Goal 2: Reduce Human Disease

Identification and validation of surrogate endpoints for long-term morbidity in Sickle Cell Disease

Research in sickle cell disease (SCD) has mostly focused on preventing or treating acute medical events, such as vaso-occlusive pain, acute chest syndrome, and, in pediatric patients, acute strokes. Chronic SCD complications such as chronic kidney disease or pulmonary hypertension, develop over decades, thus are poor choices for clinical trial endpoints. There is a great need to develop surrogate endpoints that predict negative clinical outcomes, so that clinical trials can be designed to address important questions in SCD in a feasible time frame. These surrogate endpoints could include biomarkers derived from proteomics and metabolomics studies, as well as imaging-based endpoints such as cerebral blood flow or cardiac function.

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Is this idea a Compelling Question (CQ) or Critical Challenge (CC)?: Compelling Question (CQ)

Details on the impact of addressing this CQ or CC:

Longitudinal cohorts of SCD patients, spanning childhood and adulthood, with biobanking DNA, plasma, and serum, and standardized clinical and imaging assessments will allow identification predictors of negative clinical outcomes. An NHLBI-funded national SCD clinical registry with biobanking will be necessary to validate any surrogate endpoints.

Name of idea submitter and other team members who worked on this idea: Monica Hulbert


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16 up votes
3 down votes
Idea No. 934