Long-term efficacy of oral deferiprone in management of iron overload in beta thalassemia major

Abstract

Objective: Variable response to deferiprone has been observed in the management of iron overload in patients with thalassemia major. Our objective was to assess the long-term efficacy of deferiprone in patients with thalassemia major.

Methods: We analyzed the serum ferritin levels in fifty-eight patients who were on deferiprone therapy for at least three years. We divided patients into three groups based on the initial serum ferritin level: group 1, <2000 ng/ml; group 2, 2000–4000 ng/ml; group 3, <4000 ng/ml.

Results: Repeated measurement analysis showed a fall in serum ferritin level in group 3 after 12 months but again increased after 24 months, whereas other groups showed an increase in serum ferritin after 12 months which was sustained for 3 years. The patients were divided into three groups depending on the comparison of serum ferritin at the beginning and at the end; response group I: patients remained in the same ferritin group (n=30), response group II: patients progressed to more severe group (n=17), response group III: serum ferritin decreased and patient shifted to the lower group of ferritin level (n=11). Comparing various factors which could affect the response to deferiprone among three groups, no factor other than serum ferritin at the beginning of the treatment showed significant effect. However, some patients with a serum ferritin value in the lower range also showed improvement.

Conclusion: The major factor determining the response to deferiprone was initial serum ferritin. But some patients responded well even with relatively low initial serum ferritin.

Keywords:

• THALASSEMIA MAJOR
• DEFERIPRONE
• SERUM FERRITIN
• IRON CHELATION
• PHARMACOGENETICS