Donna Oeffinger

Outcome tools used for ambulatory children with cerebral palsy: responsiveness and minimum clinically important differences

This prospective longitudinal multicenter study of ambulatory children with cerebral palsy (CP) examined changes in outcome tool score over time, tool responsiveness, and used a systematic method for defining minimum clinically important differences (MCIDs). Three hundred and eighty‐one participants with CP (Gross Motor Function Classification System [GMFCS] Levels I–III; age range 4–18y, mean age 11y [SD 4y 4mo]; 265 diplegia, 116 hemiplegia; 230 males, 151 females). At baseline and follow‐up at least 1 year later, Functional Assessment Questionnaire, Gross Motor Function Measure, Pediatric Quality of Life Inventory, Pediatric Outcomes Data Collection Instrument, Pediatric Functional Independence Measure, temporal–spatial gait parameters, and oxygen cost were collected. Adjusted standardized response means determined tool responsiveness for nonsurgical (n=292) and surgical (n=87) groups at GMFCS Levels I to III. Most scores reaching medium or large effect sizes were for GMFCS Level III. Nonsurgical group change scores were used to calculate MCID thresholds for ambulatory children with CP. These values were verified by examining participants who changed GMFCS levels. Tools measuring function were responsive when a change large enough to cause a change in GMFCS level occurred. MCID thresholds assess change in study populations over time, and serve as the basis for designing prospective intervention studies.

Gross motor function classification System and outcome tools for assessing ambulatory cerebral palsy: a multicenter study

The relationships between different levels of severity of ambulatory cerebral palsy, defined by the Gross Motor Function Classification System (GMFCS), and several pediatric outcome instruments were examined. Data from the Gross Motor Function Measure (GMFM), Pediatric Orthopaedic Data Collection Instrument (PODCI), temporal-spatial gait parameters, and oxygen cost were collected from six sites. The sample size for each assessment tool ranged from 226 to 1047 participants. There were significant differences among GMFCS levels I, II, and III for many of the outcome tools assessed in this study. Strong correlations were seen between GMFCS level and each of the GMFM sections D and E scores, the PODCI measures of Transfer and Mobility, and Sports and Physical Function, Gait Velocity, and Oxygen Cost. Correlations among tools demonstrated that the GMFM sections D and E scores correlated with the largest number of other tools. Logistic regression showed GMFM section E score to be a significant predictor of GMFCS level. GMFM section E score can be used to predict GMFCS level relatively accurately (76.6%). Study data indicate that the assessed outcome tools can distinguish between children with different GMFCS levels. This study establishes justification for using the GMFCS as a classification system in clinical studies.

Outcome assessments in children with cerebral palsy, Part I: descriptive characteristics of GMFCS Levels I to III

This prospective cross‐sectional multicenter study assessed the relationships between Gross Motor Function Classification System (GMFCS) level and scores on outcome tools used in pediatric orthopedics. Five hundred and sixty‐two participants with cerebral palsy (CP; 339 males, 223 females; age range 4‐18y, mean age 11y 1mo [SD 3y 7mo]; 400 with diplegia, 162 with hemiplegia; GMFCS Levels I‐III;) completed the study. The Functional Assessment Questionnaire (FAQ), Gross Motor Function Measure (GMFM) Dimensions D and E, Pediatric Quality of Life Inventory (PedsQL), the Pediatric Outcomes Data Collection Instrument (PODCI), Pediatric Functional Independence Measure (WeeFIM), temporal‐spatial gait parameters, and O2 cost were collected during one session. Descriptive characteristics are reported by GMFCS level clinicians can use for comparison with individual children. Tools with a direct relationship between outcome scores and GMFCS levels were the PODCI Parent and Child Global Function, Transfers & Basic Mobility, and Sports and Physical Function; PODCI Parent Upper Extremity Function; WeeFIM Self‐care and Mobility; FAQ Question 1; GMFM Dimensions D and E; GMFM‐66; O2 cost; and temporal‐spatial gait parameters. Child report scores differed significantly higher than Parent scores for six of eight PODCI subscales and three of four PedsQL dimensions. Children classified into different GMFCS levels function differently.

