Association between upper and lower respiratory disease among patients with primary ciliary dyskinesia: an international study.

Introduction: Nearly all patients with primary ciliary dyskinesia (PCD) report ear-nose-throat (ENT) symptoms. However, scarce evidence exists about how ENT symptoms relate to pulmonary disease in PCD. We explored possible associations between upper and lower respiratory disease among patients with PCD in a multicentre study. Methods: We included patients from the ENT Prospective International Cohort (EPIC-PCD). We studied associations of several reported ENT symptoms and chronic rhinosinusitis (defined using patient-reported information and examination findings) with reported sputum production and shortness of breath, using ordinal logistic regression. In a subgroup with available lung function results, we used linear regression to study associations of chronic rhinosinusitis and forced expiratory volume in 1 s (FEV1) accounting for relevant factors. Results: We included 457 patients (median age 15 years, interquartile range 10-24 years; 54% males). Shortness of breath associated with reported nasal symptoms and ear pain of any frequency, often or daily hearing problems, headache when bending down (OR 2.1, 95% CI 1.29-3.54) and chronic rhinosinusitis (OR 2.3, 95% CI 1.57-3.38) regardless of polyp presence. Sputum production associated with daily reported nasal (OR 2.2, 95% CI 1.20-4.09) and hearing (OR 2.0, 95% CI 1.10-3.64) problems and chronic rhinosinusitis (OR 2.1, 95% CI 1.48-3.07). We did not find any association between chronic rhinosinusitis and FEV1. Conclusion: Reported upper airway symptoms and signs of chronic rhinosinusitis associated with reported pulmonary symptoms, but not with lung function. Our results emphasise the assessment and management of upper and lower respiratory disease as a common, interdependent entity among patients with PCD.

Sleep problems in a population-based cohort of primary school age children with Cerebral Palsy.

AIMS: To examine sleep problems in a population-based sample of school-aged children (8-12yo) with Cerebral Palsy (CP) METHOD: Eighty-six children (mean 9 years, 5 months, SD = 1 year, 6 months; male = 60) with CP (Gross Motor Function Classification System; GMFCS I=46; II=21; III=9; IV=6; V=6) participated. Classifications/assessments included: Sleep Disturbance Scale for Children (SDSC), Gross Motor Function Measure (GMFM-66), Manual Ability Classification System (MACS), Communication Function Classification System (CFCS), Strengths and Difficulties Questionnaire (SDQ) and the Cerebral Palsy- Quality of Life (CP-QOL) Pain Impact subscale. Analysis included linear and logistic regression. RESULTS: 38 (44 %) children were within the clinical range for sleep problems. Sleep problems were significantly associated with epilepsy, (95 % CI) = 14.48 (7.95 to 21.01), gross motor function, -0.13 (-0.26 to -0.01), manual ability, 7.26 (0.82 to 13.69), communication, 10.01 (2.21 to 17.80), child behaviour, 1.134 (0.74 to 1.53), and pain related QOL, 0.33 (0.12 to 0.53). For the multivariable model, sleep problems remained significantly associated with epilepsy, b (95 % CI) = 11.72 (4.88 to 18.57), child behaviour, 1.03 (0.65 to 1.41) and pain-related QOL, 0.21 (0.29 to 0.38). CONCLUSIONS: Sleep problems are common and associated with epilepsy, child behaviour and pain related QOL.

Construct Validity of the Both Hands Assessment Using Wrist-Worn Accelerometers.

AIM: The aim of this study was to evaluate the construct validity of the Both Hands Assessment (BoHA) using activity of the upper limbs as detected by accelerometry in children with bilateral cerebral palsy (CP). METHODS: Observational study of children with CP (n = 44, n = 27 boys, aged 9.1 ± 1.6 years; Manual Ability Classification Scale I: n = 15, II: n = 22, III: n = 7) completing a BoHA assessment while wearing a triaxial accelerometer on each wrist. BoHA Each-Hand sub-scores, BoHA percentage difference between hands, BoHA Units, mean activity for each hand, mean activity asymmetry index and total mean activity were calculated. Linear regressions were used to analyze associations between measures. RESULTS: There were significant, positive associations between BoHA Units and total mean activity (B = 0.86, 95%CI: 0.32, 1.40), BoHA Percentage difference between hands and mean activity asymmetry index (B = 0.95, 95%CI: 0.75,1.15), and BoHA Each-Hand sub-score and mean activity for the non-dominant hand (B = 1.71, 95%CI: 1.16, 2.28), but not the dominant hand (B = 0.50, 95%CI: -0.45, 1.45). CONCLUSIONS: This study provides further evidence for the construct validity of the BoHA as a measure of upper limb performance. Wearable wrist sensors such as accelerometers capture and quantify gross upper limb movement in children with CP but cannot measure fine finger movements captured by the BoHA. CLINICAL TRIALS REGISTRATION: Australian New Zealand Clinical Trials Registry (ACTRN12616001488493 and ACTRN12618000164291).

