How Well Does the EQ-5D-Y-5L Describe Children With Intellectual Disability?: "There's a Lot More to My Child Than That She Can't Wash or Dress Herself."

OBJECTIVES: The EQ-5D-5L is a generic health utility instrument for measuring health-related quality of life (HRQoL), with self-report and proxy report versions for children (EQ-5D-Y-5L). Children with intellectual disability (ID) are a heterogeneous population whose impairments and comorbidities place them at risk of poor HRQoL. This study aimed to describe the content validity and suitability for children with ID of a proxy report version of the EQ-5D-Y-5L as seen by their caregivers. METHODS: A proxy report EQ-5D-Y-5L was administered to caregivers of children with ID. Using cognitive think-aloud interviewing, participants were encouraged to provide the reasoning for their choices, assess the questions' relevance, comprehensibility, and comprehensiveness, and comment on the tool's strengths and weaknesses. Qualitative content analysis used both directed (deductive) and conventional (inductive) methods. RESULTS: There were 28 interviews with 30 caregivers of children with ID (aged 8-22 years, 17 boys, with autism spectrum disorder, cerebral palsy, Down syndrome, and rare genetic disorders). The EQ-5D-Y-5L was considered clear, concise, and largely relevant, but insufficiently comprehensive for this population. Interviewees sought clarification of the definition of HRQoL, whether it included unchanging impairments (vs fluctuating health states), and what basis of comparison to use (child or peer). Many interviewees suggested inclusion of questions for other domains, including communication and social engagement, equipment and human supports required, and a wider range of mental health questions. CONCLUSIONS: The study suggests that further work is required to ensure accurate responses to the EQ-5D-Y-5L from caregivers of children with ID and to describe these children adequately.

Psychometric Properties of the EQ-5D-Y-5L for Children With Intellectual Disability.

OBJECTIVES: The EQ-5D-Y-5L is a generic preference-based measure of health-related quality of life for children. This study aimed to describe the distributional properties, test-retest reliability, and convergent validity of the EQ-5D-Y-5L in children with intellectual disability (ID). METHODS: Caregivers of children with ID (aged 4 to 18 years) completed an online survey, including a proxy-report EQ-5D-Y-5L, the Quality-of-life Inventory-Disability, and disability-appropriate measures corresponding to the EQ-5D dimensions: mobility, self-care (SC), usual activities (UA), pain/discomfort (PD), and worry/sadness/unhappiness. Twenty-one participants repeated the EQ-5D-Y-5L a few weeks later. Test-retest reliability was computed using weighted kappa and intraclass correlation coefficients, and convergent validity using Spearman's and Pearson's correlation coefficients. RESULTS: Caregivers of 234 children completed the survey, with <1% missing values. Only 1.7% reported "no problems" on all dimensions (11111). The dimensions with the lowest percentage of "no problems" were SC and UA (both 8%). Test-retest reliability coefficients were fair to substantial for 4 dimensions (weighted kappa .30 to .79) but low for PD and overall health, as measured by the visual analog scale (EQ-VAS). Convergent validity was strong (Spearman's correlation .65 to .87) for mobility, SC, and PD; moderate to strong for worry/sadness/unhappiness (.47 to .60) and the EQ-VAS (Pearson's correlation .49); and weak to moderate for UA (.21 to .52). CONCLUSIONS: Convergent validity was generally good; test-retest reliability varied. Children with ID had lower scores on SC and UA than other populations, and their EQ-VAS could fluctuate greatly, indicating poorer and less stable health-related quality of life.

Epilepsy syndromes in cerebral palsy: varied, evolving and mostly self-limited.

