Objectives: We sought to evaluate the safety and effectiveness of fecal microbiota transplantation (FMT) for recurrent Clostridioides difficile infection (CDI) in pediatric immunocompromised (IC) patients. Methods: This is a multicenter retrospective cohort study of pediatric participants who underwent FMT between March 2013 and April 2020 with 12-week follow-up. Pediatric patients were included if they met the definition of IC and were treated with FMT for an indication of recurrent CDI. We excluded patients over 18 years of age, those with incomplete records, insufficient follow-up, or not meeting study definition of IC. We also excluded those treated for Clostridioides difficile recurrence without meeting the study definition and those with inflammatory bowel disease without another immunocompromising condition. Results: Of 59 pediatric patients identified at 9 centers, there were 42 who met inclusion and no exclusion criteria. Included patients had a median age of 6.7 years. Etiology of IC included: solid organ transplantation (18, 43%), malignancy (12, 28%), primary immunodeficiency (10, 24%), or other chronic conditions (2, 5%). Success rate was 79% after first FMT and 86% after 1 or more FMT. There were no statistically significant differences in patient characteristics or procedural components when patients with a failed FMT were compared to those with a successful FMT. There were 15 total serious adverse events (SAEs) in 13 out of 42 (31%) patients that occurred during the follow-up period; 4 (9.5%) of which were likely treatment-related. There were no deaths or infections with multidrug resistant organisms during follow-up and all patients with a SAE fully recovered. Conclusions: The success rate of FMT for recurrent CDI in this pediatric IC cohort is high and mirrors data for IC adults and immunocompetent children. FMT-related SAEs do occur (9.5%) and highlight the need for careful consideration of risk and benefit.

Medical Marijuana in Pediatric Oncology: What Your Patients Are ThinkingDavid Brumbaugh, MD MSCS FAAPDepartment of Pediatrics, University of Colorado School of MedicineChildren’s Hospital Colorado13123 E. 16th Avenue, B290Aurora, CO 80045720-777-6426David.brumbaugh@childrenscolorado.orgWord count: 837Short Running Title: Medical Marijuana in Pediatric OncologyKeywords: marijuana, cannabis, complementary, pediatricsAbbreviations:MM Medical MarijuanaAYA Adolescent/young adultTHC TetrahydrocannabinolUse of complementary therapies occurs by up to 40-80% of pediatric oncology patients.1,2 Although cannabis is hardly new to the scene as a complementary treatment, legalization of both medical and recreational marijuana in many states has made these products ubiquitous. Use of and interest in medical marijuana (MM) by hospitalized pediatric patients appears to be concentrated in oncology units for the purpose of relieving symptoms such as nausea, pain, and anorexia.3 Yet clinical practitioners are still limited by the absence of high-quality research in MM to guide them. FDA approval of Epidiolex™ for specific pediatric epilepsy syndromes was an important research milestone, but marijuana remains classified as a Drug Enforcement Administration Schedule I drug, imposing an enormous barrier for clinical researchers.So how should pediatric oncology programs approach the topic of MM? In this issue of Pediatric Blood and Cancer , Ananth and colleagues used a qualitative research design to characterize patient and family perception of MM from a single institution in a state with permissive rules towards both medical and recreational marijuana. The authors interviewed both parents of younger children as well as adolescent/young adult (AYA) patients. In this cohort of pediatric oncology patients/families, although the proportion of subjects using MM was only 27%, a higher proportion were interested in MM, though with concerns about safety and effectiveness.In the Ananth study, patients/families were primarily using or interested in MM for treatment of nausea, anorexia, and anxiety. A concerning number of families in this study expressed a hope that MM would be effective as anti-cancer therapy. With the absence of high-quality randomized controlled trials of MM for treatment of cancer or treatment-related symptoms in children to inform practitioners on safety, dosing, and toxicity, there is no evidence base for pediatric oncologists to base a recommendation of MM. But should we be dissuading interested families from using MM products because they are harmful?Regarding safety of MM use, most parents and nearly all AYA patients minimized risks. When expressed, safety concerns of MM were perceived as less than with alcohol, illicit drugs, or other prescribed medications. This is not surprising, as perceived risk of marijuana in AYA has been steadily failing over last five years in the National Survey on Drug Use and Health.4 Understandably, in this study safety concerns focused on the potential for addiction, which would be associated with MM products enriched in Tetrahydrocannabinol (THC), the principal psychoactive cannabinoid found in cannabis. However, cannabis is a complex plant with over 70 distinct cannabinoids, and the MM industry now contains a broad range of different types of products that have varying concentrations of THC and consequent psychoactive potential. Carver and colleagues noted in their study of 19 hospitalized patients actively using MM, the majority were using products enriched in Cannabidiol with low concentration of THC. One limitation of the study by Ananth, et al. is that there was no attempt to classify the type of MM either being actively used or of interest to patients and parents, so the appropriateness of the concern for addiction cannot be assessed. Absent in patients/families’ perception of risk was any potential for interaction with chemotherapeutics or other prescribed medications. Since both THC and Cannabidiol can impact drug bioactivation and metabolism through multiple pathways, this potential safety concern should be known to the patient and treatment team.Despite the high level of interest in MM in their study population, the minority of patients/families had discussed MM with their oncologist and in those cases, the patient/family initiated the conversation. Absent advice from their treatment team, there was reliance on friends, family, and the internet for more information. A majority of parents desired the involvement of their physician team in any consideration of MM, and previous research has shown a high level of willingness amongst pediatric oncology providers to consider MM use by their patients, particularly when patients are seriously ill, so what stands in the way of talking about it? Providers are concerned about the absence of good research and are less knowledgeable in the domain of rules/laws regulating access to MM, particularly at the state level where there has been so much change over the last decade.5 These gaps may explain why we don’t bring up the topic of MM with our patients and families as often as they would like.Institutions may consider designating a multidisciplinary team of providers to develop greater experience in the legal and pharmacologic aspects of MM use. This team can support providers in the shared decision-making process around MM. In some institutions, it may make sense to house this expertise within the pediatric palliative care program supporting oncology patients.In summary, MM presently is an important part of the complementary therapeutic options available to pediatric oncology patients and their families, who desire the involvement of their provider team in decision making around MM. Despite the lack of evidence supporting use of MM, many patients are using MM products or may in the future, so we should invite this discussion as this will strengthen our therapeutic partnership.1. Fernandez CV, Stutzer CA, MacWilliam L, Fryer C. Alternative and complementary therapy use in pediatric oncology patients in British Columbia: prevalence and reasons for use and nonuse. J Clin Oncol. 1998;16(4):1279-1286.2. Kelly KM, Jacobson JS, Kennedy DD, Braudt SM, Mallick M, Weiner MA. Use of unconventional therapies by children with cancer at an urban medical center. J Pediatr Hematol Oncol. 2000;22(5):412-416.3. Carver AE, Jorgensen J, Barberio MW, Lomuscio CE, Brumbaugh D. A Pediatric Hospital Policy for Medical Marijuana Use. Pediatrics.2020;146(2).4. Administration SAaMHS. 2019 NSDUH Detailed Tables. samhsa.gov/data/report/2019-nsduh-detailed-tables. Published 2019. Accessed.5. Ananth P, Ma C, Al-Sayegh H, et al. Provider Perspectives on Use of Medical Marijuana in Children With Cancer. Pediatrics.2018;141(1).

