Physical Function In Children Research Articles

Good physical function but reduced quality of life in children 3 years after ACL reconstruction.

To assess children's physical function and subjective knee status 1 and 3 years after anterior cruciate ligament (ACL) reconstruction. It was hypothesised that there was no difference between the operated and normal legs in relation to physical strength and function, that there was <-2 mm side-to-side difference in knee laxity, and that the subjective knee function was better 3 years after ACL reconstruction compared to 1 year after. Children (<16 years of age) who had an ACL reconstruction had follow-up with physical function tests (four hop tests and strength measurement in a power rig [PR]), anterior knee laxity (measured using a Rolimeter) and patient-reported outcome measures (Pedi-International Knee Documentation Committee [IKDC] and Knee injury and Osteoarthritis Outcome Score [KOOS]-Child) 1 and 3 years postoperatively. Changes from 1- to 3-year follow-up were evaluated with a paired t test. Out of 148 ACL reconstructed children, 60 had all measures. The four hop tests and the PR all yielded a Limb Symmetry Index >90% at both follow-ups. There was a significant improvement from 1- to 3-year follow-up in two-hop tests (6 m on time and crossover hop). Side-to-side knee laxity was >2 mm in four children at 1- and 3-year tests. Pedi-IKDC scores increased, and KOOS-Child improved significantly from 1 to 3 years in two of the five domains: 'Sport' and 'Quality of life', but scores were lower than in a cohort of normal children. The children had good objective physical function 1 and 3 years after ACL reconstruction. However, scores from the KOOS-Child sport-specific function and quality of life domains were lower than in normal children. Level II.

Impact of a Pilates intervention on physical function in children with generalised joint hypermobility and chronic musculoskeletal pain: A single-case experimental design

AimTo assess the impact of a Physiotherapist-led Pilates Intervention for school aged children with Generalised Joint Hypermobility (GJH) on pain, physical function and Health Related Quality of Life (HRQoL). MethodsThree children aged 8–12 years with GJH participated in an 8 week Physiotherapist-led Pilates Intervention within a single-case experimental design (multiple baseline design). Repeated measures were collected during baseline, intervention, withdrawal and follow-up, for: (i) pain, (ii) physical function as measured by muscle strength, postural control, fatigue and activity levels and (iii) HRQoL. ResultsWithin the intervention phase, two children showed reduced fatigue and one child improved in muscle group strength of hip abduction (gluteus medius) and scapula adduction/rotation (rhomboideus major/minor) and HRQoL. No improvements were seen in pain or postural control. Within the early withdrawal phase all children showed improved strength for at least two muscle groups and one child showed reduced fatigue, pain (worst in last week) and improved postural control (functional reach lateral). ConclusionsPilates may provide an effective intervention for children with GJH to reduce fatigue and improve muscle strength and HRQoL. Limited conclusions can be made regarding pain and postural control. Further research with a longer Pilates duration is needed to confirm the dose and benefits for this population.

Psychostimulant Medications for Physical Function and Spasticity in Children With Cerebral Palsy: Protocol for a Randomized Controlled Trial.

Cerebral palsy (CP) is a prevalent nonprogressive disorder that leads to impaired movement (ie, spasticity), posture, and balance, which affects functions such as walking and upper extremity tasks. Current medical treatments show efficacy in improving motor performance but have considerable side effects. Emerging off-label use of central nervous system (CNS) medications for improving motor performance has shown promising results in children with CP and other populations. The aim of this study is to describe a protocol for a pilot randomized controlled trial (RCT) to examine the safety, tolerability, and efficacy of methylphenidate (MPH) and modafinil on spasticity and motor performance in children with CP. This will be a protocol study for a pilot, triple-masked, placebo-controlled RCT (a class I trial following the American Academy of Neurology criteria) with blinded patients, outcome assessors, and intervention delivery team. Eligible children should be diagnosed with CP levels I or II based on the Gross Motor Function Classification System and be aged between 7 and 12 years. Thirty-six children with CP will be randomized into 3 groups to receive (1) MPH (2.5 mg of MPH + 100 mg placebo), (2) modafinil (100 mg modafinil + 2.5 mg placebo), or (3) a placebo (2.5 mg placebo + 100 mg placebo), in addition to physical therapy for 12 weeks. Primary outcomes include the Gross Motor Function Measure-66 and the Modified Ashworth Scale. Secondary outcomes include the Timed Up and Go test, 5 Time Sit to Stand test, Modified Clinical Test for Sensory Interaction of Balance, and 10-Meter Walk Test. The protocol has been accepted by Kuwait University (VDR/EC-225) and the Ministry of Health of Kuwait (2022/2157). The inclusion of participants will start in June 2024. The combination of CNS stimulant medications and controlling for rehabilitation has not been studied yet. The findings of this study may determine if using CNS stimulant medications is beneficial for the reduction of spasticity and improvement of physical function in children with spastic CP. ClinicalTrials.gov NCT05675098; https://clinicaltrials.gov/study/NCT05675098. PRR1-10.2196/53728.

