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Aim: To evaluate an innovative paediatric neurorehabilitation model in relation to improving quality of neurorehabilitation and reducing length of stay (LOS) for children with acquired brain injury.Method: A process evaluation approach was conducted in line with Medical Research Council evaluation of complex interventions guidance. Analysis was conducted on routinely collected patient data from 2017 to 2018, including LOS and family feedback. Descriptive and inferential statistics were used for quantitative analysis and qualitative data was analysed thematically.Results: Outcomes for 70 children (0-16y, median age 5y, IQR 1-11y, 46 males, 24 females) referred to the service indicated improved function and reduced complexity of need. The mean LOS was 10.6 days compared to baseline mean LOS of 41 days (2011-2012). High satisfaction from the families was recorded; however, ongoing needs and service gaps regarding long-term support were identified.Interpretation: This service model is effective in delivering quality paediatric neurorehabilitation, demonstrating a sustained impact on LOS, and positive patient outcome data and family feedback for this group of patients. What this paper adds Investment in early intensive neurorehabilitation and supported discharge impacts length of stay (LOS) for children with acquired brain injury. Early intensive neurorehabilitation and supported discharge is effective. This is demonstrated by a sustained reduction in LOS, positive patient outcomes, and family feedback. [ABSTRACT FROM AUTHOR]

Aim: To assess the efficacy, safety, and tolerability of oromucosal nabiximols cannabinoid medicine as adjunct therapy for children with spasticity due to cerebral palsy/traumatic central nervous system injury with inadequate response to existing treatment.Method: Overall, 72 patients (mean [SD] age 12y 4mo [3y 1mo], range 8-18y) were randomized at a ratio of 2:1 to receive nabiximols (n=47; 29 males, 18 females) or placebo (n=25; 15 males, 10 females) for 12 weeks (12 sprays/day max. based on clinical response/tolerability). The primary outcome was change from baseline in level of spasticity on a 0 to 10 Numerical Rating Scale (NRS), assessed by the primary caregiver at 12 weeks. Secondary outcomes included additional measures for spasticity, sleep quality, pain, health-related quality of life, comfort, depression, and safety.Results: There was no significant difference in the spasticity 0 to 10 NRS between nabiximols versus placebo groups after 12 weeks. No statistically significant differences were observed for any secondary endpoint. Adverse events were predominantly mild or moderate in severity; however, three cases of hallucinations were reported.Interpretation: Nabiximols was generally well tolerated; however, neuropsychiatric adverse events were observed. No significant reduction in spasticity with nabiximols treatment versus placebo was observed.What This Paper Adds: Oromucosal nabiximols is generally well tolerated by paediatric patients. However, three cases of hallucinations were observed, one of which involved auditory hallucinations and a suicide attempt. Oromucosal nabiximols versus placebo did not reduce cerebral palsy/central nervous system injury-related spasticity. [ABSTRACT FROM AUTHOR]

Aim: To examine the impact of engagement in a self-chosen community-based activity on three relevant body functions (motor, cognitive, and affective) as well as on the performance of the selected activity.Method: An individual-based interrupted time series design with multiple baselines was used. Seven young people (four males, three females) aged 15 to 25 years (median 18y; interquartile range 17-20y) with physical disabilities participated in an 8-week community activity of choice (e.g. swimming, playing piano). Change in three relevant body functions, underpinning the specific chosen activity, including motor (e.g. Functional Reach Test, Trunk Impairment Scale, dynamometers), cognitive and affective (Behavior Assessment System for Children), as well as activity performance (Canadian Occupational Performance Measure) were measured repeatedly, providing individual outcome trajectories. Linear and mixed-effects models were used.Results: Significant improvements in at least one aspect of motor function (6 out of 6), cognition (3 out of 3), affect (5 out of 7), and performance (7 out of 7) were observed. Specifically, the intervention had a moderate to large effect on hyperactivity (1.45, 95% confidence interval [CI] 1.0-1.9) with a smaller effect on anxiety (0.21, 95% CI 0.10-0.32) and inadequacy (0.21, 95% CI 0.02-0.39). Concurrently, a notable effect size for activity performance (4.61, 95% CI 0.76-8.46) was observed. Average change across motor outcomes was substantial (3.7 SDs from baseline), yet non-significant.Interpertation: Findings provide initial evidence of the benefits resulting from participation-based interventions, emphasizing the merit of meaningful 'real-life' young people-engaging therapy.What This Paper Adds: Participation-based interventions can impact body-function level outcomes. Significant improvements in the performance of chosen activities were observed. Significant improvements were also seen in cognitive and affective body functions. Improvements in motor-related outcomes were substantial but not statistically significant. [ABSTRACT FROM AUTHOR]