In early 2020, hospitals across the US, including Children's Hospital Colorado (CHCO), began preparing for the regional impact of the severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2) pandemic via an Incident Command Team (ICT), a unified approach to hospital operations including the command, control, and coordination of incident management.1Farcas A. Ko J. Chan J. Malik S. Nono L. Chiampas G. Use of incident command system for disaster preparedness: a model for an emergency department COVID-19 response.Disaster Med Public Health Prep. 2020; 24: 1-6Crossref Scopus (27) Google Scholar, 2Persoff J. Ornoff D. Little C. The role of hospital medicine in emergency preparedness: a framework for hospitalist leadership in disaster preparedness, response, and recovery.J Hosp Med. 2018; 13: 713-718Crossref PubMed Scopus (19) Google Scholar, 3Rimstad R. Braut G.S. Literature review on medical incident command.Prehosp Disaster Med. 2015; 30: 205-215Crossref PubMed Scopus (27) Google Scholar The ICT at CHCO recognized the need for a scientific partnership for rapid uptake and synthesis of quickly accruing medical and public health literature to inform institutional policies and clinical care. Therefore, the ICT established a coronavirus disease 2019 (COVID-19) Scientific Advisory Council (SAC),4University of Colorado Anschutz Medical Campus, COVID-19 Scientific Advisory Council. Available at: https://medschool.cuanschutz.edu/pediatrics/sections/emergency-medicine/research-and-quality-improvement/covid-19-scientific-advisory-council. Accessed December 4, 2020.Google Scholar a multidisciplinary team of clinician–scientists from relevant CHCO clinical divisions and CHCO's Clinical Effectiveness (CE) team members.5Calhoun W.J. Wooten K. Bhavnani S. Anderson K.E. Freeman J. Brasier A.R. The CTSA as an exemplar framework for developing multidisciplinary translational teams.Clin Transl Sci. 2013; 6: 60-71Crossref PubMed Scopus (40) Google Scholar The SAC leadership developed an academic–hospital partnership and set of processes for identification of issues, priority-setting, rapid evidence assessment, as well as synthesis and dissemination of findings. To support other organization's efforts to create a similar academic–operational partnership, this paper describes the SAC's innovative infrastructure: the diverse team and the processes the SAC developed for rapid evidence synthesis, development of recommendations and guideline documents, and dissemination of findings: the Children's Colorado Rapid Evidence Analysis and Dissemination System (CCREADS). We used the Replicating Effective Programs (REP) framework to organize this report. The REP framework is an implementation science framework commonly used in quality improvement and health services research to guide selection, adaptation, and implementation of evidence-based interventions in real-world care settings.6Kwan B.M. Dickinson L.M. Glasgow R.E. Sajatovic M. Gritz M. Holtrop J.S. et al.The Invested in Diabetes Study Protocol: a cluster randomized pragmatic trial comparing standardized and patient-driven diabetes shared medical appointments.Trials. 2020; 21: 65Crossref PubMed Scopus (11) Google Scholar, 7Taylor L.J. Adkins S. Hoel A.W. Hauser J. Suwanabol P. Wood G. et al.Using implementation science to adapt a training program to assist surgeons with high-stakes communication.J Surg Educ. 2019; 76: 165-173Crossref PubMed Scopus (11) Google Scholar, 8Hastings S.N. Choate A.L. Mahanna E.P. Floegel T.A. Allen K.D. Van Houtven C.H. et al.Early mobility in the hospital: lessons learned from the STRIDE program.Geriatrics (Basel). 2018; 3PubMed Google Scholar The REP has 4 phases: the preconditions phase involves identification of the need and determining most effective strategies; preimplementation incorporates the development, orientation, and logistics of the program; the implementation phase encompasses the roll out and dissemination of the various components; and the maintenance and evolution phase involves organizational steps to sustain the intervention in the short and long term. Figure 1 (available at www.jpeds.com) summarizes application of each phase for development and implementation of CCREADS. In March 2020, the ICT called for rapid review of the emerging literature for high-priority questions, including screening and treatment of children with suspected or confirmed SARS-CoV-2 infection and protection of healthcare workers. To achieve this goal, CHCO assembled a team of clinician–scientists by issuing a hospital-wide request for volunteers representing all CHCO clinical and scientific sections. Soon after SAC co-leaders were selected, they met with ICT to establish a mission statement:The mission of the SAC is to advise CHCO incident command, faculty providers, and referring provider community on clinical aspects of the COVID-19 epidemic. The SAC will review existing literature and provide timely feedback on high priority questions to incident command. Based on input from its members, the SAC mission evolved from purely addressing the needs of the ICT to developing and updating clinical guidance for COVID-19 evaluation, management, and healthcare worker protection that were not already being addressed by other hospital teams. We considered potential barriers to implementation of SAC and CCREADS, including the rapid growth of the literature, including accelerated peer-reviewed publications from diverse international settings and preprints not yet peer-reviewed, required a rapid evidence assessment approach. Such an approach needed to evolve from traditional literature review standards, be nimble and efficient, and consider varied levels of evidence, settings, and study populations. Although rapidly evolving, the literature featured minimal evidence specific to pediatric populations, requiring the SAC to make recommendations at times in the absence of robust data, with the need to re-evaluate recommendations as new scientific literature emerged. In a novel pandemic context, the hospital's existing clinical pathways planning and development processes needed to be significantly accelerated to respond to demand from clinicians given limited local experience, the challenges associated with a rapidly-evolving body of literature, and the absence of published guidelines. The SAC aligned with the ICT goals of team member safety and an evidence-based approach to patient care. In addition, the SAC aligned with the CHCO mission to encompass research and education as well as clinical and operational needs; and with the academic mission of the University of Colorado Anschutz Medical Campus. The SAC leveraged Campus scientific resources including health sciences librarians, ethicists, and dissemination and implementation scientists. The SAC team initially assembled by the ICT was a multidisciplinary translational team, characterized by diversity in: (1) clinical setting—inpatient, critical care, emergency department, outpatient; (2) subspecialty—infectious disease, rheumatology, surgery, etc; (3) research expertise across the entire translational spectrum, from basic science to clinical science to public health; (4) training—including medical and surgical subspecialties, microbiology, epidemiology, respiratory therapy, pharmacology, health psychology, nursing, public health, communications, and CE; and (5) career stage—including senior and mid-career researchers as well as junior faculty, trainees and students. All who volunteered to be on the SAC were included. The SAC leadership worked with hospital and academic division leaders to fill gaps in representation by setting and specialty. The SAC leadership recognized that the ICT's evidentiary needs generally fell into 4 categories and established 4 corresponding working groups: Clinical Course and Epidemiology, Clinical Treatment, Diagnostic Testing, and Infection Control. Members of the SAC were asked to select 1 or more working groups and then to decide on a leader within each group. Within working groups, smaller groups with relevant expertise reviewed publications as they were released to identify those of highest possible quality and relevance to the clinical questions being addressed by the SAC. After the ICT presented the first set of urgent questions to the SAC, SAC leadership developed a process for clarifying the scientific issues, priority-setting, rapid evidence assessment, and reporting findings to the ICT (Figure 2). The SAC and ICT leadership recognized the need for an organized set of tools for the organization and dissemination of the work products, which overlapped with existing functions of the hospital's CE program. The CE team includes clinical and operational leaders, process improvement professionals, and data analysts working collaboratively with front-line caregivers to develop and oversee the Clinical Pathways program, lead large-scale quality improvement initiatives, and align clinical outcomes with emerging payment models. CE identified and assigned key team members to facilitate SAC efforts, including adaptation of existing templates and web-based development tools to improve effectiveness and efficiency overall. The CE and SAC teams developed an internal guidance to this set of processes and templates—the CCREADS—on a team web-based collaborative platform site. The SAC assessed existing resources available for addressing hospital needs and overcoming barriers. We then identified the most effective approach to rapid assessment of the literature and for adapting CE workflows to meet SAC goals. A process for rapid review relevant to COVID-19 was established (Table I; available at www.jpeds.com), and was reviewed and refined by a team of Dissemination and Implementation experts at the University of Colorado, with resources suggested by evidence review experts at the Agency for Healthcare Research and Quality. CCREADS includes intranet Web site resources, templates, project management tracking tools, and a standardized approval process flow—all documented on the team's Team collaboration software site and accessible to SAC team members. Because of the rapidly evolving evidence, the SAC developed a scheduled updating process that included timestamps on each guidance update. The SAC's rapid guidance development documents and processes were adapted from existing CE templates and workflows, including templates for internal communication, rapid evidence review, and recommendations/guidance documents. For clinical guidelines, we created AgileMD9AgileMD Integrated Clinical Pathways 2020.https://www.agilemd.comGoogle Scholar clinical pathway algorithms and embedded in these