Seizures occur in approximately one-third of children with cerebral palsy. This study aimed to determine epilepsy syndromes in children with seizures and cerebral palsy due to vascular injury, anticipating that this would inform treatment and prognosis. We studied a population-based cohort of children with cerebral palsy due to prenatal or perinatal vascular injuries, born 1999-2006. Each child's MRI was reviewed to characterize patterns of grey and white matter injury. Children with syndromic or likely genetic causes of cerebral palsy were excluded, given their inherent association with epilepsy and our aim to study a homogeneous cohort of classical cerebral palsy. Chart review, parent interview and EEGs were used to determine epilepsy syndromes and seizure outcomes. Of 256 children, 93 (36%) had one or more febrile or afebrile seizures beyond the neonatal period and 87 (34%) had epilepsy. Children with seizures were more likely to have had neonatal seizures, have spastic quadriplegic cerebral palsy and function within Gross Motor Function Classification System level IV or V. Fifty-six (60%) children with seizures had electroclinical features of a self-limited focal epilepsy of childhood; we diagnosed these children with a self-limited focal epilepsy-variant given the current International League Against Epilepsy classification precludes a diagnosis of self-limited focal epilepsy in children with a brain lesion. Other epilepsy syndromes were focal epilepsy-not otherwise specified in 28, infantile spasms syndrome in 11, Lennox-Gastaut syndrome in three, genetic generalized epilepsies in two and febrile seizures in nine. No epilepsy syndrome could be assigned in seven children with no EEG. Twenty-one changed syndrome classification during childhood. Self-limited focal epilepsy-variant usually manifested with a mix of autonomic and brachio-facial motor features, and occipital and/or centro-temporal spikes on EEG. Of those with self-limited focal epilepsy-variant, 42/56 (75%) had not had a seizure for >2 years. Favourable seizure outcomes were also seen in some children with infantile spasms syndrome and focal epilepsy-not otherwise specified. Of the 93 children with seizures, at last follow-up (mean age 15 years), 61/91 (67%) had not had a seizure in >2 years. Children with cerebral palsy and seizures can be assigned specific epilepsy syndrome diagnoses typically reserved for normally developing children, those syndromes commonly being age-dependent and self-limited. Compared to typically developing children with epilepsy, self-limited focal epilepsy-variant occurs much more commonly in children with cerebral palsy and epilepsy. These findings have important implications for treatment and prognosis of epilepsy in cerebral palsy, and research into pathogenesis of self-limited focal epilepsy.

Neonatal well-being and timing of brain injury in persons with cerebral palsy born at term or late preterm.

AIM: To describe the distribution of neuroimaging patterns in a term/late preterm population-based cohort with cerebral palsy (CP), ascertain associations between neuroimaging patterns and neonatal well-being, estimate the proportion with antenatal or perinatal timing of neuropathology, and apply this information to the understanding of common mechanisms of brain injury and causal pathways. METHOD: The cohort for this observational study comprised 1348 persons born between 1999 and 2017 in Victoria, Australia. Using algorithms designed for the study, neonatal well-being and timing of brain injury were tabulated for the whole cohort and across neuroimaging patterns and birth epochs. RESULTS: Clinical and demographic profiles, neonatal well-being, and timing of brain injury differed across neuroimaging patterns. An estimated 57% of the cohort had a complicated neonatal period. Timing of brain injury was antenatal in 57% and perinatal in 41%. A decrease in the relative proportions of perinatal timing of brain injury was observed over a period when the rates of CP in live births at term decreased. INTERPRETATION: This study begins to bridge the knowledge gap about causation in CP, moving towards better description of the main mechanisms of brain injury and their contribution within CP cohorts, and facilitating the ability to monitor changes over time and the success of preventive measures. WHAT THIS PAPER ADDS: In a population-based, term/late preterm cohort with cerebral palsy, 57% had a complicated neonatal period. In the same cohort, 57% had presumed antenatal timing of brain injury. The relative proportion with perinatal injury decreased over time.

Development of algorithms for estimating the Child Health Utility 9D from Caregiver Priorities and Child Health Index of Life with Disability.