Aim: To determine whether epilepsy surgery improved health-related quality of life (HRQoL) and whether seizure freedom after surgery mediated the improvement in HRQoL.Method: This multicenter cohort study compared HRQoL after epilepsy surgery to pharmacological management in children with drug-resistant epilepsy (DRE). HRQoL was measured using the Quality of Life in Childhood Epilepsy (QOLCE) questionnaire at baseline and 1-year follow-up. The mediator between treatment type and HRQoL was seizure freedom.Results: Two hundred and thirty-seven patients were recruited (surgery group: n=147 [92 males, 45 females]; pharmacological group: n=90 [53 males, 37 females]). Mean age at seizure onset was 6 years (SD 4y 4mo) in the surgical group and 6 years 1 month (SD 4y) in the pharmacological group. The odds ratio of seizure freedom was higher for the surgery versus pharmacological group (β=4.24 [95% confidence interval {CI}: 2.26-7.93], p<0.001). Surgery had no direct effect on total QOLCE score at 1-year (β=0.24 [95% CI -2.04 to 2.51], p=0.839) compared to pharmacological management, but had an indirect effect on total QOLCE that was mediated by seizure freedom (β=0.92 [95% CI 0.19-1.65], p=0.013), adjusting for baseline total QOLCE score. Surgery had a direct effect on improving social function (p=0.043), and an indirect effect on improving physical function (p=0.016), cognition (p=0.042), social function (p=0.012) and behavior (p=0.032), mediated by seizure freedom.Interpretation: Greater seizure freedom achieved through epilepsy surgery mediated the improvement in HRQoL compared to pharmacological management in children with DRE.What This Paper Adds: Seizure freedom is higher after pediatric epilepsy surgery compared to pharmacologically managed epilepsy. Surgery indirectly improves health-related quality of life (HRQoL) mediated by seizure freedom compared to pharmacological management. Surgery has a direct effect on improving social function relative to pharmacological management. Baseline HRQoL was an important predictor of HRQoL after treatment. [ABSTRACT FROM AUTHOR]

Aim: To assess the responsiveness, concurrent validity, and feasibility of the Dyskinesia Impairment Scale (DIS) in non-ambulatory patients with dyskinetic cerebral palsy (CP).Method: The study is a secondary analysis of data collected in the IDYS trial, a randomized controlled trial on the effects of intrathecal baclofen (ITB). The DIS and Barry-Albright Dystonia Scale (BADS) were conducted at baseline and after 3 months of ITB or placebo treatment. Responsiveness was assessed by comparing the effect sizes and correlation of change after treatment between the DIS and BADS. Concurrent validity was evaluated by assessing the correlations between scales. Feasibility was evaluated for each DIS item by the number of participants who successfully accomplished the item.Results: Thirty-three non-ambulatory patients (9 females, 24 males) with dyskinetic CP (ITB-treated: n=17, mean [SD] age: 14y 1mo [4y 1mo]; placebo-treated: n=16, mean [SD] age: 14y 7mo [4y]) were included in the study. The effect sizes for BADS and DIS were similar in The ITB-treated group (-0.29 and -0.22 respectively). Changes after treatment on the DIS dystonia subscale correlated with changes on the BADS (r=0.64; p<0.001). The DIS dystonia subscale and BADS correlated at baseline and follow-up (r=0.78; p<0.001 and r=0.79; p<0.001). Not all DIS activity items could be performed in this sample of patients.Interpretation: For non-ambulatory patients with dyskinetic CP, the responsiveness of the DIS equalled the responsiveness of BADS. Concurrent validity was adequate. Feasibility for activity items was restricted in patients with severe dyskinetic CP.What This Paper Adds: The Dyskinesia Impairment Scale (DIS) and Barry-Albright Dystonia Scale showed similar responsiveness in non-ambulatory patients with dyskinetic cerebral palsy (CP). No floor or ceiling effect was observed for DIS in non-ambulatory participants. The concurrent validity of DIS was adequate in non-ambulatory participants. Patients with dyskinetic CP in Gross Motor Function Classification System levels IV and V could not perform all DIS activity items. [ABSTRACT FROM AUTHOR]

