Updated consensus guidelines on the management of Phelan-McDermid syndrome.

Phelan-McDermid syndrome (PMS) is a genetic condition caused by SHANK3 haploinsufficiency and characterized by a wide range of neurodevelopmental and systemic manifestations. The first practice parameters for assessment and monitoring in individuals with PMS were published in 2014; recently, knowledge about PMS has grown significantly based on data from longitudinal phenotyping studies and large-scale genotype-phenotype investigations. The objective of these updated clinical management guidelines was to: (1) reflect the latest in knowledge in PMS and (2) provide guidance for clinicians, researchers, and the general community. A taskforce was established with clinical experts in PMS and representatives from the parent community. Experts joined subgroups based on their areas of specialty, including genetics, neurology, neurodevelopment, gastroenterology, primary care, physiatry, nephrology, endocrinology, cardiology, gynecology, and dentistry. Taskforce members convened regularly between 2021 and 2022 and produced specialty-specific guidelines based on iterative feedback and discussion. Taskforce leaders then established consensus within their respective specialty group and harmonized the guidelines. The knowledge gained over the past decade allows for improved guidelines to assess and monitor individuals with PMS. Since there is limited evidence specific to PMS, intervention mostly follows general guidelines for treating individuals with developmental disorders. Significant evidence has been amassed to guide the management of comorbid neuropsychiatric conditions in PMS, albeit mainly from caregiver report and the experience of clinical experts. These updated consensus guidelines on the management of PMS represent an advance for the field and will improve care in the community. Several areas for future research are also highlighted and will contribute to subsequent updates with more refined and specific recommendations as new knowledge accumulates.

Stratification of a Phelan-McDermid Syndrome Population Based on Their Response to Human Growth Hormone and Insulin-like Growth Factor.

Phelan-McDermid syndrome (PMS), caused by pathogenic variants in the SHANK3 gene or 22q13 deletions, is characterized by intellectual disability, autistic features, developmental delays, and neonatal hypotonia. Insulin-like growth factor 1 (IGF-1) and human growth hormone (hGH) have been shown to reverse neurobehavioral deficits in PMS. We assessed the metabolic profiling of 48 individuals with PMS and 50 controls and determined subpopulations by taking the top and bottom 25% of responders to hGH and IGF-1. A distinct metabolic profile for individuals with PMS showed a reduced ability to metabolize major energy sources and a higher metabolism of alternative energy sources. The analysis of the metabolic response to hGH or IGF-1 highlighted a major overlap between both high and low responders, validating the model and suggesting that the two growth factors share many target pathways. When we investigated the effect of hGH and IGF-1 on the metabolism of glucose, the correlation between the high-responder subgroups showed less similarity, whereas the low-responders were still relatively similar. Classification of individuals with PMS into subgroups based on responses to a compound can allow an investigation into pathogenic mechanisms, the identification of molecular biomarkers, an exploration of in vitro responses to candidate drugs, and eventually the selection of better candidates for clinical trials.

Identifying Patient-Centered Outcomes for Caregivers and Children With Musculoskeletal Infections.

Background: Musculoskeletal infections (MSKI), including osteomyelitis and septic arthritis, are among the most common invasive infections in children and have the potential to cause significant morbidity. Guidelines have been developed to optimize care based on clinician-developed endpoints. Patient-centered outcomes have not been defined for children with MSKI. This study identified outcomes most important to caregivers and patients with MSKI. Methods: This was a single-center prospective qualitative study of children 6 months to 18 years of age hospitalized with MSKI from November 2019 to September 2021. Using design-research methods, patients and caregivers participated in interviews and/or completed journals to describe their experiences during acute infection and recovery from MSKI. Results: A total of 51 patient/caregivers were approached to participate in the study, 35 of whom declined to participate, resulting in 8 interviews conducted and 14 journals collected from 16 patient/caregivers. From these, a journey map was created highlighting points of stress during the onset of symptoms, through hospitalization, and returning home with new challenges. In addition, patient-centered outcomes were identified. For caregivers, these included managing mental health, managing responsibilities, and receiving support. Both caregivers and patients shared the importance of understanding of treatment plans and responsibilities. For patients, improving mental and physical health was important. Conclusions: Management of children with MSKI is complex and requires a multidisciplinary team approach. Validation of the outcomes identified and development of a measurement tool are needed. Addressing the patient-centered outcomes we identified in this study can greatly improve the holistic care of children with MSKI.

