Bone Marrow Transplantation in Children with Hunter Syndrome: Outcome after 7 to 17 Years
Bone Marrow Transplantation in Children with Hunter Syndrome: Outcome after 7 to 17 Years
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DOI：
10.1016/j.jpeds.2008.11.041
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发表时间：
2009-05-01
影响因子：
5.1
通讯作者：
Froissart, Roseline
中科院分区：
文献类型：
--
作者：
Guffon, Nathalie;Bertrand, Yves;Froissart, Roseline
目的探讨骨髓移植治疗Hunter综合征的疗效，研究设计1990年至2000年，8例3 ~ 16岁男孩接受骨髓移植。6例供者为HLA相合同胞，1例为HLA相合非血缘供者，1例为不相合非血缘供者。一个完整的多学科评估每年进行year.Results移植成功，在所有患者中实现，与供体细胞移植后1个月的比例达到>= 95%，在所有患者。患者随访7 - 1.7年，除1名男孩在10岁时死于不相关原因外，其余均存活。所有患者的心血管异常均稳定。肝脾肿大消退，关节僵硬改善，感知听力缺陷保持稳定，传输听力缺陷改善。只有1名儿童需要后续手术来矫正脊柱后凸。结论骨髓移植对无神经心理症状的Hunter病患者是有效的。（J Pediatr 2009;154：733-7）
Objective To evaluate the effect of bone marrow transplantation in children with Hunter syndrome.Study design Eight boys received it bone marrow graft between the ages of 3 and 16 years front 1990 to 2000. In 6 cases, the donor was a sibling with identical HLA status, in 1 case art unrelated donor with HLA-compatible, and in 1 case a mismatched unrelated donor. A complete multidisciplinary evaluation was performed yearly.Results Successful engraftment was achieved in all patients, with the proportion of donor cells reaching >= 95% 1 month after transplantation in all patients. Patients have been followed front between 7 and 1.7 years and all are still alive, except for 1 boy who died at the age of 10 front unrelated causes. Cardiovascular abnormalities stabilized in all patients. hepatosplenomegaly resolved, and joint stiffness improved, Perceptual hearing defects remained stable, and transmission hearing defects improved. Only 1 child required subsequent surgery to correct kyphosis. Neuropsyhological outcome was variable and appeared to be related to the severity of the syndrome.Conclusions Bone marrow transplantation is effective on the no neuropsychological symptoms of Hunter disease. (J Pediatr 2009;154:733-7)