The aim of this study was to evaluate tolerability of and response to rituximab in children with myelin oligodendrocyte glycoprotein (MOG) antibody-positive relapsing neuroinflammatory disease. This was an observational study of prospectively collected data on 12 consecutive children (eight females, four males; median age at onset 10y 6mo [interquartile range {IQR} 7y 2mo-12y 5mo], median follow-up 2y 1mo [IQR 1y 7mo-2y 6mo]) with central nervous system inflammation and persistent serum MOG immunoglobulin G positivity more than 12 weeks after clinical presentation. Patients received a standardized rituximab treatment protocol. MOG antibody testing was performed following standardized cell-based methods. Median clinical follow-up after rituximab induction was 2 years (IQR 1y 7mo-2y 10mo). The relapse rate in the first 12 months posttreatment was 0 (IQR 0-0). After rituximab, two patients relapsed during B-cell suppression and four showed clinical or radiological disease recurrences at B-cell reconstitution. Mild-to-moderate infusion related adverse events occurred in two patients. Leukopenia developed in seven patients and serum immunoglobulin suppression in five patients with no significant age effect on the risk of their development. None developed severe life-threatening events. Rituximab-induced B-cell suppression was associated with absence of relapses in 10 patients who were MOG-positive with recurrent disease. Rituximab was well tolerated. The most frequent adverse effects were hypogammaglobulinemia and leukopenia. We recommend monitoring of complete blood counts and immunoglobulins in this population. WHAT THIS PAPER ADDS: Rituximab appears to control disease in most anti-myelin oligodendrocyte glycoprotein-positive patients with relapsing neuroinflammatory disease. Rituximab was associated with transitory, mild-to-moderate infusion-related effects. Half of patients treated with rituximab developed leukopenia or hypogammaglobulinemia. No opportunistic infections were observed. [ABSTRACT FROM AUTHOR]

Aim: To investigate long-term development of hand function after repeated botulinum neurotoxin A (BoNT-A) and occupational therapy at a young age.Method: Twenty children with unilateral spastic cerebral palsy (CP) (14 males, six females; median inclusion age 3y 1mo, range 1y 11mo-4y 3mo) participated in this longitudinal study. Ten children received occupational therapy after a randomized controlled trial and 10 repeated BoNT-A plus occupational therapy during 1-year. The Assisting Hand Assessment (AHA) and active supination, assessed the following 3 years. The assessments were compared with data from a reference group to investigate development over time.Results: The improvement in AHA (7.5 AHA units) after BoNT-A plus occupational therapy was maintained at final follow-up. The occupational therapy group, unchanged after 1-year, improved by 5 AHA units (96% confidence interval [CI] 2-10), thus there was no difference between the groups. Median active supination increased in comparison with the reference group. In the BoNT-A/occupational therapy group, 9 out of 10 (97.85% CI 45 115) children improved in active supination. In the occupational therapy group, 7 out of 10 (97.85% CI -2 to 68) children improved in active supination. No correlation between active supination and AHA was found.Interpretation: Bimanual performance achieved after BoNT-A plus occupational therapy was maintained, while it increased by follow-up in the occupational therapy group, suggesting that combined intervention gave earlier access to bimanual skills. Active supination was unrelated to AHA.What This Paper Adds: Children whose bimanual performance improved after botulinum neurotoxin A and/or occupational therapy, maintained skills or progressed during follow-up. Bimanual performance increased with age, similar to a quality-register reference group. Increased active supination after intervention improved at follow-up but was not related to bimanual performance. [ABSTRACT FROM AUTHOR]