Retrograde stent with external string for pediatric robotic pyeloplasty: does it reduce cost and complications?

Robotic-assisted pyeloplasty (RAP) is a mainstay in the treatment of ureteropelvic junction obstruction (UPJO) in children. At our institution, to limit planned operating rooms visits we have placed a ureteral stent with an external string (SWES) immediately prior to RAP. In this study, we sought to quantify the operative time, complications, and costs associated with this approach compared to the traditional approach, requiring subsequent stent removal in the operating room. We hypothesized the SWES cohort would have decreased cost, yet with similar operative time and complications. We retrospectively collected all RAPs performed at our institution using the SWES approach (Aug 2012-July 2017). We excluded those with a redo pyeloplasty, and/or a percutaneous nephrostomy tube for post-operative drainage. We collected 30-day costs linked to the patients' MRN using the Pediatric Health Information System (PHIS) database. We compared 30-day healthare costs for all patients following RAP. We compared our SWES group to a national cohort of all pediatric RAP during the same time period. Lastly, we sent an anonymous, electronic survey to urologists of all PHIS institutions to identify the predominant postoperative drainage, nationally. Within our institution, we reviewed all those treated with SWES (n = 85) (Table 1). The median 30-day cost was $10,548 among those with SWES (Table 2). This was significantly less than the overall, national cohort of all pediatric RAP during the same period ($14,119, p < 0.001). There was a 15.5 % rate of unplanned return to the hospital in the SWES group. Of those unplanned returns, 8.2 % (7/85) had unplanned return for a procedure (3 for unplanned stent removal, 2 for nephrostomy tube for persistent obstruction, 1 for omental hernia, and 1 for stent replacement). With a 42.5 % (37/87) response rate, our nationwide survey found 84.6 % primarily leave stents WITHOUT a string, 7.7 % left nephrostomy tubes, and 7.7 % stents with strings. During pediatric RAP, placement of a SWES takes little time, carries a risk of unplanned visit to the operating room, saves the patient a certain, second anesthetic for stent removal, and amounts to a cost savings of approximately 25 %.

Association Between Continuous Kidney Replacement Therapy Clearance and Outcome in Pediatric Patients With Hyperammonemia Not Due to Inborn Error of Metabolism.

OBJECTIVES: To describe a single-center experience of pediatric patients with hyperammonemia not due to inborn errors of metabolism and determine the association between use of continuous kidney replacement therapy (CKRT) treatment and outcomes. DESIGN: Retrospective cohort study. SETTING: Tertiary-care children's hospital. PATIENTS: All children less than 21 years old admitted to the hospital with hyperammonemia defined as an elevated ammonia levels (>100 µmol/L) not due to inborn error of metabolism. INTERVENTIONS: None. MEASURES AND MAIN RESULTS: Of 135 children with hyperammonemia, the most common reason for admission was infection in 57 of 135 (42%), congenital heart disease in 20 of 135 (14%), and bone marrow transplantation in 10 of 135 (7%). The overall mortality was 61% (82 of 135), which increased with degree of hyperammonemia (17 of 23 [74%] in those with ammonia >250 µmol/L). After multivariable regression, hyperammonemia severity was not associated with mortality (aOR, 1.4; 95% CI, 0.92-2.1; p = 0.11). Of the 43 patients (32%) receiving CKRT, 21 were prescribed standard clearance and 22 high clearance. The most common indications for CKRT were fluid overload in 17 of 43 (42%) and acute kidney injury or uremia in 16 of 43 (37%). Mean CKRT duration was 13 days. There was no difference between standard and high clearance groups in risk of death (76% vs 86%; p = 0.39), cerebral edema on CT scan (19% vs 27%; p = 0.52), nor decrease in ammonia levels after 24 or 48 hours of CKRT ( p = 0.20, p = 0.94). Among those receiving CKRT, we failed to find an association between high clearance and decreased risk of death in multivariable analysis (aOR, 1.2; 95% CI, 0.64-2.3; p = 0.55). CONCLUSIONS: In our single-center retrospective study, we failed to find an association between clearance on CKRT and improved survival nor decreased cerebral edema on head imaging. In fact, we failed to find an association between ammonia level and mortality, after controlling for illness severity.

