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Objective: To assess the performance of the SD Bioline Cholera Ag O1/O139 rapid diagnostic test (RDT) compared to a reference standard combining culture and PCR for the diagnosis of cholera cases during an outbreak.Methods: RDT and bacterial culture were performed on site using fresh stools collected from cholera suspected cases, and from stools enriched in alkaline peptone water. Dried stool samples on filter paper were tested for V. cholerae by PCR in Lusaka (as part of a laboratory technology transfer project) and at a reference laboratory in Paris, France. A sample was considered positive for cholera by the reference standard if any of the culture or PCR tests was positive for V. cholerae O1 or O139.Results: Among the 170 samples tested with SD Bioline and compared to the reference standard, the RDT showed a sensitivity of 90.9% (95% CI: 81.3-96.6) and specificity of 95.2% (95% CI: 89.1-98.4). After enrichment, the sensitivity was 95.5% (95% CI: 87.3-99.1) and specificity 100% (95% CI: 96.5-100).Conclusion: The observed sensitivity and specificity were within recommendations set by the Global Task Force for Cholera Control on the use of cholera RDT (sensitivity = 90%; specificity = 85%). Although the sample size was small, our findings suggest that the SD Bioline RDT could be used in the field to rapidly alert public health officials to the likely presence of cholera cases when an outbreak is suspected. [ABSTRACT FROM AUTHOR]

Aim: This cross-sectional study aimed to examine the preliteracy abilities of young children with neurofibromatosis type 1 (NF1) and to identify which of these abilities best predicted conventional literacy (spelling).Method: Forty-two children with NF1 (23 males, 19 females; mean age [SD] 5y 6mo [6mo]) were compared with 32 unaffected children (15 males, 17 females; mean age [SD] 5y 4mo [6mo]). All children completed a comprehensive cognitive assessment including measures of phonological processing (phonological awareness, phonological memory, rapid automatic naming) and letter-sound knowledge.Results: Children with NF1 performed significantly poorer than the comparison group across all cognitive and preliteracy domains, with specific weaknesses evident in phonological awareness (F1,68 =14.13, p<0.001, partial η2 =0.17), phonological memory (F1,68 =13.87, p<0.001, partial η2 =0.17), and letter-sound knowledge (F1,71 =5.65, p=0.020, partial η2 =0.07). Within the group with NF1 group, over a third of children demonstrated impairment in at least one phonological processing domain and the risk of phonological impairment was 5.60 times that of unaffected children. Children's letter-sound knowledge was the strongest predictor of conventional literacy (spelling).Interpretation: This study establishes that preliteracy deficits are present and detectable in young children with NF1. As a result of the high incidence of preliteracy impairment, we recommend screening phonological awareness and letter-sound knowledge to identify risk of future learning disorders.What This Paper Adds: Young children with neurofibromatosis type 1 are at elevated risk of preliteracy deficits. The most affected domains are phonological awareness and phonological memory. Letter-sound knowledge is the strongest predictor of conventional literacy (spelling). [ABSTRACT FROM AUTHOR]

Aim: Health-related quality of life (HRQoL) and peer relationships were investigated in adolescents with developmental coordination disorder (DCD) and attention-deficit-hyperactivity disorder (ADHD).Method: Adolescents with DCD (n=9), ADHD (n=9), DCD and ADHD (n=10), and typically developing adolescents (n=16) completed the following questionnaires: KIDSCREEN-52 Health-Related Quality of Life Questionnaire and Peer Relations Questionnaire for Children. Twenty-five participants took part in semi-structured interviews.Results: Adolescents with DCD and ADHD had lower HRQoL on the mood and emotions, school environment, and financial resources scales of the KIDSCREEN-52 than adolescents in the DCD and typically developing groups (all p<0.05). On the Peer Relations Questionnaire for Children, the DCD and ADHD group reported significantly higher victimization compared with those in the typically developing (p=0.030) and DCD (p=0.010) groups. Qualitative interviews among young people with DCD and ADHD revealed feelings of marginalization and victimization. Descriptors such as 'misfits', 'oddballs', 'weird', and 'the rejects' were used to describe themselves.Interpretation: HRQoL and peer relationships are negatively affected in adolescents with DCD and ADHD. WHAT THIS PAPER ADDS?: Children with developmental coordination disorder (DCD) do not display poorer overall health-related quality of life (HRQoL) versus typically developing controls. Having DCD and attention-deficit-hyperactivity disorder (ADHD) was associated with poorer HRQoL. Adolescents with DCD and ADHD experience significantly higher levels of peer victimization than typically developing adolescents. HRQoL and peer relationships are significantly associated in adolescent respondents. [ABSTRACT FROM AUTHOR]

Aim: If increased muscle stiffness and contractures in children with cerebral palsy (CP) are related to impaired muscle growth, reduced muscle growth should precede or coincide with increased muscle stiffness during development. Here, we compared the volume of the medial gastrocnemius muscle and the passive (non-neural) stiffness of the triceps surae musculotendinous unit in typically developing children and children with CP from birth until 4 years of age.Method: Forty-one children with CP and 45 typically developing children were included. Freehand three-dimensional ultrasound was used to evaluate the volume of the medial gastrocnemius muscle. Biomechanical and electrophysiological measures were used to determine passive and reflex mediated stiffness of the triceps surae musculotendinous unit.Results: Medial gastrocnemius muscle volume increased with the same rate in typically developing and children with CP until 12 months of age, when a significant smaller rate of growth was observed in children with CP. Passive stiffness of the triceps surae musculotendinous unit showed a linear increase with age in typically developing children. Children with CP older than 27 months showed a significant increase in passive stiffness. Reflex mediated stiffness was only pathologically increased in four children with CP.Interpretation: The deviation of medial gastrocnemius muscle volume, earlier than musculotendinous unit stiffness, is consistent with the hypothesis. The data also point out that muscle atrophy and muscle stiffness already develops within the first 1 to 2 years. This emphasizes the necessity of early interventions to promote lower limb muscle growth in this population.What This Paper Adds: Medial gastrocnemius muscle growth is reduced in children with cerebral palsy (CP) around 12 months after birth. Triceps surae musculotendinous unit stiffness is increased in children with CP around 27 months after birth. Reflex excitability is rarely increased in children with CP. Reduced muscle growth may be involved in the pathophysiology of contractures. [ABSTRACT FROM AUTHOR]

Objectives: MEETINGDEM investigated whether the Dutch Meeting Centres Support Programme (MCSP) could be implemented in Italy, Poland, and the UK with comparable benefits. This paper reports on the impact on people living with dementia attending pilot Meeting Centres in the 3 countries.Methods: Nine pilot Meeting Centres (MCs) participated (Italy-5, Poland-2, UK-2). Effectiveness of MCSP was compared with Usual Care (UC) on outcomes measuring behavioural and psychological symptoms (NPI), depression (CSDD), and quality of life (DQoL, QOL-AD), analysed by ANCOVAs in a 6-month pre-test/post-test controlled trial.Results: Pre/post data were collected for 85 people with dementia and 93 carers (MCSP) and 74 people with dementia /carer dyads' receiving UC. MCSP showed significant positive effects for DQoL [Self-esteem (F = 4.8, P = 0.03); Positive Affect (F = 14.93, P < 0.00); Feelings of Belonging (F = 7.77, P = 0.01)] with medium and large effect sizes. Higher attendance levels correlated with greater neuropsychiatric symptom reduction (rho = 0.24, P = 0.03) and a greater increase in feelings of support (rho = 0.36, P = 0.001).Conclusions: MCSPs showed significant wellbeing and health benefits compared with UC, building on the evidence of effectiveness from the Netherlands. In addition to the previously reported successful implementation of MCSP in Italy, Poland, and the UK, these findings suggest that further international dissemination of MCSP is recommended. [ABSTRACT FROM AUTHOR]

How health care providers are paid affects how medicine is practiced. It is thus important to assess provider payment models not only from the economic perspective but also from the ethical perspective. China recently started to reform the provider payment model in the health care system from fee-for-service to case-based payment. This paper aims to examine this transition from an ethical perspective. We collected empirical studies on the impact of case-based payment in the Chinese health care system and applied a systematic ethical matrix that integrates clinical ethics and public health ethics to analyze the empirical findings. We identified eleven prominent ethical issues related to case-based payment. Some ethical problems of case-based payment in China are comparable to ethical problems of managed care and diagnosis related groups in high-income countries. However, in this paper we discuss in greater detail four specific ethical issues in the Chinese context: professionalism, the patient-physician relationship, access to care and patient autonomy. Based on the analysis, we cautiously infer that case-based payment is currently more ethically acceptable than fee-for-service in the context of China, mainly because it seems to lower financial barriers to access care. Nonetheless, it will be difficult to justify the implementation of case-based payment if no additional measures are taken to monitor and minimize its existing negative ethical implications. [ABSTRACT FROM AUTHOR]

Recent research and theorizing has characterized contemporary mothering as medicalized, intensive and risky. The rate of diagnosis of children's mental health issues has grown rapidly, particularly since the 90s. This paper examines the construction of mothering in regard to children's behaviours and mental health through an exploratory and qualitative content analysis of the portrayal of advice to mothers in Chatelaine Magazine, the premier women's magazine published in Canada. The time periods chosen for comparison were 1945-1956 and 1990-2010. The first period was selected because it was a time of dramatic changes that occurred in family, occupational and domestic life for women immediately after World War II. The second represents the modern period. The findings of the paper suggest that mothering was intensive, medicalized and risky in both periods although the conceptualizations of problems differed. [ABSTRACT FROM AUTHOR]

