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Title: Sequential Deferiprone-Deferoxamine Tops Deferiprone Alone in Treatment of Thalassemia Major: Presented at ASH
 "Sequential Deferiprone-Deferoxamine Tops Deferiprone Alone in Treatment of Thalassemia Major: Presented at ASH"


By Sophie Bainbridge ATLANTA, GA -- December 13, 2007 -- Sequential treatment with deferiprone plus deferoxamine is more effective than deferiprone alone -- the current "gold standard" -- in reducing ferritin levels in patients with thalassemia major, according to a multicenter, randomized, 5-year trial. At 1 year, combination treatment reduced serum ferritin levels by a mean of 409 ng/mL compared with a mean reduction of 105 ng/mL with deferiprone alone, study investigators reported here at the 49th American Society of Hematology (ASH) Annual Meeting and Exposition. The superiority of sequential deferiprone-deferoxamine persisted throughout the entire study, said the study's principal investigator Aurelio Maggio, MD, Hematologist, Azienda Ospedaliera "Vincenzo Cervello," Palermo, Italy. "Our long-term evidence-based results strongly suggest that this sequential treatment is safe and effective," he said in a presentation on December 10. The study, conducted under the auspices of the Society for the Study of Thalassemia and Hemoglobinopathies (SoSTE), based in Rome, Italy, randomized 140 consecutive patients with thalassemia major to deferiprone (n = 69) or to sequential deferiprone-deferoxamine (n = 71) and followed them for 5 years. All patients had similar mean baseline ferritin levels -- 1,859 ng/mL in those randomized to deferiprone alone and 1,778 ng/mL in patients receiving the sequential therapy. Their mean age was 23 years and mean baseline hemoglobin level was 10 g/dl. Eleven of the deferiprone-alone patients had a splenectomy, compared with 16 of the sequential deferiprone-deferoxamine group, but this difference was not statistically significant, Dr. Maggio said. In addition to the greater reduction in serum ferritin levels, which was the trial's primary endpoint, incidence of adverse drug reactions were significantly lower with sequential treatment; high transaminase levels developed in 18.8% of monotherapy patients and 7% of sequentially-treated patients. There were two treatment failures in the sequential arm and eight in the deferiprone-alone arm. No cases of agranulocytosis were reported and discontinuation rates were similar for both groups (55.6% and 57.7%, respectively; [P =.86).

Dr. Maggio said he had no conflicts of interest to declare.


[Presentation title: Deferiprone Versus Sequential Deferiprone-Deferoxamine Treatment in Thalassemia Major: A Five Year Multicenter Randomized Clinical Trial Under the Auspices of the Society for the Study of Thalassemia and Hemoglobinopathies (SoSTE). Abstract 575]






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