Clinical trial of deferiprone iron chelation therapy in β‐thalassaemia/haemoglobin E patients in Thailand

Abstract

Summary. Nine patients with either b-thalassaemia/haemoglobin E (7) or homozygous b-thalassaemia (2) not requiring regular transfusions were treated with the oral iron chelator, deferiprone 25-50 mg/kg/d for between 17 and 86 weeks (mean 49 weeks). There were significant decreases in serum ferritin (initial mean ± standard deviation 2168 ± 1142, final 418 ± 247 lg/l; t-test for paired samples, P ¼ 0AE005), hepatic iron (initial 20AE3 ± 6AE26, final 11AE7 ± 4AE83 mg/g/dry weight; P ¼ < 0AE02), red cell membrane iron (initial 76AE2 ± 3AE64, final 7AE2 ± 0AE56 mmol/mg protein; P ¼ < 0AE0005) and serum non-transferrin bound iron (initial 9AE0 ± 0AE56, final 5AE9 ± 0AE89 lmol/l; P ¼ < 0AE0005). There was also a significant rise in serum erythropoietin (initial 240 ± 195AE1, final 433AE2 ± 269AE2 U/l; P ¼ 0AE034). The haemoglobin level rose in three patients and transfusion requirements were reduced substantially in four patients. Serum thiobarbituric acid reactive substance (TBARS) also fell in six of eight patients. Patients generally improved clinically, with weight gain observed. Side-effects were mild and included gastrointestinal symptoms (6) and arthralgia (1), not requiring withdrawal of the drug. One patient died at 17 weeks of therapy as a result of an intercurrent infection. His neutrophil count was normal. We conclude that deferiprone is an effective, well-tolerated iron chelator for patients with thalassaemia intermedia. Further studies are needed to determine the optimum dose and length of treatment needed to reduce iron burden to a safe level in these patients.

Deferiprone versus deferoxamine in thalassemia intermedia: Results from a 5‐year long‐term Italian multicenter randomized clinical trial

“…This study confirms these previous reports [12][13][14][15][16]. However, it additionally suggests that neutropenia and agranulocytosis may be detected (Supporting Information Table 2).…”

“…DFP use has been reported in small cohorts of patients with HbE/beta-thalassemia, HbH disease, and TI. Significant reductions in serum ferritin levels have been observed in these studies, with the primary related AEs being gastrointestinal symptoms [12][13][14][15][16].…”

“…Previous reports suggested that DFP administration is well tolerated, with the primary AEs being gastrointestinal symptoms and joint pain or arthralgia [12][13][14][15][16].…”

“…However, it additionally suggests that neutropenia and agranulocytosis may be detected (Supporting Information Table 2). It is possible that the lack of neutropenia and agranulocytosis in previous publications may be due to the small numbers of patients enrolled in these studies [12][13][14][15][16].…”

Non Transferrin Bound Iron: Nature, Manifestations and Analytical Approaches for Estimation

“…with and without oxalate mobilization were found [20]. Pootrakul et al [50] estimated the levels of LPI and DCI separately in iron overloaded patients and found that both are highly correlated fractions. To a small extent where they differ is 25 % lower values of LPI which may stand for minor component of DCI which is not redox active.…”