A randomized controlled 1-year study of daily deferiprone plus twice weekly desferrioxamine compared with daily deferiprone monotherapy in patients with thalassemia major

Deferiprone (GPO‐L‐ONE®) monotherapy reduces iron overload in transfusion‐dependent thalassemias: 1‐year results from a multicenter prospective, single arm, open label, dose escalating phase III pediatric study (GPO‐L‐ONE; A001) from Thailand

“…These groups of patients were inadequately treated or chelation naive with very high levels of baseline serum ferritin and high iron intake from blood transfusion [11,[36][37][38][39][40][41]. Furthermore our findings that SF values were more likely to decrease in those patients who began with the highest SF values and lower iron intake from lower transfusion rate (<0.25 mg/kg/day in our study) are consistent with previous individual studies [42][43][44] and review of trends from several studies [38]. Also in the view of without a strong evidence to suggest that deferiprone has superior efficacy than desferoxamine, together with its associated adverse effects, some can be fatal, deferiprone remains the second line therapy for thalassemia major patients whose desferoxamine is inadequate or contraindicated as reviewed [45,46] and documented in all clinical practice guidelines around the world [47][48][49] including the latest registration filed by the US-FDA [50].…”

Prion Protein Promotes Kidney Iron Uptake via Its Ferrireductase Activity

“…However, the universality of this phenomenon regardless of the microenvironment suggests an inherent susceptibility of PrP C to metal-induced oxidative stress. Such a response of PrP C in PT cells has physiological implications as NTBI and hemin are nephrotoxic and are likely to leak into the glomerular filtrate under a variety of pathological conditions (47)(48)(49). Aggregation of PrP C in response to these agents is, therefore, likely to serve a protective role by reducing further iron uptake by the kidney.…”

Toward optimizing the use of deferasirox: potential benefits of combined use with deferoxamine

“…Subsequent long-term clinical studies have substantiated these expectations. 9,[27][28][29][30][31][32][33] The approval of deferasirox (DFX) as an orally effective iron-chelating drug in 2005 promised to improve the management of iron overload as this drug could be taken once daily and apparently had few side effects. 34,35 Moreover, it proved to be non-inferior to DFO in a large, multicenter, randomized controlled trial involving roughly 600 patients; the doses of DFO and DFX ranged up to 60 and 30 mg/kg/day, respectively.…”