Abstract: Objective: Non-invasive sampling of human genitals to identify high-risk individuals with subclinical oncogenic HPV infection remains a challenge. The study was designed to see if self-collected urine can be used as a simple, non-invasive sampling for screening HPV, particularly for screening/monitoring general population or young adolescents or infants, if they are to be immunized by HPV vaccines. Method: Self-collected urine samples from 100 sexually unexposed college going girls and cervical scrapes from 104 normal healthy sexually active married women were used in this study. Additionally, a group of 55 women were recruited for collecting first urine and later scraped cervical cells to validate urine sampling by directly comparing HPV positivity between the two types of biological specimens. A dry ‘paper smear’ method for specimen collection and a simple single tube protocol was employed for PCR detection of HPV infection. Results: Out of 100 sexually inexperienced college going girls, only 6 (6%) were positive for HPV infection as revealed by L1 consensus primer and 4 (4%) of them were positive for HPV 16 but none was found positive for HPV 18 DNA. Out of 104 sexually active married women who were cytologically reported as negative by Pap test, 11 (10.5%) were found HPV positive and 7 (6.7%) of them had infection of high-risk HPV type 16. Both urine and later cervical scrapes from a group of 55 women collected as dry ‘paper smear’ showed perfect matching positivity for HPV between urine and cervical scrape. Conclusions: The use of urine coupled with its dry collection as ‘paper smear’ facilitating their easy transport, storage and direct PCR detection of HPV DNA opens up an alternative non-invasive approach for population screening of HPV infection, at least in such cases as children and infants in whom invasive samples are difficult to obtain. [Copyright &y& Elsevier]

Neuronal ceroid lipofuscinoses (NCLs) are rare, progressive disorders. Through this series of 20 patients with NCL, we illustrate differences between subtypes in their presenting symptoms and clinical, imaging, and electrophysiological results to raise awareness of symptom diversity. Data were available on presenting symptoms, genetics, magnetic resonance imaging (MRI), electroencephalography (including with low-frequency intermittent photic stimulation), visual responses, and electron microscopy. Causal mutations were identified in 10 patients. Eleven patients had neuronal ceroid lipofuscinosis type 2 (CLN2) disease and their most common presenting symptom was seizures, although motor and language defects were also reported. Five patients with CLN2 disease showed abnormalities at initial MRI, but only three showed a photic response with low-frequency stimulation. Seizures were not as common a presenting symptom in other NCL subtypes. Patients with NCLs present with diverse symptoms, which may not be characteristic in early disease stages. These signs and symptoms should lead to rapid diagnostic confirmatory testing for NCLs. WHAT THIS PAPER ADDS: Disease presentation is not uniform for neuronal ceroid lipofuscinoses. Characteristic clinical test results may not be identified in early disease stages. [ABSTRACT FROM AUTHOR]

International research is an essential means of reducing health disparities between and within countries and should do so as a matter of global justice. Research funders from high-income countries have an obligation of justice to support health research in low and middle-income countries (LMICs) that furthers such objectives. This paper investigates how their current funding schemes are designed to incentivise health systems research in LMICs that promotes health equity. Semi-structured in-depth interviews were performed with 16 grants officers working for 11 funders and organisations that support health systems research: the Alliance for Health Policy and Systems Research, Comic Relief, Doris Duke Foundation, European Commission, International Development Research Centre, Norwegian Agency for Development Cooperation, Research Council of Norway, Rockefeller Foundation, UK Department of International Development, UK Medical Research Council, and Wellcome Trust. Thematic analysis of the data demonstrates their funding schemes promote health systems research with (up to) five key features that advance health equity: being conducted with worst-off populations, focusing on research topics that advance equitable health systems, having LMIC ownership of the research agenda, strengthening LMIC research capacity, and having an impact on health disparities. The different types of incentives that encouraged proposed projects to have these features are identified and classified by their strength (strong, moderate, weak). It is suggested that research funders ought to create and maintain funding schemes with strong incentives for the features identified above in order to more effectively help reduce global health disparities. [ABSTRACT FROM AUTHOR]

Introduction: Percutaneous transluminal angioplasty (PTA) and surgical bypass (BYP) are treatment options for symptomatic peripheral arterial disease (PAD). PTA and BYP have different clinical outcomes and cost implications. This paper aims to compare hospital-related costs of PTA and BYP for PAD of the lower limbs in an Australian health service.Methods: A retrospective cost analysis using clinical and financial data from an urban, tertiary hospital was performed. Patient cohorts were matched to existing published studies and 3-year findings were calculated. Outcomes measured were mean initial admission cost; mean bed stay; mean complication rate; mean cost of re-intervention at 12 months and extrapolated mean cost at 3 years.Results: The mean total admission costs for PTA compared to BYP were $8758 vs. $27,849 (P < 0.001). Patients undergoing BYP were admitted for 10.25 vs. 3.77 nights (P < 0.001). The complication rate was greater in the BYP group for infection only. Re-intervention was required by 13% of the PTA group and 16% of the BYP group, at a mean cost of $11,798 and $14,728, respectively (P = 0.453). The extrapolated total mean cost at 3 years was higher in the BYP group for patients with both intermittent claudication ($26,764 vs. $11,402) and critical limb ischaemia ($27,719 vs. $12,655).Conclusions: In this cohort, PTA is a favourable alternative to BYP for PAD of the lower limbs as it is less costly, does not result in a greater re-intervention rate at 1 year and has been previously demonstrated to have comparable clinical outcomes. Given the limitations of this retrospective analysis, a prospective cost-effectiveness analysis is recommended. [ABSTRACT FROM AUTHOR]

Aim: To examine the long-term consequences of glycogen storage in the central nervous system (CNS) for classic infantile Pompe disease using enzyme replacement therapy.Method: Using neuropsychological tests and brain magnetic resonance imaging (MRI), we prospectively assessed a cohort of 11 classic infantile Pompe patients aged up to 17 years.Results: From approximately age 2 years onwards, brain MRI showed involvement of the periventricular white matter and centrum semiovale. After 8 years of age, additional white-matter abnormalities occurred in the corpus callosum, internal and external capsule, and subcortical areas. From 11 years of age, white-matter abnormalities were also found in the brainstem. Although there seemed to be a characteristic pattern of involvement over time, there were considerable variations between patients, reflected by variations in neuropsychological development. Cognitive development ranged from stable and normal to declines that lead to intellectual disabilities.Interpretation: As treatment enables patients with classic infantile Pompe disease to reach adulthood, white-matter abnormalities are becoming increasingly evident, affecting the neuropsychological development. Therefore, we advise follow-up programs are expanded to capture CNS involvement in larger, international patient cohorts, to incorporate our findings in the counselling of parents before the start of treatment, and to include the brain as an additional target in the development of next-generation therapeutic strategies for classic infantile Pompe disease.What This Paper Adds: In our long-term survivors treated intravenously with enzyme replacement therapy, we found slowly progressive symmetric white-matter abnormalities. Cognitive development varied from stable and normal to declines towards intellectual disabilities. [ABSTRACT FROM AUTHOR]

Aim: As there is little evidence for concurrent validity of the Eating and Drinking Ability Classification System (EDACS), this study aimed to determine its concurrent validity and reliability in children and adolescents with cerebral palsy (CP).Method: After an extensive translation procedure, we applied the German language version to 52 participants with CP (30 males, 22 females, mean age 9y 7mo [SD 4y 2mo]). We correlated (Kendall's tau or Kτ ) the EDACS levels with the Bogenhausener Dysphagiescore (BODS), and the EDACS level of assistance with the Manual Ability Classification System (MACS) and the item 'eating' of the Functional Independence Measure for Children (WeeFIM). We further quantified the interrater reliability between speech and language therapists (SaLTs) and between SaLTs and parents with Kappa (κ).Results: The EDACS levels correlated highly with the BODS (Kτ =0.79), and the EDACS level of assistance correlated highly with the MACS (Kτ =0.73) and WeeFIM eating item (Kτ =-0.80). Interrater reliability proved almost perfect between SaLTs (EDACS: κ=0.94; EDACS level of assistance: κ=0.89) and SaLTs and parents (EDACS: κ=0.82; EDACS level of assistance: κ=0.89).Interpretation: The EDACS levels and level of assistance seem valid and showed almost perfect interrater reliability when classifying eating and drinking problems in children and adolescents with CP.What This Paper Adds: The Eating and Drinking Ability Classification System (EDACS) correlates well with a dysphagia score. The EDACS level of assistance proves valid. The German version of EDACS is highly reliable. EDACS correlates moderately to highly with other classification systems. [ABSTRACT FROM AUTHOR]

