Motor Proficiency of People With Bleeding Disorders Using the BOT-2 (TM)
Condition: Hemophilia
MeSH terms: Hemostatic Disorders; Hemophilia A; Blood Coagulation Disorders
Study type: Observational [Patient Registry]

Summary: 
      The purpose of this study is to find out if gross motor skills of children and young adults
      with bleeding disorders are different from those without bleeding disorders. The
      investigators will use the standardized motor test the Bruininks-Oseretsky Test of Motor
      Proficiency, Second Edition (BOT-2 tm). The second purpose is to establish if history of
      joint disease secondary to bleeding affects gross motor skills.

        1. Participants and setting: Subjects will include up to 100 male youth and young adults,
           ages 4 to 21, recruited from the patient population of the Hemophilia Treatment Center
           at OHSU. A mailing that describes the study may be sent to all potential subjects who
           receive care through the Hemophilia Treatment Center at OHSU. Data collection will occur
           at either clinic visits or in the home. Information, including history of change in
           joint structure and synovium, from existing bleeding disorder repositories at OHSU will
           also be used.

        2. Design and Procedures: Research will be done using a prospective, cross-sectional study
           design to examine any relationship between a diagnosis of bleeding disorder and gross
           motor development. The gross motor ability of children and young adults with bleeding
           disorders who meet the inclusion criteria will be compared to sex-specific normative
           data from a standardized motor test. Information about age, type of hemophilia, presence
           of an inhibitor, type of management used, body composition, range of motion, and hand
           strength will also be collected through direct measurement and chart review. The
           information collected will be entered into a repository. A subject may choose to opt out
           of the repository while still participating in the research study. In addition,
           information from a current hemophilia repository will be compared to gross motor ability
           scores to determine if joint disease is related to skill level in this group of people.

        3. Proposed analyses: Two-tailed t tests and logistic regression will be used to determine
           if there are any significant differences.
    

Detailed description: 
      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.

      Recruitment Methods Potential subjects will be recruited during any clinic visit to the
      Hemophilia Treatment Center at OHSU. An IRB-approved recruitment letter may also be mailed to
      the Hemophilia Treatment Center's mailing list to recruit potential subjects.

      Consent Process Informed, face-to-face consent will be sought prior to the collection of any
      data. Following a detailed explanation of the components of the study, the parent or legal
      guardian of the subject, if the subject is under 17, or the subject themselves may sign the
      informed consent. Assent will be obtained from patients aged 7-17. All children eligible for
      participation in the study and under the age of 17 will be assessed for their understanding
      prior to signing the assent form. Should the subject or their parents be non-English
      speaking, an in-person interpreter will be present for the entire consenting process to
      ensure understanding.

      Study Procedures Data collection will take place in one clinic visit or one home visit by
      OHSU personnel. An interpreter will be scheduled should the family require an interpreter
      during a home visit or clinic visit. If data collection is not completed in one visit, a
      return visit, either to the clinic or to the home, will be conducted.

      Clinical Data Current clinical data will be collected from the subject's medical record.
      Clinical data includes body composition recorded as Body Mass Index (BMI), type of bleeding
      disorder, severity, presence of inhibitor, method of management, and age. If a current height
      and weight is not available in the chart and the data collection occurs in the subject's
      home, appropriate devices for measurement of height and weight will be supplied to the
      investigators for this visit. This data will be transcribed onto the data collection sheet
      for each subject. Collected data will then be deposited into the data collection spreadsheet.

      In addition, existing hemophilia repositories IRB # e5102 & IRB # e7074 will be accessed for
      each subject in the study to collect the following data: clinical joint health, as documented
      using the Hemophilia Joint Health Score (HJHS), and presence of chronic synovitis. Collected
      data will be deposited into the data collection spreadsheet.

      Range of Motion During the subject's clinic or home visit, assessments of range of motion of
      hip flexion and extension, knee flexion and extension, ankle dorsiflexion and plantarflexion,
      elbow flexion and extension, forearm pronation and supination, and shoulder flexion will be
      taken using a goniometer and musculoskeletal landmarks according to standard clinical
      practice at the Hemophilia Treatment Center at OHSU.

      Strength Subjects grip strength will be measured with hand held dynamometry. The Jamar
      Hydraulic Hand Dynamometer will be used to assess the bilateral grip strength in each
      subject, using the protocol set forth in the Jamar Owner's Manual.

      Gross Motor Proficiency Gross motor proficiency will be assessed utilizing the following
      subtests of the Bruininks-Oseretsky Test of Motor Proficiency, Second Edition (BOT-2™):
      Upper-Limb Coordination, Bilateral Coordination, Balance, Running Speed, and Agility and
      Strength. Prior to administration of the subtests, the researcher will determine the
      subject's hand and foot preferences for writing, throwing, and kicking, to be used within
      each subtest that requires use of a preferred extremity. Testing environment and test
      administration will conform to the protocol set out in the BOT-2™ Administration Manual.
    

Observational model: Case-Control
Time perspective: Cross-Sectional
Sampling method: Non-Probability Sample
Study population: 
        Children and young adults with hemophilia A or hemophilia B, between the ages of 4 years, 0
        months to 21 years, 11 months.
      
Keywords: hemophilia; joint health; motor development; motor proficiency