Evolving understanding of CP phenotypes: the importance of dystonia.

Cerebral palsy (CP) is the core neurodevelopmental disorder affecting movement. Several distinct movement disorders can occur in people with cerebral palsy. Dystonia is a movement disorder that causes non-velocity-dependent hypertonia and/or abnormal, often repetitive, twisting movements, and/or postures. Dystonia occurs more frequently in patients with CP than has been recognized previously, and is treated differently than other aspects of CP. Dystonia is an important cause of chronic pain, hospitalization, and musculoskeletal complications. We describe recent advances in dystonia diagnosis in patients with cerebral palsy and highlight focus areas for ongoing research and clinical care. IMPACT: Dystonia is a movement disorder that is more common in people with cerebral palsy (CP) than previously thought. Dystonia contributes to hospitalization, chronic pain, and complications in CP patients. People with dystonic CP require different tools to diagnose and treat their condition. We summarize current state of the art in dystonia in CP and identify areas of focus for future work.

Pharmacological and neurosurgical management of cerebral palsy and dystonia: Clinical practice guideline update.

Dystonia, typically characterized by slow repetitive involuntary movements, stiff abnormal postures, and hypertonia, is common among individuals with cerebral palsy (CP). Dystonia can interfere with activities and have considerable impact on motor function, pain/comfort, and ease of caregiving. Although pharmacological and neurosurgical approaches are used clinically in individuals with CP and dystonia that is causing interference, evidence to support these options is limited. This clinical practice guideline update comprises 10 evidence-based recommendations on the use of pharmacological and neurosurgical interventions for individuals with CP and dystonia causing interference, developed by an international expert panel following the Grading of Recommendations, Assessment, Development and Evaluations (GRADE) approach. The recommendations are intended to help inform clinicians in their use of these management options for individuals with CP and dystonia, and to guide a shared decision-making process in selecting a management approach that is aligned with the individual's and the family's values and preferences.

Cognitive behavioral therapies for individuals with cerebral palsy: A scoping review.

AIM: To synthesize the evidence about the main intervention characteristics of cognitive behavioral therapies (CBTs) for individuals with cerebral palsy and identify barriers and facilitators to their success, focusing on aspects of feasibility and markers of success. METHOD: A scoping review methodology informed a literature search for papers published between 1991 and 2021. Articles were screened, reviewed, and categorized using the DistillerSR systematic review software, and critically appraised for quantitative and/or qualitative criteria. RESULTS: Out of 1265 publications identified, 14 met the inclusion criteria. Elements associated with the specific study participant characteristics (46% female; aged 6-65 years), type of CBT techniques used (third-wave [n = 6], cognitive [n = 3], cognitive and behavioral [n = 2], biofeedback training [n = 2]), and features of the study context and methodological quality (two randomized clinical trials and small sample sizes [n ≤ 12]), were identified. Most studies had psychological targets of intervention (n = 10) and secondary physiological (n = 3) or social (n = 2) objectives. Feasibility indicators were described in nearly one-third of the papers. INTERPRETATION: This study highlights the high flexibility within CBT interventions, enabling their adaptation for individuals with cerebral palsy. However, relatively little, and only low-certainty evidence was identified. More high-quality research in terms of specific CBT techniques, optimal treatment doses, and detailed population characteristics are needed.

Clinical course of pain intensity in individuals with cerebral palsy: A prognostic systematic review.

AIM: To describe the clinical course of pain intensity in individuals with cerebral palsy (CP) resulting from usual care or specific interventions. METHOD: We conducted an exploratory prognostic systematic review searching electronic databases from inception to 31st December 2021. Evidence from low and moderate risk-of-bias studies was synthesized. RESULTS: We retrieved 2275 citations; 18 studies met the inclusion criteria and 10 were synthesized. The course of pain intensity in children with CP receiving usual care was stable over 15 weeks (χ2 [2]  = 1.8, p = 0.5). Children who received continuous intrathecal baclofen (CITB) reported significant pain intensity reduction (visual analogue scale [VAS] = -4.2 out of 10, 95% confidence interval [CI] = -6.3 to -2.1]) 6 months postinsertion but similar children receiving usual care had no significant change over 6 months (VAS = 1.3 out of 10, 95% CI = -1.3 to 3.6). Children receiving botulinum neurotoxin A (BoNT-A) injections had significant decreases in pain after 1 month (numeric rating scale = -6.5, 95% CI = -8.0 to -5.0). Adults with chronic pain receiving usual care reported stable pain intensity over time; pain intensity improved in ambulatory adults exercising and those treated surgically for cervical myelopathy. INTERPRETATION: The course of pain intensity in individuals with CP is unclear. Evidence suggests that children and adults receiving usual care had stable pain intensity over the short or long term. Interventions (CITB and BoNT-A in children and exercise and surgical treatment for cervical myelopathy in adults) had pain intensity reduction. Larger study samples are needed to confirm these results. WHAT THIS PAPER ADDS: Pain intensity was stable in children with cerebral palsy (CP) receiving usual care. Adults with CP and chronic pain receiving usual care had stable, persistent pain intensity. Children receiving continuous intrathecal baclofen via pump and botulinum neurotoxin A reported significantly lower pain intensities. Adults with chronic pain and dyskinetic CP and cervical myelopathy reported significantly lower pain intensity with exercise or cervical decompression. Limited high-quality evidence exists describing non-procedural pain changes in individuals with CP.

