Practical Guidance for the Use of Voxelotor in the Management of Sickle Cell Disease

Sickle cell disease (SCD) is one of the most common inherited blood disorders. Deoxygenated hemoglobin S (HbS) polymerizes and causes anemia and various end organ effects. Voxelotor acts by increasing HbS oxygen affinity, decreasing anemia and hemolysis. Voxelotor is approved for use in individuals...

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Published in:Journal of blood medicine 2022-01, Vol.13, p.739-745
Main Authors: Barriteau, Christina M, Badawy, Sherif M
Format: Article
Language:English
Subjects:
adolescents
adult
Age
Anemia
children
Clinical outcomes
Clinical trials
Diarrhea
disease-modifying therapy
Drug dosages
Enzymes
Evidence-based medicine
FDA approval
Glycosylated hemoglobin
Hemoglobin
Hemoglobin S
hemoglobinopathy
Leg ulcers
Medical research
Medicine, Experimental
Mental depression
oxbryta
Pathophysiology
Patients
pediatric
Polymerization
Quality of life
Review
sickle cell
Sickle cell anemia
Sickle cell disease
Teenagers
voxelotor
young adults
Youth
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Badawy, Sherif M
description Sickle cell disease (SCD) is one of the most common inherited blood disorders. Deoxygenated hemoglobin S (HbS) polymerizes and causes anemia and various end organ effects. Voxelotor acts by increasing HbS oxygen affinity, decreasing anemia and hemolysis. Voxelotor is approved for use in individuals with SCD age 4 years and older. Phase 3 trials demonstrated an increase in hemoglobin levels and a decrease in markers of hemolysis; however, data or benefits related to clinical and quality of life outcomes are relatively limited and varied across different studies. This review summarizes the published clinical trials and research studies focused on the use of voxelotor in SCD to provide an evidence-based practical guide for hematology providers on its utilization in clinical settings, including physicians and independent licensed practitioners.
doi_str_mv 10.2147/JBM.S362222
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subjects adolescents
adult
Age
Anemia
children
Clinical outcomes
Clinical trials
Diarrhea
disease-modifying therapy
Drug dosages
Enzymes
Evidence-based medicine
FDA approval
Glycosylated hemoglobin
Hemoglobin
Hemoglobin S
hemoglobinopathy
Leg ulcers
Medical research
Medicine, Experimental
Mental depression
oxbryta
Pathophysiology
Patients
pediatric
Polymerization
Quality of life
Review
sickle cell
Sickle cell anemia
Sickle cell disease
Teenagers
voxelotor
young adults
Youth
title Practical Guidance for the Use of Voxelotor in the Management of Sickle Cell Disease
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