Deferiprone versus Deferoxamine in Patients with Thalassaemia Major: A Randomized Clinical Trial

Authors
Aurelio Maggio, Gennaro D’Amico, Alberto Morabito, Marcello Capra, Calogero Ciaccio, Paolo Cianciulli, Felicia Di Gregorio, Giovanni Garozzo, Roberto Malizia, Carmelo Magnano, Antonio Mangiagli, Giovanni Quarta, Michele Rizzo, Domenico Giuseppe D’Ascolaa, Aroldo Rizzo and Massimo Midiri.

Journal
Blood Cells, Molecules and Diseases (2002) 28(2) Mar/Apr: 196-208

Background
Although deferoxamine represents standard chelation therapy, it requires patients to administer overnight subcutaneous infusions that are painful and associated with serious side effects. It would be a major advance in the treatment of thalassaemia to provide patients with an oral iron chelator that offers similar safety and efficacy to deferoxamine. The objective of the study was to compare the effectiveness of deferiprone and deferoxamine in reducing iron overload or to prevent its increase.

Methods

• A randomized trial of deferiprone versus deferoxamine in 144 patients with serum ferritin levels £ 3000 ng/ml at the onset of the trial. All patients were receiving deferoxamine at 50 mg/kg subcutaneously over a 12-hour period 5 days a week prior to the start of the trial.
• Patients were randomized into the deferiprone group who were dosed 75 mg/kg/day in three equal doses, or the deferoxamine group who were dosed at 50 mg/kg subcutaneously over a 12 hour period 5 days a week.
• The main measure of efficacy in the trial was the difference between serum ferritin concentration before and after 1 year of treatment. There were a number of secondary efficacy measures including liver iron concentration (LIC). Liver biopsies were obtained before and after treatment in all patients who accepted this. The Ishak system was used to score the inflammation and fibrosis of the liver samples.

Results – deferiprone lowers serum ferritin concentration.

Deferiprone-treated patients had an overall reduction in mean serum ferritin concentration that was comparable to the deferoxamine group after 1 year of treatment. 



Results – deferiprone lowers liver iron concentration.

For patients who agreed to repeat liver biopsies, deferiprone-treated patients had an overall reduction in LIC similar to the deferoxamine group after 1 year of treatment.

Results – No appreciable difference in liver fibrosis was found.

For patients who agreed to repeat liver biopsies, the mean fibrosis scores before treatment were 2.1 ± 1.3 (median 2, range 1.5 – 2.7) in the deferiprone group and 2.2 ± 1.3 (median 2, range 1.5 – 3.05) in the deferoxamine group (p = 0.77). The corresponding scores after treatment were not different from the pretreatment scores: 2.1 ± 1.5 (median 2, range 1.4 – 2.8) in the deferiprone group and 2.2 ± 1.2 (median 2, range 1.5 – 2.9) in the deferoxamine group (p = 0.84; t = -0.20). Overall fibrosis scores increased in 7 of the 21 deferiprone-treated patients and in 4 of the 15 deferoxamine-treated patients.

Conclusions
Over a short time period in patients with serum ferritin concentrations < 3000 ng/ml deferiprone has an ability to reduce iron load similar to that of deferoxamine. Deferiprone proved to be safe effective.

• If you would like to read the entire Maggio study (external site - membership required), click here.

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