Aim: To analyze the clinical outcome and neuroimaging over a long duration follow-up in the currently largest series of acute encephalopathy after status epilepticus in patients with Dravet syndrome.Method: Clinical and neuroimaging data of patients with Dravet syndrome with a history of acute encephalopathy (coma >24h) after status epilepticus from February 2005 to December 2016 at Peking University First Hospital were reviewed retrospectively.Results: Thirty-five patients (15 males, 20 females) with a history of acute encephalopathy were enrolled from a total of 624 patients with Dravet syndrome (5.6%). The median onset age of acute encephalopathy was 3 years 1 month. The duration of status epilepticus varied between 40 minutes to 12 hours. Thirty-four patients had a high fever when status epilepticus occurred, and only one had a normal temperature. Coma lasted from 2 to 20 days. Twelve patients died and 23 survived with massive neurological regression. The median follow-up time was 2 years 1 month. Neuroimaging of 20 out of 23 survivors during the recovery phase showed diverse degrees of cortical atrophy with or without subcortical lesions.Interpretation: Acute encephalopathy after status epilepticus is more prone to occur in patients with Dravet syndrome who had a high fever. The mortality rate is high in severe cases. Survivors are left with severe neurological sequelae but often with either no seizure or low seizure frequency.What This Paper Adds: Acute encephalopathy is more prone to occur in patients with Dravet syndrome with a high fever. The mortality rate is high for acute encephalopathy after status epilepticus in patients with Dravet syndrome. Survivors have neurological sequelae. [ABSTRACT FROM AUTHOR]

Aim: Adolescents with physical disabilities may have co-occurring chronic pain, but the prevalence and specific associated factors are unknown. The aims of this study were to determine (1) the prevalence of chronic pain in adolescents with physical disabilities and (2) whether known correlates of chronic pain in the general population are also present in young people both with physical disability and with chronic pain relative to peers.Method: We conducted a secondary analysis of cross-sectional nationally representative data from the National Longitudinal Study of Adolescent to Adult Health. Multivariate linear regression analysis was used to identify demographic and psychosocial factors associated with chronic pain.Results: A total of 989 (4.3%) adolescents reported physical disabilities. They had a significantly higher rate of pain (27.2%) compared with able-bodied peers (15.6%, χ2 =86.3550, p<0.001). There was no significant interaction between physical disability status and chronic pain in relation to depressive symptoms, anxiety, or insomnia.Interpretation: Adolescents with physical disabilities experience chronic pain at a significantly higher rate than able-bodied peers, but the comorbidity of physical disability and chronic pain is not related to depression, anxiety, or insomnia. Evaluation of chronic pain and tailored pain interventions need to be developed for this population.What This Paper Adds: Chronic pain and its correlates are important problems for adolescents with physical disabilities. These adolescents present with higher rates of chronic pain than other young people. Chronic pain is associated with increased levels of depressive symptoms, anxiety, and insomnia regardless of disability status. [ABSTRACT FROM AUTHOR]

Aim: To assess long-term safety and efficacy of stiripentol as an antiepileptic medication for people with Dravet syndrome.Method: A prospective, observational open-label study (2003-2015) of the efficacy and long-term safety of stiripentol in patients with Dravet syndrome and ongoing seizures. Frequency of generalized tonic-clonic seizures, focal seizures, status epilepticus, and adverse events were recorded.Results: Forty-one patients started stiripentol, with median age at enrolment 5 years 7 months (range 11mo-22y) and median duration of treatment 37 months (range 2-141mo). Twenty out of 41 patients had greater than or equal to 50% long-term reduction in frequency of generalized tonic-clonic seizures. Frequency of focal seizures was decreased by greater than or equal to 50% in 11 out of 23 patients over the long-term. Frequency of status epilepticus was decreased by 50% or more in 11 out of 26 patients. The most common adverse events were anorexia, weight loss, sedation, and behavioural changes. One patient had worsening of absence and myoclonic seizures. Another developed recurrent pancreatitis on concurrent valproate.Interpretation: Stiripentol improves long-term seizure frequency in approximately 50% of patients with Dravet syndrome, when used as part of unrestricted polytherapy. Long-term use appears safe. In more than 40% of patients, episodes of status epilepticus markedly decrease after stiripentol initiation. What this paper adds Frequency of status epilepticus is reduced in 40% of patients with Dravet syndrome after stiripentol initiation. Stiripentol is effective for generalized tonic-clonic and focal seizures. Stiripentol can be safely used with a range of antiepileptic drugs. [ABSTRACT FROM AUTHOR]

Objective: Distress screening programs aim to ensure appropriate psychooncological support for cancer patients, but many eligible patients do not use these services. To improve distress management, we need to better understand patients' supportive care needs. In this paper, we report the first key finding from a longitudinal study that focused on patients' intentions to use psychooncological support and its association with distress and uptake of the psychooncology service.Methods: We conducted a prospective, observational study in an Oncology Outpatient Clinic and assessed distress, intention to use psychooncological support, and uptake of the psychooncology service by using the Distress Thermometer, a semistructured interview, and hospital records. We analyzed data with a mixed-methods approach.Results: Of 333 patients (mean age 61 years; 55% male; 54% Distress Thermometer ≥ 5), 25% intended to use the psychooncology service (yes), 33% were ambivalent (maybe), and 42% reported no intention (no). Overall, 23% had attended the psychooncology service 4 months later. Ambivalent patients reported higher distress than patients with no intention (odds ratio = 1.18, 95% confidence interval [1.06-1.32]) but showed significantly lower uptake behavior than patients with an intention (odds ratio = 14.04, 95% confidence interval [6.74-29.24]). Qualitative analyses revealed that ambivalent patients (maybe) emphasized fears and uncertainties, while patients with clear intentions (yes/no) emphasized knowledge, attitudes, and coping concepts.Conclusions: We identified a vulnerable group of ambivalent patients with high distress levels and low uptake behavior. To optimize distress screening programs, we suggest addressing and discussing patients' supportive care needs in routine clinical practice. [ABSTRACT FROM AUTHOR]

Introduction and Aims: Take-home naloxone (THN) programs commenced in Australia in 2012 in the Australian Capital Territory and programs now operate in five Australian jurisdictions. The purpose of this paper is to record the progress of THN programs in Australia, to provide a resource for others wanting to start THN projects, and provide a tool for policy makers and others considering expansion of THN programs in this country and elsewhere.Design and Methods: Key stakeholders with principal responsibility for identified THN programs operating in Australia provided descriptions of program development, implementation and characteristics. Short summaries of known THN programs from each jurisdiction are provided along with a table detailing program characteristics and outcomes.Results: Data collected across current Australian THN programs suggest that to date over 2500 Australians at risk of overdose have been trained and provided naloxone. Evaluation data from four programs recorded 146 overdose reversals involving naloxone that was given by THN participants.Discussion and Conclusions: Peer drug user groups currently play a central role in the development, delivery and scale-up of THN in Australia. Health professionals who work with people who use illicit opioids are increasingly taking part as alcohol and other drug-related health agencies have recognised the opportunity for THN provision through interactions with their clients. Australia has made rapid progress in removing regulatory barriers to naloxone since the initiation of the first THN program in 2012. However, logistical and economic barriers remain and further work is needed to expand access to this life-saving medication. [ABSTRACT FROM AUTHOR]

Introduction and Aims: Tranquilizer use is associated with negative health outcomes among people who use drugs. This paper aims to estimate the incidence rate (initiation) and identify predictors of tranquilizer misuse (TM) among cocaine users.Design and Methods: A prospective cohort study was conducted in Montreal, Canada. Interviewer-administered questionnaires were carried out at 3-month intervals. Initiation was defined as misusing a tranquilizer for the first time during follow-up. 'Lasting-initiation', defined as reporting TM at the next visit following first use, was also examined. Cox proportional hazard regression analyses were carried out to assess predictors of initiation to TM.Results: Among the 245 participants who were eligible for the initiation analyses, 123 started TM during follow-up for an incidence rate of 40.49 per 100 person-years (95% confidence interval, CI: 33.80-48.15). Of the 123 initiates, 35.7% were still using at the interview following initiation for an incidence rate of 14.70 per 100 person-years (95% CI: 10.82-19.56). Multivariate models showed that independent predictors of initiation and 'lasting initiation' were identical as having had a criminal activity as the main source of income, having been recently in treatment for a substance use disorder (SUD) and reporting non-medical use of prescription opioids.Discussion and Conclusions: The incidence rate of TM initiation was high among a sample of cocaine users. Initiation was predicted by a problematic drug use pattern involving polydrug use, involvement in the street economy and having been in treatment for a SUD. These findings have implications for prevention and harm reduction strategies. [ABSTRACT FROM AUTHOR]

Aim: To explore the evolution of self-care and functional mobility after multilevel surgery in children and adolescents with spastic diplegic cerebral palsy and to identify factors affecting these outcomes.Method: Thirty-four participants (22 males, 12 females) were evaluated before surgery, and at 2 months, 6 months, 1 year, 18 months, and 2 years after surgery. Self-care was assessed with the Pediatric Evaluation of Disability Inventory Dutch edition. The Mobility Questionnaire47 (MobQues47) and Functional Mobility Scale (FMS) were used to measure functional mobility.Results: All outcomes revealed a significant decrease 2 months after single-event multilevel surgery (SEMLS) (p-value between <0.001 and 0.02) followed by a significant increase at 6 months (p<0.001 and p=0.045). Between 6 months and 1 year, a significant increase was also revealed for Mobques47 (p<0.001), FMS (p≤0.008), and the Pediatric Evaluation of Disability Inventory Functional Skills Scale (PEDI-FSS) (p=0.001). Improvement continued until 18 months for the PEDI-FSS. Initial score, initial muscle strength, Gross Motor Function Classification System level, age, and number of surgical interventions significantly influenced time trends for self-care and/or functional mobility.Interpretation: Most preoperative scores are regained at 6 months after SEMLS. Further improvement is seen until 18 months. Participants with a higher functional level before surgery will temporarily lose more than participants with lower initial functional ability, but they also fast regain their function.What This Paper Adds: Self-care and functional mobility decrease significantly in the first months after single-event multilevel surgery (SEMLS). Six months after SEMLS most preoperative scores are regained. Impact of SEMLS is more pronounced for functional mobility than for self-care. Muscle strength and functionality at baseline are important influencing factors on the evolution after SEMLS. [ABSTRACT FROM AUTHOR]

