The Effect of Knee Height Asymmetry on Gait Biomechanics.

BACKGROUND: Though the primary goal for limb length discrepancy (LLD) management is to equalize the leg lengths, symmetry between corresponding long bones is usually not achieved, leading to knee height asymmetry (KHA). To date, there is minimal information on what effect KHA has on gait biomechanics and joint loading. Thus, the purpose of this study is to determine the impact of KHA on gait biomechanics. METHODS: Seventeen subjects with KHA after limb equalizing surgery and 10 healthy controls were enrolled. Subjects participated in 3D gait analysis collected using self-selected speed. Lower extremity kinematics, kinetics, work generated/absorbed, and total work were calculated. Standing lower limb x-rays and scanograms were used to measure LLD and calculate the tibia-to-femur (TF) ratio for each limb. Two sample t tests were used to compare differences in standing LLD, TF ratio, and work between groups. Bivariate correlation using Pearson correlation coefficients was conducted between TF ratio and total mechanical work, as well as between knee height asymmetry indices and total work asymmetry (α=0.05). RESULTS: Among participants, there were no differences between LLD; however, there were differences between TF ratio and knee height asymmetry. We found a nonsignificant relationship between TF ratio and total mechanical work for individual lower extremities. Therefore, the length of individual bones (TF ratio) relative to each other within the individual lower extremity was not associated with the amount of work produced. However, when a difference exists between sides (asymmetry, ie, TF ratio asymmetry), there were associated differences in work (work asymmetry) produced between sides (r=0.54, P =0.003). In other words, greater knee height asymmetry between limbs resulted in more asymmetrical mechanical work during walking. CONCLUSIONS: These findings may have implications for the management of LLD. Asymmetrical total mechanical work could lead to atypical joint loading during gait. Surgeons may want to consider prioritizing achieving knee height symmetry as a postoperative goal when correcting limb length discrepancy. LEVEL OF EVIDENCE: Level III, Case Control Study.

Kinematic changes in gait in boys with Duchenne Muscular Dystrophy: Utility of the Gait Deviation Index, the Gait Profile Score and the Gait Variable Scores.

BACKGROUND: Duchenne Muscular Dystrophy (DMD) is an X-linked muscle disorder caused by a mutation or deletion in the dystrophin gene. In boys with DMD, muscle weakness progresses in a proximal to distal pattern, leading to gait abnormalities at all joints, in all planes of motion. Longitudinal studies are imperative to quantify changes in gait function due to DMD and are of particular importance when examining the efficacy of treatment interventions. RESEARCH QUESTION: The purpose of this study was to examine the sensitivity of the Gait Deviation Index (GDI) and Movement Analysis Profile (Gait Profile Score (GPS) and Gait Variable Score (GVS)) to quantify the longitudinal ambulatory decline in boys with DMD. A secondary aim was to quantify the effect of corticosteroid (CS) treatment. METHODS: The gait patterns of 75 boys were assessed longitudinally, 11 were steroid naïve (SN), and 64 received CS treatment. Joint kinematics were collected using either a VICON 612 or a Motion Analysis Corporation 3-D system. Representative trials were used to compute the GDI, GPS and the nine GVS for each boy for each visit. RESULTS: At baseline, GVS for the boys with DMD revealed abnormalities in all lower extremity joints and in all planes of movement compared to TD peers. GDI and GPS indices verified that the overall quality of gait in boys with DMD decreases at a significant rate with age. Boys who were steroid naïve changed at a rate 3 times greater than boys on CS in coronal plane hip motion. SIGNIFICANCE: The gait indices of GDI and GPS are able to identify changes in the quality of gait patterns in boys with DMD. Although boys on steroids had greater gait deviations than boys who were SN at baseline, the rate of decline in gait quality was slower in boys on CS.

Surgical outcomes for spinal deformity in osteogenesis imperfecta.

