Fidelity of Delivery in a Multisite Randomized Clinical Trial of Intervention Efficacy for Infants With Unilateral Cerebral Palsy.

PURPOSE: To investigate the reliability of a measure of fidelity of therapist delivery, quantify fidelity of delivery, and determine factors impacting fidelity in the Rehabilitation EArly for Congenital Hemiplegia (REACH) clinical trial. METHODS: Ninety-five infants (aged 3-9 months) with unilateral cerebral palsy participated in the REACH clinical trial. The Therapist Fidelity Checklist (TFC) evaluated key intervention components. Video-recorded intervention sessions were scored using the TFC. RESULTS: Inter- and intrarater reliability was percentage agreement 77% to 100%. Fidelity of delivery was high for 88.9% of sessions and moderate for 11.1% of sessions. Sessions with moderate scores included infants receiving infant-friendly bimanual therapy and occurred at the intervention midpoint or later. No significant relationships were found for TFC scores and infant age, manual ability, or parent engagement. CONCLUSIONS: Fidelity of delivery was high for the REACH trial in most intervention sessions. Standardized therapist training with intervention manuals and monthly peer-to-peer support likely contributed to these results.

Screening and Evaluation of Neurodevelopmental Impairments in Infants Under 6 Months of Age with Congenital Heart Disease.

Clinical evaluation of neurodevelopmental impairments before 6 months of age is needed in congenital heart disease (CHD) to promote early referral to developmental interventions. The objective was to identify the risk of cerebral palsy (CP) and to compare neurodevelopment outcomes in infants with and without CHD. In a longitudinal study, 30 infants with CHD and 15 infants without CHD were assessed at 1 month, 3 months, and 6 months of age. Included measures were General Movement Assessment (GMA), Test of Infant Motor Performance (TIMP) and the Bayley Scale of Infant Development, third edition (Bayley-III), selected to identify the risk of CP, document neurodevelopmental impairments and infants' eligibility for early intervention services. Abnormal GMA categories were found in the CHD group where 48% had poor repertoire and 15% were at high risk of CP. At 3 months of age, CHD group had significantly lower TIMP scores compared to infants without CHD [t(41) = 6.57, p = 0.01]. All infants in the study had higher Bayley-III scores at 6 months than at 3 months of age. Infants with CHD had lower gross motor, fine motor and cognitive Bayley-III scores compared to their peers without CHD. Over time infants without CHD outperformed the CHD group in the gross motor skills [F(1,41) = 11.76, p = .001]. Higher prevalence of abnormal GMs, lower TIMP and Bayley-III were found in infants with single ventricle physiology compared to two-ventricle physiology. The risk of CP exists in infants with CHD, and these infants have worse outcomes compared to their peers without CHD. These differences are intensified in the single ventricle population.Clinical Trial Registration National Institute of Health, Unique identifier: NCT03104751; Date of registration-April 7, 2017.

Daily Outpatient Physical Therapy for a Toddler With a Neurodegenerative Disease: A Case Report.

PURPOSE: This case report highlights the potential value of delivering a high-dose physical therapy (PT) intervention for a child with a neurodegenerative disease. We include developmental outcomes for a 23-month-old toddler with biallelic TBCD gene mutations following daily outpatient PT. SUMMARY OF KEY POINTS: The child had clinical improvements in gross and fine motor, cognition, expressive and receptive language, socioemotional, and adaptive behavior function as determined through Goal Attainment Scaling, Gross Motor Function Measure, and Bayley Scales of Infant and Toddler Development following daily PT intervention. STATEMENT OF CONCLUSION AND RECOMMENDATIONS FOR CLINICAL PRACTICE: High-dose outpatient PT may be beneficial for a child with a neurodegenerative disease at some time frames. In selected cases, if the neurodegenerative disease slowly progresses, high-dose PT may be a treatment option to promote motor change.

3D Motion Capture May Detect Spatiotemporal Changes in Pre-Reaching Upper Extremity Movements with and without a Real-Time Constraint Condition in Infants with Perinatal Stroke and Cerebral Palsy: A Longitudinal Case Series.