Relationship between exercise habits and physical function in children aged 9-12 years.

Exercise improves physical health and positively impacts physical functions in children. Additionally, the habitual exercise established during childhood often continues into adulthood. Therefore, childhood is an important period for establishing healthy habits. Investigating the relationship between the presence or absence of daily exercise and physical functions may provide important information to confirm poor physical function in children with poor exercise habits. Therefore, this study examined the relationship between exercise habits as defined by the Japanese Ministry of Health, Labor, and Welfare and physical function in children aged 9-12 years. Approximately 239 children were included in this study. Participants answered a questionnaire about their exercise habits. We evaluated the skeletal muscle mass index, grip strength, standing broad jump, one-leg standing time, and gait deviation index. Logistic regression analyses were performed to assess exercise habit association with skeletal muscle mass index, physical function, and gait deviation index after adjusting for sex. Of the 239 children, 75.5% (n = 178) had exercise habits. A significant association was noted between exercise habits and skeletal muscle mass index and standing broad jump (skeletal muscle mass index: OR, 1.84; 95% CI, 1.01-3.36 and p<0.05; standing long jump: OR, 1.02; 95% CI, 1.00-1.04 and p<0.05). In conclusion, exercise regardless of exercise level for at least 30 min per day, 2 days per week, for at least 1 year is important for skeletal muscle mass and instantaneous lower limb muscular strength development in school-aged children.

Revving up possibilities: can psychostimulants enhance physical function in children with cerebral palsy?

Revving up possibilities: can psychostimulants enhance physical function in children with cerebral palsy?

Effect of psychostimulant medications on physical function in children with cerebral palsy: scoping review.

The aim of this scoping review is to examine the extent and depth of the literature on effects of central nervous system (CNS) stimulant medications on physical function in children with cerebral palsy (CP). A systematic search for relevant peer-reviewed studies was conducted of PubMed, CINAHL, Cochrane, SPORTDiscus, Embase, & Scopus (January 2002 & August 2022). We included studies that examined the effects of CNS stimulants on physical function in children with CP. Four studies met our selection criteria. All studies explored the effect of Modafinil on physical function outcomes. Three studies of the four included studies reported positive effects of Modafinil on spasticity, motor performance, and gait, whereas one study reported no significant effects of Modafinil. Our findings suggest that there is very low-quality evidence that suggests that Modafinil may enhance physical improvements in body structure and function, including reduction in spasticity and improvements in gait parameters. IMPACT: Central nervous system stimulants were examined for efficacy on physical function and spasticity in children with cerebral palsy. The evidence on the effects of central nervous system stimulants on physical function in children with CP is limited and inconsistent.

Physical function in children and adolescents pre- and 1-year post-liver transplant.

