Optimizing hydroxyurea therapy for sickle cell anemia.

Abstract

Hydroxyurea has proven efficacy in numerous clinical trials as a disease-modifying treatment for patients with sickle cell anemia (SCA) but is currently under-used in clinical practice. To improve the effectiveness of hydroxyurea therapy, efforts should be directed toward broadening the clinical treatment indications, optimizing the daily dosage, and emphasizing the benefits of early and extended treatment. Here, various issues related to hydroxyurea treatment are discussed, focusing on both published evidence and clinical experience. Specific guidance is provided regarding important but potentially unfamiliar aspects of hydroxyurea treatment for SCA, such as escalating to maximum tolerated dose, treating in the setting of cerebrovascular disease, switching from chronic transfusions to hydroxyurea, and using serial phlebotomy to alleviate iron overload. Future research directions to optimize hydroxyurea therapy are also discussed, including personalized dosing based on pharmacokinetic modeling, prediction of fetal hemoglobin responses based on pharmacogenomics, and the risks and benefits of hydroxyurea for non-SCA genotypes and during pregnancy/lactation. Another critical initiative is the introduction of hydroxyurea safely and effectively into global regions that have a high disease burden of SCA but limited resources, such as sub-Saharan Africa, the Caribbean, and India. Final considerations emphasize the long-term goal of optimizing hydroxyurea therapy, which is to help treatment become accepted as standard of care for all patients with SCA.

• Authors: Russell E Ware
• Journal: Hematology. American Society of Hematology. Education Program (Hematology Am Soc Hematol Educ Program) Vol. 2015 Pg. 436-43 ( 2015) ISSN: 1520-4383 [Electronic] United States
• PMID: 26637755 (Publication Type: Journal Article)
• Copyright: © 2015 by The American Society of Hematology. All rights reserved.

Chemical References

• Antisickling Agents
• Fetal Hemoglobin
• Hydroxyurea

Topics

• Anemia, Sickle Cell (drug therapy)
• Antisickling Agents (therapeutic use)
• Blood Transfusion
• Cerebrovascular Disorders (therapy)
• Drug Administration Schedule
• Female
• Fetal Hemoglobin
• Genotype
• Global Health
• Health Services Accessibility
• Humans
• Hydroxyurea (therapeutic use)
• Iron Overload (therapy)
• Male
• National Heart, Lung, and Blood Institute (U.S.)
• Phlebotomy
• Practice Guidelines as Topic
• Pregnancy
• United States