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Most Human African Trypanosomiasis (HAT) control programmes in areas endemic for Trypanosoma brucei gambiense rely on a strategy of active mass screening with the Card Agglutination Test for Trypanosomiasis (CATT)/T. b. gambiense. We evaluated the performance, stability and reproducibility of the CATT/T. b. gambiense on blood-impregnated filter papers (CATT-FP) in Kajo-Keji County, South-Sudan, where some areas are inaccessible to mobile teams. The CATT-FP was performed with a group of 100 people with a positive CATT on whole blood including 17 confirmed HAT patients and the results were compared with the CATT on plasma (CATT-P). The CATT-FP was repeated on impregnated filter papers stored at ambient and refrigerated temperature for 1, 3, 7 and 14 days. Another 82 patients with HAT, including 78 with a positive parasitology, were tested with the CATT-FP and duplicate filter paper samples were sent to a reference laboratory to assess reproducibility. The CATT-FP was positive in 90 of 99 patients with HAT (sensitivity: 91%). It was less sensitive than the CATT-P (mean dilution difference: -2.5). There was no significant loss of sensitivity after storage for up to 14 days both at ambient and cool temperature. Reproducibility of the CATT-FP was found to be excellent (kappa: 0.84). The CATT-FP can therefore be recommended as a screening test for HAT in areas where the use of CATT-P is not possible. Further studies on larger population samples in different endemic foci are still needed before the CATT-FP can be recommended for universal use. [ABSTRACT FROM AUTHOR]

Cysticercosis is an infestation of Cysticercus cellulosae. When it occurs in the brain, chronic neurological complications can ensue, most commonly seizures. Neurocysticercosis is usually diagnosed by neuroimaging, a technique not available in most endemic countries. Hence immunological tests are valuable for diagnosis and epidemiological surveys. We evaluated the suitability of paper for storing blood and cerebrospinal fluid (CSF) until subsequent testing by enzyme-linked immunosorbent assay (ELISA), by testing whole blood samples on filter paper from 305 patients and CSF samples from 117 patients stored on ordinary white typing paper and on filter paper. Optimal preservation of biological samples is achieved when whole blood is stored on filter paper, CSF on white paper, and when samples are frozen within 1 week after collection. Our results could improve diagnostic capabilities and facilitate epidemiological surveys in endemic countries where immunodiagnostic tests cannot be rapidly performed because of inadequate laboratory infrastructure. [ABSTRACT FROM AUTHOR]

Aim: To determine if pain is associated with 12-month incidence of mood affective disorders (MAD) in adults with cerebral palsy (CP).Method: Data from Optum Clinformatics® Data Mart (2013-2016) were used for this retrospective cohort study. Diagnostic codes were used to identify adults (≥18y) with CP, incident cases of MAD, and covariates (other neurodevelopmental conditions, sleep disorders, arthritis). Pain (any type, location) was identified between 1st October 2014 and 30th September 2015. The pain group was divided into new or consistent pain if they had a history of pain (i.e. consistent) in the 12 months before their first pain claim date. Crude incidence rates of MAD (expressed per 100 person-years) were estimated. Cox regression was used to estimate hazard ratio (95% confidence interval [CI]) of MAD after adjusting for covariates.Results: Adults that had new pain (n=859; incidence rate=15.5) or consistent pain (n=1303; incidence rate=17.9) had greater crude incidence rate of MAD compared to adults without pain (n=3726; incidence rate=5.9). The elevated rate of MAD remained after adjusting for covariates, for new pain (hazard ratio=2.4; 95% CI=1.9-3.0) and consistent pain (hazard ratio=2.1; 95% CI=1.7-2.7).Interpretation: Pain is associated with greater incidence of MAD in adults with CP. This association remained after accounting for potential confounding factors.What This Paper Adds: What this paper adds Pain was associated with higher 12-month incidence of mood affective disorders (MAD). The 12-month MAD incidence was similar between new and consistent pain groups. The MAD incidence remained higher adjusting for neurodevelopmental comorbidities, sleep disorders, and arthritis. [ABSTRACT FROM AUTHOR]

Aim: To validate the Whitney Comorbidity Index (WCI), which was recently developed to monitor disease status for adults with cerebral palsy (CP), and to identify WCI scores associated with an increased mortality risk using a representative sample of adults with CP.Method: Data from 2016 to 2018 were used from a random 20% sample from the fee-for-service Medicare database for this retrospective cohort study. The WCI was examined as unweighted (WCIunw ) and weighted (WCIw ) among adults at least 18 years old with CP. Cox regression models were developed with mortality as the outcome after adjusting for demographics. A concordance statistic (C-statistic) of at least 0.70 was considered as showing sufficient validity. The hazard ratio of mortality for each WCI score was estimated. Secondary analyses were performed for subgroups with co-occurring epilepsy and/or intellectual disabilities.Results: For the entire group (n=16 728) and subgroups, the WCI showed sufficient validity (C-statistic 0.73-0.81). For the entire group, the mortality rate was elevated for a score of 1 compared with 0 from the WCIunw (hazard ratio 3.06; 95% confidence interval [CI] 1.52-6.17) and WCIw (hazard ratio 4.08; 95% CI 1.69-9.85), and became larger with each WCI score. Results were similar for the subgroups.Interpretation: The WCI is a valid marker for health/disease status for adults with CP. Several WCI score thresholds were identified to assist in clinical decision making for preventive medicine and intervention implementation. What this paper adds The Whitney Comorbidity Index (WCI) is valid among 16 728 adults with CP. The WCI is valid for those with co-occurring epilepsy and/or intellectual disabilities. Thresholds of the WCI score were identified to assist clinical decision making. [ABSTRACT FROM AUTHOR]

Aim: To determine if walking ability and presence of intellectual disability and epilepsy change from childhood to 50 years of age in individuals with cerebral palsy (CP), and if such changes are related to age, sex, or CP subtype.Method: This was a population-based follow-up study of 142 adults born from 1959 to 1978 (82 males, 60 females; mean age 48y 4mo, range 37-58y; 44% unilateral, 35% bilateral, 17% dyskinetic, and 4% ataxic CP) listed in the CP register of western Sweden. We compared childhood data with a follow-up assessment in 2016.Results: At follow-up, walking ability had changed significantly (p<0.001). The proportion of participants walking without aids had decreased from 71% to 62%, and wheelchair ambulation increased from 18% to 25%. Walking ability was related to subtype (p=0.001), but not to age, sex, pain, fatigue, or body mass index. The proportion classified as having intellectual disability had increased from 16% to 22% (p=0.039) and the proportion with epilepsy from 9% to 18% (p=0.015). Of those with childhood epilepsy, 46% were seizure-free without medication.Interpretation: Walking ability and the presence of intellectual disability and epilepsy had changed significantly since childhood. Life-long access to specialized health care is warranted for re-evaluation of impairments, treatment, and assistance. What this paper adds Changes in impairments in individuals with cerebral palsy (CP) over time are related to CP subtype. After 50 years, walking ability in CP may have deteriorated or improved. Intellectual disability in CP may not always be detected in early childhood assessments. Epilepsy in CP may develop after childhood or may be outgrown. [ABSTRACT FROM AUTHOR]

Aim: We explored the psychometric properties of the recently developed Tyneside Pegboard Test (TPT) for unimanual and bimanual dexterity in children with unilateral cerebral palsy (CP) and investigated the impact of sensorimotor impairments on manual dexterity.Method: In this cross-sectional study, the TPT was assessed in 49 children with unilateral CP (mean age 9y 8mo, SD 1y 11mo, range 6-15y; 30 males, 19 females; 23 with right unilateral CP). All participants additionally underwent a standardized upper limb evaluation at body function and activity level. We investigated: (1) known-group, concurrent, and construct validity and (2) impact of sensorimotor impairments including spasticity, grip force, stereognosis, and mirror movements using analysis of covariance, Spearman's rank correlation (r), and multiple linear regression (R2 ) respectively.Results: TPT outcomes significantly differed according to the Manual Ability Classification System (p<0.001, known-group validity). Relationships were found between the unimanual TPT tasks and the Jebsen-Taylor Hand Function Test (r=0.86-0.88, concurrent validity). Bimanual TPT tasks were negatively correlated with the Assisting Hand Assessment, ABILHAND-Kids, and Children's Hand-use Experience Questionnaire (r=-0.38 to -0.78, construct validity). Stereognosis was the main determinant influencing all tasks (p<0.001, R2 =37-50%). Unimanual dexterity was additionally determined by grip strength (p<0.05, R2 =8-9%) and mirror movements in the more impaired hand (p<0.05, R2 =4-8%). Bimanual dexterity was also explained by mirror movements in the more impaired hand (p<0.01, R2 =10-16%) and spasticity (p=0.04, R2 =5%).Interpretation: The TPT is a valid test to measure unimanual and bimanual dexterity in unilateral CP. The results further emphasize the importance of somatosensory impairments in children with unilateral CP. What this paper adds The Tyneside Pegboard Test is valid for measuring unimanual and bimanual dexterity in unilateral cerebral palsy. Children with poorer manual ability show worse unimanual and bimanual dexterity. Stereognosis is the main predictor of both unimanual and bimanual dexterity. Stronger mirror movements in the more impaired hand result in worse bimanual dexterity. [ABSTRACT FROM AUTHOR]

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]