Gross motor change after inpatient rehabilitation for children with acquired brain injury: A 10-year retrospective review.

AIM: To estimate gross motor change in inpatient school-aged children with subacute acquired brain injury (ABI), identify factors associated with gross motor change, and describe inpatient physiotherapy focus. METHOD: This retrospective chart review involved inpatient children (5-18 years) with subacute ABI who had either two Gross Motor Function Measure (GMFM-88) assessments or one GMFM-88 with another pre/post gross motor outcome measure. Outcome change scores and Goal Attainment Scaling (GAS) T scores were calculated. Regression analyses examined factors predicting gross motor change. GAS goal areas were analysed to determine physiotherapy focus. RESULTS: Of the 546 charts screened, 266 (118 female) met study criteria. The GMFM-88 was generally administered first, followed by other measures. GMFM-88 (n = 202), Community Balance and Mobility Scale (n = 89), and Six-Minute Walk Test (6MWT) (n = 98) mean change scores were 18.03% (SD 19.34), 17.85% (SD 10.77), and 142.3 m (SD 101.8) respectively. The mean GAS T score was 55.06 (SD 11.50). Lower baseline scores and increased time between assessments were most predictive of greater GMFM-88 change (r ≥ 0.40). Twenty-five percent of GAS goals were ambulation-based. INTERPRETATION: Appropriate outcome measure selection is integral to detecting gross motor change in pediatric inpatient ABI rehabilitation. Mean change score estimates can be used to compare standard inpatient rehabilitation with new treatment approaches.

Integrating Transcranial Direct Current Stimulation into an Existing Inpatient Physiotherapy Program to Enhance Motor Learning in an Adolescent with Traumatic Brain Injury: A Case Report.

PURPOSE: Describe how transcranial direct current stimulation (tDCS) was incorporated into an inpatient physiotherapy program for an adolescent with severe traumatic brain injury (TBI), detail the motor learning focus of the physiotherapy sessions, and summarize gross motor progress. METHOD: This case report describes an adolescent who received 20 minutes of anodal tDCS immediately prior to 16 physiotherapy sessions over four weeks. Potential side effects were tracked pre/post tDCS. Gross motor outcomes were measured pre-intervention, post-intervention, and three months post-intervention. Physiotherapy session content was analyzed using therapist documentation and the Motor Learning Strategies Rating Instrument. RESULTS: The youth tolerated tDCS well. The primary side effect was itchiness under the electrodes during tDCS sessions. His mobility progressed from wheelchair use pre- 'tDCS + physiotherapy' to ambulation with a walker post-intervention. His Gross Motor Function Measure score increased 33.1% points pre/post intervention. Session tasks often had several foci (e.g., skill acquisition, strength, and balance) with task focus changing as the youth progressed. Various motor learning strategies were layered within tasks to support performance and learning. CONCLUSIONS: tDCS was successfully integrated into an existing inpatient physiotherapy program for an adolescent with TBI. This protocol provides a structure for implementing, monitoring, and measuring tDCS + physiotherapy in pediatric rehabilitation.

Carrying the Burden: Informal Care Requirements by Caregivers of Children with Intestinal Failure Receiving Home Parenteral Nutrition.