STUDY DESIGN: IRB approved retrospective review of the the SHOnet (Shriners Health Outcomes Network). OBJECTIVES: Osteogenesis imperfecta (OI) is the most prevalent genetic disorder of bone and connective tissue in the pediatric population, with an incidence of 1/15,000-20,000. Scoliosis has been reported to be present in 39-100% of OI patients and may continue to progress into adulthood but there is little information on the perioperative outcomes, rates of revision surgery and complications in this fragile population. The purpose of this study is to examine the prevalence of scoliosis in the OI population across a tertiary care multihospital system. The rate at which these patients progress to surgery, the perioperative complication rate and if there is an association between complications and age at surgical intervention, pre-operative Cobb angle, number of fractures, and type of OI. Incidence of scoliosis in OI may be lower than previously reported, many may never require surgical intervention and those that do require surgical intervention have an 8% reoperation rate at an average of 3.3 years postop. METHODS: IRB approved retrospective review of the the SHOnet (Shriners Health Outcomes Network), electronic health record data warehouse from January 1, 2011 and December 31, 2017. Inclusion criteria included International Classification of Diseases (ICD) code for osteogenesis imperfecta and scoliosis. The data warehouse was queried for age, gender, presence of vertebral body fractures, proximal junctional kyphosis, basilar invagination, bisphosphonate use, and perioperative complications including postoperative infection. RESULTS: There were 2372 patients with osteogenesis imperfecta, 429 or 18.1% also had a diagnosis of scoliosis, while 81.9% did not (see Table 1). Only 74 patients (17.2%) of the patients that had scoliosis underwent spine surgery, 12 of which had staged surgery with an average preop thoracic Cobb 58.18 (range 7-115), and thoracolumbar Cobb 59.83 (range 5-145). Six patients (8%) required revision spine surgery. Average time to revision was 3.88 years (6.9-69mo). Bisphosphonate use was present in 35.5% of patients that did not require surgery and in 40.5% that did. CONCLUSION: With over 2300 patients, this is the largest study to date on scoliosis in patients with OI. We found that contrary to prior studies which had indicated the incidence of scoliosis ranges from 39 to 100% that it was only present in 18% of our patients. This indicates that the incidence of scoliosis in OI is potentially lower than previously reported, though due to the nature of this study it may be underreported. Furthermore, only 17% of those patients in our study with scoliosis eventually underwent surgery. It is important to note that many patients treated non-operatively had deformity within the operative range, which may indicate that they were not considered good candidates for surgical intervention. Lastly, we found that bisphosphonate use did not appear to affect the likelihood of progression to surgery in this group of patients, though this may be due to later initiation of the bisphosphonate use.

The incidence of avascular necrosis in children with cerebral palsy after hip containment surgery.

Purpose: To establish the rate of avascular necrosis after hip reconstruction surgery in children with cerebral palsy and to identify risk factors that influence the development of avascular necrosis in this population. Methods: An institutional review board-approved retrospective review was conducted on children with cerebral palsy who underwent hip containment surgery at a single institution. Radiographs were evaluated at three time points. The Reimer's migration percentage, neck shaft angle, epiphyseal shaft angle, acetabular index, center edge angle, and acetabular angle were measured. The presence of avascular necrosis was evaluated and graded by the Bucholz/Ogden and the Kalamchi/MacEwen classification systems. Multivariate logistic regression was performed to identify risk factors associated with the development of avascular necrosis. Results: A total of 154 children with cerebral palsy underwent hip containment surgery on 223 hips. Twenty-nine children (18.8%) underwent both pelvic and femoral procedures; 36 children (23.4%) had only femoral procedures; 47 children (30.5%) had femoral and soft tissue; and 42 children (27.3%) had pelvic, femoral, and soft tissue procedures. Using the Bucholz and Ogden or the Kalamchi classifications, the rate of avascular necrosis was 24.7% (38/154). Of the variables evaluated, preoperative Reimers was found to be significant predictors of avascular necrosis. The rate of avascular necrosis was 26.7% for Gross Motor Functional Classification System level III, 24.1% for Gross Motor Functional Classification System level IV, and 27.3% for Gross Motor Functional Classification System level V. Conclusion: The overall rate of avascular necrosis in children undergoing hip containment surgery was 26.7%. Together, age at surgery, open reduction, previous surgery, preoperative Reimers, and estimated blood loss contributed to the development of postoperative avascular necrosis; however, only preoperative Reimers significantly contributed to the development of avascular necrosis in children with cerebral palsy undergoing hip containment procedures.