algorithms hyperlinks to longer-form SAC guidance statements. This approach allowed us to include the more rapidly updating details of clinical management in the hyper-linked statements with fewer changes to the base algorithm. The process we developed for the SAC and ICT to review guidance documents and select dissemination strategies included a process flow map and involved coordination with CHCO communications team members. To support other academicly-affiliated hospitals in implementing an SAC to support ICT decision-making, we developed a toolkit that includes SAC processes, spreadsheets, and document templates. The SAC toolkit is available for free download from the CHCO Web site (available at: childrenscolorado.org/ScientificAdvisoryToolkit). The entire SAC met between twice weekly to once monthly depending on the level of ICT response activation to review the status of guidance documents being developed, updates by the working groups, and incoming questions; to set team priorities; and to address working group needs for support from SAC leadership or the CE team. All SAC members reviewed each document, and feedback was used to improve the scientific rigor of each guidance document as well as the templates and processes used in their preparation. The SAC leadership met with the CE team after each meeting to further discuss implementation of the action items from each meeting and to prepare for the next ICT report-out. Report-out meetings with ICT were initially twice weekly and included a report-out template. By leveraging the collective expertise of a diverse group of experts, the SAC iteratively developed 20 clinical guidance resources to guide local care teams on screening, monitoring, treatment,10Agile MD. Available at: https://www.agilemd.com/meta/viewer/organizations/or_1016532754403149/modules/mo_114e15541dc02da0. Accessed December 4, 2020.Google Scholar and escalation of patients with suspected or confirmed SARS-CoV-2 or multisystem inflammatory syndrome in children (MIS-C), including 3 institutional clinical pathways (Table II; available at www.jpeds.com). Clinical pathways translate best-available evidence into an actionable format to help physicians make decisions in specific clinical circumstances.11Rotter T. Kinsman L. James E. Machotta A. Gothe H. Willis J. et al.Clinical pathways: effects on professional practice, patient outcomes, length of stay and hospital costs.Cochrane Database Syst Rev. 2010; : CD006632PubMed Google Scholar These pathways include dynamic algorithms integrated in the electronic health record and available at the point-of care, as well as externally available to peer organizations via the hospital's Web site. They required weekly reassessment and amendment incorporating emerging scientific literature, guidelines, and local institutional data. Updates to guidance documents were logged in a SAC tracking document. This document provided the input for each SAC team meeting agenda, and action items from each meeting were added to the tracking document. New incoming questions were adapted based on a template. As a result, a rapid-cycle improvement framework was applied using CE and research community expertise to identify, critically appraise and apply findings from key studies to the local context. The SAC infrastructure afforded opportunities to conduct expedited research and quality improvement to answer questions related to COVID-19 in pediatric populations. For example, despite an extensive rapid evidence assessment, there remained uncertainty regarding the reliability of preoperative SARS CoV-2 testing in the pediatric setting, which was affecting hospital policies regarding personal protective equipment, clearance wait times, and testing protocols. We formed a perioperative taskforce comprising members of the SAC and conducted a quality assurance initiative by comparing preoperative upper respiratory samples with intraoperative upper and lower respiratory tract samples. After obtaining necessary approvals, we were able to rapidly design a protocol, implement the collection of samples, and analyze data over a 2-month period (publication under review). The SAC's multidisciplinary team meetings enable a cross-pollination of ideas and research questions, with opportunities for ongoing future collaborations. To date, SAC members have published 4 peer-reviewed manuscripts related to SAC work, as well as 2 non–peer-reviewed articles, and 2 non–peer-reviewed meeting abstracts.12Bailey L.C.R.H. Burrows E.K. Bunnell H.T. Camacho P.E.F. Christakis D.A. Eckrich D. et al.Assessment of 56,441 Pediatric patients tested for SARS-CoV-2 across the United States.JAMA Pediatr. 2020; https://doi.org/10.1001/jamapediatrics.2020.5052Crossref PubMed Scopus (158) Google Scholar, 13Francom C.R. Javia L.R. Wolter N.E. Lee G.S. Wine T. Morrissey T. et al.Pediatric laryngoscopy and bronchoscopy during the COVID-19 pandemic: a four-center collaborative protocol to improve safety with perioperative management strategies and creation of a surgical tent with disposable drapes.Int J Pediatr Otorhinolaryngol. 2020; 134: 110059Crossref PubMed Scopus (47) Google Scholar, 14Loi M. Branchford B. Kim J. Self C. Nuss R. COVID-19 anticoagulation recommendations in children.Pediatr Blood Cancer. 2020; : e28485PubMed Google Scholar, 15White A. Mukherjee P. Stremming J. Sherlock L.G. Reynolds R.M. Smith D. et al.Neonates hospitalized with community-acquired SARS-CoV-2 in a Colorado neonatal intensive care unit.Neonatology. 2020; https://doi.org/10.1159/000508962Crossref PubMed Scopus (16) Google Scholar, 16Dove M.L. Jaggi P. Kelleman M. Abuali M. Ang J.Y. Ballan W. et al.Multisystem inflammatory syndrome in children: survey of early hospital evaluationand management.https://www.medrxiv.org/content/10.1101/2020.07.29.20164459v1.full.pdfDate: 2020Google Scholar Because of the benefit of the SAC to hospital operations, the SAC's structure and processes have been written into the institution's "Pandemic Playbook." In the case of another novel threat, the SAC can then be stood up in a shorter amount of time. This was agreed upon by hospital leadership based on the following value added: (1) the SAC demonstrated that it could rapidly assess the evidence and release recommendations in a short time period—sometimes as short as 3-4 days, in contrast to the institution's typical time period of 8 months—and (2) the SAC allowed for rapid collaborations on scientific grants and national organization statements that gave the institution academic exposure. The SAC created no additional cost to the hospital as the SAC drew on members of the existing CE team. For faculty members of the SAC, the time invested was time they would have spent figuring out COVID-19–related policies and procedures for their own divisions; the SAC coordinated those efforts so that divisions were not duplicating efforts. The SAC also provided faculty the opportunity to pursue several activities relevant to academic advancement, including working on clinical committees and policy development. This contribution was supported in a letter of recognition SAC leadership sent to the department chair on behalf of each member. The SAC's evidence synthesis process became the basis for a medical student elective and included several postgraduate trainees. When traditional third-year clinical rotations were closed because of social distancing, the School of Medicine designed a COVID-19 elective course that placed students in diverse COVID-19–related settings including the SAC. Students, residents, and subspecialty fellows on the SAC worked in project teams to help research and draft initial SAC guidance. Students and trainees were mentored by senior SAC members, providing an opportunity for professional development and academic mentorship. Additional approaches to disseminating information from the SAC's clinical guidance documents and pathways included multiple lectures given within the University. Updates on SAC activities and relevant documents were presented at section meetings, as well as the hospital's medical staff, epidemiology, and pediatric community Town Hall meetings. Materials from SAC guidance documents were incorporated into the hospital's internal frequently-asked-questions site for staff. Information generated for SAC guidance documents were incorporated into national guidelines through its members who were involved in several national organizations, including the Sharing Antimicrobial Reports for Pediatric Stewardship (SHARPS) collaborative and the International PICU COVID-19 Collaborative.17International PICU COVID-19 Collaborative 2020.https://www.openpediatrics.org/group/international-picu-covid-19-collaborativeGoogle Scholar Since inception, the median number of times SAC pathway documents are opened is 368 per month, with an IQR from 333 to 523. The most frequently accessed document is the clinical pathway for patients suspected to have MIS-C. Given the rapidly changing evidence related to COVID-19, the SAC continues to update its guidance statements quarterly or as new key evidence is released. Content experts continue to monitor evolving evidence and update guidance and pathways monthly or more often as applicable. The SAC is also using process and outcome metrics to further adapt the COVID-19 and MIS-C clinical pathways. Based on epidemiologic projections and the clinical research pipeline—including more than 2900 COVID-19–related studies in ClinicalTrials.gov—the SAC projects that the COVID-19 pandemic curve and ongoing COVID-related scientific discovery will extend into 2022. Anticipating the potential need to scale up efforts in response to pandemic surges, the SAC will operate with flexibility depending on institutional needs as well as the pace of scientific discovery to meet future needs. The SAC was developed in response to our health system–driven demand for evidence. We engaged a diverse team of scientific experts and hospital leaders in a well-coordinated and timely pandemic response that met operational and academic goals with an infrastructure that is customizable. We have developed a process for prioritizing clinical needs, developed a rapid evidence assessment method, and adapted existing CE processes for rapid implementation and dissemination. Our iterative experience can inform not only our institution's CE processes to match future evidence delivery needs, but, in addition, through sharing our processes and templates on public Web sites, we plan to disseminate our learned experience with other healthcare systems interested in replicating the SAC's framework in their specific settings.