Aims: Patients surviving an acute myocardial infarction (AMI) are at risk of developing symptomatic heart failure (HF) or premature death. We hypothesized that sacubitril/valsartan, effective in the treatment of chronic HF, prevents development of HF and reduces cardiovascular death following high-risk AMI compared to a proven angiotensin-converting enzyme (ACE) inhibitor. This paper describes the study design and baseline characteristics of patients enrolled in the Prospective ARNI vs. ACE inhibitor trial to DetermIne Superiority in reducing heart failure Events after Myocardial Infarction (PARADISE-MI) trial.Methods and Results: PARADISE-MI, a multinational (41 countries), double-blind, active-controlled trial, randomized patients within 0.5-7 days of presentation with index AMI to sacubitril/valsartan or ramipril. Transient pulmonary congestion and/or left ventricular ejection fraction (LVEF) ≤40% and at least one additional factor augmenting risk of HF or death (age ≥70 years, estimated glomerular filtration rate <60 mL/min/1.73 m2 , diabetes, prior myocardial infarction, atrial fibrillation, LVEF <30%, Killip class ≥III, ST-elevation myocardial infarction without reperfusion) were required for inclusion. PARADISE-MI was event-driven targeting 708 primary endpoints (cardiovascular death, HF hospitalization or outpatient development of HF). Randomization of 5669 patients occurred 4.3 ± 1.8 days from presentation with index AMI. The mean age was 64 ± 12 years, 24% were women. The majority (76%) qualified with ST-segment elevation myocardial infarction; acute percutaneous coronary intervention was performed in 88% and thrombolysis in 6%. LVEF was 37 ± 9% and 58% were in Killip class ≥II.Conclusions: Baseline therapies in PARADISE-MI reflect advances in contemporary evidence-based care. With enrollment complete PARADISE-MI is poised to determine whether sacubitril/valsartan is more effective than a proven ACE inhibitor in preventing development of HF and cardiovascular death following AMI. [ABSTRACT FROM AUTHOR]

Aim: To evaluate if autism symptoms and diagnoses are more common in children with neurofibromatosis type 1 (NF1) than in typically developing children, to which levels, and to determine if co-occurring attention-deficit/hyperactivity disorder (ADHD) symptomatology accounts for this increase.Method: We searched hospital electronic medical records (EMR) for International Classification of Diseases, 10th Revision NF1 and co-occurring diagnoses codes. We recruited a subsample of 45 children (mean age 9y 2mo; SD 2y 7mo; range 5-12y; 22 males, 23 females) and collected parental reports of autism symptomatology, adaptive behavior, and behavioral problems that were compared to those of 360 age- and sex-matched controls from the Simons Simplex Collection (SSC) with autism spectrum disorder (ASD; SSC-ASD) or typically developing (SSC-TD).Results: The EMR search identified 968 children with NF1; 8.8% had ADHD and 2.1% had ASD co-occurring diagnoses. In the subsample, the mean autism scale score for participants with NF1 was below cut-off for significant autism symptoms. Participants with NF1 had significantly more autism and behavioral symptoms than SSC-TD participants, and significantly less than SSC-ASD participants, with one exception: ADHD symptom levels were similar to those of SSC-ASD participants. In analyses that controlled for internalizing, ADHD, and communication scores, the difference in autism symptom levels between participants with NF1 and typically developing controls disappeared almost entirely.Interpretation: Our results do not support an association between NF1 and autism, both at the symptom and disorder levels.What This Paper Adds: Diagnoses of attention-deficit/hyperactivity disorder (ADHD) were more common in children with neurofibromatosis type 1 (NF1) than in the general child population. Diagnoses of autism spectrum disorder were no more common in children with NF1 than in the general child population. Increases in autism symptoms did not reach clinically significant thresholds. Co-occurring ADHD symptoms accounted for increased autism questionnaire scores. Adaptive behavior in participants with NF1 showed normal socialization but lower communication proficiency. [ABSTRACT FROM AUTHOR]

Objective: There is an urgent need to test and incorporate new molecules with promising efficacy and novel mode of action to control insecticide-resistant mosquito vectors for disease control. We tested a new compound, clothianidin (SumiShield 50 WG), for its efficacy as an indoor residual spray (IRS) for the control of pyrethroid-resistant Anopheles culicifacies (Diptera: Culicidae) in comparison with pirimiphos methyl (Actellic CS) as a positive control.Methods: Ten villages were selected, five each for IRS with clothianidin (300 mg AI/m2 ) and pirimiphos methyl (1000 mg AI/m2 ) in Almatti Dam catchment area in Karnataka state, India. Entomological parameters were monitored in these sprayed villages using standard methods. Assessment of quality of spray was performed by analysing the insecticide content in the filter paper samples collected from sprayed houses. Perceptions of spray men and inhabitants were recorded post-spray on safety of these molecules.Results: The mean applied to target ratio of content was 1.7 (n = 29) for clothianidin and 1.8 (n = 50) for pirimiphos methyl on filter paper samples analysed. Residual activity (≥80% mortality in exposed mosquitoes) after 24 h post-exposure of SumiShield WG was 5 months and increased to 6 months when the holding period was extended to 120 h and that of Actellic CS was 3 months at 24-h holding period and extended to 4 months at 120-h extended holding period. The mean densities of An. culicifacies in both arms fell drastically post-spray. In light trap collections, density of mosquitoes collected indoors was lower than outdoors in both arms indicating effectiveness of IRS. SumiShield WG was more efficacious in reducing the per-structure density than Actellic CS. The proportion of nulliparous mosquitoes was higher than that of parous mosquitoes during post-spray collections in both arms. The majority of adverse events reported were transitory and subsided without medication.Conclusion: Indoor residual spraying with SumiShield WG was found effective, operationally feasible and safe, and it is effective for up to 6 months. [ABSTRACT FROM AUTHOR]

Aim: To investigate the efficacy of waiting room media for reducing anxiety and increasing satisfaction at a paediatric rehabilitation hospital.Method: In this clustered, parallel, randomized controlled trial, 310 young people with disabilities (age range 5-19y) and their parents attending outpatient clinics were assigned to interactive media (n=113), a silent nature video (n=97), or media-free comparison (n=100) groups. Young person and parent anxiety was reported using the State-Trait Anxiety Inventory (STAI) on arrival and after 10 minutes in the waiting space. Questionnaires measured young person, parent, and staff satisfaction.Results: Young people exposed to interactive media reported a postexposure state anxiety that was 1.1 raw points (2.7 standardized points) lower on the STAI than the comparison group (95% confidence interval [CI] -1.9 to -0.22). There was no difference in postexposure state anxiety between the passive media and comparison groups (95% CI -0.64 to 1.1). Parents' state anxiety did not differ between conditions, but interactive media were associated with greater satisfaction (p=0.009). Of 120 staff, 119 reported that interactive media improved the clinic experience for families.Interpretation: Interactive media designed for accessible, hands-free play mitigate waiting anxiety and increases satisfaction. This paper provides evidence to guide design and decision-making around the use of interactive media in health care spaces.What This Paper Adds: Interactive media reduced preclinic waiting anxiety for young people with disabilities. Interactive media were accessible to young people with a range of mobility. Interactive media increased parental and staff satisfaction in the clinic. Guidelines for the design of hands-free, inclusive interactive media for health care facilities are presented. [ABSTRACT FROM AUTHOR]

Aim: To investigate the utility of the Insight Inventory (a structured clinical inventory completed by caregivers) for assessment of children with cerebral visual impairment; and to investigate effectiveness of tailored habilitational strategies derived from the responses to the Insight Inventory.Method: Fifty-one eligible children (26 males, 25 females; mean age 9y 5mo, SD 3y, range 5-16y) were recruited from Great Ormond Street Hospital, London. They underwent baseline assessment including neuro-ophthalmological and neuropsychological evaluations, and parent- and child-reported ratings on a questionnaire-based measure of quality of life. Parents also completed the Insight Inventory. On the basis of responses to the Inventory, families received individualized habilitational strategies. Follow-up assessments 6 months later included repeating the Insight Inventory and quality of life questionnaires.Results: Correlations were found between the Insight Inventory and the Wechsler Intelligence Scale for Children, Fourth Edition, the Beery-Buktenica Test of Visual-Motor Integration, and the Benton Facial Recognition Test, suggesting that the Insight Inventory is an effective tool to estimate visual-perceptual difficulties. At 6 months follow-up, caregiver reports indicated significant improvements in the quality of life of children below the age of 12 years.Interpretation: The Insight Inventory is a simple questionnaire which covers practical aspects of cognitive visual function in everyday life. It provides in-depth information about the aspects that children struggle with. It can also guide programmes of individualized habilitation strategies, which may enhance the quality of life of younger children.What This Paper Adds: Questionnaire scores demonstrate biologically plausible correlations with formal neuropsychological tests of visual function. After administration of matched practical habilitational strategies, younger children showed improvement in quality of life and functional vision scores. [ABSTRACT FROM AUTHOR]

Aim: To analyse the development of motor skill and executive function in school-aged children with and without developmental coordination disorder (DCD).Method: Using a longitudinal design, 186 children (86 males, 100 females) aged 6 to 11 years at Time 1 were tested over a 2-year period, 52 of whom were diagnosed with DCD at Time 1 (27 males, 25 females; mean age 8y 5mo, SD 1y 6mo) using DSM-5 criteria. The McCarron Assessment of Neuromuscular Development assessed motor status at Time 1 and at 2-year follow-up (Time 2). Executive function was assessed using a well-validated measure, the Groton Maze Learning Test.Results: The DCD cohort at Time 1 had moderate incidence of executive function deficit (41%). Most importantly, at a group level, children with persisting DCD (across Times 1 and 2) also showed significantly lower levels of executive function than children with typical motor development at both time points. At an individual level, around 26% of children in this group had persisting executive function deficits relative to normal ranges of performance.Interpretation: Children with persisting DCD are at significant risk of executive function issues. The combination of motor and cognitive issues as a potential risk factor in the longer-term development of children is discussed.What This Paper Adds: Around half of children initially diagnosed with developmental coordination disorder (DCD) had the same diagnosis at 2-year follow-up. 41% of children with DCD have impaired executive function. Children with persisting DCD show poorer executive function than those with typical motor development or remitting DCD. [ABSTRACT FROM AUTHOR]