OBJECTIVE: To measure the time that caregivers spend on tasks related to providing care to their child with intestinal failure receiving home parenteral nutrition (PN). STUDY DESIGN: We conducted an exploratory cross-sectional study of caregivers of children with intestinal failure receiving long-term PN followed by our intestinal rehabilitation program. Caregivers completed a daily diary of care-related tasks. Data were analyzed using descriptive statistics. Exploratory models were completed to evaluate factors that influenced the amount of time that caregivers spent providing care. SAS University Edition 2018 (SAS Institute, Cary, NC) was used for data analysis with a P value of less than .05 considered significant. RESULTS: Thirty-four caregivers of children with intestinal failure consented with response rates of 85%. The mean age of the primary caregiver was 37 ± 7.9 years of age with 97% being the child's mother. The median PN exposure was 1239 days (IQR, 432-3012). Caregivers reported a median of 29.2 hours per week (IQR, 20.8-45.7 hours per week) of direct medical care. The majority of time was spent on providing PN and care of the central venous catheter (6.1 hours; IQR, 5.2-8.8). CONCLUSIONS: Caregivers of children with intestinal failure receiving long-term PN provide a significant amount of care to ensure their child remains healthy at home. The most significant amounts of time were spent on the administration of the PN and care of the central venous catheter.

Pain trajectories and well-being in children and young people with cerebral palsy: A cohort study.

AIM: To identify 5-week pain intensity trajectories and their association with physical and psychological well-being in children/young people with cerebral palsy (CP). METHOD: A cohort study was conducted with 101 Canadian children/young people with CP, of whom 49 were female, with an overall mean age of 12 years 11 months (SD 3 years 1 month), range of 8 to 18 years, and classified in any Gross Motor Function Classification System level. Self-reported pain intensity (Faces Pain Scale - Revised) was collected weekly for 5 weeks and physical and psychological well-being (KIDSCREEN-27) at baseline and 5 weeks. Statistical analyses included latent class growth and general linear models. RESULTS: All Gross Motor Function Classification System levels were represented (I = 40.6%; II = 15.8%; III = 20.8%; IV = 13.9%; V = 8.9%). Five pain intensity trajectories were identified. Three trajectories had very low (35.4%), low (32.4%), or high (4.9%) mean stable pain. Two trajectories had moderate changing pain (16.8%) and high pain decreasing to moderate levels (10.5%) respectively. Trajectory participants with stable high pain had the lowest physical well-being (adjusted ß = -10.01; 95% confidence interval [CI] = -19.37 to -0.66). Those in the three trajectories with the highest mean baseline pain intensity (>3 out of 10) had the lowest psychological well-being (adjusted ß = -8.27, 95% CI = -14.84 to -1.70; ß = -6.74, 95% CI = -12.43 to -1.05; ß = -5.82, 95% CI = -15.34 to 3.71). INTERPRETATION: Almost one-third of participants had moderate-to-high pain intensity trajectories. Membership in the higher pain intensity trajectories was associated with lower physical and psychological well-being. WHAT THIS PAPER ADDS: Five distinct 5-week pain intensity trajectories were identified in children/young people with cerebral palsy. Thirty-two per cent of participants had moderate-to-high pain intensity trajectories. Participants in the trajectories with higher pain intensity reported lower physical and psychological well-being.

OBJETIVO: Identificar trajetórias de intensidade de dor de 5 semanas e sua associação com o bem-estar físico e psicológico em crianças/jovens com paralisia cerebral (PC). MÉTODO: Foi realizado um estudo de coorte com 101 crianças/jovens canadenses com PC, sendo 49 do sexo feminino, com média de idade geral de 12 anos e 11 meses (DP 3 anos 1 mês), faixa de 8 a 18 anos, e classificados em qualquer nível do Sistema de Classificação da Função Motora Grossa. A intensidade da dor autorreferida (Faces Pain Scale - Revised) foi coletada semanalmente por 5 semanas e o bem-estar físico e psicológico (KIDSCREEN-27) no início e 5 semanas. As análises estatísticas incluíram crescimento de classe latente e modelos lineares gerais. RESULTADOS: Todos os níveis do Sistema de Classificação da Função Motora Grossa (GMFCS) foram representados (I = 40,6%; II = 15,8%; III = 20,8%; IV = 13,9%; V = 8,9%). Cinco trajetórias de intensidade de dor foram identificadas. Três trajetórias tiveram muito baixa (35,4%), baixa (32,4%) ou alta (4,9%) média de dor estável. Duas trajetórias apresentaram dor moderada em mudança (16,8%) e dor alta diminuindo para níveis moderados (10,5%), respectivamente. Os participantes com trajetória com dor alta estável tiveram o menor bem-estar físico (ß ajustado = -10,01; intervalo de confiança de 95% [IC] = -19,37 a -0,66). Aqueles nas três trajetórias com a maior intensidade média de dor na linha de base (> 3 em 10) tiveram o menor bem-estar psicológico (ß ajustado = -8,27, IC 95% = -14,84 a -1,70; ß = -6,74, 95% IC = -12,43 a -1,05; ß = -5,82, IC 95% = -15,34 a 3,71). INTERPRETAÇÃO: Quase um terço dos participantes tiveram trajetórias de intensidade de dor moderada a alta. A participação nas trajetórias de maior intensidade de dor foi associada a menor bem-estar físico e psicológico.

