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.

Improving Clinical Trials for Anticomplement Therapies in Complement-Mediated Glomerulopathies: Report of a Scientific Workshop Sponsored by the National Kidney Foundation.

Blocking the complement system as a therapeutic strategy has been proposed for numerous glomerular diseases but presents myriad questions and challenges, not the least of which is demonstrating efficacy and safety. In light of these potential issues and because there are an increasing number of anticomplement therapy trials either planned or under way, the National Kidney Foundation facilitated an all-virtual scientific workshop entitled "Improving Clinical Trials for Anti-Complement Therapies in Complement-Mediated Glomerulopathies." Attended by patient representatives and experts in glomerular diseases, complement physiology, and clinical trial design, the aim of this workshop was to develop standards applicable for designing and conducting clinical trials for anticomplement therapies across a wide spectrum of complement-mediated glomerulopathies. Discussions focused on study design, participant risk assessment and mitigation, laboratory measurements and biomarkers to support these studies, and identification of optimal outcome measures to detect benefit, specifically for trials in complement-mediated diseases. This report summarizes the discussions from this workshop and outlines consensus recommendations.

Extent of hypertension and renal injury in children surviving acute lymphoblastic leukemia.

BACKGROUND: Childhood acute lymphoblastic leukemia (ALL) fortunately has high survival rates, and understanding longer term implications of therapy is critical. In this study, we aimed to investigate kidney health outcomes by assessing the prevalence of renal dysfunction and hypertension (HTN) in children with ALL at 1-5 years after ALL diagnosis. METHODS: This was a single-center, cross-sectional study of children with ALL who were 1-5 years post diagnosis. Glomerular filtration rate (GFR) measurements were calculated, and urine samples were collected to assess for protein/creatinine and albumin/creatinine. Blood pressure (BP) was determined by standard oscillometric technique, and children ≥6 years of age were eligible for ambulatory blood pressure monitoring (ABPM). RESULTS: Forty-five patients enrolled in the study, and 21 completed ABPMs. Fifteen patients (33%, 95% CI: 20%-49%) developed acute kidney injury (AKI) at least once. Thirteen (29%, 95% CI: 16%-44%) had hyperfiltration, and 11 (24%) had abnormal proteinuria and/or albuminuria. Prevalence of HTN based on clinic measurements was 42%. In the 21 ABPM patients, 14 had abnormal results (67%, 95% CI: 43%-85%), with the majority (11/21) demonstrating abnormal nocturnal dipping pattern. CONCLUSIONS: Among children with ALL, there is a high prevalence of past AKI. The presence of hyperfiltration, proteinuria, and/or albuminuria at 1-5 years after ALL diagnosis suggests real risk of developing chronic kidney disease (CKD) over time. There is a high prevalence of HTN on casual BP readings and even higher prevalence of abnormal ABPM in this group. The high prevalence of impaired nocturnal dipping by ABPM indicates an increased risk for future cardiovascular or cerebral ischemic events.

Pediatric CKD-MBD: existing and emerging treatment approaches.

The effects of bone and mineral metabolism on skeletal formation, as well as vascular and soft tissue calcifications, define chronic kidney disease-metabolic bone disease (CKD-MBD). Treatment recommendations center on establishing adequate vitamin D status, phosphate control through diet restriction and phosphate binders, and the use of vitamin D analogs for specific indications. Several emerging bone-promoting therapies have now been studied in adults with CKD, including bisphosphonates and denosumab. These approaches are associated with improved bone mass and, in some cases, decreased fracture rates in adults with CKD-MBD and are of potential interest for some children with CKD-MBD. In children with CKD and immobilization and/or muscle weakness, bisphosphonates appear to be an effective treatment to increase bone mass; baseline assessment and careful monitoring of bone density and/or bone biopsy findings are important in consideration of any new bone therapies for children with CKD-MBD.

Defining kidney outcomes in children with acute lymphoblastic leukemia in the modern era.