Aim: To evaluate the properties of the Infant Motor Activity Log (IMAL), a caregiver-report for frequency and quality of use of more affected upper extremity in infants with neurological and functional impairments.Method: This was a prospective cohort study of 66 children (34 females, 32 males) aged 6 to 24 months (mean age [SD] 13.7mo [5.3]) with neurological and functional impairments and a confirmed cerebral palsy diagnoses after 2 years, and 51 age-matched typically developing children. The IMAL was administered at baseline and 4 weeks later. Typically developing infants were tested with randomly assigned 'more affected' upper extremity. Psychometric properties were evaluated using Spearman's correlation coefficient, Cronbach's alpha, and Jonckheere-Terpstra tests.Results: In the children with impairments, the IMAL showed internal consistency (alpha≥0.88) for the How Well Scale (HWS) and How Often Scale (HOS). Test-retest reliability was 0.64 (HOS) and 0.70 (HWS), demonstrating stability over time. Correlation with Bayley Scales of Infant and Toddler Development, Third Edition more affected arm raw scores were 0.70 (HOS) and 0.72 (HWS) (p<0.001) demonstrating construct validity. Both scale scores decreased with increasing Gross Motor Function Classification System and Mini-Manual Ability Classification System (p<0.001) levels, supporting discriminative validity. Discrimination between typically developing infants and infants with impairments was high (HWS: area under the receiver operating characteristic curve [AUC] 0.96, 95% confidence interval [CI] 0.94-0.99 and HOS AUC=0.95, CI 0.92-0.99).Interpretation: The IMAL is a valid and reliable discriminative caregiver measure of upper limb performance and may complement measures of capacity in infants with neurological and functional impairments.What This Paper Adds: The Infant Motor Activity Log (IMAL) is a valid and reliable measure of caregiver perception of upper limb function. The IMAL fills a measurement gap for infant motor performance in children with impairments. The IMAL discriminates among motor function levels. [ABSTRACT FROM AUTHOR]

Aim: To describe the trajectory of clinical signs in children who developed human immunodeficiency virus encephalopathy (HIVE) after starting early antiretroviral therapy (ART).Method: This was a retrospective case-cohort description of HIVE among Cape Town participants from the Children with HIV Early AntiRetroviral treatment (CHER) trial. Criteria for HIVE diagnosis were at least two of: (1) acquired central motor deficit, (2) impaired brain growth, and (3) failure to attain or loss of developmental milestones in the absence of an alternative aetiology.Results: Of 133 surviving participants who initiated ART at a median age of 9 weeks and who were followed until a median age of 6 years, 20 (12%) developed HIVE at a median age 31 months (interquartile range 19-37). In these, the first neurological deterioration was noticed at a median age of 19 months, when 16 were on ART and nine had undetectable HIV viral load for a median of 12 months. Signs of upper motor neurons were present in 18, of whom 12 resolved and four had persistent spastic diplegia; 19 had motor delay, of whom 14 resolved; 12 had language delay, of whom 11 resolved; and 16 had impaired brain growth, of whom only five recovered. For the 16 participants already on ART at HIVE diagnosis, regimens were not altered in response to diagnosis.Interpretation: HIVE may occur despite early ART initiation and virological suppression and then resolve on unchanged ART, most likely as intrathecal inflammation subsides.What This Paper Adds: Despite suppressive antiretroviral therapy, children can develop human immunodeficiency virus encephalopathy, The most common manifestations are motor deficits and impaired brain growth. Most experience improvement, with many resolving without additional intervention. [ABSTRACT FROM AUTHOR]

Aim: To examine the efficacy of Hand-Arm Bimanual Intensive Therapy (HABIT) on daily functioning, unimanual dexterity, and bimanual performance of children with bilateral cerebral palsy (CP) compared with customary care.Method: Forty-one children with bilateral CP, aged 4 to 16 years, classified in levels I to III of the Manual Ability Classification System, were randomly assigned to HABIT (90h) (n=21) or to customary care (4.5h) (n=20). Participants' daily functioning (Pediatric Evaluation of Disability Inventory [PEDI], Canadian Occupational Performance Measure [COPM]), unimanual dexterity (Jebsen-Taylor Test of Hand Function, Box and Blocks Test [BBT]), and bimanual performance (Both Hands Assessment) were assessed pre-, post-, and 6 months after the intervention. Linear mixed-effects models were used for inferential analysis.Results: Children participating in HABIT showed greater improvements in daily functioning (COPMperformance : χ12 =9.50, p<0.01; COPMsatisfaction : χ12 =5.07, p<0.05; PEDIfunctional skills : χ12 =6.81, p<0.01; PEDIcaregiver assistance : χ12 =6.23, p<0.05) and in the dexterity of the dominant hand (BBT: χ12 =3.99, p<0.05) compared with children maintaining customary care. Group or time effects did not explain any variance in bimanual performance or in the dexterity of the non-dominant hand.Interpretation: HABIT may be beneficial for children with bilateral CP, with benefits evidenced for daily functioning outcomes.What This Paper Adds: Hand-Arm Bimanual Intensive Therapy (HABIT) improved daily functioning of children with bilateral cerebral palsy (CP). Bimanual performance, measured by the Both Hands Assessment, did not change after HABIT in children with bilateral CP. Children with asymmetric and symmetric hand use exhibited similar improvements after HABIT. [ABSTRACT FROM AUTHOR]

Aim: To evaluate pain prevalence and characteristics in children and adolescents with predominant dyskinetic and mixed (dyskinetic/spastic) cerebral palsy (CP) motor types.Method: Seventy-five participants with a diagnosis of CP and confirmed dyskinetic or mixed (dyskinetic/spastic) motor type took part in a multisite cross-sectional study. The primary outcome was carer-reported pain prevalence (preceding 2wks) measured using the Health Utilities Index-3. Secondary outcomes were chronicity, intensity, body locations, quality of life, and activity impact.Results: Mean participant age was 10 years 11 months (SD 4y 2mo, range 5-18y). There were 44 males and 31 females and 37 (49%) had predominant dyskinetic CP. Pain was prevalent in 85% and it was chronic in 77% of participants. Fifty-two per cent experienced moderate-to-high carer-reported pain intensity, which was significantly associated with predominant dyskinetic motor types (p=0.008). Pain occurred at multiple body locations (5 out of 21), with significantly increased numbers of locations at higher Gross Motor Function Classification System levels (p=0.02). Face, jaw, and temple pain was significantly associated with predominant dyskinetic motor types (p=0.005). Poorer carer proxy-reported quality of life was detected in those with chronic pain compared to those without (p=0.03); however, chronic pain did not affect quality of life for self-reporting participants.Interpretation: Pain was highly prevalent in children and adolescents with predominant dyskinetic and mixed (dyskinetic/spastic) motor types, highlighting a population in need of lifespan pain management.What This Paper Adds: Chronic pain prevalence in children and adolescents with predominant dyskinetic and mixed (dyskinetic/spastic) motor types is high. Pain occurs across multiple body locations in predominant dyskinetic and mixed (dyskinetic/spastic) motor types. Less recognized locations of pain include the face, jaw, and temple for predominant dyskinetic motor types. [ABSTRACT FROM AUTHOR]

Variants in the gene SCN1A are a common genetic cause for a wide range of epilepsy phenotypes ranging from febrile seizures to Dravet syndrome. Focal onset seizures and structural lesions can be present in these patients and the question arises whether epilepsy surgery should be considered. We report eight patients (mean age 13y 11mo [SD 8y 1mo], range 3-26y; four females, four males) with SCN1A variants, who underwent epilepsy surgery. Outcomes were variable and seemed to be directly related to the patient's anatomo-electroclinical epilepsy phenotype. Patients with Dravet syndrome had unfavourable outcomes, whilst patients with focal epilepsy, proven to arise from a single structural lesion, had good results. We conclude that the value of epilepsy surgery in patients with an SCN1A variant rests on two issues: understanding whether the variant is pathogenic and the patient's anatomo-electroclinical phenotype. Careful evaluation of epilepsy phenotype integrated with understanding the significance of genetic variants is essential in determining a patient's suitability for epilepsy surgery. Patients with focal onset epilepsy may benefit from epilepsy surgery, whereas those with Dravet syndrome do not. WHAT THIS PAPER ADDS: Patients should not automatically be excluded from epilepsy surgery evaluation if they carry an SCN1A variant. Patients with focal epilepsy may benefit from epilepsy surgery; those with Dravet syndrome do not. [ABSTRACT FROM AUTHOR]

