Efficacy and safety of Deferasirox therapy in a group of beta thalassemia patients during three years follow up in Babylon thalassemic center in Babylon Province in Iraq

الملخص EN

Objective: To assess the efficacy and safety of 3 years deferasirox therapy in group of patients with β thalassemia.

Methods: One hundred and thirty two patients with beta-thalassemia major, (2-16 years old), had been started treatment with deferasirox in Babylon Center of Hereditary Blood disorders in Babylon governorate- Iraq.

They were followed prospectively from March 2012 till March 2015.

The dose of deferasirox was adjusted up to 40 mg/kg according to the response Serum ferritin, liver enzymes, urine for albumin and renal function tests were regularly checked.

Results: Out of 132 patients, 105 patients (71%) had decreased level of serum ferritin (less than 1000 ng/dl), (p-value<0.05).

The mean serum ferritin at basal, 12, 24 and 36 months were 3120, 2077, 1402 and 1075 respectively, (p-value<0.05).

The most effective deferasirox dose which was used to reduce ferritin level was between 30-30.79 mg/kg from 6-12 month of treatment, while 25.68-30.42 mg/kg after 12 month of therapy.

All patients had normal liver enzymes and renal function tests except three patients who experienced mild elevation of liver enzymes and one patient had mild disturbance in creatinine level.

Conclusions: Deferasirox significantly reduces serum ferritin in the majority of patients with betathalassemia major.

A dose of ≥30 mg/kg is required to achieve a negative iron balance.

It appears to be safe, well tolerated in pediatric patients and efficacious even in patients with very high iron load.

Deferiprone (Ferriprox) is bidenate has been introduced since 1987, oral chelation therapy used as second line therapy when desferrioxamine is contraindicated.

It is effective in removing cardiac iron but associated with other side effect like hepatic fibrosis, erosive arthritis and granulocytosis, so this needs close monitoring.

Deferasirox (Exjade) is a tridenate once-daily oral chelation therapy approved by FDA in 2005 used effectively and safely in transfusion-dependent thalassemia in pediatric and adult with half-life 8-16 hours, it is excreted in stool.

Deferasirox once-daily dosing permits circulating drug at all tissue scavenge non-transferrin bound (labile plasma iron), the chemical species responsible for tissue damage in iron-overloaded patient