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21 世纪重型β地中海贫血症的成功螯合疗法。

Successful chelation in beta-thalassemia major in the 21st century.

机构信息

Thalassemia Unit, "Aghia Sofia" Children's Hospital, Athens, Greece.

Thalassemia Unit, "G. Gennimatas" General Hospital, Athens, Greece.

出版信息

Medicine (Baltimore). 2023 Oct 13;102(41):e35455. doi: 10.1097/MD.0000000000035455.

Abstract

This century has seen a revolution the management of beta-thalassemia major. Over a 12-year period to 2016, we aimed to analyze the benefits of such advances. In 209 patients, independent of the chelation regimen, ferritin, cardiac T2* and liver iron concentration changes were evaluated. We defined chelation success (ChS) as no iron load in the heart and acceptable levels in the liver. Over 3 early magnetic resonance imagings, the same parameters were assessed in 2 subgroups, the only 2 that had sufficient patients continuing on 1 regimen and for a significant period of time, 1 on deferrioxamine (low iron load patients n = 41, Group A) and 1 on deferoxamine-deferiprone (iron overloaded n = 60, Group B). Finally, 28 deaths and causes were compared to those of an earlier period. The 209 patients significantly optimized those indices, while the number of patients with chelation success, increased from 6% to 51% (P < .0001). In group A, ChS after about 8 years increased from 21 to 46% (P = .006), while in Group B, from 0% to 60% (P < .001) after about 7 years. Deaths over the 2 periods showed significant reduction. Combined clearance of cardiac and liver iron (ChS) is feasible and should become the new target for all patients. This requires, serial magnetic resonance imagings and often prolonged intensified chelation for patients.

摘要

本世纪见证了β-地中海贫血重症管理的一场革命。在截至 2016 年的 12 年期间,我们旨在分析这些进展的益处。在 209 名患者中,无论螯合方案如何,均评估铁蛋白、心脏 T2*和肝脏铁浓度变化。我们将螯合成功(ChS)定义为心脏无铁负荷和肝脏可接受的水平。在 3 次早期磁共振成像中,在 2 个亚组中评估了相同的参数,这是仅有的 2 个具有足够数量的患者继续使用 1 种方案且持续时间较长的亚组,1 种使用去铁胺(低铁负荷患者 n=41,A 组),1 种使用去铁胺-地拉罗司(铁过载患者 n=60,B 组)。最后,将 28 例死亡及其原因与早期进行了比较。209 名患者显著优化了这些指标,而螯合成功的患者数量从 6%增加到 51%(P<0.0001)。在 A 组中,大约 8 年后,ChS 从 21%增加到 46%(P=0.006),而在 B 组中,大约 7 年后,ChS 从 0%增加到 60%(P<0.001)。两个时期的死亡人数均显著减少。心脏和肝脏铁的联合清除(ChS)是可行的,应成为所有患者的新目标。这需要对患者进行连续磁共振成像,并且通常需要延长强化螯合治疗。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/d523/10578721/5bbef87234e0/medi-102-e35455-g001.jpg

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