Search

Recent advances in rare disease research are accelerated by the work of consortia that have been supported by the National Institutes of Health. Development of such consortia rely on multidisciplinary relationships and engagement with patient advocacy groups, as well as the National Institutes of Health and industry and academic partners. In this rostrum we present the development of such a process that focuses on eosinophilic gastrointestinal diseases. Principal investigators, patient advocacy groups, research assistants, and trainees work together to perform natural history studies that promote clinical trial readiness tools, conduct clinical trials, train a new generation of investigators, and perform innovative pilot studies.

IntroductionThe aim of this feasibility and proof-of-concept study was to examine the use of a novel wearable device for automatic food intake detection to capture the full range of free-living eating environments of adults with overweight and obesity. In this paper, we document eating environments of individuals that have not been thoroughly described previously in nutrition software as current practices rely on participant self-report and methods with limited eating environment options.MethodsData from 25 participants and 116 total days (7 men, 18 women, Mage = 44 ± 12 years, BMI 34.3 ± 5.2 kg/mm2), who wore the passive capture device for at least 7 consecutive days (≥12h waking hours/d) were analyzed. Data were analyzed at the participant level and stratified amongst meal type into breakfast, lunch, dinner, and snack categories. Out of 116 days, 68.1% included breakfast, 71.5% included lunch, 82.8% included dinner, and 86.2% included at least one snack.ResultsThe most prevalent eating environment among all eating occasions was at home and with one or more screens in use (breakfast: 48.1%, lunch: 42.2%, dinner: 50%, and snacks: 55%), eating alone (breakfast: 75.9%, lunch: 89.2%, dinner: 74.3%, snacks: 74.3%), in the dining room (breakfast: 36.7%, lunch: 30.1%, dinner: 45.8%) or living room (snacks: 28.0%), and in multiple locations (breakfast: 44.3%, lunch: 28.8%, dinner: 44.8%, snacks: 41.3%).DiscussionResults suggest a passive capture device can provide accurate detection of food intake in multiple eating environments. To our knowledge, this is the first study to classify eating occasions in multiple eating environments and may be a useful tool for future behavioral research studies to accurately codify eating environments.

We aimed to determine whether results of our prior randomized control trial [RCT; NCT02301195, (1)] of Therapeutic Horseback Riding (THR) for children and adolescents with autism spectrum disorder (ASD) could be replicated at a different riding center and if treatment effects also included differences in the expression of associations between problem behavior and the activity of the hypothalamic-pituitary-adrenal (HPA) axis. Participants with ASD (N = 16) ages 6-16 years were randomized by nonverbal intelligence quotient to either a 10-week THR group (n = 8) or no horse interaction barn activity (BA) control group (n = 8). Outcome measures were a standard speech-language sample and caregiver-report of aberrant and social behaviors. Participants' saliva was sampled weekly at a consistent afternoon time immediately pre- and 20 min' post-condition (later assayed for cortisol). Intent-to-treat analysis revealed that compared to controls, THR participants had significant improvements in hyperactivity, and social awareness, and significant improvements at the 0.1 significance level in irritability and social communication behaviors. There were no significant improvements in number of words or new words spoken during the standard language sample. Linear mixed effects model analysis indicated that greater weekly pre-lesson irritability levels were associated with smaller post-lesson reduction in salivary cortisol levels, and greater weekly pre-lesson hyperactivity levels were associated with smaller cortisol reduction in the THR group, but not in the BA control group. The findings represent a partial replication of prior results (1), extend prior observations to include THR effects on biobehavioral relationships and suggest that cortisol could be a target mediator for THR effects on irritability and hyperactivity behaviors in youth with ASD. Clinical Trial Registration: Trial of Therapeutic Horseback Riding in Children and Adolescents with Autism Spectrum Disorder; http://clinicaltrials.gov, identifier: NCT02301195.

