University of Missouri–Kansas City

There is a growing body of clinical research on the utility of synthetic data derivatives, an emerging research tool in medicine. In nephrology, clinicians can use machine learning and artificial intelligence as powerful aids in their clinical decision-making while also preserving patient privacy. This is especially important given the epidemiology of chronic kidney disease, renal oncology, and hypertension worldwide. However, there remains a need to create a framework for guidance regarding how to better utilize synthetic data as a practical application in this research.

Presenting posters at scientific conferences is often viewed as a valuable way for professionals, researchers, and students to cultivate presentation skills. Furthermore, the exchange of ideas between presenters and attendees frequently stimulates new perspectives. Coupled with in-person poster discussion, our recent poster presentation at the National Association of School Nurses (NASN) conference amassed 110 qualitative feedback comments from attendees who viewed it online. Given the substantial feedback received, our team decided to conduct a thematic analysis to evaluate the impact of the presentation and examine whether any helpful information was provided. As the data were not solicited by the authors, we did not anticipate that attendee feedback regarding our poster would lead to this spontaneous research inquiry. Thus, this commentary paper was developed without the context of a predisposing research question or hypothesis. From the submitted data, five dominant themes emerged that were agreed upon by all authors: Awareness, motivation, implementation, evaluation, and COVID-19 issues. Based on this experience, we affirm the value of conference poster presentations that invite presenter evaluation feedback, offer multiple options for viewing presentations (e.g. virtual viewing in addition to in-person presentations), and award attendees continuing education credit in exchange for viewing and providing constructive comments.

Background Individuals living with multiple chronic conditions (MCC; e.g., obesity, hypertension, diabetes, dyslipidemia) are at an increased risk for occupational injuries, decreased work productivity, and early departure from the workforce. Methods A cross-sectional online survey of 200 working adults with obesity and hypertension, diabetes, and/or dyslipidemia was conducted to examine the relationships across self-efficacy, physical function, and healthy behaviors in addition to their relationship with the work environment. Findings Self-efficacy was found to have a statistically significant positive association with physical function ( p < .001) as well as with eating behaviors ( p < .001). Full-time workers reported greater physical function than part-time workers ( p < .001), and those working in active/manual labor positions reported more physical activity compared to those working in sedentary positions ( p = .001). Conclusion Individuals with greater self-efficacy for managing their chronic conditions also had higher physical function capabilities and healthier eating behaviors. In addition, individuals who work full-time reported higher physical function capabilities, and those in active work positions had higher physical activity scores. Future research is needed to implement and evaluate the effectiveness of interventions. Application to Practice Occupational health nurses need to work with employees with MCC to help accommodate healthcare provider visits and/or work restrictions to allow employees to maintain full-time work in addition to encouraging sedentary workers to increase physical activity.

Evinacumab, an angiopoietin‐like 3 inhibitor, significantly reduces low‐density lipoprotein cholesterol (LDL‐C) in patients with homozygous familial hypercholesterolemia (HoFH). Herein, we report pharmacokinetic and efficacy analyses of evinacumab in < 5‐year‐old patients with HoFH. Population pharmacometric models characterizing evinacumab exposure and LDL‐C response accounting for lipoprotein apheresis effect in ≥ 5‐year‐old patients were adapted for growth and maturation to predict and compare evinacumab and LDL‐C concentrations across age/weight groups in virtual ≥ 6‐month‐old patients receiving 15 mg/kg evinacumab intravenous (iv) infusions every 4 weeks (q4w). As expected from allometric theory, weight‐based dosing resulted in decreasing evinacumab exposures with declining body weight. Consistent with trends observed in > 5‐year‐old patients, the predicted percent change from LDL‐C baseline (%∆LDL‐C) was generally comparable or even higher in < 5‐year‐old patients (63.0%–68.5%) than in 5‐ to < 18‐year‐old patients (61.3%–67.8%) or adults (51.7%), with the predicted percentages of patients achieving %∆LDL‐C > 50% also higher in < 5‐year‐old patients (82.0%–86.9%) versus 5‐ to < 18‐year‐old patients (72.0%–84.5%) and adults (54.8%). Through a managed access program, six 1‐ to < 5‐year‐old patients received between 5 and 23 iv infusions of 15 mg/kg evinacumab q4w. Rapid and clinically meaningful LDL‐C reductions were observed, with %∆LDL‐C at the last reported dose ranging from 41.3% to 77.3%. Based on the actual patient dosing and plasmapheresis history, model‐predicted evinacumab and LDL‐C concentrations were comparable to the observed data collected in the managed access program. Overall, this analysis provides evidence for the use of evinacumab 15 mg/kg iv q4w dosing regimen in 6‐month‐old to 5‐year‐old patients.