Female and male obese Zucker rats display differential inflammatory mediator and long non-coding RNA profiles.

AIMS: The goal of this study was to identify mediators in peri-lymphatic adipose tissue (PLAT) that are altered in obese versus lean Zucker rats, with focus on potential sex differences MAIN METHODS: Mesenteric PLAT was analyzed with protein and lncRNA arrays. Additional RT-PCR confirmation was performed with epididymal/ovarian fat. KEY FINDINGS: MCP-1, TCK-1, Galectin-1, Galectin-3, and neuropilin-1 were elevated in PLAT from obese rats of both sexes. However, 11 additional proteins were elevated only in obese males while 24 different proteins were elevated in obese females. Profiling of lncRNAs revealed lean males have elevated levels of NEAT1, MALAT1 and GAS5 compared to lean females. NEAT1, MALAT1, and GAS5 were significantly reduced with obesity in males but not in females. Another lncRNA, HOTAIR, was higher in lean females compared to males, and its levels in females were reduced with obesity. Obese rats of both sexes had similar histologic findings of mesenteric macrophage crown-like structures and hepatocyte fat accumulation. SIGNIFICANCE: While obese male and female Zucker rats both have increased inflammation, they have distinct signals. Future studies of the proteome and lncRNA landscape of obese males vs. females in various animal models and in human subjects are warranted to better guide development of therapeutics for obesity-induced inflammation.

Sinonasal disease among patients with primary ciliary dyskinesia: an international study.

Background: Sinonasal symptoms are a common feature of primary ciliary dyskinesia (PCD); however, literature about their severity and frequency, particularly during the life course, is scarce. Using baseline data from the Ear, nose and throat (ENT) Prospective International Cohort of PCD patients, we describe sinonasal disease in PCD. Methods: We included participants who had a routine sinonasal examination during which they completed a symptoms questionnaire. We compared frequency of reported symptoms and examination findings among children and adults, and identified characteristics potentially associated with higher risk of sinonasal disease using ordinal regression. Results: 12 centres contributed 384 participants; median age was 16 years (IQR 9-22), and 54% were male. Chronic nasal problems were the most common feature, reported by 341 (89%). More adults (33; 24%) than children (10; 4%) described hyposmia. Quality of life was moderately affected by rhinosinusitis among 136 participants with completed SNOT-22 questionnaires (median score 31; IQR 23-45). Examinations revealed nasal polyps among 51 of 345 participants (15%) and hypertrophic inferior nasal turbinates among 127 of 341 participants (37%). Facial pain was detected in 50 of 342 participants (15%). Nasal polyps, hypertrophic turbinates, deviated septum and facial pain were found more commonly in adults than children. The only characteristic associated with higher risk of sinonasal disease was age 10 years and older. Conclusions: Based on our findings, regular sinonasal examinations are relevant for patients with PCD of all ages. There is a need for improved management of sinonasal disease supported by evidence-based guidelines.

Characteristics of Otologic Disease Among Patients With Primary Ciliary Dyskinesia.

