Implementing Entrustable Professional Activities in Pediatric Fellowships: Facilitating the Process.

BACKGROUND AND OBJECTIVES: Entrustable professional activities (EPAs) will be used for initial certification by the American Board of Pediatrics by 2028. Less than half of pediatric fellowships currently use EPAs for assessment, yet all will need to adopt them. Our objectives were to identify facilitators and barriers to the implementation of EPAs to assess pediatric fellows and to determine fellowship program directors' (FPD) perceptions of EPAs and Milestones. METHODS: We conducted a survey of FPDs from 15 pediatric subspecialties. EPA users were asked about their implementation of EPAs, barriers encountered, and perceptions of EPAs. Nonusers were queried about deterrents to using EPAs. Both groups were asked about potential facilitators of implementation and their perceptions of Milestones. RESULTS: The response rate was 65% (575/883). Of these, 344 (59.8%) were EPA users and 231 (40.2%) were nonusers. Both groups indicated work burden as a barrier to implementation. Nonusers reported more barriers than users (mean [SD]: 7 [3.8] vs 5.8 [3.4], P < .001). Both groups identified training materials and premade assessment forms as facilitators to implementation. Users felt that EPAs were easier to understand than Milestones (89%) and better reflected what it meant to be a practicing subspecialty physician (90%). In contrast, nonusers felt that Milestones were easy to understand (57%) and reflected what it meant to be a practicing subspecialist (58%). CONCLUSIONS: Implementing EPA-based assessment will require a substantial investment by FPDs, facilitated by guidance and easily accessible resources provided by multiple organizations. Perceived barriers to be addressed include FPD time constraints, a need for additional assessment tools, and outcomes data.

Exploring Factors for Implementation of EPAs in Pediatric Subspecialty Fellowships: A Qualitative Study of Program Directors.

OBJECTIVE: To understand fellowship program directors' (FPDs) perspectives on facilitators and barriers to using entrustable professional activities (EPAs) in pediatric subspecialty training. METHODS: We performed a qualitative study of FPDs, balancing subspecialty, program size, geographic region and current uses of EPAs. A study coordinator conducted 1-on-1 interviews using a semistructured approach to explore EPA use or nonuse and factors supporting or preventing their use. Investigators independently coded transcribed interviews using an inductive approach and the constant comparative method. Group discussion informed code structure development and refinement. Iterative data collection and analysis continued until theoretical sufficiency was achieved, yielding a thematic analysis. RESULTS: Twenty-eight FPDs representing 11 pediatric subspecialties were interviewed, of whom 16 (57%) reported current EPA use. Five major themes emerged: (1) facilitators including the intuitive nature and simple wording of EPAs; (2) barriers such as workload burden and lack of a regulatory requirement; (2) variable knowledge and training surrounding EPAs, leading to differing levels of understanding; (3) limited current use of EPAs, even among self-reported users; and (4) complementary nature of EPAs and milestones. FPDs acknowledged the differing strengths of both EPAs and milestones but sought additional knowledge about the value added by EPAs for assessing trainees, including the impact on outcomes. CONCLUSIONS: Identified themes can inform effective and meaningful EPA implementation strategies: Supporting and educating FPDs, ongoing assessment of the value of EPAs in training, and practical integration with current workflow. Generating additional data and engaging stakeholders is critical for successful implementation for the pediatric subspecialties.

Relationship between epa level of supervision with their associated subcompetency milestone levels in pediatric fellow assessment.