Aim: To describe trends in gastrostomy tube insertion in children with developmental or acquired disorders in Sweden and assess their demographic characteristics.Method: Children aged 0 to 18 years with gastrostomy tube insertions recorded between 1998 and 2014 were identified in the Swedish National Patient Register. Associations between disorder type and year of surgery, as well as age at surgery, were analysed using linear regression analyses. The association between disorder type and mortality 2 years from gastrostomy tube insertion was also analysed using logistic regression analysis.Results: The data for 4112 children (2182 males, 1930 females), with a median age of 2 years (interquartile range=1-8y), were analysed. Children who presented with developmental disorders were the largest group (n=3501, 85%). The most common diagnosis in children with developmental disorders was cerebral palsy (n=165, 4%). In children with acquired disorders, acute lymphoblastic leukaemia (n=117, 3%) was the most common diagnosis. Gastrostomy tube insertions increased from 1998 to 2014, with the greatest increase in children with developmental disorders, who were younger than children with acquired disorders when the gastrostomy tube was first inserted. Age at tube insertion decreased in both groups during the study period. Mortality was higher in children with acquired disorders, suggesting that gastrostomy tube insertion should be part of a palliative care approach.Interpretation: Child characteristics differed depending on whether the underlying disorder was developmental or acquired, suggesting a need for clinical health care guidelines related to the specific goals of gastrostomy tube insertion.What This Paper Adds: Gastrostomy tube insertions increased by 140% from 1998 to 2014 in Sweden. The age of children with developmental disorders decreased by 1 month per year during the study period. Children presenting with developmental disorders were younger than children with acquired disorders when the gastrostomy tube was first inserted. Mortality was higher in children with acquired disorders. [ABSTRACT FROM AUTHOR]

Aim: To explore whether increasing parental education has a causal effect on risk of cerebral palsy (CP) in the child, or whether unobserved confounding is a more likely explanation.Method: We used data from Norwegian registries on approximately 1.5 million children born between 1967 and 2011. We compared results from a traditional cohort design with results from a family-based matched case-control design, in which children with CP were matched to their first cousins without CP. In addition, we performed a simulation study to assess the role of unobserved confounding.Results: In the cohort design, the odds of CP were reduced in children of mothers and fathers with higher education (adjusted odds ratio [OR] 0.67, 95% confidence interval [CI] 0.60-0.75 for maternal education, and adjusted OR 0.75, 95% CI 0.67-0.85 for paternal education). In the family-based case-control design, only an association for maternal education remained (adjusted OR 0.80, 95% CI 0.64-0.99). Results from a simulation study suggested that this association could be explained by unobserved confounding.Interpretation: A causal effect of obtaining higher education on risk of CP in the child is unlikely. Results stress the importance of continued research on the role of genetic and environmental risk factors that vary by parents' educational level.What This Paper Adds: Children of higher-educated parents had significantly lower odds of cerebral palsy (CP). There was no evidence of difference in risk of CP within first cousins whose mothers or fathers had different educational levels. Association between parental education and odds of CP did not reflect a causal effect. [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 assess effects of growth on lower limb maximal isometric muscle strength (MIMS) development in children with developmental coordination disorder (DCD).Method: This observational study used hand-held dynamometry to evaluate MIMS (hip abductors, flexors, extensors; knee flexors and extensors; ankle dorsiflexors) in children with DCD (n=33, 12 females, 21 males, 6-12y, mean [SD] age 9y [2y]). Regression analysis compared changes in MIMS for similar changes in growth (height or body mass) for children with DCD and typically developing children (pre-existing database, n=183), controlling for age and sex.Results: For the same height gain, the gain in muscle strength was 37.3% to 69.2% less in children with DCD compared with typically developing children, with significantly lower slopes (p-value between <0.001-0.042) in all muscle groups tested except knee extensors and ankle dorsiflexors in females. Strength gains related to body mass gains were not different for children with DCD compared to typically developing children.Interpretation: Even when growing at a similar rate, children with DCD do not develop muscle strength gains at the same rate as their typically developing peers.What This Paper Adds: Strength gains with growth (height) may be reduced in children with developmental coordination disorder. [ABSTRACT FROM AUTHOR]

Aim: To assess the neural and non-neural contributions to spasticity in the impaired ankle of children with cerebral palsy (CP).Method: Instrumented tapping of the Achilles tendon was done isometrically to minimize non-neural contributions and elicit neural contributions. Robot-controlled ankle stretching was done at various velocities, including slow stretching, with minimized neural contributions. Spasticity was assessed as having neural (phasic and tonic stretch reflex torque, tendon reflex gain, contraction rate, and half relaxation rate) and non-neural origin (elastic stiffness and viscous damping) in 17 children with CP (six females and 11 males; mean age [SD] 10y 8mo [3y 11mo], range 4y-18y) and 17 typically developing children (six females and 11 males; mean age [SD] 12y 7mo [2y 9mo], range 7y-18y). All torques were normalized to weight×height.Results: Children with CP showed increased phasic and tonic stretch reflex torque (p=0.004 and p=0.001 respectively), tendon reflex gain (p=0.02), contraction rate (p=0.038), half relaxation rate (p=0.02), elastic stiffness (p=0.01), and viscous damping (p=0.01) compared to typically developing children.Interpretation: Controlled stretching and instrumented tendon tapping allow the systematic quantification of various neural and non-neural changes in CP, which can be used to guide impairment-specific treatment.What This Paper Adds: Ankle spasticity is associated with increased phasic and tonic stretch reflexes, tendon reflex gain, and contraction and half relaxation rates. Ankle spasticity is also associated with increased elastic stiffness and viscous damping. [ABSTRACT FROM AUTHOR]

Aim: To evaluate the feasibility, acceptability, and preliminary effects of Participation and Environment Measure-Plus (PEM+) 2.0, an optimized version of a web-based, participation-focused, care-planning tool.Method: Twenty-two caregivers of children aged 0 to 5 years receiving rehabilitation services, who reported dissatisfaction with their child's participation, had internet access, and could read and write English, were recruited for this 2-week, single-arm pilot trial. Feasibility was assessed through retention rates, completion time, percentage of care plans developed relative to caregiver reported need, and independent completion of PEM+ 2.0. Acceptability was assessed by the Usefulness, Satisfaction, and Ease of Use Questionnaire. Preliminary effects were assessed by two items on caregiver reported impact of PEM+ 2.0 on confidence for addressing their child's participation.Results: Eighteen caregivers completed at least one iteration of PEM+ 2.0; of those, 17 were female and 15 were 30 to 39 years old. The median completion time was 12.99 minutes (quartile 1, 6.30; quartile 3, 17.33), mean care plan creation relative to need was 50% (standard deviation [SD] 31), and 17 completed PEM+ 2.0 independently. Mean acceptability scores were 3.80 to 4.97 (SD 1.25-1.97) and mean preliminary effect scores were 4.61 to 4.72 (SD 1.85-2.24), out of 7.0. There were strong and significant positive associations between two of the three estimates of PEM+ 2.0 acceptability and caregiver confidence (r=0.577-0.793, p<0.01).Interpretation: Electronic health tools have the potential for facilitating family-centered care in pediatric rehabilitation. PEM+ 2.0 is a feasible tool within pediatric rehabilitation and has potential to be an acceptable tool for improving caregiver confidence for promoting their child's participation in valued activities.What This Paper Adds: Participation and Environment Measure-Plus (PEM+) 2.0 is feasible for collaboratively engaging caregivers in the care-planning process. Caregivers perceived PEM+ 2.0 as helpful for thinking about their child's participation and what can be done to improve it. Feasibility and acceptability results will inform PEM+ 2.0 optimizations. [ABSTRACT FROM AUTHOR]

Aim: To describe leisure participation in adolescents with congenital heart defects (CHD) and identify factors associated with intensity of participation.Method: Eighty adolescents with CHD were recruited (39 males, 41 females; mean age [SD] 15y 8mo [1y 8mo] range 11y 5mo-19y 11mo) of whom 78 completed the Children's Assessment of Participation and Enjoyment (CAPE) outcome measure of leisure participation. The measure has five subscales: recreational, active-physical, social, skill-based, and self-improvement. Associations between the CAPE and age, sex, and development were examined. Motor ability (Movement Assessment Battery for Children, Second Edition), cognition (Leiter International Performance Scale-Revised), behavior (Strengths and Difficulties Questionnaire), and motivation (Dimensions of Mastery Questionnaire) were assessed.Results: Participants exhibited impaired motor (43.5%), behavioral (23.7%), and cognitive (29.9%) development. The most intense participation was in social (mean [SD] 3.3 [0.99]) and recreational (2.9 [0.80]) activity types on the CAPE. Male sex (p<0.05) and younger age were associated with greater physical activity (<15y: 1.87; ≥15y: 1.31, p<0.05). Greater engagement in social activities was related to better cognition (r=0.28, p<0.05), higher motor function (r=0.30-0.36, p<0.01), and fewer behavioral difficulties (r=-0.32 to -0.47, p<0.01). Cognitive ability (r=0.27, p<0.05), dexterity and aiming/catching (r=0.27-0.33, p<0.05), and behavior problems (r=0.38-0.49, p=0.001) were correlated with physical activity participation. Persistence in tasks, an aspect of motivation, correlated with physical (r=0.45, p<0.001) and social activity involvement (r=0.28, p<0.05).Interpretation: Ongoing developmental impairments in adolescents with CHD are associated with decreased active-physical and social engagement, putting them at risk of poor physical and mental health. Health promotion strategies should be considered.What This Paper Adds: Adolescents with congenital heart defects (CHD) have limited engagement in active-physical leisure activities. Cognitive, motor, and behavioral impairments are associated with decreased participation in leisure in children with CHD. Female sex and older age are associated with less engagement in leisure. Mastery motivation correlates with participation, suggesting an avenue for intervention. [ABSTRACT FROM AUTHOR]