Perinatal stroke (PS), occurring between 20 weeks of gestation and 28 days of life, is a leading cause of hemiplegic cerebral palsy (HCP). Hallmarks of HCP are motor and sensory impairments on one side of the body-especially the arm and hand contralateral to the stroke (involved side). HCP is diagnosed months or years after the original brain injury. One effective early intervention for this population is constraint-induced movement therapy (CIMT), where the uninvolved arm is constrained by a mitt or cast, and therapeutic activities are performed with the involved arm. In this preliminary investigation, we used 3D motion capture to measure the spatiotemporal characteristics of pre-reaching upper extremity movements and any changes that occurred when constraint was applied in a real-time laboratory simulation. Participants were N = 14 full-term infants: N = six infants with typical development; and N = eight infants with PS (N = three infants with PS were later diagnosed with cerebral palsy (CP)) followed longitudinally from 2 to 6 months of age. We aimed to evaluate the feasibility of using 3D motion capture to identify the differences in the spatiotemporal characteristics of the pre-reaching upper extremity movements between the diagnosis group, involved versus uninvolved side, and with versus and without constraint applied in real time. This would be an excellent application of wearable sensors, allowing some of these measurements to be taken in a clinical or home setting.

Reliability and Responsiveness of the Timed Up and Go Test in Children With Cerebral Palsy.

PURPOSE: The purpose of this study was to determine the absolute reliability and responsiveness of the Timed Up and Go (TUG) test, as measured using minimal detectable change (MDC) and minimal clinical important difference (MCID) values. METHODS: Prospective observational study of children aged 3 to 10 years with cerebral palsy (CP) in Gross Motor Function Classification System levels I-III who completed the TUG test. Minimal detectable change estimates were calculated using baseline data. MCID estimates for each Gross Motor Function Classification System (GMFCS) level were calculated using distribution- and anchor-based methods. RESULTS: Minimal detectable change values ranged from 1.40 to 8.74 seconds and MCID estimates ranged from 0.22 to 5.31 seconds. CONCLUSIONS: The TUG test is a reliable and responsive measure of balance and mobility for children with CP between 3 and 10 years of age in GMFCS levels I-III. Study results support improved use of the TUG test in clinical and research settings by providing reliability values and estimates of meaningful change. VIDEO ABSTRACT: For more insights from the authors, see Supplemental Digital Content 1, available at http://links.lww.com/PPT/A117.

Technology for Children With Brain Injury and Motor Disability: Executive Summary From Research Summit IV.

Advances in technology show promise as tools to optimize functional mobility, independence, and participation in infants and children with motor disability due to brain injury. Although technologies are often used in adult rehabilitation, these have not been widely applied to rehabilitation of infants and children. In October 2015, the Academy of Pediatric Physical Therapy sponsored Research Summit IV, "Innovations in Technology for Children With Brain Insults: Maximizing Outcomes." The summit included pediatric physical therapist researchers, experts from other scientific fields, funding agencies, and consumers. Participants identified challenges in implementing technology in pediatric rehabilitation including accessibility, affordability, managing large data sets, and identifying relevant data elements. Participants identified 4 key areas for technology development: to determine (1) thresholds for learning, (2) appropriate transfer to independence, (3) optimal measurement of subtle changes, and (4) how to adapt to growth and changing abilities.

Early results of neurodevelopment following hybrid stage I for hypoplastic left heart syndrome.

Motor skills and neurodevelopment in infants with hypoplastic left heart syndrome (HLHS) who have undergone Hybrid Stage I palliation is unknown. The purpose of this study is to assess early neurodevelopment in infants with HLHS after Hybrid Stage I palliation. Developmental assessment was performed in HLHS infants who underwent Hybrid Stage I palliation at 2 and 4 months of age using the Test of Infant Motor Performance, and at 6 months of age, prior to undergoing the second staged surgery, using the Bayley Scales of Infant and Toddler Development, 3rd edition (Bayley-III). Results were compared to healthy control subjects and norm-referenced data. The HLHS group scored between -1 and -2 standard deviations (SD) below the mean at 2 months of age (p = 0.002), and within -1 SD of the mean, at 4 months of age (p = 0.0019), on the TIMP. Compared to the control group, composite motor skills were significantly lower at 6 months of age on the Bayley-III in the HLHS group (p = 0.0489), however, not significant for cognitive (p = 0.29) or language (p = 0.68). Percentile rank motor scores were 17 ± 20 % in the HLHS group compared to 85 ± 12 % for the healthy age-matched control group. Infants with HLHS who undergo Hybrid Stage I palliation score lower on standardized motor skill tests compared to healthy age-matched controls and the norm-referenced population. This suggests that infants with HLHS have poorer motor skill performance than typically developing infants at 6 months of age.