BACKGROUND: Entrustable Professional Activities (EPA) and competencies represent components of a competency-based education framework. EPAs are assessed based on the level of supervision (LOS) necessary to perform the activity safely and effectively. The broad competencies, broken down into narrower subcompetencies, are assessed using milestones, observable behaviors of one's abilities along a developmental spectrum. Integration of the two methods, accomplished by mapping the most relevant subcompetencies to each EPA, may provide a cross check between the two forms of assessment and uncover those subcompetencies that have the greatest influence on the EPA assessment. OBJECTIVES: We hypothesized that 1) there would be a strong correlation between EPA LOS ratings with the milestone levels for the subcompetencies mapped to the EPA; 2) some subcompetencies would be more critical in determining entrustment decisions than others, and 3) the correlation would be weaker if the analysis included only milestones reported to the Accreditation Council for Graduate Medical Education (ACGME). METHODS: In fall 2014 and spring 2015, the Subspecialty Pediatrics Investigator Network asked Clinical Competency Committees to assign milestone levels to each trainee enrolled in a pediatric fellowship for all subcompetencies mapped to 6 Common Pediatric Subspecialty EPAs as well as provide a rating for each EPA based upon a 5-point LOS scale. RESULTS: One-thousand forty fellows were assessed in fall and 1048 in spring, representing about 27% of all fellows. For each EPA and in both periods, the average milestone level was highly correlated with LOS (rho range 0.59-0.74; p < 0.001). Correlations were similar when using a weighted versus unweighted milestone score or using only the ACGME reported milestones (p > 0.05). CONCLUSIONS: We found a strong relationship between milestone level and EPA LOS rating but no difference if the subcompetencies were weighted, or if only milestones reported to the ACGME were used. Our results suggest that representative behaviors needed to effectively perform the EPA, such as key subcompetencies and milestones, allow for future language adaptations while still supporting the current model of assessment. In addition, these data provide additional validity evidence for using these complementary tools in building a program of assessment.

Premature adrenarche in Prader-Willi syndrome is associated with accelerated pre-pubertal growth and advanced bone age.

OBJECTIVES: Prader-Willi syndrome (PWS) is characterized by obesity, growth hormone deficiency, hypogonadism, and a high prevalence of premature adrenarche despite reported hypothalamic-pituitary-adrenal axis dysfunction. While idiopathic premature adrenarche is associated with accelerated pre-pubertal growth and advanced bone age, the consequences of elevated adrenal androgens on growth and bone maturation in PWS remain unknown. This study therefore sought to describe age-related changes in dehydroepiandrosterone sulfate (DHEAS) and their effects on growth and bone maturation in PWS. METHODS: This retrospective observational study included 62 children with PWS. Simple and multiple regression models were constructed to relate age and BMI-SDS with DHEAS levels. Height velocity was compared to age and sex-based norms with t-tests and two-way ANOVA. Patterns in bone age Z-score were examined with two-way ANOVA, and the contributions of age, BMI-SDS, and DHEAS to bone age Z-score were analyzed with multiple regression. RESULTS: DHEAS levels rose earlier and were less strongly correlated with age in males and females with PWS (R2=0.12 and 0.30) compared to healthy controls (R2=0.89 and 0.88) in a pattern unrelated to BMI-SDS (adjusted R2=0.076, p=0.10 for age, and 0.29 for BMI-SDS). Mid-childhood height velocity was increased in males and preserved in females with PWS before declining at the age of expected puberty (p<0.0001). Peri-adrenarchal bone age was advanced in a manner associated with DHEAS but not BMI-SDS (p<0.0001; adjusted R2=0.48, p=0.0014 for DHEAS, and 0.78 for BMI-SDS). CONCLUSIONS: An obesity-independent increase in adrenal androgens is associated with accelerated mid-childhood growth and bone maturation in PWS.

Clinical competency committee perceptions of entrustable professional activities and their value in assessing fellows: A qualitative study of pediatric subspecialty program directors.