Idiopathic Toe Walking: An Update on Natural History, Diagnosis, and Treatment.

Toe walking is a common presenting report to an orthopaedic practice. Evaluation of a child with toe walking includes a thorough history and physical examination to elucidate the diagnosis. When no other diagnosis is suspected, a diagnosis of idiopathic toe walking is often given. Despite the high prevalence of the condition, there is notable controversy of the nomenclature of the disease. Recent research has shed more light on both the natural history and the genetic basis. The use of motion analysis, including EMG and multisegment foot model, may help both differentiate children with mild forms of cerebral palsy and evaluate outcomes after treatment. Early treatment for young children with adequate range of motion (ROM) is generally reassurance because most young children with idiopathic toe walking are expected to resolve spontaneously. When the toe walking persists, treatment options include both surgical and nonsurgical management. Nonsurgical management centers on obtaining ROM through stretching and serial casting, followed by gait retraining and bracing treatment. Surgical lengthening at either the Achilles or gastrocnemius level improves the ROM of the ankle and then similarly requires therapy and bracing treatment to obtain a more normalized gait.

Patient-Reported Outcome Measurement Information System (PROMIS) Scores in Pediatric Idiopathic Toe Walkers.

BACKGROUND: Idiopathic toe walking (ITW) can result in early contact with the health care system and be distressing for patients and their families. The natural history of ITW is poorly characterized. Deciding how and when to intervene can be difficult.Patient-reported outcomes are utilized in the clinical setting to assess patient factors and indications that may better inform treatment plans. Patient-Reported Outcomes Measurement Information System (PROMIS) is an instrument designed to collect patient-reported outcomes. Minimum clinically important differences in PROMIS metrics have been established to facilitate clinical relevance and utility of these metrics. The purpose of this study was to characterize the patient perspective of ITW by utilizing the PROMIS scores. METHODS: Retrospective chart review was performed to identify children aged 5 to 17 with a diagnosis of ITW treated at a single tertiary care center between 2017 and 2020. Inclusion criteria were a diagnosis of ITW and completion of a PROMIS questionnaire. Exclusion criteria were neurologic disease, autism, and previous surgical treatment. Demographic, physical exam, treatment, and available motion analysis data were collected. PROMIS scores for the following domains were available: Mobility, Peer Relationships, and Pain Interference. RESULTS: Forty-five children were enrolled. Seventy-three percent of PROMIS scores were patient reported while the remainder were parent reported. PROMIS score means for the cohort by domain were Mobility: 45.2±8.2 ( P <0.000); Peer Relationships: 46.4±11.6 ( P =0.047); and Pain Interference: 47.4±9.5 ( P =0.67). Motion analysis data, available for 11 children, noted age-matched gait velocity negatively correlated ( rs =-0.652, P =0.03) with Peer Relationships. No correlations were found between other aspects of gait, body mass index, or limitations in dorsiflexion and PROMIS domains. Parents reported lower Mobility scores than children did. There were no other significant differences between patient-reported and parent-reported PROMIS scores. PROMIS scores did not differ significantly between those <10 years and those ≥10 years. CONCLUSION: In this cohort of 45 otherwise healthy children without other neurologic diagnoses, there are both clinically and statistically significant differences in PROMIS means between our cohort and the healthy age-matched population. These differences manifested in worse peer relationships and mobility scores. LEVEL OF EVIDENCE: Level IV.

The SHOnet learning health system: Infrastructure for continuous learning in pediatric rehabilitation.

