Emerging trends in transplantation of inherited metabolic diseases

被引：54
作者：
Prasad, V. K. [1 ]
Kurtzberg, J. [1 ]
机构：
[1] Duke Univ, Med Ctr, Pediat Blood & Marrow Transplant Program, Durham, NC 27710 USA
来源：
BONE MARROW TRANSPLANTATION | 2008年 / 41卷 / 02期
关键词：
inherited metabolic diseases; lysosomal and peroxisomal storage disease; hematopoietic stem cell transplantation; umbilical cord blood transplantation;
D O I：
10.1038/sj.bmt.1705970
中图分类号：
Q6 [生物物理学];
学科分类号：
071011 ;
摘要：
Allogeneic hematopoietic stem cell transplantation (HSCT) can prolong life and improve its quality in patients with inherited metabolic diseases. HSCT offers a permanent source of enzyme replacement therapy and also might mediate nonhematopoietic cell regeneration or repair. Unrelated cord blood is an exciting newer graft source for treatment of patients with these fatal disorders, providing increased access to donors and significant clinical efficacy, particularly when transplantation is performed in early stages. Pre-transplant performance status is highly predictive of overall survival.
引用
收藏
页码：99 / 108
页数：10
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