OBJECTIVES: To examine the composition and processes of Clinical Competency Committees (CCCs) assigning entrustable professional activity (EPA) levels of supervision for pediatric subspecialty fellows and to examine fellowship program director (FPD) perspectives about using EPAs to determine fellows' graduation readiness. METHODS: A qualitative study was performed using one-on-one interviews with a purposeful sample of pediatric subspecialty FPDs to yield a thematic analysis. Semi-structured interview guides were used for participants who self-identified as EPA users or non-users. Inductive analysis and coding were performed on transcripts until theoretical sufficiency was attained. RESULTS: Twenty-eight FPDs were interviewed. There was significant variability in the composition and processes of CCCs across subspecialties. FPDs felt that CCCs intuitively understand what entrustment means, allowing for ease of application of level of supervision (LOS) scales and consensus. FPDs perceived that EPAs provided a global assessment of fellows and are one tool to determine graduation readiness. CONCLUSIONS: Although there was variability in the makeup and processes of CCCs across subspecialties, FPDs believe EPAs are intuitive and relatively easy to implement. Consensus can be reached easily using EPA-specific LOS scales focusing on entrustment. FPDs desire a better understanding of how EPAs should be used for graduation.

Linking oxytocin and arginine vasopressin signaling abnormalities to social behavior impairments in Prader-Willi syndrome.

Prader-Willi syndrome (PWS) is a genetic neurodevelopmental disorder. Global hypothalamic dysfunction is a core feature of PWS and has been implicated as a driver of many of PWS's phenotypic characteristics (e.g., hyperphagia-induced obesity, hypogonadism, short stature). Although the two neuropeptides (i.e., oxytocin [OXT] and arginine vasopressin [AVP]) most implicated in mammalian prosocial functioning are of hypothalamic origin, and social functioning is markedly impaired in PWS, there has been little consideration of how dysregulation of these neuropeptide signaling pathways may contribute to PWS's social behavior impairments. The present article addresses this gap in knowledge by providing a comprehensive review of the preclinical and clinical PWS literature-spanning endogenous neuropeptide measurement to exogenous neuropeptide administration studies-to better understand the roles of OXT and AVP signaling in this population. The preponderance of evidence indicates that OXT and AVP signaling are indeed dysregulated in PWS, and that these neuropeptide pathways may provide promising targets for therapeutic intervention in a patient population that currently lacks a pharmacological strategy for its debilitating social behavior symptoms.

Fellow Entrustment for the Common Pediatric Subspecialty Entrustable Professional Activities Across Subspecialties.

OBJECTIVE: To determine the relationship between level of supervision (LOS) ratings for the Common Pediatric Subspecialty Entrustable Professional Activities (EPAs) with their associated subcompetency milestones across subspecialties and by fellowship training year. METHODS: Clinical Competency Committees (CCCs) in 14 pediatric subspecialties submitted LOS ratings for 6 Common Subspecialty EPAs and subcompetency milestone levels mapped to these EPAs. We examined associations between these subcompetency milestone levels and LOS ratings across subspecialty training year by fitting per-EPA linear mixed effects models, regressing LOS rating on milestone level and on training year. RESULTS: CCCs from 211 pediatric fellowship programs provided data for 369 first, 336 second, and 331 third year fellows. Mean subcompetency milestone levels increased similarly among subspecialties for most EPAs compared with the reference, Adolescent Medicine. Mean subcompetency milestones mapped to each EPA and mean EPA LOS ratings generally increased by training year across all subspecialties. CONCLUSIONS: Subcompetency milestones levels mapped to each Common Subspecialty EPA and the EPA LOS ratings increase similarly across subspecialties and by training year, providing validity evidence for using EPA LOS to assess pediatric subspecialty trainee performance. This study supports the development of tools to facilitated the CCC evaluation process across all pediatric subspecialties.

Achieving Entrustable Professional Activities During Fellowship.