BACKGROUND: To define the incidence of acute kidney injury (AKI), chronic kidney disease (CKD), and hypertension (HTN) in pediatric patients diagnosed with acute lymphoblastic leukemia (ALL) over a recent 9-year period. METHODS: This study is a retrospective cohort study of all pediatric patients diagnosed with ALL at Nationwide Children's Hospital from January 1, 2008, to December 31, 2016. Patient demographic and clinical data including serum creatinine and blood pressure were collected at diagnosis up to 9 years post diagnosis. RESULTS: A total of 222 patients were identified for this study. The overall incidence of AKI in our cohort was high, with 101 subjects (45.5%, CI 38.8-52.3%) developing AKI at least once. CKD status could only be determined in 214 patients due to limited later GFR data. The incidence of CKD was low with only 5 of 214 patients developing CKD (2.3%, CI: 0.8-5.4%). The overall incidence of HTN at diagnosis was 45.6% (95% CI: 59.1-72%), and at 1 month post diagnosis was 65.8% (95% CI: 59.1-72.0%). Chronic HTN could only be determined in 216 patients due to limited blood pressure data. Chronic HTN was noted in 34.3% of patients (74/216, 95% CI: 28-41%). CONCLUSIONS: Among children with ALL, the incidence of AKI is relatively high at the time of diagnosis. However, development of CKD is relatively rare, suggesting good mid-term kidney prognosis. There is a high incidence of HTN at the time of diagnosis, 1 month post diagnosis, and chronic HTN that often goes untreated. A higher resolution version of the Graphical abstract is available as Supplementary information.

Low albumin levels are independently associated with neonatal acute kidney injury: a report from AWAKEN Study Group.

BACKGROUND: Data from adult and pediatric literature have shown an association between albumin levels and AKI. Whether hypoalbuminemia and neonatal AKI are associated has not been studied. METHODS: We evaluated the association of albumin with early (during the first postnatal week) and late (after the first postnatal week) AKI for 531 neonates from the Assessment of Worldwide AKI Epidemiology in Neonates (AWAKEN) database and for 3 gestational age (GA) subgroups: < 29, 29 to < 36, and ≥ 36 weeks GA. RESULTS: Low albumin levels were associated with increased odds of neonatal AKI; for every 0.1 g/dL decrease in albumin, the odds of late AKI increased by 12% on continuous analysis. After adjustment for potential confounders, neonates with albumin values in the lowest quartiles (< 2.2 g/dL) had an increased odds of early [Adjusted Odd Ratio (AdjOR) 2.5, 95% CI = 1.1-5.3, p < 0.03] and late AKI [AdjOR 13.4, 95% CI = 3.6-49.9, p < 0.0001] compared to those with albumin in the highest quartile (> 3.1 g/dL). This held true for albumin levels 2.3 to 2.6 g/dL for early [AdjOR 2.5, 95% CI = 1.2-5.5, p < 0.02] and late AKI [AdjOR 6.4, 95% CI = 1.9-21.6, p < 0.01]. Albumin quartiles of (2.7 to 3.0 g/dL) were associated with increased odds of late AKI. Albumin levels of 2.6 g/dL and 2.4 g/dL best predicted early (AUC = 0.59) and late AKI (AUC = 0.64), respectively. Analysis of albumin association with AKI by GA is described. CONCLUSIONS: Low albumin levels are independently associated with early and late neonatal AKI. Albumin could be a potential modifiable risk factor for neonatal AKI.

Results of the PROPINE randomized controlled trial: determining the ever-elusive target, the optimal plan for relapses of nephrotic syndrome in children.

Best treatments for initial presentation and relapses in children with nephrotic syndrome (NS) are still to be defined. The PROPINE study, published in this issue of Kidney International, demonstrates for relapse of childhood NS, the non-inferiority of a short taper (over 36 days) after remission with steroids. This study reinforces the need for more well-designed studies and the incorporation of predictive biomarkers, genetic studies, and other details to personalize treatment for each child with idiopathic NS.

Low hemoglobin levels are independently associated with neonatal acute kidney injury: a report from the AWAKEN Study Group.

