Sickle Cell Disease: New Frontiers

Recent progress in genetic epidemiology, molecular diagnostics, and targeted therapeutics has led to a surge of new knowledge and developments in sickle cell disease (SCD) and sickle cell trait. This session will focus on recent advances in the understanding of clinical implications of sickle cell trait, as well as promising diagnostic approaches in clinical care and research of SCD. Lastly, this session will review exciting progress in the development of therapeutics for SCD.

Dr. Rakhi Naik will review the current landscape of research in clinical complications of sickle cell trait, including exertion-related injury, chronic kidney disease, and venous thromboembolism. She will discuss the role of epidemiology and precision medicine in evaluating the risk of complications in individuals with sickle cell trait. Dr. Naik will also highlight opportunities for further discovery and guideline development in this emerging field.

Dr. Enrico Novelli will summarize existing evidence and promising data on biomarkers for clinical care and research in SCD. He will discuss pitfalls in biomarker development and the recent progress in molecular and imaging diagnostics in the new era of personalized medicine. Dr. Novelli will also review how these modalities can be applied to a precision medicine approach for both the care of patients and in the design of clinical trials in SCD.

Dr. Deepa Manwani will discuss the considerable progress in the development of treatments for SCD. She will outline the complex pathophysiology of SCD, including blood cell adhesion, inflammation and hemostatic system activation, as potential targets for therapeutics. Dr. Manwani will also highlight the challenges in selection of study end points in the development of novel therapeutic strategies in SCD.