Proactive measurement of infliximab drug levels in children with Crohn's disease.

BACKGROUND: Proactively monitoring infliximab levels is an emerging area of interest in pediatric Crohn's disease. There are only limited data on therapeutic drug monitoring for children with Crohn's disease. The goal of our study was to determine the utility of therapeutic drug monitoring in achieving clinical remission in a cohort of pediatric Crohn's disease patients receiving infliximab. METHODS: This prospective single-center study enrolled 37 patients with Crohn's disease at the start of infliximab infusions and monitored trough levels at 6-month intervals for 18 months. Each participant was matched to a historic control for the modified pediatric Crohn's disease activity index (mPCDAI) at baseline, age and sex. The primary outcome was an mPCDAI score of ≤7.5 at 6, 12 and 18 months. A multivariate logistic regression analysis was performed. RESULTS: Data were available for all 37 cases at 6 and 12 months and for 34 cases at 18 months. Demographics and disease characteristics were similar between groups. All 34 cases demonstrated clinical remission at 18 months (100% vs. 88%, P=0.114). Univariate and multivariate analyses did not show statistical significance. Dose intensification was seen more often in the cases at 18 months. CONCLUSION: All of our moderate-to-severe pediatric Crohn's disease patients who received prospective therapeutic drug monitoring of infliximab were in clinical remission at follow up, but this was not statistically significantly different from the 88% clinical remission rate of the control group.

Phelan-McDermid syndrome: a classification system after 30 years of experience.

Phelan-McDermid syndrome (PMS) was initially called the 22q13 deletion syndrome based on its etiology as a deletion of the distal long arm of chromosome 22. These included terminal and interstitial deletions, as well as other structural rearrangements. Later, pathogenetic variants and deletions of the SHANK3 gene were found to result in a phenotype consistent with PMS. The association between SHANK3 and PMS led investigators to consider disruption/deletion of SHANK3 to be a prerequisite for diagnosing PMS. This narrow definition of PMS based on the involvement of SHANK3 has the adverse effect of causing patients with interstitial deletions of chromosome 22 to "lose" their diagnosis. It also results in underreporting of individuals with interstitial deletions of 22q13 that preserve SHANK3. To reduce the confusion for families, clinicians, researchers, and pharma, a simple classification for PMS has been devised. PMS and will be further classified as PMS-SHANK3 related or PMS-SHANK3 unrelated. PMS can still be used as a general term, but this classification system is inclusive. It allows researchers, regulatory agencies, and other stakeholders to define SHANK3 alterations or interstitial deletions not affecting the SHANK3 coding region.

Inadequate Bowel Preparation in Pediatric Colonoscopy-Prospective Study of Potential Causes.