BACKGROUND: Studies in adults showed a relationship between low hemoglobin (Hb) and acute kidney injury (AKI). We performed this study to evaluate this association in newborns. METHODS: We evaluated 1891 newborns from the Assessment of Worldwide AKI Epidemiology in Neonates (AWAKEN) database. We evaluated the associations for the entire cohort and 3 gestational age (GA) groups: <29, 29-<36, and ≥36 weeks' GA. RESULTS: Minimum Hb in the first postnatal week was significantly lower in neonates with AKI after the first postnatal week (late AKI). After controlling for multiple potential confounders, compared to neonates with a minimum Hb ≥17.0 g/dL, both those with minimum Hb ≤12.6 and 12.7-14.8 g/dL had an adjusted increased odds of late AKI (aOR 3.16, 95% CI 1.44-6.96, p = 0.04) and (aOR 2.03, 95% CI 1.05-3.93; p = 0.04), respectively. This association was no longer evident after controlling for fluid balance. The ability of minimum Hb to predict late AKI was moderate (c-statistic 0.68, 95% CI 0.64-0.72) with a sensitivity of 65.9%, a specificity of 69.7%, and a PPV of 20.8%. CONCLUSIONS: Lower Hb in the first postnatal week was associated with late AKI, though the association no longer remained after fluid balance was included. IMPACT: The current study suggests a possible novel association between low serum hemoglobin (Hb) and neonatal acute kidney injury (AKI). The study shows that low serum Hb levels in the first postnatal week are associated with increased risk of AKI after the first postnatal week. This study is the first to show this relationship in neonates. Because this study is retrospective, our observations cannot be considered proof of a causative role but do raise important questions and deserve further investigation. Whether the correction of low Hb levels might confer short- and/or long-term renal benefits in neonates was beyond the scope of this study.

Promoting bone health in children and adolescents following solid organ transplantation.

Solid organ transplantation in children and adolescents provides many benefits through improving critical organ function, including better growth, development, cardiovascular status, and quality of life. Unfortunately, bone status may be adversely affected even when overall status is improving, due to issues with pre-existing bone disease as well as medications and nutritional challenges inherent post-transplantation. For all children and adolescents, bone status entering adulthood is a critical determinant of bone health through adulthood. The overall health and bone status of transplant recipients benefits from attention to regular physical activity, good nutrition, adequate calcium, phosphorous, magnesium and vitamin D intake and avoidance/minimization of soda, extra sodium, and obesity. Many immunosuppressive agents, especially glucocorticoids, can adversely affect bone function and development. Minimizing exposure to "bone-toxic" medications is an important part of promoting bone health in children post-transplantation. Existing guidelines detail how regular monitoring of bone status and biochemical markers can help detect bone abnormalities early and facilitate valuable bone-directed interventions. Attention to calcium and vitamin D supplementation, as well as tapering and withdrawing glucocorticoids as early as possible after transplant, can provide best bone outcomes for these children. Dual-energy X-ray absorptiometry can be useful to detect abnormal bone mass and fracture risk in this population and newer bone assessment methods are being evaluated in children at risk for poor bone outcomes. Newer bone therapies being explored in adults with transplants, particularly bisphosphonates and the RANKL inhibitor denosumab, may offer promise for children with low bone mass post-transplantation.

Mechanisms and management of edema in pediatric nephrotic syndrome.

Edema is the abnormal accumulation of fluid in the interstitial compartment of tissues within the body. In nephrotic syndrome, edema is often seen in dependent areas such as the legs, but it can progress to cause significant accumulation in other areas leading to pulmonary edema, ascites, and/or anasarca. In this review, we focus on mechanisms and management of edema in children with nephrotic syndrome. We review the common mechanisms of edema, its burden in pediatric patients, and then present our approach and algorithm for management of edema in pediatric patients. The extensive body of experience accumulated over the last 5 decades means that there are many options, and clinicians may choose among these options based on their experience and careful monitoring of responses in individual patients.

Diuretic therapy and acute kidney injury in preterm neonates and infants.

BACKGROUND: Acute kidney injury (AKI) in preterm infants is associated with prolonged hospitalization and high mortality. Diuretic therapy has been used to enhance urine output in preterm infants with AKI. Treatment with diuretics, prescription patterns, and relationship with length of stay (LOS), mechanical ventilation (MV), and mortality in preterm infants who also had AKI have not been fully evaluated. METHODS: This multicenter retrospective study used the Pediatric Hospital Information System database. We included 2121 preterm infants with AKI diagnosis from 46 hospital Neonatal Intensive Care Units (NICUs) born <37 weeks gestational age (GA). Treatment with diuretics, practice patterns across 46 NICUs in the USA, and associated outcomes including LOS, MV, and mortality were evaluated. RESULTS: Seventy-six percent of infants received at least one dose of diuretics (median treatment 18 days). Diuretic prescription varied significantly across hospitals and ranged from 42 to 96%. Diuretics were used more frequently in infants with younger GA and smaller birth weight. Infants with older GA who received diuretics at or before 28 days postnatally had worse survival even after adjusting for known confounders. CONCLUSIONS: Preterm infants with AKI diagnosis were frequently treated with diuretics. Moreover, infants with younger GA and smaller birth weight were more likely to receive diuretics. Worse survival in infants with older GA who received diuretics could be the result of more underlying severe illness in these infants and not the cause of more severe illness. Prospective studies are needed to best determine patient safety and outcomes with diuretic treatment in preterm infants with AKI. A higher resolution version of the Graphical abstract is available as Supplementary information.

