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论文摘要

去铁胺、去铁酮治疗重型β地中海贫血铁过载患者的临床研究

Clinical study of deferoxamine and deferiprone in severe β-thalassemia patients with iron overload

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收稿日期:2019-11-23          年卷(期)页码:2020,43(02):148-154

期刊名称:国际输血及血液学杂志

Journal Name:International Journal of Blood Transfusion and Hematology

关键字:β地中海贫血,铁超负荷,铁蛋白质类,去铁胺,去铁酮

Key words:Beta-thalassemia|Iron overload|Ferritins|Deferoxamine|Deferipron

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中文摘要

英文摘要

ObjectiveTo investigate the efficacy of deferoxamine and deferiprone in treating severe β-thalassemia patients with iron overload.

MethodsFrom June 1, 2015 to May 31, 2016, a total of 52 children with severe β-thalassemia complicated iron overload who were diagnosed and treated in the First Affiliated Hospital of Guangxi Medical University were collected in the study.According to the different treatment methods, they were divided into desferrioxamine group(take desferrioxamine,n=17), deferiprone group(take deferiprone,n=20), and combined treatment group(take desferrioxamine and deferiphone,n=15). Changes in MRI T2*values of cardiac and liver before and 12 months after treatment were observed, as well as changes in serum ferritin (SF) values before and 3, 6, 9, 12 months after treatment were observed among 3 groups.The paired samplettest was used for comparison between before and 12 months after treatment within group. The one-way variance analysis was used for comparison among 3 groups. The pairwise comparison was performed using the least significant difference (LSD) method. The procedure of this study is accordance with the requirement of the revisedWorld Medical Association Declaration of Helsinkiin 2013. Consent form was obtained from all subjects.

Results① After 12 months of treatment, the cardiac MRI T2*values of patient were (22.71±2.57), (26.50±3.32) and (34.93±8.26)ms in three groups, respectively, which were obviously higher than those before treatment[(13.76±3.85), (13.60±3.73) and (14.30±2.95)ms]. The differences were significant(t=-11.697, -11.352, -9.449;P<0 .001, 0.001, 0.001). after 12 months of treatment, the total differences of the cardiac mri t2*value was statistically significant among 3 groups(F=23.885,P<0 .001). meanwhile the cardiac mri t2*value in combined treatment group were improved than ferritamine group and deferiprone group(combined treatment groupvsferritamine group, LSD-t=-5.806,P<0 .001; combined treatment groupvsdeferiprone group, LSD-t=-5.213,P<0 .001). ②after 12 months of treatment, the liver mri t2*values of patient were(8.30±1.07), (8.27±1.25) and (9.04±1.01)ms, respectively, which were higher than those before treatment[(4.27±1.06), (4.24±1.01), (4.23±0.99)ms ], and the differences were significant(t=-12.378, -13.384, -12.619;P<0 .001, 0.001, 0.001). after 12 months of treatment, there was no significant difference in the liver mri t2*values among 3 groups (F= 2.423,P=0.099). But the liver MRI T2*in combined treatment group was significantly higher than those of deferoxamine group and deferiprone group(combined treatment groupvsferritamine group, LSD-t=-2.339,P=0.026; combined treatment groupvsdeferiprone group, LSD-t=-2.057,P=0.048). ③After 12 months of treatment, there were significant differences in SF values between before treatment [(3 051.88±233.44), (2 891.70±101.54), (2 800.60±202.99)μg/L] and 12 months after treatment[(3 442.88±137.91), (3 443.30±150.79), (3 460.27±227.52)μg/L] in 3 groups(t=22.33, 6.142, 22.744;P<0 .001, 0.001, 0.001), respectively. after 12 months treatment, the total differences of sf values were statistically significant among three groups(F=7.825,P= 0.001). The SF value in combined treatment group were uniformly improved than ferritamine group and deferiprone group(combined treatment groupvsferritamine group, LSD-t=2.855,P=0.008; combined treatment groupvsdeferiprone group, LSD-t=2.359,P=0.024).

ConclusionsDeferoxamine, deferiprone and combined treatment can effectively alleviate the children with severe β-thalassemia complicated iron overload. The combined treatment of deferoxamine and deferiprone can significantly reduce the level of SF value, better the cardiac iron overload, in of these patients, and the effect of cardiac iron overload is better.

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