BACKGROUND AND OBJECTIVES The American Board of Pediatrics requires that proposed changes to the duration of pediatric subspecialty training must include a framework for competency assessment with a measurement component. We analyzed the clinical Entrustable Professional Activity (EPA) level of supervision ratings across 3-year pediatric fellowships to determine if trainees met the minimum thresholds for graduation after 2 years of fellowship training. METHODS From spring 2019 through spring 2022, Clinical Competency Committees (CCCs) reported fellow supervision level ratings for all clinical EPAs, fellowship program directors (FPDs) assessed the scholarship EPA supervision level, and fellows self-reported their required level of supervision for all EPAs. Ratings were compared with minimum supervision level thresholds for fellow graduation previously identified by FPDs. We analyzed the proportion of fellows achieving these EPA supervision level thresholds after 2 and 3 years of training. RESULTS CCCs reported ratings for 1538 second-year and 1505 third-year fellows. Fewer than 50% of fellows met clinical EPA supervision level thresholds for graduation after 2 years of training, increasing to 86%–100% across subspecialties at 3 years. Fellow self-assessment aligned well with CCC ratings. FPDs reported that 64%–68% of fellows across subspecialties met the scholarship EPA supervision level threshold for graduation after 2 years compared with 99%–100% at 3 years. CONCLUSIONS As pediatric fellowships are currently structured and using an EPA assessment framework, many trainees are not ready to graduate after 2 years.

The Society for Craniofacial Genetics and Developmental Biology (SCGDB) hosted its 47th Annual Meeting on September 9–10, 2024, at the Stowers Institute for Medical Research and Children's Mercy Research Institute in Kansas City, Missouri. On the opening day, Drs. Jean‐Pierre Saint‐Jeannet and Elizabeth Leslie received the SCGDB Distinguished Scientist Awards in recognition of their exceptional contributions to craniofacial biology. Additionally, Dr. Daniel Jensen discussed his unique perspective on Treacher Collins syndrome, speaking as both a physician and a patient. Over the next 2 days, five sessions showcased groundbreaking research on cell signaling and genomic mechanisms regulating craniofacial development, human genetics, translational and regenerative approaches, and clinical management of craniofacial differences. The meeting also featured interactive workshops on engaging with journal editors during the manuscript review process and empowering mentees to take an active role in maximizing their mentorship experience. A poster session further fostered meaningful interactions among the attendees, who represented diverse career stages and research backgrounds in developmental biology and genetics, further strengthening the SCGDB community.

Objective Children with 22q11.2 deletion syndrome (22q11.2DS) present with complex medical challenges and often are followed by multiple specialists. Healthcare for these patients may be influenced by demographic and socioeconomic factors. This study's aim is to identify factors correlated with no-show clinic visits for these children. Design Retrospective cohort study Setting Tertiary care hospital Participants Pediatric patients with a diagnosis of 22q11.2DS. Patients without appointment attendance or socioeconomic data were excluded. Interventions None Main Outcomes and Measures To identify potential healthcare disparities, the patient's home address was cross-referenced with the GeoMarker database to define socioeconomic variables. We analyzed the rate of no-show clinic appointments to identify associations with socioeconomic, racial, or ethnic factors. Results Of the 198 patients with 22q11.2DS were included in this study. These patients were scheduled for 2049 visits with the 22q11.2 multidisciplinary clinic (22qMDC) and 8708 additional clinic visits within the institution. The median 22qMDC no-show rate was 0% [interquartile range—IQR: 0, 9.78] and 4.35% [IQR: 0, 15.00] for other clinic appointments. Socioeconomic factors associated with clinic no-show included Hispanic or Latino ethnicity, lower median household income, lower fraction of population with high school diploma, higher fraction of population below the poverty line, and higher fraction of population that required assisted income. Conclusions These findings highlight potential influences of socioeconomic factors on no-show clinic appointments in children with 22q11.2DS. These factors can be used to develop targeted interventions aimed at improving clinic attendance and ultimately elevating patient outcomes.

