Sickle cell disease (SCD) is an inherited monogenic disease characterized by misshapen red blood cells that causes vaso-occlusive disease, vasculopathy, and systemic inflammation. Approximately 300,000 infants are born per year with SCD globally. Acute, chronic, and acute-on-chronic complications contribute to end-organ damage and adversely affect quantity and quality of life. Hematopoietic stem cell transplantation is the only cure available today, but is not feasible for the vast majority of people suffering from SCD. Fortunately, new therapies are in late clinical trials and more are in the pipeline, offering hope for this unfortunate disease, which has increasing global burden.
Keywords: Hemoglobinopathy; Hydroxyurea; Iron overload; Review; Sickle cell anemia; Sickle cell disease.
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