Several studies describe poorer motor developmental motor outcomes post-liver transplant (LT) in younger children. Limited studies examine physical function in older children and adolescents pre- and post-LT. Retrospective review of standard of care physical function outcome measures pre- and 1-year post-LT in children ≥6 years at LT. Measures include: 6-minute walk test (6MWT), grip strength, Bruininks-Oseretsky Test of Motor Proficiency-2 (BOT-2) components, Physical Activity Questionnaire (PAQ), and Paediatric Quality of Life Multidimensional Fatigue Scale. Association of medical variables with outcomes was explored. The study cohort included 23 (8 male, median (interquartile range) age 11.67 (8.25, 13.92) years at LT) participants. Top two primary diagnoses included biliary atresia (30.4%) and fulminant hepatic failure (21.7%). At 1-year post-LT, over one-third (36%) were overweight or obese. Compared with healthy norms, children had significantly lower pre-LT PAQ scores (p = .002), pre- and post-6MWT scores (p < .001) and post-LT BOT-2 strength and agility scores (p < .001). Pre-LT, lower balance scores were associated with abdominal distention/ascites (p = .009) and splenomegaly (p = .017). Lower pre-LT platelet count correlated with poorer balance (r = .532, p = .017) and lower strength and agility scores (r = .446, p = .043). Significant moderate inverse correlations were found between weight/body mass index z-scores and BOT-2 components. Post-LT children continue to demonstrate decreased levels of motor proficiency and functional capacity but report less fatigue and increased physical activity. Older children and adolescents undergoing LT are at risk of decreased physical function, highlighting the need for pre- and post-LT rehabilitation to optimize long term outcomes.

Skeletal muscle single fiber force production declines early in juvenile male mice with chronic kidney disease.

Children with chronic kidney disease (CKD) frequently exhibit delayed physical development and reduced physical performance, presumably due to skeletal muscle dysfunction. However, the cellular and molecular basis of skeletal muscle impairment in juvenile CKD remains poorly understood. Cellular (single fiber) and molecular (myosin-actin interactions and myofilament properties) function was examined ex vivo in slow (soleus) and fast (extensor digitorum longus) contracting muscles of juvenile male (6 weeks old) CKD and control mice. CKD was induced by 0.2% adenine diet for 3 weeks starting at 3 weeks of age. Specific tension (maximal isometric force divided by cross-sectional area) was reduced in larger myosin heavy chain (MHC) I and IIA fibers and in all IIB fibers in juvenile male mice with CKD due to fewer strongly bound myosin-actin cross-bridges. Fiber cross-sectional area in juvenile CKD mice was unchanged in MHC I and IIB fibers and increased in MHC IIA fibers, compared to controls. CKD slowed cross-bridge kinetics (slower rate of myosin force production and longer myosin attachment time, ton ) in MHC IIA fibers, and accelerated kinetics (shorter ton ) in MHC IIB fibers, which may indicate fiber type dependent shifts in contractile velocity in juvenile CKD. Overall, our findings show that single fiber myopathy is an early event during juvenile CKD, manifesting prior to the development of cellular atrophy as reduced force generation due to fewer strongly bound myosin heads. These results warrant clinical translation and call for early interventions to preserve physical function in children with CKD.

Effectiveness of eAsthmaCare on Symptoms, Childhood Asthma Control Test, and Lung Function among Asthmatic Children.

A web-based self-health management system-eAsthmaCare, was developed as an intervention for asthmatic children. A randomized controlled trial was performed. Consent was obtained for 98 children with asthma to participate in the study and the pre- and post-test data collection process. The experimental group was given access to eAsthmaCare online management, the control group was subjected to general asthma management. The experimental and control groups' asthma symptoms, Childhood Asthma Control Test (C-ACT) scores, and lung function were evaluated, and their pre- and 3-month post-test results were compared. The following records were maintained: (1) medication record (2) daily asthma symptoms log (3) monthly C-ACT and lung function records. The C-ACT results indicated a p-value of < .01 for: overall improvements to childhood asthma symptoms, time effect, group and time interaction effects, and group and time interaction effects in relation to sleeping condition on the previous day; cough symptom time effect, and group and time interaction effects; the two groups' time effect in relation to cough symptoms; the two groups' time effect in relation to monthly activity restrictions (number of days); and the two groups' time effect in relation to nasal symptoms; the two groups' time effect; and group and time interaction effects (p < .01). In terms of the predictive values for lung function (FVC, FEV1, PEFR), the improvements in both groups were not statistically significant. The implementation of the eAsthmaCare intervention might have a positive impact on pediatric patients, making it an effective management tool for monitoring asthmatic children's physical function and discomfort.