Importance: Otologic disease is common among people with primary ciliary dyskinesia (PCD), yet little is known about its spectrum and severity. Objective: To characterize otologic disease among participants with PCD using data from the Ear-Nose-Throat Prospective International Cohort. Design, Setting, and Participants: This cross-sectional analysis of baseline cohort data from February 2020 through July 2022 included participants from 12 specialized centers in 10 countries. Children and adults with PCD diagnoses; routine ear, nose, and throat examinations; and completed symptom questionnaires at the same visit or within 2 weeks were prospectively included. Exposures: Potential risk factors associated with increased risk of ear disease. Main Outcomes and Measures: The prevalence and characteristics of patient-reported otologic symptoms and findings from otologic examinations, including potential factors associated with increased risk of ear inflammation and hearing impairment. Results: A total of 397 individuals were eligible to participate in this study (median [range] age, 15.2 [0.2-72.4] years; 186 (47%) female). Of the included participants, 204 (51%) reported ear pain, 110 (28%) reported ear discharge, and 183 (46%) reported hearing problems. Adults reported ear pain and hearing problems more frequently when compared with children. Otitis media with effusion-usually bilateral-was the most common otoscopic finding among 121 of 384 (32%) participants. Retracted tympanic membrane and tympanic sclerosis were more commonly seen among adults. Tympanometry was performed for 216 participants and showed pathologic type B results for 114 (53%). Audiometry was performed for 273 participants and showed hearing impairment in at least 1 ear, most commonly mild. Season of visit was the strongest risk factor for problems associated with ear inflammation (autumn vs spring: odds ratio, 2.40; 95% CI, 1.51-3.81) and age 30 years and older for hearing impairment (41-50 years vs ≤10 years: odds ratio, 3.33; 95% CI, 1.12-9.91). Conclusion and Relevance: In this cross-sectional study, many people with PCD experienced ear problems, yet frequency varied, highlighting disease expression differences and possible clinical phenotypes. Understanding differences in otologic disease expression and progression during lifetime may inform clinical decisions about follow-up and medical care. Multidisciplinary PCD management should be recommended, including regular otologic assessments for all ages, even without specific complaints.

School readiness of children at high risk of cerebral palsy randomised to early neuroprotection and neurorehabilitation: protocol for a follow-up study of participants from four randomised clinical trials.

INTRODUCTION: School readiness includes cognitive, socio-emotional, language and physical growth and development domains which share strong associations with life-course opportunities. Children with cerebral palsy (CP) are at increased risk of poor school readiness compared with their typically developing peers. Recently, earlier diagnosis of CP has allowed interventions to commence sooner, harnessing neuroplasticity. First, we hypothesise that early referral to intervention for children at-risk of CP will lead to improved school readiness at 4-6 years relative to placebo or care as usual. Second, we hypothesise that receipt of early diagnosis and early intervention will lead to cost-savings in the form of reduced healthcare utilisation. METHODS AND ANALYSIS: Infants identified as at-risk of CP ≤6 months corrected age (n=425) recruited to four randomised trials of neuroprotectants (n=1), early neurorehabilitation (n=2) or early parenting support (n=1) will be re-recruited to one overarching follow-up study at age 4-6 years 3 months. A comprehensive battery of standardised assessments and questionnaires will be administered to assess all domains of school readiness and associated risk factors. Participants will be compared with a historical control group of children (n=245) who were diagnosed with CP in their second year of life. Mixed-effects regression models will be used to compare school readiness outcomes between those referred for early intervention versus placebo/care-as-usual. We will also compare health-resource use associated with early diagnosis and intervention versus later diagnosis and intervention. ETHICS AND DISSEMINATION: The Children's Health Queensland Hospital and Health Service, The University of Queensland, University of Sydney, Monash University and Curtin University Human Research Ethics Committees have approved this study. Informed consent will be sought from the parent or legal guardian of every child invited to participate. Results will be disseminated in peer-reviewed journals, scientific conferences and professional organisations, and to people with lived experience of CP and their families. TRIAL REGISTRATION NUMBER: ACTRN12621001253897.

Development of social functioning in children with cerebral palsy: A longitudinal study.