Aim: To assess the reliability and to evaluate the responsiveness of both the Jebsen-Taylor Test of Hand Function (JTTHF) and the Box and Block Test (BBT) in children with cerebral palsy (CP).Method: In this retrospective study, the reliability analyses were conducted with paired t-tests considering a short (mean 14d) and a long (mean 120d) time in between two assessment periods. In addition, an intraclass correlation coefficient (ICC) was used to assess the level of congruency. The responsiveness to therapy was conducted with a paired t-test in the whole sample regarding the age, the manual ability level as classified with the Manual Ability Classification System (MACS), and the topography.Results: Our main results confirmed the tests' reliability in a short time period for the JTTHF in both hands and for the BBT on the less affected hand. These results were consistent with the ICC. The responsiveness was confirmed, except on the less affected hand for the JTTHF, with similar results for age, MACS, and topography approach.Interpretation: This study supports the use of the JTTHF and the BBT to examine changes after short-term interventions for children with CP. These results should be interpreted with association to normative values or with a control group when used over long assessment periods.What This Paper Adds: The Box and Block Test (BBT) is reliable in a brief period of assessment in children with cerebral palsy (CP). The Jebsen-Taylor Test of Hand Function (JTTHF) is reliable in a brief period of assessment in children with CP. The JTTHF and BBT are responsive to changes in a brief period of intensive therapy in children with CP. The reliability and responsiveness of the JTTHF and BBT are weak over long assessment periods. [ABSTRACT FROM AUTHOR]

Aim: To test the hypothesis that improvements in gait and function following individualized interdisciplinary interventions consisting of physical therapy, orthotics, spasticity management, and orthopaedic surgery using instrumented gait analysis are superior to 'usual care' in children with cerebral palsy (CP).Method: This was a prospective, single-blind, parallel-group, randomized controlled trial investigating the effectiveness of interventions based on the use of gait analysis. Primary outcome was gait (Gait Deviation Index) and secondary outcomes were walking and patient-reported outcome measures of function, disability, and health-related quality of life. Follow-ups were done at 26 weeks (questionnaires) and at the primary end point of 52 weeks (all outcomes).Results: Sixty participants with CP (39 males, 21 females, mean age 6y 10mo, standard deviation 1y 3mo, range 5y-9y 1mo) in Gross Motor Function Classification System levels I or II, were randomized to interventions with or without gait analysis. No significant or clinically relevant between-group differences in change scores of the primary or secondary outcomes were found. The recommended categories of interventions were dominated by non-surgical interventions and were applied in 36% to 86% of the participants.Interpretation: Interventions using gait analysis were not superior to 'usual care' on gait, walking, or patient-reported outcomes in a sample of relatively young and independently walking children with CP not expected to need surgery.What This Paper Adds: Gait analysis in children with cerebral palsy in Gross Motor Function Classification System levels I or II recommends interdisciplinary interventions. Compliance to interventions recommended after gait analysis was low. No statistically significant advantages were identified for the intervention group versus the control group. [ABSTRACT FROM AUTHOR]

Aim: To evaluate short-term (1y postoperatively; E1) and long-term (at least 4y postoperatively; E2) changes in hamstring muscle-tendon length (MTL) and lengthening velocity after hamstring lengthening in children with bilateral cerebral palsy (CP).Method: Three-dimensional gait analysis was performed in 19 children (16 males, 3 females; 36 limbs; mean age at surgery 9y [SD 3y]; range 6-10y) with flexed knee gait, preoperative ankle dorsiflexion lower than 20 degrees, and CP before bilateral hamstring lengthening (E0), at E1 and E2. Hamstring MTL (normalized by leg length) and velocity were assessed via OpenSim software.Results: MTL increased from E0 to E1 (p=0.004) and decreased from E1 to E2 (p<0.020). Hamstring lengthening velocity did not change. In the subgroup with short, not slow hamstrings, the increase in MTL was maintained at E2.Interpretation: Hamstring lengthening is an efficient procedure to lengthen short and/or slow hamstrings short-term. The desired outcome with maintenance of the postoperative changes in hamstring MTL is only achieved for preoperatively short, not slow hamstrings.What This Paper Adds: Surgical hamstring lengthening can be confirmed via musculoskeletal modelling in OpenSim software. Surgical hamstring lengthening in cerebral palsy does not change hamstring lengthening velocity. Short, not slow hamstrings present a long-lasting muscle-tendon length (MTL) increase after hamstring lengthening. Changes in MTL after hamstring lengthening cannot be maintained for slow hamstrings. MTL does not change after hamstring lengthening for neither short nor slow hamstrings. [ABSTRACT FROM AUTHOR]

