Abstract
Sickle cell disease (SCD) is one of the world’s most common severe monogenic disorders affecting millions of people worldwide and represents a significant public health problem. The last two decades have seen a major increase in knowledge about the cascade of events that follow the polymerization of hemoglobin, the main pathophysiological event in SCD, including impaired biorheology and increased adhesion-mediated vaso-occlusion, hemolysis-mediated endothelial dysfunction, and inflammation. As a result, several distinctive therapeutic targets have been discovered, and a few drugs with innovative mechanisms of action are already on the Market. In contrast, several others are the focus of ongoing trials. This narrative review aims to describe some of the more recent data in the SCD literature regarding pathophysiology and novel treatments.