Non-attendance at outpatient clinic appointments by children with cerebral palsy.

AIM: To determine factors that influence non-attendance at outpatient clinics by children with cerebral palsy (CP). METHOD: This was a retrospective cohort study of 1395 children with CP (59.6% male; born 2005 to 2017) identified from the New South Wales (NSW)/Australian Capital Territory CP Register, who had scheduled appointments at outpatient clinics at two NSW tertiary paediatric hospitals between 2012 and 2019. Associations between sociodemographic, clinical, and process-of-care factors and non-attendance were examined using multivariate logistic regression with generalized estimating equations. RESULTS: A total of 5773 (12%) of 50 121 scheduled outpatient days were not attended. Non-attendance increased over time (average increase 5.6% per year, 95% confidence interval [CI]: 3.7-7.3). Older children aged 5 to 9 years (adjusted odds ratio [aOR] 1.11; 95% CI: 1.02-1.22) and 10 to 14 years (aOR 1.17; 95% CI: 1.03-1.34), socioeconomic disadvantage (aOR 1.29; 95% CI: 1.11-1.50), previous non-attendance (aOR 1.38; 95% CI: 1.23-1.53), and recent rescheduled or cancelled appointments (aOR 1.08; 95% CI: 1.01-1.16) were associated with increased likelihood of non-attendance. INTERPRETATION: One in eight outpatient appointments for children with CP were not attended. Non-attendance was associated with increasing age, socioeconomic disadvantage, previous non-attendance, and recent rescheduled or cancelled appointments. Identifying specific barriers and interventions to improve access to outpatient services for these groups is needed. WHAT THIS PAPER ADDS: Twelve per cent of scheduled appointments for children with cerebral palsy are not attended. Proportions of appointments not attended has increased over the last decade. Increasing age and socioeconomic disadvantage increase the likelihood of non-attendance. Previous non-attendance and recent cancelled or rescheduled appointments increase the likelihood of further non-attendance.

A pilot feasibility study of gabapentin for managing pain in children with dystonic cerebral palsy.

BACKGROUND: Gabapentin is often used to manage pain in children with dystonic cerebral palsy, however the evidence for its effectiveness in this population is limited. The primary objective of this feasibility pilot study was to assess the factors which might impact on a future randomised controlled trial including the ability to recruit and retain participants, assess adherence/compliance to the prescribed intervention, and ability to complete all outcome assessments. The secondary objective was to gather preliminary evidence for the effectiveness of gabapentin at reducing pain, improving comfort and reducing dystonia in children with dystonic cerebral palsy. METHODS: This open label pilot study recruited children aged 5-18 years with dystonic cerebral palsy and accompanying pain affecting daily activities from four centres around Australia. Children were prescribed gabapentin for 12 weeks and were assessed at baseline, 6 weeks and 12 weeks. The primary outcome was feasibility of the protocol. Secondary outcomes were pain behaviour, pain intensity, care and comfort, individualised goal setting and dystonia severity. RESULTS: Thirteen children (mean age 10.4 years (SD 2.4yrs), 9 females) were recruited from 71 screened over 15 months. Two children withdrew while eight children experienced side effects. There were issues with adherence to medication dosage regimens and data collection. Improvements were seen in pain behaviour, comfort and pain related goals at 12 weeks. Dystonia was not significantly changed. CONCLUSIONS: Whilst gabapentin has potential to improve pain and comfort in children with dystonic CP, the feasibility of implementing a definitive randomised controlled trial is low. Alternative trials designs are required to further examine the effectiveness of gabapentin in this heterogeneous population. TRIAL REGISTRATION: The trial was registered with the Australian Clinical Trial Registry ( ACTRN12616000366459 ) on 22/03/2016 and the Therapeutic Goods Administration (CT-2016-CTN-00500-1) on 22/06/2016.

Adverse events associated with paediatric intrathecal baclofen in Australia: 5 years of data collection.

AIM: This study aims to examine the adverse event (AE) rate for intrathecal baclofen (ITB) therapy in an Australian paediatric population and to clarify type and frequency of AEs. METHODS: AE data were extracted from the Australian Paediatric ITB Research Group national database, to include the first 5 years of data collection. Raw data were collated and analysed descriptively. RESULTS: Data were collected for 40 patients. Forty-seven AEs in 23 patients were reported. Ten (25%) patients required surgical intervention related to their AE. Five patients (12.5%) required pump removal. The most frequent ITB-related AEs were catheter dysfunction (24%), drug overdose, withdrawal or sensitivity (19%), seromas and haematomas (15%) and infections (13%). CONCLUSIONS: The AE rate for ITB therapy is high and needs to be considered when counselling patients regarding ITB as a therapeutic option.

