Gross motor and gait abilities of children with Hurler syndrome, pre and post umbilical cord blood transplant

Children with Hurler syndrome have significant impairments to body structure and function that result from glycosaminoglycans accumulating in cells throughout the body. Little is known about activity limitations or more specifically gross motor and gait abilities in this population. The advent of new treatments such as umbilical cord blood transplantation (UCBT) is increasing the lifespan of children with Hurler syndrome. Information on gross motor and gait abilities with and without medical interventions such as UCBT will enable the medical community, therapists, and families to help children with Hurler syndrome maximize their motor abilities. The purpose of this dissertation was to describe the gross motor and gait abilities of children with Hurler syndrome pre and post UCBT. The first study presents a case series of 4 children who had not received medical intervention to alter their enzyme levels. The second study describes changes in gross motor abilities over time for 21 children who received UCBT. The third study describes changes in selected gait parameters of 18 children with Hurler syndrome post UCBT. The combined results of these studies indicate that children with Hurler syndrome have below average gross motor abilities and significant joint range of motion impairments by 10 months of age. Gross motor abilities are most delayed in the area of locomotion prior to and after UCBT. Following UCBT, children with Hurler syndrome gain locomotor and object manipulation abilities at the same or a faster rate than typically developing children. However, they gain stationary balance abilities at a rate slower than their peers. In addition, children with Hurler syndrome post UBCT walk with age appropriate velocity and step length by 48 months of age, after having immature gait at 24 and 36 months of age. The findings from this dissertation suggest that children with Hurler syndrome do have the ability to gain new gross motor abilities and improve gait velocity and step length with increasing time post-UCBT. However, significant discrepancies in the children's abilities on various gross motor domains maybe related to orthopedic conditions, strength, and balance deficits. There findings warrant further investigation.