There is preliminary research suggesting that animal-assisted activities can improve social interactions of children with autism spectrum disorder. This pilot study sought to investigate the benefits of animal-assisted activities with dogs and psychiatrically hospitalized youth with autism spectrum disorder. Participants were recruited from a specialized inpatient psychiatric hospital unit for youth with autism spectrum disorder and other developmental disabilities. Utilizing a crossover design, participants served as their own control by engaging in two 10-min conditions: an experimental dog and handler interaction (animal-assisted activities) and a novel toy and handler control (control). Of the 142 youth aged 6-8 years screened for participation, 47 completed both conditions. Participants' behavioral data were captured via video and coded using the Observation of Human-Animal Interaction for Research, a tool specifically developed to capture human behavioral interactions in the presence of animals. Overall, social-communication behaviors significantly improved in the animal-assisted activities experimental condition compared to the control condition (p = 0.0001). Specifically, participants in the animal-assisted activities experimental condition displayed more positive emotional facial expressions (p ? 0.0001), talking (p = 0.0408), use of gestures (p = 0.032), and looking at both adults and peers (p ? 0.0001). In addition, a higher frequency of constant motion (p = 0.003) was observed in the animal-assisted activities experimental condition. Results suggest that animal-assisted activities with a dog may promote social-communication behaviors in psychiatrically hospitalized youth with autism spectrum disorder. Given the fact that social and communication behaviors can facilitate treatment engagement for this population, we recommend future studies examine how such improvements can positively affect the psychiatric treatment of this population.

BACKGROUND: Patient-reported outcome metrics for eosinophilic esophagitis (EoE) have been developed and validated but not used in a multicenter pediatric population or systematically aligned with histology. OBJECTIVE: We sought to understand (1) the potential of caregiver report to predict patient self-reported symptoms and (2) the correlation of patient-reported outcome domains with histology. METHODS: Patients with EoE (n = 310) and their parents participating in the Consortium of Gastrointestinal Eosinophilic Disease Researchers (CEGIR) observational clinical trial were queried for baseline patient symptoms and quality of life (QOL) by using the Pediatric Eosinophilic Esophagitis Symptom Score, version 2 (PEESSv2.0), and the Pediatric QOL EoE module (PedsQL-EoE), and biopsy specimens were analyzed by using the EoE Histology Scoring System. RESULTS: PEESSv2.0 parental and child reports aligned across all domains (r = 0.68-0.73, P

BACKGROUND:Patient-reported outcome metrics for eosinophilic esophagitis (EoE) have been developed and validated but not used in a multicenter pediatric population or systematically aligned with histology. OBJECTIVE:We sought to understand (1) the potential of caregiver report to predict patient self-reported symptoms and (2) the correlation of patient-reported outcome domains with histology. METHODS:Patients with EoE (n = 310) and their parents participating in the Consortium of Gastrointestinal Eosinophilic Disease Researchers (CEGIR) observational clinical trial were queried for baseline patient symptoms and quality of life (QOL) by using the Pediatric Eosinophilic Esophagitis Symptom Score, version 2 (PEESSv2.0), and the Pediatric QOL EoE module (PedsQL-EoE), and biopsy specimens were analyzed by using the EoE Histology Scoring System. RESULTS:PEESSv2.0 parental and child reports aligned across all domains (r = 0.68-0.73, P

