Autism screening at 18 months of age: a comparison of the Q-CHAT-10 and M-CHAT screeners.

Funder: Peterborough NHS Foundation TrustFunder: Collaboration for Leadership in Applied Health Research and Care - Greater ManchesterFunder: NIHR Cambridge Biomedical Research CentreBACKGROUND: Autism screening is recommended at 18- and 24-month pediatric well visits. The Modified Checklist for Autism in Toddlers-Revised (M-CHAT-R) authors recommend a follow-up interview (M-CHAT-R/F) when positive. M-CHAT-R/F may be less accurate for 18-month-olds than 24-month-olds and accuracy for identification prior to two years is not known in samples that include children screening negative. Since autism symptoms may emerge gradually, ordinally scoring items based on the full range of response options, such as in the 10-item version of the Quantitative Checklist for Autism in Toddlers (Q-CHAT-10), might better capture autism signs than the dichotomous (i.e., yes/no) items in M-CHAT-R or the pass/fail scoring of Q-CHAT-10 items. The aims of this study were to determine and compare the accuracy of the M-CHAT-R/F and the Q-CHAT-10 and to describe the accuracy of the ordinally scored Q-CHAT-10 (Q-CHAT-10-O) for predicting autism in a sample of children who were screened at 18 months. METHODS: This is a community pediatrics validation study with screen positive (n = 167) and age- and practice-matched screen negative children (n = 241) recruited for diagnostic evaluations completed prior to 2 years old. Clinical diagnosis of autism was based on results of in-person diagnostic autism evaluations by research reliable testers blind to screening results and using the Autism Diagnostic Observation Schedule-Second Edition (ADOS-2) Toddler Module and Mullen Scales of Early Learning (MSEL) per standard guidelines. RESULTS: While the M-CHAT-R/F had higher specificity and PPV compared to M-CHAT-R, Q-CHAT-10-O showed higher sensitivity than M-CHAT-R/F and Q-CHAT-10. LIMITATIONS: Many parents declined participation and the sample is over-represented by higher educated parents. Results cannot be extended to older ages. CONCLUSIONS: Limitations of the currently recommended two-stage M-CHAT-R/F at the 18-month visit include low sensitivity with minimal balancing benefit of improved PPV from the follow-up interview. Ordinal, rather than dichotomous, scoring of autism screening items appears to be beneficial at this age. The Q-CHAT-10-O with ordinal scoring shows advantages to M-CHAT-R/F with half the number of items, no requirement for a follow-up interview, and improved sensitivity. Yet, Q-CHAT-10-O sensitivity is less than M-CHAT-R (without follow-up) and specificity is less than the two-stage procedure. Such limitations are consistent with recognition that screening needs to recur beyond this age

Emerging Language Comprehension in Toddlers with Significant Developmental Delays

Language comprehension is critical to a wide variety of child outcomes, including academic success and emotional and social well-being. Effective intervention relies on valid, reliable language comprehension data to determine the intensity and techniques that are appropriate for an individual child. The present study investigated language comprehension in a sample of 113 toddlers with significant developmental delays using IRT methods. We found that the aggregate data adequately fit the Rasch model, though each measure also contained items with poor fit. Analyses of the correspondence between item difficulties and participant abilities generally supported the appropriateness of the measures for our sample, and indicated acceptable measurement precision for the majority of participants. Examination of the relative difficulty of items revealed patterns that were largely consistent with the literature on typically developing children, with a few exceptions. Investigation of individual items showing the highest proportions of change in our sample indicated that parent-report items of moderate difficulty were most likely to reflect language comprehension improvement. Our findings inform clinical practice by underscoring the strengths and limitations of currently available measures. They also inform future measure development by emphasizing the benefits of integrating IRT methods in order to maximize both measurement precision and testing efficiency. Finally, they add to knowledge about language comprehension development in atypical populations

Early Start DENVER Model: a Meta - Analysis

Each child with Autism Spectrum Disorder has different symptoms, skills and types of impairment or disorder with other children. This is why the word “spectrum” is included in this disorder. Eapen, Crncec, and Walter, 2013 claimed that there was an emerging evidence that early interventions gives the greatest capacity of child's development during their first years of life as “brain plasticity” are high during this period. With this, the only intervention program model for children as young as 18 months that has been validated in a randomized clinical trial is “Early Start Denver Model” (ESDM). This study aimed to determine the effectiveness of the outcome of “Early Start Denver Model” (ESDM) towards young children with Autism Spectrum Disorders. This study made use of meta-analysis method. In this study, the researcher utilized studies related to “Early Start Denver Model (ESDM)” which is published in a refereed journal which are all available online. There were five studies included which totals 149 children exposed to ESDM. To examine the “pooled effects” of ESDM in a variety of outcomes, a meta-analytic procedure was performed after the extraction of data of the concrete outcomes. Comprehensive Meta Analysis Version 3.3.070 was used to analyze the data. The effectiveness of the outcome of “Early Start Denver Model” towards young children with Autism Spectrum Disorders (ASD) highly depends on the intensity of intervention and the younger child age. This study would provide the basis in effectively implementing an early intervention to children with autism such as the “Early Start Denver Model” (ESDM) that would show great outcome effects to those children that has “Autism Spectrum Disorder”

