Novel Agents in Sickle Cell Disease

Sickle cell disease is the most common inherited disorder of hemoglobin that arises from a single nucleotide variant on the beta globin. Under stressful physiologic conditions, red blood cells with sickle hemoglobin assume a rigid, elongated shape and form aggregate with one another; these dense polymers of sickle erythrocytes bind to activated platelets, white blood cells and the vascular endothelium and occlude the microvasculature. In this lecture, I discuss the mechanisms of action of the novel sickle cell disease-modifying agents (L-glutamine, crizanlizumab and voxelator) and how their therapeutic effects complement those of hydroxyurea in people with sickle cell disease

After viewing this lecture, participants should be able to:
1. Describe the pathophysiology of sickle cell disease
2. Distinguish the mechanism of action of sickle cell disease-modifying drugs
3. Interpret changes in laboratory parameters that correlate with therapeutic benefit (or adverse effects) of sickle cell disease-modifying drugs

Oyebimpe O. Adesina, MD, MS
Assistant Professor, Department of Medicine
Division of Hematology
University of Washington

02/19/20