Wynn Robert F, Mercer Jean, Page Joanne, Carr Trevor F, Jones Simon, Wraith J Edmond
Blood and Marrow Transplant Unit, Royal Manchester Children's Hospital, Manchester, UK.
J Pediatr. 2009 Jan;154(1):135-9. doi: 10.1016/j.jpeds.2008.07.004.
We describe the use of enzyme replacement therapy in conjunction with hematopoietic stem cell transplantation in 18 consecutive patients with severe mucopolysaccharidosis type I. The survival and engraftment rate was 89% overall and 93% for the 15 patients who received full-intensity conditioning.
我们描述了18例连续的重度I型黏多糖贮积症患者联合使用酶替代疗法和造血干细胞移植的情况。总体生存率和植入率为89%，接受全强度预处理的15例患者的生存率和植入率为93%。
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