Stewart Rust

A New Parenting-Based Group Intervention for Young Anxious Children: Results of a Randomized Controlled Trial

OBJECTIVE Despite recent advances, there are still no interventions that have been developed for the specific treatment of young children who have anxiety disorders. This study examined the impact of a new, cognitive-behaviorally based parenting intervention on anxiety symptoms. METHOD Families of 74 anxious children (aged 9 years or less) took part in a randomized controlled trial, which compared the new 10-session, group-format intervention with a wait-list control condition. Outcome measures included blinded diagnostic interview and self-reports from parents and children. RESULTS Intention-to-treat analyses indicated that children whose parent(s) received the intervention were significantly less anxious at the end of the study than those in the control condition. Specifically, 57% of those receiving the new intervention were free of their primary disorder, compared with 15% in the control condition. Moreover, 32% of treated children were free of any anxiety diagnosis at the end of the treatment period, compared with 6% of those in the control group. Treatment gains were maintained at 12-month follow-up. CONCLUSIONS This new parenting-based intervention may represent an advance in the treatment of this previously neglected group.

A systematic review of psychosocial outcomes within 2 years of paediatric traumatic brain injury in a school-aged population

Abstract Primary objective: To systematically review the results of investigative research into psychosocial outcomes following paediatric traumatic brain injury (TBI) in children of school-age. Methods: Searches were conducted using PsycINFO, MEDLINE and Science Direct. Three hundred and ninety papers were identified, of which 17 met inclusion criteria. Citation searches uncovered 28 further studies. All 44 studies were appraised in terms of their methodological rigour (e.g. sample characteristics, measures utilized, control groups employed). Results: The papers reviewed described outcomes across domains of behavioural, emotional, adaptive and parent/family functioning. Studies describing behavioural functioning post-injury demonstrated least consistent results. Papers examining emotional, adaptive and parent/family outcomes found that those with head injury presented with poorer functioning than other paediatric populations. Injury severity was commonly identified as a significant predictor of outcome, as were several pre-injury psychosocial factors. Conclusions: Inconsistencies in results may have arisen due to the heterogeneous populations studied and methodologies employed. Research which examines the reliability of assessment measures is needed. Given the high prevalence of difficulties, psychosocial assessment is warranted in this population.

Cognitive endpoints for therapy development for neuronopathic mucopolysaccharidoses: Results of a consensus procedure

The design and conduct of clinical studies to evaluate the effects of novel therapies on central nervous system manifestations in children with neuronopathic mucopolysaccharidoses is challenging. Owing to the rarity of these disorders, multinational studies are often needed to recruit enough patients to provide meaningful data and statistical power. This can make the consistent collection of reliable data across study sites difficult. To address these challenges, an International MPS Consensus Conference for Cognitive Endpoints was convened to discuss approaches for evaluating cognitive and adaptive function in patients with mucopolysaccharidoses. The goal was to develop a consensus on best practice for the design and conduct of clinical studies investigating novel therapies for these conditions, with particular focus on the most appropriate outcome measures for cognitive function and adaptive behavior. The outcomes from the consensus panel discussion are reported here.

Developmental outcome post allogenic bone marrow transplant for Niemann Pick Type C2.

Niemann Pick Type C2 (NPC2) is a rare autosomal recessive disease caused by mutations in the NPC2 gene (OMIM 601015). Clinically, NPC2 presents in most cases in the neonatal period with inflammatory lung disease, which may lead to death in the first year. If patients survive the neonatal period, they may develop a severe neurological disease. Here we present the developmental and neurological follow up at 5 years of age of a child with NPC2 successfully treated with allogenic bone marrow transplantation (BMT) at the age of 16 months. A homozygous p.E20X sequence variation previously associated with a severe phenotype was identified. In contrast to the previously reported patients with the same mutations, our patient has no respiratory compromise and has made some developmental progress (especially gross motor), though is significantly delayed (particularly in speech and language). Haematopoietic stem cell transplantation (HSCT) could be considered for patients with this mutation as long as performed early in the course of the disease.