PURPOSE: The primary aim was to determine Child Health Utility 9D (CHU9D) utilities from the Caregiver Priorities and Child Health Index of Life with Disabilities (CPCHILD) for non-ambulatory children with cerebral palsy (CP). METHODS: One hundred and eight surveys completed by Australian parents/caregivers of children with CP were analysed. Spearman's coefficients were used to investigate the correlations between the two instruments. Ordinary least square, robust MM-estimator, and generalised linear models (GLM) with four combinations of families and links were developed to estimate CHU9D utilities from either the CPCHILD total score or CPCHILD domains scores. Internal validation was performed using 5-fold cross-validation and random sampling validation. The best performing algorithms were identified based on mean absolute error (MAE), concordance correlation coefficient (CCC), and the difference between predicted and observed means of CHU9D. RESULTS: Moderate correlations (ρ 0.4-0.6) were observed between domains of the CHU9D and CPCHILD instruments. The best performing algorithm when considering the CPCHILD total score was a generalised linear regression (GLM) Gamma family and logit link (MAE = 0.156, CCC = 0.508). Additionally, the GLM Gamma family logit link using CPCHILD comfort and emotion, quality of life, and health domain scores also performed well (MAE = 0.152, CCC = 0.552). CONCLUSION: This study established algorithms for estimating CHU9D utilities from CPCHILD scores for non-ambulatory children with CP. The determined algorithms can be valuable for estimating quality-adjusted life years for cost-utility analysis when only the CPCHILD instrument is available. However, further studies with larger sample sizes and external validation are recommended to validate these findings.

Umbilical Cord Blood Cell Clearance Post-Infusion in Immune-Competent Children with Cerebral Palsy.

Umbilical cord blood cells have therapeutic potential for neurological disorders, through a paracrine mechanism of action. A greater understanding of the safety and immunological effects of allogeneic donor cord blood cells in the context of a healthy recipient immune system, such as in cerebral palsy, is needed. This study aimed to determine how quickly donor cord blood cells were cleared from the circulation in children with cerebral palsy who received a single intravenous infusion of 12/12 human leucocyte antigen (HLA)-matched sibling cord blood cells. Twelve participants with cerebral palsy aged 2-12 years received cord blood cell infusions as part of a phase I trial of umbilical blood infusion for cerebral palsy. Digital droplet PCR analysis of DNA copy number variants specific to donor and recipient was used to assess donor DNA clearance at five timepoints post-infusion, a surrogate measure of cell clearance. Donor cells were cleared by 3 months post-infusion in 11/12 participants. When detected, donor DNA was at a fraction of 0.01-0.31% of total DNA with no signs of graft-versus-host disease in any participant. The donor DNA clearance times provided by this study have important implications for understanding the safety of allogeneic cord blood cell infusion for cerebral palsy and translational tissue engineering or regenerative medicine research in other disorders.

Survival of individuals with cerebral palsy in Victoria, Australia: A longitudinal cohort study spanning four decades.

AIM: To provide an updated description of the rates, trends, and predictors of mortality of individuals with cerebral palsy (CP), born in the Australian state of Victoria between 1970 and 2012. METHOD: Data were extracted for 4807 individuals (2091 females; 2716 males). The probability of survival to 30th June 2017 was calculated using the Kaplan-Meier method. Mortality rates were calculated per 1000 person-years using age strata and compared with population mortality rates to produce mortality ratios. Cox proportional hazards regression was used to calculate hazard ratios for selected demographic and clinical characteristics and to estimate the effect of birth epoch on 15-year survival. RESULTS: There were 666 recorded deaths. Compared to the general population, mortality was higher for all persons with CP and highest for children aged 1 to 15 years (45-62 times). We observed 35% improvement in the probability of survival to 15 years for births in the 2000s relative to the 1970s (hazard ratio 0.65, 95% confidence interval [CI] 0.49, 0.86), but only 4% improvement for the subgroup with complex CP (hazard ratio 0.96, 95% CI 0.69, 1.33). INTERPRETATION: The observed improvements in survival for those born in the 2000s is likely related predominantly to a proportional reduction in complex CP within the cohort. WHAT THIS PAPER ADDS: Length of survival improved for Australians with cerebral palsy (CP) born this millennium. Improved survival was mainly because of a proportional reduction in complex CP. A small improvement in length of survival was seen for children with complex CP.

Developmental outcomes for survivors of placental laser photocoagulation for the management of twin-to-twin transfusion syndrome.

