Sickle cell disease
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Lancet Publishing Group
Abstract
Sickle cell disease is a common and life-threatening haematological disorder that affects millions of people worldwide. Abnormal sickle-shaped erythrocytes disrupt blood flow in small vessels, and this vaso-occlusion leads to distal tissue ischaemia and inflammation, with symptoms defining the acute painful sickle-cell crisis. Repeated sickling and ongoing haemolytic anaemia, even when subclinical, lead to parenchymal injury and chronic organ damage, causing substantial morbidity and early mortality. Currently available treatments are limited to transfusions and hydroxycarbamide, although stem cell transplantation might be a potentially curative therapy. Several new therapeutic options are in development, including gene therapy and gene editing. Recent advances include systematic universal screening for stroke risk, improved management of iron overload using oral chelators and non-invasive MRI measurements, and point-of-care diagnostic devices. Controversies include the role of haemolysis in sickle cell disease pathophysiology, optimal management of pregnancy, and strategies to prevent cerebrovascular disease. © 2017 Elsevier Ltd
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Keywords
Anemia, sickle cell, Antisickling agents, Blood transfusion, Cerebrovascular disorders, Chronic disease, Early diagnosis, Female, Genetic therapy, Global burden of disease, Hemolysis, Hemolytic plaque technique, Humans, Hydroxyurea, Iron overload, Point-of-care systems, Pregnancy, Pregnancy complications, hematologic, Stem cell transplantation, Stroke, Antisickling agent, Acute disease, Cerebrovascular disease, Gene therapy, Global disease burden, Human, Pathophysiology, Phase 1 clinical trial (topic), Phase 2 clinical trial (topic), Phase 3 clinical trial (topic), Point of care testing, Priority journal, Randomized controlled trial (topic), Review, Sickle cell anemia, Transplantation, Cerebrovascular accident, Complication, Hemolytic plaque assay, Point of care system, Pregnancy complication, Procedures