Aim: To describe the cognitive development of children with neurofibromatosis type 1 (NF1) and plexiform neurofibromas, and identify predictors of cognitive development.Method: Participants included 88 children with NF1 and plexiform neurofibromas (50 males, 38 females, aged 6-18y, mean=12y, SD=3y 7mo) on a natural history study at the National Cancer Institute. Neuropsychological assessments (e.g. IQ, academic achievement, attention, and executive functioning) were administered three times over 6 years.Results: Relative to normative peers, the total sample of children with NF1 and plexiform neurofibromas demonstrated significantly lower scores in most cognitive domains and decreasing z-scores over time in math, writing, inhibitory control, and working memory. Children who had parents with (vs without) NF1 were more likely to experience decreased z-scores in performance IQ, reading, writing, attention, and working memory. Higher (vs lower) parental education was related to higher levels of IQ, math, reading, and cognitive flexibility and a slower decrease in math z-scores. Children's sex and the number of NF1 disease-related complications were not related to most cognitive outcomes.Interpretation: Children with NF1 and plexiform neurofibromas are at high risk for cognitive difficulties and declining z-scores in various domains of cognitive functioning over time. The findings highlight the need for a better understanding of the within-group differences in these children and their need for individualized educational plans.What This Paper Adds: Math, writing, inhibitory control, and working memory scores decreased over time. The proportion of children with clinically significant cognitive deficits increased over time. Parental neurofibromatosis type 1 and low education were related to greater cognitive difficulties in children. [ABSTRACT FROM AUTHOR]

Aim: To investigate the underlying factor structure of the 46-item Flemish cerebral visual impairment (CVI) questionnaire, differentiate the factor scores of children with and without CVI, and examine the impact of comorbidities on factor scores.Method: The records of 630 children (386 males, 244 females; median age 77mo; interquartile range 63-98mo) who visited the CVI clinic and the Centre for Developmental Disabilities at the University Hospitals of Leuven from 2001 to 2018 were reviewed systematically. Inclusion criteria included an up-to-date questionnaire, a definitive diagnosis, and clinical assessment.Results: Three hundred and forty-five children (179 with CVI [108 males, 71 females; median age 74mo; interquartile range 61-93mo] and 166 without CVI [110 males, 56 females; median age 88mo; interquartile range 70-107mo]) were included. An exploratory factor analysis resulted in a 5-factor (object and face processing impairments; visual (dis)interest; clutter and distance viewing impairments; moving in space impairments; and anxiety-related behaviours) biologically and clinically plausible model, which retained 35 items and explained 56% of the total variance. Mann-Whitney U tests indicated that factors 1 to 4 were significantly higher in children with CVI compared to children without CVI (p-values ranged from p<0.001 to p<0.05; effect sizes ranged from 0.11 to 0.33); factor 5 showed no differences. Autism, developmental coordination disorder, epilepsy, and cerebral palsy impacted factor scores.Interpretation: A 5-factor structure of the Flemish CVI questionnaire differentiates children with and without CVI. Comorbidities should be accounted for when researching CVI.What This Paper Adds: Cerebral visual impairment (CVI) is characterized by impaired object and face processing and impaired visual interest. CVI is also characterized by impaired clutter and distance viewing, and impaired moving in space. All children (with or without CVI) demonstrated anxiety-related behaviours. Autism affected object/face processing, whereas developmental coordination disorder, epilepsy, and cerebral palsy affected visual interest. [ABSTRACT FROM AUTHOR]

The aim of this case series was to assess and characterize cognitive abilities, autistic traits, and adaptive behaviour in Tatton-Brown-Rahman syndrome (TBRS). The sample included 18 individuals with a clinical and genetic diagnosis of TBRS (11 males, seven females; mean age 17y 7mo, SD 9y 5mo, range 7y 2mo-33y 10mo). The British Ability Scales, Third Edition and the Autism Diagnostic Observation Schedule, Second Edition (ADOS-2) were administered to all participants. The Social Responsiveness Scale, Second Edition and the Vineland Adaptive Behaviour Scales, Third Edition were completed by a parent/caregiver. The majority of participants (n=15) had intellectual disability and General Conceptual Ability scores ranged from 39 to 76 (mean 53.17, SD 12.13). Participants displayed a profile of better verbal ability compared with non-verbal reasoning ability and spatial ability. Autistic traits were prevalent and eight participants scored above the cut-off on the ADOS-2, although symptoms were less pronounced in older individuals. Adaptive functioning was impaired but commensurate with intellectual ability. Overall, TBRS is associated with an uneven cognitive profile and a high prevalence of autistic traits. This has implications for identifying appropriate services and support that may be beneficial for individuals with TBRS. WHAT THIS PAPER ADDS: Tatton-Brown-Rahman syndrome is associated with intellectual disability and impaired adaptive functioning. Autistic traits were prevalent within the sample. Lower intellectual ability and adaptive behaviour were associated with greater severity of autistic traits. [ABSTRACT FROM AUTHOR]

Aim: To determine neurocognitive performance and behavioural problems in children with Panayiotopoulos syndrome.Method: All 18 children (10 females, 8 males; mean age 4y 7mo; SD 1y 10mo) diagnosed with Panayiotopoulos syndrome at the Kempenhaeghe Epilepsy Center in the Netherlands between 2010 and 2017 were analysed retrospectively. All underwent a neuropsychological/behavioural assessment, an academic assessment, and a 24-hour electroencephalogram.Results: Mean full-scale IQ (93.5; range 76-123; p=0.04) and performance IQ (93.2; range 76-126; p=0.04) were within the normal range, although significantly lower compared to the normative mean. Verbal IQ (96.3; range 76-118) and processing speed (96.1; range 74-114) were not significantly lower. Simple auditory/visual reaction times, visual attention, visual-motor integration, and verbal memory were significantly lower compared to normative values. On average, patients with Panayiotopoulos syndrome were 8 months behind in arithmetic speed and 11 months behind in reading speed for the number of months in school. Behavioural questionnaires revealed significantly higher scores on reported internalizing behavioural problems.Interpretation: Children with Panayiotopoulos syndrome demonstrated diffuse cognitive dysfunction in full-scale IQ, performance IQ, visual attention, visual-motor integration, and verbal memory. A high incidence of internalizing behavioural problems was reported. This strongly suggests neuropsychological and behavioural comorbidity in children with Panayiotopoulos syndrome.What This Paper Adds: Children with Panayiotopoulos syndrome are at risk for cognitive deficits in various cognitive domains. Children with Panayiotopoulos syndrome are also prone to internalizing behavioural problems. Mild-to-severe academic underachievement was present in more than half of the children with Panayiotopoulos syndrome. [ABSTRACT FROM AUTHOR]

Aim: We examined key features of two outcome measures for social dysfunction and autism spectrum disorder traits, the Social Responsiveness Scale, Second Edition (SRS-2) and the Social Skills Improvement System - Rating Scales (SSIS-RS), in children with neurofibromatosis type 1 (NF1). The aim of the study was to provide objective evidence as to which behavioural endpoint should be used in clinical trials.Method: Cross-sectional behavioural and demographic data were pooled from four paediatric NF1 tertiary referral centres in Australia and the United States (N=122; 65 males, 57 females; mean age [SD] 9y 2mo [3y], range 3-15y).Results: Distributions of SRS-2 and SSIS-RS scores were unimodal and both yielded deficits, with a higher proportion of severely impaired scores on the SRS-2 (16.4%) compared to the SSIS-RS (8.2%). Pearson's product-moment correlations revealed that both questionnaires were highly related to each other (r=-0.72, p<0.001) and to measures of adaptive social functioning (both p<0.001). Both questionnaires were significantly related to attention-deficit/hyperactivity disorder symptoms, but only very weakly associated with intelligence.Interpretation: The SRS-2 and SSIS-RS capture social dysfunction associated with NF1, suggesting both may be suitable choices for assessing social outcomes in this population in a clinical trial. However, careful thought needs to be given to the nature of the intervention when selecting either as a primary endpoint.What This Paper Adds: The Social Responsiveness Scale, Second Edition yielded a large deficit relative to population norms. The Social Skills Improvement System - Rating Scales yielded a moderate deficit relative to population norms. Both scales were highly correlated, suggesting that they are measuring a unitary construct. [ABSTRACT FROM AUTHOR]

Aim: To assess: (1) the effect on drooling of bilateral submandibular duct ligation as surgical therapy after the administration of submandibular botulinum neurotoxin A (BoNT-A) for excessive drooling and (2) the predictive value of treatment success with BoNT-A on treatment success after bilateral submandibular duct ligation.Method: This was a within-participant retrospective observational study in which 29 children with severe drooling (15 males, 14 females) received BoNT-A treatment at a mean age of 9 years 6 months (SD 2y 5mo), followed by bilateral submandibular duct ligation at a mean age of 10 years 11 months (SD 2y 4mo). Fifteen children were diagnosed with cerebral palsy (CP), with 12 children classified in Gross Motor Function Classification System levels IV and V. The 14 children without CP had non-progressive developmental disorders. The primary drooling severity outcomes were the Visual Analogue Scale (VAS; subjective assessment) and drooling quotient (objective assessment). Measurements were taken before each intervention and again at 8 and 32 weeks.Results: The VAS was significantly lower after bilateral submandibular duct ligation at follow-up compared to BoNT-A treatment (mean difference -33, p≤0.001; 95% confidence interval [CI]=-43.3 to -22.9). The mean drooling quotient did not significantly differ between BoNT-A treatment and bilateral submandibular duct ligation at follow-up (3.3, p=0.457; 95% CI=-4.35 to 9.62) or between 8 and 32 weeks (4.7, p=0.188; 95% CI=-2.31 to 11.65).Interpretation: BoNT-A treatment and bilateral submandibular duct ligation are both effective treatment modalities for drooling. At 32-week follow-up, subjective drooling severity after bilateral submandibular duct ligation was significantly lower compared to previous BoNT-A injections in participants. However, treatment success with BoNT-A is no precursor to achieving success with bilateral submandibular duct ligation.What This Paper Adds: Bilateral submandibular duct ligation is an effective therapy for drooling after treatment with botulinum neurotoxin A (BoNT-A). Treatment success with BoNT-A is not a predictor of successful therapy with bilateral submandibular duct ligation. [ABSTRACT FROM AUTHOR]

