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BACKGROUND Care for children and youth with special healthcare needs (CYSHCN) relied on a network of providers who work to address the medical, behavioral, developmental, educational, family support, social, and economic needs of the child and family. Family-directed, manually created visual depictions of care team composition (i.e. care mapping) and detailed notetaking curated by caregivers (e.g. care binders) have been shown to enhance care coordination (CC) for families of CYSHCN, but are difficult to implement in clinical settings due to lack of integration with electronic health records (EHR) and limited visibility of family generated insights for care providers. Digital health tools are a promising solution to address this. Caremap is an EHR-integrated digital personal health record mobile application designed to integrate the benefits of care mapping and care binders. Currently, there is sparse literature describing end-user participation in the co-design of digital health tools that support care coordination for families of CYSHCN. In this paper, we describe a project that evaluated app usability and proof-of-concept through end-user simulation. OBJECTIVE The goal of this study was to conduct proof-of-concept of the Caremap app to coordinate care for CYSHCN. Specific aims include 1) to engage end-users in app co-design via app simulation; 2) to evaluate the useability of the app using validated measures; and 3) to explore user perspectives on how to make further improvements to the app using qualitative and quantitative data collection. METHODS In partnership with Family Voices, a national advocacy group for families of CYSHCN, caregivers of CYSHCN were recruited and underwent a virtual simulation exercise using Caremap to coordinate care for a simulated case of a child with complex medical and behavioral needs. Caregivers completed a post-simulation questionnaire adapted from two validated surveys: the Pediatric Integrated Care Survey (PICS) and the User Version of the Mobile Application Rating Scale (uMARS). In addition, a key informant interview was conducted with a liaison to Spanish-speaking families of CYSHCN regarding app accessibility for non-English speaking users. RESULTS A Caremap simulation was successfully developed in partnership with families of CYSHCN. 38 families recruited from 19 different states participated in the simulation exercise and completed the survey. Average rating for the adapted PICS survey was 4.1/5 (range: 1-5), and average rating for the adapted uMARS survey was 4/5(range: 1-5). The highest rated app feature was the ability to track progress toward short-term, patient/ family defined care goals. CONCLUSIONS Virtual simulation successfully facilitated end-user engagement and feedback on the usability and functionality of a digital health care coordination app for families of CYSHCN. Families who completed simulation with Caremap rated the app highly across several domains for CC. Simulation study results also elucidated key areas for improvement that translated to actionable next steps in application development.

BackgroundFecal Microbiota Transplant (FMT) has proven effective in treating recurrentClostridioides difficileinfection (rCDI) and has shown some success in treating inflammatory bowel diseases (IBD). There is emerging evidence that host engraftment of donor taxa is a tenet of successful FMT. However, there is little known regarding predictors of engraftment. We undertook a double-blind, randomized, placebo-controlled pilot study to characterize the response to FMT in children and young adults with mild to moderate active Crohn’s disease (CD) and ulcerative colitis (UC).ResultsSubjects with CD or UC were randomized to receive antibiotics and weekly FMT or placebo in addition to baseline medications. The treatment arm received seven days of antibiotics followed by FMT enema and then capsules weekly for seven weeks. We enrolled four subjects with CD and 11 with UC, ages 14-29 years. Due to weekly stool sampling, we were able to create a time series of alpha diversity, beta diversity and engraftment as they related to clinical response. Subjects exhibited a wide range of microbial diversity and donor engraftment as FMT progressed. Specifically, engraftment ranged from 26% to 90% at week 2 and 3% to 92% at two months. Consistent with the current literature, increases over time of both alpha diversity (p< 0.05) and donor engraftment (p< 0.05) correlated with improved clinical response. Additionally, our weekly time series enabled an investigation into the clinical and microbial correlates of engraftment at various time points. We discovered that the post-antibiotic but pre-FMT time point, often overlooked in FMT trials, was rich in microbial correlates of eventual engraftment. Greater residual alpha diversity after antibiotic treatment was positively correlated with engraftment and subsequent clinical response. Interestingly, a transient rise in the relative abundance of Lactobacillus was also positively correlated with engraftment, a finding that we recapitulated with our analysis of another FMT trial with publicly available weekly sequencing data.ConclusionsWe found that higher residual alpha diversity and Lactobacillus blooms after antibiotic treatment correlated with improved engraftment and clinical response to FMT. Future studies should closely examine the host microbial communities pre-FMT and the impact of antibiotic preconditioning on engraftment and response.

