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 μg/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 μg/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 μg/l at 18 months, P = 0·0008). Nine of 16 patients had levels below 397 μg/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 (T1-SIR) and gradient echo T 2-signal intensity ratio (T2-SIR) increased with treatment. T2-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. © 2006 Blackwell Publishing Ltd.link_to_subscribed_fulltex