Describing Overarching Curricular Goals for Individualized Education.

Phenomenon: The phenomenon of individualized education, an essential component of competency-based medical education, addresses individual learner needs while working toward standardized learning outcomes. One challenge with broadly implementing individualized education is the lack of a pragmatic operational definition. To formalize expectations for individualized education, the Accreditation Council of Graduate Medical Education in 2013 began requiring six months of individualized curriculum (IC) during pediatric residency; however, there is not a national standard of formal curricular goals for the IC as an educational entity. Examining and describing the overarching curricular goals of IC could provide a framework for discourse about and further study of individualized education in medicine across disciplines and the continuum of medical education. Thus, we aimed to describe the phenomenon of individualized education through the lens of the goals of the IC in pediatric residency in the United States. Approach: In 2017, a purposeful sample of Pediatric Residency leaders were recruited to represent a diverse sample of program sizes, regions of the country, and importance of the IC to the program leadership. They completed an online survey with open-ended questions describing formal and implicit goals of their program's IC. The authors analyzed responses initially using conventional content analysis, then investigated whether the themes for program goals aligned with any existing educational theory. The concepts and language aligned with the principles of self-determination theory (SDT); therefore, the IC goals were subsequently grouped using the SDT domains of relatedness, autonomy, and competence. A focus group with a subset of survey respondents was conducted for member checking and elaboration of concepts. Findings: Program leaders from a diverse sample of 36 programs participated in the survey and a subset of 11 programs participated in the focus group. The common goals across all programs are listed in parentheses and organized by domains of SDT: 1)Relatedness goals (engage in mentorship, select a career) cultivate resident's professional identity based on their desired future career path; 2)autonomy goals (create a learning plan, practice accountability) help residents plan their path; and fulfillment of their plans lead to 3)competence goals (develop targeted clinical skills and knowledge, ensure comprehensive exposure, address learning gaps), ensuring they have a comprehensive skill set for their chosen identity. Insights: This study provides a framework to describe the phenomenon of individualized education through the lens of IC curricular goals in pediatric residency. The goals for IC that emerged from this study serve as a pragmatic framework for implementation of individualized education. They provide a common language and structure to promote more rigorous and collaborative study of individualized education across programs, disciplines, and settings in medicine. They may function as a roadmap for learners to navigate educational activities and for programs to help shape the experiences of their learners and examine outcomes of individualized education in their programs. The framework can also help individual pediatric residency programs structure improvements to their IC. Disciplines beyond pediatrics may also use this framework to better structure elective experiences to capitalize on the benefits of individual education.

The impact of fluid balance on outcomes in premature neonates: a report from the AWAKEN study group.

BACKGROUND: We evaluated the epidemiology of fluid balance (FB) over the first postnatal week and its impact on outcomes in a multi-center cohort of premature neonates from the AWAKEN study. METHODS: Retrospective analysis of infants <36 weeks' gestational age from the AWAKEN study (N = 1007). FB was defined by percentage of change from birth weight. OUTCOME: Mechanical ventilation (MV) at postnatal day 7. RESULTS: One hundred and forty-nine (14.8%) were on MV at postnatal day 7. The median peak FB was 0% (IQR: -2.9, 2) and occurred on postnatal day 2 (IQR: 1,5). Multivariable models showed that the peak FB (aOR 1.14, 95% CI 1.10-1.19), lowest FB in first postnatal week (aOR 1.12, 95% CI 1.07-1.16), and FB on postnatal day 7 (aOR 1.10, 95% CI 1.06-1.13) were independently associated with MV on postnatal day 7. In a similar analysis, a negative FB at postnatal day 7 protected against the need for MV at postnatal day 7 (aOR 0.21, 95% CI 0.12-0.35). CONCLUSIONS: Positive peak FB during the first postnatal week and more positive FB on postnatal day 7 were independently associated with MV at postnatal day 7. Those with a negative FB at postnatal day 7 were less likely to require MV.