Background Children with inflammatory bowel disease [IBD] are disproportionally affected by recurrent Clostridioides difficile infection [rCDI]. Although faecal microbiota transplantation [FMT] has been used with good efficacy in adults with IBD, little is known about outcomes associated with FMT in paediatric IBD. Methods We performed a retrospective review of FMT at 20 paediatric centres in the USA from March 2012 to March 2020. Children with and without IBD were compared with determined differences in the efficacy of FMT for rCDI. In addition, children with IBD with and without a successful outcome were compared with determined predictors of success. Safety data and IBD-specific outcomes were obtained. Results A total of 396 paediatric patients, including 148 with IBD, were included. Children with IBD were no less likely to have a successful first FMT then the non-IBD affected cohort [76% vs 81%, p = 0.17]. Among children with IBD, patients were more likely to have a successful FMT if they received FMT with fresh stool [p = 0.03], were without diarrhoea prior to FMT [p = 0.03], or had a shorter time from rCDI diagnosis until FMT [p = 0.04]. Children with a failed FMT were more likely to have clinically active IBD post-FMT [p = 0.002] and 19 [13%] patients had an IBD-related hospitalisation in the 3-month follow-up. Conclusions Based on the findings from this large US multicentre cohort, the efficacy of FMT for the treatment of rCDI did not differ in children with IBD. Failed FMT among children with IBD was possibly related to the presence of clinically active IBD.

Digital therapeutics is a newly described concept in healthcare which is proposed to change patient behavior and treat medical conditions using a variety of digital technologies. However, the term is rarely defined with criteria that make it distinct from simply digitizedversions of traditional therapeutics. Our objective is to describe a more valuable characteristic of digital therapeutics, which is distinct from traditional medicine or therapy: that is, the utilization of artificial intelligence and machine learning systems to monitor and predict individual patient symptom data in an adaptive clinical feedback loop via digital biomarkers to provide a precision medicine approach to healthcare. Artificial intelligence platforms can learn and predict effective interventions for individuals using a multitude of personal variables to provide a customized and more tailored therapy regimen. Digital therapeutics coupled with artificial intelligence and machine learning also allows more effective clinical observations and management at the population level for various health conditions and cohorts. This vital differentiation of digital therapeutics compared to other forms of therapeutics enables a more personalized form of healthcare that actively adapts to patients’ individual clinical needs, goals, and lifestyles. Importantly, these characteristics are what needs to be emphasized to patients, physicians, and policy makers to advance the entire field of digital healthcare.

The results of medical procedures can often be difficult to translate into comprehensible and engaging information for patients. This randomized controlled trial evaluated the satisfaction and perceived value of a technology, called HealthVoyager, which creates a personalized virtual reality (VR) experience of a patient's endoscopy or colonoscopy findings in comparison to the standard practice (ie, reviewing printed reports). The platform allows gastroenterologists to create a customized VR patient report to help translate medical knowledge and procedural information to the patient. Forty-one patients (17 HealthVoyager [test]; 24 standard practice [control]) completed a self-report survey assessing their experience for receiving medical information. Results demonstrated that patients were significantly more satisfied in learning about their gastrointestinal condition and procedural results using HealthVoyager rather than with the standard of care. These results have implications for improving the knowledge translation of medical findings between healthcare providers and patients in various disease states and patient populations.