BACKGROUND AND OBJECTIVES: Entrustable Professional Activities (EPAs) were developed to assess pediatric fellows. We previously showed that fellowship program directors (FPDs) may graduate fellows who still require supervision. How this compares with their expectations for entrustment of practicing subspecialists is unknown. METHODS: We surveyed US FPDs in 14 pediatric subspecialties through the Subspecialty Pediatrics Investigator Network between April and August 2017. For each of 7 common pediatric subspecialty EPAs, we compared the minimum level of supervision that FPDs required for graduation with the level they expected of subspecialists for safe and effective practice using the Friedman rank sum test and paired t test. We compared differences between subspecialties using linear regression. RESULTS: We collected data from 660 FPDs (response rate 82%). For all EPAs, FPDs did not require fellows to reach the level of entrustment for graduation that they expected of subspecialists to practice (P < .001). FPDs expected the least amount of supervision for the EPAs consultation and handovers. Mean differences between supervision levels for graduation and practice were smaller for clinical EPAs (consultation, handovers, lead a team) when compared with nonclinical EPAs (quality improvement, management, lead the profession and scholarship; P = .001) and were similar across nearly all subspecialties. CONCLUSIONS: Fellowship graduates may need continued development of clinical and nonclinical skills in their early practice period, underscoring a need for continued assessment and mentoring. Graduation readiness must be based on clear requirements, with alignment of FPD expectations and regulatory standards, to ensure quality care for patients.

Agreement of Program Directors With Clinical Competency Committees for Fellow Entrustment.

OBJECTIVES: Fellowship program directors (FPD) and Clinical Competency Committees (CCCs) both assess fellow performance. We examined the association of entrustment levels determined by the FPD with those of the CCC for 6 common pediatric subspecialty entrustable professional activities (EPAs), hypothesizing there would be strong correlation and minimal bias between these raters. METHODS: The FPDs and CCCs separately assigned a level of supervision to each of their fellows for 6 common pediatric subspecialty EPAs. For each EPA, we determined the correlation between FPD and CCC assessments and calculated bias as CCC minus FPD values for when the FPD was or was not a member of the CCC. In addition, we examined the effect of program size, FPD understanding of EPAs, and subspecialty on the correlations. Data were obtained in fall 2014 and spring 2015. RESULTS: A total of 1040 fellows were assessed in the fall and 1048 in the spring. In both periods and for each EPA, there was a strong correlation between FPD and CCC supervision levels (P < .001). The correlation was somewhat lower when the FPD was not a CCC member (P < .001). Overall bias in both periods was small. CONCLUSIONS: The correlation between FPD and CCC assignment of EPA supervision levels is strong. Although slightly weaker when the FPD is not a CCC member, bias is small, so this is likely unimportant in determining fellow entrustment level. The similar performance ratings of FPDs and CCCs support the validity argument for EPAs as competency-based assessment tools.

Improving pediatric endocrinology trainees' knowledge about insulin pumps and continuous glucose monitors with online spaced education: Technology Knowledge Optimization in T1D (TeKnO T1D).

OBJECTIVE: We explored the impact of TeKnO T1D, an online, case-based, spaced education curriculum about insulin pump and continuous glucose monitor (CGM) use in pediatric type 1 diabetes management. METHODS: Pediatric endocrinology fellows (n = 64) were randomized to receive an educational curriculum focused on either insulin pumps or CGMs. Fellows received interactive questions twice weekly via email or mobile app. Median time to completion was 76.5 days. The primary outcome was change in knowledge as measured by performance on multiple-choice questions (MCQ) from the pre-test to the post-test. RESULTS: Forty-eight of 64 (75%) learners completed the curriculum and assessments. The pump group improved from 35.0 ± 15% on the pre-test MCQs to 61.1 ± 17% on the post-test, a 12.2 absolute percentage point greater improvement on pump-specific items than the CGM group (P = .03). The CGM group improved from 30.3 ± 15% on the pre-test MCQs to 61.4 ± 21% on the post-test, a 28.7 absolute percentage point greater improvement on CGM-specific items than the pump group (P < .001). Both groups were more likely to report an appropriate level of understanding of their respective technologies after completing the corresponding curriculum. In thematic analysis of qualitative data, fellows indicated that knowledge gains led to improved patient care. There was universal agreement about enjoyment and effectiveness of the curricula. CONCLUSIONS: TeKnO T1D proved to be an engaging, effective way to improve endocrinology fellows' knowledge and confidence about insulin pumps and CGM use in the management of pediatric type 1 diabetes.

Prader Willi syndrome: endocrine updates and new medical therapies.