Aim: This study examined the effectiveness of the Pathways and Resources for Engagement and Participation (PREP) intervention in improving the participation of adolescents in community-based activities.Method: Twenty-eight adolescents (14 males, 14 females), aged 12 to 18 years (mean 14y 6mo, SD 21.6mo) with moderate physical disabilities participated in a 12-week PREP intervention. An interrupted time series design with multiple baselines was employed, replicating the intervention effect across three chosen activities and all participants. An occupational therapist worked individually with adolescents and parents to identify and implement strategies to remove environmental barriers that impede participation in selected activities. Activity performance was repeatedly measured using the Canadian Occupational Performance Measure (COPM) at baseline, intervention, and follow-up (20wks). For each activity, the trajectory representing change in performance was analyzed descriptively. Segmented regression combined with a mixed-effects modeling approach was used to statistically estimate the overall effectiveness of the intervention within and across 79 activities.Results: A statistically significant improvement (B=2.08, p<0.001) was observed across all activities, 59 per cent of which also indicated a clinically significant change of more than 2 points on the COPM scale. Levels of performance were maintained during follow-up with an additional increase of 0.66 points on the COPM scale (t=3.04, p=0.004). Intervention was most effective for males and those with a higher number of functional issues.Interpretation: Findings illustrate that participation can be improved by changing the environment only. Such evidence further supports emerging therapeutic approaches that are activity-based, goal-oriented, and ecological in nature.What This Paper Adds: Environment-based intervention strategies, guided by the Pathways and Resources for Engagement and Participation, are effective in improving and maintaining adolescent participation. Intervention was most effective for males and those with a higher number of functional issues. The study design serves as an example for future pragmatic studies accounting for individual-based changes and contexts. [ABSTRACT FROM AUTHOR]

Aim: To describe the development of bimanual performance among young children with unilateral or bilateral cerebral palsy (CP).Method: A population-based sample of 102 children (53 males, 49 females), median age 28.5 months (interquartile range [IQR] 16mo) at first assessment and 47 months (IQR 18mo) at last assessment, was assessed half-yearly with the Assisting Hand Assessment (AHA) or the Both Hands Assessment (BoHA) for a total of 329 assessments. Developmental limits and rates were estimated by nonlinear mixed-effects models. Developmental trajectories were compared between levels of manual ability (Mini-Manual Ability Classification System [Mini-MACS] and MACS) and AHA or BoHA performance at 18 months of age (AHA-18/BoHA-18) for both CP subgroups, and additionally between children with bilateral CP with symmetric or asymmetric hand use.Results: For both CP subgroups, children classified in Mini-MACS/MACS level I, and those with high AHA-18 or BoHA-18 reached the highest limits of performance. For children with bilateral CP the developmental change was small, and children with symmetric hand use reached the highest limits.Interpretation: Mini-MACS/MACS levels and AHA-18 or BoHA-18 distinguished between various developmental trajectories both for children with unilateral and bilateral CP. Children with bilateral CP changed their performance to a smaller extent than children with unilateral CP.What This Paper Adds: Manual Ability Classification System levels and Assisting Hand Assessment/Both Hands Assessment performance at 18 months are important predictors of hand use development in cerebral palsy (CP). Children with bilateral CP improved less than those with unilateral CP. Children with bilateral CP and symmetric hand use reached higher limits than those with asymmetry. [ABSTRACT FROM AUTHOR]

Abstract: Background: Due to civil war, Somalis immigrants have settled in Norway and the United States. Large families are valued among Somalis and Somali women continue to give birth in their new countries. Research studies have been conducted with Somali immigrant new mothers in Norway and Minnesota, United States to understand perceptions of their childbirth experience. Purpose: The purpose of this qualitative study was to compare the childbirth experiences of Somali immigrant mothers in the United States and Norway to make recommendations to improve health care practice. Design: This paper presents a comparison of two research studies in order to identify and explain similarities and differences between cross‐cultural immigrant populations. Results: Both studies identified themes related to the importance of family support in the postpartum period, fear of Cesarean delivery, and relationships with nurses. These studies give recommendations for nurses on how to provide culturally sensitive care for Somali new mothers. Conclusions: Implications for practice are that nurses should develop trusting relationships with Somali mothers and facilitate cultural and religious practices. Information regarding support resources in the community should be provided to Somali mothers throughout the perinatal period. [ABSTRACT FROM AUTHOR]

Objectives: Suicide among nursing home residents is a growing public health concern, currently lacking in empirical research. This study aims to describe the frequency and nature of suicide among nursing home residents in Australia.Methods: This research comprised a national population-based retrospective analysis of suicide deaths among nursing home residents in Australia reported to the Coroner between July 2000 and December 2013. Cases were identified using the National Coronial Information System, and data collected from paper-based coroners' records on individual, incident, and organizational factors, as well as details of the medico-legal death investigation. Data analysis comprised univariate and bivariate descriptive statistical techniques; ecological analysis of incidence rates using population denominators; and comparison of age and sex of suicide cases to deaths from other causes using logistic regression.Results: The study identified 141 suicides among nursing home residents, occurring at a rate of 0.02 deaths per 100 000 resident bed days. The ratio of deaths from suicide to deaths from any other cause was higher in males than females (OR = 3.56, 95%CI = 2.48-5.12, P = <0.001). Over half of the residents who died from suicide had a diagnosis of depression (n = 93, 66.0%) and had resided in the nursing home for less than 12 months (n = 71, 50.3%). Common major life stressors identified in suicide cases included the following: health deterioration (n = 112, 79.4%); isolation and loneliness (n = 60, 42.6%); and maladjustment to nursing home life (n = 42, 29.8%).Conclusions: This research provides a foundational understanding of suicide among nursing home residents in Australia and contributes important new information to the international knowledge base. [ABSTRACT FROM AUTHOR]

Aim: To compare three reaching movements made in two planes between a low-cost, game-based virtual reality and a matched physical environment in typically developing children and children with cerebral palsy (CP). To determine if differences in kinematics are related to sensory deficits.Method: An observational study in which 27 children (typically developing, n=17, mean age 13y, [SD] 2y 2mo, range 9y 3mo-17y 2mo; CP, n=10, mean age 13y 8mo, [SD] 1y 8mo, range 11y 1mo-17y 1mo, Manual Ability Classification System levels I-II) performed 15 trials of three gestures in each of a virtual reality and a matched physical environment. Upper-limb and trunk kinematics were recorded using an electromagnetic system (G4, Polhemus, six markers, 120Hz).Results: Compared to the physical environment, movements in virtual reality made by typically developing children were slower (p=0.002), and involved less trunk flexion (p=0.002) and rotation (p=0.026). Children with CP had more curved trajectories (p=0.005) and used less trunk flexion (p=0.003) and rotation (p=0.005). Elbow and shoulder kinematics differed from 2.8% to 155.4% between environments in both groups. Between groups, there were small, clinically insignificant differences with only the vertical gesture being longer in typically developing children. Children with CP who had greater tactile impairment used more trunk displacement.Interpretation: Clinicians and researchers need to be aware of differences in movement variables when setting goals or designing protocols for improving reaching in children with CP using low-cost, game-based virtual reality systems.What This Paper Adds: Upper-limb kinematics differed in each group when reaching in physical versus virtual environments. There were small differences in movements made by children with mild unilateral cerebral palsy (CP) compared to typically developing children. Differences in reaching kinematics should be considered when goal setting using virtual reality interventions for children with mild unilateral CP. [ABSTRACT FROM AUTHOR]

Aim: To describe health-related quality of life of Australian children and adolescents with Down syndrome and compare it with norm-referenced data.Method: A cross-sectional survey was conducted with parents of 75 children and adolescents (43 males, 32 females) with Down syndrome aged 5 to 18 years (mean age 13y 2mo, SD 4y 8mo). The proxy-report KIDSCREEN-27 questionnaire was administered and five dimensions of health-related quality of life were measured. Data were analysed descriptively and compared with normative data.Results: Total group mean scores for psychological well-being, autonomy and parent relation, and school environment dimensions were within normal threshold values, whereas mean scores for physical well-being, and social support and peers dimensions, were poorer. For participants with Down syndrome aged 8 to 18 years, the difference with normative data for proxy-reported physical well-being, psychological well-being, and social support and peers dimensions favoured typically developing children. Adolescents (13-18y) with Down syndrome scored poorer on all dimensions than children (5-12y) with Down syndrome.Interpretation: Our findings assist a better understanding of the lived experiences of children and adolescents with Down syndrome, as perceived by their parents, and suggest aspects of health that could be influenced to optimize their quality of life.What This Paper Adds: Proxy-reported psychological well-being and autonomy were within the normal range for children with Down syndrome. Physical well-being and social support scores were significantly lower than normative data. Proxy-reported scores for adolescents with Down syndrome were consistently poorer than for children with Down syndrome and the differences were clinically important. [ABSTRACT FROM AUTHOR]

