Hydroxyurea in the sickle cell disease modern era

Area of Science:

• Hematology
• Pharmacology
• Genetics

Background:

• Sickle cell disease (SCD) is an inherited blood disorder causing red blood cell polymerization, vaso-occlusion, and hemolytic anemia, leading to severe acute and chronic complications.
• Millions are affected globally, particularly in sub-Saharan Africa, highlighting the need for effective and accessible treatments.
• Hydroxyurea was the first FDA-approved medication for SCD, primarily used to reduce painful crises and the need for blood transfusions in patients with severe disease.

Purpose of the Study:

• To review current clinical data, safety information, and evidence supporting hydroxyurea's role in managing sickle cell disease.
• To examine the evolving therapeutic landscape of sickle cell disease in light of new drug approvals.
• To provide a comprehensive overview of hydroxyurea's continued relevance and expanded research areas.

Main Methods:

• A narrative review approach was employed.
• A comprehensive literature search was conducted across PubMed and Cochrane Library databases.
• The search encompassed publications from 1963 to 2024, focusing on hydroxyurea in sickle cell disease.

Main Results:

• Hydroxyurea is recognized as the gold standard treatment for sickle cell disease, even with the advent of new medications.
• It functions as a disease modifier with additional clinical benefits, characterized by its affordability and established safety profile.
• Ongoing research is exploring hydroxyurea's underutilization and pharmacogenomic aspects.

Conclusions:

• Hydroxyurea remains a vital therapeutic agent in sickle cell disease management due to its efficacy, safety, and cost-effectiveness.
• Its long-standing record supports its continued use as a key treatment option.
• Expanded research into its application and personalized medicine approaches is warranted.