Introduction: To describe the development and implementation of learning health system (LHS) infrastructure for a pediatric specialty care health system to support LHS research in pediatric rehabilitation settings. Methods: An existing pediatric common data model (eg, PEDSnet) of standardized medical terminologies for research was expanded and leveraged for this stud, and applied to SHOnet, a clinical research data resource consisting of deidentified data extracted from the electronic health record (EHR) from the Shriners Hospitals for Children speacialty pediatric health care system. We mapped EHR data for laboratory, procedures, drugs, and conditions to standardized vocabularies including ICD-10, CPT, RxNorm, and LOINC to the common data model using an established extraction-transformation-loading process. Rigorous quality checks were conducted to ensure a high degree of data conformance, completeness, and plausibility. SHOnet data elements from all sources are de-identified and the server is managed by the SHC Information Systems Department. SHOnet data are refreshed monthly and data elements are continually expanded based on new research endeavors. Interventions: Not applicable. Results: The Shriners Health Outcomes Network (SHOnet) includes data for over 10 000 distinct observational data elements based on over two million patient encounters between 2011 and present. Conclusion: The systematic process to develop SHOnet is replicable and flexible for other pediatric rehabilitation research settings interested in building out their LHS capabilities. Challenges and facilitators may arise for building such LHS infrastructure for rehabilitation in areas of (a) data capture, curation, query, and governance, (b) generating knowledge from data, and (c) dissemination and implementation of new institutional knowledge. Further research studies are needed to evaluate these data resources for scalable system-learning endeavors.SHOnet is an exemplar of an LHS for rehabilitation and specialty care settings. The success of an LHS is dependent on engagement of multiple stakeholders, shared governance, effective knowledge translation, and deep commitment to long-term strategies for engaging clinicians, administration, and families in leveraging knowledge to improve clinical outcomes.

The Effects of Lower Extremity Rotational Malalignment on Pediatric Patient-reported Outcomes Measurement and Information System (PROMIS) Scores.

BACKGROUND: There is sparse literature demonstrating the relationship between lower limb pediatric idiopathic rotational malalignment (IRM) and patient-reported outcomes measurement information system (PROMIS) scores. Our goal is to determine and quantify the amount that IRM deformities, as measured with the 3D gait analysis, affect childrens' pain interference, mobility, and peer relationship PROMIS domains. Secondary outcomes include investigating the potential relationships between IRM and various subgroups (Pediatric Outcomes Data Collection Instrument (PODCI), gender, Body Mass Index (BMI), femur Versus tibia). We also examine whether the PROMIS domains correlate with PODCI in this population. METHODS: This study was a retrospective cohort, single institution, and consecutively recruited cases series. We identified 47 children over a 3-year period who were evaluated at the motion analysis center at our tertiary care hospital, with increased torsion of the femur or tibia. After exclusions, 25 children with IRM, documented PROMIS data and gait analysis were considered. RESULTS: Femoral malrotation had a significant relationship with female gender ( P =0.001) and increased BMI ( P <0.001). Femoral malrotation had a correlation with PROMIS pain interference ( P =0.016), whereas tibial rotation did not achieve significance ( P =0.084). In the ANOVA regression analysis, there was a strong prediction of the PROMIS mobility domain when both malrotation and pain interference were present ( P =0.007). There were Pearson Correlations of PROMIS and PODCI domains for Mobility Versus Sports ( P =0.007) and Pain Interference Versus Comfort/Pain ( P =0.004), respectively. CONCLUSION: The evident relationship between lower limb rotational malalignment and PROMIS scores signifies the likelihood for gait and pain disturbance. This in turn could show us that children are likely to be more debilitated and thus may benefit from timely correction. We aim to conduct this as a multicentre trial to confirm our findings. LEVEL OF EVIDENCE: Level IV.

A Longitudinal Study of Quantitative Muscle Strength and Functional Motor Ability in Ambulatory Boys with Duchenne Muscular Dystrophy.

BACKGROUND: Duchenne muscular dystrophy (DMD) is an X-linked recessive genetic disorder, that is characterized by progressive muscle degeneration and loss of ambulation between 7-13 years of age. Novel pharmacological agents targeting the genetic defects and disease mechanisms are becoming available; however, corticosteroid (CS) therapy remains the standard of care. OBJECTIVE: The purpose of this longitudinal study was to elucidate the effect of CS therapy on the rate of muscle strength and gross motor skill decline in boys with DMD and assess the sensitivity of selected outcome measures. METHODS: Eighty-four ambulatory boys with DMD (49-180 months), 70 on CS, 14 corticosteroid naïve (NCS), participated in this 8-year multi-site study. Outcomes included; isokinetic dynamometry, the Standing (STD) and Walking/Running/jumping (WRJ) dimensions of the Gross Motor Function Measure (GMFM), and Timed Function Tests (TFTs). Nonlinear mixed modeling procedures determined the rate of change with age and the influence of steroids. RESULTS: Despite CS therapy the rate of decline in strength with age was significant in all muscle groups assessed. CS therapy significantly slowed decline in knee extensor strength, as the NCS group declined at 3x the rate of the CS group. Concurrently, WRJ skills declined in the NCS group at twice the rate of the CS group. 4-stair climb and 10 meter walk/run performance was superior in the boys on CS therapy. CONCLUSION: CS therapy slowed the rate of muscle strength decline and afforded longer retention of select gross motor skills in boys on CS compared to boys who were NCS. Isokinetic dynamometry, Walk/Run/Jump skills, and select TFTs may prove informative in assessing the efficacy of new therapeutics in ambulatory boys with DMD.

