Bone Marrow Transplantation in Children with Hunter Syndrome: Outcome after 7 to 17 Years

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)