Orthopedic Outcomes Measures for NF Betty Schorry, M.D. Cincinnati Children’s Hospital 1 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 2 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 3 Scales of PODCI • • • • • • 1. 2. 3. 4. 5. 6. Upper extremity physical function Transfer and basic mobility scale Sports and physical functioning scale Pain/ comfort scale Happiness core scale Global functioning scale – Higher score indicates higher functioning. 4 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 5 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 6 7 Gait analysis set-up 8 9 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. 10 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 11 Timed 25 Foot Walk (T25FW) • Endpoints – 20% increase/decrease as clinically significant change – % of subjects with faster walking speed during intervention over baseline 12 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). 13