Daily Intervention for Young Children With Cerebral Palsy in GMFCS Level V: A Case Series.

PURPOSE: To describe a daily physical therapy (PT) intervention program and outcomes for 2 young children with spastic quadriplegia, Gross Motor Function Classification System (GMFCS) level V, and to evaluate the feasibility of using a daily program in an urban children's hospital outpatient setting. SUMMARY OF KEY POINTS: Two young children, GMFCS level V, received 2 hours of PT intervention based on motor learning principles 5 days a week for 4 consecutive weeks. Gross Motor Functional Measure (GMFM-66, GMFM-88) and the Bayley Scales of Infant Development, Third Edition, were used as pre- and postoutcome measures. The daily, high intensity intervention was well tolerated. Improvements in motor function, language, and cognitive skills were found. STATEMENT OF CONCLUSION: A daily PT program appears feasible and may improve overall development in young children with cerebral palsy in GMFCS level V.

Manual Abilities and Cognition in Children with Cerebral Palsy: Do Fine Motor Skills Impact Cognition as Measured by the Bayley Scales of Infant Development?

Manual ability may be an important consideration when measuring cognition in children with CP because many items on cognitive tests require fine motor skills. This study investigated the association of fine motor dependent (FMD) and fine motor independent (FMI) items within the cognitive domain (COG) of the Bayley Scales of Infant Development-Third Edition (Bayley-III) and Manual Ability Classification System (MACS) in children with cerebral palsy. Children aged 2 to 8 (3.96 ± 1.68) years were included in this study. MACS levels were assigned at baseline. COG was administrated at baseline (n = 61) and nine months post-baseline (n = 28). The 91 items were classified into FMD (52) and FMI (39). Total raw score, FMD, and FMI scores were calculated. The association between MACS and cognitive scores (total, FMD, and FMI) were evaluated using linear regression and Spearman correlation coefficients. We found total, FMD, and FMI scores decrease significantly as the MACS level increases at the baseline. Both FMD and FMI scores decreased as MACS levels increased (worse function). There was a significant difference between the two slopes, with the FMD scores having a steeper slope. Similar patterns were observed nine months post-baseline. Children with lower manual ability scored lower in the cognitive domain at baseline and 9 months post-baseline. The significant difference in the performance of FMD items and FMI items across MACS levels with a steeper slope of changes in FMD items suggests fine motor skills impact cognition.

Task-Related Differences in End-Point Kinematics in School-Age Children with Typical Development.

Understanding whether and how children with typical development adapt their reaches for different functional tasks could inform a more targeted design of rehabilitation interventions to improve upper extremity function in children with motor disabilities. This prospective study compares timing and coordination of a reach-to-drink, reach-to-eat, and a bilateral reaching task in typically developing school-aged children. Average speed, straightness, and smoothness of hand movements were measured in a convenience sample of 71 children, mean age 8.77 ± 0.48 years. Linear mixed models for repeated measures compared the variables by task, phases of the reach, task x phase interactions, and dominant versus non-dominant hands. There were significant main effects for task and phase, significant task x phase interactions (p < 0.05), and a significant difference between the dominant and non-dominant hand for straightness. Hand movements were fastest and smoothest for the reach-to-eat task, and least straight for the bilateral reaching task. Hand movements were also straighter in the object transport phases than the prehension and withdrawal phases. These results indicate that children with typical development change their timing and coordination of reach based on the task they are performing. These results can inform the design of rehabilitation interventions targeting arm and hand function.

Preliminary evidence of an association between spontaneous kicking and learning in infants between 3-4 months of age.

