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

2. 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

3. 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 min. to complete

4. 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.

5. 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

6. 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

8. Gait analysis set-up

10. 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.

11. 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

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

13. 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).