Assessment of Motor Proficiency in People With Bleeding Disorders Using the Bruininks-Oseretsky Test of Motor Proficiency, Second Edition (BOT-2™)

The primary purpose of this study is to determine whether gross motor proficiency of children and young adults with hemophilia falls below average, sex-specific normative data, using the standardized motor assessment the Bruininks-Oseretsky Test of Motor Proficiency, Second Edition (BOT-2™). The primary hypothesis is that children and young adults with hemophilia perform at a lower level of gross motor proficiency compared to same-aged peers, despite advances in care.

The secondary purpose is to establish whether there is a relationship between joint health and level of gross motor proficiency. The secondary hypothesis is that history of hemophilia-caused ankle, knee, or elbow arthropathy will result in significant differences in gross motor proficiency within and between the populations of children and young adults with hemophilia.

Background/justification/rationale With the advent of widely-used prophylactic treatment for people with bleeding disorders, activity-limiting joint disease may be significantly reduced. Research investigating joint health and aerobic capacity of boys with hemophilia, an X-linked bleeding disorder, have shown no significantly different levels of functional ability when compared to peers, though other parameters may continue to be lower. However, a recent unpublished report of preliminary data using the Bruinicks-Oseretsky Test of Motor Proficiency (BOT-2 tm), a standardized gross motor assessment tool, suggests that male children and young adults with bleeding disorders function at a significantly lower level of gross motor skills compared to normative, sex-specific data, established when standardizing the BOT-2. This question is important in the determination of prognosis and intervention in this population.

Study Design Research will be conducted utilizing a prospective, cross-sectional study design to analyze the relationship between a diagnosis of bleeding disorder and gross motor development in male children and young adults as well as the association between joint health and gross motor development. As diagnosis of hemophilia is extremely rare in women and statistical analysis requires use of sex-specific normative data, this research study will focus strictly on the population of boys and young men with a diagnosis of hemophilia.

Once informed consent and assent (if applicable) is obtained, data will be collected by OHSU research personnel during a regular clinic visit at the Hemophilia Treatment Center (HTC). If a subject is unable to visit the clinic, informed consent/assent and study visit may be performed at the subject's home.