In June 2015, Ethan, an 11-year-old boy whose “light shone from his eyes,” began suffering severe headaches accompanied by fever. Clinicians repeatedly misdiagnosed Ethan over several weeks as migraine headaches. An insidious, more dangerous condition remained undetected despite his parents repeatedly questioning the migraine diagnosis. Below, David and Farren Wyner, Ethan’s parents, describe the missteps in Ethan’s diagnostic journey. Their experience highlights the National Academy of Medicine’s challenge to the health care community to begin addressing diagnostic safety as laid out in Improving Diagnosis in Health Care.1 We share how Children’s Hospital Colorado responded to the challenge, in partnership with the Wyners.When Ethan was ∼7 years old, he experienced several sharp, localized, short-lived headaches. A neurologist at Children’s Hospital Colorado (CHCO) diagnosed “ice pick headaches” thought to be related to our family’s migraine history. She explained the headaches may evolve into traditional migraines when Ethan reached puberty.In late June 2015, Ethan, at age 11, developed another headache with photophobia. His pediatrician examined him and explained he probably had a migraine. She recommended over-the-counter pain medications until the headache resolved.One week later, Ethan was still experiencing an unremitting headache as well as mild fever. His pediatrician referred us to CHCO’s 24/7 urgent care. The staff took a family history, performed a neurologic examination, and began a “migraine protocol” of intravenous medications and fluids. When the protocol appeared to offer relief, the staff evidently cemented the migraine diagnosis. When we asked staff repeatedly across multiple shifts if migraines could last for weeks without resolution, we were assured they could. Because that assurance contradicted Ethan’s father’s lifelong experiences with migraines, we asked if imaging was warranted. The providers confidently replied that imaging was not indicated, that computed tomography (CT) would expose Ethan to unnecessary radiation, and that he would require sedation for an MRI, something they were hesitant to do. We were discharged, with recommendations to continue over-the-counter medications and follow-up with a neurologist.When Ethan’s headache continued unabated for another 2 days, we returned to urgent care. Our second experience, including 2 shift changes, was almost exactly like our previous visit: the same family history, neurologic examination, and “migraine protocol.” We again asked if migraines could last 2 weeks. We again asked about the benefit of advanced imaging. We again received the same answers. When the migraine protocol provided no relief, intravenous prednisone was prescribed, leading to a dramatic improvement in Ethan’s discomfort. Rather than considering what other conditions might produce such drastic relief with steroids, the staff was reassured by his improvement. We went home with 5 days of steroids and instructions to return for further treatment if the headaches continued.On prednisone, Ethan’s headaches minimally improved but persisted. Less than 24 hours after taking his last steroid, Ethan’s headache returned in full force. Now 6 days after the last urgent care visit and nearly a month since the headache began, we went to the CHCO emergency department. The staff assumed the accuracy of the migraine diagnosis that was, by now, written in Ethan’s chart from previous visits. Ethan received the exact same migraine protocol. When that did not help, they admitted Ethan for observation. Once Ethan was moved to a medical ward, his condition deteriorated dramatically over the next 3 hours. The staff decided to transfer him to the ICU. In route, Ethan’s condition deteriorated further, and he underwent emergent CT.The CT revealed a mass, presumably a brain tumor. He was taken straight to the operating room after the CT, without us being able to see him. We learned Ethan was being prepared for emergency surgery. After a shorter-than-expected surgery, we learned the mass was not a tumor but an abscess, likely present for some time. Ethan never regained consciousness after surgery and died 5 days later.We understand how doctors initially reached the faulty diagnosis. Ethan had a history of headaches. His dad has migraines, as do both of Ethan’s grandmothers. His symptoms mimicked migraines. We also understand how rare his true diagnosis was. But it is not the job of medical professionals to blindly accept convenient answers. We acknowledge that we did not hear every conversation that happened between clinicians. We accept that mistakes happen. We know medical professionals are fallible. But this was not a 1-time error made by 1 professional. This was a system-wide failure with multiple components. Numerous clinicians exhibited the same faulty reasoning. We were not provided the opportunity to share in the decision-making about a CT scan. The migraine diagnosis documented in the chart became established fact without being questioned during subsequent encounters. Our opportunities to contribute to the diagnostic process diminished at each encounter as the migraine diagnosis became solidified in the collective consciousness of the system.Rather than feeling like partners or, at least, valued contributors in the diagnostic process, we felt steamrolled by the army of white coats and scrubs, all apparently eager to corroborate and support each other’s theories. As parents, it is not our job to be medical experts, but it is our job to ask hard questions and push for better answers. Time and again, when we did just that, we felt dismissed by people we relied on to be the experts.We needed a creative thinker, a maverick, who thought outside the box and who entertained possibilities other than the obvious written into the chart. Had we had a physician who said, “It is probably a migraine, but Ethan’s felt bad for a few weeks. I would really like to know what an MRI would show,” then the outcome may have been different.In the absence of that maverick (or, maybe, in addition), Ethan needed a system with room for doubt built in, room to question its own biases, that had sufficient checks and balances to explore the possibilities of rare situations. Without that system, all we got was shift change after shift change that simply inherited the previous diagnosis without question. Indeed, the focus seemed to be on confirming that diagnosis, oblivious to the possibility that the reasoning was clouded by confirmation bias. As Ethan’s parents, we were powerless. We were at the mercy of the health professionals. If Ethan had a doctor who thought creatively and autonomously and encouraged by a supportive medical system, he may have come home.Our goal in partnering with Children’s Colorado is to turn our avoidable tragedy into a meaningful contribution to patient safety. We want Ethan’s story to lead to changes in the way the medical system approaches diagnosis so that no parents have to bury their child because of a diagnostic error.Improving Diagnosis in Health Care places the patient at the center of the diagnostic team because “[p]atients hold critical knowledge that informs the diagnostic process” and “bear the ultimate risk of harm from a diagnostic error.”1 This central placement requires the rest of the diagnostic team to listen actively and communicate effectively to ensure the best outcome from the diagnostic process. Farren and David felt dismissed and disconnected from the process once the clinicians conferred a diagnosis on Ethan. Their experience is not unique.Ethan’s story exemplifies key barriers to effective partnership with parents previously highlighted in Family Partnerships: fear of being labeled a “difficult parent”; insufficient confidence to understand medical jargon; and exclusion from conversations between clinicians.2 Knowledge asymmetry between patients and clinicians creates power imbalances that impede effective communication and imply that, once made, a diagnosis is right rather than a working hypothesis.3 Knowledge and power imbalances that hamper communication are supported by evidence that 50% of patient narratives regarding diagnostic errors involve ignoring the patient’s knowledge (eg, worrisome symptoms and failure to improve).4 These experiences underpin diagnostic errors for many Americans.5The first step in our journey addressed a culture reticent to discuss diagnostic errors.6 Dr Grubenhoff and the Wyners have presented the consequences of not acknowledging parents’ contribution to the diagnostic process to clinicians, nurses, administrators, and a national patient safety organization. We have also recorded an educational video that relays Ethan’s story, followed by introduction of concepts, including cognitive bias and diagnostic safety, that has been shared locally and nationally. A patient’s outcome cannot be dependent on “mavericks” comfortable challenging confirmation bias and diagnosis momentum when parents ask tough questions about the accuracy of diagnoses. Diagnostic safety requires being receptive when asked tough questions and welcoming of improvement opportunities. However, clinicians must ask uncomfortable questions about our diagnostic errors, even when patients and families feel unable to speak up. Through the Wyner’s partnership, CHCO has begun asking tough questions about failures in the diagnostic process at both individual and institutional levels.However, awareness does not necessarily prompt change. The commitment CHCO made to Ethan’s parents mandates taking an honest look at our diagnostic performance as a system. A year after Ethan’s death, CHCO invited David and Farren to share their experience during a hospital-wide collaborative case review (CCR) as part of a 2-day kickoff to discuss strategies to improve culture surrounding diagnostic errors conversations. CCR can promote system-wide improvements and may be better than root cause analysis (RCA) for identifying opportunities to improve the diagnostic process.7,8 Now held quarterly for 3 years, CCR focuses on how systems, communication, infrastructure, and vulnerabilities in human reasoning create risks to diagnostic safety. During our regular diagnostic safety program updates with the Wyners, they challenged us to incorporate the patient perspective in CCR. We now include patient liaisons in these conferences to provide the patient and family voice. Feedback from participants consistently confirms that we have created psychological safety for the difficult task of reviewing human contributions to misdiagnosis.After CHCO disclosed the diagnostic error in Ethan’s case to Farren and David they asked: “Is this going to happen to someone else’s child?” As Chief Medical Officer and Medical Director for Diagnostic Safety (DB and JAG), we wish we could definitively answer, “No!” An inherent barrier to reducing diagnostic errors lies in the relative obscurity of latent vulnerabilities to reliable diagnostic performance. Although CCRs and RCAs identify highly impactful diagnostic errors, the rarity of such tragic cases potentially leads safety leaders to focus on the wrong threats. The risks that expose patients to more frequent but less severe consequences remain hidden. Diagnostic errors compose only a small fraction of medical errors reported to passive incident reporting systems.9 To address this, we now review all admissions occurring within 10 days of an emergency department or urgent care visit to screen for potential diagnostic errors. This has unearthed patterns repeated in multiple patient encounters allowing recognition of diagnostic safety improvement opportunities. For instance, we discovered nearly a dozen cases like Ethan’s. None were reviewed by CCR or RCA. Aggregating cases previously considered isolated events reveals repetitive patterns of misdiagnosis. This knowledge informed a migraine clinical care pathway that specifically excludes patients like Ethan. Such institutional learning opens new possibilities for identifying possible diagnostic missteps amenable to systematic improvement efforts.Although less tangible than specific programs, the Wyners’ partnership has proved invaluable for maintaining our hospital’s commitment to diagnostic safety. Patients want clinicians to accompany them on their journey after adverse events helping them better understand what has happened “to allow patients to…feel support and empathy from healthcare providers”; patients also desire trust from their clinical companions.10 The Wyner’s partnership continually renews our commitment to diagnostic safety.Our journey and partnership have only begun. As the Wyners point out, a culture persists that marginalizes patients and families to recipients of a diagnosis rather than engaging them as coequal members of the diagnostic team. Partnership with patients and families requires humility and active listening from health care providers and committed engagement to find solutions together. Ethan’s, it turns out, was not as rare a situation as any of us had hoped. But partnering with David and Farren preserves our accountability to the commitment we made 6 years ago to ask hard questions and push for better answers.

