Long-term hydroxyurea therapy in beta-thalassaemia patients

Area of Science:

• Hematology
• Pharmacology
• Genetics

Background:

• Beta-thalassemia (beta-thal) is a group of inherited blood disorders characterized by reduced or absent synthesis of beta-globin chains.
• Current management for beta-thal major often involves regular blood transfusions and iron chelation therapy.
• Hydroxyurea (HU) has demonstrated efficacy in other hemoglobinopathies, such as sickle cell disease, by increasing fetal hemoglobin (HbF) production.

Purpose of the Study:

• To investigate the efficacy and hematological effects of hydroxyurea (HU) in patients with beta-thalassemia major and intermedia.
• To assess changes in hemoglobin levels, fetal hemoglobin, and other hematological parameters during HU therapy.
• To evaluate the safety and tolerability of HU in this patient population.

Main Methods:

• A total of 11 patients (4 beta-thal major, 7 beta-thal intermedia) received oral hydroxyurea at doses of 10-20 mg/kg/day.
• Complete blood counts, fetal hemoglobin (HbF), liver enzymes, and serum creatinine were monitored before and during treatment.
• Response to therapy was assessed after 6 months of treatment.

Main Results:

• One beta-thal major patient experienced a significant increase in hemoglobin (Hb) of 4.1 g/dL, enabling cessation of transfusions.
• In beta-thal intermedia patients, 3 out of 7 (42.9%) showed increased Hb levels (1.3-2.0 g/dL) during HU therapy.
• Mean Hb, mean corpuscular hemoglobin (MCH), and HbF levels increased significantly (P ≤ 0.05), while reticulocyte counts decreased (P = 0.03). No significant toxicity was observed.

Conclusions:

• Hydroxyurea (HU) may offer a therapeutic benefit by improving hemoglobin levels in beta-thalassemia patients.
• The observed increase in Hb and HbF suggests a potential mechanism for HU's efficacy.
• Larger clinical trials are warranted to definitively establish the role of HU in beta-thalassemia management.