Abstract

At follow up, 4 patients were either wheelchair bound or required a walking frame to mobilize in the community. At follow up, all hips were in-joint. All hips displayed characteristic medial flattening of the femoral head. Ten hips (of 26 hips) showed radiological degenerative changes with loss of joint space < 2mm. Conclusion(s): This is the first study to report both longterm functional and radiological outcomes in this population. Despite the surgical provision of stable well-covered hips, active intervention did not prevent the development of radiological deterioration and clinically significant hip arthritis. The authors recommend that pediatric hip surgery in Hurler syndrome is designedwith the possibility of early hip replacement in mind. Implications: The lifespan and long-term functional requirements of children with MPS-1 are unknown. HSCT has revolutionized the care of children with MPS-1. However HSCT has presented health professionals treating these patients with a challenge, in that the natural history of the musculoskeletal aspects of this condition post HSCT is entirely unknown. The authors have found that achieving good radiological correction of hip dysplasia in patients did not prevent these patients from developing clinically significant arthritis in young adulthood. Recommendations are that the outcomes of all children internationally (both operatively and non-operatively treated) be pooled to facilitate a greater understanding of both the natural history and results of interventions in this rare metabolic disorder.

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