Advances in the use of hydroxyurea.

Area of Science:

• Hematology
• Pharmacology
• Genetics

Background:

• Hydroxyurea has 25 years of clinical experience in managing sickle cell disease (SCD).
• Current evidence supports hydroxyurea's safety, tolerability, and efficacy in most SCD patients.
• It is approved for adult severe SCD but remains off-label for pediatric use.

Purpose of the Study:

• To review the established clinical efficacy and safety of hydroxyurea for sickle cell disease.
• To discuss indications for hydroxyurea treatment beyond recurrent vaso-occlusive events.
• To highlight the underutilization of hydroxyurea in SCD and identify barriers to its prescription.

Main Methods:

• Review of accumulated clinical experience and evidence over 25 years.
• Analysis of hematological parameters and clinical outcomes in patients with SCD treated with hydroxyurea.
• Examination of current treatment guidelines and prescribing patterns.

Main Results:

• Hydroxyurea demonstrates efficacy in reducing acute vaso-occlusive events, including pain episodes and acute chest syndrome.
• Treatment leads to beneficial hematological changes: increased fetal hemoglobin (HbF), hemoglobin, and MCV; decreased WBC, ANC, reticulocytes, LDH, and bilirubin.
• Despite proven benefits, hydroxyurea is underprescribed in the SCD population.

Conclusions:

• Hydroxyurea is a well-tolerated, safe, and effective therapy for sickle cell disease.
• Indications for hydroxyurea include recurrent vaso-occlusive events, disease severity markers, and organ dysfunction.
• Addressing barriers to hydroxyurea prescription is crucial to enhance its effectiveness in managing SCD.