BACKGROUND: Fetoscopic laser coagulation of placental anastomoses reverses the pathological process in twin-to-twin transfusion syndrome, thereby increasing survival, but there are a paucity of studies addressing long-term neurodevelopmental outcome of survivors. This study aimed to ascertain the presence of neurodevelopmental disabilities in child survivors of monochorionic pregnancies managed by placental laser photocoagulation in the Australian state of Victoria. METHODS: All pregnancies undergoing placental laser photocoagulation with the Victorian Fetal Therapy Service between 2006-2017 were included. Information on each surviving child, including demographics, perinatal course, and developmental progress was collected from parents, and consent was sought to complete the Child Behaviour Checklist. Interviewers evaluated whether this information was consistent with a diagnosis of any of 14 neurodevelopmental conditions. A three-tiered outcome measure was allocated for each child: (1) unimpaired or developmentally normal, (2) mild or moderate neurological impairment, or (3) severe neurological impairment. Clinical predictors for adverse outcome were identified. RESULTS: Of 116 pregnancies (113 twin, 3 triplet), 96 (83%) resulted in 1 + surviving fetuses. 57/113 (50%) twin pregnancies resulted in 2 survivors, 36 (32%) in 1 survivor, and 20 (18%) in no survivors. Of the 235 fetuses, 154 (65.5%) survived to follow-up. Survival increased from 59% in 2006-2008 to 73% in 2015-2017. 90/154 (58%) survivors were followed up at a mean age of 7.5 [SD 3.0] years. Based on parental interview and Child Behaviour Checklist data, 28/90 (31%) participants were assessed as having neurodevelopmental impairment, 27 of mild-moderate severity and 1 severe. Speech/language disorders, attention deficit (hyperactivity) disorders, and fine motor impairment were most common. Neonatal length of stay conferred the highest risk of impairment. CONCLUSION: Substantial variation exists between fetal therapy services in the type and length of neonatal follow-up following fetoscopic laser coagulation, contributing to a lack of data on long-term outcomes. The findings from this study support increasingly urgent calls to undertake systematic and sustained follow-up of fetoscopic laser coagulation survivors until school age. Information from this study may assist parents in their decision-making when offered fetal surgery. Importantly, it highlights a group for targeted surveillance and early intervention.

Safety of sibling cord blood cell infusion for children with cerebral palsy.

Cerebral palsy (CP) is a nonprogressive neurological disorder and the most common physical disability of childhood. There is no cure for CP, but stem cells have the potential to improve brain injury and hence function. This phase 1 clinical trial investigated the safety of the intravenous infusion of full-matched sibling cord blood cells for children with CP aged 1 to 16 years. Preliminary efficacy outcomes were also investigated. Twelve participants received 12/12 HLA-matched sibling cord blood cell infusions. One treatable serious adverse reaction to cryoprotectant was observed, and no adverse reactions occurred beyond 24 h after infusion. Gross motor function measure (GMFM-66) scores did not improve compared with baseline beyond what could be expected from developmental levels, and participants had varied changes in the Quality of Upper Extremity Skills Test (QUEST) and Vineland Adaptive Behavior Scales (VABS-II) scores. In conclusion, matched sibling cord blood cell infusion for children with CP is relatively safe when conducted in an appropriate facility. Australian and New Zealand Clinical Trials Registry (ACTRN12616000403437) and Clinicaltrials.gov (NCT03087110).

Modelling quality of life in children with intellectual disability using regression trees.

AIM: To identify factors associated with quality of life (QoL) in children with intellectual disability. We aimed to identify patterns of association not observable in previous hypothesis-driven regression modelling using the same data set from a cross-sectional observational study. METHOD: A questionnaire was completed by 442 caregivers of children with confirmed intellectual disability and a diagnosis of autism spectrum disorder, cerebral palsy, Down syndrome, or Rett syndrome. The Quality of Life Inventory-Disability (QI-Disability) questionnaire was used to assess child QoL. Independent variables described the child's health, functional abilities, community participation, and sociodemographics. The R package rpart was used to build the regression trees. RESULTS: The mean total QI-Disability score was 69.2 out of a maximum 100. The subgroup with the lowest QoL scores comprised children with a high degree of daytime sleepiness (n=74, mean 57.5) while the subgroup with the highest QoL scores (n=91, mean 80.3) comprised children with little daytime sleepiness who participated more frequently in community activities and displayed good eye contact while listening. INTERPRETATION: Regression tree analysis provides insights into the relative importance of associated factors. Sleep problems and community participation were more important than functional abilities in accounting for differences in QoL. WHAT THIS PAPER ADDS: A hypothesis-free regression tree analysis enables examination of multiple factors potentially influencing quality of life (QoL) in children with intellectual disability. Functional abilities were less strongly associated with QoL than sleep problems and community participation.