Aim: This cross-sectional investigation evaluated whether recurrent botulinum neurotoxin A (BoNT-A) interventions to the medial gastrocnemius have an influence on muscle morphology, beyond Gross Motor Function Classification System (GMFCS) level.Method: A cohort of typically developing children (n=67; 43 males, 24 females; median age 9y 11mo [range 7y 10mo-11y 6mo]), a cohort of children with spastic cerebral palsy (CP) naive to BoNT-A interventions (No-BoNT-A; n=19; 10 males, nine females; median age 9y 3mo [range 8y 5mo-10y 10mo]) and a cohort of children with spastic CP with a minimum of three recurrent BoNT-A interventions to the medial gastrocnemius (BoNT-A; n=19; 13 males, six females; median age 9y 8mo [range 7y 3mo-10y 7mo]) were recruited. Three-dimensional freehand ultrasound was used to estimate medial gastrocnemius volume normalized to body mass and echo-intensity.Results: Normalized medial gastrocnemius volume and echo-intensity significantly differed between the two spastic CP cohorts (p≤0.05), with the BoNT-A cohort having larger alterations. Associations between normalized medial gastrocnemius volume and echo-intensity were highest in the No-BoNT-A cohort, followed by the BoNT-A cohort. Multiple regression analyses revealed that both GMFCS level and BoNT-A intervention history were significantly associated with smaller normalized medial gastrocnemius volume and higher echo-intensity.Interpretation: Recurrent BoNT-A interventions may induce alterations to medial gastrocnemius volume and echo-intensity beyond the natural history of the spastic CP pathology.What This Paper Adds: In spastic cerebral palsy, medial gastrocnemius volumes are smaller and echo-intensities higher compared with typical development. Alterations after botulinum neurotoxin A intervention (BoNT-A) are larger than in no BoNT-A intervention. Gross Motor Function Classification System level and BoNT-A history significantly associate with medial gastrocnemius and echo-intensity alterations. [ABSTRACT FROM AUTHOR]

Aim: To gather data on mycophenolate mofetil (MMF) in paediatric autoimmune/immune-mediated central nervous system (CNS) conditions, focusing on safety and factors that may affect MMF efficacy.Method: Retrospective, multicentre study based on four paediatric neurology centres.Results: Forty-four children were included (30 females, 14 males): 19 had proven/suspected autoimmune encephalitis, 14 had inflammatory demyelinating CNS diseases, and 11 had other autoimmune/immune-mediated CNS conditions. Before MMF, all received first-line immune therapies, and 17 had second-line rituximab and/or cyclophosphamide. MMF was started at a median of 9.5 months from disease onset (range 1-127mo) (median age 9y 4mo, range 1y 5mo-16y 5mo), and was used for median 18 months (range 0.3-73mo). On MMF, 31 patients were relapse-free, whereas eight relapsed (excluding patients with chronic-progressive course). Relapses on MMF were associated with medication weaning/cessation, or with suboptimal MMF dosage/duration. Adverse events of MMF occurred in eight patients: six moderate (gastrointestinal, movement disorder, dermatological) and two severe (infectious).Interpretation: MMF use in paediatric neuroimmunology is heterogeneous, although relatively safe. We have identified factors that may affect MMF efficacy and provide recommendations on MMF usage.What This Paper Adds: Mycophenolate mofetil (MMF) use was heterogeneous with relatively common adverse events, although mostly not severe. MMF treatment reduced median annualized relapse rate, although 20% of patients relapsed on MMF. A high relapse rate pre-MMF and late MMF start were associated with higher probability of relapsing on MMF. Most relapses were associated with suboptimal MMF dosage, short MMF duration, or concurrent medication weaning/discontinuation. [ABSTRACT FROM AUTHOR]