OBJECTIVES: Inadequate bowel preparation (IBP) for colonoscopy leads to missed diagnosis, longer anesthesia time, higher chance of complications and increased costs. Adult studies have demonstrated that patient characteristics such as male gender and obesity are associated with IBP. Little is known about factors affecting bowel preparation in children. Our aim was to determine factors associated with IBP in children. METHODS: We prospectively enrolled children undergoing outpatient colonoscopy. Quality of bowel preparation was assessed using Boston Bowel Preparation Scale (BBPS) score (range 0-9). Data collected included patient demographics, indication, and type of insurance. Patients were divided into two groups based on BBPS score-adequate (BBPS score > 5) and inadequate (BBPS score < 5) and groups were compared using Student t-test and chi-square test. Possible predictors were analyzed using multivariate logistic regression models. RESULTS: A total of 334 children were prospectively enrolled of whom 321 were studied further (age range 2-18 years; mean age 12.4 years; 60.4% female; 85.9% Caucasian). The mean BBPS score was 6.8 (standard deviation of ±2). IBP was reported in 12.8% (41/321). Multivariable logistic regression analysis did not show statistical differences between the groups in studied patient factors including age, gender, obesity, race, insurance type, and indication for colonoscopy. CONCLUSION: Contrary to several adult studies, the results of our prospective study did not show any relationship between examined patient factors and IBP in children. Interestingly, IBP was less prevalent in our pediatric study compared to published adult data (12.8% vs 20-40%).

Factors Affecting Higher Readmission Rates and Costs in Pediatric Patients With Hirschsprung Disease.

BACKGROUND: Hirschsprung disease is a relatively uncommon disorder of the developing gastrointestinal tract that requires surgical intervention to restore long-term function. While readmission for Hirschsprung-related complications is a known concern in these patients, we sought to identify patient-level factors associated with a prolonged hospital stay, increased costs at the time of a pull-through operation, as well as the risk for all-cause inpatient readmission after surgery. We hypothesized that higher level of care requirement during the operative stay, age at operation, and length of stay (LOS) would portend increased readmissions and disease-related complications such as Hirschsprung-associated enterocolitis. METHODS: Data was obtained from the Pediatric Health Information System database on all Hirschprung patients who underwent a pull-through operation between 2004 and 2019. Regression analyses were performed on this cohort of 3345 patients. Multivariable regression models were utilized to analyze the key outcome variables of postoperative LOS and adjusted charges. RESULTS: Post-operative LOS was significantly increased by the presence of a surgical complication, congenital/genetic defect, or neurologic/neuromuscular defect. Increased LOS was also seen in Black patients. The cost of pull-through operations was significantly higher in patients admitted to the NICU and ICU during index hospitalization, with a cost increase of approximately $75,000 and $57,000 respectively. Presence of a surgical complication, comorbid congenital/genetic defect, and need for mechanical ventilation were associated with higher odds of inpatient readmission. CONCLUSION: The management of patients with Hirschsprung disease is longitudinal and complex. Identification of key patient metrics can aid clinicians in developing targeted care and education strategies to minimize readmission and excessive hospital charges.

The Effects of Patient Demographics on Outpatient Endoscopy Utilization in Children With Eosinophilic Esophagitis.

OBJECTIVES: Eosinophilic esophagitis (EoE) is a chronic inflammatory disease of the esophagus that requires esophagogastroduodenoscopy (EGD) for diagnosis and treatment monitoring. We aimed to identify the frequency of endoscopic monitoring in children with EoE and observe the effect of age, race, socioeconomic factors, and atopy on the rate of endoscopy. METHODS: We queried the Pediatric Health Information System over a 15-year period for ambulatory EGDs in children with EoE. Subjects with at least 1 year of data were included. Age, sex, ethnicity, race, insurance type, median household income, and atopy were collected for each subject. RESULTS: 16,517 subjects were included (mean age 8.5 years). 3211 (19%) of subjects had ≥1 EGD per subject year (EGD/SY). Subjects >13 years old were more likely to have ≥1 EGD/SY compared to children 6-12 years (odds ratio [OR] 2.29, P < 0.001, 95% confidence interval [CI] = 2.06-2.54). Males were more likely to have ≥1 EGD/SY compared to females (OR 1.19, P < 0.001, 95% CI = 1.08-1.31). African-American subjects were 16% less likely than Caucasian subjects to have ≥1 EGD/SY (OR 0.84, P = 0.05, 95% CI = 0.71-1.00). Subjects with allergic rhinitis or anaphylaxis, food allergy, and/or oral allergy syndrome were more likely to have ≥1 EGD/SY (OR 1.67, P < 0.001, 95% CI = 1.47-1.90 and OR 3.65, P < 0.001, 95% CI = 3.25-4.11, respectively). CONCLUSIONS: Nineteen percent of subjects had ≥1 EGD/SY. Older age, male sex, allergic rhinitis, and food allergies were associated with more frequent endoscopic monitoring in children with EoE. Caucasian subjects had more frequent endoscopy than African-American subjects. This study raises awareness about underrecognized variation in the care of children with EoE.