Patients are typically debriefed by their healthcare provider after any medical procedure or surgery to discuss their findings and any next steps involving medication or treatment instructions. However, without any medical or scientific background knowledge, it can feel overwhelming and esoteric for a patient to listen to a physician describe a complex operation. Instead, providing patients with engaging visuals and a virtual reality (VR) simulation of their individual clinical findings could lead to more effective transfer of medical knowledge and comprehension of treatment information. A newly developed VR technology is described, called HealthVoyager, which is designed to help facilitate this knowledge transfer between physicians and patients. The platform represents a customizable, VR software system utilizing a smartphone or tablet computer to portray personalized surgical or procedural findings as well as representations of normal anatomy. The use of such technology for eliciting medical understanding and patient satisfaction can have many practical and clinical applications for a variety of disease states and patient populations.

Background & Aims Fecal microbiota transplantation (FMT) is commonly used to treat Clostridium difficile infection (CDI). CDI is an increasing cause of diarrheal illness in pediatric patients, but the effects of FMT have not been well studied in children. We performed a multi-center retrospective cohort study of pediatric and young adult patients to evaluate the efficacy, safety, and factors associated with a successful FMT for the treatment of CDI. Methods We performed a retrospective study of 372 patients, 11 months to 23 years old, who underwent FMT at 18 pediatric centers, from February 1, 2004, to February 28, 2017; 2-month outcome data were available from 335 patients. Successful FMT was defined as no recurrence of CDI in the 2 months following FMT. We performed stepwise logistic regression to identify factors associated with successful FMT. Results Of 335 patients who underwent FMT and were followed for 2 months or more, 271 (81%) had a successful outcome following a single FMT and 86.6% had a successful outcome following a first or repeated FMT. Patients who received FMT with fresh donor stool (odds ratio [OR], 2.66; 95% CI, 1.39–5.08), underwent FMT via colonoscopy (OR, 2.41; 95% CI, 1.26–4.61), did not have a feeding tube (OR, 2.08; 95% CI, 1.05–4.11), or had 1 less episode of CDI before FMT (OR, 1.20; 95% CI, 1.04–1.39) had increased odds for successful FMT. Seventeen patients (4.7%) had a severe adverse event during the 3-month follow-up period, including 10 hospitalizations. Conclusions Based on the findings from a large multi-center retrospective cohort, FMT is effective and safe for the treatment of CDI in children and young adults. Further studies are required to optimize the timing and method of FMT for pediatric patients—factors associated with success differ from those of adult patients.

Inflammatory bowel disease (IBD) is a chronic inflammatory disease of the gastrointestinal tract, likely caused by an aberrant immune response to the microbiota and other environmental factors in genetically susceptible individuals.1,2 Oral mucosal inflammation is commonly described in patients with IBD, particularly Crohn’s disease (CD). Oral pathology has a reported prevalence of 0.5% to 80% in CD.3–8 When studied prospectively in children in collaboration with a dentist, 42% of new diagnoses of CD had oral manifestations.7 Symptoms can span from mild and nonspecific inflammation such as minor aphthous lesions, mucogingivitis, and angular cheilitis to more specific findings, such as mucosal tags, cobblestoning, deep linear ulcerations, and more severe granulomatous swelling isolated to the labia and face known as orofacial granulomatosis (OFG).3,5–7,9 Lesions of the oral mucosa may occur years before the onset of intestinal symptoms, particularly in the pediatric population.6 Specific oral mucosal findings are common in children and in 75%–100% of cases, contain disease-defining, histologic evidence of noncaseating granulomas.3,7,9 Data from animal models suggests that the microbiome is a critical factor in the pathogenesis of IBD in knockout mice that are at risk for the disease. Preliminary studies in humans have found differences in the intestinal microbial populations of IBD patients when compared with healthy controls.2,10–12 However, such studies have largely focused on the lower gastrointestinal tract. The oral cavity provides an easily accessed mucosal surface that may potentially yield valuable information about the microbiome and its interaction with the host. The mouth and its resident flora is a well-characterized microbiome with ≈600 predominant bacterial species, of which about 35% are unable to be grown in culture.13,14 This environment is unlike any other in the body, made up of diverse ecological niches including hard surfaces upon which complex biofilms flourish, anaerobic microclimates, and rapidly shedding mucosal surfaces. Given the difficulties in studying the oral microbiome using conventional, culture-based techniques, our laboratory has developed a molecular technique using a 16S rRNA-based microarray technology known as the Human Oral Microbe Identification Microarray (HOMIM). Previous investigations in our laboratory and others have implicated distinct changes in the oral microbiome in dental caries and periodontitis.13–15 Oral microbial alterations in systemic diseases have also been identified including atherosclerosis, preterm birth, and pancreatic cancer.13–21 In this case–control study, we demonstrate an overall decrease in the oral bacterial diversity of children with CD when compared with healthy controls. Furthermore, several key phyla were significantly reduced when compared with healthy subjects, as has been identified in studies of the intestinal microbiome.11,12,22–25