Health-related quality of life in glomerular disease.

There is scant literature describing the effect of glomerular disease on health-related quality of life (HRQOL). The Cure Glomerulonephropathy study (CureGN) is an international longitudinal cohort study of children and adults with four primary glomerular diseases (minimal change disease, focal segmental glomerulosclerosis, membranous nephropathy, and IgA nephropathy). HRQOL is systematically assessed using items from the Patient-Reported Outcomes Measurement Informative System (PROMIS). We assessed the relationship between HRQOL and demographic and clinical variables in 478 children and 1115 adults at the time of enrollment into CureGN. Domains measured by PROMIS items included global assessments of health, mobility, anxiety, fatigue, and sleep impairment, as well as a derived composite measure incorporating all measured domains. Multivariable models were created that explained 7 to 32% of variance in HRQOL. Patient-reported edema consistently had the strongest and most robust association with each measured domain of HRQOL in multivariable analysis (adjusted ß [95% CI] for composite PROMIS score in children, -5.2 [-7.1 to -3.4]; for composite PROMIS score in adults, -6.1 [-7.4 to -4.9]). Female sex, weight (particularly obesity), and estimated glomerular filtration rate were also associated with some, but not all, domains of HRQOL. Primary diagnosis, disease duration, and exposure to immunosuppression were not associated with HRQOL after adjustment. Sensitivity analyses and interaction testing demonstrated no significant association between disease duration or immunosuppression and any measured domain of HRQOL. Thus, patient-reported edema has a consistent negative association with HRQOL in patients with primary glomerular diseases, with substantially greater impact than other demographic and clinical variables.

The impact of fluid balance on outcomes in critically ill near-term/term neonates: a report from the AWAKEN study group.

BACKGROUND: In sick neonates admitted to the NICU, improper fluid balance can lead to fluid overload. We report the impact of fluid balance in the first postnatal week on outcomes in critically ill near-term/term neonates. METHODS: This analysis includes infants ≥36 weeks gestational age from the Assessment of Worldwide Acute Kidney injury Epidemiology in Neonates (AWAKEN) study (N = 645). Fluid balance: percent weight change from birthweight. PRIMARY OUTCOME: mechanical ventilation (MV) on postnatal day 7. RESULTS: The median peak fluid balance was 1.0% (IQR: -0.5, 4.6) and occurred on postnatal day 3 (IQR: 1, 5). Nine percent required MV at postnatal day 7. Multivariable models showed the peak fluid balance (aOR 1.12, 95%CI 1.08-1.17), lowest fluid balance in 1st postnatal week (aOR 1.14, 95%CI 1.07-1.22), fluid balance on postnatal day 7 (aOR 1.12, 95%CI 1.07-1.17), and negative fluid balance at postnatal day 7 (aOR 0.3, 95%CI 0.16-0.67) were independently associated with MV on postnatal day 7. CONCLUSIONS: We describe the impact of fluid balance in critically ill near-term/term neonates over the first postnatal week. Higher peak fluid balance during the first postnatal week and higher fluid balance on postnatal day 7 were independently associated with MV at postnatal day 7.

Treatment regimens by pediatric nephrologists in children with congenital nephrogenic diabetes insipidus: A MWPNC study
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BACKGROUND: Congenital nephrogenic diabetes insipidus (NDI) is a rare genetic disorder affecting urinary concentration. Clinicians have varied medication regimens as well as nutritional plan approaches for these children. MATERIALS AND METHODS: An electronic survey was distributed to member pediatric nephrologists of the Midwest Pediatric Nephrology Consortium via email (n = 179). Questions included types of drugs prescribed, factors contributing to drug choice, common drug combinations given, and dietary/failure to thrive interventions used. RESULTS: We analyzed results from 72 respondents (42% overall response rate). 72% treated only 1 - 3 patients with NDI per year, 12% treated 4 or more, and 17% had no NDI patients. Of providers treating NDI patients, almost all prescribed thiazides (93%), 62% prescribed amiloride, and 55% reported prescribing nonsteroidal anti-inflammatory drugs (NSAIDs) as part of their drug regimen. gastrointestinal (GI) and renal side effects (43%) were given as reasons for not prescribing indomethacin. For 70%, drug choice was determined by severity of failure to thrive (FTT). Physicians were asked to define the most common drug combinations they prescribed. 48% reported prescribing indomethacin with hydrochlorothiazide. 84% of respondents have a renal dietitian on staff, and half included appointments with a dietitian as part of FTT therapy. The most common intervention for FFT was gastrostomy tube placement (78%). CONCLUSION: Our results suggest consensus on the use of thiazides, while the use of indomethacin is limited by GI and renal side effect profile. Our results revealed that multiple drug combinations are frequently used without one specific preferred regimen.
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Utility of intermittent online quizzes as an early warning for residents at risk of failing the pediatric board certification examination.