AIM: To describe the development of social function in children with cerebral palsy (CP) classified in all levels of the Communication Function Classification System (CFCS). METHOD: This prospective, longitudinal population-based cohort study recruited children with CP born in Queensland, Australia. Social functioning was measured using the Pediatric Evaluation of Disability Inventory (PEDI) social function domain at 2 years, 2 years 6 months, 3 years, 4 years, and 5 years, and the PEDI Computer Adaptive Test (PEDI-CAT) social/cognitive domain at 8 to 12 years. RESULTS: Seventy-four children provided 356 observations. PEDI-CAT social/cognitive scaled scores at 8 to 12 years were (mean [SD] n) CFCS level I, 68.6 (2.7) 45; CFCS level II, 64.0 (3.4) 10; CFCS level III, 63.5 (3.7) 4; CFCS level IV, 56.8 (5.0) 9; CFCS level V, 47.2 (5.8) 6. Scores within expected range for age (not less than 2 SD below mean) at 8 to 12 years were achieved by 35 (78%) children in CFCS level I and four (14%) in CFCS levels II to V. Forty-nine per cent of children scored at least two standard deviations below the population mean on a proxy measure of fluid intelligence. Intellectual impairment was associated with lower PEDI-CAT social/cognitive scaled scores in univariable analysis (ß = -8.3, 95% confidence interval - 10.91 to -5.63; p < 0.001) but had a smaller effect when modelled together with CFCS. INTERPRETATION: Social function attained by 8 to 12 years of age was strongly related to level of communication function (CFCS). The small number of children classified in CFCS levels II to V necessitates caution when viewing these individual CFCS level trajectories. WHAT THIS PAPER ADDS: There is a strong relation between social functioning and Communication Function Classification System (CFCS) levels. At 8 to 12 years, 35 out of 45 children in CFCS level I met social functioning age expectations. Twenty-five out of 29 in CFCS levels II to V had social functioning below that expected for age. CFCS and age were more strongly associated with development of social functioning than Gross Motor Function Classification System or Manual Ability Classification System and age.

The behavioral and neurobiological relationships between executive function and reading: A review and preliminary findings.

Despite decades of prior research, the mechanisms for how skilled reading develops remain elusive. Numerous studies have identified word recognition and oral language ability as key components to explain later reading comprehension performance. However, these components alone do not fully explain differences in reading achievement. There is ongoing work exploring other candidate processes important for reading, such as the domain-general cognitive ability of executive function (EF). Here, we summarize our work on the behavioral and neurobiological connections between EF and reading and present preliminary neuroimaging findings from ongoing work. Together, these studies suggest 1) that EF plays a supportive and perhaps indirect role in reading achievement and 2) that EF-related brain regions interface with the reading and language networks. While further work is needed to dissect the specifics of how EF interacts with reading, these studies begin to reveal the complex role that EF plays in reading development.

Identifying a brain network for musical rhythm: A functional neuroimaging meta-analysis and systematic review.

We conducted a systematic review and meta-analysis of 30 functional magnetic resonance imaging studies investigating processing of musical rhythms in neurotypical adults. First, we identified a general network for musical rhythm, encompassing all relevant sensory and motor processes (Beat-based, rest baseline, 12 contrasts) which revealed a large network involving auditory and motor regions. This network included the bilateral superior temporal cortices, supplementary motor area (SMA), putamen, and cerebellum. Second, we identified more precise loci for beat-based musical rhythms (Beat-based, audio-motor control, 8 contrasts) in the bilateral putamen. Third, we identified regions modulated by beat based rhythmic complexity (Complexity, 16 contrasts) which included the bilateral SMA-proper/pre-SMA, cerebellum, inferior parietal regions, and right temporal areas. This meta-analysis suggests that musical rhythm is largely represented in a bilateral cortico-subcortical network. Our findings align with existing theoretical frameworks about auditory-motor coupling to a musical beat and provide a foundation for studying how the neural bases of musical rhythm may overlap with other cognitive domains.

Development of gross motor capacity and mobility performance in children with cerebral palsy: a longitudinal study.

AIM: To describe development of gross motor capacity and mobility performance in children with cerebral palsy. METHOD: This longitudinal cohort study measured gross motor capacity with the Gross Motor Function Measure and mobility performance with the Pediatric Evaluation of Disability Inventory (PEDI) between 18 months and 5 years, and the PEDI - Computer Adaptive Test (PEDI-CAT) between 8 years and 12 years. Longitudinal analyses used mixed-effects regression modelling with interaction between age and Gross Motor Function Classification System (GMFCS). Stability of GMFCS levels over time was measured using agreement. RESULTS: Two hundred and twenty-two children provided 871 observations (median 4 observations, range 1-7). Children classified in GMFCS level I improved in both capacity and performance until 8 to 12 years. Children classified in GMFCS levels II and III continued to develop mobility performance after gross motor capacity had plateaued at 5 years. Children classified in GMFCS level IV plateaued at 5 years in capacity and performance. Children classified in GMFCS level V showed no changes in capacity or performance between 18 months and 8 to 12 years. Stability of GMFCS levels was 73%. INTERPRETATION: Change in mobility performance over time somewhat reflected gross motor capacity trajectories. Continued improvement in mobility performance after plateau of gross motor capacity for children classified in GMFCS levels II and III suggests importance of other personal or environmental factors.

