title: Use of the oral chelator deferiprone in the treatment of iron overload in patients with Hb H disease.
abstract: Seventeen non-transfusion-dependent Chinese haemoglobin H (Hb H) disease patients (age 29-76 years) with serum ferritin >900 microg/l were treated with deferiprone for up to 18 months. One patient withdrew and data from 16 patients were analysed. Sixteen other Hb H patients with ferritin <900 microg/l, matched for age and genotype, acted as controls. Treatment was well tolerated except for mild arthralgia. Serum ferritin fell with treatment, reaching significance at 6 and 18 months (from 1492.3 +/- 901.4 to 519.4 +/- 405.4 microg/l at 18 months, P = 0.0008). Nine of 16 patients had levels below 397 microg/l before 18 months. Serum ferritin remained stable 6 months after stopping treatment. In contrast, there was no change in ferritin levels in the control group. Magnetic resonance imaging was used for measurement of liver iron content. Spin echo T(1)-signal intensity ratio (T(1)-SIR) and gradient echo T(2)-signal intensity ratio (T(2)-SIR) increased with treatment. T(2)-SIR rose from 0.17 +/- 0.08 pretreatment to 0.58 +/- 0.50 at 2 years (P = 0.0055). Improvement occurred in 12 of 16 patients, reaching normal in three patients. Using echocardiography, peak early diastolic : late diastolic blood flow (E/A) remained unchanged with treatment, but isovolumic relaxation time (IVRT) was prolonged at 2 years indicating mild impairment of diastolic function. All systolic function parameters were normal. A longer treatment period is desirable to demonstrate improvement in cardiac function.
authors.affiliation: Department of Medicine, Pamela Youde Nethersole Eastern Hospital, Hong Kong, China.
authors.forename: Joyce C W
authors.initials: JC
authors.lastname: Chan
authors.forename: Chor-Sang
authors.initials: CS
authors.lastname: Chim
authors.forename: Clara G C
authors.initials: CG
authors.lastname: Ooi
authors.forename: Bernard
authors.initials: B
authors.lastname: Cheung
authors.forename: Raymond
authors.initials: R
authors.lastname: Liang
authors.forename: Tai-Kwong
authors.initials: TK
authors.lastname: Chan
authors.forename: Vivian
authors.initials: V
authors.lastname: Chan
mesh_terms: D000284:Administration, Oral; D000328:Adult; D000368:Aged; D000077543:Deferiprone; D003971:Diastole; D018619:Echocardiography, Doppler, Pulsed; D005260:Female; D005293:Ferritins / Q000097:blood; D006801:Humans; D007501:Iron / Q000378:metabolism; D007502:Iron Chelating Agents / Q000009:adverse effects / Q000627:therapeutic use*; D019190:Iron Overload / Q000000981:diagnostic imaging / Q000188:drug therapy* / Q000209:etiology / Q000503:physiopathology; D008099:Liver / Q000378:metabolism; D008279:Magnetic Resonance Imaging / Q000379:methods; D008297:Male; D008875:Middle Aged; D011728:Pyridones / Q000009:adverse effects / Q000627:therapeutic use*; D016277:Ventricular Function, Left; D017085:alpha-Thalassemia / Q000150:complications* / Q000503:physiopathology
chemical_list: D007502:Iron Chelating Agents; D011728:Pyridones; D000077543:Deferiprone; D005293:Ferritins; D007501:Iron