Aim: To determine whether intrathecal baclofen (ITB) therapy improves performance and performance satisfaction in goal areas identified by patients' parents.Method: This study formed part of an ongoing multicentre national audit involving six paediatric ITB pump implant centres across Australia. The Canadian Occupational Performance Measure was the primary outcome measure utilized at baseline, 6 months, and 12 months after pump implants in paediatric patients receiving ITB therapy for the first time between 31st December 2009 and 31st December 2014.Results: Twenty-five children had goals identified (mean age 11y 1mo), 19 had a diagnosis of cerebral palsy and 22 were at Gross Motor Function Classification System level IV, V, or equivalent. Strong evidence for an improvement in goal performance (2.33, 95% CI 1.70, 2.96, p<0.001) and performance satisfaction scores (3.08, 95% CI 2.28, 3.88, p<0.001) were demonstrated at 6 months, compared to baseline. The differences were clinically significant and were sustained to 12 months.Interpretation: ITB therapy in paediatric patients with hypertonia results in clinically significant improvements in average performance and performance satisfaction scores.What This Paper Adds: The most commonly identified goals of parents of children treated with intrathecal (ITB) therapy were: improving ease of dressing, positioning, and transfers. ITB therapy is effective in improving performance and performance satisfaction in children with hypertonia. Score improvements are mainly evident within the first 6 months of therapy. [ABSTRACT FROM AUTHOR]

Aim: To review the demographics and clinical and paraclinical parameters of children with myelin oligodendrocyte glycoprotein (MOG) antibody-associated relapsing disease.Method: In this UK-based, multicentre study, 31 children with MOG antibody-associated relapsing disease were studied retrospectively.Results: Of the 31 children studied, 14 presented with acute disseminated encephalomyelitis (ADEM); they were younger (mean 4.1y) than the remainder (mean 8.5y) who presented with optic neuritis and/or transverse myelitis (p<0.001). Similarly, children who had an abnormal brain magnetic resonance imaging (MRI) at onset (n=20) were younger than patients with normal MRI at onset (p=0.001) or at follow-up (p<0.001). 'Leukodystrophy-like' MRI patterns of confluent largely symmetrical lesions was seen during the course of the disease in 7 out of 14 children with a diagnosis of ADEM, and was only seen in children younger than 7 years of age. Their disability after a 3-year follow-up was mild to moderate, and most patients continued to relapse, despite disease-modifying treatments.Interpretation: MOG antibody should be tested in children presenting with relapsing neurological disorders associated with confluent, bilateral white matter changes, and distinct enhancement pattern. Children with MOG antibody-associated disease present with age-related differences in phenotypes, with a severe leukoencephalopathy phenotype in the very young and normal intracranial MRI in the older children. This finding suggests a susceptibility of the very young and myelinating brain to MOG antibody-mediated mechanisms of damage.What This Paper Adds: Myelin oligodendrocyte glycoprotein (MOG) antibody-associated demyelination manifest with an age-related phenotype. Children with MOG antibody and 'leukodystrophy-like' imaging patterns tend to have poor response to second-line immunotherapy. [ABSTRACT FROM AUTHOR]

Aim: To systematically review evidence for pharmacological/neurosurgical interventions for managing dystonia in individuals with cerebral palsy (CP) to inform a care pathway.Method: Searches included studies with a minimum of five participants with dystonia in CP receiving oral baclofen, benzodiazepines (clonazepam, diazepam, lorazepam), clonidine, gabapentin, levodopa, trihexyphenidyl, botulinum toxin, intrathecal baclofen (ITB), or deep brain stimulation (DBS). Evidence was classified according to American Academy of Neurology guidelines.Results: Twenty-eight articles underwent data extraction: one levodopa, five trihexyphenidyl, three botulinum toxin, six ITB, and 13 DBS studies. No articles for oral baclofen, benzodiazepines, clonidine, or gabapentin met the inclusion criteria. Evidence for reducing dystonia was level C (possibly effective) for ITB and DBS; level C (possibly ineffective) for trihexyphenidyl; and level U (inadequate data) for botulinum toxin.Interpretation: For dystonia reduction, ITB and DBS are possibly effective, whereas trihexyphenidyl was possibly ineffective. There is insufficient evidence to support oral medications or botulinum toxin to reduce dystonia. There is insufficient evidence for pharmacological and neurosurgical interventions to improve motor function, decrease pain, and ease caregiving. The majority of the pharmacological and neurosurgical management of dystonia in CP is based on clinical expert opinion.What This Paper Adds: Intrathecal baclofen and deep brain stimulation are possibly effective in reducing dystonia. Current evidence does not support effectiveness of oral medications or botulinum toxin to reduce dystonia. Evidence is inadequate for pharmacological/neurosurgical interventions impact on improving motor function, pain/comfort, and easing caregiving. The majority of the care pathway rests on expert opinion. [ABSTRACT FROM AUTHOR]

Aim: To identify a cohort of children with cerebral palsy (CP) from hospital data; determine the proportion that participated in standardized educational testing and attained a score within the normal range; and describe the relationship between test results and motor symptoms.Method: This population-based retrospective cohort study used data from New South Wales, Australia. We linked hospital data for children younger than 16 years of age admitted between 1st July 2000 and 31st March 2014 to education data from 2009 to 2014. Hospital diagnosis codes were used to identify a cohort of children with CP (n=3944) and describe their motor symptoms. Educational outcomes in the CP cohort were compared with those among children without CP.Results: Of those with educational data (n=1770), 46% were exempt from reading assessment because of intellectual or functional disability, 7% were absent or withdrawn from testing and 47% participated in testing. About 30% of all children with educational data had test scores in the normal range. The proportion was greatest among those with hemiplegia (>40%) and lowest among those with tetraplegia (<10%).Interpretation: One-third of children with CP participated in standardized testing and achieved a result in the normal range. The proportions were lower in children with more severe motor symptoms.What This Paper Adds: From 2009 to 2014, most Australian children with cerebral palsy (CP) attended a mainstream school. The rate of disability-related exemption from standardized educational testing was almost 50%. Thirty per cent of children with CP achieved educational scores in the normal range. [ABSTRACT FROM AUTHOR]

Aim: To describe behavioural and psychiatric outcomes of children within 10 years of convulsive status epilepticus (CSE).Method: Children originally identified by the population-based North London Convulsive Status Epilepticus in Childhood Surveillance Study were followed-up between July 2009 and February 2013. They were grouped into epilepsy- and non-epilepsy-related CSE, and compared with population norms and healthy controls using the Strengths and Difficulties Questionnaire; the Autism Spectrum Screening Questionnaire; and the Swanson, Nolan, and Pelham questionnaire. Children who scored above recommended clinical cut-offs on any scale were invited for a neuropsychiatric assessment. Regression models were fitted to identify clinically relevant covariates associated with behavioural outcomes.Results: At a mean follow-up of 8.1 years post-CSE, 28% of enrolled children were found to have a psychiatric disorder. Children with epilepsy-related CSE scored higher than norms on all scales and children with non-epilepsy-related CSE scored higher than norms on the Strengths and Difficulties Questionnaire and the Autism Spectrum Screening Questionnaire. Presence of seizures at baseline and recurrence of CSE was associated with worse outcomes in the group with epilepsy. Intellectual abilities were associated with behavioural outcomes in all participants.Interpretation: A large proportion of children manifest behavioural issues 8 years after CSE. The present data highlight the need for behavioural screening in children with neurodevelopmental impairments post-CSE.What This Paper Adds: Eight years post convulsive status epilepticus (CSE), 37% of parents report behavioural issues. Of enrolled children, 28% were found to have a Diagnostic and Statistical Manual mental disorder. Intellectual abilities are strongly associated with behavioural outcomes in children post-CSE. [ABSTRACT FROM AUTHOR]

Based on two years' ethnographic research on doctor-patient relations in urban China, this paper examines the causes of patients' mistrust of physicians. I identify the major factors at the societal, institutional, and interpersonal levels that lead to patients' mistrust of physicians. First, I set the context by describing the extent of mistrust at the societal level. Then, I investigate the institutional sources of mistrust. I argue that the financing mechanism of public hospitals and physicians' income structures are the most crucial factors in inducing patients' mistrust. Hospitals' heavy reliance on self-finance has basically caused public hospitals to run like private hospitals, resulting in blatant conflicts of interest between hospitals and patients. Related to this is physicians' reliance on bonuses and commissions as part of their regular incomes, which has inevitably resulted in overtreatment and, hence, mistrust from the patients. At the interpersonal level, I describe how individual physicians' attitudes toward and interaction with patients may also affect patients' sense of trust or mistrust in physicians. In conclusion, I discuss the ethical implications of the mistrust problem, and suggest changes at the institutional and interpersonal levels to mitigate the problem. [ABSTRACT FROM AUTHOR]

