Hydroxyurea (hydroxycarbamide) for transfusion-dependent β-thalassaemia.

Area of Science:

• Hematology
• Pharmacology
• Genetics

Background:

• Hydroxyurea (hydroxycarbamide) stimulates fetal hemoglobin (HbF) production by reactivating gamma-globin genes.
• Clinical benefits of hydroxyurea are established in sickle cell anemia.
• Limited research exists on hydroxyurea's efficacy in beta (β)-thalassemia.

Purpose of the Study:

• To review hydroxyurea's effectiveness in reducing blood transfusion needs for transfusion-dependent β-thalassemia.
• To assess the safety and severe adverse effects of hydroxyurea in this patient population.

Main Methods:

• Systematic review of randomized controlled trials (RCTs).
• Searched multiple databases and trial registries up to March 2019.
• Included RCTs comparing hydroxyurea to placebo, standard treatment, or different doses.

Main Results:

• No trials met the inclusion criteria for this review.
• Consequently, no data on hydroxyurea's efficacy or safety in transfusion-dependent β-thalassemia was available from eligible studies.

Conclusions:

• There is currently a lack of high-quality evidence to support or refute hydroxyurea use in transfusion-dependent β-thalassemia.
• Multicenter RCTs are required to evaluate hydroxyurea's efficacy, safety, and cost-effectiveness in this condition.
• Future trials should compare hydroxyurea against placebo or current standards of care, including blood transfusions and iron chelation.