Troubleshooting problems with antegrade continent enema flushes: The Indiana university algorithm.

INTRODUCTION: Antegrade continence enemas have transformed treatment and improved the quality of life in children with neuropathic bowel, refractory constipation and fecal incontinence. However, it can often be difficult to manage problems that arise with ACE flushes. OBJECTIVE: We report the use of an online tool designed for nurses to help troubleshoot calls for problems associated with antegrade continence enema (ACE) flushes as well as update our algorithm for managing refractory constipation/fecal incontinence in a large single institution experience. STUDY DESIGN: We developed an online tool based on our management protocol for managing refractory constipation/fecal incontinence (Summary Figure). Patient frequency and bother was assessed prior to the intervention and at one month after the intervention using 5- and 4-point Likert scales respectively. Patient demographics, MACE/Chait information, type of difficulty, volume of flush, and use of additives were recorded. Nurses were also interviewed prior to using the tool and 14 months after its development with regards to taking these phone calls and the helpfulness of the tool. RESULTS: Over 14 months, the nurses received 22 patients calls via the nursing triage line regarding ACE flush problems and prospectively collected data. Half reported multiple episodes of fecal incontinence. Other complaints included no response to flush (8, 36.4%), occasional episodes of liquid fecal incontinence (2, 9.1%) and time of flush exceeding 60 min (1, 4.5%). While patients did not report decreased frequency of problems as a result of nurse troubleshooting using the ACE algorithm (2.5 vs. 2, p = 0.55), patients did report a significant improvement in their bother scores (4 vs. 2, p = 0.02). All but one patient reported that the recommendation was "some" or "a lot" helpful on follow up interview. The nurses all indicated that the tool helped "some" or "a lot." DISCUSSION: The antegrade continence enema is valuable in managing neurogenic bowel, refractory constipation, and fecal incontinence, however, some patients experience problems with flushes that can often be difficult to manage. CONCLUSION: Patients reported less bother with their bowel issues after using our algorithm for managing refractory constipation/fecal incontinence and nurses reported that the tool was helpful.

Mortality, Risk Factors and Disparities Associated with Esophageal Variceal Bleeding in Children's Hospitals in the US.

OBJECTIVES: To use a large administrative database to determine the mortality, risk factors, and comorbidities of esophageal variceal bleeding in children. STUDY DESIGN: Retrospective cohort study using Pediatric Health Information System data from 50 tertiary children's hospitals in the US. International Classification of Diseases (ICD) codes (FY 2020 ICD-10 update and revision 10 of ICD-9) from 2004 through 2019 identified children 18 years and younger with variceal bleeding and complications. Univariate analyses used the Student t -test for continuous variables (age) and the χ2 test for categorical variables (all others). A mixed-effects linear regression was performed for multiple variables. RESULTS: There were 1902 patients who had 3399 encounters for esophageal variceal bleeding. The mortality rate for variceal bleeding was 7.3%, increasing to 8.8% by 6 weeks; any mortality during the study was 20.1%. Transfusion was required in 54.7% of encounters, and 42.6% were admitted to the intensive care unit. Variceal bleeding encounters were complicated by peptic ulcer disease (6.9%), bacteremia (11.4%), acute renal failure (5.1%), mechanical ventilation (18%), ascites (21.3%), and peritonitis (3.3%). Multivariable mixed-effects logistic regression showed that Black race (OR, 2.59; P < .001) or Hispanic ethnicity (OR, 2.31; P = .001), but not sex, household income, or insurance type, were associated with increased mortality. Bacteremia, peritonitis, mechanical ventilation, acute renal failure, and transfusion were associated with higher mortality (ORs of 2.29, 2.18, 1.93, 6.33, and 1.81, respectively; P < .001, .005, .011, <.001, and .005, respectively). CONCLUSIONS: The 6-week mortality rate for variceal bleeding in children is 8.8%. Black or Hispanic children are at higher risk of dying. Serious morbidities associated with variceal hemorrhage impact mortality. These data can inform consideration of prophylactic or therapeutic interventions for children at risk.