Aim: This study aimed to track alterations in muscle volume for 6 months in children with cerebral palsy (CP) after the first exposure to botulinum neurotoxin A (BoNT-A), a commonly used focal spasticity treatment.Method: Eleven ambulant children (eight males, three females) with spastic CP, mean age 8 years 10 months (SD 3y 1mo) participated. Participants received injections to the affected gastrocnemius. The muscle volume of the gastrocnemius, soleus, tibialis anterior, and hamstrings was measured using magnetic resonance imaging. Muscle volume was normalized to bone length, and changes analysed relative to baseline. Assessments were conducted 1 week before, and 4 weeks, 13 weeks, and 25 weeks after BoNT-A treatment.Results: All children demonstrated positive clinical and functional gains. Muscle volume of the injected gastrocnemius was found to be significantly reduced at 4 weeks (-5.9%), 13 weeks (-9.4%), and 25 weeks (-6.8%). Significant increases in normalized soleus muscle volume were identified at each follow-up, while hamstrings showed significant increase at 4 weeks only.Interpretation: Absolute and normalized muscle volume of the injected muscle reduces after first BoNT-A exposure, and does not return to baseline volume by 25 weeks. Hypertrophy is seen in the soleus up to 25 weeks; the volume of the plantar flexor compartment is stable.What This Paper Adds: Muscle atrophy after first botulinum neurotoxin A (BoNT-A) exposure in children with cerebral palsy is noted. Mild BoNT-A-induced muscle atrophy is still apparent 6 months after BoNT-A exposure. Hypertrophy is evident in soleus after gastrocnemius BoNT-A exposure. Total plantarflexor volume is unchanged. [ABSTRACT FROM AUTHOR]

Aim: To investigate the responsiveness of the ACTIVLIM-CP questionnaire after two evidence-based interventions for children with cerebral palsy (CP).Method: Seventy-five children with CP either participated in an intensive motor-skill learning intervention (hand-arm bimanual intensive therapy including lower extremities [HABIT-ILE], n=47) or received botulinum neurotoxin-A (BoNT-A) injection(s) into lower extremities combined with conventional physical therapy (n=28). All children were assessed three times: at baseline (T0 ; before HABIT-ILE/the day of BoNT-A injection), at T1 (last day of HABIT-ILE/6wks after BoNT-A injection), and at follow-up (T2 ; 3-4mo after the beginning of intervention). Parents completed ACTIVLIM-CP and three other activity questionnaires. Responsiveness was analysed using group (based on intervention), subgroup (based on gross motor function level), and individual approaches.Results: For the HABIT-ILE group, significant improvements in ACTIVLIM-CP were observed for the T0 -T1 period (p<0.001) but not for the T1 -T2 period. No significant changes were found in the BoNT-A group during assessments (p=0.84). In the subgroup analysis for the HABIT-ILE group (T0 -T1 ), greater changes were demonstrated for children in Gross Motor Function Classification System levels III and IV (p<0.001, effect size=1.36). The individual approach was congruent with the group approach.Interpretation: ACTIVLIM-CP demonstrated high responsiveness after HABIT-ILE, showing that this scale may be used to investigate global activity performance in clinical trials focusing on improving daily life activities.What This Paper Adds: Good responsiveness of ACTIVLIM-CP questionnaire during intensive motor-skill learning intervention. Higher responsiveness for children in Gross Motor Function Classification System (GMFCS) levels III and IV versus I and II after intensive intervention. ACTIVLIM-CP is useful to identify children improving their performance after botulinum neurotoxin-A injection. [ABSTRACT FROM AUTHOR]