From Timid to Tiger: A Treatment Manual for Parenting the Anxious Child

DESCRIPTION This book is an essential manual for mental health professionals who work with young anxious children and their parents. Organised into a 10-session parenting-based course, the book provides parents with simple cognitive behavioural techniques for helping their children to manage their worries and fears. • The first manual designed specifically to help therapists take parents through a step-by-step approach to managing young anxious children • The manuals empirical focus is highly effective in treating anxiety disorders in children under the age of 10 • The provision of scripts throughout the book offer realistic illustrations of the techniques described • Stories and analogies included to explain the more complex concepts • Includes handouts which can be photocopied and useful additional materials

Recommendations on clinical trial design for treatment of Mucopolysaccharidosis Type III

BackgroundMucopolysaccharidosis type III is a progressive, neurodegenerative lysosomal storage disorder for which there is currently no effective therapy. Though numerous potential therapies are in development, there are several challenges to conducting clinical research in this area. We seek to make recommendations on the approach to clinical research in MPS III, including the selection of outcome measures and trial endpoints, in order to improve the quality and impact of research in this area.ResultsAn international workshop involving academic researchers, clinical experts and industry groups was held in June 2015, with presentations and discussions on disease pathophysiology, biomarkers, potential therapies and clinical outcome measures. A set of recommendations was subsequently prepared by a working group and reviewed by all delegates. We present a series of 11 recommendations regarding the conduct of clinical research, outcome measures and management of natural history data in Mucopolysaccharidosis type III.ConclusionsImproving the quality of clinical research in Mucopolysaccharidosis type III will require an open, collaborative and systematic approach between academic researchers, clinicians and industry. Natural history data should be published as soon as possible and ideally collated in a central repository. There should be agreement on outcome measures and instruments for evaluation of clinical outcomes to maximise the effectiveness of current and future clinical research.

Vineland adaptive behavior scales to identify neurodevelopmental problems in children with Congenital Hyperinsulinism (CHI)

BackgroundCongenital Hyperinsulinism (CHI) is a disease of severe hypoglycaemia caused by excess insulin secretion and associated with adverse neurodevelopment in a third of children. The Vineland Adaptive Behavior Scales Second Edition (VABS-II) is a parent report measure of adaptive functioning that could be used as a developmental screening tool in patients with CHI. We have investigated the performance of VABS-II as a screening tool to identify developmental delay in a relatively large cohort of children with CHI. VABS-II questionnaires testing communication, daily living skills, social skills, motor skills and behaviour domains were completed by parents of 64 children with CHI, presenting both in the early neonatal period (Early-CHI, n = 48) and later in infancy (Late-CHI, n = 16). Individual and adaptive composite (Total) domain scores were converted to standard deviation scores (SDS). VABS-II scores were tested for correlation with objective developmental assessment reported separately by developmental paediatricians, clinical and educational psychologists. VABS-II scores were also investigated for correlation with the timing of hypoglycaemia, gender and phenotype of CHI.ResultsMedian (range) total VABS-II SDS was low in CHI [-0.48 (-3.60, 4.00)] with scores < -2.0 SDS in 9 (12%) children. VABS-II Total scores correctly identified developmental delay diagnosed by objective assessment in the majority [odds ratio (OR) (95% confidence intervals, CI) 0.52 (0.38, 0.73), p < 0.001] with 95% specificity [area under curve (CI) 0.80 (0.68, 0.90), p < 0.001] for cut-off < -2.0 SDS, although with low sensitivity (26%). VABS-II Total scores were inversely correlated (adjusted R2 = 0.19, p = 0.001) with age at presentation (p = 0.024) and male gender (p = 0.036), males having lower scores than females in those with Late-CHI [-1.40 (-3.60, 0.87) v 0.20 (-1.07, 1.27), p = 0.014]. The presence of a genetic mutation representing severe CHI also predicted lower scores (R2 = 0.19, p = 0.039).ConclusionsThe parent report VABS-II is a reliable and specific tool to identify developmental delay in CHI patients. Male gender, later age at presentation and severity of disease are independent risk factors for lower VABS-II scores.

Autism Spectrum Disorder symptomatology in children with Mucopolysaccharide Disease Type III

Symptoms of autism spectrum disorder (ASD) are present in children with mucopolysaccharidosis type III (MPS III). Questionnaires measuring the two core domains of ASD (social responsiveness and repetitive behaviour) were completed by N = 17 parents of children with MPS III. Results indicated that scores consistent with ASD were reported by all parents on a measure of social responsiveness but lower scores were reported for repetitive behaviour. The findings provide support for the provision of additional interventions, focusing on social and communication difficulties, for children with MPS III across all phases of their disorder.