BACKGROUND: The timing and coordination of infant kicking may allow for activities that facilitate learning and cognitive development. OBJECTIVE: This study examined spontaneous kicking and associations with changes in kicking during a learning paradigm in typically developing infants. METHODS: Ten healthy full-term infants participated in two experiments at 3 months of age: spontaneous kicking and the mobile paradigm. The inter-limb, intra-limb, and spatiotemporal parameters during spontaneous kicking were collected by 3D motion capture. Learning was measured in the mobile paradigm where an infant's leg was tethered to an overhead mobile. The mobile offered visual and auditory reinforcement when the infant kicked. Changes in kicking rate indicate learning. Friedman tests were used to determine the dominant inter-/intra-limb kicking patterns. Spearman's rank correlation coefficients were used to assess the correlations between spontaneous kicking and performance in the mobile paradigm. RESULTS: A significant negative correlation (r = -0.72, p = 0.03) was observed between the percentages of unilateral kicking and normalized kicking rate during the extinction phase of the paradigm. There was a trend of positive correlation (r = 0.58, p < 0.10) between dissociated hip-ankle joint coupling and the last three-minute of the acquisition phase of the paradigm. CONCLUSION: Exploratory kicking behaviors elicited by visual and auditory feedback may be related to lower extremity movement control. Enhancing movement experience through appropriate external feedback may be critical in treatment programs to support infant development. Future studies to assess how exploratory motor behaviors contribute to development in motor and other domains are warranted.

Using the PODCI to Measure Motor Function and Parent Expectations in Children With Cerebral Palsy.

OBJECTIVE: Involving parents in the evaluation of their child with cerebral palsy (CP) is associated with enhanced neurodevelopmental outcomes. The pediatric outcomes data collection instrument (PODCI) is a patient-reported outcome measure primarily used to assess motor function following orthopedic surgical intervention or for older children with more independent motor function. The PODCI expectations scale has infrequently been reported in previous studies. This study aims to determine the relationship between parent-reported motor performance using the PODCI and motor capacity assessed by pediatric therapists for young children with CP across all ability levels and to explore the use of the PODCI expectations scale for quantifying therapy-related parent expectations. METHODS: This prospective cohort study included 108 participants with CP, 2 to 8 years of age, gross motor function classification systems (GMFCS) levels I to V. Measures included the PODCI, gross motor function measure (GMFM), and GMFCS. RESULTS: There were moderate (r = 0.513) to large (r = 0.885) relationships between PODCI and GMFM scores. PODCI scores were significantly different across GMFCS levels. Weak, significant relationships (r = -0.28) were found between function expectations scores and measures of function. CONCLUSION: The PODCI, GMFM, and GMFCS provide different, but strongly related, information about the abilities of young children. The GMFM measures motor capacity. Parents report daily function and health-related quality of life for their child using the PODCI. Parent expectations for intervention outcomes may relate to a child's motor function. IMPACT: These study results are consistent with those for older children with greater independent mobility, indicating an opportunity for expanded use of the PODCI for measuring motor performance for younger children with CP across all ability levels. A strategy is provided for using the PODCI expectations scale to quantify parent therapy-related expectations in future research and clinical settings. Therapy-related expectations may relate to child outcomes.

Single-centre parental survey of paediatric rehabilitation services for children with cerebral palsy.

Cerebral palsy (CP) is the most common childhood motor disability. The dose of usual care for rehabilitation therapies is unknown. The purpose of this study was to describe current dosage of rehabilitation services for children with CP recruited from a paediatric hospital system in the USA. 96 children with CP were included in this cross-sectional survey. Parents reported frequency, intensity, time and type of therapy services. Weekly frequency was the most common. Children with CP received 0.9-1.2 hours/month of each discipline in the educational setting and 1.5-2.0 hours/month in the clinical setting, lower than the recommendations for improvements in motor skills.

Job role and stress influence student movement during postpartum haemorrhage simulation: an exploratory study.