Aim: To develop an instrument (Paediatric Rehabilitation Ingredients Measure [PRISM]) for quantitative estimation of contents of interdisciplinary neurorehabilitation for use in studies of relationships between rehabilitation treatment delivered and severity-adjusted outcomes after acquired brain injury (ABI).Method: The measure was developed using an ingredients-mediators-outcomes model consistent with the International Classification of Functioning, Disability and Health, a literature review, and other current initiatives in the development of rehabilitation treatment taxonomies, with item codevelopment in workshops with rehabilitation professionals. Interrater reliability was assessed in inpatient and residential paediatric rehabilitation settings.Results: Although sometimes an initially unfamiliar perspective on rehabilitation practice, PRISM's acceptability amongst professionals was excellent. Internal consistency of scores was sometimes an issue for users unfamiliar with the tool; however, this improved with practice and interrater reliability (assessed by Kendall's W) was good. The tool was felt to have particular value in facilitating interdisciplinary communication and working. Modifications to the design of the tool have improved internal consistency.Interpretation: PRISM supports identification of the 'active ingredients' of an interdisciplinary rehabilitation package and facilitates interdisciplinary communication. It also has potential as a research tool examining relationships between rehabilitation delivered and severity-adjusted outcomes observed after paediatric ABI.What This Paper Adds: Identifying contribution of rehabilitation to outcomes after acquired brain injury requires quantification of rehabilitation 'dose' and 'content'. Previous approaches to 'parsing' of rehabilitation dose and content may have overemphasized one-to-one sessions with therapists. We present a novel, holistic tool for identification of ingredients of an interdisciplinary rehabilitation package. It supports interdisciplinary communication and has potential as a research tool. [ABSTRACT FROM AUTHOR]

Aim: The aims of this study were twofold: first, to develop and validate a timed test of unimanual and bimanual dexterity suitable for those with disability affecting hand function; second, to explore relationships between unimanual and bimanual completion times.Method: We developed the Tyneside Pegboard Test (TPT), an electronically timed test with three peg sizes, incorporating an asymmetrical bimanual task. Nine hundred and seventy-four participants (455 males, 519 females; age range 4-80y) provided normative data. Test-retest reliability and construct validity were assessed (50 adults: 14 males, 36 females; 15-73y) on two occasions 2 weeks apart. Bimanual and unimanual completion times were measured in 87 children (51 males, 36 females) with unilateral cerebral palsy (CP) and 498 individuals in a comparison group (238 males, 260 females; 5-15y).Results: The comparison group showed an asymmetrical U-shaped relationship between completion times and age. Intraclass correlation coefficients ranged from 0.74 to 0.91, indicating moderate test-retest reliability. There was a negative relationship between average TPT bimanual times and Purdue pegboard bimanual scores (Spearman's rho -0.611, degrees of freedom 44, p<0.001). Children with unilateral CP had greater prolongation of bimanual than unimanual completion times compared with the comparison group (mean difference 20.31s, 95% confidence interval 18.13-22.49, p<0.001).Interpretation: The TPT is accessible for those with impaired hand function. Children with unilateral CP demonstrated disproportionate bimanual deficits, even allowing for unimanual dexterity: this has implications for therapy.What This Paper Adds: We developed an adapted, electronically timed 9-hole pegboard test. Our modifications facilitate use by those with disability affecting hand function. The test incorporates an asymmetrical bimanual task. Children with unilateral cerebral palsy showed disproportionate bimanual dexterity deficits even allowing for unimanual dexterity. [ABSTRACT FROM AUTHOR]

Aim: This study examined cross-sectional and longitudinal patterns of parenting stress, adult anxiety, and depression in mothers of children with profound or severe visual impairment (PVI or SVI) at 1 year and 2 years of age.Method: Mothers of a national longitudinal cohort (OPTIMUM Project) of infants with congenital disorders of the peripheral visual system and PVI (light perception at best) or SVI (basic 'form' vision of non-light reflecting objects) participated. Infant age at baseline (T1 ) was 8 to 16 months. Mothers completed the Parenting Stress Index - Short Form and the Hospital Anxiety and Depression Scale at T1 (n=79) and at follow-up 12 months later (T2 ) (n=73).Results: Mothers of the total group had higher parenting stress levels (34.6% in clinical range) than community normative data at T1 (p=0.017). Mothers of infants in the PVI subgroup had elevated stress at T1 (p=0.014) and T2 (p=0.009). The PVI subgroup was also elevated in the Difficult Child subscale at T2 (p=0.001). Within-sample differences in parenting stress between the visual impairment subgroups were found at T2 only: the PVI subgroup scored higher than the SVI subgroup (p=0.029). Adult anxiety and depression in the total group were not elevated compared with community normative data at T1 and T2 ; however, higher parenting stress was related to raised adult anxiety and depression levels at T1 and T2 (p=0.001). Regression analysis found parenting stress and lower child vision level (T1 ) predicted parenting stress (T2 ) (p=0.001; 42% variance).Interpretation: Mothers of 1-year-old infants with visual impairment showed raised risk for parenting stress, which continued to be elevated for children with PVI and those perceived as 'difficult' at 2 years. This was also a psychological risk, with greater adult anxiety and depression in those mothers with raised parenting stress. The clinical significance is that identification of parenting stress and targeted parenting, and behavioural support of the child in the first years of life is highly indicated.What This Paper Adds: Mothers of infants with visual impairment are at increased risk of parenting stress. Parenting stress was higher in mothers of children with profound visual impairment than those with severe visual impairment. High levels of parenting stress and lower infant vision at 1 year of age predicted higher parenting stress at 2 years of age. [ABSTRACT FROM AUTHOR]

Aim: Executive function impairments have been identified in children with poor motor skills, with and without a diagnosis of developmental coordination disorder (DCD). However, most studies are cross-sectional. This study investigates the development of executive function in children with poor motor skills over 2 years.Method: Children aged 7 to 11 years (n=51) were assessed twice, 2 years apart, on verbal and nonverbal measures of executive functions: executive-loaded working memory (ELWM); fluency; response inhibition; planning; and cognitive flexibility. Typically developing children (n=17) were compared with those with a clinical diagnosis of DCD (n=17) and those with identified motor difficulties (n=17) but no formal diagnosis of DCD.Results: Developmental gains in executive function were similar between groups, although a gap between children with poor motor skills and typically developing children on nonverbal executive functions persisted. Specifically, children with DCD performed significantly more poorly than typically developing children on all nonverbal executive function tasks and verbal fluency tasks at both time points; and children with motor difficulties but no diagnosis of DCD showed persistent executive function problems in nonverbal tasks of ELWM and fluency.Interpretation: Children with DCD and motor difficulties demonstrated executive function difficulties over 2 years, which may affect activities of daily living and academic achievement, in addition to their motor deficit.What This Paper Adds: Executive function difficulties in children with poor motor skills persist throughout middle childhood. Children with motor difficulties, without a developmental coordination disorder (DCD) diagnosis, demonstrate less pervasive executive function difficulties than those with DCD. Executive function problems in the groups with motor difficulties and DCD affect mostly nonverbal domains. All groups showed similar developmental gains in executive function. [ABSTRACT FROM AUTHOR]

Aim: Difficulties in reading comprehension can arise from either word reading or listening comprehension difficulties, or a combination of the two. We sought to determine whether children with rolandic epilepsy had poor reading comprehension relative to typically developing comparison children, and whether such difficulties were associated with word reading and/or general language comprehension difficulties.Method: In this cross-sectional study, children with rolandic epilepsy (n=25; 16 males, 9 females; mean age 9y 1mo, SD 1y 7mo) and a comparison group (n=39; 25 males, 14 females; mean age 9y 1mo, SD 1y 3mo) completed assessments of reading comprehension, listening comprehension, word/non-word reading, speech articulation, and Non-verbal IQ.Results: Reading comprehension and word reading were worse in children with rolandic epilepsy (F1,61 =6.89, p=0.011, ηp2=0.10 and F1,61 =6.84, p=0.011, ηp2=0.10 respectively), with listening comprehension being marginal (F1,61 =3.81, p=0.055, ηp2=0.06). Word reading and listening comprehension made large and independent contributions to reading comprehension, explaining 70% of the variance.Interpretation: Children with rolandic epilepsy may be at risk of reading comprehension difficulties. Thorough assessment of individual children is required to ascertain whether the difficulties lie with decoding text, or with general comprehension skills, or both.What This Paper Adds: Children with rolandic epilepsy may be at risk of poor reading comprehension. This was related to poor word reading, poor listening comprehension, or both. Reading comprehension interventions should be tailored to the profile of difficulties. [ABSTRACT FROM AUTHOR]

Aim: This study examined whether late-preterm birth (34+0 to 36+6wks+d gestational age) was associated with neurocognitive deficit in young adulthood, and whether small for gestational age (SGA) birth amplified any adversity.Method: Participants derived from the prospective regional cohort study, the Arvo Ylppö Longitudinal Study (n=786; 398 females, 388 males) (mean age 25y 4mo, SD 8mo), born 1985 to 1986 late-preterm (n=119; 21 SGA, <-2 SD) and at term (37+0 to 41+6wks+d; n=667; 28 SGA) underwent tests of intelligence, executive functioning, attention, and memory, and reported their education.Results: Those born late-preterm scored -3.71 (95% confidence interval [CI] -6.71 to -0.72) and -3.11 (95% CI -6.01 to -0.22) points lower on Full-scale and Verbal IQ than peers born at term. Compared with those born at term and appropriate for gestational age (≥-2 to <2 SD) Full-scale, Verbal, and Performance IQ scores of those born late-preterm and SGA were -9.45 to -11.84 points lower. After adjustments, differences were rendered non-significant, except that scores in Full-scale and Performance IQ remained lower among those born late-preterm and SGA.Interpretation: Late-preterm birth, per se, may not increase the risk of poorer neurocognitive functioning in adulthood. But the double burden of being born late-preterm and SGA seems to increase this risk.What This Paper Adds: Late-preterm birth did not increase the risk of poorer neurocognitive functioning in adulthood. But the double burden of being born late-preterm and being small for gestational age did increase this risk. [ABSTRACT FROM AUTHOR]