Longitudinal changes in energy cost during walking in boys with Duchenne Muscular Dystrophy (DMD).

BACKGROUND: In boys with DMD, muscle weakness progresses in a proximal to distal pattern, leading to compensatory gait strategies, including hyperlordosis and equinus, that increase energy cost and accelerate the loss of walking capacity. RESEARCH QUESTION: The purpose of this study was to determine the changes in the energy cost of walking that occur with disease progression and to determine the optimal normalization scheme for the longitudinal assessment of the energy cost of walking in boys with DMD. METHODS: Energy cost was assessed with the COSMED K4b2. Three normalization schemes were examined: gross energy cost (EC), net non-dimensional oxygen cost (NNcost) and speed-matched control energy cost (SMC-EC). Nonlinear mixed modeling procedures determined the rate of change with age. Linear regression was used to asses the relationship between each normalization scheme and age and body height. RESULTS: 74 boys with DMD were assessed for the energy cost of walking. Velocity decreased at a significant rate (-.00245/month, p = .03) across time; (Fig. 2), while gross EC (.003248/month, p = 0.0026), NNcost (.006155/month, p < 0.0001) and SMC-EC (.001690/month, p = 0.03) all increased significantly. Age and height were significantly associated with NNcost and SMC-EC. The sensitivity of NNcost and SMC-EC to age over time were similar, while SMC-EC was less sensitive to changes in height over time than NNcost. SIGNIFICANCE: In contrast to able-bodied peers, boys with DMD decrease their velocity while all walking energy cost measures increased over time. Both SMC-EC and NNcost proved appropriate normalization schemes for boys with DMD. Compared to gross EC, both NNcost and SMC-EC were less sensitive to changes in age over time, while SMC-EC was less sensitive to changes in height than NNcost. Therefore, both NNCost and SMC-EC are suggested normalization schemes for the longitudinal assessment of energy cost in boys with DMD.

Correlation of collagen X biomarker (CXM) with peak height velocity and radiographic measures of growth in idiopathic scoliosis.

STUDY DESIGN: Prospective comparative study. OBJECTIVES: Evaluate the correlation of CXM with established measures of growth. Theoretically higher CXM levels would correlate with rapid longitudinal bone growth and lower levels with growth cessation. Assessment of growth status in patients with pediatric spinal deformity is critical. The current gold standards for assessing skeletal maturity are based on radiographic measures and have large standard errors (SE). Type X collagen (COLX) is produced in the growing physis during enchondral ossification. CXM is a COLX breakdown product that can be measured in blood products. CXM, thus, is a direct measure of enchondral ossification. METHODS: IRB-approved prospective study. Q6mo anthropometrics and spine PA biplanar slot scanner images including the hand were assessed for major Cobb, Risser score (RS), triradiate cartilage status (TRC), Greulich and Pyle bone age (BA), and Sanders Score (SS). Serial dried blood spots (DBS) to obtain CXM levels were collected 3 consecutive days Q1-2 months based on SS. RESULTS: 47 idiopathic scoliosis patients, Cobb ≥ 20 were enrolled. Mean enrollment age was 11.8 years (range 7.1-16.6 years). 3103 DBS samples were assayed in quadruplicate. CXM results were highly reproducible with a 3% intraassay coefficient of variation (CV), and 12% interassay CV%. The CXM 3-day average was significantly correlated with BA R = 0.9, p < 0.001, RS R = 0.6, p < 0.001, SS R = 0.7, p < 0.001 and with height R = 0.7, p < 0.001. No patient with a CXM level < 5 ng/ml had remaining growth. CONCLUSION: CXM is the first identifiable biomarker specific to longitudinal bone growth. Early results indicate that it is a patient-specific, real-time measure of growth velocity with high correlation to the established anthropometric and radiographic measures of growth. It is predictive of cessation of growth. It is highly reproducible with a low SE. Long-term follow-up is required to determine the ability of CXM to guide clinical decision-making.