Translating Promoting Factors and Behavior Change Principles Into a Blended and Technology-Supported Intervention to Stimulate Physical Activity in Children With Asthma (Foxfit): Design Study.

BackgroundChildren with asthma can decrease the impact of their disease by improving their physical activity (PA). However, health care providers lack interventions for children with asthma that effectively increase their PA levels and achieve behavior change. A technology-supported approach can positively influence PA and physical functioning in children.ObjectiveThe aims of this study were to develop a technology-supported intervention that facilitates health care providers in promoting PA for children (aged 8 to 12 years) with asthma and to systematically describe this developmental process.MethodsIntervention mapping (IM) was applied to develop a blended and technology-supported intervention in cocreation with children with asthma, their parents, and health care providers. In accordance with the IM framework, the following steps were performed: conduct a needs assessment; define the intervention outcome, performance objectives, and change objectives; select theory-based intervention methods and strategies; create components of the intervention and conduct pilot tests; create an implementation plan; and create an evaluation plan.ResultsWe developed the blended intervention Foxfit that consists of an app with a PA monitor for children (aged 8 to 12 years) with asthma and a web-based dashboard for their health care provider. The intervention focuses on PA in everyday life to improve social participation. Foxfit contains components based on behavior change principles and gamification, including goal setting, rewards, action planning, monitoring, shaping knowledge, a gamified story, personal coaching and feedback, and a tailored approach. An evaluation plan was created to assess the intervention’s usability and feasibility for both children and health care providers.ConclusionsThe IM framework was very useful for systematically developing a technology-supported intervention and for describing the translational process from scientific evidence, the needs and wishes of future users, and behavior change principles into this intervention. This has led to the technology-supported intervention Foxfit that facilitates health care providers in promoting PA in children with asthma. The structured description of the development process and functional components shows the way behavior change techniques are incorporated in the intervention.Trial RegistrationInternational Clinical Trial Registry Platform NTR6658; https://tinyurl.com/3rxejksf

Vitamin D supplementation and increased dairy protein intake do not affect muscle strength or physical function in healthy 6-8-year-old children: the D-pro randomized trial.

PurposeTo investigate separate and combined effects of vitamin D supplementation during the extended winter and increased dairy protein intake on muscle strength and physical function in children, and furthermore to explore potential sex differences.MethodsIn a 2 × 2-factorial, randomized winter trial, 183 healthy, 6–8-year-old children received blinded tablets with 20 µg/day vitamin D3 or placebo, and substituted 260 g/day dairy with yogurts with high (HP, 10 g protein/100 g) or normal protein content (NP, 3.5 g protein/100 g) for 24 weeks during winter at 55° N. We measured maximal isometric handgrip and leg press strength, and physical function by jump tests and a 30 s sit-to-stand test. Physical activity was measured by 7-day accelerometry.ResultsBaseline (mean ± SD) serum 25-hydroxyvitamin D was 80.8 ± 17.2 nmol/L, which increased to 88.7 ± 17.6 nmol/L with vitamin D supplementation and decreased to 48.4 ± 19.2 nmol/L with placebo. Baseline protein intake was 15.5 ± 2.4 E%, which increased to 18.4 ± 3.4 E% with HP and was unchanged with NP. We found no separate or combined effects of vitamin D supplementation and/or increased dairy protein intake on muscle strength or physical function (all P > 0.20). There was an interaction on the sit-to-stand test (Pvitamin×yogurt = 0.02), which however disappeared after adjusting for physical activity (P = 0.16). Further, vitamin D supplementation increased leg press strength relatively more in girls compared to boys (mean [95% CI] 158 [17, 299] N; Pvitamin×sex = 0.047).ConclusionOverall, vitamin D and dairy protein supplementation during the extended winter did not affect muscle strength or physical function in healthy children. Potential sex differences of vitamin D supplementation should be investigated further.Registered at clinicaltrials.govNCT0395673.Supplementary InformationThe online version contains supplementary material available at 10.1007/s00394-022-02912-0.