Social and Community Participation of Children and Youth With Cerebral Palsy Is Associated With Age and Gross Motor Function Classification

Background Through social and community participation, children and youth with cerebral palsy (CP) form friendships, gain knowledge, learn skills, express creativity, and determine meaning and purpose in life. Objective The purposes of this study were: (1) to determine whether social and community participation of children and youth with CP differ based on age, sex, and gross motor function, and (2) to identify the types of activities in which social and community participation are highest. Design and Methods A prospective cross-sectional analytic design was used. The participants were a sample of convenience of 291 children (6–12 years of age) and 209 youth (13–21 years of age) with CP (55.4% males, 44.6% females) receiving services from 7 childrens hospitals. Participants completed the Childrens Assessment of Participation and Enjoyment (CAPE) by structured interview. Gross Motor Function Classification System (GMFCS) level was determined by the researchers. Results Youth did a higher percentage of activities with friends and others and outside the home than children. Children and youth in level I did a higher percentage of activities with friends and others compared with children and youth in levels II and III and in levels IV and V. Children and youth in level I and in levels IV and V did a higher percentage of activities outside the home than children and youth in levels II and III. Differences were not found between females and males. The percentage of activities done with friends and others and outside the home was highest for physical and skill-based activities. Limitations Findings cannot be attributed only to GMFCS level. Conclusions The ability to walk without restrictions is desirable for social and community participation. For children and youth with CP who have limitations in mobility, physical therapists have roles as consultants for accessibility, activity accommodations, and assistive technology and as advocates for inclusive environments.

Comparison of gait with and without shoes in children

Full body gait analysis was used to determine if differences exist in kinematic, kinetic, and temporal-spatial data with and without shoes in able-bodied children. The greatest difference noted between conditions was an increase in stride length with shoes. Minimal changes were seen in kinematics and kinetics with the addition of shoes. Due to the very tight standard deviations of the data, these minimal changes in the magnitude of the curves resulted in statistically significant differences, yet these changes do not appear to be clinically significant. It is believed that this study establishes that barefoot gait analysis is sufficient for most clinical studies, and an additional assessment undertaken while wearing shoes is not necessary.

Relationships among functional outcome measures used for assessing children with ambulatory CP

In ambulatory children with cerebral palsy (CP), practitioners often examine outcomes using measures related to functions necessary for daily life. The Gross Motor Function Measure (GMFM) Dimensions D and E, Pediatric Outcomes Data Collection Instrument (PODCI) Parent and Child versions, Gillette Functional Assessment Questionnaire (FAQ) Walking subscale, Functional Independence Measure for Children (WeeFIM), Pediatric Quality of Life Inventory (PedsQL), temporal‐spatial gait parameters, and O2 cost during ambulation were selected for study. Cross‐sectional data were collected in a prospective multicenter study of 562 participants with CP (339 males, 223 females), between 4 and 18 years of age (mean age 11y 1mo). There were 240 classified as Gross Motor Function Classification System Level I, 196 as Level II, and 126 as Level III. The tools that had the best interrelationships and underlying constructs predominately measured changes in physical function. These included portions of the FAQ, Parent PODCI, WeeFIM, and GMFM. GMFM Dimensions D and E exhibited a very strong relationship. Temporal‐spatial gait parameters and O2 cost measures represented a different construct of physical function. The Child PODCI reports and both the Parent and Child PedsQL reports did not relate well to other measures, suggesting a pattern of answers not related to question content. The Parent PODCI, the FAQ Walking subscale, and GMFM Dimension E were found to be an appropriate minimum set of instruments for assessment of functional outcomes in patients with ambulatory CP.

Comparing functional profiles of children with hemiplegic and diplegic cerebral palsy in GMFCS Levels I and II: are separate classifications needed?

The goal was to compare children with hemiplegia with those with diplegia within Gross Motor Functional Classification System (GMFCS) levels using multiple validated outcome tools. Specifically, we proposed that children with hemiplegia would have better gait and gross motor function within levels while upper extremity function would be poorer. Data were collected on 422 ambulatory children with cerebral palsy: 261 with diplegia and 161 with hemiplegia, across seven centers. Those with hemiplegia in each level performed significantly and consistently better on gait or lower extremity function and poorer on upper extremity and school function than those with diplegia. In GMFCS Level II, the group with hemiplegia walked faster (p = 0.017), scored 6.6 points higher on Dimension E of the Gross Motor Function Measure (p = 0.017), 6.7 points lower on Upper Extremity subscale of the Pediatric Outcomes Data Collection Instrument, and 9.1 points lower on WeeFIM self-care (p = 0.002). Basing motor prognosis on GMFCS level alone may underestimate lower extremity skills of children with hemiplegia, and overestimate those of children with diplegia.

