Sickle cell disease (SCD) is a beta hemoglobinopathy that occurs in approximately
100,000 Americans and affects 1 in 365 African-American and 1 in 16,300 Hispanic-American
births [
[1]
]. It is characterized by the presence of hemoglobin S (HbS) which contains a single
base substitution (A-T) in the β-globin gene that leads to a single amino acid change.
HbS polymerizes under deoxygenated conditions causing the red blood cell (RBC) to
assume a sickle shape. These rigid sickle RBCs cause a number of adverse events including
vaso-occlusion, intravascular hemolysis, endothelial injury, end-organ damage, and
anemia [
2
,
3
]. Despite the simplicity of its genetic etiology, the clinical manifestations of SCD
are highly heterogeneous and complex. Studies continue to be performed to better understand
the pathophysiology of SCD to not only help target drug development but also to develop
diagnostic assays that would enable early detection of the disease, predict the occurrence
of adverse events, and to monitor the effectiveness of therapies. To date, there are
a number of treatment options available for managing patients with SCD but not all
are readily accessible and some may be associated with significant morbidity and mortality
which decrease their appeal. This issue of Transfusion and Apheresis Science focuses
on the advancements in treatment options for SCD and laboratory testing methodologies
for monitoring the efficacy of the different treatment strategies.To read this article in full you will need to make a payment
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References
Sickle Cell Disease (SCD). Center for Disease Control and Prevention.
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- Impact of sickle cell disease on patients' daily lives, symptoms reported, and disease management strategies: results from the international Sickle Cell World Assessment Survey (SWAY).Am J Hematol. 2021; 96: 404-417
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- Mortality in sickle cell disease. Life expectancy and risk factors for early death.N Engl J Med. 1994; 330: 1639-1644
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Jensen K. Bluebird proposes installment plan for LentiGlobin gene therapy 2019.
- Curative therapy for hemoglobinopathies: an International Society for Cell & Gene Therapy Stem Cell Engineering Committee review comparing outcomes, accessibility and cost of ex vivo stem cell gene therapy versus allogeneic hematopoietic stem cell transplantation.Cytotherapy. 2022; 24: 249-261
Article Info
Publication History
Published online: August 28, 2022
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