Therapeutic drug monitoring (TDM) for dose modification of biologics has the potential to improve patient outcomes. The US Food and Drug Administration (FDA) and the American Association of Pharmaceutical Scientists (AAPS) hosted the first US-based public workshop on TDM of biologics with contributions from a broad array of interested parties including healthcare providers, clinical pharmacologists, test developers, bioanalysis and immunogenicity scientists, health economics and outcomes research (HEOR) experts and regulators. The key insight was that despite a body of evidence to support TDM in certain therapeutic areas, there remain substantial challenges to widespread clinical implementation. There is a lack of consensus regarding the integration of TDM in clinical guidelines, and a lack of consensus on the cost-effectiveness of TDM; both factors contribute to the difficulty that healthcare providers face in obtaining reimbursement for TDM (both coverage of testing itself, and coverage of potential dosing modifications). The HEOR experts outlined alternative routes to obtaining reimbursement and suggested advocating for changes in coverage policies to promote TDM use in the clinic. Reaching alignment across policy makers, patients and advocacy groups, payers, and healthcare providers, on specific treatment settings where TDM will be clearly beneficial, was identified as an important step to advancing TDM implementation for the benefit of patients.

Objectives To assess the frequency and yield of retinal examination in children below 2 years old undergoing abuse evaluations in the setting of skull fracture(s) and small underlying intracranial hemorrhage. Methods This cross-sectional study used CAPNET, a multicenter child physical abuse network, to identify children below 2 years with a skull fracture(s) and intracranial injury limited to an underlying small focal intracranial hemorrhage undergoing subspeciality child abuse evaluations. Our outcomes of interest were (1) the performance of a retinal examination, (2) the identification of retinal hemorrhages, and (3) associations of clinical factors and CAPNET site with the performance of retinal examinations. We hypothesized that retinal hemorrhages would be identified in <5% of patients. Results Of 242 children who met inclusion criteria, the majority (189, 78.1%) presented with a reported history of accidental trauma, and most (211, 87.2%) lacked additional injuries. Only 9 (3.7%) had loss of consciousness and/or seizures/seizure-like activity. The majority (201, 83.1%) had low concern for abuse. Overall, 104 (43.0%) children underwent retinal examinations, of which 0 had retinal hemorrhages (one-sided 95% CI: 0-2.8%). Children without a reported accidental mechanism of injury ( P =0.004), those with intermediate/high concern for abuse ( P <0.001), and children with occipital fractures ( P =0.008) were more likely than their counterparts to undergo retinal examination. The proportion of children undergoing retinal examination varied by CAPNET site ( P <0.001). Conclusions Our findings suggest that it may be reasonable to forgo retinal examinations in children below 2 years of age with skull fracture(s) and intracranial injury limited to an underlying small focal hemorrhage who are overall neurologically well-appearing.

Background During adolescence, accurate diagnostic criteria and/or identification of adolescents “at risk” of polycystic ovary syndrome (PCOS) are critical to establish appropriate screening, treatment, and lifelong health plans. The 2023 International Evidence-Based Guideline for PCOS aimed to provide the most up-to-date evidence-based recommendations to improve health outcomes for individuals with PCOS, emphasizing accurate and timely diagnosis of PCOS from adolescence. Methods The best practice methods following the Appraisal of Guidelines for Research and Evaluation (AGREE-II) criteria were applied. Healthcare professionals and patients/consumers reviewed extensive evidence synthesis/meta-analysis for 55 prioritized clinical questions. Databases (OVID MEDLINE, All EBM, PsycInfo, EMBASE, CINAHL) were searched until August 2022 as part of the 2023 update of the Guideline. The Grading of Recommendations, Assessment, Development, and Evaluation (GRADE) framework guided experts on evidence quality, feasibility, acceptability, cost, implementation, and ultimately recommendation strength. Results This manuscript focuses on the adolescent-specific recommendations of the 2023 Guideline. The diagnosis is based on the presence of both irregular menstrual cycles (defined according to the time postmenarche) and clinical/biochemical hyperandrogenism following the exclusion of other disorders that mimic PCOS. Adolescents with only one of these features can be considered “at risk” of PCOS requiring the management of symptoms and ongoing follow-up. Polycystic ovarian morphology on pelvic ultrasonography or anti-Müllerian hormone levels should not be used for diagnosis during adolescence. Lifelong health planning is recommended to include healthy lifestyles, screening for depression and metabolic features and the transition to adult care, all underpinned by shared decision-making. Healthcare professionals should explain weight-related health risks to adolescents, while minimizing weight stigma. In adolescents with PCOS or “at risk” of PCOS, combined oral contraceptive pills are indicated for menstrual irregularity and clinical hyperandrogenism, focusing on low dose preparations, and metformin could be considered for metabolic features and cycle regulation. Overall, the evidence is limited in adolescents with PCOS, and recommendations are based on low to moderate certainty evidence. Conclusions Extensive international engagement and rigorous processes generated International Guideline diagnostic criteria for adolescents that differ from adult criteria and clarified appropriate screening and management strategies for PCOS during adolescence.

