地中海贫血的孩子长大成人。

Renzo Galanello
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引用次数: 0

摘要

在过去的几十年里,随着红细胞输血的进步和铁螯合疗法的引入,地中海贫血患者的治疗发生了很大的变化。这一进展大大增加了地中海贫血儿童在成年后生活质量良好的可能性。目前,地中海贫血重症患者的预后是“开放式”的。依从性常规治疗和心理支持是获得良好效果的关键。对高质量长期生存的期望鼓励患者计划他们未来的生活,有工作,有家庭,经常还有孩子。严重地中海贫血的最佳治疗是昂贵的,因此,不幸的是,世界上只有少数患者可以获得治疗。尽管取得了重大进展,但预计其他进展将进一步提高生存率和生活质量。主要目标是治愈这种疾病,增加使用hla匹配的非亲属供体进行骨髓移植的可能性,并有希望在未来进行基因治疗。然而,即使是常规治疗,特别是铁螯合治疗也有望改善。西方国家以及国际卫生和经济组织应作出努力,为在世界所有地方实现对地中海贫血的高标准管理提供持续和具体的支持。
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A thalassemic child becomes adult.

In the last decades, treatment of patients with beta-thalassemia has changed considerably, with advances in red cell transfusion and the introduction of iron chelation therapy. This progress has greatly increased the probability for a thalassemic child to reach adult age with a good quality of life. At present, the prognosis for thalassemia major patients is "open-ended". Compliance with the conventional treatment and psychological support are critical to obtain good results. The expectancy of a long survival of good quality encourages the patients to plan their future life, having a job, a family and often children. Optimal treatment of thalassemia major is expensive and for this reason, unfortunately, available only for a minority of patients in the world. Despite the significant advances, other progresses are expected to further improve survival and quality of life. The major aim is the cure of the disease, increasing the possibility of bone marrow transplantation using HLA-matched unrelated donors, and hopefully, in the future, gene therapy. However, even the conventional treatment and in particular iron chelation is expected to improve. Efforts should be made by the Western countries, and by the international health and economic organizations to provide continuous and concrete support for achieving a high standard of management for thalassemia in all places of the world.

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