Stability of the Gross Motor Function Classification System over time in children with cerebral palsy.

AIM: To assess the stability of the Gross Motor Functional Classification System (GMFCS) in children with cerebral palsy (CP) from time of preliminary diagnosis (~2 years of age) to time of diagnosis (~5 years of age), and to examine factors associated with reclassification. METHOD: We conducted a longitudinal study using a sample from the Canadian CP Registry. Stability was analysed by using the percentage of agreement between timepoints and a weighted prevalence and bias adjusted kappa statistic. Univariate and multivariate logistic regressions were performed to identify variables associated with reclassification. RESULTS: The study included 1670 children (857 males, 713 females) with a mean age of 11 years 4 months (SD 4 years, range 3 years 5 months-20 years 1 month) at time of data extraction (3rd September 2019), of which 1435 (85.9%) maintained a stable GMFCS, with a weighted kappa of 0.91 (95% confidence interval 0.89-0.92). Univariate logistic regression showed that initial GMFCS level, CP subtype, and the presence of cognitive impairment were associated with the likelihood of change in the GMFCS level (p < 0.1). In the multivariate analysis, however, the likelihood was associated with initial GMFCS level only (odds ratio 7.10-8.88, p < 0.00). INTERPRETATION: The GMFCS has good stability in early childhood. For the majority of children, it is predictive of their long-term motor function. WHAT THIS PAPER ADDS: The Gross Motor Function Classification System (GMFCS) rating in early childhood is stable over time. There is no directionality in the reclassification of the GMFCS. The initial GMFCS level was related to the likelihood of change in follow-up GMFCS level.

Feasibility of High Repetition Upper Extremity Rehabilitation for Children with Unilateral Cerebral Palsy.

AIMS: In pediatric upper extremity rehabilitation, feasible repetition rates are unknown. Our objectives were to examine repetition rates during rehabilitation and their impact on outcomes. METHODS: Children with unilateral cerebral palsy due to perinatal stroke (n = 55, median 10 y 7 mo, 30 males) received Constraint-Induced Movement Therapy (CIMT) followed by Bimanual Therapy, each for 5 days. Repetitions were documented during one-on-one therapy (1.5 h/day). Outcomes included the Assisting Hand Assessment (AHA), Jebsen Taylor Test of Hand Function (JTTHF), and Box and Block Test (BBT). Means and standard deviations for motor outcomes and frequencies for repetition rates were calculated. Factors associated with repetition rates and outcome change were explored using standard linear regression. RESULTS: Repetitions/hour averaged 365 ± 165 during CIMT and 285 ± 103 during Bimanual Therapy. Higher repetition rates were associated with higher baseline function by older age, a main effect of younger age, and improving motor skill (p < .05). Higher repetition rates corresponded with improvement of the AHA and BBT (p < .05, standardized ß = 0.392, 0.358). CONCLUSIONS: Results suggest high repetition therapy is feasible in school-aged children with perinatal stroke, albeit with high individual variability. Multiple associations between repetition rates and baseline function and change point to the clinical importance of this measurable and potentially modifiable factor.

Randomized Trial of Oral Iron and Diet Advice versus Diet Advice Alone in Young Children with Nonanemic Iron Deficiency.

OBJECTIVE: To compare the effects of 2 treatment options on neurodevelopmental and laboratory outcomes in young children with nonanemic iron deficiency. STUDY DESIGN: A blinded, placebo-controlled, randomized trial of children 1-3 years with nonanemic iron deficiency (hemoglobin ≥110 g/L, serum ferritin <14 µg/L) was conducted in 8 primary care practices in Toronto, Canada. Interventions included ferrous sulfate or placebo for 4 months; all parents received diet advice. The primary outcome was the Early Learning Composite (ELC) using the Mullen Scales of Early Learning (mean 100, SD 15). Secondary outcomes included serum ferritin. Measurements were obtained at baseline and 4 and 12 months. Sample size was calculated to detect a between-group difference of 6-7 points in ELC. RESULTS: At enrollment (n = 60), mean age was 24.2 (SD 7.4) months and mean serum ferritin was 10.0 (SD 2.4) µg/L. For ELC, the mean between-group difference at 4 months was 1.1 (95% CI -4.2 to 6.5) and at 12 months was 4.1 (95% CI -1.9 to 10.1). For serum ferritin, at 4 months, the mean between-group difference was 16.9 µg/L (95% CI 6.5 to 27.2), and no child randomized to ferrous sulfate had a serum ferritin <14 µg/L (0% vs 31%, P = .003). CONCLUSIONS: For young children with nonanemic iron deficiency, treatment options include oral iron and/or diet advice. We remain uncertain about which option is superior with respect to cognitive outcomes; however, adding ferrous sulfate to diet advice resulted in superior serum ferritin outcomes after 4 months. Shared decision-making between practitioners and parents may be considered when selecting either option. TRIAL REGISTRATION: Clinicaltrials.gov: NCT01481766.