Aim: To evaluate the efficacy of clobazam treatment in reducing epileptiform discharges and modifying neuropsychological function in continuous spike-wave during slow wave sleep.Method: We performed a prospective clinical trial in patients with continuous spike-wave during sleep aged 4 to 10 years. Patients underwent neuropsychological assessment and overnight electroencephalographic monitoring before treatment, and subsequent repeat assessment and overnight electroencephalographic monitoring 3 months after treatment. Treatment consisted of 1mg/kg clobazam up to a maximum dose of 30mg during the first night, followed by 0.5mg/kg nightly for 3 months.Results: Nine patients completed the study and had pre- and post-neuropsychological evaluation. There was a qualitative reduction in median (p25 -p75 ) spike percentage after 3 months (72.2 [68.0-75.8] vs 32.7 [4.7-81.7]). There were no marked changes in median (p25 -p75 ) IQ comparing pre- and post-clobazam treatment (80.0 [74.0-88.0] vs 80.0 [67.0-89.0]). There was a qualitative increase in Verbal IQ (83.0 [69.0-92.0] vs 95.0 [83.0-99.0]) and a qualitative decrease in Non-verbal IQ (84.0 [74.0-87.0] vs 71.0 [60.0-84.0]).Interpretation: Qualitative improvements in epileptiform activity and cognition occurred in patients treated with clobazam for 3 months and the relationship between epileptiform activity and cognitive outcome should be studied in larger studies.What This Paper Adds: Verbal IQ in patients with continuous spike-wave during sleep improved following short-term treatment with clobazam. Other neuropsychological improvements were observed, but varied by patient. Cognitive improvement was observed despite some worsening of epileptiform discharges. [ABSTRACT FROM AUTHOR]

Drug-drug interactions (DDIs) are a common cause of adverse drug events (ADEs). The electronic medical record (EMR) database and the FDA's adverse event reporting system (FAERS) database are the major data sources for mining and testing the ADE associated DDI signals. Most DDI data mining methods focus on pair-wise drug interactions, and methods to detect high-dimensional DDIs in medical databases are lacking. In this paper, we propose 2 novel mixture drug-count response models for detecting high-dimensional drug combinations that induce myopathy. The "count" indicates the number of drugs in a combination. One model is called fixed probability mixture drug-count response model with a maximum risk threshold (FMDRM-MRT). The other model is called count-dependent probability mixture drug-count response model with a maximum risk threshold (CMDRM-MRT), in which the mixture probability is count dependent. Compared with the previous mixture drug-count response model (MDRM) developed by our group, these 2 new models show a better likelihood in detecting high-dimensional drug combinatory effects on myopathy. CMDRM-MRT identified and validated (54; 374; 637; 442; 131) 2-way to 6-way drug interactions, respectively, which induce myopathy in both EMR and FAERS databases. We further demonstrate FAERS data capture much higher maximum myopathy risk than EMR data do. The consistency of 2 mixture models' parameters and local false discovery rate estimates are evaluated through statistical simulation studies. [ABSTRACT FROM AUTHOR]

Aim: To monitor the evolution of the motor function of ambulatory patients with Duchenne muscular dystrophy (DMD) treated by corticosteroids for 2 years in comparison with untreated patients.Method: This observational, multicentre cohort study explores the evolution of the motor function measure (MFM) over a 24-month period for 29 ambulant corticosteroids-treated and 45 ambulant untreated patients with DMD.Results: Significant differences were found between mean MFM scores in corticosteroids-treated and untreated groups for domain 1 of the MFM (standing position and transfers; D1), domain 2 of the MFM (axial and proximal motor function; D2), and domain 3 of the MFM (distal motor function; D3). Subscores were between 0 months and 6 months, and 0 months and 24 months. For the D1 subscore specifically, there was a significant increase in the corticosteroids-treated group (mean±standard deviation [SD] slope of change=12.6±15.5%/y), while a decrease was observed in the untreated group (-17.8±17.7%/y) between 0 months and 6 months (p<0.001). Sensitivity to change as assessed by standardized response means was high between 12 months and 24 months for D1 of both corticosteroids-treated and untreated groups (1.0 and 1.2 respectively), and low for D2 and D3 of both treated and untreated groups.Interpretation: Patients with DMD treated by corticosteroids present a different course of the disease as assessed by MFM, confirming the sensitivity to change of the MFM in this population.What This Paper Adds: Corticosteroids have a quantitative impact on muscle strength 6 to 24 months after starting treatment. Motor function measure is a valid outcome measure in Duchenne muscular dystrophy patients under corticosteroid treatment. [ABSTRACT FROM AUTHOR]

Aim: Many children with severe developmental and epileptic encephalopathies experience significant sleep disturbance, causing major disruption to the family's quality of life. We aimed to determine the frequency and nature of sleep problems in individuals with Dravet syndrome.Methods: The Sleep Disturbance Scale for Children and a seizure questionnaire were distributed to the parents/guardians of 96 patients with Dravet syndrome. Sixteen patients had two nights of home oximetry.Results: Fifty-seven out of 96 questionnaires were completed. Forty-three out of 57 (75%) individuals had sleep problems. Twenty-five out of 57 (44%) individuals had an abnormal total sleep score, with difficulty initiating and maintaining sleep (22 out of 57, 39%), sleep-wake transition disorders (20 out of 57, 35%), and sleep breathing disorders (19 out of 57, 33%). Twenty-two out of 57 (39%) individuals took medication to assist sleep, predominantly melatonin (n=14). Thirty out of 57 (53%) recently had nocturnal seizures. Overnight oximetry showed 14 out of 16 (88%) had a higher oxygen desaturation index (>3%), and six out of 16 (38%) had higher mean pulse rates than normative values. Home oximetry was normal or inconclusive in all patients.Interpretation: Seventy-five per cent of individuals with Dravet syndrome had sleep problems, highlighting the importance of routinely assessing sleep and initiating appropriate behavioural and pharmacological interventions to improve the patient and family's quality of life. A high oxygen desaturation index and mean pulse rates on pulse oximetry may reflect unrecognized nocturnal seizures.What This Paper Adds: More than 70% of patients with Dravet syndrome have sleep problems. Difficulty initiating and maintaining sleep was most common, particularly in those older than 20 years. Second most common were sleep-wake transition disorders, affecting more than 50% of those younger than 5 years. Sleep breathing disorders were a frequent problem across all age groups. Oximetry was not diagnostic of sleep-disordered breathing or obvious seizures. [ABSTRACT FROM AUTHOR]

Objective: Cancer in adolescents and young adults (AYAs) can interrupt important developmental milestones. Absence from school and time lost from work, together with the physical impacts of treatment on energy and cognition, can disrupt educational and vocational goals. The purpose of this paper is to report on AYA cancer survivors' experiences of reintegration into school and/or work and to describe perceived changes in their educational and vocational goals.Methods: Adolescents and young adults recruited from 7 hospitals in Australia, aged 15 to 26 years and ≤24 months posttreatment, were interviewed using the psychosocial adjustment to illness scale. Responses were analysed to determine the extent of, and explanations for, cancer's effect on school/work.Results: Forty-two AYA cancer survivors (50% female) participated. Compared with their previous vocational functioning, 12 (28.6%) were scored as experiencing mild impairment, 14 (33.3%) moderate impairment, and 3 (7.1%) marked impairment. Adolescents and young adults described difficulties reintegrating to school/work as a result of cognitive impacts such as concentration problems and physical impacts of their treatment, including fatigue. Despite these reported difficulties, the majority indicated that their vocation goals were of equal or greater importance than before diagnosis (26/42; 62%), and most AYAs did not see their performance as compromised (23/42; 55%). Many survivors described a positive shift in life goals and priorities. The theme of goal conflict emerged where AYAs reported compromised abilities to achieve their goals.Conclusions: The physical and cognitive impacts of treatment can make returning to school/work challenging for AYA cancer survivors. Adolescents and young adults experiencing difficulties may benefit from additional supports to facilitate meaningful engagement with their chosen educational/vocational goals. [ABSTRACT FROM AUTHOR]