Despite ongoing efforts of the national Button Battery Task Force (BBTF), cases of major morbidity and mortality continue to be reported at a higher rate after ingestion of higher voltage and larger button batteries. Initiation of sucralfate or honey shortly after some button battery ingestions is crucial to prevent further injury while awaiting emergent removal. Endoscopic acetic acid irrigation post removal of button battery may halt deeper injury. Gastroenterologists need to be aware of the updates to the National Capital Poison Center algorithm for button battery ingestion.

Most US states have now legalized medical marijuana (MMJ) use, giving new hope to families dealing with chronic illness, despite only limited data showing efficacy. Access to MMJ has presented several challenges for patients and families, providers, and pediatric hospitals, including the discrepancy between state and federal law, potential patient safety issues, and drug interaction concerns. Colorado was one of the first states to legalize MMJ and has remained at the forefront in addressing these challenges. Children's Hospital Colorado has created and evolved its MMJ inpatient use policy and has developed a unique consultative service consisting of a clinical pharmacist and social worker. This service supports patients and families and primary clinical services in situations in which MMJ is actively being used or considered by a pediatric patient. The first 50 patients seen by this consultative service are reported. Eighty percent of patients seen had an oncologic diagnosis. Symptoms to be ameliorated by active or potential MMJ use included nausea and vomiting, appetite stimulation, seizures, and pain. In 64% of patients, MMJ use was determined to be potentially unsafe, most often because of potential drug-drug interactions. In 68% of patients, a recommendation was made to either avoid MMJ use or adjust its administration schedule. As pediatric hospitals address the topic of MMJ use in their patients, development of institutional policy and clinical support services with specific expertise in MMJ is a recommended step to support patient and families and hospital team members.

Background/PurposeThe surgical morbidity and mortality (M&M) conferences at a regional children's hospital achieved the goals of case by case peer review and education for trainees but provided limited data for trending and analysis. In 2019, an institution-wide effort was initiated to create an electronic case review system with the goals of improving event capture and real-time practice performance feedback. Surgical M&M was migrated to this structured case review format to provide a platform for surgical performance improvement.MethodsAn online secure database was created with a 3-step classification system based on Clavien-Dindo severity score, peer review, and causality fishbone analysis. The data entered were available in an interactive dashboard. Retrospective tabulation of the 2018 M&M data was performed using the archived paper system used prior to 2019.ResultsFor the calendar year of 2019, the division of pediatric surgery captured and categorized 193 complications in the case review system. The capture rate was 50 per 1000 surgical procedures. For a similar time frame in 2018, the capture rate was 35 per 1000 surgical procedures. The dashboard provided run charts of the incidence and types of complications by procedure and by surgeon. Similar trend data were not available in 2018. The dashboard output has made possible the creation of (non- risk adjusted) individual surgeon performance reports. The output has been used to direct process improvement projects and educational content.ConclusionCreation of an online database with interactive dashboard has allowed surgical M&M to evolve into a systematic case review that greatly facilitates quality improvement efforts. This system increased the event capture rate and provided novel practice performance feedback, resulting in process improvement projects and educational objectives predicated on the trending data. These electronic reporting tools are now available to all surgical divisions and represent a transformative approach to surgical case review.Type of StudyRetrospective Historical control; Quality improvement.Level of EvidenceLevel III.