Pharmacological and neurosurgical interventions for individuals with cerebral palsy and dystonia: a systematic review update and meta-analysis.

AIM: To update a systematic review of evidence published up to December 2015 for pharmacological/neurosurgical interventions among individuals with cerebral palsy (CP) and dystonia. METHOD: Searches were updated (January 2016 to May 2020) for oral baclofen, trihexyphenidyl, benzodiazepines, clonidine, gabapentin, levodopa, botulinum neurotoxin (BoNT), intrathecal baclofen (ITB), and deep brain stimulation (DBS), and from database inception for medical cannabis. Eligible studies included at least five individuals with CP and dystonia and reported on dystonia, goal achievement, motor function, pain/comfort, ease of caregiving, quality of life (QoL), or adverse events. Evidence certainty was evaluated using GRADE. RESULTS: Nineteen new studies met inclusion criteria (two trihexyphenidyl, one clonidine, two BoNT, nine ITB, six DBS), giving a total of 46 studies (four randomized, 42 non-randomized) comprising 915 participants when combined with those from the original systematic review. Very low certainty evidence supported improved dystonia (clonidine, ITB, DBS) and goal achievement (clonidine, BoNT, ITB, DBS). Low to very low certainty evidence supported improved motor function (DBS), pain/comfort (clonidine, BoNT, ITB, DBS), ease of caregiving (clonidine, BoNT, ITB), and QoL (ITB, DBS). Trihexyphenidyl, clonidine, BoNT, ITB, and DBS may increase adverse events. No studies were identified for benzodiazepines, gabapentin, oral baclofen, and medical cannabis. INTERPRETATION: Evidence evaluating the use of pharmacological and neurosurgical management options for individuals with CP and dystonia is limited to between low and very low certainty. What this paper adds Meta-analysis suggests that intrathecal baclofen (ITB) and deep brain stimulation (DBS) may improve dystonia and pain. Meta-analysis suggests that DBS may improve motor function. Clonidine, botulinum neurotoxin, ITB, and DBS may improve achievement of individualized goals. ITB and DBS may improve quality of life. No direct evidence is available for oral baclofen, benzodiazepines, gabapentin, or medical cannabis.

Neurodevelopmental profiles of children with unilateral cerebral palsy associated with middle cerebral artery and periventricular venous infarctions.

AIM: To compare the neurodevelopment of children with unilateral cerebral palsy (CP) with middle cerebral artery (MCA) and periventricular venous infarctions (PVIs). METHOD: In this cross-sectional study, children with unilateral CP completed a neurological exam, unimanual Quality of Upper Extremity Skills Test, hand usage questionnaires, and IQ test. Neuroimaging was obtained from health records. RESULTS: Two hundred and forty-five participants with unilateral CP had neuroimaging (151 [61.9%] male, ages 2-18y, median=7y 6mo, interquartile range [IQR]=6y 7mo, with 93.6% in Gross Motor Function Classification System level I/II and 78.8% in Manual Ability Classification System level I/II). Ninety-seven (39.6%) had MCA injuries and 106 (43.3%) had periventricular white matter injuries, of which 48 (45.3%) were PVIs. Median Quality of Upper Extremity Skills Test for the MCA group was 49.2 (IQR=55.8), PVI 79.9 (IQR=23.6) (Mann-Whitney U=988.50, p<0.001). Bimanual hand usage (Children's Hand-use Experience Questionnaire) (Mann-Whitney U=425, p<0.001) and light touch (odds ratio=9.12, 95% confidence interval 1.28-400.76, Fisher's exact test p=0.017) were lower in the MCA compared to the PVI group. Full-scale IQ median centile score for the MCA group was 18.0 (IQR=35.5) and 50.0 (IQR=30.0) for the PVI group (Mann-Whitney U=382, p<0.001). INTERPRETATION: Children with unilateral CP and MCA injuries demonstrated lower hand function and usage, decreased light touch, and lower IQs compared to the PVI group. This study aids in defining rehabilitation needs informed by brain injury patterns.