Aim: To examine the differential effects of an early intervention programme for infants born preterm on neurodevelopment and parental mental health according to family social risk.Method: One hundred and twenty infants born earlier than 30 weeks' gestation were randomized to early intervention (n=61) or control groups (n=59). Cognitive, language, and motor outcomes were assessed by blinded assessors at 2 years, 4 years, and 8 years, and primary caregivers completed questionnaires on their anxiety and depression. Outcomes at each time point were compared between groups using linear regression with an interaction term for social risk (higher/lower).Results: There was evidence of interactions between intervention group and social risk for cognition at 2 years and 4 years, motor function at 4 years, and language at 8 years, with a greater intervention effect in children from higher social risk environments. In contrast, the impact of early intervention on parental depressive symptoms was greater for parents of lower social risk than for those of higher social risk.Interpretation: Effects of early intervention on outcomes for children born preterm and their caregivers varied according to family social risk. Family social risk should be considered when implementing early intervention programmes for children born preterm and their families.What This Paper Adds: Intervention is associated with better early cognitive functioning for children in higher social risk families. Positive effects of intervention for the high risk group were not sustained at school-age. Intervention has a greater effect on primary caregiver mental health in the lower social risk group compared with higher social risk. [ABSTRACT FROM AUTHOR]

Aim: To test the hypothesis that higher seizure burden in Dravet syndrome is associated with increased comorbidities and lower quality of life (QoL) in a large cohort of patients with Dravet syndrome and their caregivers in Europe.Method: An extensive survey of caregivers of patients with Dravet syndrome on experiences of diagnosis, seizure burden, management, social and financial impact, and health services use was administered online in 10 languages.Results: The survey received 584 unique responses from caregivers of paediatric (83%) and adult (17%) patients with Dravet syndrome (aged <1-48y). Despite broadly following current treatment guidance, less than 10% of patients were seizure free in the previous 3 months. Nearly all (99.6%) patients aged 5 years or older experienced at least one or more motor, speech, learning, or behavioural impairment. High seizure frequency was related to more reports of emergency treatment, comorbidities, and a lower QoL (as measured by the standardized instrument EQ-5D-5L). If not diagnosed at the first instance, the majority (83%) of adults, but less than 20% of 6- to 11-year-olds were diagnosed after 4 or more years.Interpretation: Patients with Dravet syndrome with the highest current seizure frequency suffer from more comorbidities and have a lower QoL. Therefore, more effective antiepileptic treatments are needed.What This Paper Adds: The survey captured about 15% of all patients with Dravet syndrome in Europe. Less than 10% of patients had current seizure freedom. Patients with a high current seizure burden have more comorbidities and lower quality of life. [ABSTRACT FROM AUTHOR]

Aim: We investigated the long-term efficacy and safety of multilevel surgery (MLS) in ambulatory children with bilateral spastic cerebral palsy (CP).Method: Two hundred and thirty-one children were evaluated at short term (1.1y, SD 0.4) and long term (9.1y, SD 3.0) follow-up using clinical examination and gait analysis. MLS was investigated by studying changes in the Gait Profile Score (GPS) referenced to the minimally important clinical difference.Results: Ambulatory children aged 10 years and 7 months (SD 2y 11mo) at MLS in Gross Motor Function Classification System levels I (19), II (144), and III (68) showed a decrease (improvement) in preoperative GPS from 16.3° (SD 4.8) to 11.3° (SD 3.2) at short-term follow-up, an improvement of 5°. At long-term follow-up, GPS was maintained at 11.4° (SD 3.1). Overall, 177 (76.6%) children maintained their improvement in GPS after 9 years.Interpretation: Multilevel surgery is a safe and effective surgical intervention, which leads to a significant improvement in gait kinematics in children with bilateral spastic CP. This study improves our understanding of MLS in the long term and will help to inform families and children when planning for MLS.What This Paper Adds: Largest study of multilevel surgery (MLS) for children with bilateral spastic cerebral palsy, with longest follow-up. MLS resulted in significant long-term improvements in gait function. Minor adverse events were common, while events requiring intervention were uncommon (4% of children). Thirty-nine per cent of children required additional surgery during follow-up. 'Single-event multilevel surgery' was changed to the more realistic term 'multilevel surgery'. [ABSTRACT FROM AUTHOR]

Purpose The aim of this study was to compare the Mother-Infant/Toddler Feeding Scale (MITFS) and the Nursing Child Assessment Feeding Scale (NCAFS). Specific questions were as follows: (1) Are there differences between the results of the MITFS and the NCAFS tools in terms of rating infant feeding interactions? And (2) does one tool provide a more detailed, nuanced overview of the quality of feeding interactions than the other? Design and methods This comparative descriptive study is a secondary analysis of a study evaluating a massage intervention for infants with symptoms of gastroesophageal reflux disease (GERD). A feeding observation for each dyad was scored using both the NCAFS and MITFS. Infants were 6-18 weeks of age. Results There were moderate correlations ( r = 0.3-0.8) between 11 of 20 possible NCAFS and MITFS mother-related subscales, and between 3 of 10 possible NCAFS and the MITFS infant-related subscales. A total of 19 dyads (83%) had one or more NCAFS subscale scores that were at least 1 standard deviation (SD) below normative scores, and 21 dyads (91%) had one or more MITFS subscale T-scores that deviated 1 SD from the normative T-scores. Agreement between the two instruments on scores deviating from the norm was 78%. Practice implications Both instruments discussed in this paper are standardized ways of assessing an infant feeding. Use of a standardized feeding tool is an objective way to evaluate feedings and can potentially identify specific areas of concern to guide an individualized intervention for a mother-infant dyad. Thus, nurses and other health professionals working with mothers and babies, whether in the hospital, pediatrician offices, or clinics would benefit from learning how to use a standardized feeding tool. Use of a feeding tool also provides the opportunity for nurses to understand the basis of important qualities of a feeding interaction, and a way for nurses who regularly feed infants to self-evaluate their feeding methods. This may minimize less than optimal interactions and allow for optimal support for infants during feedings by mothers and nurses. [ABSTRACT FROM AUTHOR]

Aim: The aim of this study was to develop a descriptive and evaluative assessment of upper limb function for infants aged 3 to 12 months and to investigate its internal scale validity for use with infants at risk of unilateral cerebral palsy.Method: The concepts of the test items and scoring criteria were developed. Internal scale validity and aspects of reliability were investigated on the basis of 156 assessments of infants at 3 to 12 months corrected age (mean 7.2mo, SD 2.5) with signs of asymmetric hand use. Rasch measurement model analysis and non-parametric statistics were used.Results: The new test, the Hand Assessment for Infants (HAI), consists of 12 unimanual and five bimanual items, each scored on a 3-point rating scale. It demonstrated a unidimensional construct and good fit to the Rasch model requirements. The excellent person reliability enabled person separation to six significant ability strata. The HAI produced an interval-level measure of bilateral hand use as well as unimanual scores of each hand, allowing a quantification of possible asymmetry expressed as an asymmetry index.Interpretation: The HAI can be considered a valid assessment tool for measuring bilateral hand use and quantifying side difference between hands among infants at risk of developing unilateral cerebral palsy.What This Paper Adds: The Hand Assessment for Infants (HAI) measures the use of both hands and quantifies a possible asymmetry of hand use. HAI is valid for infants at 3 to 12 months corrected age at risk of unilateral cerebral palsy. [ABSTRACT FROM AUTHOR]

Aim: To characterize carpal tunnel syndrome (CTS) in patients with mucopolysaccharidosis I (MPS I).Method: Data were included for patients with MPS I who had either nerve conduction examination that included a diagnosis of CTS or who had CTS release surgery. Although this represented a subset of patients with CTS in the MPS I Registry, the criteria were considered the most objective for data analysis.Results: As of March 2016, 994 patients were categorized with either severe (Hurler syndrome) or attenuated (Hurler-Scheie or Scheie syndromes) MPS I. Among these, 291 had a CTS diagnosis based on abnormal nerve conduction (n=54) or release surgery (n=237). Median ages (minimum, maximum) at first CTS diagnosis were 5 years 2 months (10mo, 16y 2mo) and 9y 11mo (1y 8mo, 44y 1mo) for patients with severe and attenuated MPS I respectively. Most patients had their first CTS diagnosis after MPS I diagnosis (94%) and treatment (hematopoietic stem cell transplant and/or enzyme replacement therapy) (74%). For 11% of patients with attenuated disease, CTS diagnosis preceded MPS I diagnosis by a mean of 7 years 6 months.Interpretation: CTS is a rare complication in pediatric patients and should alert medical care providers to the potential diagnosis of MPS I. Significant delays exist between diagnosis of CTS and MPS I for patients with attenuated disease.What This Paper Adds: There are significant delays in diagnosing carpal tunnel syndrome (CTS) in patients with mucopolysaccharidosis I (MPS I). Enzyme replacement therapy or hematopoietic stem cell transplant do not prevent the development of CTS. Testing for CTS in patients with MPS I is recommended to prevent irreparable damage. CTS in pediatric patients should alert physicians to potential diagnosis of MPS I. [ABSTRACT FROM AUTHOR]

Pharmacokinetic studies aim to study how a compound is absorbed, distributed, metabolised, and excreted. The concentration of the compound in the blood or plasma is measured at different time points after administration and pharmacokinetic parameters such as the area under the curve (AUC) or maximum concentration (Cmax ) are derived from the resulting concentration time profile. In this paper, we want to compare different methods for collecting concentration measurements (traditional sampling versus microsampling) on the basis of these derived parameters. We adjust and evaluate an existing method for testing superiority of multiple derived parameters that accounts for model uncertainty. We subsequently extend the approach to allow testing for equivalence. We motivate the methods through an illustrative example and evaluate the performance using simulations. The extensions show promising results for application to the desired setting. [ABSTRACT FROM AUTHOR]