Study protocol: the ear-nose-throat (ENT) prospective international cohort of patients with primary ciliary dyskinesia (EPIC-PCD).

INTRODUCTION: Primary ciliary dyskinesia (PCD) is a rare, genetic, multiorgan disease with an estimated prevalence of 1 in 10 000. It affects mainly the upper and lower airways due to impaired mucociliary clearance. Almost all patients have sinonasal or otologic (ear-nose-throat, ENT) problems, although the ENT clinical phenotype may present great variability. Despite that, data on PCD ENT manifestations are scarce and based on small single-centre studies. To date, we know little about the spectrum and severity of PCD ENT disease, its association with lung disease, its course over life and its determinants of prognosis.This study protocol describes the aims and methods of the first prospective, observational, multinational cohort study focusing on ENT disease in patients with PCD. METHODS AND ANALYSIS: The ENT prospective international cohort of patients with PCD (EPIC-PCD) is a prospective standardised observational clinical cohort set up as a multinational multicentre study, embedded into routine patient care. It aims to longitudinally characterise ENT disease in patients with PCD and its association with lung disease, and to identify determinants of its prognosis. Patients of all ages, diagnosed with PCD who undergo an ENT clinical assessment at least once a year at one of the participating centres will be invited to participate. Collected data include diagnostic test results, results of ENT examinations, lung function measurements, information on management of ENT disease and patient-reported data on clinical symptoms and health-related quality of life (QoL). Data are collected using the standardised PCD-specific FOLLOW-PCD form and the validated QoL-PCD questionnaire. ETHICS AND DISSEMINATION: The study has been reviewed and approved by the Human Research Ethics Committees at all participating centres, based on local legislation. The results of the study will be published in scientific journals, presented at scientific conferences and disseminated to participants and national patient organisations. TRIAL REGISTRATION: NCT04611516.

Preschool HABIT-ILE: study protocol for a randomised controlled trial to determine efficacy of intensive rehabilitation compared with usual care to improve motor skills of children, aged 2-5 years, with bilateral cerebral palsy.

INTRODUCTION: Young children with bilateral cerebral palsy (BCP) often experience difficulties with gross motor function, manual ability and posture, impacting developing independence in daily life activities, participation and quality of life. Hand Arm Bimanual Intensive Training Including Lower Extremity (HABIT-ILE) is a novel intensive motor intervention integrating upper and lower extremity training that has been developed and tested in older school-aged children with unilateral and BCP. This study aims to compare an adapted preschool version of HABIT-ILE to usual care in a randomised controlled trial. METHODS AND ANALYSIS: 60 children with BCP aged 2-5 years, Gross Motor Function Classification System (GMFCS) II-IV will be recruited. Children will be stratified by GMFCS and randomised using concealed allocation to either receive Preschool HABIT-ILE or usual care. Preschool HABIT-ILE will be delivered in groups of four to six children, for 3 hours/day for 10 days (total 30 hours). Children receiving Preschool HABIT-ILE be provided a written home programme with the aim of achieving an additional 10 hours of home practice (total dose 40 hours). Outcomes will be assessed at baseline, immediately following intervention and then retention of effects will be tested at 26 weeks. The primary outcome will be the Peabody Developmental Motors Scales-Second Edition to evaluate gross and fine motor skills. Secondary outcomes will be gross motor function (Gross Motor Function Measure-66), bimanual hand performance (Both Hands Assessment), self-care and mobility (Pediatric Evaluation of Disability Inventory-Computer Adapted Test), goal attainment (Canadian Occupational Performance Measure), global performance of daily activities (ACTIVLIM-CP), cognition and adaptive function (Behavior Rating Inventory of Executive Function-Preschool Version), habitual physical activity (ActiGraph GT3X+) and quality of life (Infant Toddler Quality of Life Questionnaire and Child Health Utility Index-9). Analyses will follow standard principles for RCTs using two-group comparisons on all participants on an intention-to-treat basis. Comparisons between groups for primary and secondary outcomes will be conducted using regression models. ETHICS AND DISSEMINATION: Ethics approval has been granted by the Medical Research Ethics Committee Children's Health Queensland Hospital and Health Service Human Research Ethics Committee (HREC/19/QCHQ/59444) and The University of Queensland (2020000336/HREC/19/QCHQ/59444). TRIAL REGISTRATION NUMBER: ACTRN126200000719.