Identifying pain trajectories in children and youth with cerebral palsy: a pilot study.

BACKGROUND: Although chronic pain is common in children with cerebral palsy (CP), little is known about short-term pain fluctuations and their impact on children's well-being. High-quality cohort studies are needed to understand the clinical course of pain in this population. We aimed to determine the feasibility of conducting a multicentre cohort study. In this pilot study we assessed: 1) study processes, 2) resource and 3) management indicators including recruitment and follow-up rates, data completeness, participant characteristics, and successes and barriers in the study conduct. METHODS: A multi-centre pilot cohort study was conducted with 10 Canadian children/youth with CP attending one of two children's rehabilitation centers. We collected self-reported pain intensity (Faces Pain Scale-Revised [FPS-R], Numeric Rating Scale [NRS]); pain interference (PROMIS PI); pain location (pain diagram); physical and psychological well-being (KIDSCREEN-27), sleep characteristics, preceding months' interventions, and some clinical characteristics at baseline. Average pain intensity was reported weekly for five weeks. Well-being, sleep and interventions were measured at baseline and again at five weeks. We used feasibility indicators to evaluate:1) study processes (e.g. recruitment, attrition rates); 2) resources (e.g. data completion, budgetary challenges); and 3) management (e.g. data optimization, variability of participants and pain scores). RESULTS: Between March and May 2019, 24 children and their parents/guardians were contacted and 20 met eligibility criteria. Of those, 10 agreed to in-person screening (50%) and were subsequently enrolled. The follow-up rate was 90% and self-reported missing data was minimal. Ninety percent of participants chose e-questionnaire follow-ups versus mailed paper questionnaires. Sixty percent required reminders to complete e-follow-ups. Participants were aged 8-17 years, five were female, GMFCS levels I-IV (none with level V), 90% had spastic CP and 80% reported having pain in the preceding week. Pain intensity (FPS-R) between participants ranged from 0-8/10 at baseline and 0-6/10 across all four weekly follow-ups. CONCLUSIONS: This pilot study demonstrates the feasibility of conducting a multicentre cohort study to identify short-term pain trajectories and measure their association with well-being in children and youth with CP. Additional strategies to improve recruitment and accessibility for those with GMFCS levels V should be implemented in future studies.

Current Referral Practices for Diagnosis and Intervention for Children with Cerebral Palsy: A National Environmental Scan.

OBJECTIVES: To describe current physician referral practices with respect to age at referral to medical specialists for initial diagnosis of cerebral palsy (CP) and rehabilitation specialists for intervention and to identify factors associated with delayed referral. STUDY DESIGN: National environmental scan of 455 children diagnosed with CP who were born in Canada between 2008 and 2011, selected from 4 sites within the Canadian CP Registry (Edmonton, Calgary, Toronto, and Montreal). Two sources of information were used-children's medical charts and the population-based registry, which provided corresponding data for each child. Primary outcomes extracted from the charts were age at referral for diagnostic assessment, age at diagnosis, age at referral for rehabilitation services, and age at initial rehabilitation intervention. Twelve variables were explored as potential predictors. Descriptive statistics, bivariate analyses, and multiple linear regressions were conducted. RESULTS: Median age (in months) at referral for diagnostic assessment was 8 (mean: 12.7 ± 14.3), diagnosis 16 (mean: 18.9 ± 12.8), referral for rehabilitation services 10 (mean: 13.4 ± 13.5), and rehabilitation initiation 12 (mean: 15.9 ± 12.9). Lower maternal education, mild severity of motor dysfunction, type of CP, early discharge after birth, and region of residence explained between 20% and 32% of the variance in age at referral for assessment, diagnosis, referral for rehabilitation, and rehabilitation initiation. CONCLUSIONS: Findings suggest wide variability exists in the age at which young children with CP are referred to specialists for diagnosis and intervention. User-friendly tools are therefore needed to enhance early detection and referral strategies by primary care practitioners, to ensure early interventions to optimize developmental outcomes and enhance opportunities for neural repair at a younger age.

Documenting change with the Canadian Occupational Performance Measure for children with cerebral palsy.