OBJECTIVES/SPECIFIC AIMS: Clostridium difficile infection (CDI) is the most common cause of antibiotic-associated diarrhea and an increasingly common infection in children in both hospital and community settings. Between 20% and 30% of pediatric patients will have a recurrence of symptoms in the days to weeks following an initial infection. Multiple recurrences have been successfully treated with fecal microbiota transplantation (FMT), though the body of evidence in pediatric patients is limited primarily to case reports and case series. The goal of our study was to better understand practices, success, and safety of FMT in children as well as identify risk factors associated with a failed FMT in our pediatric patients. METHODS/STUDY POPULATION: This multicenter retrospective analysis included 373 patients who underwent FMT for CDI between January 1, 2006 and January 1, 2017 from 18 pediatric centers. Demographics, baseline characteristics, FMT practices, C. difficile outcomes, and post-FMT complications were collected through chart abstraction. Successful FMT was defined as no recurrence of CDI within 60 days after FMT. Of the 373 patients in the cohort, 342 had known outcome data at two months post-FMT and were included in the primary analysis evaluating risk factors for recurrence post-FMT. An additional six patients who underwent FMT for refractory CDI were excluded from the primary analysis. Unadjusted analysis was performed using Wilcoxon rank-sum test, Pearson χ2 test, or Fisher exact test where appropriate. Stepwise logistic regression was utilized to determine independent predictors of success. RESULTS/ANTICIPATED RESULTS: The median age of included patients was 10 years (IQR; 3.0, 15.0) and 50% of patients were female. The majority of the cohort was White (89.0%). Comorbidities included 120 patients with inflammatory bowel disease (IBD) and 14 patients who had undergone a solid organ or stem cell transplantation. Of the 336 patients with known outcomes at two months, 272 (81%) had a successful outcome. In the 64 (19%) patients that did have a recurrence, 35 underwent repeat FMT which was successful in 20 of the 35 (57%). The overall success rate of FMT in preventing further episodes of CDI in the cohort with known outcome data was 87%. Unadjusted predictors of a primary FMT response are summarized. Based on stepwise logistic regression modeling, the use of fresh stool, FMT delivery via colonoscopy, the lack of a feeding tube, and a lower number of CDI episodes before undergoing FMT were independently associated with a successful outcome. There were 20 adverse events in the cohort assessed to be related to FMT, 6 of which were felt to be severe. There were no deaths assessed to be related to FMT in the cohort. DISCUSSION/SIGNIFICANCE OF IMPACT: The overall success of FMT in pediatric patients with recurrent or severe CDI is 81% after a single FMT. Children without a feeding tube, who receive an early FMT, FMT with fresh stool, or FMT via colonoscopy are less likely to have a recurrence of CDI in the 2 months following FMT. This is the first large study of FMT for CDI in a pediatric cohort. These findings, if confirmed by additional prospective studies, will support alterations in the practice of FMT in children.