Autism in regional and remote communities: Examining the effect of an autism spectrum disorder diagnosis on regional and remote families and innovative therapies

This PhD project aims to address therapy service access barriers for families of children with autism spectrum disorder living in regional and remote Western Australia. Therefore the feasibility, effectiveness and appropriateness of an information communication technology based intervention, the Therapy Outcomes By Your Playpad Application were examined. The findings provide partial support for the Therapy Outcomes By Your Playpad Application as a feasible, effective and appropriate intervention for families of children with autism spectrum disorder

Mathematical modeling: a valuable training aid for new medical devices

[EN] The paper deals with biomedical modeling in general and in particular how modeling techniques can be used to improve the training of clinical practitioners in the use of new medical devices. The Bioheat Equation is given as an example to show how, even though mathematical models are based on complex equations, the terms of the equations represent phenomena which can be intuitively understood. The paper also describes examples in which mathematical modeling was used to study the performance of medical devices, especially those related with radiofrequency ablation.[ES] Este artículo trata sobre la modelización biomédica en general y cómo las técnicas de modelización pueden ayudar para mejorar la práctica clínica en el uso de nuevos dispositivos médicos en particular. La ecuación “Bioheat” o de difusión del calor en tejido biológico se utiliza como ejemplo para mostrar como, incluso en el caso en que los modelos matemáticos estén basados en ecuaciones complejas, los términos de dichas ecuaciones representan fenómenos que pueden ser entendidos de forma intuitiva. El artículo también presenta ejemplos en los cuales el modelado matemático se usa para estudiar la adecuación de los dispositivos médicos, especialmente aquellos relacionados con la ablación por radiofrecuencia.Berjano, E. (2010). Mathematical modeling: a valuable training aid for new medical devices. Modelling in Science Education and Learning. 3:55-65. doi:10.4995/msel.2010.3112SWORD5565

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

Measuring Change in Social Communication Behaviors: Reliability, Validity, and Application

Purpose: The field of Autism Spectrum Disorder (ASD) intervention research is in need of treatment response measures that are sensitive to change and flexible enough to be used across studies. The Brief Observation of Social Communication Change (BOSCC) was developed to address this need. The purpose of this work is to examine the initial reliability and validity of the BOSCC in two samples of children with ASD. Method: In a sample of 56 children participating in ongoing early intervention, the primary objectives of Study 1 were to 1) determine items for inclusion in the BOSCC coding scheme, 2) explore the relationships among items using factor analysis, 3) assess inter-rater and test-retest reliability, and 4) explore change over time. Using a sample of school-age, minimally-verbal children, the primary objectives of Study 2 were to extend the results of Study 1 to a new sample to 1) assess BOSCC changes over time, 2) compare changes in BOSCC to clinician determinations of improvement, 3) examine the relationship between change in BOSCC scores with changes in baseline cognitive skills, adaptive functioning, and ASD severity, and 4) compare changes in BOSCC scores in children who did and did not change on other standard measures. Results: Study 1 revealed that the BOSCC has high to excellent inter-rater and test-retest reliability and shows convergent validity with measures of language and communication skills. The BOSCC Core total demonstrated statistically significant amounts of change over time while the ADOS Calibrated Severity Score over the same period did not. Results of Study 2 confirmed excellent inter-rater reliability but the BOSCC did not change significantly over time. Most children were identified by clinicians as improving in response to treatment. However, only 15% of children changed significantly on the BOSCC over 16 weeks of intervention. Limitations: Both studies had small samples of predominantly male, Caucasian children. When interpreting the results of these studies, it is important to consider the differences between samples, including the shorter time of treatment and more cognitively and language impaired children in Study 2. Conclusions: These studies are a first step in the development of a novel outcome measure for social-communication behaviors with applications to clinical trials and longitudinal studies. Future work should continue to explore the benefits and limitations of the BOSCC in larger independent samples

Neuro-Immune Abnormalities in Autism and Their Relationship with the Environment: A Variable Insult Model for Autism

Autism spectrum disorder (ASD) is a heterogeneous condition affecting an individual’s ability to communicate and socialize and often presents with repetitive movements or behaviors. It tends to be severe with less than 10% achieving independent living with a marked variation in the progression of the condition. To date, the literature supports a multifactorial model with the largest, most detailed twin study demonstrating strong environmental contribution to the development of the condition. Here, we present a brief review of the neurological, immunological, and autonomic abnormalities in ASD focusing on the causative roles of environmental agents and abnormal gut microbiota. We present a working hypothesis attempting to bring together the influence of environment on the abnormal neurological, immunological, and neuroimmunological functions and we explain in brief how such pathophysiology can lead to, and/or exacerbate ASD symptomatology. At present, there is a lack of consistent findings relating to the neurobiology of autism. Whilst we postulate such variable findings may reflect the marked heterogeneity in clinical presentation and as such the variable findings may be of pathophysiological relevance, more research into the neurobiology of autism is necessary before establishing a working hypothesis. Both the literature review and hypothesis presented here explore possible neurobiological explanations with an emphasis of environmental etiologies and are presented with this bias

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

AbstractThe 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