Aim: We explored relationships of school-based physical therapy to standardized outcomes of students receiving physical therapy.Method: Using a practice-based evidence research design, School Function Assessment (SFA) outcomes of 296 students with disabilities (mean age 7y 4mo [standard deviation 2y]; 166 males, 130 females), served by 109 physical therapists, were explored. After training, therapists completed 10 SFA scales on students at the beginning and end of the school year. Therapists collected detailed weekly data on services (activities, interventions, types, student participation) using the School-Physical Therapy Interventions for Pediatrics (S-PTIP) system. Stepwise linear regressions were used to investigate S-PTIP predictors of SFA outcomes.Results: Predictors of SFA section outcomes varied in strength, with the coefficient of determination (R2 ) for each outcome ranging from 0.107 to 0.326. Services that correlated positively with the SFA outcomes included mobility, sensory, motor learning, aerobic/conditioning, functional strengthening, playground access interventions, and higher student participation during therapy (standardized β=0.11-0.26). Services that correlated negatively with the SFA outcomes included providing services within student groups, within school activity, with students not in special education, during recreation activities, and with positioning, hands-on facilitation, sensory integration, orthoses, and equipment interventions (standardized β=-0.14 to -0.22).Interpretation: Consideration of outcomes is prudent to focus services. Overall results suggest we should emphasize active mobility practice by using motor learning interventions and engaging students within therapy sessions.What This Paper Adds: No specific interventions predicted positively on all School Function Assessment (SFA) outcomes. Active movement practice seems related to overall better SFA outcomes. Active mobility practice improved SFA participation, mobility, recreation, and activities of daily living. Engaging students in therapy activities and interventions improved outcomes. [ABSTRACT FROM AUTHOR]

Aim: To assess long-term safety and efficacy of stiripentol as an antiepileptic medication for people with Dravet syndrome.Method: A prospective, observational open-label study (2003-2015) of the efficacy and long-term safety of stiripentol in patients with Dravet syndrome and ongoing seizures. Frequency of generalized tonic-clonic seizures, focal seizures, status epilepticus, and adverse events were recorded.Results: Forty-one patients started stiripentol, with median age at enrolment 5 years 7 months (range 11mo-22y) and median duration of treatment 37 months (range 2-141mo). Twenty out of 41 patients had greater than or equal to 50% long-term reduction in frequency of generalized tonic-clonic seizures. Frequency of focal seizures was decreased by greater than or equal to 50% in 11 out of 23 patients over the long-term. Frequency of status epilepticus was decreased by 50% or more in 11 out of 26 patients. The most common adverse events were anorexia, weight loss, sedation, and behavioural changes. One patient had worsening of absence and myoclonic seizures. Another developed recurrent pancreatitis on concurrent valproate.Interpretation: Stiripentol improves long-term seizure frequency in approximately 50% of patients with Dravet syndrome, when used as part of unrestricted polytherapy. Long-term use appears safe. In more than 40% of patients, episodes of status epilepticus markedly decrease after stiripentol initiation. What this paper adds Frequency of status epilepticus is reduced in 40% of patients with Dravet syndrome after stiripentol initiation. Stiripentol is effective for generalized tonic-clonic and focal seizures. Stiripentol can be safely used with a range of antiepileptic drugs. [ABSTRACT FROM AUTHOR]

Aim: This study examined the effectiveness of the Pathways and Resources for Engagement and Participation (PREP) intervention in improving the participation of adolescents in community-based activities.Method: Twenty-eight adolescents (14 males, 14 females), aged 12 to 18 years (mean 14y 6mo, SD 21.6mo) with moderate physical disabilities participated in a 12-week PREP intervention. An interrupted time series design with multiple baselines was employed, replicating the intervention effect across three chosen activities and all participants. An occupational therapist worked individually with adolescents and parents to identify and implement strategies to remove environmental barriers that impede participation in selected activities. Activity performance was repeatedly measured using the Canadian Occupational Performance Measure (COPM) at baseline, intervention, and follow-up (20wks). For each activity, the trajectory representing change in performance was analyzed descriptively. Segmented regression combined with a mixed-effects modeling approach was used to statistically estimate the overall effectiveness of the intervention within and across 79 activities.Results: A statistically significant improvement (B=2.08, p<0.001) was observed across all activities, 59 per cent of which also indicated a clinically significant change of more than 2 points on the COPM scale. Levels of performance were maintained during follow-up with an additional increase of 0.66 points on the COPM scale (t=3.04, p=0.004). Intervention was most effective for males and those with a higher number of functional issues.Interpretation: Findings illustrate that participation can be improved by changing the environment only. Such evidence further supports emerging therapeutic approaches that are activity-based, goal-oriented, and ecological in nature.What This Paper Adds: Environment-based intervention strategies, guided by the Pathways and Resources for Engagement and Participation, are effective in improving and maintaining adolescent participation. Intervention was most effective for males and those with a higher number of functional issues. The study design serves as an example for future pragmatic studies accounting for individual-based changes and contexts. [ABSTRACT FROM AUTHOR]