AIM: To assess the Canadian Occupational Performance Measure's (COPM) ability to document change over 3 years in children with cerebral palsy (CP). METHOD: This was a prospective study with ambulatory children with CP, aged 2 to 6 years. Caregivers set one to three COPM goals which were rescored annually over 3 years. A ceiling effect for performance goals was operationalized as a score of 8. A Wald χ2 generalized estimating equations model adjusted for age, sex, and Gross Motor Function Classification System (GMFCS) level, evaluated change over time. RESULTS: In total, 124 children (47 [37.9%] females, 77 [62.1%] males; mean age 3y 11mo [SD 1y 1mo]; GMFCS level I [n=78, 62.9%], II [n=21, 16.9%], and III [n=25, 20.2%]) were set 345 COPM goals at baseline. By Year 3, 106 participants (85.5%) rescored 287 of the goals (83.2%). Performance scores increased between baseline mean (SD) 2.93 (0.56), Year 1 5.98 (0.58) with 34.8% at ceiling; Year 2 6.74 (0.60) 48.3% at ceiling; and Year 3 7.37 (0.60) 59.6% at ceiling (Wald χ2 [3]=607.18, p<0.001). Satisfaction scores increased between baseline 4.42 (0.59), Year 1 6.82 (0.60) with 48% at ceiling; Year 2 7.53 (0.60) with 62.2% at ceiling (Wald χ2 [3]=208.48, p<0.001); with no significant increase by Year 3 7.82 (0.62) with 66.9% at ceiling. INTERPRETATION: COPM performance scores increased steadily over 3 years. By Year 2, a ceiling effect was seen in about half of the goals. The COPM may have utility to measure change over 3 years; periodic resetting of the descriptors of goal success are required to minimize ceiling.

International expert recommendations of clinical features to prompt referral for diagnostic assessment of cerebral palsy.

AIM: To establish international expert recommendations on clinical features to prompt referral for diagnostic assessment of cerebral palsy (CP). METHOD: An online Delphi survey was conducted with international experts in early identification and intervention for children with CP, to validate the results obtained in two previous consensus groups with Canadian content experts and knowledge users. We sent two rounds of questionnaires by e-mail. Participants rated their agreement using a 4-point Likert scale, along with optional open-ended questions for additional feedback. Additionally, a panel of experts and knowledge-users reviewed the results of each round and determined the content of subsequent surveys. RESULTS: Overall, there was high-level of agreement on: (1) six clinical features that should prompt referral for diagnosis; (2) two 'warning sign' features that warrant monitoring rather than immediate referral for diagnosis; and (3) five referral recommendations to other healthcare professionals to occur simultaneously with referral for diagnosis. INTERPRETATION: There was high agreement among international experts, suggesting that the features and referral recommendations proposed for primary care physicians for early detection of CP were broadly generalizable. These results will inform the content of educational tools to improve the early detection of CP in the primary care context. WHAT THIS PAPER ADDS: International experts provide strong agreement on clinical features to detect cerebral palsy. Consensus on clinical 'warning signs' to monitor over time. Referral recommendations from primary care to specialized health services are identified.

RECOMENDACIONES DE EXPERTOS INTERNACIONALES SOBRE CARACTERÍSTICAS CLÍNICAS PARA UNA DERIVACIÓN RÁPIDA PARA LA EVALUACIÓN DIAGNÓSTICA DE LA PARÁLISIS CEREBRAL: OBJETIVO: Establecer recomendaciones de expertos internacionales sobre características clínicas para iniciar derivación para la evaluación diagnóstica de la parálisis cerebral (PC). MÉTODO: Se realizó una encuesta online tipo Delphi con expertos en identificación e intervención temprana de niños con PC a fin de validar los resultados obtenidos en dos grupos de consenso realizados previamente en Canadá con expertos en contenidos y usuarios. Se enviaron dos rondas de cuestionarios por correo electrónico. Los participantes calificaron su acuerdo con un puntaje de 4 puntos en una escala Likert y con preguntas opcionales de respuesta abierta para comentarios adicionales. Además, un panel de expertos y usuarios revisaron los resultados de cada ronda y determinaron el contenido de las encuestas subsiguientes. RESULTADOS: En general, hubo un alto nivel de acuerdo sobre: ​​(1) seis características clínicas que requieren derivación rápida para el diagnóstico, (2) dos características de "señales de advertencia" que requieren monitoreo en lugar de referencia inmediata para el diagnóstico, y (3) cinco recomendaciones de referencia a otros profesionales de la salud que deben realizarse simultáneamente con la derivación para el diagnóstico. INTERPRETACIÓN: Hubo gran acuerdo entre los expertos internacionales, sugiriendo que las características y recomendaciones de referencia propuestas para los médicos de atención primaria para la detección de PC fue ampliamente generalizable. Estos resultados informarán el contenido de herramientas educativas para mejorar la detección precoz de PC en el contexto de atención primaria.