Aim: To explore how patients with cerebral palsy (CP) perceive their gait and evaluate associations between subjective gait perception and: objective gait parameters, endurance, pain, and fatigue.Method: Sixty-two patients (21 females and 41 males; mean [SD] age 20y [5y 1mo], range 15-29y) performed a clinical gait analysis. Self-selected walking speed, Gait Profile Score, and Gait Variable Score were calculated. Subjective gait perception was assessed with a visual analogue scale using the question: 'On a scale from 0 (worst) to 10 (optimal), how would you describe your walking today?'. A 6-minute walk test (6MWT) measured endurance; the 36-Item Short Form Health Survey (SF-36) evaluated quality of life. T-tests, Pearson correlations, and univariate and multiple linear regression models were used to compare and find associations between the data.Results: Overall mean (SD) subjective gait perception was 7.5 (1.8) and was significantly higher for patients in Gross Motor Function Classification System (GMFCS) level I (7.9 [1.5]) than for patients in GMFCS levels II and III (5.9 [2.0]). Positive correlations were found between subjective gait perception and gait scores, walking speed, 6MWT distance, and SF-36 score. Only walking speed was a significant predictor of subjective gait perception.Interpretation: Subjective gait perception was influenced by GMFCS level and linked partially with the walking speed. The gait quality did not explain subjective gait perception. It is important to combine subjective and objective gait scores to develop personalized therapeutic goals.What This Paper Adds: Subjective gait perception is influenced by the physical impairment levels of patients with cerebral palsy. Subjective gait perception and objective gait scores are associated. Walking speed is the only predictor of gait perception. [ABSTRACT FROM AUTHOR]

This case series aimed to characterize the clinical features, management, and outcomes of apnea in infants with trisomy 18. Participants in this study were infants with trisomy 18 who were born alive and admitted to the neonatal intensive care unit in Kyushu University Hospital from 2000 to 2018. Retrospective analysis was performed on clinical data recorded in our department. Twenty-seven infants with trisomy 18 were admitted to our hospital during the study period, of which 25 (nine males, 16 females) were enrolled as eligible participants in this study. Among them, 14 started presenting with apnea from median 3.5 days of age (range 0-47d). In these infants with apnea, eight received respiratory support of positive pressure ventilation (PPV). The 1-year survival rate of infants in the PPV group was higher than that of non-PPV-supported infants (5 out of 8 vs 0 out of 6 infants). Five PPV-supported infants received a diagnosis of epilepsy, which was controlled by antiepileptic drugs. Postnatal respiratory intervention provides better prognosis in infants with trisomy 18. Improved survival leads to accurate diagnosis and treatment of apneic events in association with epilepsy. WHAT THIS PAPER ADDS: Respiratory support is effective against apnea in infants with trisomy 18. Intervention with ventilation provides a higher chance of prolonged survival. Improved survival leads to the accurate diagnosis and treatment of epilepsy-associated apnea. [ABSTRACT FROM AUTHOR]

Aim: To: (1) investigate the relationship between upper-limb impairment and health-related quality of life (HRQoL) for children with cerebral palsy and (2) develop a mapping algorithm from the Cerebral Palsy Quality of Life Questionnaire for Children (CPQoL-Child) onto the Child Health Utility 9D (CHU9D) measure.Method: The associations between physical and upper-limb classifications and HRQoL of 76 children (40 females, 36 males) aged 6 to 15 years (mean age 9 years 7 months [SD 3y]) were assessed. Five statistical techniques were developed and tested, which predicted the CHU9D scores from the CPQoL-Child total/domain scores, age, and sex.Results: Most participants had mild impairments. The Manual Ability Classification System (MACS) level was significantly negatively correlated with CHU9D and CPQoL-Child (r=-0.388 and r=-0.464 respectively). There was a negative correlation between the Neurological Hand Deformity Classification (NHDC) and CPQoL-Child (r=-0.476, p<0.05). The generalized linear model with participation, pain domain, and age had the highest predictive accuracy.Interpretation: The weak negative correlations between classification levels and HRQoL measures may be explained by the restricted range of impairment levels of the participants. The MACS and NHDC explained the impact of upper-limb impairment on HRQoL better than the other classifications. The generalized linear model with participation, pain, and age is the suggested mapping algorithm. The suggested mapping algorithm will facilitate the use of CPQoL-Child for economic evaluation and can be used to conduct cost-utility analyses.What This Paper Adds: The Manual Ability Classification System and Neurological Hand Deformity Classification were the best predictors of health-related quality of life measures. Age and Cerebral Palsy Quality of Life Questionnaire for Children participation and pain domain scores can predict Child Health Utility 9D scores. [ABSTRACT FROM AUTHOR]

Aim: To evaluate cortical excitability with transcranial magnetic stimulation (TMS) in children with new-onset epilepsy before and after antiepileptic drugs (AEDs).Method: Fifty-five drug-naïve patients (29 females, 26 males; 3-18y), with new-onset epilepsy were recruited from 1st May 2014 to 31st October 2017 at the Child Neurology Department, Queen Silvia's Children's Hospital, Gothenburg, Sweden. We performed TMS in 48 children (23 females, 25 males; mean [SD] age 10y [3y], range 4-15y) with epilepsy (27 generalized and 21 focal) before and after the introduction of AEDs. We used single- and paired-pulse TMS. We used single-pulse TMS to record resting motor thresholds (RMTs), stimulus-response curves, and cortical silent periods (CSPs). We used paired-pulse TMS to record intracortical inhibition and facilitation at short, long, and intermediate intervals.Results: There were no differences in cortical excitability between children with generalized and focal epilepsy at baseline. After AED treatment, RMTs increased (p=0.001), especially in children receiving sodium valproate (p=0.005). CSPs decreased after sodium valproate was administered (p=0.050). As in previous studies, we noted a negative correlation between RMT and age in our study cohort. Paired-pulse TMS could not be performed in most children because high RMTs made suprathreshold stimulation impossible.Interpretation: Cortical excitability as measured with RMT decreased after the introduction of AEDs. This was seen in children with both generalized and focal epilepsy who were treated with sodium valproate, although it was most prominent in children with generalized epilepsy. We suggest that TMS might be used as a prognostic tool to predict AED efficacy.What This Paper Adds: Resting motor threshold (RMT) correlated negatively with age in children with epilepsy. No differences in cortical excitability were noted between patients with generalized and focal epilepsy. Treatment with antiepileptic drugs decreased cortical excitability as measured with transcranial magnetic stimulation (TMS). Decreased cortical excitability with increased RMT was recorded, especially after sodium valproate treatment. Paired-pulse TMS was difficult to perform because of high RMTs in children. [ABSTRACT FROM AUTHOR]

Aim: To assess the reliability and predictive validity of the developmental and socio-emotional scales of the Standardized Infant NeuroDevelopmental Assessment (SINDA).Method: To assess reliability, two sets of three assessors forming eight assessor-pairs independently rated the developmental and socio-emotional scales of 60 infants. To evaluate predictive validity, 223 infants (gestational age 30wks [range 23-41wks]; 117 males, 106 females) attending a non-academic outpatient clinic were assessed by different assessors with SINDA's neurological, developmental, and socio-emotional scales. Atypical neurodevelopmental outcome at a corrected age of 24 months or older implied a Bayley Mental or Psychomotor Developmental Index score of less than 70 or neurological disorder (including cerebral palsy). Behavioural and emotional disorders were classified according to the International Classification of Diseases, 10th Revision. Predictive values were calculated from SINDA (2-12mo corrected age, median 7mo) and typical versus atypical outcome, and for intellectual disability only (Mental Developmental Index <70).Results: Assessors highly agreed on the developmental and socio-emotional assessments (developmental scores: Spearman's rank correlation coefficient ρ=0.972; single socio-emotional behaviour items: Cohen's κ=0.783-0.896). At 24 months or older, 65 children had atypical outcome. Atypical neurological scores predicted atypical outcome (sensitivity 83%, specificity 96%); atypical developmental scores predicted intellectual disability (sensitivity 77%, specificity 92%). Atypical emotionality and atypical self-regulation were associated with behavioural and emotional disorders.Interpretation: SINDA's three scales are reliable, and have a satisfactory predictive validity for atypical developmental outcome at 24 months or older in a non-academic outpatient setting. SINDA's developmental scale has promising predictive validity for intellectual disability. SINDA's socio-emotional scale is a tool for caregiver counselling.What This Paper Adds: Standardized Infant NeuroDevelopmental Assessment (SINDA)'s developmental and socio-emotional scales have excellent interrater reliability. Replication of the satisfactory validity of SINDA's neurological scale for atypical outcome. [ABSTRACT FROM AUTHOR]