The concern of childcare and education site about the “abnormalities in physical function” among children based on the results of ‘The questionnaire about physical function of children in 2020’

The concern of childcare and education site about the “abnormalities in physical function” among children based on the results of ‘The questionnaire about physical function of children in 2020’

OEIS Syndrome: Omphalocele, Exstrophy of the Cloaca, Imperforate Anus, and Spinal Defects

OEIS Syndrome: Omphalocele, Exstrophy of the Cloaca, Imperforate Anus, and Spinal Defects

Physical Functioning After Admission to the PICU: A Scoping Review.

To conduct a scoping review to 1) describe findings and determinants of physical functioning in children during and/or after PICU stay, 2) identify which domains of physical functioning are measured, 3) and synthesize the clinical and research knowledge gaps. A systematic search was conducted in PubMed, Embase, Cumulative Index to Nursing and Allied Health Literature, and Cochrane Library databases following the Preferred Reporting Items for Systematic Reviews and Meta-analyses extension for Scoping Reviews guidelines. Two investigators independently screened and included studies against predetermined criteria. One investigator extracted data with review by a second investigator. A narrative analyses approach was used. A total of 2,610 articles were identified, leaving 68 studies for inclusion. Post-PICU/hospital discharge scores show that PICU survivors report difficulties in physical functioning during and years after PICU stay. Although sustained improvements in the long-term have been reported, most of the reported levels were lower compared with the reference and baseline values. Decreased physical functioning was associated with longer hospital stay and presence of comorbidities. A diversity of instruments was used in which mobility and self-care were mostly addressed. The results show that children perceive moderate to severe difficulties in physical functioning during and years after PICU stay. Longitudinal assessments during and after PICU stay should be incorporated, especially for children with a higher risk for poor functional outcomes. There is need for consensus on the most suitable methods to assess physical functioning in children admitted to the PICU.

Relationship between the skeletal muscle mass index and physical activity of Japanese children: A cross-sectional, observational study.

Regular physical activity is an important component of physical health of children and has been associated with increasing skeletal muscle mass and muscle strength. Children with low levels of physical activity may experience health problems, such as loss of muscle mass, later in life. Thus, it may be valuable to identify declining physical function in children who do not perform the recommended amount of physical activity. Therefore, we aimed to evaluate the relationship between the amount of physical activity performed for ≥60 min per day for ≥5 days per week and the skeletal muscle mass index and physical function in young children. In total, 340 typically developing children aged 6-12 years (175 girls; average age, 9.5±1.9 years) were included in this cross-sectional study. We evaluated the proportion of children performing the recommended minimum of 60 min of daily moderate-to-vigorous physical activity at least 5 days per week. The skeletal muscle mass and Gait Deviation Index scores, gait speed, grip strength, Five Times Sit-to-Stand test results, Timed Up-and-Go test results, one-leg standing time, and gait efficiency were evaluated. Multiple logistic regression analyses were performed to assess the association of moderate-to-vigorous physical activity with the skeletal muscle mass index, percent body fat, and physical function, after controlling for confounding factors (age and sex). A logistic regression analysis revealed that the skeletal muscle mass index was independently associated with moderate-to-vigorous physical activity (odds ratio, 2.34; 95% confidence interval, 1.17-4.71; P = 0.017). Performance of moderate-to-vigorous physical activity for ≥5 days per week for ≥60 min per day was associated with the skeletal muscle mass index score of Japanese children. Our findings highlighted the importance of performing moderate-to-vigorous physical activity for the development of skeletal muscle mass in children.

