Li Changgang, Liu Sixi, Wang Ying, Wen Feiqiu, Gao Hongying, Chen Guangfu, Li Chunfu, Wu Xuedong, Fang Jianpei, Hao Wenge, Liu Riyang, Zhang Xinhua, Chu Chiuwing Winnie, Au WingYan
Department of Hematology and Oncology, Shenzhen Children's Hospital, Shenzhen 518026, China.
Zhonghua Er Ke Za Zhi. 2014 Feb;52(2):90-3.
To observe the status of iron deposition in patient with β thalassemia major, and to formulate appropriate treatment strategies.
The data of status of transfusion and chelation in 135 patients aged from 6 years and 4 months to 17 years and 11 months with β thalassemia major were collected and analyzed. Serum ferritin levels were determined and cardiac and hepatic iron deposition was determined using MRI T2(*) technology.
Of the 135 cases studied, 66 were male, and 69 were female, their average age was 12.1 years. Serum ferritin (SF) was determined for 111 cases, it varied from 1 086.8 µg/L to 15 011.5 µg/L. Among them, 16 cases had SF level <2 000 µg/L (14.5%) , in 41 cases SF were between 2 000 and 4 000 µg/L (36.0%) ;in 54 cases SF >4 000 µg/L (48.7%) . Liver MRI T2() results showed that in only 8 cases (5.9%) iron content in the liver was in normal range, 19 cases (14.9%) showed mild liver iron deposition;34 (25.2%) moderate and 74 (54.8%, the youngest one was only 6 years and 4 months of age) had severe iron deposition respectively. Cardiac MRI T2() showed that in 89 cases (65.9%) iron content in the heart was in normal range;19 cases (14.1%) had mild cardiac iron deposition and 27 (20.0%) presented severe iron deposition (the youngest one was only 9 years and 3 months of age) . SF level was obviously related to liver and cardiac iron deposition (MRI T2(*)) r and P value were -0.284, 0.003 and -0.374, 0.000 respectively. In 108 cases regular transfusion and chelation were delayed due to financial problem. The late and insufficient dosage administered and irregular chelation caused the higher SF level and the severe iron deposition.
The survival status of β thalassemia major in China is worrisome. Majority of them had not received regular transfusion and chelation. Liver and cardiac iron deposition occur early and had a high incidence.
观察重型β地中海贫血患者铁沉积状况，制定合适的治疗策略。
收集并分析135例年龄在6岁4个月至17岁11个月的重型β地中海贫血患者的输血及螯合治疗状况数据。测定血清铁蛋白水平，并使用MRI T2（*）技术测定心脏和肝脏的铁沉积情况。
在研究的135例患者中，男性66例，女性69例，平均年龄12.1岁。对111例患者测定了血清铁蛋白（SF），其范围为1 086.8 μg/L至15 011.5 μg/L。其中，16例SF水平<2 000 μg/L（14.5%），41例SF在2 000至4 000 μg/L之间（36.0%）；54例SF>4 000 μg/L（48.7%）。肝脏MRI T2（）结果显示，仅8例（5.9%）肝脏铁含量在正常范围，19例（14.9%）显示轻度肝脏铁沉积；34例（25.2%）中度和74例（54.8%，最小的仅6岁4个月）分别有重度铁沉积。心脏MRI T2（）显示，89例（65.9%）心脏铁含量在正常范围；19例（14.1%）有轻度心脏铁沉积，27例（20.0%）有重度铁沉积（最小的仅9岁3个月）。SF水平与肝脏和心脏铁沉积（MRI T2（*））明显相关，r和P值分别为-0.284、0.003和-0.374、0.000。108例患者因经济问题输血及螯合治疗不规律。后期剂量不足及螯合治疗不规律导致SF水平升高及铁沉积严重。
中国重型β地中海贫血患者的生存状况令人担忧。大多数患者未接受规律的输血及螯合治疗。肝脏和心脏铁沉积出现早且发生率高。