Aim: To explore the evolution of self-care and functional mobility after multilevel surgery in children and adolescents with spastic diplegic cerebral palsy and to identify factors affecting these outcomes.Method: Thirty-four participants (22 males, 12 females) were evaluated before surgery, and at 2 months, 6 months, 1 year, 18 months, and 2 years after surgery. Self-care was assessed with the Pediatric Evaluation of Disability Inventory Dutch edition. The Mobility Questionnaire47 (MobQues47) and Functional Mobility Scale (FMS) were used to measure functional mobility.Results: All outcomes revealed a significant decrease 2 months after single-event multilevel surgery (SEMLS) (p-value between <0.001 and 0.02) followed by a significant increase at 6 months (p<0.001 and p=0.045). Between 6 months and 1 year, a significant increase was also revealed for Mobques47 (p<0.001), FMS (p≤0.008), and the Pediatric Evaluation of Disability Inventory Functional Skills Scale (PEDI-FSS) (p=0.001). Improvement continued until 18 months for the PEDI-FSS. Initial score, initial muscle strength, Gross Motor Function Classification System level, age, and number of surgical interventions significantly influenced time trends for self-care and/or functional mobility.Interpretation: Most preoperative scores are regained at 6 months after SEMLS. Further improvement is seen until 18 months. Participants with a higher functional level before surgery will temporarily lose more than participants with lower initial functional ability, but they also fast regain their function.What This Paper Adds: Self-care and functional mobility decrease significantly in the first months after single-event multilevel surgery (SEMLS). Six months after SEMLS most preoperative scores are regained. Impact of SEMLS is more pronounced for functional mobility than for self-care. Muscle strength and functionality at baseline are important influencing factors on the evolution after SEMLS. [ABSTRACT FROM AUTHOR]

Aim: To systematically review evidence for pharmacological/neurosurgical interventions for managing dystonia in individuals with cerebral palsy (CP) to inform a care pathway.Method: Searches included studies with a minimum of five participants with dystonia in CP receiving oral baclofen, benzodiazepines (clonazepam, diazepam, lorazepam), clonidine, gabapentin, levodopa, trihexyphenidyl, botulinum toxin, intrathecal baclofen (ITB), or deep brain stimulation (DBS). Evidence was classified according to American Academy of Neurology guidelines.Results: Twenty-eight articles underwent data extraction: one levodopa, five trihexyphenidyl, three botulinum toxin, six ITB, and 13 DBS studies. No articles for oral baclofen, benzodiazepines, clonidine, or gabapentin met the inclusion criteria. Evidence for reducing dystonia was level C (possibly effective) for ITB and DBS; level C (possibly ineffective) for trihexyphenidyl; and level U (inadequate data) for botulinum toxin.Interpretation: For dystonia reduction, ITB and DBS are possibly effective, whereas trihexyphenidyl was possibly ineffective. There is insufficient evidence to support oral medications or botulinum toxin to reduce dystonia. There is insufficient evidence for pharmacological and neurosurgical interventions to improve motor function, decrease pain, and ease caregiving. The majority of the pharmacological and neurosurgical management of dystonia in CP is based on clinical expert opinion.What This Paper Adds: Intrathecal baclofen and deep brain stimulation are possibly effective in reducing dystonia. Current evidence does not support effectiveness of oral medications or botulinum toxin to reduce dystonia. Evidence is inadequate for pharmacological/neurosurgical interventions impact on improving motor function, pain/comfort, and easing caregiving. The majority of the care pathway rests on expert opinion. [ABSTRACT FROM AUTHOR]