Foreign body (FB) and caustic ingestion events are common conditions managed by pediatric gastroenterologists. Several publicly accessible options exist for providers to report pediatric ingestions. However, industry has deemed existing data sources insufficient for some hazards to justify product safety changes. The objectives of this study were: 1) to capture ingestion injury reporting practices among pediatric gastroenterologists and 2) to discuss a solution.

The incidence of childhood obesity continues to rise and alarmingly, 40% are already estimated to have nonalcoholic fatty liver disease (NAFLD), the leading cause of liver transplantation. An early origin exposure that contributes to NAFLD may be maternal obesity, diet, triglycerides (TG) and free fatty acids (FFA) in-utero, particularly in offspring of mothers with diabetes or gestational diabetes (GDM) who are at high risk for childhood metabolic disorders. In this pilot study, we explored the hypothesis that maternal TG in diet-controlled GDM (A1GDM) is associated with offspring intrahepatic lipid (IHL) accumulation. We randomized newly diagnosed A1GDM women (n=13, BMI 32±2 [mean±SEM]) to eucaloric diets at 32 weeks. Fasting and postprandial (PP) glucose, insulin, TG, FFA, and glycerol were measured in a breakfast test meal study (hourly x5) at baseline and 37 weeks. Newborn IHL content (Magnetic Resonance Spectroscopy; MRS) and body composition (%fat, PEAPOD) were measured at ~10 days. At 32 weeks, but not at 37 weeks, maternal fasting TG (199±17 mg/dL) and the 5-hour TG area-under-the-curve (AUC) were strongly associated with newborn IHL (r=0.69, p=0.01; r=0.75, p=0.003, respectively). The 1-hour and 2-hour PPTG (205-206±19-20 mg/dL) were highly associated with IHL (r=0.69-0.73; p≤0.01) as was the 5-hour glycerol AUC (r=0.65, p=0.01). There was no relationship between IHL and subcutaneous %fat by PEAPOD. We found no associations between maternal pre-pregnancy BMI, gestational weight gain (11±1.6 kg), glucose, insulin, or FFA and IHL. These data are the first in humans to support that maternal TG exposure at the time of GDM diagnosis, when fetal subcutaneous fat depots are early in development, may result in increased liver lipid deposition. If confirmed, MRS newborn liver fat measures and early interventions targeting maternal TG may be indicated to lower fetal liver fat deposition that could potentially be a developmental risk for pediatric NAFLD. Disclosure T.L. Hernandez: None. S.S. Farabi: None. N. Hirsch: None. E.Z. Dunn: None. E.A. Haugen: None. D. Brumbaugh: None. M.S. Brown: None. J.E. Friedman: Consultant; Self; Janssen Research & Development. L.A. Barbour: None. Funding National Institutes of Health

It is my great honor to introduce my friend and mentor, Dr Michael Narkewicz, (Fig. 1) for the NASPGHAN 2018 Distinguished Service Award.FIGURE 1: Dr. Michael Narkewicz.Mike was born to Richard and Carol Narkewicz. Dr Richard Narkewicz was actually the first pediatrician in the family, attending the School of Medicine of the University of Vermont on an ROTC scholarship and training at Lackland Air Force Base in San Antonio. After completing his military service, the family returned to Burlington Vermont, where Mike's father set up a solo pediatric practice. He enlisted Mike's services early. Mike recalls his first summer job at 13 years old updating problem lists in patients’ charts in his father's practice. Notable families in the elder Dr Narkewicz's practice included the Von Trapp family, of Sound of Music fame, who emigrated from outside of Salzburg to Vermont, as well as the kids of Ben and Jerry (of ice cream fame). Mike kept himself busy playing on the family farm outside of Burlington, where they raised chickens, hogs, cattle, and horses, and where Mike learned to hunt and fish, which remains one of his passions today. Mike attended Dartmouth College, majoring in chemistry and environmental studies. He decided late on medicine but followed his father's footsteps into medical school at the University of Vermont. Mike is most often the smartest guy in the room and is not afraid to let you know. One medical school classmate remembered that Mike would complete every examination with an audible flourish, just to let everyone know that he was the first to finish. Mike gravitated towards pediatrics. One of his mentors in Vermont, Dr Buzz Land, was an old pal of Bill Balistreri and arranged for Mike to enter into a month-long, fourth year externship at Cincinnati Children's on the inpatient gastrointestinal (GI) service. At that time, future colleagues Ron Sokol and Fred Suchy were at Cincinnati, where Ron was a senior fellow and Fred a junior attending. Sue Moyer and John Bucuvalas were first-year fellows at that time. Even as a medical student, he made quite an impression on the inpatient residents. After Mike left at the end of the month, Fred recalls the house staff asking, “Hey, where did the third fellow go?” Mike then moved to Denver, completing his pediatrics residency at the University of Colorado, and became the program's first GI fellow. He trained under an exceptional faculty—Arnie Silverman, Judy Sondheimer, and Ron Sokol. Half a salary was cobbled together, and Mike had to moonlight at Denver General Hospital that first year to make ends meet. Arnie taught the art of clinical GI and Ron the science of GI, whereas Judy taught him that patience was a virtue. Mike's father had built a successful pediatric practice and was a civic leader in Burlington, but then he also served his patients and the children of Vermont as a leader in the American Academy of Pediatrics (AAP). Dr Richard Narkewicz rose through leadership at the AAP and eventually served as President of the AAP. Mike joined the AAP as a Fellow in 1988, during his father's tenure as AAP President. As far as we know, Mike is the only AAP Fellow whose certificate is signed by his father! After spending his first year of fellowship on half a salary, Mike was smart enough to apply for the Physician Scientist Training Program and was selected to spend his second and third year fellowship years solely dedicated to research. He moved to Paris, France, to work in the laboratory of Jean Girard. In Paris, Mike developed a passion for science, for speaking French, and good wine, not necessarily in order of importance. The work hours in Paris were quite a favorable improvement from his first year of fellowship. Paris was a wonderful place for Mike and Dana, newlyweds, to spend their first 2 years together. Mike returned to Colorado to join the faculty as a physician scientist. His research contributions have been prolific, ranging from basic mechanisms of hepatic amino acid metabolism to clinical research in children on the topics of viral hepatitis, acute liver failure, and the development of cystic fibrosis-related liver disease. Mike's service contributions, which are the focus of this award, have been similarly prolific. I will focus on 3 areas: service to children with cystic fibrosis (CF). Mike stumbled into CF research, but during his career his passion for this disease grew, and he has developed an important partnership with the Cystic Fibrosis Foundation. Mike has created awareness of the clinical importance of CF-liver disease, through organization of symposia at the NIH and North American CF conference. He conceived and has organized the CFLD research network, supported by the NIH and CF Foundation that has driven multicenter research producing seminal observations forming our understanding of the evolution of CF-liver disease. He serves on the Clinical Research Committee and Data Safety Monitoring Board of the CF Foundation. Service to our organization: Mike has been a key contributor and/or Chair to an amazing breadth of NASPGHAN committees, including Research, Hepatology, Training and Education, Program Planning, and Finance. He left a lasting impact on the Teaching and Tomorrow and Fellows’ Conference programs, so impactful to our members. Mike was elected to Secretary/Treasurer of NASPGHAN in 2014. The Secretary/Treasurer role, in the words of Dr Carlo DiLorenzo, is a job “with tremendous responsibilities and little visibility.” In this role, Mike rolled up his shirt sleeves and focused on the gritty details, pushing through changes in organizational by-laws that expanded membership opportunities. He created a task force to focus on our organization's investment allocations, leading to the adoption of a new investment strategy that has allowed for more earning potential now and for years ahead, fueling many NASPGHAN-supported programs in education and research. Service locally in Colorado: Mike was the inaugural GI/Hepatology fellow and then went on to transform the training program by establishing a T32 training grant and serving as training program director for 8 years. He served as Medical Director of the section. Mike served as President of the Medical Board and on the Hospital's Board of Directors and serves currently as one of the top 2 physician executives at Children's Hospital Colorado, responsible for execution of major parts of the hospital's strategic plan. Mike is full professor with tenure at the University of Colorado School of Medicine, previously serving as Associate Dean for Pediatric Clinical Affairs, and serves on the Board of Directors of the Faculty practice for the University of Colorado. As well, Mike is a superb clinician and a resource for questions on difficult cases that span all domains of gastroenterology and hepatology. Mike is a master generalist gastroenterologist/hepatologist in an age of sub-sub specialization. How does one man do all this? Mike has a tireless work ethic and is focused and organized like few people. Mike gets the big picture of health care. He operates as easily at the health care system level as he does at the bedside. Over his career, he has allowed the practice of medicine to teach him and has grown wiser as a result. He relishes the success of his colleagues and has mentored scores of fellows in Colorado and around the country. And like his father, service to children always remain at his center. Mike's family keeps him going. His legendary intensity is balanced by his wife of 31 years Dana, who is among the most laid back people in Colorado. Mike and Dana have 2 wonderful sons Andrew and Jeff, who share Mike's love of the outdoors. Mike relaxes by going back outside, using those lessons learned as a youth to fish and traveling around the Rocky Mountain region to find the best trout streams. Despite a tough exterior, Mike has a well-known soft spot for kids. Mike serves on the Medical Advisory Board for Round Up River Ranch in Colorado, a summer camp for children with chronic illness. Mike never misses a summer at the Ranch, where he is known to loosen up a bit to the delight of the children. Mike's commitment to service is deserving of this important recognition. Congratulations!

