The reputation of deferiprone, the controversial drug at the heart of one of Canada's bitterest academic rows (see CMAJ 2002;166[4]:452-3), has been buoyed by 2 new studies.
Deferiprone is an orally active iron chelator used to treat patients with thalassemia major. The drug, which is given orally, is better tolerated than deferoxamine, which must be given 5 to 7 days per week by prolonged subcutaneous infusion.
In 1998, Toronto researcher Nancy Olivieri and colleagues (N Engl J Med 1998; 339:417-23) concluded that the drug may worsen the hepatic fibrosis that is typical of the iron-overloaded state endured by these children. The study involved 14 patients.
One of the new studies, by Ian Wanless and colleagues, involved an analysis of the largest collection of liver biopsies (112) from patients receiving deferiprone. It concluded that patients taking it show “no evidence of deferiprone-induced progression of hepatic fibrosis during long-term therapy.” The study was prepublished online (Blood 2002;100:1566-9). The authors write: “The differing conclusion of Olivieri et al may be related to the small number of patients [14] in their study and to the difficulties of grading fibrosis in the small biopsies.”
The second study, by Lisa Anderson and associates (Lancet 2002;360:516-20), involved a comparison of myocardial iron content and cardiac function in 15 patients receiving long-term deferiprone therapy and 30 matched controls receiving long-term treatment with deferoxamine. The authors report that deferiprone was more effective than deferoxamine in removing iron from the heart — an important finding because heart failure is the most frequent cause of death among thalassemia patients — but less effective than deferoxamine in removing iron from the liver.
In an accompanying commentary in The Lancet, Des Richardson notes that it may be time to try a combination of the 2 drugs and that it is time for a randomized clinical trial. — CMAJ