Daily alternating deferasirox and deferiprone therapy for ‘hard-to-chelate' β-thalassemia major patients

Manuela Balocco (1) , Paola Carrara (1) , Valeria Pinto (1) , Gian Luca Forni (2)

1 Centro della Microcitemia e delle Anemie Congenite
2 Centro della Microcitemia e delle Anemie Congenite

Summary Mono- and combination therapy with chelating agents such as deferoxamine (DFO), deferasirox (DFX), and deferiprone (DFP) has made a significant difference to the quality of life and the life expectancy of patients who need to undergo regular blood transfusions due to thalassemia major. Serious adverse reactions that can limit uptake of these therapies are not unknown, however. We present two case studies of patients with thalassemia major who have experienced improvements in their ferritin levels and liver iron concentration (LIC) with DFP and DFX on alternating days over 1 year, despite having previously experienced serious adverse events with all three monotherapies. The success of this novel use for chelating agents suggests a need for larger studies to determine if this approach might be an option for otherwise untreatable thalassemia patients.

Mots clés: Medicine

Manuela Balocco, Paola Carrara, Valeria Pinto, Gian Luca Forni. Daily alternating deferasirox and deferiprone therapy for ‘hard-to-chelate' β-thalassemia major patients. American Journal of Hematology, 2010, ⟨10.1002/ajh.21711⟩. ⟨hal-00552318⟩