Can empirical research guide clinicians in how to conduct effective couple therapy? While we know that couple therapy works, understanding the mechanisms of action—how and why it works—has been the focus of research for several decades. Using Doss's framework for understanding the key components of the change process—therapy change processes, client change processes, mediators, and outcomes—we reviewed 48 quantitative couple therapy process studies over a 40‐year period. The results reveal a fragmented knowledge base. No single study examines the entire process of change, and although several findings show promise, none have been replicated. Additionally, only a limited number of hypothesized associations achieved statistical significance, with neither theory nor empirical evidence adequately explaining why some hypotheses were supported while others were not. Current couple therapy process research cannot yet guide clinicians on how to conduct effective therapy. Until it does, the divide between research and practice will persist.

Objective Neurodevelopmental follow-up programmes for children with CHD help identify neurodevelopmental impairments and support the delivery of recommended interventions. The Cardiac Neurodevelopmental Outcome Collaborative, Diversity, Equity, and Inclusion Special Interest Group surveyed members to assess perceived patient barriers to neurodevelopmental follow-up, previous diversity and inclusion education, and confidence in caring for historically marginalised populations. Methods A link to a Redcap online survey was emailed to Cardiac Neurodevelopmental Outcome Collaborative members on 23 April 2022, with 4 weeks given to complete. Results Eighty-four participants from 37 institutions in North America completed the survey. Respondents acknowledged that education on the importance of neurodevelopmental follow-up and frequent accommodations for cancellations or rescheduling clinic visits is essential. Language interpretation and written materials were available in languages other than English, but a limited number provided fully translated evaluation reports. Driving distance and the caregiver’s lack of understanding of the rationale for neurodevelopmental follow-up were the top perceived barriers to programme attendance. At the institutional level, training for cultural competency was typically provided, and most respondents felt comfortable caring for patients from diverse backgrounds. However, many agreed their programmes could do more to make evaluations accessible to historically marginalised/underserved populations. Conclusions Multiple barriers exist to cardiac neurodevelopmental follow-up, particularly for patients from under-represented minorities and for those whose primary language is not English. Surveying families will be valuable to understand how we may overcome these barriers. Further education about the importance of neurodevelopmental follow-up programmes continues to be essential.

Background: Necrotizing pancreatitis (NP), a severe form of pancreatitis characterized by necrosis of pancreatic tissue, is associated with a significant health care burden worldwide. In this study, we assess early readmissions of NP in the US. Methods: The National Readmission Database from 2016 to 2020 was utilized to identify all index and 30-day readmissions of NP in the US. Hospitalization characteristics, readmission rates, clinical outcomes, predictors of readmissions, and health care burden were assessed. P-values <0.05 were statistically significant. Results: From 2016 to 2020, 43,968 index admissions for NP were identified. Of which, 18.6% were readmitted within 30 days. There was a higher proportion of males on index and 30-day readmission. On readmission, NP was identified as the admitting diagnosis in only 27.64% of the patients, followed by pancreatitis without necrosis (17.7%), sepsis (8.8%), pancreatic pseudocyst (6.85%), and chronic pancreatitis (2.5%). Biliary pancreatitis (aHR 1.46, 95% CI 1.30-1.65, P<0.001), idiopathic pancreatitis (aHR 1.45, 95% CI 1.33-1.57, P<0.001), and other etiologies of pancreatitis (aHR 1.74, 95% CI 1.46-2.06, P<0.001) had a higher risk of 30-day readmission compared with alcohol-induced pancreatitis. We noted lower inpatient mortality (2.11 vs. 2.97%, aOR:0.65, 95% CI 0.50-0.83, P=0.001), mean length of stay (7.36 vs. 10.97 days, mean difference 3.86, 95% CI 4.25-3.48, P<0.001), and total hospitalization charges ($75,282 vs.$125,480, mean difference $53,979, 95$59,417-$48,541, P<0.001) for 30-day readmissions compared with index NP hospitalizations. Conclusion: About one-fifth of NP patients were readmitted within 30 days. However, these patients had lower mortality and health care burden compared with index hospitalization.