Abstract P162: Comorbid Asthma And Allergies Are Common In Children With Congenital Heart Disease And Related To Decreased Physical Functioning

Background: Congenital heart defects (CHD) are the most common birth defect and asthma and allergies are common conditions in childhood. The co-occurrence of these is not well understood but may be increased due to similar risk factors or immune dysregulation related to early life inflammation. This study aimed to compare the prevalence of asthma and allergies between children with CHD and their unaffected siblings and assess their impact on physical functioning in children with CHD. Methods: Parent-reported data was collected on allergy medications and asthma diagnosis, physical functioning (PedsQL physical health), and characteristics in children (age 12-17) with surgically treated CHD and an unaffected sibling (age 11-18). The McNemar test compared the comorbidities between siblings. Among children with CHD, logistic and linear regression were used to estimate the association of each comorbidity with child characteristics and physical functioning respectively. Models included CHD severity, SES, age, BMI, and sex. Results: Among 218 sibling pairs, the children with CHD had a higher, but not statistically significant, prevalence of asthma and allergies (Figure 1). In all 497 children with CHD, single ventricle CHD had a 2.5 times greater odds of allergies compared with non-critical (95%CI=1.0,6.0) with no difference in asthma. Asthma was associated with decreased physical functioning (8.7 point decrease vs no asthma, 95%CI=-14.5,-2.9). Conclusions: Children with CHD and their siblings had similar levels of allergies and asthma. However, as symptoms of these conditions may overlap, studies should further investigate a possible link with CHD, especially given the lower physical health in patients with CHD and asthma.

The relationship between quality of life and coping strategies of children with EB and their parents

BackgroundEpidermolysis bullosa (EB) is a group of rare genetic skin disorders that primarily manifest as blisters and erosions following mild mechanical trauma. Despite the crucial role of the parents of children with EB in managing the disease, studies focusing on the parent–child relationship remain a gap in the literature. To address this gap, the current quantitative study, involving 55 children with all types of EB and 48 parents, assessed the relationship between their quality of life and coping strategies. Quality of life was measured with the Pediatric Quality of Life Inventory and TNO-AZL Questionnaire for Adult’s Health- related Quality of Life, and coping strategies were assessed with the Coping with a Disease Questionnaire. The majority of the analyses were descriptive and the results were interpreted qualitatively because of the small sample size.ResultsOverall, the quality of life of children with EB and that of their parents was somewhat lower compared with the quality of life of healthy children and adults. Children with EB who more frequently used emotional reactions and cognitive-palliative strategies to cope with the disease demonstrated lower levels of emotional and social functioning, while children who showed more acceptance and distancing showed higher levels of functioning on all domains. Parents who frequently demonstrated emotional reactions reported lower levels of social functioning and experienced more depressive emotions and anger. Parents who used more avoidance showed higher levels of positive emotions. Within parent–child dyads, acceptance, cognitive-palliative strategies and distancing were positively related. Children’s emotional and social functioning were negatively associated with their parents’ depressive emotions. Parents’ acceptance was linked to higher physical functioning in children, whereas children’s avoidance was linked to a lower level of anger in parents.ConclusionChildren who are able to accept the disease or distance themselves from it appear to be better off in contrast to those who tend to engage in the cognitive-palliative strategies and expressing emotional reactions. Parents seem to be better off when they are able to use avoidance in contrast to those who tend to show emotional reactions. Further research is needed to substantiate these findings.

Evaluating the Impact of a Pilates Intervention on Physical Function in Children with Hypermobility Spectrum Disorder: A Study Protocol Using Single-Case Experimental Design

Background: Pilates has been shown to be an effective intervention for adults with musculoskeletal conditions with only a few examples available in the literature for children. As musculoskeletal pain is a major symptom experienced by children with Hypermobility Spectrum Disorder (HSD), they may benefit from practicing Pilates to improve postural alignment, strength and motor control to effectively distribute movement load and decrease adverse load through involved joints. Method: This study aims to evaluate the impact of a Physiotherapy-led Pilates intervention on school aged children with HSD and the benefits of this approach on pain, physical function and quality of life when delivered in a community-based model of care. A single-case experimental design (SCED) that incorporates a multiple baseline design will be used. Children with HSD, aged from 8 to 12 years, will commence concurrently in this study. Participants will undergo multiple assessments through all phases of the study which incorporates an A-B-A withdrawal design. The initial baseline period will be randomised from 5 to 7 weeks duration, then participants will enter the intervention period for 8 weeks followed by a withdrawal period of 5 weeks. The Physiotherapy-led Pilates intervention will consist of individual, 45 minute bi-weekly sessions, performed on both mat and the Pilates Reformer with an additional home program of mat exercises performed weekly. The study hypotheses are that children will show: 1) a decrease in pain; 2) an increase in their physical function as measured by muscle strength, postural control, fatigue and physical activity levels; and 3) an improvement in their Health Related Quality of Life in the domains of physical, emotional, social and school functioning. Conclusion: The findings will add specific responsiveness information to the scientific evidence for Physiotherapy-led Pilates for children with HSD.

