OBJECTIVES: The benefits of combined chelation therapy with daily deferiprone (DFP) and subcutaneous desferrioxamine (DFO) have been widely reported in literature. We retrospectively evaluated the efficacy of different schedules of combined chelation therapy and the incidence of adverse events. METHODS: We evaluated 36 patients affected by thalassemia major treated with combined chelation therapy. Patients were subdivided into four treatment arms according to severity of iron overload and previous onset of adverse events to DFP therapy: Group 1 (13 pts) DFP 75 mg/kg per d plus DFO (25-35 mg/kg per d for 5 d); Group 2 (6 pts) DFP 50 mg/kg per d plus DFO (25-35 mg/kg for 5 d), Group 3 (10 pts) DFP 75 mg/kg per d plus DFO (25-35 mg/kg for 3 d), and Group 4 (7 pts) DFP 50 mg/kg per d plus DFO (25-35 mg/kg for 3 d). Change in serum ferritin level was evaluated in all patients. RESULTS: Overall, ferritin decreased from 2592 +/- 1701 to 899 +/- 833 ng/mL (P < 0.001). All treatments were able to reduce ferritin levels, but in patients of group 1 and group 2 the highest mean decrease in serum ferritin level and the greatest improvement in liver iron concentration (LIC) and in T2* values were observed. CONCLUSIONS: This study showed that the administration of DFO for 5 d a wk in combination with daily administration of DFP at 75 mg/Kg seemed to be the most efficacy and rapid method for reducing iron overload at liver and heart level. Furthermore, the use of different schedules of combined DFO and DFP administration was not associated with different incidence of adverse effects between the groups.
OBJECTIVES: The benefits of combined chelation therapy with daily deferiprone (DFP) and subcutaneous desferrioxamine (DFO) have been widely reported in literature. We retrospectively evaluated the efficacy of different schedules of combined chelation therapy and the incidence of adverse events. METHODS: We evaluated 36 patients affected by thalassemia major treated with combined chelation therapy. Patients were subdivided into four treatment arms according to severity of iron overload and previous onset of adverse events to DFP therapy: Group 1 (13 pts) DFP 75 mg/kg per d plus DFO (25-35 mg/kg per d for 5 d); Group 2 (6 pts) DFP 50 mg/kg per d plus DFO (25-35 mg/kg for 5 d), Group 3 (10 pts) DFP 75 mg/kg per d plus DFO (25-35 mg/kg for 3 d), and Group 4 (7 pts) DFP 50 mg/kg per d plus DFO (25-35 mg/kg for 3 d). Change in serum ferritin level was evaluated in all patients. RESULTS: Overall, ferritin decreased from 2592 +/- 1701 to 899 +/- 833 ng/mL (P < 0.001). All treatments were able to reduce ferritin levels, but in patients of group 1 and group 2 the highest mean decrease in serum ferritin level and the greatest improvement in liver iron concentration (LIC) and in T2* values were observed. CONCLUSIONS: This study showed that the administration of DFO for 5 d a wk in combination with daily administration of DFP at 75 mg/Kg seemed to be the most efficacy and rapid method for reducing iron overload at liver and heart level. Furthermore, the use of different schedules of combined DFO and DFP administration was not associated with different incidence of adverse effects between the groups.
Authors: Francesca Vinchi; Richard Sparla; Sara T Passos; Richa Sharma; S Zebulon Vance; Hala S Zreid; Hesham Juaidi; Deepa Manwani; Karina Yazdanbakhsh; Vijay Nandi; André M N Silva; Anand R Agarvas; Eitan Fibach; John D Belcher; Gregory M Vercellotti; Husam Ghoti; Martina U Muckenthaler Journal: Br J Haematol Date: 2021-03-15 Impact factor: 6.998