What's Inside of a AA Battery? An Unusual Caustic Ingestion in an Infant.

Current guidelines for the management of battery ingestions in children focus on button batteries due to the risk of morbidity and mortality. In our review of the literature, there is little information on the ingestion of cylindrical AA or AAA battery contents. We report a case of an 11-month-old female who ingested the internal alkaline contents of a AA battery. The ingestion resulted in oropharyngeal and esophageal caustic injuries visualized on upper endoscopy. Imaging has long been used for localizing ingested whole batteries. In our case, standard radiograph confirmed that internal battery contents were ingested. Advanced imaging modalities, including computed tomography, have been suggested as methods to investigate the degree of caustic injury and were utilized in this case. Our case is one of the few reported cases of the ingestion of alkaline battery contents alone.

A feasibility study of telemedicine for paediatric sickle cell patients living in a rural medically underserved area.

INTRODUCTION: Sickle cell disease (SCD) is the most common inherited haematological disease, with potentially devastating complications. Improvements in therapies have increased the life span of patients with SCD, but this is contingent on receiving timely evidence-based medical care, including regular evaluations with haematologists, disease-specific education and psychosocial care. Our objective was to evaluate the feasibility of utilizing telemedicine for the provision of subspecialty paediatric SCD care in a rural medically underserved area. METHODS: This was a cross-sectional, observational, feasibility study. All patients 0-21 years old with SCD seen at Riley Hospital for Children Comprehensive Pediatric Sickle Cell Clinic who lived within 30 miles of the spoke telemedicine facility were eligible for recruitment. The Telehealth Satisfaction Scale (TeSS) was adapted for the SCD population and administered at each visit. RESULTS: Ten SCD patients, ranging in age from 10 months to 18 years old, initiated telemedicine visits during this timeframe. Some 60% were lost to follow-up or did not attend >50% of scheduled visits prior to beginning telemedicine visits. Following initiation of telemedicine, all Hb SS patients were started and/or maintained on hydroxyurea. Nine out of 10 patients who participated during this timeframe had a 100% follow-up rate. All who participated rated the comfort and ease of using the telehealth system as good or excellent and would do a telemedicine visit again. DISCUSSION: This study provides critical information to determine the feasibility and acceptability of a telemedicine intervention to aid in SCD care. To our knowledge, this is the first study to examine the effectiveness of telemedicine to deliver comprehensive paediatric SCD care. Future research with a larger sample size is needed to confirm findings of our study, including expansion of telemedicine sites to include more urban areas.

Tranexamic Acid in Infantile Craniosynostosis Surgery: Friend or Foe?