RECOMENDAÇÕES DE ESPECIALISTAS INTERNACIONAIS SOBRE ASPECTOS CLÍNICOS DISPARADORES DE ENCAMINHAMENTO PARA AVALIAÇÃO DIAGNÓSTICA EM PARALISIA CEREBRAL: OBJETIVO: Estabelecer recomendações de especialistas internacionais sobre os aspectos clínicos disparadores de encaminhamento para avaliação diagnóstica em paralisia cerebral (PC). MÉTODO: Um levantamento online internacional do tipo Delphi foi realizado com especialistas em identificação e intervenção precoce para crianças com PC, para validar os resultados obtidos em dois consensos prévios com especialistas no conteúdo e usuários canadenses. Enviamos duas rodadas de questionários por email. Os participantes pontuaram sua concordância usando uma escala Likert de 4 pontos, junto com questões abertas opcionais para informações adicionais. Além disso, um painel de especialistas e usuários revisaram os resultados de cada rodada, e determinaram o conteúdo das pesquisas subsequentes. RESULTADOS: Em geral, houve alto nível de concordância em: 1) seis aspectos clínicos que devem disparar encaminhamento para diagnóstico. 2) dois 'sinais de alerta' que merecem monitoramento mas não encaminhamento imediato para diagnóstico, e 3) cinco recomendações de encaminhamento para outros profissionais da saúde simultaneamente ao encaminhamento para diagnóstico. INTERPRETAÇÃO: Houve alta concordância entre especialistas internacionais, sugerindo que os aspectos e recomendações para encaminhamento propostos para médicos na atenção básica para a identificação precoce da PC foram amplamente generalizáveis. Estes resultados informarão o conteúdo de ferramentas educacionais para melhorar a detecção precoce de PC no contexto da atenção básica.

Use of consensus methods to determine the early clinical signs of cerebral palsy.

OBJECTIVES: To develop expert-informed content regarding the early motor attributes of cerebral palsy (CP) that should prompt physician referral for diagnostic assessment of CP, as well as concurrent referral recommendations. This content will be used in the creation of knowledge translation (KT) tools for primary care practitioners and parents. METHODS: Two nominal group processes were conducted with relevant stakeholders, representing Canadian 'content experts' and 'knowledge-users', using an integrated KT approach. RESULTS: Six attributes were identified that should prompt referral for diagnosis. If the child demonstrates: Early handedness <12 months; stiffness or tightness in the legs between 6 and 12 months; persistent fisting of the hands >4 months; persistent head-lag >4 months; inability to sit without support >9 months; any asymmetry in posture or movement. Five referral recommendations were agreed upon: Motor intervention specialist (physical therapy and/or occupational therapy) for ALL; speech-language pathology IF there is a communication delay; audiology IF there is parental or healthcare professional concern regarding a communication delay; functional vision specialist (e.g., optometrist or occupational therapist) IF there is a vision concern (e.g., not fixating, following, or tracking); feeding specialist (e.g., occupational therapist, speech-language pathologist) IF there are feeding difficulties (e.g., poor sucking, poor swallowing, choking, and/or not gaining weight). CONCLUSION: Rigorous consensus methods provided the initial evidence necessary to inform the content of tools to assist primary care providers in the early detection of CP. Results will be validated through a Delphi process with international experts, and user-friendly formats of this KT tool will be developed collaboratively with stakeholders.

Deep brain stimulation for pediatric dystonia: a meta-analysis with individual participant data.

AIM: We performed a meta-analysis with individual participant data of deep brain stimulation (DBS) for dystonia in children and young people. METHOD: Three databases (PubMed, Embase, and Web of Science) were queried from January 1999 to August 2017 with no language restrictions to identify case studies and cohort studies reporting on pediatric patients (age ≤21y) with dystonia. The primary outcomes were changes in Burke-Fahn-Marsden (BFM) or Barry-Albright Dystonia Scale scores. A mixed-effects regression was used to identify associations between clinical covariates and outcomes. RESULTS: Of 2509 citations reviewed, 72 articles (321 children) were eligible. At last follow-up (median 12mo, 25th centile=9.0; 75th centile=32.2), 277 (86.3%) patients showed improvement in dystonia, while 66.1 percent showed clinically significant (>20%) BFM Dystonia Rating Scale-motor improvement. On multivariable hierarchical regression, older age at dystonia onset, inherited dystonia without nervous system pathology and idiopathic dystonia (vs inherited with nervous system pathology or acquired dystonia), and truncal involvement indicated a better outcome (p<0.05). INTERPRETATION: The data suggest that DBS is effective and should be considered in selected children with inherited or idiopathic dystonia. WHAT THIS PAPER ADDS: Deep brain stimulation is effective in selected children with inherited or idiopathic dystonia.