Maintenance of Normal Range Body Iron Store Levels for up to 4.5 Years in Thalassemia Major Patients Using Deferiprone Monotherapy

Abstract

New gold standard protocols are tested for the complete removal of iron overload in thalassemia using the International Committee on Chelation (ICOC) Maintaining Normal Body Iron combination protocol therapy of deferiprone (L1)/deferoxamine (DFO) and maintenance of normal range body iron store levels (NRBISL) using L1 monotherapy. Deferiprone (80–100 mg/kg/day) was administered for a mean of 21.3 months (range 7–91, total 171) in eight thalassemia major patients (four males and four females, 29–47 years) who have achieved serum ferritin (mean 108.5 μg/L, range 25–408), cardiac T2* (mean 31.5 ms, range 24–41) and liver T2* (30.1 ms, range 9.1–41) magnetic resonance imaging (MRI) relaxation times. At the end of the study normal range MRI T2* relaxation time was maintained in all eight patients with a cardiac mean of T2* 30.3 ms (range 23–37), liver T2* 28.8 ms (range 9.8–43) and serum ferritin with a mean of 173.7 μg/L (range 86–406). In one patient, this NRBISL was maintained for more than 4.5 years. No toxic side effects have been observed during the L1 monotherapy period.

Keywords:

• Thalassemia
• Iron overload
• Chelation therapy
• Deferiprone (L1)
• Deferoxamine (DFO)
• Deferasirox (DFRA)
• Normal iron stores

ACKNOWLEDGMENTS

We would like to thank Dr Charalambos Economides and Eleni Eracleous at the Agios Therissos Medical Diagnostic Centre, Nicosia, Cyprus, for the MRI measurements.

Declaration of Interest: The authors report no conflicts of interest. The authors alone are responsible for the content and writing of this article.