was established decades ago, existing therapies have varied effectiveness and side effects, and development of novel therapies has been slow. The limitations of existing treatment options highlight the need for new therapies that are aligned with the desires of the community. To date, little has been done to systematically seek and report the opinions and experiences of people with SCD regarding clinical research. In 2019, the American Society of Hematology Research Collaborative conducted 8 community workshops across the United States engaging 472 people, including persons with SCD and caregivers of those living with the disease. The workshop goals included assessing understanding, awareness, and perceptions of clinical research; and identifying the most critical clinical trial considerations of this community. Participants were asked about their experiences living with SCD and their satisfaction with treatment options. Pain and fatigue were reported as symptoms requiring better therapies. Although few participants reported being asked to enroll in a clinical trial, they expressed conditional willingness to participate. A majority were willing to share personal health information to further research and improve health outcomes. To actively engage the SCD community and increase enrollment and retention in clinical trials, researchers should address the treatment priorities of this population and ensure they have access to trusted information about clinical research and opportunities for participation.

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http://www.ncbi.nlm.nih.gov/pmc/articles/PMC9153049PMC
http://dx.doi.org/10.1182/bloodadvances.2020003434DOI Listing

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