Allogeneic Hematopoietic Stem Cell Transplantation in Thirty-Four Pediatric Cases of Mucopolysaccharidosis—A Ten-Year Report from the China Children Transplant Group
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- 作者：Jianmin Wang1 ; &dagger ; ; Zuo Luan2 ; &dagger ; ; Hua Jiang3 ; &dagger ; ; Jianpei Fang4 ; Maoquan Qin5 ; Vincent Lee6 ; Jing Chen1 ; chenjing@scmc.com.cn" class="auth_mail" title="E-mail the corresponding author
- 关键词：Mucopolysaccharidosis ; Allogeneic hematopoietic stem cell transplantation ; Children ; Multicenter
- 刊名：Biology of Blood and Marrow Transplantation
- 出版年：2016
- 出版时间：November 2016
- 年：2016
- 卷：22
- 期：11
- 页码：2104-2108
- 全文大小：145 K
文摘
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We reported multicenter results of allogeneic hematopoietic stem cell transplantation for mucopolysaccharidosis throughout China
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This study included not only mucopolysaccharidosis IH but also other types that were reported rarely
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Overall survival, full donor chimerism, and normal enzyme levels were high and transplantation-related mortality was low
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Hematopoietic stem cell transplantation was helpful to improve multisystem symptoms of mucopolysaccharidosis