National surveillance of oral medication prescription for children with dystonic cerebral palsy.

AIM: Oral medications are often first-line medical management for children with cerebral palsy who have generalised dystonia; however, evidence for their effectiveness is limited and dosing practices are inconsistent. As a first step to improve consistency, this study aimed to examine current clinical practice of expert doctors for prescribing medications for children with dystonic cerebral palsy including prescribing patterns and combinations of medications used. METHODS: This was a prospective surveillance study of medical doctors working in major Australian centres who manage children with cerebral palsy. Each week over a continuous 6-month period, doctors completed a custom developed online survey for children seen that week with dystonic cerebral palsy for whom they prescribed a new medication to treat dystonia. RESULTS: Twenty-five doctors consented to participate, 16 of whom prescribed new medications for dystonia in children with cerebral palsy over the study period. There were 77 children who were prescribed new medications. Baclofen and gabapentin were prescribed most, followed by levodopa, trihexyphenidyl and diazepam. The most common combinations of medications were baclofen and diazepam or baclofen and gabapentin. Dosage regimens were variable, particularly for trihexyphenidyl and diazepam. CONCLUSION: Inconsistencies in dosing regimens remain for oral medication prescription by Australian doctors when managing dystonia in cerebral palsy. Future studies using the consensus of expert clinicians will be conducted to develop guidelines in an area where the evidence for individual medications is extremely limited.

Transition to adult care for young people with cerebral palsy.

Cerebral palsy (CP) is associated with a high burden of comorbid respiratory disease subsequent to multiple risk factors associated with increasing levels of disability. Correspondingly, respiratory disease is the leading cause of death in CP, including amongst young people who are transitioning or who have just transitioned between paediatric and adult healthcare services. Therefore, consideration of both preventive and therapeutic respiratory management is integral to transition in patients with CP, as summarised in this review.

Global mapping for the epidemiology of paediatric spinal cord damage: towards a living data repository.

STUDY DESIGN: Literature review. OBJECTIVES: Globally map key paediatric spinal cord damage epidemiological measures and provide a framework for an ongoing repository of data. SETTING: Worldwide, initiative of ISCoS Prevention Committee. METHODS: Literature search of Medline (1946-March 2017) and Embase (1974-March 2017). Relevant articles in any language regarding children with spinal cord damage included. Stratification of data quality into Green/Yellow /Red 'zones' facilitated comparison between countries. RESULTS: A total of 862 abstracts were reviewed and data from 25 articles were included from 14 countries in 6 of the 21 Global Regions. Fourteen studies involved paediatric traumatic spinal cord injury (SCI) and seven were regarding non-traumatic spinal cord dysfunction (SCDys). An additional four articles provided both paediatric SCI and SCDys data. The median SCI incidence rates in Global Regions were: Asia, East 5.4/million population/year; Australasia 9.9/million population/year; Western Europe 3.3/million population/year and North America, High Income 13.2 million population/year. The median SCDys incidence rates in Global Regions were: Australasia 6.5/million population/year; Western Europe 6.2/million population/year and North America, High Income 2.1/million population/year. SCI was mostly due to land transport (46-74%), falls (12-35%) and sport/recreation (10-25%) and SCDys was mostly caused by tumours (30-63%) and inflammatory/autoimmune causes (28-35%). CONCLUSIONS: There is a scarcity of quality epidemiology studies of paediatric SCD regarding incidence, prevalence, aetiology and survival. Recent ISCoS frameworks provide guidance for researchers to use established classification of SCDys and age group cut-off levels in future studies, thereby improving the ability to compare and combine data.

Orthopaedic Surgery in Dystonic Cerebral Palsy.