Ten-year follow-up study found that motor-free intelligence quotient declined in children with mild-to-moderate cerebral palsy.

AIM: This 10-year follow-up study examined cognitive change in a cohort of children with cerebral palsy from preschool to adolescence at the group and individual levels. METHODS: The Wechsler Preschool and Primary Scale of Intelligence was administered to 80 children with cerebral palsy (mean = 4 years 6 months, standard deviation = 7 months) at baseline (Time 1). At 10-year follow-up (Time 2), 28 adolescents (mean = 14 years 6 months, standard deviation = 9 months) returned for assessment with the Wechsler Intelligence Scale for Children. Motor-free intelligence quotient (IQ) scores were calculated and paired-samples t-tests and the Reliable Change Index (RCI) were used to investigate change in IQ over time. RESULTS: At the group level, nonverbal IQ scores declined significantly. At the individual level, RCI indicated nine and 11 children showed a clinically significant decline in Full Scale IQ (FSIQ) and nonverbal IQ scores, respectively. Decline in FSIQ was related to a history of seizures whereas decline in nonverbal IQ was associated with higher initial IQ. CONCLUSION: Cognitive abilities in children with cerebral palsy evolve over time and selective deficits may not be observable until a later age, highlighting the importance of repeated cognitive assessment throughout childhood and adolescence.

Functioning, participation, and quality of life in children with intellectual disability: an observational study.

AIMS: To investigate associations between functioning, community participation, and quality of life (QoL) and identify whether participation mediates the effects of functioning on QoL. METHOD: The caregivers of 435 children (211 females, 224 males; mean age 12y; SD 3y 11mo; age range 5-18y) with intellectual disability and autism spectrum disorder, cerebral palsy, Down syndrome, or Rett syndrome reported on their child's functioning (dependence for managing personal needs, mobility, communication, eye contact when speaking), frequency of participation, and QoL. Linear regression and mediation analyses were used to evaluate the relationships between child functioning, participation, and QoL. RESULTS: Children with greater dependency for managing personal needs and limited eye contact when speaking experienced poorer QoL. Less impaired functioning was associated with more frequent participation, which, in turn, was associated with a 3-point gain in QoL for each additional point in frequency of participation (coefficient=2.67, 95% confidence interval 1.56-3.78). The effect of impaired functioning on QoL was partially mediated by participation in children with greater dependency in managing personal needs and those with mildly impaired communication. INTERPRETATION: Greater levels of impairments with poorer functioning, notably a high level of dependence, were associated with poorer QoL. Poorer QoL can be partly explained by less frequent community participation.

Experiences and barriers to accessing mental health support in mothers of children with a disability.

BACKGROUND: Despite evidence for high levels of mental health problems in mothers of children with a disability and the potential impact on caring for their child, very little is known about mothers' experience in accessing professional mental health support. This study aimed to explore mothers' views and experience on seeking help for their mental health. METHODS: Semi-structured interviews were conducted with 25 mothers of children with a disability. Thematic analysis was completed. RESULTS: Mothers experienced significant barriers when accesing support at the personal, professional and system level. Personal barriers included the need for competency and stigma about mental illness: professional barriers included the lack of discussions about mental health and interpersonal factors that hindered disclosure. System barriers included feeling invisible to the health services, paediatric care focusing on the child rather than the family and limitations to the type of mental health support available. CONCLUSION: Mothers perceive substantial barriers in accessing support for their mental health. It is important that strategies are designed so the importance of mentally healthy mothers is understood and to normalize a need for assistance when you are the mother of a child with additional support needs. Strategies are also needed to encourage mothers to seek help and to assist professionals having discussions about maternal wellbeing. Improvements are also required in the accessibility of service supports.

Comorbidities and quality of life in children with intellectual disability.

