EFFICACY AND SAFETY OF DEFERASIROX THERAPY ON Β-THALASSEMIA MAJOR PATIENTS IN BABYLON THALASSEMIA CENTER

Ahmed Shemran Al-Wataify *

* Department of pediatrics, Faculty of Medicine, Babylon University. Babylon Pediatric and Maternal Teaching Hospital.


EFFICACY AND SAFETY OF DEFERASIROX THERAPY ON Β-THALASSEMIA MAJOR PATIENTS IN BABYLON THALASSEMIA CENTER


ABSTRACT


Background
Chronic iron overload from frequent blood transfusion leads to significant morbidity and mortality. Iron chelation therapy is an important part of management of those patients. Deferasirox is once daily oral iron chelator that is now widely used for treatment of transfusion hemosiderosis and represents a significant advance in treatment.

Objectives
The aim of this study is to assess the efficacy and safety of deferasirox therapy in a group of patients with β-thalassemia major.

Patients and Methods
A prospective study of 160 patients with β-thalassemia major for one year duration in Babylon thalassemia center, whose age ranged from 2—15 years with mean age of 6.7 ± 2.3years, have their serum ferritin level ranged from 1000—4000 ng/ml. The initial usage of Deferasirox dose depended on serum ferritin level ranged from 20 - 40 mg/kg/day, the patients classified into 2 groups based on serum ferritin either higher or lower than 3000ng/ml ( 137 patients with level of < 3000ng/ml, 18 patients>3000ng/ml and only 5 patients stopped treatment due to poor Compliance ), the initial dose for first group is ≥20-<30mg/kg/day and the dose adjustment was performed in steps of 5-10 mg/kg/day every 3 months based on serum ferritin and safety marker of the drug, while the initial dose for the second group is 40mg/kg/day.

Results
The median base serum ferritin level was 2836.456±1244 ng/ml decreased to 2000.56±531.1 ng/ml after 12 months of deferasirox therapy (p value<.001). The optimum dose in reducing serum ferritin in the first group is ≥30-<40mg/kg/day in 65.69%. While, for the second group is 40mg/kg/day in 62.5% and 23 patients discontinued treatment. The commonest adverse effect is gastro-intestinal upset in 38.7%.

Conclusion
Deferasirox is effective and tolerable chelation in treatment of β-thalassemia major with iron overload.

Keywords: Deferasirox, β thalassemia major, Babylon thalassemia center.


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Kewan Omer,
Jan 22, 2015, 10:51 AM