Department of Pediatrics, Northwestern University Feinberg School of Medicine, Chicago, IL; and Division of Hematology, Oncology, and Stem Cell Transplant, Ann and Robert H. Lurie Children's Hospital of Chicago, Chicago, IL.
Hematology Am Soc Hematol Educ Program. 2021 Dec 10;2021(1):196-205. doi: 10.1182/hematology.2021000252.
Patients with sickle cell disease (SCD) have significant impairment in their quality of life across the life span as a consequence of serious disease burden with several SCD-related complications. A number of disease-modifying therapies are currently available, yet long-term clinical benefits in real-world settings remain unclear. Over the past few years, a number of important initiatives have been launched to optimize clinical trials in SCD in different ways, including: (1) established panels through a partnership between the American Society of Hematology (ASH) and the US Food and Drug Administration; (2) the ASH Research Collaborative SCD Clinical Trials Network; (3) the PhenX Toolkit (consensus measures for Phenotypes and eXposures) in SCD; and (4) the Cure Sickle Cell Initiative, led by the National Heart, Lung, and Blood Institute. Electronic patient-reported outcomes assessment is highly recommended, and patient-reported outcomes (PROs) should be evaluated in all SCD trials and reported using Standard Protocol Items Recommendations for Interventional Trials guidelines. Patient-centered outcomes research (PCOR) approaches and meaningful stakeholder engagement throughout the process have the potential to optimize the execution and success of clinical trials in SCD with considerable financial value. This article reviews several clinical trial considerations in SCD related to study design and outcomes assessment as informed by recent initiatives as well as patient-centered research approaches and stakeholder engagement. A proposed hematology stakeholder-engagement framework for clinical trials is also discussed.
患有镰状细胞病 (SCD) 的患者由于严重的疾病负担和多种 SCD 相关并发症，在整个生命周期中都存在显著的生活质量受损。目前有许多疾病修正疗法，但在真实环境中的长期临床获益仍不清楚。在过去几年中，已经启动了许多重要的倡议，以通过多种方式优化 SCD 临床试验，包括：(1) 通过美国血液学会 (ASH) 与美国食品和药物管理局之间的合作建立专门小组；(2) ASH 研究协作 SCD 临床试验网络；(3) SCD 中的 PhenX 工具包（表型和暴露的共识措施）；(4) 由美国国立心肺血液研究所领导的治愈镰状细胞病倡议。强烈建议使用电子患者报告结局评估，并且应该在所有 SCD 试验中评估患者报告结局 (PRO)，并使用干预性试验标准议定书项目建议报告。以患者为中心的结局研究 (PCOR) 方法和整个过程中的有意义的利益相关者参与有可能以相当大的经济价值优化 SCD 临床试验的执行和成功。本文综述了最近的倡议以及以患者为中心的研究方法和利益相关者参与所告知的 SCD 临床试验中的几个临床考虑因素和结局评估。还讨论了一个拟议的血液学利益相关者参与临床试验的框架。