BACKGROUND: Traditionally, quizzes have been applied as a tool for summative assessment, though literature suggests their use as a formative assessment can improve motivation and content retention. With this premise, we implemented a series of intermittent, online quizzes known as the Board Examination Simulation Exercise (BESE). We sought to demonstrate an association between BESE participation and scores and performance on the American Board of Pediatrics (ABP) Certifying Examination (CE). METHODS: Residents were assigned online quizzes on a single topic at 2 week intervals that consisted of 20 multiple choice questions written by the study authors. This analysis includes graduates of 3 Pediatric and Internal Medicine-Pediatrics residency programs. RESULTS: Data were available for 329 residents. The overall BESE score weakly correlated with ABP CE score (n = 287; r = 0.39, p < 0.0001). ABP CE pass rates increased from 2009 to 2016 at all programs combined (p = 0.0001). A composite BESE score ≤ 11 had sensitivity of 54% and specificity of 80% for predicting ABP CE failure on the first attempt. There was no difference in ABP CE failure rates or scores by number of completed quizzes. CONCLUSION: Intermittent online quizzes implemented at three pediatric residency programs were associated with overall increasing ABP CE pass rates. BESE increased program emphasis on board preparation. Residents with lower BESE scores more often failed ABP CE. Though additional data are needed, BESE is a promising tool for pediatric resident learning and board preparation. It may also aid in earlier identification of residents at higher risk of failing the ABP CE and facilitate targeted interventions.

Responsiveness of the PROMIS® measures to changes in disease status among pediatric nephrotic syndrome patients: a Midwest pediatric nephrology consortium study.

BACKGROUND: Nephrotic syndrome represents a condition in pediatric nephrology typified by a relapsing and remitting course, proteinuria and the presence of edema. The PROMIS measures have previously been studied and validated in cross-sectional studies of children with nephrotic syndrome. This study was designed to longitudinally validate the PROMIS measures in pediatric nephrotic syndrome. METHODS: One hundred twenty seven children with nephrotic syndrome between the ages of 8 and 17 years participated in this prospective cohort study. Patients completed a baseline assessment while their nephrotic syndrome was active, a follow-up assessment at the time of their first complete proteinuria remission or study month 3 if no remission occurred, and a final assessment at study month 12. Participants completed six PROMIS measures (Mobility, Fatigue, Pain Interference, Depressive Symptoms, Anxiety, and Peer Relationships), the PedsQL version 4.0, and two global assessment of change items. RESULTS: Disease status was classified at each assessment: nephrotic syndrome active in 100% at baseline, 33% at month 3, and 46% at month 12. The PROMIS domains of Mobility, Fatigue, Pain Interference, Depressive Symptoms, and Anxiety each showed a significant overall improvement over time (p < 0.001). When the PROMIS measures were compared to the patients' global assessment of change, the domains of Mobility, Fatigue, Pain Interference, and Anxiety consistently changed in an expected fashion. With the exception of Pain Interference, change in PROMIS domain scores did not correlate with changes in disease activity. PROMIS domain scores were moderately correlated with analogous PedsQL domain scores. CONCLUSION: This study demonstrates that the PROMIS Mobility, Fatigue, Pain Interference, and Anxiety domains are sensitive to self-reported changes in disease and overall health status over time in children with nephrotic syndrome. The lack of significant anchoring to clinically defined nephrotic syndrome disease active and remission status may highlight an opportunity to improve the measurement of HRQOL in children with nephrotic syndrome through the development of a nephrotic syndrome disease-specific HRQOL measure.