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Orthopedic Outcomes Measures for NF

Betty Schorry, M.D.Cincinnati Children’s Hospital

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PODCI

• Pediatric Outcomes Data Collection Instrument– Developed by POSNA (Pediatric orthopaedic society of

North America) in 1994– Measure of functional health in children and adolescents

with musculoskeletal conditions– Validated on group of 530 pediatric patients and parents– All scales with good – exc. internal reliability (>0.80)– Good-exc. test-retest reliability– Published extensively

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PODCI – cont.

• Pediatric questionnaire (ages 2-10 years)• Adolescent (self-reported) questionnaire (ages

11-18)• Parent-reported Adolescent questionnaire• Total of 83 – 86 questions– 4 or 5 point Likert scale– Takes 15-20 min. to complete

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Scales of PODCI

• 1. Upper extremity physical function• 2. Transfer and basic mobility scale• 3. Sports and physical functioning scale• 4. Pain/ comfort scale• 5. Happiness core scale• 6. Global functioning scale – Higher score indicates higher functioning.

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Advantages of PODCI

• Extensive questionnaire• Valuable for studies where primary

assessment involves musculoskeletal system– Tibial dysplasia; scoliosis; paraspinal tumors;

motor coordination

• DISADVANTAGES of PODCI– Length of questionnaire– Not available for adults

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Functional Orthopaedic Measures for NF Studies

• Clinical Gait Analysis– Used in some rehabilitation programs; athletes– Uses computerized system with multiple cameras– Marks are placed on different joints– Can calculate kinematic data– Beginning to be used in research

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Gait analysis set-up

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Data available

• Kinematics – 3-dimensional movement of joints

• Kinetics - study of forces involved in walking• Can add dynamic EMG data• One study of gait analysis in 12 children with

repaired tibial pseudarthrosis (1998)• Could have potential future use in clinical

trials of musculoskeletal complications of NF.

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Timed 25 Foot Walk (T25FW)

• Time taken to walk 25 feet• Represented as time (sec) or velocity

(feet/sec)• No learning effect• Used in studies of multiple sclerosis, spinal

cord injury, Friedrich’s ataxia

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Timed 25 Foot Walk (T25FW)

• Endpoints– 20% increase/decrease as clinically significant

change– % of subjects with faster walking speed during

intervention over baseline

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Walking (from approval of ampyra)

• Inclusion criteria included the ability to walk 25 feet in 8–45 seconds

• The primary measure of efficacy in both trials was walking speed (in feet per second) as measured by the Timed 25-foot Walk (T25W), using a responder analysis.

• A responder was defined as a patient who showed faster walking speed for a least three visits out of a possible four during the double-blind period than the maximum value achieved in the five non-double-blind no treatment visits (four before the double-blind period and one after).