Intrathecal baclofen therapy in paediatrics : A study protocol for an Australian multicentre, 10-year prospective audit

Introduction Increasing clinical use of Intrathecal baclofen (ITB) in Australian tertiary paediatric hospitals, along with the need for standardised assessment and reporting of adverse events, saw the formation of the Australian Paediatric ITB Research Group (APIRG). APIRG developed a National ITB Audit tool designed to capture clinical outcomes and adverse events data for all Australian children and adolescents receiving ITB therapy. Methods and analysis The Australian ITB Audit is a 10 year, longitudinal, prospective, clinical audit collecting all adverse events and assessment data across body functions and structure, participation and activity level domains of the ICF. Data will be collected at baseline, 6 and 12 months with ongoing capture of all adverse event data. This is the first Australian study that aims to capture clinical and adverse event data from a complete population of children with neurological impairment receiving a specific intervention between 2011 and 2021. This multi-centre study will inform ITB clinical practice in children and adolescents, direct patient selection, record and aid decision making regarding adverse events and investigate the impact of ITB therapy on family and patient quality of life. Ethics and dissemination This project was approved by the individual Human Research Ethics committees at the six Australian tertiary hospitals involved in the study. Results will be published in various peer reviewed journals and presented at national and international conferences. Trial registration number ACTRN 12610000323022; Pre-results

Blinding and bias in randomized controlled trials: when to measure the effectiveness of blinding

This article is commented on by Day on page 159 of this issue

Development of a Novel Ceramic Support Layer for Planar Solid Oxide Cells

OBJECTIVE: To determine safety of intramuscular botulinum toxin A (BoNT-A) injections to reduce spasticity and improve care and comfort of nonambulatory children with cerebral palsy (CP)

Evaluation of the effects of botulinum toxin A injections when used to improve ease of care and comfort in children with cerebral palsy whom are non-ambulant: a double blind randomized controlled trial

Abstract Background Children with cerebral palsy (CP) whom are non-ambulant are at risk of reduced quality of life and poor health status. Severe spasticity leads to discomfort and pain. Carer burden for families is significant. This study aims to determine whether intramuscular injections of botulinum toxin A (BoNT-A) combined with a regime of standard therapy has a positive effect on care and comfort for children with CP whom are non-ambulant (GMFCS IV/V), compared with standard therapy alone (cycle I), and whether repeated injections with the same regime of adjunctive therapy results in greater benefits compared with a single injecting episode (cycle II). The regime of therapy will include serial casting, splinting and/or provision of orthoses, as indicated, combined with four sessions of goal directed occupational therapy or physiotherapy. Method/design This study is a double blind randomized controlled trial. Forty participants will be recruited. In cycle I, participants will be randomized to either a treatment group who will receive BoNT-A injections into selected upper and/or lower limb muscles, or a control group who will undergo sham injections. Both groups will receive occupational therapy and /or physiotherapy following injections. Groups will be assessed at baseline then compared at 4 and 16 weeks following injections or sham control. Parents, treating clinicians and assessors will be masked to group allocation. In cycle II, all participants will undergo intramuscular BoNT-A injections to selected upper and/or lower limb muscles, followed by therapy. The primary outcome measure will be change in parent ratings in identified areas of concern for their child’s care and comfort, using the Canadian Occupational Performance Measure (COPM). Secondary measures will include the Care and Comfort Hypertonicity Scale (ease of care), the Cerebral Palsy Quality of Life Questionnaire (CP QoL–Child) (quality of life), the Caregiver Priorities and Child Health Index of Life with Disabilities Questionnaire (CPCHILD©) (health status) and the Paediatric Pain Profile (PPP) (pain). Adverse events will be carefully monitored by a clinician masked to group allocation. Discussion This paper outlines the theoretical basis, study hypotheses and outcome measures for a trial of BoNT-A injections and therapy for children with non-ambulant CP. Trial registration Australia New Zealand Clinical Trials Registry:N12609000360213</p

Efficacy of cycling interventions to improve function in children and adolescents with cerebral palsy: a systematic review and meta-analysis

Objectives: The aim of this study was to determine the efficacy of cycling to improve function and reduce activity limitations in children with cerebral palsy; the optimal training parameters for improved function; and whether improvements in function can be retained. Method: Six databases were searched (until February 2019) and articles were screened in duplicate. Randomized or quasi-randomized controlled trials and pre–post studies were included. Methodological quality was assessed using the Downs and Black scale. Outcomes were reported under the International Classification of Functioning, Disability and Health domains of body functions and activity limitations. Quantitative analyses were completed using RevMan V5.3. Results: A total of 533 articles were identified and 9 studies containing data on 282 participants met full inclusion criteria. Methodological quality ranged from low (14 of 32) to high (28 of 32). Significant improvements were reported for hamstring strength (effect size = 0.77–0.93), cardiorespiratory fitness (effect size = 1.13–1.77), balance (effect size = 1.03–1.29), 3-minute walk test distance (effect size = 1.14) and gross motor function (effect size = 0.91). Meta-analysis suggested that cycling can improve gross motor function (standardized mean difference = 0.35; 95% confidence interval = (−0.01, 0.70); P = 0.05); however, the effect was insignificant when a poor-quality study was omitted. Conclusion: Cycling can improve muscle strength, balance and gross motor function in children with cerebral palsy; however, optimal training doses are yet to be determined. There was insufficient data to determine whether functional improvements can be retained. Conclusions were limited by small sample sizes, inconsistent outcome measures and a lack of follow-up testing

