TY - JOUR
T1 - Iron-chelation therapy with oral deferiprone in patients with thalassemia major
AU - Olivieri, Nancy F.
AU - Brittenham, Gary M.
AU - Matsui, Doreen
AU - Berkovitch, Matitiahu
AU - Blendis, Laurence M.
AU - Cameron, Ross G.
AU - Mcclelland, Robert A.
AU - Liu, Peter P.
AU - Templeton, Douglas M.
AU - Koren, Gideon
PY - 1995/4/6
Y1 - 1995/4/6
N2 - 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. 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. 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. 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.
AB - 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. 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. 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. 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.
UR - http://www.scopus.com/inward/record.url?scp=0028898064&partnerID=8YFLogxK
U2 - 10.1056/NEJM199504063321404
DO - 10.1056/NEJM199504063321404
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C2 - 7877649
AN - SCOPUS:0028898064
SN - 0028-4793
VL - 332
SP - 918
EP - 922
JO - New England Journal of Medicine
JF - New England Journal of Medicine
IS - 14
ER -