Hand Function in 8- to 12-Year-Old Children with Bilateral Cerebral Palsy and Interpretability of the Both Hands Assessment.

AIM: To describe bimanual performance in a sample of Australian children with bilateral cerebral palsy (CP) and, examine the qualitative meaning (or interpretability) of scores on the Both Hands Assessment (BoHA). METHODS: Children with bilateral CP aged 8-12 years (n = 54) classified Manual Ability Classification System (MACS) level I = 20, II = 18, III = 16 were examined using the BoHA. RESULTS: Bimanual performance was significantly different across MACS levels I-III (p < 0.001). Mean (95%CI) BoHA-unit for each MACS level were I = 85 (81-89), II = 72 (68-76) and III = 53 (49-56). Children with asymmetrical hand use (≥ 20% difference between upper limbs, n = 10) were classified MACS levels II and III and had a mean (95%CI) BoHA-unit of 56 (51-62). Children with symmetrical hand use were classified in MACS level I-III and had a mean (95%CI) BoHA-unit of 74 (70-79). CONCLUSIONS: The BoHA quantified observations of bimanual performance for children with bilateral CP, differentiated between MACS levels I-III and provided clinically meaningful information. The BoHA may facilitate tailoring of upper limb intervention. Future research is recommended to examine inter-rater and intra-rater reliability and responsiveness of the BoHA, as well as longitudinal studies of bimanual hand skill development in children with bilateral CP.

Hand function and self-care in children with cerebral palsy.

AIM: To examine the relationship between self-care and bimanual performance in children aged 8 to 12 years with cerebral palsy (CP). METHOD: This was a cross-sectional study of 74 children with CP (unilateral n=30, bilateral n=44; 48 males, 26 females; median age 9y 8mo [25th, 75th centiles 9y 1mo, 10y 8mo], Manual Abilities Classification System level I=30, II=28, III=16). Self-care was measured using the Pediatric Evaluation of Disability Inventory-Computer Adaptive Test (PEDI-CAT), and bimanual performance using the Assisting Hand Assessment (AHA) and Both Hands Assessment (BoHA). Measures of cognition, behavioural regulation, inattention, and gross motor function were included. Analyses used a directed acyclic graph to select variables for linear regression modelling. RESULTS: Higher AHA and BoHA scores were associated with higher PEDI-CAT scores. An increase of 1 AHA unit was associated with an increase of 0.12 PEDI-CAT scores, and a 1 BoHA unit increase was associated with an increase of 0.17 PEDI-CAT scores. The BoHA accounted for 57% of variance in PEDI-CAT scores for children with bilateral CP, while BoHA and cognition accounted for 68% of variance. The AHA accounted for 40% of variance in PEDI-CAT scores for unilateral CP with no effect of cognition on self-care. INTERPRETATION: Self-care was strongly and positively associated with bimanual performance. Associations between self-care and bimanual performance differed for those with unilateral and bilateral CP. WHAT THIS PAPER ADDS: There is a strong positive relationship between self-care and bimanual performance for unilateral and bilateral cerebral palsy (CP). Both Hands Assessment (BoHA) scores have a stronger association with self-care than Assisting Hand Assessment scores. BoHA scores also account for more variation in self-care. There is a strong positive relationship between self-care and cognition overall. The effect of cognition on self-care performance differed for bilateral and unilateral CP.

Self-care performance in children with cerebral palsy: a longitudinal study.