BACKGROUND: Many children with intellectual disability live with medical comorbidities. This study examined the impacts of comorbidities on quality of life (QOL) of children with intellectual disabilities and whether impacts varied with caregiver perceptions that medical needs had been met. METHODS: Primary caregivers of 447 children (aged 5-19 years) with an intellectual disability reported on their child's medical comorbidities and the extent to which they perceived their child's medical needs had been met in a cross-sectional observational study. The Quality of Life Inventory-Disability was used to measure QOL on a 100-point scale. Linear regression models including interaction terms were used to evaluate their associations. RESULTS: Parent-reported recurrent child pain (-4.97, 95% CI -8.21, -1.72), night-time sleep disturbances (-4.98, 95% CI -7.23, -2.73), daytime somnolence (-8.71, 95% CI -11.30, -2.73), seizures that occurred at least weekly (-7.59, 95% CI -13.50, -1.68) and conservatively managed severe scoliosis (-7.39, 95% CI -12.97, -1.81) were negatively associated with child QOL. Despite the majority of parents (~70%) perceiving that their child's medical needs had been met to a great extent, this did not significantly moderate the association between any comorbidities and QOL. CONCLUSIONS: Comorbidities were common and had marked associations with QOL. Evaluation and management of pain and sleep disturbance continue to be high priorities in improving QOL of young people with intellectual disabilities. Further research on the optimal methods of managing these comorbidities is warranted.

Successfully Negotiating Life Challenges: Learnings From Adults With Cerebral Palsy.

Despite facing multidimensional inequalities, some adults with cerebral palsy achieve positive social outcomes (e.g., independent living, employment, and romantic relationships). We interviewed 23 adults (aged 23-47 years) about how they successfully negotiated the challenges of adulthood. Common to all life situations was doing what others (people without cerebral palsy) do. Origins of success lay in their formative experiences (e.g., typical parental expectations with commensurate support and acceptance and supported involvement in school life). Their present functioning (e.g., positive self-concept, well-honed social skills, and physical fitness) combined with present contexts (e.g., expectations of involvement in adult activities and accessible and accommodating environments) enabled them to find ways of negotiating challenges. Finding a way included drawing on knowledge and skills, gravitating toward those who are comfortable with difference, maintaining function, and using natural and paid supports. The findings suggest multiple avenues for supporting people with cerebral palsy to achieve positive social outcomes.

Anticholinergic medications for reducing drooling in children with developmental disability.

AIM: To determine: the effectiveness of three anticholinergic medications in reducing drooling in children with developmental disabilities (such as cerebral palsy, intellectual disability, and autism spectrum disorder), the frequency and nature of side effects, and their impact on treatment discontinuation. METHOD: After prescription of benzhexol hydrochloride, glycopyrrolate, or scopolamine patches at a tertiary saliva control clinic, all carers of 110 consecutive, eligible patients were recruited over a 5-year period. They provided data for 52 weeks, or until drug discontinuation, on compliance, drooling, adverse effects, and reasons for cessation. We evaluated and compared best drooling response, side effects, and drug cessation rates using survival analysis, and the effect of baseline variables on the discontinuation rate using proportional hazards regression. RESULTS: Among 110 participants (71 males, 39 females; mean age 8y 5mo [SD 4y 3mo], range 1y 11mo-18y 11mo), benzhexol, glycopyrrolate, and scopolamine were prescribed 81, 62, and 17 times respectively, with respective response rates of 85%, 75%, and 65%. Poor head control and poor oromotor function were predictive of poor response. Side effects frequently prompted drug cessation in males more than females (hazard ratio 1.8 [95% confidence interval 1.0-3.2], p=0.048). Glycopyrrolate had the fewest side effects. INTERPRETATION: Benzhexol, glycopyrrolate, and scopolamine reduce drooling, but improvement is offset by adverse side effects. Overall, glycopyrrolate performs best. WHAT THIS PAPER ADDS: In drooling, glycopyrrolate produced the greatest improvement with fewer side effects compared with benzhexol and scopolamine. Poor head control and poor oromotor function were associated with poor response. Medication side effects were common and often led to treatment discontinuation. Behavioural issues instigated cessation of benzhexol more often in males than females.

