Use of the oral chelator deferiprone in the treatment of iron overload in patients with Hb H disease
Use of the oral chelator deferiprone in the treatment of iron overload in patients with Hb H disease
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DOI：
10.1111/j.1365-2141.2006.05984.x
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发表时间：
2006-04-01
影响因子：
6.5
通讯作者：
Chan, V
中科院分区：
文献类型：
--
作者：
Chan, JCW;Chim, CS;Chan, V
17例血清铁蛋白> 900 μ g/l的非输血依赖型中国血红蛋白H病患者（年龄29-76岁）接受去铁酮治疗长达18个月。1例患者退出研究，分析了16例患者的数据。另外16名年龄和基因型匹配的铁蛋白< 900 μ g/l的Hb H患者作为对照。除轻度关节痛外，治疗耐受性良好。血清铁蛋白随治疗而下降，在6个月和18个月时达到显著性（从1492.3 +/- 901.4至18个月时的519.4 +/- 405.4 μ g/l，P = 0.0008）。16名患者中有9名在18个月前的水平低于397 μ g/l。停药后6个月血清铁蛋白保持稳定。相反，对照组的铁蛋白水平没有变化。磁共振成像用于测量肝脏铁含量。自旋回波T-1信号强度比（T-1-SIR）和梯度回波T-2信号强度比（T-2-SIR）随治疗增加。T-2-SIR从治疗前的0.17 +/- 0.08上升至2年时的0.58 +/- 0.50（P = 0.0055）。16例患者中有12例出现改善，3例患者达到正常。使用超声心动图，舒张早期峰值：舒张晚期血流（E/A）与治疗保持不变，但等容舒张时间（IVRT）在2年时延长，表明舒张功能轻度受损。所有收缩功能参数均正常。需要更长的治疗期来证明心脏功能的改善。
Seventeen non-transfusion-dependent Chinese haemoglobin H (Hb H) disease patients (age 29-76 years) with serum ferritin > 900 mu g/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 mu g/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 mu g/l at 18 months, P = 0.0008). Nine of 16 patients had levels below 397 mu g/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.