Efficacy of corticosteroid in decreasing scoliosis and extending time to loss of ambulation in a single clinic: an effectiveness trial.

PURPOSE: Pharmacologic doses of corticosteroid (CS) have been shown to ameliorate the progression of Duchenne muscular dystrophy (DMD) preserving strength, pulmonary function and ambulation as well as reducing the incidence of scoliosis. However, there are serious side effects of CS, which may impact dose tolerance. The purpose of this study was to compare the magnitude of positive CS effects on patients in our clinic to those reported in the literature. METHODS: We retrospectively reviewed medical records and radiographs of 142 DMD patients who were seen between 1st January 1991 and 31st December 2017. RESULTS: In total, 101 boys met study inclusion criteria. Of these 32 were steroid naïve, 37 took the recommended dose (standard of care, SOC) of Prednisone or Deflazacort, and 32 took a lower dose (LD). Following initiation of CS, both treatment groups showed an increase in weight velocity and decrease in linear growth velocity. Although there was a trend to later loss of ambulation (LOA) in the SOC group relative to the naïve group by one year, this was not significant, however, a small subgroup of boys on Deflazacort showed a 3.4 year later LOA than the naïve group. The incidence of scoliosis was reduced from 69% in the naïve, to 41% in the LD and 47% in the SOC group. CONCLUSIONS: Although there was a reduction in the incidence of scoliosis, it was not as robust as seen elsewhere. Many published studies have inadequate data on scoliosis probably due to the lack of inclusion of orthopaedists in the study group. LEVEL OF EVIDENCE: IV.

Image Distortion in Biplanar Slot Scanning: Part 2 Technology-specific Factors.

BACKGROUND: Biplanar digital slot scanning technology has become the standard of care in the treatment of scoliosis. Yet, the amount of distortion and reproducibility of this type of imaging modality has yet to be fully investigated. In our paper "Image distortion in biplanar slot scanning: part 1 patient-specific factors" we found that there was potentially clinically impactful interimage distortion. The purpose of this study was to evaluate the degree to which this image distortion was secondary to the image acquisition process. METHODS: Four 25 mm radio-opaque markers were placed at C3, T1, T12, and L5 on a full-length skeleton model. The skeleton was imaged in 10 different positions within the scanner. Five posteroanterior and 5 lateral images were obtained in each position. Two orthopaedic attending physicians and 3 orthopaedic resident physicians measured the markers for a total of 3200 measurements. Intraclass correlation coefficients (ICCs) and 95% confidence intervals were used to examine image distortion. RESULTS: Average marker size was 24.77, with a standard error of measurement of 0.00493. Image distortion and standard error of measurement accounted for ∼0.5% to 1.5% of total the measurement. Overall, there was good reliability and consistency when looking at markers in different views (ICC 0.790), planes, and locations within the image. Horizontal measurements were found to be more consistent and have better reliability (ICC 0.881) than vertical measurements (ICC 0.386). Position within the scanner had minimal impact on the accuracy of the measurements. CONCLUSIONS: This study demonstrates that there is minimal error due to image acquisition and measurement when using a biplanar slot scanner. Biplanar slot scanning technology tended to underestimate the size of the marker; however, the least accurate measurements only erred by 1.5% from the true length. This indicates that unlike traditional radiographs the sources of error in biplanar slot scanning images are not due to parallax and are likely due to patient-specific factors and rather than the technology itself.

Image Distortion in Biplanar Slot Scanning: Patient-specific Factors.