MEDICAMENTOS ANTICOLINÉRGICOS PARA REDUCIR EL BABEO EN NIÑOS CON TRASTORNOS DEL DESARROLLO: OBJETIVO: Determinar: la eficacia de tres medicamentos anticolinérgicos en la reducción del babeo en niños con trastornos del desarrollo (como parálisis cerebral, discapacidad intelectual y el trastorno del espectro autista), la frecuencia y la naturaleza de los efectos secundarios y su impacto en la interrupción del tratamiento. MÉTODO: Después de la prescripción de trihexifenidilo, glicopirrolato o parches de escopolamina en una clínica terciaria de control de saliva, fueron reclutados los cuidadores de 110 pacientes elegibles durante un período de 5 años. Estos proporcionaron datos sobre el cumplimiento, babeo, efectos adversos y razones para el cese, durante 52 semanas o hasta la interrupción del medicamento. Se evaluó y comparó la mejor respuesta al babeo, efectos secundarios y tasas de cese de drogas, utilizando el análisis de supervivencia y el efecto de las variables basales en la tasa de interrupción utilizando el modelo de riesgos proporcionales. RESULTADOS: Entre 110 participantes (71 varones y 39 mujeres; edad promedio de 8 años 5 meses [DE 4 años 3 meses], con rango de 1 año 11 meses - 18 años 11 meses), se prescribió trihexifenidilo, glicopirrolato y escopolamina, 81, 62, y 17 veces respectivamente, con tasas de respuesta respectivas de 85%, 75% y 65%. El pobre control cefálico y la mala función oromotora eran predictores de una respuesta deficiente. Los efectos secundarios provocaron el cese de las drogas con más frecuencia en los varones que en las mujeres (relación de riesgo 1,8 [intervalo de confianza del 95% 1,0-3,2], p 0,048). Glicopirrolato tuvo el menor número de efectos adversos INTERPRETACIÓN: El trihexifenidilo, el glicopirrolato y la escopolamina reducen el babeo, sin embargo, la mejora se contrarresta por los efectos secundarios. En general, el glicopirrolato tiene mejores resultados.

MEDICAÇÕES ANTICOLINÉRGICAS PARA REDUZIR SIALORRÉIA EM CRIANÇAS COM DEFICIÊNCIAS DESENVOLVIMENTAIS: OBJETIVO: Determinar: a efetividade de três medicações anticolinérgicas na redução da sialorréia em crianças com deficiências desenvolvimentais (como paralisia cerebral, deficiência intelectual, e transtorno do espectro autista), a frequência e natureza dos efeitos colaterais, e seu impacto na descontinuação do tratamento. MÉTODO: Após prescrição de hidroclorido benzexol, glicopirrolato, ou faixas de escopolamina em uma clínica terciária de controle da salivação, todos os cuidadores de 110 pacientes elegíveis consecutivos foram recrutados em um período de 5 anos. Eles forneceram dados de 52 semanas, ou até a descontinuação da medicação, sobre adesão, sialorréia, efeitos adversos, e razões para interrupção. Avaliamos e comparamos a melhor resposta para salivação, efeitos colaterais e interrupção da medicação usando análise de sobrevivência, e o efeito das variáveis na linha de base na taxa de descontinuação usando regressão de riscos proporcionais. RESULTADOS: Entre 110 participantes (71 do sexo masculino, 39 do sexo feminino; média de idade 8a 5m [DP 4a 3m], variação 1a 11m-18a 11m), benzexol, glicopirrolato, e escopolamina foram prescritos 81, 62, e 17 vezes respectivamente, com as respectivas taxas de resposta de 85%, 75%, and 65%. Pouco controle de cabeça e função oromotora foram preditivos de pior resposta. Efeitos colaterais mais frequentemente causaram interrupção da medicação em meninos do que em meninas (taxa de risco 1,8 [intervalo de confiança a 95% 1,0-3,2], p=0,048). Glicopirrolato teve menos efeitos colaterais. INTERPRETAÇÃO: Benzexol, glicopirrolato, ou escopolamina reduzem a sialorréia, mas as melhoras são atenuadas pelos efeitos colaterais. Em geral, glicopirrolato teve o melhor desempenho.

