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Haematopoietic stem cell transplantation for thalassaemia major.

机译：重型地中海贫血的造血干细胞移植。

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摘要

Haematopoietic stem cell transplantation (HSCT) is the only curative treatment for thalassaemia major (TM) patients. Modern conventional therapy with safe transfusion and effective iron chelation can lead to prolonged survival in TM patients. It was aimed at studying the benefits of HSCT as compared with conventional treatment in Hong Kong Chinese children.;In the second part of study on BMT, it demonstrated that 86% of patients could be cured by BMT. A more immunosuppressive preparative regimen including anti-thymocyte globulin reduced graft rejection without increase in toxicity. Age and hepatic fibrosisckli/MD was found to be important prognostic factors. Monitoring the busulphan levels with adjustment of dosage during the preparative period might reduce the early mortality. The survivors were free from further blood transfusion and its related complications. Body iron could be reduced to a safe level by desferrioxamine or phlebotomy, complications such as cardiomyopathy were prevented. After transplant liver function and growth improved, diabetes mellitus and hypothyroidism was prevented. However ovarian failure was universal in girls due to busulphan toxicity. Immune function after transplant was normal in most aspects except lower natural killer cells and delayed response to vaccine. However TM patients on conventional treatment showed progressive decrease in natural killer cells and low T-helper cells with age.;In summary, HSCT can achieve cure for this severe hereditary disease and also improves the quality of life. If HLA identical sibling is available, BMT is the recommended treatment in young patients as they have excellent long-term disease-free survival, over 90%.;The first part of this study on non-transplant patients demonstrated that morbidity and mortality was still common due to poor compliance to chelation therapy. Patients were at risk of complications due to iron overload. Patients older than 10 years have higher chance of cardiomyopathy (19%) and diabetes mellitus (11%), delayed puberty was still common. Cardiomyopathy was the leading cause of death followed by infection. Patients received chelation therapy from young age had 90% chance of surviving up to 20 years. However impaired immunity and progressive liver diseases were common, and these abnormalities may become more important as these patients grow older.
机译：造血干细胞移植（HSCT）是重型地中海贫血（TM）患者的唯一治疗方法。具有安全输血和有效铁螯合作用的现代常规疗法可导致TM患者的生存期延长。目的在于研究HSCT与香港中国儿童相比常规治疗的益处。；在BMT研究的第二部分中，它表明86％的患者可以通过BMT治愈。包括抗胸腺细胞球蛋白在内的更具免疫抑制作用的制备方案可减少移植排斥，但不会增加毒性。发现年龄和肝纤维化ckli / MD是重要的预后因素。在准备期间通过调整剂量来监测白头翁水平可能会降低早期死亡率。幸存者没有进一步输血及其相关并发症。可以通过去铁敏或静脉放血将人体铁含量降低到安全水平，可以预防诸如心肌病的并发症。移植肝功能和生长改善后，可预防糖尿病和甲状腺功能减退症。然而，由于busulphan毒性，卵巢衰竭在女孩中普遍存在。移植后的免疫功能在大多数方面都是正常的，除了较低的自然杀伤细胞和延迟的疫苗反应。然而，接受常规治疗的TM患者显示随着年龄的增长自然杀伤细胞和低T辅助细胞逐渐减少。总之，HSCT可以治愈这种严重的遗传性疾病并改善生活质量。如果可获得HLA同胞兄弟姐妹，则BMT是年轻患者的推荐治疗方法，因为它们具有优异的长期无病生存率（超过90％）.;本项非移植患者研究的第一部分表明发病率和死亡率仍然很高常见由于对螯合疗法的依从性差。由于铁过载，患者有发生并发症的风险。年龄大于10岁的患者发生心肌病的几率更高（19％）和糖尿病（11％），青春期延迟仍然很常见。心肌病是导致死亡然后感染的主要原因。从年轻时开始接受螯合疗法的患者有90％的机会存活至20年。但是，免疫力受损和进行性肝病很常见，随着这些患者年龄的增长，这些异常可能变得更加重要。

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  • 作者

    Li, Chi Kong.;

  • 作者单位

    The Chinese University of Hong Kong (Hong Kong).;

  • 授予单位 The Chinese University of Hong Kong (Hong Kong).;
  • 学科 Health Sciences Medicine and Surgery.;Health Sciences Public Health.
  • 学位 M.D.
  • 年度 2002
  • 页码 281 p.
  • 总页数 281
  • 原文格式 PDF
  • 正文语种 eng
  • 中图分类
  • 关键词

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