Functional electrical stimulation cycling, goal‐directed training, and adapted cycling for children with cerebral palsy: a randomized controlled trial

Aim: To test the efficacy of functional electrical stimulation (FES) cycling, goal-directed training, and adapted cycling, compared with usual care, to improve function in children with cerebral palsy (CP). Method: The intervention was delivered between 2017 and 2019 and included three sessions per week for 8 weeks (2×1h sessions at a children’s hospital, and 1h home programme/week). Hospital sessions included 30 minutes of FES cycling and 30 minutes of goal-directed training. Home programmes included goal-directed training and adapted cycling. The comparison group continued usual care. Primary outcomes were gross motor function assessed by the Gross Motor Function Measure (GMFM) and goal performance/satisfaction assessed using the Canadian Occupational Performance Measure (COPM). Secondary outcomes were sit-to-stand and activity capacity, participation in home, school, and community activities, and power output. Linear regression was used to determine the between-group mean difference immediately post-training completion after adjusting for baseline scores. Results: This randomized controlled trial included 21 participants (mean age=10y 3mo, standard deviation [SD]=3y; Gross Motor Function Classification System level: II=7, III=6, IV=8) who were randomized to the intervention (n=11) or usual care group (n=10). Between-group differences at T2 favoured the intervention group for GMFM-88 (mean difference=7.4; 95% confidence interval [CI]: 2.3–12.6; p=0.007), GMFM-66 (mean difference=5.9; 95% CI: 3.1–8.8;

ACTIVE STRIDES-CP: protocol for a randomised trial of intensive rehabilitation (combined intensive gait and cycling training) for children with moderate-to-severe bilateral cerebral palsy

Introduction For children with cerebral palsy (CP), who are marginally ambulant, gross motor capacity peaks between 6 and 7 years of age with a subsequent clinical decline, impacting their ability to engage in physical activity. Active Strides-CP is a novel package of physiotherapy targeting body functions, activity and participation outcomes for children with bilateral CP. This study will compare Active Strides-CP to usual care in a multisite randomised waitlist-controlled trial.Methods and analysis 150 children with bilateral CP (5–15 years), classified in Gross Motor Function Classification System (GMFCS) levels III and IV will be stratified (GMFCS III vs IV, age 5–10 years; 11–15 years and trial site) and randomised to receive either (1) 8 weeks of Active Strides-CP two times/week for 1.5 hours in clinic and one time/week for 1 hour alternating home visits and telehealth (total dose=32 hours) or (2) usual care. Active Strides-CP comprises functional electrical stimulation cycling, partial body weight support treadmill training, overground walking, adapted community cycling and goal-directed training. Outcomes will be measured at baseline, immediately post-intervention at 9 weeks primary endpoint and at 26 weeks post-baseline for retention. The primary outcome is the Gross Motor Function Measure-66. Secondary outcomes include habitual physical activity, cardiorespiratory fitness, walking speed and distance, frequency/involvement of community participation, mobility, goal attainment and quality of life. Analyses will follow standard principles for randomised controlled trials 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. A within-trial cost utility analysis will be performed.Ethics and dissemination The Children’s Health Queensland Hospital and Health Service, The University of Queensland, The University of Melbourne and Curtin University Human Research Ethics Committees have approved this study. Results will be disseminated as conference abstracts and presentations, peer-reviewed articles in scientific journals, and institution newsletters and media releases.Trial registration number ACTRN12621001133820

Evaluation of group versus individual physiotherapy following lower limb intra-muscular Botulinum Toxin-Type A injections for ambulant children with cerebral palsy: A single-blind randomized comparison trial

© 2016 Elsevier Ltd. This study aimed to evaluate efficacy of group (GRP) versus individual (IND) physiotherapy rehabilitation following lower limb intramuscular injections of Botulinum Toxin-Type A (BoNT-A) for ambulant children with cerebral palsy (CP). Following lower limb BoNT-A injections, 34 children were randomly allocated to GRP (n = 17; mean age 7y8m SD 2.0; 13 males; Gross Motor Function Classification System (GMFCS) I = 5, II = 8, III = 4) or IND physiotherapy (n = 17; mean age 8y7m SD 2.0; 11 males; GMFCS I = 9, II = 5, III = 3). Primary outcomes were the Canadian Occupational Performance Measure (COPM) and Edinburgh Visual Gait Score (EVGS) assessed at baseline, 10 and 26 weeks post intervention. There were no baseline differences between groups. GRP intervention had greater, but not clinically meaningful, improvement in COPM satisfaction (estimated mean difference EMD 1.7, 95% CI 0.4-3.1; p &lt; 0.01) at 26 weeks. Both groups demonstrated clinically significant improvements in COPM performance and satisfaction, but minimal change in quality of gait (EVGS). Six hours of direct physiotherapy (either GRP or IND) with an additional indirect dose (median 16 episodes) of individualized home programme activities following lower limb BoNT-A injections, however, was inadequate to drive clinically meaningful changes in lower limb motor outcomes