Introduction: Postpartum haemorrhage is the leading cause of maternal death. Healthcare simulations are an educational tool to prepare students for infrequent high-risk emergencies without risking patient safety. Efficiency of movement in the simulation environment is important to minimize the risk of medical error. The purpose of this study was to quantify the movement behaviours of the participants in the simulation and evaluate the relationship between perceived stress and movement. Methods: N=30 students participated in 10 high-fidelity medical simulations using an adult patient simulator experiencing a postpartum haemorrhage. The participants completed the State-Trait Anxiety Inventory prior to the simulation to measure perceived stress. Physical movement behaviours included walking around the simulation, time spent at bedside, arm movements, movements without purpose, looking at charts/vitals and total movement. Results: Midwife (MW) students spent significantly more time walking (p=0.004) and looking at charts/vitals (p=<0.001) and significantly less time at bedside (p=<0.001) compared to obstetric (OB) students. The MW students demonstrated significantly more total movements compared to the OB students (p=<0.001). There was a significant, moderate, positive relationship between perceived stress and total movement during the simulation for the MW group (r=0.50, p=0.05). There was a trend for a moderate, positive relationship between perceived stress and total movement during the simulation for the OB group (r=0.46, p=0.10). Conclusions: Physical movement during a simulation varies by job role and is influenced by perceived stress. Improved understanding of physical movement in the simulation environment can improve feedback, training and environmental set-up.

Gene therapy for aromatic L-amino acid decarboxylase deficiency by MR-guided direct delivery of AAV2-AADC to midbrain dopaminergic neurons.

Aromatic L-amino acid decarboxylase (AADC) deficiency is a rare genetic disorder characterized by deficient synthesis of dopamine and serotonin. It presents in early infancy, and causes severe developmental disability and lifelong motor, behavioral, and autonomic symptoms including oculogyric crises (OGC), sleep disorder, and mood disturbance. We investigated the safety and efficacy of delivery of a viral vector expressing AADC (AAV2-hAADC) to the midbrain in children with AADC deficiency (ClinicalTrials.gov Identifier NCT02852213). Seven (7) children, aged 4-9 years underwent convection-enhanced delivery (CED) of AAV2-hAADC to the bilateral substantia nigra (SN) and ventral tegmental area (VTA) (total infusion volume: 80 µL per hemisphere) in 2 dose cohorts: 1.3 × 1011 vg (n = 3), and 4.2 × 1011 vg (n = 4). Primary aims were to demonstrate the safety of the procedure and document biomarker evidence of restoration of brain AADC activity. Secondary aims were to assess clinical improvement in symptoms and motor function. Direct bilateral infusion of AAV2-hAADC was safe, well-tolerated and achieved target coverage of 98% and 70% of the SN and VTA, respectively. Dopamine metabolism was increased in all subjects and FDOPA uptake was enhanced within the midbrain and the striatum. OGC resolved completely in 6 of 7 subjects by Month 3 post-surgery. Twelve (12) months after surgery, 6/7 subjects gained normal head control and 4/7 could sit independently. At 18 months, 2 subjects could walk with 2-hand support. Both the primary and secondary endpoints of the study were met. Midbrain gene delivery in children with AADC deficiency is feasible and safe, and leads to clinical improvements in symptoms and motor function.

Differences in coordination and timing of pre-reaching upper extremity movements may be an indicator of cerebral palsy in infants with stroke: A preliminary investigation.

BACKGROUND: Neonatal stroke is a leading cause of hemiplegic cerebral palsy that occurs around the time of birth. Infants are diagnosed with cerebral palsy when motor impairments become clinically apparent, months or years after the stroke. Tools/methods for identifying high risk or diagnosis of cerebral palsy in infancy are improving. METHODS: We measured spatial and temporal kinematics of pre-reaching upper extremity movements in 2-3 month old infants with neonatal stroke and typical development. We aimed to evaluate the feasibility of applying kinematics in this population and collect preliminary data to explore (1) if asymmetries are present in the infants with neonatal stroke, particularly those with a later diagnosis of cerebral palsy, and (2) to compare differences in the timing and coordination of their movements to infants with typical development, and infants with stroke and no cerebral palsy. Participants were 21 full-term infants, 10 with stroke (4 who later received a cerebral palsy diagnosis) age 72.1 (SD 9.3) days, and 11 typically developing, age 74.3 (SD 9.3) days. FINDINGS: Results showed that infants with stroke and cerebral palsy demonstrated significant asymmetry in the average movement length (p = 0.0089) and hand path length (p = 0.0275) between their involved and uninvolved sides and moved less frequently (p = 0.09) and slower (p = 0.041) than infants with stroke and no cerebral palsy. INTERPRETATION: Results suggest that kinematic analysis might detect asymmetries and motor impairment indicative of hemiplegic cerebral palsy earlier than current assessments and that asymmetry in speed, length and frequency of arm movements may be early indicators. This study is preliminary, limiting interpretation of the results.