Health-related quality of life and upper-limb impairment in children with cerebral palsy: developing a mapping algorithm.

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

Speech in children with cerebral palsy.

AIM: To examine the frequency, characteristics, and factors associated with speech delay and disorder in a community sample of children with cerebral palsy (CP). METHOD: Participants were 84 children (37 females, 47 males; aged between 4y 11mo-6y 6mo) with CP identified through a population-based registry. Speech and oromotor function were systematically evaluated to provide a differential diagnosis of articulation, phonological, and motor speech disorders. RESULTS: In total, 82% (69/84) of participants had delayed or disordered speech production, including minimally verbal presentations (n=20). Verbal participants (n=64) presented with dysarthria (78%), articulation delay or disorder (54%), phonological delay or disorder (43%), features of childhood apraxia of speech (CAS) (17%), or mixed presentations across these conditions. Speech intelligibility was poorest in those with dysarthria and features of CAS. Speech delay or disorder in verbal participants was associated with language impairment (p=0.002) and reduced health-related quality of life (p=0.04) (Fisher's exact test). Poorer speech accuracy (i.e. lower percentage consonants correct) correlated with greater impairments in both language (p<0.001) and oromotor function (p<0.001) (Spearman's test). INTERPRETATION: The speech profile of children with CP is characterized by impairment at multiple levels of speech production (phonetic, cognitive-linguistic, neuromuscular execution, and high-level planning/programming), highlighting the importance of a personalized differential diagnosis informing targeted treatment.

More than an X-ray: Experiences and perspectives of parents of children with cerebral palsy when engaging in hip surveillance.

AIM: We explored the experiences of parents of children with cerebral palsy (CP) when engaging in hip surveillance for their child and aimed to identify the barriers and facilitators they encounter. METHODS: We conducted a pragmatic qualitative study through five focus groups conducted with 23 parents and primary care givers of young people with cerebral palsy. A semi-structured topic guide was used to facilitate discussion. Recordings were transcribed verbatim and transcripts analysed using content analysis. RESULTS: Six major categories emerged: (i) hip surveillance is a shared responsibility; (ii) knowledge is empowering; (iii) hip surveillance should be proactive rather than reactive; (iv) consistency and support from health professionals is valuable; (v) good communication is crucial; and (vi) challenges associated with having an X-ray may not be appreciated. Participants made recommendations related to: service model enhancements, information provision and improving both communication and the experience of having an X-ray. CONCLUSION: Despite having a good understanding of the need and importance of hip surveillance for their child, several barriers to parent engagement exist. Findings will inform the implementation of a family-centred model for hip surveillance and may be relevant to those undertaking or planning the implementation of hip surveillance in other areas.

Sleep problems and solution seeking for children with cerebral palsy and their parents.

AIM: Sleep problems are common in school-aged children with cerebral palsy (CP). Despite the significant impact of sleep disturbance and deprivation, there is a paucity of research in the area. The aims of this study were to (i) investigate the frequency of sleep problems in children with CP and their parents and (ii) understand what happens when parents ask for help with sleep problems from their health-care professionals. METHODS: This was a prospective cohort study using an online survey sent to parents/primary care givers of children with CP aged 6-12 years recruited through the Victorian Cerebral Palsy Register. The following sleep assessment tools: The Children's Sleep Habits Questionnaire, the Pittsburgh Sleep Quality Index and the Gross Motor Function Classification System Parent Rating Tool were administered along with custom-designed questions that were informed by a preceding qualitative scoping study. RESULTS: Complete data sets were received from 126 parents/care givers. Almost half (46%) of the parents reported their child had sleep problems. Of the 64 parents who reported seeking help for a child's sleep problem, only 21 indicated that their attempt was successful. If a child had poor sleep, the parent was more likely to have a sleep problem than parents who did not report poor child sleeping. CONCLUSION: Sleep problems are common in children with CP and their parents. Parents do not always seek help, and those who do may not find an effective solution. Future research should explore how sleep problems can be effectively prioritised for children with CP and their parents/care givers.