PURPOSE OF REVIEW: Prader Willi syndrome is characterized not only by hyperphagia frequently resulting in obesity, but also by endocrine dysfunction across a variety of axes. This article reviews the most recent literature regarding possible causes of hyperphagia and the nature of endocrinopathies seen in Prader Willi syndrome, as well as current research into possible therapies. RECENT FINDINGS: Investigation into neurologic, metabolic and hormonal drivers of hyperphagia and obesity has revealed new insights and clarified underlying pathophysiology. Additional studies continue to elucidate the hormonal deficiencies seen in the syndrome, allowing for improvements in clinical care. SUMMARY: The underlying causes of the hyperphagia and progressive obesity frequently seen in Prader Willi Syndrome are largely unknown and likely multifactorial. Understanding the hormonal and metabolic drivers at work in PWS, as well as the nature of other hormonal dysfunction seen in the syndrome is necessary to guide current management and future research directions.

Pediatric Endocrinology Trainees' Education and Knowledge About Insulin Pumps and Continuous Glucose Monitors.

BACKGROUND: Recent data demonstrating a lack of improvement in average hemoglobin A1c levels despite the increased use of insulin pumps and continuous glucose monitors (CGMs) suggest that patients are not using these technologies optimally. Suboptimal provider understanding of these devices may be a contributing factor. METHODS: We sought to assess fellows' knowledge, attitudes, and practices regarding insulin pumps and CGMs using a mixed-methods survey. We polled 42 pediatric endocrinology fellows and 69 attending physicians in pediatric endocrinology using items on a five-point Likert scale. RESULTS: Perceived fellow knowledge of insulin pumps and CGMs was only 3.6 ± 1.0 and 3.6 ± 0.9, respectively. Despite consensus about the need for pediatric endocrinologists to understand these technologies, only 14.7% of fellows reported the presence of a formal curriculum about these technologies at their institutions. Potential gaps identified in fellows' knowledge include general use and troubleshooting, advanced insulin pump features, infusion sets and dermatological complications, systematic approach to data, interpretation and application of CGM data, and keeping pace with technological advances. CONCLUSIONS: Our data suggest suboptimal fellow knowledge and understanding of insulin pumps and CGMs in pediatric type 1 diabetes management, which is likely attributable to inadequate education in fellowship training programs. The development of formal educational curricula targeting areas of weakness identified in this survey may improve clinician knowledge of these technologies and ultimately impact patient education and care.

The High Direct Medical Costs of Prader-Willi Syndrome.

OBJECTIVE: To assess medical resource utilization associated with Prader-Willi syndrome (PWS) in the US, hypothesized to be greater relative to a matched control group without PWS. STUDY DESIGN: We used a retrospective case-matched control design and longitudinal US administrative claims data (MarketScan) during a 5-year enrollment period (2009-2014). Patients with PWS were identified by Classification of Diseases, Ninth Revision, Clinical Modification diagnosis code 759.81. Controls were matched on age, sex, and payer type. Outcomes included total, outpatient, inpatient and prescription costs. RESULTS: After matching and application of inclusion/exclusion criteria, we identified 2030 patients with PWS (1161 commercial, 38 Medicare supplemental, and 831 Medicaid). Commercially insured patients with PWS (median age 10 years) had 8.8-times greater total annual direct medical costs than their counterparts without PWS (median age 10 years: median costs $14 907 vs $819; P < .0001; mean costs: $28 712 vs $3246). Outpatient care comprised the largest portion of medical resource utilization for enrollees with and without PWS (median $5605 vs $675; P < .0001; mean $11 032 vs $1804), followed by mean annual inpatient and medication costs, which were $10 879 vs $1015 (P < .001) and $6801 vs $428 (P < .001), respectively. Total annual direct medical costs were ∼42% greater for Medicaid-insured patients with PWS than their commercially insured counterparts, an increase partly explained by claims for Medicaid Waiver day and residential habilitation. CONCLUSION: Direct medical resource utilization was considerably greater among patients with PWS than members without the condition. This study provides a first step toward quantifying the financial burden of PWS posed to individuals, families, and society.

