Use of the oral chelator deferiprone in the treatment of iron overload in patients with Hb H disease

• Published in: British journal of haematology Vol. 133; no. 2; pp. 198 - 205
• Main Authors: Chan, Joyce C. W., Chim, Chor‐Sang, Ooi, Clara G. C., Cheung, Bernard, Liang, Raymond, Chan, Tai‐Kwong, Chan, Vivian
• Format: Journal Article
• Language: English
• Published: Oxford, UK Blackwell Publishing Ltd 01.04.2006; Blackwell
• Subjects: Administration, Oral; Adult; Aged; alpha-Thalassemia - complications; alpha-Thalassemia - physiopathology; Anemias. Hemoglobinopathies; Biological and medical sciences; Diastole; Diseases of red blood cells; Echocardiography, Doppler, Pulsed; Female; Ferritins - blood; haemoglobin H disease; Hematologic and hematopoietic diseases; Hematology; Humans; Iron - metabolism; Iron Chelating Agents - adverse effects; Iron Chelating Agents - therapeutic use; iron overload; Iron Overload - diagnostic imaging; Iron Overload - drug therapy; Iron Overload - etiology; Iron Overload - physiopathology; Liver - metabolism; Magnetic Resonance Imaging - methods; Male; Medical sciences; Middle Aged; oral chelator; Pyridones - adverse effects; Pyridones - therapeutic use; Ventricular Function, Left; Human; Hemoglobinopathy; iron overload; Hematology; oral chelator; Hemopathy; Deferiprone; Iron; Genetic disease; Unstable hemoglobin; Treatment; Overload; haemoglobin H disease; Chelating agent; Hemoglobin H
• ISSN: 0007-1048; 1365-2141
• DOI: 10.1111/j.1365-2141.2006.05984.x

Summary 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 T1‐signal intensity ratio (T1‐SIR) and gradient echo T2‐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.