AIM: To investigate self-care developmental trajectories in children with cerebral palsy (CP) across all functional ability levels, according to Manual Ability Classification System (MACS) levels. METHOD: This was a prospective longitudinal population-based study of 71 children aged from 2 years 6 months to 12 years, with CP (47 [66%] males, 24 [34%] females). Pediatric Evaluation of Disability Inventory (PEDI) measures were taken at 2 years 6 months, 3, 4, and 5 years, and the PEDI - Computer Adaptive Test (PEDI-CAT) between 8 and 12 years. At 8 to 12 years, children were classified in MACS levels I (21; 30%), II (22; 31%), III (16; 23%), IV (6; 8%), and V (6; 8%). Longitudinal analysis of the PEDI Functional Skills Scale self-care and PEDI-CAT daily activities domains used the published linking equation, and multilevel mixed-effects regression modelling with interaction between age and MACS. RESULTS: Between 5 and 12 years of age, children classified in MACS levels I to III continued to show progress in self-care development (PEDI-CAT scaled scores estimated change per year: I, 0.72; II, 0.49; III, 0.48). Children classified in MACS level IV showed an upward non-significant trend between 5 and 8 to 12 years (estimated change 0.42; 95% confidence interval [CI] -0.04 to 0.88). Children in MACS level V showed a decline in self-care (estimated change: -0.65; 95% CI -1.16 to -0.14). INTERPRETATION: Self-care development attained by 8 to 12 years of age was related to the severity of manual ability impairment. Application of the linking equation from PEDI to PEDI-CAT is somewhat uncertain at the extreme lower end of the scale. Our study supports recommendations for items to be added to the PEDI-CAT to address floor effect.

A systematic review of upper limb activity measures for 5- to 18-year-old children with bilateral cerebral palsy.

AIM: To investigate measurement properties and feasibility of upper limb activity measures in children aged 5-18 years with bilateral cerebral palsy (CP). METHODS: Five electronic databases were searched to identify measures of upper limb activity with published psychometric data for children with bilateral CP aged 5-18 years. Measures included both Patient-Reported Outcome Measures (PROMs) and observational measures. The COnsensus-based Standards for selection of health Measurement Instruments checklist was used to evaluate methodological quality of studies for each measure. RESULTS: Forty-eight measures were identified, eight of which met inclusion criteria for reliability and validity. Four PROMs were included: the ABILHAND-Kids and Children's Arm Rehabilitation Measure are parent questionnaires measuring overall manual ability; the ACTIVLIM-CP is a parent questionnaire measuring global activity (upper and lower extremity) performance, and, the Pediatric Upper Limb Measure, Short Form is a child self-report questionnaire. Four observational measures were included: the Both Hands Assessment (BoHA) is an observational measure of bimanual activity performance; the Melbourne Assessment of Unilateral Upper Limb Function and the Melbourne Assessment 2 measure quality of movement of each upper limb separately, and the Peabody Developmental Motor Scales-Second Edition assesses fine motor skill capacity in young children. Based upon available evidence, the most suitable PROM for evaluation of upper limb activity in children with bilateral CP is the ACTIVLIM-CP, and the most suitable observational measure is the BoHA. CONCLUSION: Selection of upper limb measures depend on clinical information required and available resources. The BoHA is the only observational-based assessment which measures bimanual upper limb activity performance in children with bilateral CP. Recommendation for future measurement studies include familiarisation with the standards required for excellence, which include adequate sample size and content validity studies for PROMs.

Non-selective laryngeal reinnervation in a child with unilateral left vocal fold palsy utilizing laryngeal electromyography.

INTRODUCTION: The impact of unilateral vocal fold palsy (UVFP) on quality of life cannot be underestimated. Management may be complicated by difficulty in determining prognosis. Currently, there is no standardized management pathway for UVFP. Surgery is considered when speech and language therapy has not been successful or when there is significant aspiration and dysphonia. Surgical options for UVFP include injection laryngoplasty, thyroplasty and laryngeal reinnervation. METHODS: We report the case of a 6-year-old girl with a left UVFP sustained following PDA ligation. She suffered significant voice issues, aspiration and intermittent stridor limiting activities. Following multidisciplinary team reassessment including videofluoroscopy and perceptual and objective voice measures, laryngotracheobronchoscopy (LTB) and laryngeal electromyography (LEMG) with injection of Radiesse into the left vocal fold was undertaken. Consequently, she underwent non-selective laryngeal reinnervation with the aim of providing a permanent solution by the formation of an anastomosis between the ansa cervicalis and the main stump of the recurrent laryngeal nerve (RLN) resulting in improved muscle tone. RESULTS: LEMG indicated no spontaneous recovery. The laryngeal injection allowed for temporary improvement of voice and feeding. Consequently non-selective left laryngeal reinnervation using ansa cervicalis and repeat vocal cord injection was performed. Twelve months following surgery her voice remain greatly improved and there are no feeding issues or aspiration. CONCLUSION: In this case so far the use of LEMG and laryngeal reinnervation has proved successful. Non-selective laryngeal reinnervation techniques for UVFP may provide a permanent solution and should be considered in children as a management option in suitable cases.