Importance The impact of awake prone positioning (APP) on clinical outcomes in patients with COVID-19 and acute hypoxemic respiratory failure (AHRF) remains uncertain. Objective To assess the association of APP with improved clinical outcomes among patients with COVID-19 and AHRF, and to identify potential effect modifiers. Data Sources PubMed, Embase, the Cochrane Library, and ClinicalTrials.gov were searched through August 1, 2024. Study Selection Randomized clinical trials (RCTs) examining APP in adults with COVID-19 and AHRF that reported intubation rate or mortality were included. Data Extraction and Synthesis Individual participant data (IPD) were extracted according to PRISMA-IPD guidelines. For binary outcomes, logistic regression was used and odds ratio (OR) and 95% CIs were reported, while for continuous outcomes, linear regression was used and mean difference (MD) and 95% CIs were reported. Main Outcomes and Measures The primary outcome was survival without intubation. Secondary outcomes included intubation, mortality, death without intubation, death after intubation, escalation of respiratory support, intensive care unit (ICU) admission, time from enrollment to intubation and death, duration of invasive mechanical ventilation, and hospital and ICU lengths of stay. Results A total of 14 RCTs involving 3019 patients were included; 1542 patients in the APP group (mean [SD] age, 59.3 [14.1] years; 1048 male [68.0%]) and 1477 in the control group (mean [SD] age, 59.9 [14.1] years; 979 male [66.3%]). APP improved survival without intubation (OR, 1.42; 95% CI, 1.20-1.68), and it reduced the risk of intubation (OR, 0.70; 95% CI, 0.59-0.84) and hospital mortality (OR, 0.77; 95% CI, 0.63-0.95). APP also extended the time from enrollment to intubation (MD, 0.93 days; 95% CI, 0.43 to 1.42 days). In exploratory subgroup analyses, improved survival without intubation was observed in patients younger than age 68 years, as well as in patients with a body mass index of 26 to 30, early implementation of APP (ie, less than 1 day from hospitalization), a pulse saturation to inhaled oxygen fraction ratio of 155 to 232, respiratory rate of 20 to 26 breaths per minute (bpm), and those receiving advanced respiratory support at enrollment. However, none of the subgroups had significant interaction with APP treatment. APP duration 10 or more hours/d within the first 3 days was associated with increased survival without intubation (OR, 1.85; 95% CI, 1.37-2.49). Conclusions and Relevance This IPD meta-analysis found that in adults with COVID-19 and AHRF, APP was associated with increased survival without intubation and with reduced risks of intubation and mortality, including death after intubation. Prolonged APP duration (10 or more hours/d) was associated with better outcomes.

Objective Dystonia is a movement disorder defined by involuntary muscle contractions leading to abnormal postures or twisting and repetitive movements. Classically dystonia has been thought of as a disorder of the basal ganglia, but newer results in idiopathic dystonia and lesion‐induced dystonia in adults point to broader motor network dysfunction spanning the basal ganglia, cerebellum, premotor cortex, sensorimotor, and frontoparietal regions. It is unclear whether a similar network is shared between different etiologies of pediatric lesion‐induced dystonia. Methods Three cohorts of pediatric patients with lesion‐induced dystonia were identified. The lesion etiologies included hypoxia, kernicterus, and stroke versus comparison subjects with acquired lesions not associated with dystonia. Multivariate lesion‐symptom mapping and lesion network mapping were used to evaluate the anatomy and networks associated with dystonia. Results Multivariate lesion‐symptom mapping showed that lesions of the putamen and globus pallidus were associated with dystonia ( r = 0.41, p < 0.001). Lesion network mapping using normative connectome data from healthy children demonstrated that these regional findings occurred within a common brain‐wide network that involves the basal ganglia, anterior and medial cerebellum, and cortical regions that overlap the cingulo‐opercular action‐mode and somato‐cognitive‐action networks. Interpretation We interpret these findings as novel evidence for a unified dystonia brain network that involves the somato‐cognitive‐action network, which is implicated in the coordination of movement. Elucidation of this network gives insight into the functional origins of dystonia and provides novel targets to investigate for therapeutic intervention. ANN NEUROL 2025