Cure Sickle Cell Initiative recommendations on common data elements for sickle cell disease gene therapy trials

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ABSTRACT: The Cure Sickle Cell Initiative (CureSCi), created by the National Heart, Lung, and Blood Institute, has developed data standards to increase interoperability and meta-analysis of studies specifically investigating gene therapy for sickle cell disease (SCD). The aims of the CureSCi Common Data Elements (CDE) initiative are to facilitate the development of safe, effective, and accessible gene therapies. Five working groups (WGs) comprising people living with SCD, stakeholders, clinicians, and researchers reviewed current definitions used for SCD clinical studies, including those from the American Society of Hematology, US Food and Drug Administration, PhenX catalog of consensus measures for phenotypes and exposures, and others. The WGs met over 1.5 years to develop a comprehensive list of defined data points and variables recommended for any gene therapy clinical trial that incorporated feedback from a 60-day public review period. The SCD CDEs are publicly available on the CureSCi (curesickle.org/datatools-overview) and the National Institutes of Health (NIH) Repository (cde.nlm.nih.gov/home) websites. In addition to data elements, there are 42 instrument recommendations, including those from the Patient-Reported Outcomes Measurement Information System and NIH Toolbox, and 49 template case report forms for various stages of a study (eg, demographic, disease history, treatment, and outcome). CureSCi data element recommendations build upon current consensus data elements and facilitate the start-up of multicenter clinical research efforts. This standardized set of clinical research recommendations will increase the ability to compare outcomes across studies, increase data quality, and help educate new clinical investigators in the field of SCD gene therapy research.

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