Efficacy And Tolerability Of Oral Iron Chelator, Deferasirox

Area of Science:

• Hematology
• Pharmacology

Background:

• Thalassemia Major causes severe anemia and iron overload due to ineffective red blood cell destruction.
• Iron overload, characterized by nontransferrin-bound iron, leads to organ damage in liver, heart, and endocrine systems.
• Iron chelation therapy is crucial for managing hemosiderosis in Thalassemia Major patients.

Purpose of the Study:

• To evaluate the efficacy of oral Deferasirox in managing iron overload in Thalassemia Major patients.
• To assess the impact of oral Deferasirox on serum ferritin levels and identify potential side effects.

Main Methods:

• A non-randomized study enrolled 37 Thalassemia Major patients aged 2-30 years.
• Serum ferritin, ALT, and creatinine levels were monitored at baseline and at 3, 6, and 9 months.
• Patients were assessed for oral tolerability and other adverse effects of Deferasirox.

Main Results:

• Serum ferritin levels increased over the 9-month study period, with an average of 3440 at baseline to 4394 at 9 months.
• 46% of patients (17 out of 37) required alternative chelation, specifically IV Deferioxamine, due to rising serum ferritin.
• Oral Deferasirox was found to be ineffective in significantly reducing or controlling high iron overload levels.

Conclusions:

• Oral Deferasirox is not an effective iron chelator for patients with serum ferritin levels above 1500 ng/ml.
• Regular monitoring of serum ferritin (every 3 months) is recommended for patients on oral Deferasirox.
• Intravenous Deferioxamine is still the preferred treatment for effective control of iron overload and its complications in Thalassemia Major.