National Minority Quality Forum, Washington, DC.
Sickle Cell Disease Clinical Trials Network, ASH Research Collaborative, Washington, DC.
Blood Adv. 2021 Dec 14;5(23):5323-5331. doi: 10.1182/bloodadvances.2020003434.
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.
镰状细胞病 (SCD) 是美国最常见的遗传性红细胞疾病,影响 70,000 至 100,000 名美国人,并导致一系列严重的医疗并发症。尽管 SCD 的病因已确立数十年,但现有疗法的疗效和副作用各不相同,新型疗法的开发进展缓慢。现有治疗方案的局限性突出表明需要新的治疗方法,这些方法应符合社区的需求。迄今为止,很少有人系统地寻求并报告 SCD 患者对临床研究的意见和经验。2019 年,美国血液学会研究协作组织在美国各地举办了 8 次社区研讨会,共有 472 人参加,其中包括 SCD 患者及其疾病患者的护理人员。研讨会的目标包括评估对临床研究的理解、意识和看法;并确定该社区最关键的临床试验考虑因素。参与者被问及他们的 SCD 生活经历以及对治疗选择的满意度。疼痛和疲劳被报告为需要更好治疗的症状。尽管很少有参与者报告被要求参加临床试验,但他们表示有条件愿意参与。大多数人愿意分享个人健康信息,以进一步研究和改善健康结果。为了积极参与 SCD 社区并增加临床试验的参与率和保留率,研究人员应解决该人群的治疗重点,并确保他们能够获得有关临床研究和参与机会的可信信息。