U.O.D. Centro per le Microcitemie, A.O.R.N. A. Cardarelli, Napoli, Italy.
Blood Cells Mol Dis. 2013 Aug;51(2):85-8. doi: 10.1016/j.bcmd.2013.04.002. Epub 2013 Apr 28.
Transfusion and iron chelation treatment have significantly reduced morbidity and improved survival of patients with thalassemia major. However, cardiac disease continues to be the most common cause of death. We report the left-ventricular ejection fraction, determined by echocardiography, in one hundred sixty-eight patients with thalassemia major followed for at least 5years who received continuous monotherapy with deferoxamine (N=108) or deferiprone (N=60). The statistical analysis, using the generalized estimating equations model, indicated that the group treated with deferiprone had a significantly better left-ventricular ejection fraction than did those treated with deferoxamine (coefficient 0.97; 95% CI 0.37; 1.6, p=0.002). The heart may be particularly sensitive to iron-induced mitochondrial damage because of the large number of mitochondria and its low level of antioxidants. Deferiprone, because of its lower molecular weight, might cross into heart mitochondria more efficiently, improving their activity and, thereby, myocardial cell function. Our findings indicate that the long-term administration of deferiprone significantly enhances left-ventricular function over time in comparison with deferoxamine treatment. However, because of limitations related to the design of this study, these findings should be confirmed in a prospective, randomized clinical trial.
输血和铁螯合治疗显著降低了重型地中海贫血患者的发病率并提高了他们的生存率。然而，心脏疾病仍然是最常见的死亡原因。我们报告了 168 例至少接受 5 年连续单一药物治疗的重型地中海贫血患者的左心室射血分数，这些患者分别接受了去铁胺（N=108）或地拉罗司（N=60）治疗。使用广义估计方程模型的统计分析表明，接受地拉罗司治疗的患者的左心室射血分数明显优于接受去铁胺治疗的患者（系数 0.97；95%CI 0.37；1.6，p=0.002）。由于心脏中线粒体数量多且抗氧化剂水平低，心脏可能对铁诱导的线粒体损伤特别敏感。由于分子量较小，地拉罗司可能更有效地进入心脏线粒体，从而提高其活性，进而改善心肌细胞功能。我们的研究结果表明，与去铁胺治疗相比，地拉罗司的长期给药随着时间的推移显著增强了左心室功能。然而，由于这项研究设计的局限性，这些发现应在前瞻性、随机临床试验中得到证实。
