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Abstract

BACKGROUND:

X-linked adrenoleukodystrophy is the hot research in the field of genetics at home and abroad and there still lacks of the effective treatment method.

OBJECTIVE:

To analyze the efficacy and complications of allogeneic hematopoietic stem cell transplantation on the treatment of X-linked adrenoleukodystrophy in two cases.

METHODS:

Two cases of X-linked adrenoleukodystrophy treated with allogeneic hematopoietic stem cell transplantation were selected from Tongji Hospital, Tongji Medical College of Huazhong University of Science and Technology for comprehensive analysis and genetic testing. The success of the transplantation and the occurrence of complications after transplantation were observed.

RESULTS AND CONCLUSION:

Both of the two cases were childhood cerebral X-linked adrenoleukodystrophy. One case had successful allogeneic hematopoietic stem cell transplantation but with serious transplant-related complications, and the other one case was failed. Allogeneic hematopoietic stem cell transplantation is an effective method for treatment of early childhood cerebral X-linked adrenoleukodystrophy, but the ultimate effect was related with the success of transplantation and post-transplant complications and other factors, and the efficacy of allogeneic hematopoietic stem cell transplantation still needs long-term investigation.

RESULTS AND CONCLUSION:

Zhang J, Ying YQ, Luo XP, Zhang YC, Xiao Y. Allogeneic hematopoietic stem cell transplantation for the treatment of X-linked adrenoleukodystrophy.Zhongguo Zuzhi Gongcheng Yanjiu. 2013;17(10):1868-1875.

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