To the Editor A 27-month-old, 13.8 kg girl presented after swallowing multiple neodymium magnets. She underwent esophagogastroduodenoscopy (EGD) within 5 hours; however, the magnets had progressed beyond the reach of EGD. She was admitted and remained asymptomatic. Abdominal x-rays showed magnet nonprogression after 19 hours. She underwent antegrade small bowel enteroscopy for removal with single balloon overtube (outer diameter 13.2 mm). The magnet string was visualized closed across a fold of small bowel with areas of bleeding and early pressure injury. The magnets were too large to be brought through the working channel; therefore, piecemeal removal was required (Fig. 1A). The enteroscope was removed with each pass, but the overtube was left in position (Fig. 1B), allowing efficient readvancement of the enteroscope to the magnets. After 85 minutes, removal of 28 magnets was successful, and she recovered without sequelae.FIGURE 1: A, Enteroscope adjacent to magnet string. Piecemeal removal was required with removal of the enteroscope with each section of magnets. B, The overtube remained in place with enteroscope removal to allow rapid reinsertion to the magnet location.Multiple magnet ingestions beyond the reach of EGD raise difficult management decisions. Symptoms may be mild as intestinal injury is occurring (1) and can rapidly progress to intestinal perforation, volvulus, and death (2–4). Our asymptomatic patient had magnets removed within 24 hours; however, she had magnet-related mucosal damage and pressure injury. If magnets fail to progress, antegrade small bowel enteroscopy should be considered, if patient size and location are appropriate, as an alternative to continued observation or surgery. A previous US ban on small, powerful magnets (5) was overturned in 2016. Powerful magnet ingestions should be reported to the consumer product safety commission (www.cpsc.gov) with the goal that additional data and research will lead to policies that will safeguard children in the future.

Advances in treatment and multidisciplinary management have resulted in improved survival of individuals with Duchenne muscular dystrophy (DMD). Updated DMD treatment recommendations as found in the 2018 DMD Care Considerations are aimed to assist multidisciplinary care teams in providing standardized care to their patients, including attention to nutritional and gastrointestinal health. Challenges remain for care teams in accurately estimating height and nutritional status for individuals with DMD. It can be difficult for patients to maintain a healthy weight. Risk factors for obesity include glucocorticoid therapy and loss of ambulation. In contrast, in the later stages of the disease, swallowing dysfunction can lead to poor nutrition and consideration for gastrostomy tube placement. Constipation is highly prevalent, underrecognized, and undertreated in DMD. With this article, we address the assessment and management of gastrointestinal and nutritional issues, as well as clinical controversies.

Since the publication of the Duchenne muscular dystrophy (DMD) care considerations in 2010, multidisciplinary care of this severe, progressive neuromuscular disease has evolved. In conjunction with improved patient survival, a shift to more anticipatory diagnostic and therapeutic strategies has occurred, with a renewed focus on patient quality of life. In 2014, a steering committee of experts from a wide range of disciplines was established to update the 2010 DMD care considerations, with the goal of improving patient care. The new care considerations aim to address the needs of patients with prolonged survival, to provide guidance on advances in assessments and interventions, and to consider the implications of emerging genetic and molecular therapies for DMD. The committee identified 11 topics to be included in the update, eight of which were addressed in the original care considerations. The three new topics are primary care and emergency management, endocrine management, and transitions of care across the lifespan. In part 1 of this three-part update, we present care considerations for diagnosis of DMD and neuromuscular, rehabilitation, endocrine (growth, puberty, and adrenal insufficiency), and gastrointestinal (including nutrition and dysphagia) management.

OBJECTIVE:The aim of the study is to analyze a large series of esophageal balloon dilations in patients with epidermolysis bullosa (EB) to determine procedural approach and frequency of post-endoscopic adverse events (AEs). METHODS:Retrospective chart review for AE occurrence and clinical outcomes in children and adolescents with EB, age 1 to 19, who underwent esophageal dilation for esophageal stricture(s) from January 2003 to April 2016 at an academic, tertiary care, free-standing children's hospital. The primary outcome measure was occurrence of procedural AEs (defined as events occurring within 72 hours after endoscopic dilation procedure). RESULTS:A total of 231 fluoroscopy-guided esophageal balloon dilation procedures (209 anterograde, 20 retrograde, 2 both) were performed in 24 patients. Strictures were more common in the proximal portion of the esophagus with median stricture location 13 cm from the lips. From 2003 to 2012, 4.1% of dilations were retrograde. From 2013 to 2016, 20.2% of dilations were retrograde. AEs attributable to dilation occurred after 10.0% of procedures, and the most common AEs were vomiting, pain, and fever. No esophageal perforations, serious bleeding events, or deaths occurred secondary to dilation. The rate of post-dilation hospitalization was 6.9%. Dilation approach (anterograde vs retrograde) did not impact the likelihood of AEs. CONCLUSIONS:The characteristic esophageal lesion in EB is a single, proximal esophageal stricture. EB patients can safely undergo repeat pneumatic esophageal balloon dilations with minimal risk for severe complication. We observed a trend towards increased use of retrograde esophageal dilation.