Fellows as Teachers: Raising the Educational Bar.

Fellows are expected to educate trainees, peers, and patients, during and long after fellowship. However, there has been relatively little emphasis on the acquisition of teaching skills in fellowship programs. Challenges to teaching by fellows during subspecialty training include demanding clinical duties, their limited knowledge base in the field, brief contact time with learners during consultative roles, and, for new fellows, personal unfamiliarity with the learners and hospital culture. Fellows' teaching skills can be improved by formal curricula addressing teaching, and by direct observation and feedback of teaching akin to what is provided for learning clinical care. Further expansion of fellow-as-teacher programs will allow in-depth training for fellows seeking careers as medical educators. Even without such dedicated programs, emphasis on honing teaching skills during fellowship will telegraph the importance of teaching and help evolve divisional culture. Such efforts can have a positive impact on patients and learners, and enhance the teaching skills of future faculty.

Altered hypothalamic-pituitary-ovarian axis function in young female athletes: implications and recommendations for management.

Young women have become increasingly active in athletics during the 20th century. Those involved in sports that emphasize lean body type are at high risk for the development of menstrual dysfunction, including amenorrhea. This is mediated by an alteration in function of the hypothalamic-pituitary-ovarian (HPO) axis, with loss of normal secretion of luteinizing hormone, and subsequent lack of estrogen production. Disruption of the HPO axis appears to be dependent on the body's recognition of an energy imbalance, which may be due to a lack of compensatory caloric intake in the face of significant energy expenditure. Other pituitary hormones, such as triiodothyronine, growth hormone, and insulin-like growth factor-1 may also be affected. These metabolic changes have an impact on bone mineralization during a critical period in the development of bone mass. Recognition by physicians of the so-called 'female athlete triad', consisting of disordered eating, amenorrhea, and osteoporosis, may allow therapeutic intervention. Diagnosis of eating disorders and decreased bone mineral density can have significant impact on the health of the young athlete. Treatment is aimed at restoring normal menstrual function by increasing caloric intake to balance the increased energy demands of athletic participation. Concurrent treatment of the hypoestrogenemic state using estrogen replacement is controversial, but may aid in alleviating further loss of bone mass.

Adolescent androgen abnormalities.

PURPOSE OF REVIEW: Polycystic ovary syndrome is one of the most common endocrinopathies affecting premenopausal women. This review focuses on this major cause of hyperandrogenism in adolescents and young women, highlighting new diagnostic and therapeutic strategies that are under investigation. The pathophysiologic role in the disorder are the subject of several recent reports. RECENT FINDINGS: Recent studies have found a 33% prevalence of abnormal glucose tolerance in a cohort of affected adolescents, higher fasting insulin levels and lower insulin sensitivity, and that glucose tolerance testing appears to be necessary for routine screening. The effects of hyperinsulinism may be counteracted by insulin sensitizing agents. In adult women with polycystic ovary syndrome, metformin treatment reduced hyperinsulinemia and hyperandrogenemia. In some obese adolescents, metformin therapy resulted in declines in body mass index, insulin, and glucose. Restoration of regular menses may also occur after metformin treatment. Thus, data is accumulating that insulin-sensitizing agents may be helpful in decreasing the pathophysiologic effects of hyperinsulinism and insulin resistance associated with polycystic ovary syndrome. Other hormonal alterations in polycystic ovary syndrome have also been the subject of recent reports. Leptin secretion was found to be markedly irregular in these women. Elevated LH secretion may be secondary to accelerated gonadotropin-releasing hormone pulse generator activity, although the etiology of the pulse alterations is unclear. SUMMARY: Although polycystic ovary syndrome is the most common endocrine disorder affecting young women, it is one of the least understood, reflected by the wealth of research in this area. One area of focus has been the pathophysiologic link between insulin resistance and this disorder, including the effects of promising new agents to counteract these effects.