BACKGROUND: Error within imaging measurements can be due to processing, magnification, measurement performance, or patient-specific factors. Previous length measurement studies based on radiographs have shown good intraclass correlation coefficients (ICCs) on single images; but have not assessed interimage distortion. In our study, "image distortion in biplanar slot scanning: technology-specific factors" we determined that there is minimal image distortion due to the image acquisition when using biplanar slot scanning. In this study, we aim to determine the role of patient-specific factors in image distortion, specifically evaluating interimage distortion. METHODS: Digital radiographs and biplanar slot scanner images were reviewed in 43 magnetically controlled growing rod (MCGR) patients. Fifty-five postoperative anteroposterior digital radiographs, 184 follow-up biplanar slot-scanner scanner posteroanterior and 76 biplanar slot-scanner scanner laterals were measured by 2 residents and 1 attending. The manufacturer reported average actuator diameter of 9.02 mm was used as our reference width. RESULTS: Overall, within image interobserver ICC were moderate to excellent (0.635 to 0.983), but the interimage ICCs were poor (0.332). Digital radiographs consistently overestimated the MCGR actuator width (mean=9.655) and biplanar slot-scanner scanner images underestimated it (mean=8.935). The measurement range was large with biplanar slot-scanner scanner posteroanterior (up to 15%) and lateral (22%) measurements and with digital radiographs (39%). Patients with abnormal muscle tone had higher degrees of measurement variability. CONCLUSIONS: We found that neither biplanar slot scanning nor digital radiography was precise or accurate. Digital radiographs consistently overestimated MCGR actuator width and biplanar slot scanning underestimated it. The poor ICC's within and between image subtypes and large standard error of measurement reflected a magnitude of distortion that needs to be accounted for when using length measurements clinically. Unlike the clinically insignificant error that we noted in our previous study "image distortion in biplanar slot scanning: technology-specific factors" (0.5% to 1.5% of the measurement), the error noted in this study (0.2% to 38.5% of the measurement) has the potential to be clinically significant. Patients who have abnormal muscle tone had larger measurement errors, likely stemming from motion during the slot scanning process. LEVEL OF EVIDENCE: Level III.

Understanding the factors that impact the participation in physical activity and recreation in young adults with cerebral palsy (CP).

BACKGROUND: Young adults with cerebral palsy (CP) are reported to be less active than their typically developing peers, have higher rates of sedentary behaviors, engage in slower tempo activities, and overall participate in a smaller variety of physical activities (PA). Functional motor level and environmental barriers have been purported to impact the ability of young adult with CP to participate in physical activity and recreational activities. OBJECTIVE: To examine the role functional motor level and environmental barriers have on participation in PA and recreation in young adults with CP. METHODS: A survey assessing functional motor level (Gross Motor Function Classification Scale), participation in physical activity (The Physical Activity Scale for Individuals with Physical Disabilities) and recreation (The Assessment of Life Habits), and environmental barriers (The Craig Hospital Inventory of Environmental Factors) was mailed to 442 young adults with CP, between the ages of 18-30 years. RESULTS: 97 surveys were returned. No differences in PA were found between GMFCS level; however, young adults in GMFCS levels I and II participated more in recreational activities than GMFCS levels III-V. Greater environmental barriers were experienced by young adults in GMFCS II-V. CONCLUSIONS: This study showed that GMFCS level and physical/structural, policy and attitudinal barriers significantly impacted participation in recreational activities for young adults with CP. Strategies for addressing these barriers from both an individualized therapeutic and advocacy perspective are needed in order to facilitate engagement in physical activity and recreation for young adults with CP at all GMFCS levels.

A common data language for clinical research studies: the National Institute of Neurological Disorders and Stroke and American Academy for Cerebral Palsy and Developmental Medicine Cerebral Palsy Common Data Elements Version 1.0 recommendations.