We aimed to determine whether results of our prior randomized control trial [RCT; NCT02301195, (1)] of Therapeutic Horseback Riding (THR) for children and adolescents with autism spectrum disorder (ASD) could be replicated at a different riding center and if treatment effects also included differences in the expression of associations between problem behavior and the activity of the hypothalamic-pituitary-adrenal (HPA) axis. Participants with ASD (N = 16) ages 6-16 years were randomized by nonverbal intelligence quotient to either a 10-week THR group (n = 8) or no horse interaction barn activity (BA) control group (n = 8). Outcome measures were a standard speech-language sample and caregiver-report of aberrant and social behaviors. Participants' saliva was sampled weekly at a consistent afternoon time immediately pre- and 20 min' post-condition (later assayed for cortisol). Intent-to-treat analysis revealed that compared to controls, THR participants had significant improvements in hyperactivity, and social awareness, and significant improvements at the 0.1 significance level in irritability and social communication behaviors. There were no significant improvements in number of words or new words spoken during the standard language sample. Linear mixed effects model analysis indicated that greater weekly pre-lesson irritability levels were associated with smaller post-lesson reduction in salivary cortisol levels, and greater weekly pre-lesson hyperactivity levels were associated with smaller cortisol reduction in the THR group, but not in the BA control group. The findings represent a partial replication of prior results (1), extend prior observations to include THR effects on biobehavioral relationships and suggest that cortisol could be a target mediator for THR effects on irritability and hyperactivity behaviors in youth with ASD. Clinical Trial Registration: Trial of Therapeutic Horseback Riding in Children and Adolescents with Autism Spectrum Disorder; http://clinicaltrials.gov, identifier: NCT02301195.

The objective of this study was to examine how behavioral manifestations of trauma due to abuse are expressed in youth with autism spectrum disorder (ASD). Analysis of covariance (ANCOVA) compared outcomes between patients with a caregiver reported history of abuse and those without. Findings indicate that patients with ASD and reported abuse (i.e. physical, sexual, and/or emotional) have more intrusive thoughts, distressing memories, loss of interest, irritability, and lethargy than those without reported maltreatment. Those with clinical diagnoses of posttraumatic stress disorder (PTSD) had more severe and externalized symptoms than those with reported abuse not diagnosed with PTSD. Results emphasize the need for trauma screening measures to guide evidence-based treatments for children with ASD.

Aim: To examine the impact of participation in an adaptive ski and snowboard program on Health-Related Quality of Life (HRQL). Methods: Participants completed the KIDSCREEN-52 HRQL questionnaire prior to and upon completion of a winter sport season. The questionnaire was distributed for five seasons. Participants who completed three or more years were further analyzed as a long-term cohort. Results: Seventy-six athletes met inclusion criteria. After one year, athletes demonstrated significant improvement in physical well-being and bullying; guardians reported improvement in six of the 10 HRQL domains. Twenty-nine athletes met criteria for the long-term cohort, reporting significant improvements in financial resources, peers and social support, and bullying, but a significant decrease in self-perception. Parents reported improvements in financial resources and bullying. Conclusions: Perceived changes in HRQL of children with disabilities are evident after one season of participation, and guardians are more likely to report significant improvements than child-athletes. Keywords: Adaptive recreation, adaptive sports, cerebral palsy, disabilities, quality of life, skiing, Introduction The positive physical, psychological, and social benefits of sports participation in child-athletes are well documented. Despite demonstrated benefits of physical activity, studies have found that children with cerebral palsy [...]

The objective of this study was to examine how behavioral manifestations of trauma due to abuse are expressed in youth with autism spectrum disorder (ASD). Analysis of covariance (ANCOVA) compared outcomes between patients with a caregiver reported history of abuse and those without. Findings indicate that patients with ASD and reported abuse (i.e. physical, sexual, and/or emotional) have more intrusive thoughts, distressing memories, loss of interest, irritability, and lethargy than those without reported maltreatment. Those with clinical diagnoses of posttraumatic stress disorder (PTSD) had more severe and externalized symptoms than those with reported abuse not diagnosed with PTSD. Results emphasize the need for trauma screening measures to guide evidence-based treatments for children with ASD., Author(s): Jamie Brenner [sup.1] [sup.4] , Zhaoxing Pan [sup.1] , Carla Mazefsky [sup.2] , Kahsi A. Smith [sup.3] , Robin Gabriels [sup.1] Author Affiliations: (Aff1) 0000 0001 0703 675X, grid.430503.1, [...]