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The purpose of this article is threefold: (1) to describe the development, reliability, and validity of a revised physical performance measure for individuals with mucopolysaccharidosis type I (MPS I); (2) to standardize the test on a normal sample; and (3) to compare results from a selected sample of individuals with MPS I with age-based centiles. The MPS Physical Performance Measure (MPS-PPM) is composed of eight timed functional tasks (FT-8) and two endurance tasks with a modified Energy Expenditure Index for comfortable walking (CW) and fast walking (FW) speeds. Age norms were derived from a convenience sample of 150 typically developing children and adolescents (75 males, 75 females; mean age 11y 2mo [SD 4y 5mo] range 5–22y). Using a Rasch model for speed tests and confirmatory factor analysis, we established the unidimensionality of the FT-8. Interrater reliability of the FT-8 (intraclass correlation [ICC]=0.98) and test–retest reliability of the FT-8 (ICC=0.96), CW (ICC=0.91), and FW (ICC=0.83) were good. Results of the age-based profiles in 10 individuals with MPS I (five males, five females; mean age 14y 2mo [SD 7y 6mo] range 6–29y) indicate that the amount of time needed to perform functional tasks is severely affected by the disease, and most individuals were at or below the fifth centile for their age. The patterns of limitations in endurance were more varied. These results suggest the utility of using this revised MPS-PPM to identify the extent of limitation in age-expected physical performance. Implications for using the MPS-PPM for monitoring physical performance changes during clinical interventions are discussed. [ABSTRACT FROM AUTHOR]

The purpose of this article is to demonstrate: (1) the accuracy and (2) the reduction in amount of time and effort in assessing physical functioning (self-care and mobility domains) of children and adolescents using computer-adaptive testing (CAT). A CAT algorithm selects questions directly tailored to the child's ability level, based on previous responses. Using a CAT algorithm, a simulation study was used to determine the number of items necessary to approximate the score of a full-length assessment. We built simulated CAT (5-, 10-, 15-, and 20-item versions) for self-care and mobility domains and tested their accuracy in a normative sample (n=373; 190 males, 183 females; mean age 6y 11mo [SD 4y 2m], range 4mo to 14y 11mo) and a sample of children and adolescents with Pompe disease (n=26; 21 males, 5 females; mean age 6y 1mo [SD 3y 10mo], range 5mo to 14y 10mo). Results indicated that comparable score estimates (based on computer simulations) to the full-length tests can be achieved in a 20-item CAT version for all age ranges and for normative and clinical samples. No more than 13 to 16% of the items in the full-length tests were needed for any one administration. These results support further consideration of using CAT programs for accurate and efficient clinical assessments of physical functioning. [ABSTRACT FROM AUTHOR]

A consecutive series of 106 children and adolescents (mean age 10 years, 6 months; SD 4 years, 8 months) with recent traumatic brain injury admitted to a regional hospital-based rehabilitation program was assessed to determine the rate of walking recovery, and characteristics that distinguish between independent walkers, non-walkers, and device-assisted walkers at hospital discharge. Data were collected through a retrospective medical record review of patients admitted between 1994 and 2001. Mean hospital stays were 66.7 days (SD 88.5, range 7 to 140 days). All children (72 male, 34 female) had recent injuries (from 1 to 8 weeks after onset of traumatic brain injury) and were independent walkers before injury. Sixty-four children (60.4%) were discharged as independent walkers, 13 (12.3%) walked with the assistance of a device, and 29 (27.3%) were non-walkers. Non-walkers had a higher proportion of prolonged loss of consciousness, lower-extremity injury, impaired responsiveness, and lower-extremity spasticity than independent walkers. In addition, non-walkers had poorer discharge mobility and social function scores, longer average hospital stays, and a greater proportion of non-community discharges. Device-only walkers were older, more likely to be male, and had a higher proportion of lower-extremity injuries than independent walkers. Results highlight several demographic, clinical, and outcome variables that distinguish independent walkers from device-assisted walkers and non-walkers. These variables might help to determine the prognosis for ambulation, resource needs, and discharge plans for children and adolescents with traumatic brain injury after episodes of inpatient rehabilitation. [ABSTRACT FROM AUTHOR]

This study describes the physical disability of 30 children and adolescents with Pompe disease (23 males, 7 females; mean age 7 years 7 months, SD 5 years 6 months; range 6 months to 22 years 1 month) using a disease-specific functional instrument. Data were collected by telephone interview with parents using a modified version of the Pediatric Evaluation of Disability Inventory. The sample included mostly males of Caucasian origin, recruited from several countries. Disability profiles in mobility and self-care skills were heterogeneous because functional status was not related to chronological age. Most children had severe functional deficits: nearly two-thirds of the sample was non-ambulatory and could not perform age-expected self-care skills. Three-quarters of the children used a ventilator. Two children were able to participate in age-appropriate sports and peer activities. Although the mean chronological age of the sample was 7 years 7 months, the mean age-performance for self-care skills was under 2 years 6 months and under 1 year 6 months for mobility. Implications of physical disability findings for individuals with Pompe disease are discussed. [ABSTRACT FROM AUTHOR]