Aim: To evaluate presence and severity of social impairments in alternating hemiplegia of childhood (AHC) and determine factors that are associated with social impairments.Method: This was a retrospective analysis of 34 consecutive patients with AHC (19 females, 15 males; mean age: 9y 7mo, SD 8y 2mo, range 2y 7mo-40y), evaluated with the Social Responsiveness Scale, Second Edition (SRS-2).Results: SRS-2 scores, indicating level of social impairment, were higher than population means (75, SD 14 vs 50, SD 10, p<0.001). Of these, 27 out of 34 had high scores: 23 severe (>76), four moderate (66-76). All subscale domains, including social cognition, social communication, social awareness, social motivation, restricted interests, and repetitive behavior, had abnormal scores compared to population means (p<0.001). High SRS-2 scores were associated with the presence of autism spectrum disorder (ASD) and epilepsy (p=0.01, p=0.04), but not with other scales of AHC disease symptomatology. All nine patients who received formal evaluations for ASD, because they had high SRS-2 scores, were diagnosed with ASD.Interpretation: Most patients with AHC have impaired social skills involving multiple domains. ASD is not uncommon. High SRS-2 scores in patients with AHC support referral to ASD evaluation. Our findings are consistent with current understandings of the pathophysiology of AHC and ASD, both thought to involve GABAergic dysfunction.What This Paper Adds: Most patients with alternating hemiplegia of childhood (AHC) have impaired social skills involving multiple domains. These impairments are significant compared to population means. Most patients with AHC have high Social Responsiveness Scale, Second Edition (SRS-2) scores. Patients with AHC with high SRS-2 scores are likely to have autism spectrum disorder. [ABSTRACT FROM AUTHOR]

Aim: To compare anterior and posterior standing balance reactions, as measured by single-stepping thresholds, in children with and without spastic cerebral palsy (CP).Method: Seventeen ambulatory children with spastic CP (eight males, nine females) and 28 typically developing children (13 males, 15 females; age range 5-12y, mean [SD] 9y 2mo [2y 3mo]), were included in this cross-sectional, observational study. Balance reaction skill was quantified as anterior and posterior single-stepping thresholds, or the treadmill-induced perturbations that consistently elicited a step in that direction. In order to understand the underlying mechanisms of between-group differences in stepping thresholds, dynamic stability was quantified using the minimum margin of stability. Ankle muscle activation latency, magnitude, and co-contraction were assessed with surface electromyography.Results: We observed an age and group interaction for anterior thresholds (p=0.001, partial η2 =0.24). At older (≈11y; p<0.001, partial η2 =0.48), but not younger (≈7y; p=0.33, partial η2 =0.02) ages, typically developing children had larger anterior thresholds than those with CP. In response to near-threshold anterior perturbations, older typically developing children recovered from more instability than their peers with CP (p=0.004, partial η2 =0.18). Older children had no between-group differences in ankle muscle activity. No between-group differences were observed in posterior thresholds.Interpretation: The effects of CP on balance reactions are age- and direction-specific. Older typically developing children are more able or willing to withhold a step when unstable.What This Paper Adds: Children with spastic cerebral palsy have age- and direction-specific balance-reaction impairments. Lower anterior stepping thresholds were observed in older, but not younger children. Older typically developing children withheld a forward step at higher levels of instability. No between-group differences were seen in posterior stepping thresholds. [ABSTRACT FROM AUTHOR]

Aim: To test whether wearable textile electromyography (EMG) recording systems may detect differences in muscle activity levels during daily activities between children with cerebral palsy (CP) and age-matched typically developing children.Method: Wearable textile EMG recording systems were used to obtain leg muscle activity in 10 children with spastic CP (four females, six males; mean age 9y 6mo, standard deviation [SD] 2y 4mo, range: 6-13y; Gross Motor Function Classification System [GMFCS] level I and II) and 11 typically developing children (four females, seven males; mean age 9y 9mo, SD 1y 11mo, 7-12y) at rest and while performing seven daily activities.Results: Children with CP showed significantly lower absolute EMG levels during maximal voluntary contractions (MVCs) of muscles on the most affected side as compared to the least affected side and to typically developing children. None of the typically developing children or children with CP showed detectable EMG activity in resting situations. EMG activity relative to MVC was greater in children with CP during walking, jumping, and kicking on the most affected side as compared to the least affected side and to typically developing children.Interpretation: Wearable textile EMG recording systems may be used to determine differences in muscle activity during daily activities in children with CP. Children with CP showed reduced muscle activity during daily activities compared to their peers, but used a significantly larger part of their maximal voluntary muscle strength to perform these activities.What This Paper Adds: Wearable textile electromyography (EMG) systems are feasible for measurement of daily muscle activity in children with cerebral palsy (CP). Children with CP showed reduced EMG levels during maximal voluntary contractions. Neither typically developing children or children with CP showed EMG activity in resting situations. Children with CP used a larger part of their voluntary muscle strength during daily activities. [ABSTRACT FROM AUTHOR]

Aims: To describe stroke syndromes and transcranial Doppler (TCD) findings in children with human immunodeficiency virus (HIV) and examine the associations between TCD and clinical and laboratory data.Method: We enrolled 42 children (24 males, 18 females) with HIV (median age=7y 6mo; 2y 7mo-15y 6mo), with and without stroke who underwent a TCD examination of the anterior and posterior circulations to derive time-averaged maximum mean velocity (TAMMV) measurements for comparison with previous studies. Clinical and laboratory variables were extracted from the medical records.Results: Of the 42 children with HIV, five had right-sided hemiparesis, three had chronic lung disease, two occurred post-varicella infection, one after herpetic oral ulceration, and one had a poorly functioning left ventricle. Neuroimaging showed middle cerebral artery (MCA) TAMMV greater than 200cm/s, moyamoya-like arteriopathy, left basal ganglia infarction with ipsilateral stenosis, hygroma consistent with venous thrombosis, and a hyperdense left MCA. Eight neurologically asymptomatic children had atypical TCD. The CD4 cell count was non-significantly lower in 6 out of 30 children with atypical TCD (median=21.5; interquartile range=16.1-26.5) compared with the remainder (median=29; interquartile range=21.3-35.0; p=0.09).Interpretation: A variety of stroke syndromes occur in children with HIV. TCD suggests atypical intracranial vessels and/or haemodynamics in some children with HIV infection, consistent with vasculopathy, possibly related directly to immunodeficiency and/or infection.What This Paper Adds: A range of stroke syndromes are found in children with human immunodeficiency virus (HIV). Transcranial Doppler (TCD) velocities in HIV are commonly outside the range for typically developing children. TCD and neuroimaging data in children with HIV suggest intracranial vasculopathy as one mechanism for stroke. CD4 cell count is non-significantly lower in children with HIV and atypical TCD. [ABSTRACT FROM AUTHOR]

Aim: To examine the association between brain magnetic resonance imaging (MRI) characteristics and executive function and bimanual performance in children with unilateral cerebral palsy (CP).Method: Clinical MRI brain scans were classified as: (1) predominant pathological pattern (normal, white matter injury [WMI]; grey matter injury; focal vascular insults [FVI]; malformations; or miscellaneous); and (2) focal lesions (frontal, basal ganglia, and/or thalamus). Assessments included: (1) bimanual performance; (2) unimanual dexterity; and (3) executive function tasks (information processing, attention control, cognitive flexibility, and goal setting) and behavioural ratings (parent).Results: From 131 recruited children, 60 were ineligible for analysis, leaving 71 children (47 males, 24 females) in the final sample (mean age 9y [SD 2y], 6y-12y 8mo). Brain MRIs were WMI (69%) and FVI (31%); and frontal (59%), thalamic (45%), basal ganglia (37%), and basal ganglia plus thalamic (21%). Bimanual performance was lower in FVI versus WMI (p<0.003), and with frontal (p=0.36), basal ganglia (p=0.032), and thalamic/basal ganglia lesions (p=0.013). Other than information processing, executive function tasks were not associated with predominant pattern. Frontal lesions predicted attention control (p=0.049) and cognitive flexibility (p=0.009) but not goal setting, information processing, or behavioural ratings.Interpretation: Clinical brain MRI predicts cognitive and motor outcomes when focal lesions and predominate lesion patterns are considered. What this paper adds Early brain magnetic resonance imaging (MRI) predicts bimanual performance and cognitive outcomes. Brain MRI may identify children requiring targeted interventions. Basal ganglia with/without thalamic lesions predicted bimanual performance. Frontal lesions were associated with attention control and cognitive flexibility. Brain MRI predominant patterns predicted motor, not cognitive outcomes, other than information processing. [ABSTRACT FROM AUTHOR]