BACKGROUND: Recent studies support the prophylactic use of tranexamic acid during craniosynostosis surgery to reduce blood loss. The study aims to assess national trends and outcomes of tranexamic acid administration. METHODS: The Pediatric Health Information System database was used to identify patients who underwent craniosynostosis surgery over a 9-year period (2010 to 2018). Search criteria included patients younger than 2 years with a primary diagnosis of craniosynostosis (International Classification of Diseases, Ninth Revision, 756.0; International Classification of Diseases, Tenth Revision, Q75.0) and CPT code for craniotomy (61550 to 61559). Tranexamic acid use, complications, length of stay, and transfusion requirements were recorded. Subgroup analysis was performed for fronto-orbital advancements and single-suture surgery. RESULTS: A total of 1345 patients were identified. Mean patient age was 229 ± 145 days. Four hundred fifty-four patients (33.7 percent) received tranexamic acid. Tranexamic acid use increased from 13.1 percent in 2010 to 75.6 percent in 2018 (p = 0.005), and mean blood products per patient increased from 1.09 U to 1.6 U (p = 0.009). Surgical complication rate was higher in those receiving tranexamic acid (16.7 percent versus 11.1 percent; p = 0.004). Tranexamic acid administration was associated with increased transfusion requirements on univariate and multivariate analysis (1.76 U versus 1.18 U; OR, 2.03; p < 0.001). In the fronto-orbital advancement subgroup, those receiving tranexamic acid received more total blood products (2.2 U versus 1.8 U; p = 0.02); this difference was present but not significant within the single-suture group (0.69 U versus 0.50 U; p = 0.06). CONCLUSIONS: Tranexamic acid use in craniosynostosis surgery has increased dramatically since 2010. However, it was associated with higher transfusion and complication rates in this data set. Optimization of its use and blood loss mitigation in infant craniosynostosis deserve continued research. CLINICAL QUESTION/LEVEL OF EVIDENCE: Therapeutic, III.

Cholangitis in Patients With Biliary Atresia Receiving Hepatoportoenterostomy: A National Database Study.

INTRODUCTION: Biliary atresia (BA) is a progressive form of liver disease in the neonatal period usually requiring hepatoportoenterostomy (HPE). Cholangitis is a common sequelae of HPE but data about which patients are at risk for this complication are limited. OBJECTIVE: The objective of the study was to determine risk factors associated with cholangitis in a large retrospective cohort after HPE. METHODS: The Pediatric Health Information System (PHIS) was queried for BA (ICD-9 975.61) and HPE (ICD-9-CM 51.37) admissions from 2004 to 2013. We performed univariate analysis and linear regression with dependent variables of ≥ 2 or ≥ 5 episodes of cholangitis, and independent variables of age at time of HPE, race, ethnicity, gender, insurance, ursodeoxycholic acid (UDCA) use, steroid use, presence of esophageal varices (EV), and portal hypertension (PH). RESULTS: We identified 1112 subjects with a median age at HPE of 63 days and median number of cholangitis episodes of 2 within 2 years. On multiple regression analysis, black race (odds ratio (OR) 1.51, P = 0.044) and presence of PH (OR 2.24, P < 0.001) were associated with increased risk of ≥ 2 episodes of cholangitis, whereas HPE at >90 days was associated with less risk (OR 0.46, P = 0.001). Among those with ≥5 episodes, Asian race (OR 2.66, P = 0.038), public insurance (OR 1.72, P = 0.043), EV (OR 1.81, P = 0.017), and PH (OR 2.88, P < 0.001) were associated with higher risk. CONCLUSIONS: Complications, such as cholangitis remain a common problem for patients, after HPE, with median of 2 episodes within 2 years. Higher rates of cholangitis are associated with portal hypertension whereas lower rate is associated with age at HPE of >90 days. Asians, patients with public insurance, and those with portal hypertension are more likely to have recurrent cholangitis.

Insurance Denials of Care Amount to Unlicensed Medical Practice.

DISCLOSURES: No funding supported the writing of this article. The author has nothing to disclose.

Variation in hospital admission from the emergency department for children with cancer: A Pediatric Health Information System study.

