Progress in the management of iron overload in erytrocyte disorders

M. D. Cappellini, E. Cassinerio, L. Zanaboni, G. Fabio, P. Pattoneri

Research output: Contribution to journalArticlepeer-review

Abstract

Although blood transfusions are essential for patients with hemoglobinopathies (ie Thalassemia, Sickle Cell Disease) or with rare anemias (ie Fanconi, Blackfan Diamond, PK deficiency), chronic transfusions inevitably lead to iron overload as humans cannot actively remove excess iron. The cumulative effects of iron overload lead to significant morbidity and mortality, if untreated. Desferrioxamine (DFO) is the reference-standard iron chelator whose safety and efficacy profile has been established through many years of clinical use. DFO side effects are accettable and manageable however the prolonged subcutaneous infusion regimen of 5-7 days per week is very demanding and results in poor adherence to therapy. Deferiprone (Ferriprox, L1) is a bidentate molecule, orally administrable three-times/day, licensed in Europe and in other regions but in the USA and Canada, for the treatment of iron overload in patients for whom DFO therapy is contraindicated or inadequate. Preliminary evidences suggest that deferiprone may be more effective than DFO in chelating cardiac iron. The side effects include gastrointestinal symptoms, liver dysfunction, joint pain, neutropenia and agranulocytosis. A weekly assessment of white blood cell counts is recommended because of the risk of agranulocytosis. Deferasirox is a new, convenient, once-daily oral iron chelator that has demonstrated in various clinical trials good efficacy and acceptable safety profile in adult and pediatric patients affected by transfusion-dependent thalassemia major and by different chronic anemias (SCD, BDA, MDS). The long half-life of deferasirox (16-18 hours) provides sustained 24 hr iron chelation coverage. The efficacy and safety profile have been evaluated in more than 3000 patients in clinical trials. Patient satisfaction with deferasirox was superior than with DFO therapy.

Original languageEnglish
Pages (from-to)6-13
Number of pages8
JournalHaematologica Meeting Reports
Volume2
Issue number5
Publication statusPublished - 2008

ASJC Scopus subject areas

  • Hematology

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