Oral deferiprone for iron chelation in people with thalassaemia

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

“…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]. In this regard, our study has further confirmed that efficacy of deferiprone monotherapy is rather limited and should not be used as a first-line treatment in patients with transfusion-dependent thalassemia including severe Hb E/b thalassemia.…”

Thalassemia and its Management during Pregnancy

“…Due to the lack of physiological process for removing excessive iron from the body due to multiple transfusions, transfusion-dependent patients require treatment with an iron chelator between 5 and 8 years of age. 52 Generally, chelation therapy is started at an age of 2 to 4 years after 20 to 25 RBC units have been transfused, serum ferritin levels are more than 1000 μg/dL, and LIC estimated by liver biopsy or by noninvasive hepatic T 2 MRI is more than 3 mg iron/gm dw. 53 Various chelating agents, which are currently available, are as follows ( …”

Management of the Thalassemias

“…The long-term effectiveness of a variety of binary combinations of chelating agents presently administered in off-label uses remains uncertain due to the absence of unequivocal evidence of the superiority of any specific combination over treatment with a single agent (Roberts et al 2007;Brittenham 2011).…”