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Sickle Cell Disease and Hematopoietic Stem Cell Transplantation pp 87–110Cite as

Risk-Based Therapies for Sickle Cell Disease

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Abstract

Sickle cell severity varies among genotypes, with HbSS and HbSβ0thalassemia (sickle cell anemia, SCA) classically having more serious complications compared to patients with HbSC or HbSβ+thalassemia. However, clinical complications vary dramatically, even among patients with the same genotype. One of the most fascinating and frustrating aspects of sickle cell care is how people with the same single amino acid substitution can have different clinical complications of differing severity and at different time points. The search for a predictor of disease severity has been ongoing for more than 30 years, and the only currently available validated predictor of a severe outcome of sickle cell disease is transcranial Doppler (TCD). TCD, however, only identifies children with SCA who are at the highest risk of stroke, so predictors for recurrent painful crises or episodes of acute chest syndrome are not available. With the advent of new treatments for people with sickle cell disease like hematopoietic stem cell transplant, risk stratifiers are needed to help clinicians and families make treatment decisions. This chapter reviews work to date on disease severity predictors and highlights how therapeutic improvements for infants with sickle cell disease have made identifying high-risk children more challenging.

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Abbreviations

ACS:

Acute chest syndrome

ARC:

Absolute reticulocyte count

CAR:

Central African Republic

CSSCD:

Cooperative Study of Sickle Cell Disease

G6PD:

Glucose-6-phosphate dehydrogenase

GVHD:

Graft-versus-host disease

HbF:

Fetal hemoglobin

HbS:

Sickle hemoglobin

HSCT:

Hematopoietic stem cell transplant

SCA:

Sickle cell anemia (HbSS or HbSβ0thalassemia)

SCD:

Sickle cell disease

SCI:

Silent cerebral infarct

SITT:

Silent infarct transfusion trial

SNP:

Single-nucleotide polymorphism

STOP:

Stroke prevention trial in sickle cell anemia

TCD:

Transcranial Doppler

TNF-A:

Tumor necrosis factor-alpha

VOC:

Vaso-occlusive crisis

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Authors and Affiliations

  1. Indiana Hemophilia and Thrombosis Center, 8326 Naab Road, Indianapolis, IN, 46260, USA

    Emily Riehm Meier

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  1. Indiana Hemophilia and Thrombosis Center, Indianapolis, Indiana, USA

    Emily Riehm Meier

  2. Division of Blood and Marrow Transplantation, Children’s National Medical Center, Washington, DC, USA

    Allistair Abraham

  3. Department of Pathology and Laboratory Medicine, Emory University School of Medicine, Atlanta, Georgia, USA

    Ross M. Fasano

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Meier, E.R. (2018). Risk-Based Therapies for Sickle Cell Disease. In: Meier, E., Abraham, A., Fasano, R. (eds) Sickle Cell Disease and Hematopoietic Stem Cell Transplantation . Springer, Cham. https://doi.org/10.1007/978-3-319-62328-3_4

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