Objectives

To build a registry that includes longitudinal data of individuals with sickle cell disease in order to better understand the natural history of the disease.

Background

The abnormally shaped red blood cells (RBCs) in individuals with sickle cell disease (SCD) results in increased hemolysis and vaso-occlusion, which produce the clinical symptoms of SCD, such as episodic pain, splenic dysfunction and associated vulnerability to bacterial infections, strokes, and renal dysfunction. Treatment for SCD has been aimed at avoiding crises, relieving symptoms, and preventing complications. Bone marrow or stem cell transplantation is currently the only approved cure for SCD. However, because of the risks associated with a bone marrow transplant the procedure is recommended only for people who have significant symptoms and complications of SDC. Research on genetic therapies in SCD is ongoing.

The SCD NHDR was initiated to better understand the natural history of the disease through the collection of longitudinal data. The registry also provides a cohort of well-characterized participants to serve as comparator groups for gene therapy or other clinical trials or comparative effectiveness studies.

Participants

Eligible participants were at least 14 years of age, English speaking, with a confirmed SCD diagnosis. Participants with a successful bone marrow transplant were not eligible.

The registry includes 1220 participants that were previously enrolled in the SCDIC Registry, as well as 450 new participants.

Design

The SCD NHDR is a longitudinal observational cohort study. Recruitment of both new participants and those that were enrolled in the SCDIC Registry occurred at the SCDIC participating Clinical Centers. Standardized data, such as co-morbidities, medications, and laboratory measurements, were extracted from medical records. Standard patient reported outcome measures from ASCQ-Me, PROMIS, and Neuro-QoL were collected through self-reported participant surveys.

The registry was designed to be of sufficient size to detect differences among treatment or outcome groups. There was no primary outcome as the data can be used to answer many research questions.

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