Oral iron chelation with 1,2-dimethyl-3-hydroxypyrid-4-one (L1) in iron loaded thalassemia patients.
Despite the successes of deferoxamine (DFO) in the treatment and prevention of iron overload, an effective orally available iron chelating drug is needed, since erratic compliance with irritating, cumbersome parenteral infusions still results in fatal iron accumulation in many patients. Disorders of increased iron absorption should also benefit from the development of safe and effective iron chelating agents. Individuals with non-transfusion-dependent thalassemia (thalassemia "intermedia"), exhibit excessive dietary iron absorption that can lead to serious iron loading by the second or third decade of life. An orally effective iron-chelating drug would have major therapeutic advantages for all these patients.
Department of Pediatrics and Clinical Biochemistry, Hospital for Sick Children, Toronto, Canada.
N F
NF
Olivieri
D
D
Matsui
P P
PP
Liu
L
L
Blendis
R
R
Cameron
R A
RA
McClelland
D M
DM
Templeton
G
G
Koren
D000284:Administration, Oral; D015913:Chelation Therapy; D000077543:Deferiprone; D006801:Humans; D007501:Iron / Q000652:urine; D007502:Iron Chelating Agents / Q000008:administration & dosage* / Q000627:therapeutic use; D007592:Joint Diseases / Q000139:chemically induced; D010349:Patient Compliance; D011728:Pyridones / Q000008:administration & dosage* / Q000627:therapeutic use; D017086:beta-Thalassemia / Q000188:drug therapy*
D007502:Iron Chelating Agents; D011728:Pyridones; D000077543:Deferiprone; D007501:Iron