Aim: To explore cortical activation during bimanual tasks and functional correlates in unilateral cerebral palsy (CP).Method: This cross-sectional study included eight participants with unilateral CP (six females, two males; mean age [SD] 20y 10mo [5y 10mo], 13y 8mo-31y 6mo) in Manual Ability Classification System levels II to III and nine age-matched participants with typical development (seven females, two males; mean age [SD] 17y 8mo [5y 7mo], 9y 4mo-24y 2mo). They performed bimanual symmetric squeezing (BSS) and bimanual asymmetric squeezing (BAS) tasks at 1Hz, and a pouring task with dominant hand (DPour) and a pouring task with non-dominant hand (NDPour) at 0.67Hz, all while a custom array of functional near-infrared spectroscopy (fNIRS) optodes were placed over their sensorimotor area. Mixed-effects were used to contrast groups, tasks, and hemispheres (corrected p-values [q] reported). Analysis of variance and t-tests compared performance measures across groups and tasks.Results: Participants with unilateral CP showed greater activation in both hemispheres during BAS (non-lesioned: q<0.001; lesioned: q<0.001), and in the lesioned hemisphere during BSS (q<0.001), DPour (q=0.02), and NDPour (q=0.02) than those with typical development. The lesioned hemisphere in unilateral CP showed more activity than the non-lesioned one (BSS: q=0.01; BAS: q=0.009; NDPour: q=0.04). During BAS, higher cortical activity correlated with more synchronous arm activation (r=0.79; p=0.02); activity lateralized towards the non-lesioned hemisphere correlated with better Pediatric Evaluation of Disability Inventory computer adaptive test scores (r=0.81; p=0.03).Interpretation: Results suggest abnormally increased sensorimotor cortical activity in unilateral CP, with implications to be investigated.What This Paper Adds: Cortical activity in manual tasks is described with functional near-infrared spectroscopy in typical and atypical cohorts. Activation levels in unilateral cerebral palsy appear to escalate with task difficulty. Increased brain activity may be associated with poorer selective manual control. Specific patterns of brain activity may be related to impaired bimanual function. [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 examine self- and proxy-reported symptoms of depression in children with epilepsy.Method: This was a prospective longitudinal cohort study of children with epilepsy. Participants were treated at six Canadian tertiary-care centers and followed over 28 months with repeated assessments of child self-reported symptoms of depression using the Children's Depression Inventory Short-Form (CDI-S). Trajectories of symptoms of depression were estimated using linear mixed effects (LME) modeling.Results: At baseline, 477 children had complete data (mean age [SD] 11y 5mo [2y 1mo], range 7y 7mo-15y 1mo; 234 females, 243 males). Mean CDI-S T score at baseline was 45.7 (SD=7.5) and at 28 months was 44.9 (SD=8.2), both were within the 'average' range. Results from LME modeling revealed mean raw CDI-S score of 1.897, corrected for age 10 years (corresponding to T scores slightly below the normed mean of 50), with no significant change over three measurements (slope=-0.113, p=0.135), indicating that CDI-S scores were stable over 28 months. Children with high initial CDI-S scores had lower subsequent scores, as demonstrated by the correlation of -0.827 between intercept and slope (p<0.001). Parents reported comparable findings.Interpretation: Self- and proxy-reported symptoms of depression were generally low and stable over an extended follow-up period. Normalization of scores was seen upon repeated assessment, even in children with higher scores of symptoms of depression at one point. These findings speak to the value and importance of repeated assessment over time.What This Paper Adds: In children with epilepsy, self- and proxy-reported symptoms of depression were generally low and stable over 28 months. The trajectory of symptoms of depression was not associated with seizure severity, whether considering the frequency or type of seizures. Parents' reports of symptoms of depression were comparable to the children's self-evaluations. [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 report the differences between Performance of Upper Limb (PUL) versions 1.2 and 2.0, compare the measurement ability of the two versions, and compare their longitudinal performance in Duchenne muscular dystrophy.Method: Rasch analysis was performed on the dual data from three centres to confirm whether the two scales measure the same construct. Change scores in natural history for the different domains were compared for the two versions.Results: Rasch analysis demonstrated that both versions measure the same construct and that the PUL 2.0 was a better fit to the construct of motor performance and better able to detect change at 12 months in all levels of ability than the PUL 1.2. This was also true when change scores were reviewed over 2 years.Interpretation: Our results confirm that the PUL 1.2 and 2.0 versions detect change in all domains over 2 years. They also demonstrate that simplifying the original scoring of the PUL 1.2 for the revised PUL 2.0 maintains the validity of the construct and enhances the scale measurement qualities.What This Paper Adds: The original and revised Performance of Upper Limb (PUL) scales measure the same construct. Both scales detected change in all domains over 2 years. The PUL 2.0 enhances the measurement qualities of the scale. [ABSTRACT FROM AUTHOR]

Aim: To develop a detailed priority list of family-centred rehabilitation goals on the activity level within the International Classification of Functioning, Disability and Health (ICF) chapters d4 'Mobility' and d5 'Self-care' in a paediatric population with a broad range of health conditions.Method: Twenty-two months after implementing a systematic, family-centred, goal-setting process, the rehabilitation goals of 212 inpatients were retrospectively allocated to the most detailed level of ICF categories by two independent researchers. The overall frequencies of these goals were calculated and stratified by health condition, functional independence, and age.Results: Ninety-three females and 119 males were included in the study (mean age 10y 9mo, SD 4y 5mo, range 2y 1mo-21y 5mo). The five most frequent rehabilitation goals were ICF codes d4500 'Walking short distances' (11%), d4200 'Transferring oneself while sitting' (9%), d5400 'Putting on clothes' (7%), d451 'Going up and down stairs' (6%), and d4153 'Maintaining a sitting position' (5%). These top goals varied in the subgroups with regard to the underlying health condition, functional independence, and age.Interpretation: The findings of this study are not generalizable due to the large heterogeneity in priorities. However, they can be used to incorporate families' needs into future research designs and the development of new technologies.What This Paper Adds: Walking short distances is the most frequent mobility/self-care goal of paediatric rehabilitation. The top goals depend on health condition, functional independence, and age. Priorities vary considerably between children undergoing rehabilitation. Rehabilitation goals need to be assessed individually for each child. [ABSTRACT FROM AUTHOR]

Aim: To compare the rate of falls between adults with and without cerebral palsy (CP).Method: We used primary care data on 1705 adults with CP and 5115 adults without CP matched for age, sex, and general practice attended. We compared odds of experiencing a fall between adults with and without CP using conditional logistic regression. We compared the rate of falls using a negative binomial model.Results: Participants were 3628 males (53%) and 3192 females (47%) (median age 29y, interquartile range 20-42y) at the start of follow-up. Follow-up was 14 617 person-years for adults with CP and 56 816 person-years for adults without CP. Of adults with CP, 15.3% experienced at least one fall compared to 5.7% of adults without CP. Adults with CP had 3.64 times (95% confidence interval [CI] 2.98-4.45) the odds of experiencing a fall compared to adults without CP. The rate of falls was 30.5 per 1000 person-years and 6.7 per 1000 person-years for adults with and without CP respectively (rate ratio 5.83, 95% CI 4.84-7.02) INTERPRETATION: Adults with CP are more likely to fall, and fall more often, than adults without CP. The causes and consequences of falls in adults with CP need examination.What This Paper Adds: Twenty adults with CP and 5.3 adults without CP experienced at least one fall per 1000 person-years. Adults with CP experienced 30.5 falls per 1000 person-years compared to 6.7 falls per 1000 person-years among adults without CP. Adults with CP had 3.64 times the odds of experiencing a fall compared to adults without CP. Adults with CP experienced 5.83 times more falls than adults without CP. [ABSTRACT FROM AUTHOR]

Aim: To determine the proportion of children with cerebral palsy (CP) who had major congenital anomalies, describe the types of disorders, and report on the children's functional outcomes.Method: Data were extracted from the Bangladesh Cerebral Palsy Register (BCPR). Descriptive analyses were conducted on children with CP and major congenital anomalies. Odds ratios (ORs) with 95% confidence intervals (CIs) were calculated to measure the association between major congenital anomalies, clinical severity, and presence of comorbidities.Results: Between January 2015 and December 2016, 726 children with CP were newly registered with the BCPR (277 females, 449 males; mean age [SD] at registration 90mo [54mo], 4mo-18y). Seventy-eight children (11%) had a major congenital anomaly. Neurological (86%) and musculoskeletal congenital anomalies (10%) were the most common. Microcephaly was the most common congenital anomaly (83%). The odds of severe functional motor limitations (OR=2.4, 95% CI=1.9-2.9), epilepsy (OR=1.6, 95% CI=1.1-2.1), visual impairment (OR=2.6, 95% CI=2.0-3.2), presence of strabismus (OR=3.9, 95% CI=3.8-4.4), hearing (OR=1.2, 95% CI=0.6-1.9), speech (OR=5.4, 95% CI=4.6-6.2), and intellectual impairments (OR=2.3, 95% CI=1.8-2.8) were higher in children with congenital anomalies compared to children without.Interpretation: The proportion of children with major congenital anomalies in the BCPR (11%) was lower than that identified in higher-income countries. This may be because of differences in how congenital anomalies are diagnosed as well as the impact of survival bias. In Bangladesh, children with CP and major congenital anomalies are more likely to have severe functional motor limitations and associated comorbidities.What This Paper Adds: Eleven per cent of children with cerebral palsy (CP) in Bangladesh had major congenital anomalies. Neurological and musculoskeletal congenital anomalies were the most common. Severe functional motor limitations and associated comorbidities were more common in children presenting with CP and major congenital anomalies. [ABSTRACT FROM AUTHOR]

Aim: To determine whether, and how, neonatal intensive care unit (NICU) parents want to receive early neurodevelopmental screening information about their child's future risk of cerebral palsy and other disabilities.Method: This was a qualitative interview study. Parents of hospitalized infants born preterm completed semi-structured interviews. Data were analysed using a directed content analysis approach.Results: Thematic saturation was achieved after 19 interviews. Four themes characterized parent perceptions of early neurodevelopmental screening: (1) acceptability: most parents were in favour of neurodevelopmental screening if parents could refuse; (2) disclosure of results: parents want emotional preparation for results, especially false positives; (3) emotional burden of uncertainty: parents of children in the NICU balance taking their infant's illness 'day by day' and preparing for an uncertain future. Parents expressed distress with screening that increased uncertainty about the future; and (4) disability: prior experience with disability informs parent concerns.Interpretation: Parents interpret the risks and benefits of NICU developmental screening through the lens of prior experiences with disability. Most expressed interest in screening and emphasized a desire for autonomy, pretest counselling, and emotional preparation.What This Paper Adds: Most parents with infants in the neonatal intensive care unit expressed interest in early screening for developmental disability. Prior experience with disability informed concerns about specific deficits. Parents emphasized a desire for autonomy, pretest counselling, and emotional preparation. [ABSTRACT FROM AUTHOR]