BACKGROUND: Outcomes after orthopaedic interventions in patients with dystonic cerebral palsy (DCP) are historically regarded as unpredictable. This study aims to evaluate the overall outcome of orthopaedic surgery in children with DCP. METHOD: Children with DCP who underwent lower limb orthopaedic surgery with a minimum follow-up of 12 months were included. Data collected included age at time of surgery, surgical procedures performed, Gross Motor Function Classification System (GMFCS) level, and Barry Albright Dystonia Scale (BADS) score. The cohort was divided into 2 groups. Group 1 (GMFCS levels I to III), mean age 12 years 7 months and group 2 (GMFCS levels IV to V), mean age 10 years 7 months. Group 1 had surgery aimed at deformity correction to improve gait and mobility, and group 2 for the management or prevention of hip displacement. Outcome measures analyzed were: the incidence of unpredictable results related to surgery and early recurrence of deformity in both groups. Functional mobility scale scores were evaluated for group 1 and hip migration percentage for group 2. Linear mixed models were used to take into account repeated measures over time and correlations between measurements from the same patient. RESULTS: Group 1 (n=18); had low BADS scores and were considered to have mild dystonia. Three children experienced unpredictable results, 2 had early recurrence of deformity, 3 had a decline, and 1 child improved in the functional mobility scale.Group 2 (n=19); had high BADS scores and were considered to have moderate to severe dystonia. Nine surgical events involved bony procedures and 15 were soft tissue surgery only. One surgical event lead to unpredictable results and 2 children had early recurrence of deformity. Postoperatively, a linear trend of increasing migration percentage [0.49% (95% confidence interval, 0.23-0.74; P=0.0002)] was seen up to 21 months. There was no significant change after 21 months [-0.08% (95% confidence interval, -0.24 to +0.041; P=0.18)]. CONCLUSIONS: This study suggests that unpredictable results and early recurrence of deformity following orthopaedic surgery in children with DCP are not as common as previously regarded. Furthermore, functional mobility and hip morphology can be improved. LEVEL OF EVIDENCE: Level IV-this is a case-series.

Evidence- and Consensus-Based Guidelines for the Management of Communication and Swallowing Disorders Following Pediatric Traumatic Brain Injury.

OBJECTIVE: Evidence-based management guidelines for communication and swallowing disorders following pediatric traumatic brain injury (TBI) are scarcely available, potentially resulting in suboptimal outcomes. To improve clinical care of this population, a multidisciplinary guideline development committee was formed to develop evidence-based recommendation (EBR) and consensus-based recommendation (CBR) for the management of speech, language, and swallowing disorders during the first year of recovery. METHODS: A 3-round Delphi survey was completed by the committee to reach agreement (80% consensus) for the CBRs. Systematic review evidence guided development of EBRs, devised using the National Health and Medical Research Council statement form. RESULTS: Altogether, 30 recommendations (5 EBRs and 25 CBRs) were developed to guide management of speech, language, and swallowing disorders, including prediction of these disorders; health team required, optimal timing of assessment; assessment tools; intervention strategies and commencement of treatment; and key information to support parents. CONCLUSION: The developed recommendations provide a basis for the systematic management of communication and swallowing disorders to be refined as new evidence emerges. Key recommendations include screening of children with moderate/severe TBI for these disorders acutely using specified measures. Patients with severe TBI and prolonged ventilation are a particular at-risk group and should be considered for early referral to speech-language pathology to support timely diagnosis and management. No evidence was identified to support an EBR for treatment, highlighting a key area for research.

Central nervous system stimulants for secondary attention deficit-hyperactivity disorder after paediatric traumatic brain injury: a rationale and protocol for single patient (n-of-1) multiple cross-over trials.

BACKGROUND: It is estimated that 22,800 children were living with an Acquired Brain Injury (ABI) (0.6% of children aged under 15 years) in Australia during 2003. Many children after a traumatic brain injury will experience difficulties with attention and concentration; a condition termed secondary Attention Deficit-Hyperactivity Disorder. There is conflicting evidence on whether treatment with stimulant therapy with medications such as methylphenidate or dexamphetamine will improve the attention and behavior of children with this condition. METHODS/DESIGN: Single patient trials (n-of-1s or SPTs) evaluate the effect of titrated doses of psychostimulants methylphenidate or dexamphetamine compared to placebo on attention and behavior, in children with TBI and secondary ADHD. The aggregation of multiple SPTs will produce a population estimate of the benefit. Forty-two children will be registered into the trial through rehabilitation services at three large children's hospitals in Australia. Patients will complete up to 3 cycles of treatment. Each cycle is 2 weeks long comprising seven days each of treatment and placebo, with the first two days of each cycle considered a washout period and the data not analysed. The order of treatment and placebo is randomly allocated for each cycle. The Conners' Parent Rating Scales long forms will be employed to measure change in attention-deficit/hyperactivity and related problems of the child, and the primary outcome measure is the Conners' Global Index Parent Version. Secondary outcomes include the teacher and child (if aged > 12 years) Conners' Rating Scales, the Behaviour Rating Inventory of Executive Function among other measures. This study will provide high-level evidence using a novel methodological approach to inform clinicians about the most appropriate treatment for individual children. Through aggregation of individual trials, a population estimate of treatment effect will be provided to guide clinical practice in the treatment of children with secondary ADHD after a traumatic brain injury. DISCUSSION: This study employs an innovative methodological approach on the effectiveness of CNS stimulants for secondary ADHD from a brain injury. The findings will both guide clinicians on treatment recommendations, and inform the concept and acceptance of SPTs in paediatric research. TRIAL REGISTRATION: Australian New Zealand Clinical Trials Registry. ACTRN12609000873224.