Dual X-ray absorptiometry has limited utility in detecting bone pathology in children with hypophosphatasia: A pooled post hoc analysis of asfotase alfa clinical trial data.

Asfotase alfa is an enzyme replacement therapy approved for treatment of patients with pediatric-onset hypophosphatasia (HPP), a rare, inherited, systemic disease causing impaired skeletal mineralization, short stature, and reduced physical function in children. The role of dual X-ray absorptiometry (DXA) in the assessment of children with HPP has been insufficiently explored. This post hoc analysis included pooled DXA data from 2 open-label, multicenter studies in 19 children with HPP. The study population was aged ≥5 to <18years and had received asfotase alfa for ≤6.6years at enrollment (male: 79%; median age at enrollment: 10.4 y [range: 5.9-16.7]; treatment duration: 6.3 y [range: 0.1-6.6]. Baseline height Z-scores indicated short stature (median [min, max]: -1.26 [-6.6, 0]); mean [SD]: -2.30 [1.97]), thus requiring height adjustment of DXA Z-scores. At Baseline, few patients had height-adjusted bone mineral density (BMDht) Z-scores of -2 or less for whole body (n=3) or lumbar spine (n=5). In treated patients, mean whole body and lumbar spine BMDhtZ-scores did not change over time, but whole body and lumbar spine height- adjusted bone mineral content (BMCht) Z-scores increased significantly from Baseline to Last Assessment (P≤0.0056). Improvements in Radiographic Global Impression of Change (RGI-C) scale scores correlated significantly with increases in whole body and lumbar spine BMChtZ-scores (P<0.05) but not BMDhtZ-Scores. Improvements in Rickets Severity Score (RSS) correlated significantly with increases in lumbar spine BMDhtZ-scores and whole body BMCht Z-scores (P<0.05). No significant correlations were observed between any DXA and bone histomorphometry measure. These findings suggest that DXA BMD Z-scores, which are commonly used in clinical practice, have limited utility in assessing deficient bone mineralization in patients with HPP. Although BMChtZ-scores increased significantly over time with asfotase alfa therapy, the lack of significant changes in more than one DXA parameter suggests that this tool may not be useful in everyday clinical practice. Furthermore, the use of BMC as an independent metric is not typical or recommended by guidelines. Complementary measures, such as skeletal radiographs supplemented with age-appropriate functional assessments, should be considered.

Assessing Physical Function and Mobility following Pediatric Traumatic Brain Injury with the NIH Toolbox Motor Battery: A Feasibility Study

Aims Traumatic brain injury (TBI) can impair physical function in children. The NIH Toolbox Motor Battery (NIHTB-M) was designed to be a brief assessment of physical function, but no studies have examined its use in children with TBI. This study aims to describe the feasibility of using the NIHTB-M to assess children with TBI. Methods The NIHTB-M was administered to children with TBI 2 weeks (n = 22) and/or 6 months (n = 23) following injury. This descriptive study summarizes participant performance, administration challenges, and the association between NIHTB-M scores, participant characteristics, and subjective report of physical function. Results Of the NIHTB-M domains, deficits in endurance and balance were most prevalent. Children aged 5 to 16 years could complete the assessment per administration guidelines, except for a few cases (n = 3) where orthopedic injuries limited participation. Younger children (aged 3 to 4) had difficulty following the NIHTB-M directions. Technological issues impacted balance assessment in several cases (n = 6). Conclusion The NIHTB-M is brief to administer, generally well tolerated by school-aged children and, despite occasional technological challenges, is a feasible performance-based battery for assessment of children with TBI for clinical and research purposes. Additional investigation of psychometric properties and ceiling and floor effects is needed.