Abstract
High fetal hemoglobin (HbF) levels inhibit the polymerization of sickle hemoglobin (HbS) and reduce the complications of sickle cell disease. Pharmacologic agents that can reverse the switch from γ- to β-chain synthesis — γ-globin chains characterize HbF, and sickle β-globin chains are present in HbS — or selectively increase the proportion of adult erythroid precursors that maintain the ability to produce HbF are therapeutically useful. Hydroxyurea promotes HbF production by perturbing the maturation of erythroid precursors. This treatment increases the total hemoglobin concentration, reduces the vaso-occlusive complications of pain and acute chest syndrome, and attenuates mortality in adults. It is a promising beginning for pharmacologic therapy of sickle cell disease. Still, its effects are inconsistent, trials in infants and children are ongoing, and its ultimate value — and peril — when started early in life are still unknown.
Highlights
Hydroxyurea Treatment for Sickle Cell DiseaseM.D. Professor of Medicine and Pediatrics, Boston University School of Medicine, 88 E
The new millennium begins an era of remarkable promise for patients with sickle cell disease
This review addresses the evolution of pharmacologic treatment for sickle cell disease focused on increasing the concentration of fetal hemoglobin (HbF) in erythrocytes
Summary
M.D. Professor of Medicine and Pediatrics, Boston University School of Medicine, 88 E. High fetal hemoglobin (HbF) levels inhibit the polymerization of sickle hemoglobin (HbS) and reduce the complications of sickle cell disease. Hydroxyurea promotes HbF production by perturbing the maturation of erythroid precursors. This treatment increases the total hemoglobin concentration, reduces the vaso-occlusive complications of pain and acute chest syndrome, and attenuates mortality in adults. It is a promising beginning for pharmacologic therapy of sickle cell disease. DOMAINS: gene expression, molecular and gene therapy, hematology, clinical medicine, medical research, clinical trials, medical care
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