Orthopedic Outcomes Measures for NF Betty Schorry, M.D. Cincinnati Children’s Hospital

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

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

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.

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

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

Gait analysis set-up

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.

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

Timed 25 Foot Walk (T25FW) Endpoints 20% increase/decrease as clinically significant change % of subjects with faster walking speed during intervention over baseline

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).