Iron Chelation Effects of Different Treatment Protocols in Thalassaemia Major: Comparison by Magnetic Resonance T2* over Two Years
DD Rasalkar, RKL Lee, V Lee, A Leung, FWT Cheng, WWM Lam, CK Li, WCW Chu
Hong Kong J Radiol 2012;15:88-95
Objective: Myocardial haemosiderosis leading to cardiac dysfunction is a major complication in transfusiondependent thalassaemia. Our objective was to compare the iron chelation efficacy of the recently launched deferasirox (ICL670) with conventional deferoxamine (DFO) and the combined regimen of deferiprone (L1) and DFO in thalassaemia major patients, based on our local experience.
Methods:This was a retrospective study. Nineteen thalassaemia major patients received ICL670 for one year (as part of a therapeutic trial). All underwent magnetic resonance imaging assessment of T2* for myocardial and hepatic iron content and left ventricular ejection fraction at baseline and after one year. Twelve and 21 patients with similar T2* cardiac or liver values at baseline were selected as controls, who received DFO and combination therapy (DFO+L1), respectively. Changes in T2* values, left ventricular ejection fraction, and serum ferritin were evaluated at baseline and follow-up and the efficacy of each type of treatment was compared (analysis of variance with one factor).
Results:In all three treatment groups, there was significant improvement of mean myocardium T2* (p<0.05), and the DFO+L1 and ICL670 groups showed significant improvements in mean left ventricular ejection fraction (p<0.05), as well as liver T2* and ferritin levels.
Conclusion: This study suggests that ICL670 is as efficient as DFO and DFO+L1 for iron chelation in the heart. ICL670 is comparable to combination therapy in improving both left ventricular ejection fraction and iron chelation of the liver.
中文摘要
針對重型地中海貧血患者的不同治療方案中鐵螯合劑效果的比較： 連續兩年T2加權磁共振成像的研究
DD Rasalkar、李嘉樂、李偉生、梁永堃、鄭偉才、林慧雯、李志光、朱昭穎
目的：對於依靠輸血的地中海貧血患者來說，心肌血鐵質沉着病引致心功能不全屬嚴重併發症。本 文在香港地中海貧血患者中，比較三種療法的去鐵效果，它們分別為：最新推出的口服鐵螯合劑（deferasirox, ICL670）、傳統的去鐵胺（deferoxamine, DFO），以及排鐵劑deferiprone（L1）與DFO 的聯合療法。
方法：本回顧研究的對象為19名重型地中海貧血患者。作為治療試驗的一部分，他們均接受ICL670 已達一年。在開始治療前和接受治療一年後，用磁共振成像(MR) T2* 量度患者的心肌和肝的鐵含 量，以及左心室射血分數。與此同時，安排兩組對照組，其中一組是接受DFO的12名患者，另一組 是接受L1及DFO聯合療法的21名患者。在開始試驗前，兩對照組的心肌T2*值和肝的鐵含量都與實 驗組相似。利用ANOVA比較三組患者在治療開始前和治療後的T2*值、左心室射血分數、及血清鐵 蛋白值的轉變。並比較每種治療的效用。
結果：三組患者心肌T2*值均有顯著改善（p<0.05）。接受ICL670的患者及接受L1及DFO聯合療法的 患者在左心室射血分數有明顯改善（p<0.05），而兩組在肝T2*值和鐵蛋白水平方面亦有改善。
結論：本研究顯示ICL670在心臟的鐵螯合治療方面，可媲美DFO和L1及DFO聯合療法。對於改善左 心室射血分數及肝的鐵螯合治療方面，ICL670可媲美L1及DFO聯合療法。