Objective To assess the safety, efficacy, and relative expense of a nurse-led fecal microbiota transplantation (FMT) program for the treatment of recurrent Clostridium difficile infection (CDI). Study design Retrospective cohort study design in children aged 1-18 years with recurrent CDI. The intervention was an intragastric FMT with stool derived from a donor stool bank. Primary outcome was resolution of diarrhea at 3 months post-transplantation. A secondary analysis compared charge data associated with FMT by intragastric delivery vs administration by colonoscopy or nasoduodenal tube. Results A total of 47 intragastric FMT procedures were performed in 42 children (median age 9 years) with recurrent CDI. Response to treatment varied by disease status, with 94% success in previously healthy children, 75% in medically complex children, and 54% in children with inflammatory bowel disease (P = .04). FMT via intragastric delivery showed lower facility and professional charges by 85% and 78% compared with delivery via colonoscopy and radiology-placed nasoduodenal tube, respectively. The use of stool derived from a donor stool bank decreased charges by 49% compared with charges associated with the use of a donor who was a relative. Conclusion A nurse-led intragastric FMT procedure using stool derived from a donor stool bank is a relatively inexpensive and efficacious treatment for recurrent CDI in children. Intragastric FMT success in children was attenuated by the presence of underlying disease, particularly inflammatory bowel disease.

OBJECTIVES:Bacterial colonization and succession of the human intestine shape development of immune function and risk for allergic disease, yet these processes remain poorly understood. We investigated the relations between delivery mode, initial bacterial inoculation of the infant oropharynx (OP), and intestinal colonization.METHODS:We prospectively collected maternal rectal and vaginal swabs, infant OP aspirates, and infant stool from 23 healthy mother/infant pairs delivering by cesarean (CS) or vaginal delivery (VD) in an academic hospital. Bacterial abundance (16S rRNA sequencing) and community similarity between samples were compared by delivery mode. Shotgun DNA metagenomic sequencing of infant stool was performed.RESULTS:VD infants had higher abundance of Firmicutes (mainly lactobacilli) in OP aspirates whereas CS OP aspirates were enriched in skin bacteria. OP aspirates were more similar to maternal vaginal and rectal microbiomes in VD compared with CS. Bacteroidetes were more abundant through 6 weeks in stool of VD infants. Infant fecal microbiomes in both delivery groups did not resemble maternal rectal or vaginal microbiomes. Differences in fecal bacterial gene potential between CS and VD at 6 weeks clustered in metabolic pathways and were mediated by abundance of Proteobacteria and Bacteroidetes.CONCLUSIONS:CS infants exhibited different microbiota in the oral inoculum, a chaotic pattern of bacterial succession, and a persistent deficit of intestinal Bacteroidetes. Pioneer OP bacteria transferred from maternal vaginal and intestinal communities were not prominent constituents of the early infant fecal microbiome. Oral inoculation at birth may impact the intestinal microenvironment, thereby modulating early succession of intestinal bacteria.

Gastrointestinal injuries secondary to button battery ingestions in children have emerged as a dangerous and difficult management problem for pediatricians. Implementation of a multidisciplinary team approach, with rapid and coordinated care, is paramount to minimize the risk of negative outcomes. In addition to providing a comprehensive review of the topic, this article outlines the authors' referral center's experience with patients with severe battery ingestion, highlighting the complications, outcomes, and important lessons learned from their care. The authors also propose an algorithm for clinical care that may be useful for guiding best management of pediatric button battery ingestion.

Steven Abman Anthony Absalom Thomas Abshire Wiliam Ackerman Margaret Adam Shawn K. Ahlfeld S.Faisal Ahmed Joseph L. Alcorn Eileen Steinle Alexander Omar Ali Geoffrey Ambler Istvan Arany J. Arciero Petra Clara Arck Moshe Arditi Guillaume Arlet H. Artac Stephen Ashwal Sara Aspberg Bakri Assas Chun Ting Au Renata Auricchio Richard Auten John E Baatz Tiziana Bachetti Carl Backes Praveen Ballabh Robert S. Baltimore Siddharth Banka Laura Banks Piers Barker James Barkovich Alberto Battezzati Beau Batton Olivier Baud Michel Baum Catherine Beauchemin S. Patricia Becerra Manon J. Benders Pere Berbel Anne Berg Ina bergheim Janet Elizabeth Berrington Carol Berseth Enrico Bertini Julia Beulertz Flavia Bezerra Vinod K Bhutani Marc B Bierings Stella Tracey Bjorkman Robert Black Heather Louise Blackmore Jorge Blanco Francis G Blankenberg David Bleich Joseph M. Bliss Jacky Bonaventure Avihu Boneh Riccardo Bonfanti Osvaldo Borrelli Arend F Bos Rodolfo Bracci Paolo Brambilla C Brendel Dora Brites Frank Brozovich David Elliott Brumbaugh Giacomina Brunetti Luc Bruyndonckx Karen Jane Bryson Vittoria Buccigrossi Sara Buckelew Daniel Bulte Giuseppe Buonocore David Burgner Jane C. Burns Will Bush Kara Calkins Angelo Salvatore Campanozzi Deborah Campbell Philippe Campeau Michael S. Caplan PJ Carey Waldemar A. Carlo Susan E. Carlson Virgilio P. Carnielli Marco Carotenuto David J Carr William J. Cashore Kevin A Cassady Gustavo Osvaldo Castaño Marco Castori Brian Catchpole Corrado Cecchetti Gediminas Cepinskas Lina F Chalak Wei-Chiao Chang Jennifer R Charlton Christiane Charriaut-Marlangue Raul Chavez-Valdez Fook-Choe Cheah Paul Checchia Henry Cheng Yiu Fai Cheung Terry W. Chin Valerie Y. Chock Rolando Cimaz Nevio Cimolai Tereza Cindrova-Davies Erika C. Claud Nelson Claure Pilar Codoñer-Franch Martine Cohen Solal John Connolly James Connor Giangennaro Coppola Luc Cornette Serena Counsell John M. Dagle Yun Dai Alexis S Davis Miles De Blasio Sarah D. De Ferranti Nicola De Luca Emanuella De Lucia Rolfe Linda S. de Vries Koert de Waal Charlotte Dean Justin Dean Sean Deane Roberta DeBiasi Tamas Decsi Andre Dejam Paul Delgado Olguin Christian Delles Eugene M. Dempsey Matthew Derrick Prasad Devarajan George A. Diaz Juliann DiFiore James DiNardo Lili Ding Jens Christian Djurhuus Franc C.L. Donkers Birgit Cornelia Donner Benard Dreyer Emma Gail Duerden Elia J. Duh Olivier Dulac Galit Dunietz Asim K K Duttaroy Michael B Dwinell David Eaton Danielle Ehret Barbara E Ehrlich Thomas Eiwegger Dawn Elder Maurizio Elia Nicholas Embleton Vibeke Ramsgaard Eriksen Erica A. Eugster Karen D. Fairchild Lir-Wan Fan Kathryn Noel Farrow Jia Feng Wu Rômulo Fernandes Mary S. Fewtrell Jeffrey R. Fineman Neil N. Finer I Fischbein Roslyn Holly Fitch V Flamand Brian W. Fleck Bobbi Fleiss Steven J. Fliesler Anna Forsberg P Forsythe William D. Foulkes Maria V. Fraga Mark Rohit Francis Axel Franz Mhoyra Fraser David Frommhold Richard Eugene Frye G Fueller Drude Fugelseth Ryoichi Fujiwara Camille Fung John Fuqua Sheila Gahagan Robert Galinsky Jorge Gallego Pediatr Res