Outcome assessments in children with cerebral palsy, Part II: discriminatory ability of outcome tools

Discriminatory ability of several pediatric outcome tools was assessed relative to Gross Motor Function Classification System (GMFCS) level in patients with cerebral palsy. Five hundred and sixty‐two patients (400 with diplegia, 162 with hemiplegia; 339 males, 223 females; age range 4‐18y, mean 11y 1mo [SD 3y 7mo]), classified as GMFCS Levels I to III, participated in this prospective multicenter, cross‐sectional study. All tools were completed by parents and participants when appropriate. Effect size indices (ESIs) for parametric variables and odds ratios for non‐parametric data quantified the magnitude of differences across GMFCS levels. Binary logistic regression models determined discrimination, and receiver operating characteristic curves addressed sensitivity and specificity. Between Levels I and II, the most discriminatory tools were Gross Motor Function Measure (GMFM‐66), velocity, and WeeFIM Mobility. Between Levels II and III, the most discriminatory tools were GMFM Dimension E, Pediatric Functional Independence Measure (WeeFIM) Self‐Care and Mobility, cadence, and Gillette Functional Assessment Questionnaire Question 1. Large ESIs were noted for Parent and Child reports of Pediatric Outcomes Data Collection Instrument (PODCI) Sports & Physical Function, Parent report of PODCI Global Function, GMFM Dimension E, and GMFM‐66 across all GMFCS level comparisons. The least discriminatory tools were the Quality of Life and cognition measures; however, these are important in comprehensive assessments of treatment effects.

Foot pressure and radiographic outcome measures of lateral column lengthening for pes planovalgus deformity.

The purpose of this study was to establish and characterize the relationship between foot pressure analysis and radiographic measurements in children that underwent a lateral column lengthening procedure for pes planovalgus (PPV). Eight children (13 feet) with PPV that had failed non-operative treatment underwent a lateral column lengthening procedure. Pre-operative and post-operative standing AP and lateral radiographs and foot pressure data were obtained. The relationships between the radiographic and foot pressure measurements were investigated. The findings from this study demonstrated strong relationships between these parameters. The relationships seen in this study indicate that the addition of foot pressure analysis provides objective documentation of the improvement in foot pressure distribution following a lateral column lengthening. Also, there is a direct, positive relationship between the key radiographic and foot pressure measurements.

A prospective cohort study of the effects of lower extremity orthopaedic surgery on outcome measures in ambulatory children with cerebral palsy.

Background Lower-extremity musculotendinous surgery is standard treatment for ambulatory children with deformities such as joint contractures and bony torsions resulting from cerebral palsy (CP). However, evidence of efficacy is limited to retrospective, uncontrolled studies with small sample sizes focusing on gait variables and clinical examination measures. The aim of this study was to prospectively examine whether lower-extremity musculotendinous surgery in ambulatory children with CP improves impairments and function measured by gait and clinical outcome tools beyond changes found in a concurrent matched control group. Methods Seventy-five children with spastic CP (Gross Motor Function Classification System levels I to III, age 4 to 18 y) that underwent surgery to improve gait were individually matched on the basis of sex, Gross Motor Function Classification System level, and CP subtype to a nonsurgical cohort, minimizing differences in age and Gross Motor Function Measure Dimension E. At baseline and at least 12 months after baseline or surgery, participants completed gait analysis and Gross Motor Function Measure, and parents completed outcome questionnaires. Mean changes at follow-up were compared using analysis of covariance adjusted for baseline differences. Results Surgery ranged from single-level soft tissue release to multilevel bony and/or soft tissue procedures. At follow-up, after correcting for baseline differences, Gillette Gait Index, Pediatric Outcomes Data Collection Instrument Expectations, and Pediatric Quality of Life Inventory (PedsQL) Physical Functioning improved significantly for the surgical group compared with the nonsurgical group, which showed minimal change. Conclusions On the basis of a matched concurrent data set, there was significant improvement in function after 1 year for a surgical group compared with a nonsurgical group as measured by the Gillette Gait Index, with few significant changes noted in outcome measures. Changes over 1 year are minimal in the nonsurgical group, supporting the possibility of ethically performing a randomized controlled trial using nonsurgical controls. Level of Evidence Therapeutic level 2. Prospective comparative study.