BACKGROUND: Children with cancer experience a wide range of conditions that require urgent evaluation in the emergency department (ED), yet variation in admission rates is poorly documented. PROCEDURE: We performed a retrospective cohort study using the Pediatric Health Information System of ED encounters by children with cancer between July 2012 and June 2015. We compared demographics for admitted versus discharged using univariate statistics, and calculated admission rates by hospital, diagnosis, day of the week, and weekend versus weekday. We assessed the degree of interhospital admission rates using the index of dispersion (ID). RESULTS: Children with cancer had 60 054 ED encounters at 37 hospitals. Overall, 62.5% were admitted (range 43.2%-92.1%, ID 2.6) indicating overdispersed admission rates with high variability. Children with cancer that visited the ED for a primary diagnosis of fever experienced the largest amount of variability in admission with rates ranging from 10.4% to 74.1% (ID 8.1). Less variability existed among hospital admission rates for both neutropenia (range 60%-100%, ID 1.0) and febrile neutropenia (FN) (range 66.7%-100%, ID 0.83). Admission rates by day of the week did not demonstrate significant variability for any of the scenarios examined (overall P = 0.91). There were no differences by weekend versus weekday either (overall P = 0.52). CONCLUSION: The percentage of children with cancer admitted through the ED varies widely by institution and diagnosis. Standardization of best practices for children with cancer admitted through the ED should be an area of continued improvement.

Variation in hospital admission of sickle cell patients from the emergency department using the Pediatric Health Information System.

BACKGROUND: Universal newborn screening and improved treatment options have led to increased survival in sickle cell disease (SCD). However, patients with SCD still rely heavily on acute care services. OBJECTIVE: To determine the variation seen in hospitalizations for the top complaints for ED visits for children with SCD nationally. METHODS: We performed a retrospective review of the Pediatric Health Information Systems (PHIS) Database between October 2011 and September 2015. Emergency department (ED) encounters were selected by using International Classification of Diseases, Ninth Edition, Clinical Modification (ICD-9-CM) codes for SCD with and without crisis, fever, and pain. Univariate analyses were performed, as well as index of dispersion (ID) to assess variation by day of the week and region. ANOVA and t-test were used to determine statistical significance. RESULTS: A total of 68 661 ED encounters at 36 hospitals met the criteria for inclusion. Of those encounters, 50.1% were admitted to the hospital. Pain and fever were the most common primary diagnoses among this population. Although variation in hospitalization was seen overall, as well as for a primary diagnosis of pain or fever, this variation was not explained by weekday/weekend designation. CONCLUSION: The results of our study confirm pain and fever as the most common primary diagnoses for children with SCD who seek acute care, as well as demonstrate that while significant variation in hospitalization exists, it is not associated with day of the week. Further studies to elucidate patient- and hospital-level factors that influence admission variation are necessary.

A Retrospective Cohort Study of Growth in the First 2 Years of Life in Preterm Infants With Cystic Fibrosis.

BACKGROUND: Late preterm infants (born 34-36 weeks gestation) without cystic fibrosis (CF) are at risk for growth failure during the first 2 years of life. Infants with CF are at risk of being born premature, and thus at risk for growth failure. The aim for this study was to assess weight-for-length (WFL) at 2 years of age for late preterm infants compared with term infants with CF. METHODS: Data were collected from the US CF Foundation Patient Registry. We compared growth parameters between late preterm and term infants with CF born from 2010 to 2013. Our primary outcome was WFL <10 and <50 percentile at 2 years of age. A multivariate logistical regression analysis evaluated late preterm gestation and WFL<10 or <50 percentile. RESULTS: A total of 2955 infants were born from 2010 to 2013 with CF. Eight percentage late preterm. Forty-five percentage late preterm versus 43% term were below the 50th percentile for WFL at age 2 years (P = 0.75). Twelve percentage late preterm versus 6% term for WFL <10 percentile at age 2 years (P = 0.010). The multivariate regression model identified 2-fold increased odds of being <10th percentile for WFL at age 2 years (P = 0.025) for preterm over term. Late preterm infants used higher calorie dense feeds and more feeding tubes (P = 0.035 and P = 0.006). CONCLUSIONS: Late preterm infants with CF are at higher risk of being below the 10th percentile for WFL at 2 years of age compared with their term peers. This indicates a population that is at risk for growth failure.