Collaborating Studies

Registry Expansion Analyses to Learn (REAL Answers)

With funding from NHLBI, Jeffery Glassberg, MD (Icahn School of Medicine at Mount Sinai) is leading REAL Answers, a prospective target-trial-emulation cohort study of individuals with SCD. It is a longitudinal observational study that follows patients as they cycle on and off various regiments. There are 2 aims to the study.

Aim 1

Compare the effect of new SCD medications - crizanlizumab, voxelotor, and L-glutamine – on clinical outcomes in individuals with SCD.

Aim 2

Identify genetic and genomic predictors of response to crizanlizumab, voxelotor, and L-glutamine.

REAL Answers leverages SCDIC infrastructure to achieve specific research goals of comparing the efficacy of new medicines and using genomics to personalize treatment. The study operates at the same 8 centers and co-enrolls many patients. REAL Answers uses common data elements with SCDIC and the data are entered into the same REDCap database to facilitate cross-study analyses.

Enrolled participants are followed regularly each year of this 5-year study, with data collected on the following forms. There is also a specimen collection at enrollment for all patients and at follow up for a select small number.

Medical record abstraction (e.g. co-morbidities, complications) 1 time a year
Patient surveys 3 times a year
Laboratory values 3 times a year
Prescription information regularly throughout the year
Health encounters (e.g. acute care visits, transfusions) regularly throughout the year