title: Normalisation of total body iron load with very intensive combined chelation reverses cardiac and endocrine complications of thalassaemia major.
abstract: Cardiac and endocrine disorders are common sequelae of iron overload in transfused thalassaemia patients. Combined chelation with desferrioxamine (DFO) and deferiprone (DFP) is well tolerated and produces an additive/synergistic effect superior to either drug alone. 52 thalassaemia major patients were transitioned from DFO to combined chelation with DFO and DFP. Serum ferritin, cardiac and hepatic iron levels were monitored regularly for up to 7 years, as were cardiac and endocrine function. Patients' iron load normalized, as judged by ferritin and cardiac and hepatic magnetic resonance imaging findings. In all 12 patients receiving treatment for cardiac dysfunction, symptoms reversed following combined chelation, enabling nine patients to discontinue heart medications. In the 39 patients with abnormal glucose metabolism, 44% normalized. In 18 requiring thyroxine supplementation for hypothyroidism, 10 were able to discontinue, and four reduced their thyroxine dose. In 14 hypogonadal males on testosterone therapy, seven stopped treatment. Of the 19 females, who were hypogonadal on DFO monotherapy, six were able to conceive. Moreover, no patients developed de novo cardiac or endocrine complications. These results suggest that intensive combined chelation normalized patients' iron load and thereby prevented and reversed cardiac and multiple endocrine complications associated with transfusion iron overload.
authors.affiliation: Transfusion Department and Thalassaemia Unit, General Hospital of Corinth, Leoforos Athinon 53, Corinth 20100, Greece. stheni@otenet.gr
authors.forename: Kallistheni
authors.initials: K
authors.lastname: Farmaki
authors.forename: Ioanna
authors.initials: I
authors.lastname: Tzoumari
authors.forename: Christina
authors.initials: C
authors.lastname: Pappa
authors.forename: Giorgos
authors.initials: G
authors.lastname: Chouliaras
authors.forename: Vasilios
authors.initials: V
authors.lastname: Berdoukas
mesh_terms: D000293:Adolescent; D000328:Adult; D001786:Blood Glucose / Q000378:metabolism; D009202:Cardiomyopathies / Q000188:drug therapy* / Q000209:etiology; D002648:Child; D000077543:Deferiprone; D003676:Deferoxamine / Q000009:adverse effects / Q000627:therapeutic use; D004334:Drug Administration Schedule; D004359:Drug Therapy, Combination; D004700:Endocrine System Diseases / Q000188:drug therapy* / Q000209:etiology; D005260:Female; D005293:Ferritins / Q000097:blood; D005500:Follow-Up Studies; D006801:Humans; D007037:Hypothyroidism / Q000188:drug therapy / Q000209:etiology; D007502:Iron Chelating Agents / Q000009:adverse effects / Q000627:therapeutic use*; D019190:Iron Overload / Q000097:blood / Q000150:complications / Q000188:drug therapy*; D008099:Liver / Q000378:metabolism; D008297:Male; D011728:Pyridones / Q000009:adverse effects / Q000627:therapeutic use; D013974:Thyroxine / Q000008:administration & dosage; D065227:Transfusion Reaction; D055815:Young Adult; D017086:beta-Thalassemia / Q000150:complications / Q000628:therapy*
chemical_list: D001786:Blood Glucose; D007502:Iron Chelating Agents; D011728:Pyridones; D000077543:Deferiprone; D005293:Ferritins; D003676:Deferoxamine; D013974:Thyroxine