To increase the efficiency and effectiveness of clinical research studies, cerebral palsy (CP) specific Common Data Elements (CDEs) were developed through a partnership between the National Institute of Neurological Disorders and Stroke (NINDS) and the American Academy of Cerebral Palsy and Developmental Medicine (AACPDM). International experts reviewed existing NINDS CDEs and tools used in studies of children and young people with CP. CDEs were compiled, subjected to internal review, and posted online for external public comment in September 2016. Guided by the International Classification of Functioning, Disability and Health framework, CDEs were categorized into six domains: (1) participant characteristics; (2) health, growth, and genetics; (3) neuroimaging; (4) neuromotor skills and functional assessments; (5) neurocognitive, social, and emotional assessments; and (6) engagement and quality of life. Version 1.0 of the NINDS/AACPDM CDEs for CP is publicly available on the NINDS CDE and AACPDM websites. Global use of CDEs for CP will standardize data collection, improve data quality, and facilitate comparisons across studies. Ongoing collaboration with international colleagues, industry, and people with CP and their families will provide meaningful feedback and updates as additional evidence is obtained. These CDEs are recommended for NINDS-funded research for CP. WHAT THIS PAPER ADDS: This is the first comprehensive Common Data Elements (CDEs) for children and young people with CP for clinical research. The CDEs for children and young people with CP include common definitions, the standardization of case report forms, and measures. The CDE guides the standardization for data collection and outcome evaluation in all types of studies with children and young people with CP. The CDE ultimately improves data quality and data sharing.

An exploratory study investigating the multidimensional factors impacting the health and well-being of young adults with cerebral palsy.

BACKGROUND: For young adults with cerebral palsy, changes in psychological and social development, in conjunction with the progression of musculoskeletal deformities and the onset of secondary conditions, make the transition to adulthood a difficult developmental phase. Preliminary evidence shows that many of the physical impairments reported in adults with cerebral palsy begin during late adolescence; however, there is little information about prevalence of impairments and the combined role impairments, psychological and social factors have on the health and well-being of young adults with cerebral palsy. METHODS: A cross-sectional, multidimensional survey approach was used to examine the ambulatory decline, pain, pain interference, depression, fatigue, locus of control, emotional support, overall health status and satisfaction with life of young adults with cerebral palsy, age 18-30 years. RESULTS: Ninety-seven surveys (57 self-report and 40 proxy report) were completed across all gross motor function classification system levels. No significant differences were found amongst functional levels for pain, pain interference, fatigue or depression. Only pain interference significantly contributed to the variance in health status, while emotional support significantly contributed to the variance in satisfaction with life. CONCLUSIONS: The large percentage of young adults in this study reporting pain, fatigue and depression indicates that the onset of these impairments may begin at an earlier age. This study found that emotional support from family facilitates improved health status and enhanced satisfaction with life in young adults with cerebral palsy. Similar to physical impairments, social and psychological factors also contribute to the health and well-being of young adults with cerebral palsy, a holistic approach to care that includes preventative strategies to address both mental and physical health outcomes should begin well in advance to their transition into young adulthood in order to mitigate the impact these factors have on health and well-being during this critical developmental time. Implications for Rehabilitation Pain, fatigue and depression were reported for all levels of GMFCS and should be assessed and addressed with appropriate treatment early in order to determine whether there are surgical, pharmacological, rehabilitative or counseling services that could be implemented at a younger age to improve outcomes in young adulthood. For young adults with CP, emotional support plays a significant role in the health status and satisfaction with life and strategies to enhancing support beyond the family could enhance health status and satisfaction with life.

Longitudinal community walking activity in Duchenne muscular dystrophy.

INTRODUCTION: Natural history studies for Duchenne muscular dystrophy (DMD) have not included measures of community ambulation. METHODS: Step activity (SA) monitors quantified community ambulation in 42 boys (ages 4-16 years) with DMD with serial enrollment up to 5 years by using a repeated-measures mixed model. Additionally, data were compared with 10-meter walk/run (10mWR) speed to determine validity and sensitivity. RESULTS: There were significant declines in average strides/day and percent strides at moderate, high and pediatric high rates as a function of age (P < 0.05). Significant correlations for 10mWR versus high and low stride rates were found at baseline (P < 0.05). SA outcomes were sensitive to change over 1 year, but the direction and parameter differed by age group (younger vs. older). Changes in strides/day and percentages of high frequency and low frequency strides correlated significantly with changes in 10mWR speed (P < 0.05). DISCUSSION: Community ambulation data provide valid and sensitive real-world measures that may inform clinical trials. Muscle Nerve 57: 401-406, 2018.