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Previous Volume 332:918-922 April 6, 1995 Number 14 Next

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ABSTRACT

Background To determine whether the orally active iron chelator deferiprone (1,2-dimethyl-3-hydroxypyridin-4-one) is efficacious in the treatment of iron overload in patients with thalassemia major, we conducted a prospective trial of deferiprone in 21 patients unable or unwilling to use standard chelation therapy with parenteral deferoxamine.

Methods Hepatic iron stores were determined yearly by chemical analysis of liver-biopsy specimens or magnetic-susceptibility measurements. Detailed clinical and laboratory studies were used to monitor safety and compliance.

Results The patients received deferiprone therapy for a mean (±SE) of 3.1±0.3 years. Ten patients in whom previous chelation therapy with deferoxamine had been ineffective had initial hepatic iron concentrations of at least 80 µmol per gram of liver, wet weight — values associated with complications of iron overload. Hepatic iron concentrations decreased in all 10 patients, from 125.3±11.5 to 60.3±9.6 µmol per gram (P<0.005), with values that were less than 80 µmol per gram in 8 of the 10 patients (P<0.005). In all 11 patients in whom deferoxamine therapy had previously been effective, deferiprone maintained hepatic iron concentrations below 80 µmol of iron per gram.

Conclusions Oral deferiprone induces sustained decreases in body iron to concentrations compatible with the avoidance of complications from iron overload. The risk of agranulocytosis associated with deferiprone may restrict its administration to patients who are unable or unwilling to use deferoxamine.

Source Information

From the Department of Pediatrics, Hospital for Sick Children (N.F.O., D.M., M.B., G.K.); the Departments of Medicine (N.F.O., L.M.B., P.P.L.) and Pathology (R.G.C.), Toronto Hospital; and the Departments of Chemistry (R.A.M.) and Clinical Biochemistry (D.M.T.), University of Toronto — all in Toronto; and the Department of Medicine, MetroHealth Medical Center and Case Western Reserve University, Cleveland (G.M.B.).

Address reprint requests to Dr. Olivieri at the Hemoglobinopathy Program, Hospital for Sick Children, 555 University Ave., Toronto, ON M5G 1X8, Canada.

Full Text of this Article

Related Letters:

Deferiprone in Iron Overload
Mehta J., Singhal S., Mehta B.C., Adhikari D., Hoffbrand A.V., Wonke B., Nathan D. G.
Extract | Full Text  
N Engl J Med 1995; 333:597-599, Aug 31, 1995. Correspondence

Iron Chelation with Oral Deferiprone in Patients with Thalassemia
Tricta F., Spino M., Callea F., Nathan D. G., Weatherall D. J., Stella M., Pinzello G., Maggio A., Wonke B., Telfer P., Hoffbrand A.V., Grady R. W., Giardina P. J., Cohen A. R., Martin M. B., Brittenham G. M., Fleming K. A., Templeton D. M., Olivieri N. F., Kowdley K. V., Kaplan M. M.
Extract | Full Text  
N Engl J Med 1998; 339:1710-1714, Dec 3, 1998. Correspondence

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