Case Discussions and Radiographic Illustration of Magnet-Related Injuries in Children.

BACKGROUND: Injuries from multiple magnet ingestions in the pediatric population have been increasing in both incidence and morbidity. This trend will likely continue after a 2017 court ruling that overturned a ban on the sale of magnet sets marketed as "adult desk toys." Depending on the arrangement of the ingested magnets in the gastrointestinal tract, the consequences can range from benign to life threatening. OBJECTIVE: This review of cases aims to help clinicians recognize this pathology and help them appreciate the unique management of this type of foreign body ingestion. DISCUSSION: Several cases are presented that individually illustrate an arm of the most comprehensive management algorithm, proposed by the North American Society for Pediatric Gastroenterology, Hepatology, and Nutrition. The management is largely driven by the clinical appearance of the child as well as information obtained through abdominal radiographs. Imaging variables that factor into management include the location of the magnets, the number of magnets, and the progression of magnets on serial radiographs. CONCLUSION: This article uses cases and illustrative medical imaging to describe the most common scenarios and their management. This is especially relevant considering recent U.S. court rulings that overturned the U.S. Consumer Product Safety Commission's ban on the sale of toys containing multiple miniature magnets.

Assessing Competence in Emergency Radiology Using an Online Simulator.

RATIONALE AND OBJECTIVES: Traditional assessments in radiology residency focus on the Medical Expert CanMEDS role and typically rely upon a single or limited static images. We designed an Emergency Radiology Simulator that aimed to assess the breadth of competencies required across Medical and NonMedical Expert domains. MATERIAL AND METHODS: An online simulator with typical emergency cases was administered in October 2015 to Post Graduate Year (PGY) 2-5 residents in Radiology. Residents provided preliminary reports, which were graded for style and content. The simulation also included prioritization, protocoling, counseling, and handover exercises geared to assess NonMedical Expert roles. RESULTS: Fourty eight residents participated in the simulation. Level of resident was 11 PGY-2, 17 PGY-3, 13 PGY-4, and 7 PGY-5. There was a significant difference in resident performance between PGY-2 residents and those more senior in terms of the Medical Expert role (findings, diagnosis, recommendations, and clinical relevance of reports). Differences in performance between PGY levels were not seen in the NonMedical Expert roles (prioritization, protocoling, counseling, and handover). CONCLUSION: Simulation provides an opportunity to assess radiology resident performance across multiple domains. PGY-2 residents performed worse on the Medical Expert domains, although performance did not significantly vary between the other years. This may suggest that competence in Emergency Radiology is achieved early in residency, possibly related to the importance placed on developing skills related to on-call performance during the PGY-2 year. The simulator should be extended to other areas of Radiology, in order to assess the ability to discriminate performance in other subspecialties.

Preventing the Progression of Intestinal Failure-Associated Liver Disease in Infants Using a Composite Lipid Emulsion: A Pilot Randomized Controlled Trial of SMOFlipid.

BACKGROUND: To examine whether SMOFlipid prevents progression of intestinal failure-associated liver disease (IFALD) in parenteral nutrition (PN)-dependent infants with early IFALD (conjugated bilirubin 17-50 µmol/L, 1-3 mg/dL). STUDY DESIGN: Pilot multicenter blinded randomized controlled trial comparing SMOFlipid with Intralipid. Patients received the trial lipid for up to 12 weeks, unless they achieved full enteral tolerance sooner. The primary clinical outcome was the serum conjugated bilirubin. RESULTS: Twenty-four infants (mean age, 6 weeks) participated in the trial (13 Intralipid and 11 SMOFlipid). At the time of trial enrollment, patients in both groups were receiving 90% of their calories by PN. Mean duration on trial was 8 weeks and did not differ according to treatment ( P = .99). At trial conclusion, patients who received SMOFlipid had a lower conjugated bilirubin than those who received Intralipid (mean difference, -59 µmol/L; P = .03). Patients receiving SMOFlipid were also more likely to have a decrease in serum conjugated bilirubin to 0 µmol/L than those in the Intralipid group over the entire observation period (hazard ratio, 10.6; 95%; P = .03). The time to achievement of full enteral tolerance did not differ statistically (hazard ratio, 1.3; P = .59) between the groups. There was no significant difference in safety outcomes between the groups. CONCLUSIONS: Compared with Intralipid, SMOFlipid reduces the risk of progressive IFALD in children with intestinal failure. This trial was registered at clinicaltrials.gov as NCT00793195.

Alternative designs for clinical trials in rare diseases.

Evidence-based medicine requires strong scientific evidence upon which to base treatment. In rare diseases, study populations are often small, and thus this evidence is difficult to accrue. Investigators, though, should be creative and develop a flexible toolkit of methods to deal with the problems inherent in the study of rare disease. This narrative review presents alternative clinical trial designs for studying treatments of rare diseases, including cross-over and n-of-1 trials, randomized placebo-phase design, enriched enrollment, randomized withdrawal design, and classes of adaptive designs. Examples of applications of these designs are presented along with their advantages and disadvantages. Additional analytical considerations such as Bayesian analysis, internal pilots, and use of biomarkers as surrogate outcomes are further discussed. A framework for selecting appropriate clinical trial design is proposed to guide investigators in the process of selecting alternative designs for rare diseases. © 2016 Wiley Periodicals, Inc.

A new toolkit for conducting clinical trials in rare disorders.

Evidence based medicine requires strong scientific evidence upon which to base treatment. Because the available study populations for rare diseases are small, this evidence is difficult to accrue. Investigators need to consider a flexible toolkit of methods to deal with the problems inherent in the study of rare disease. We present some potential solutions in this paper.

Defining consensus: a systematic review recommends methodologic criteria for reporting of Delphi studies.

OBJECTIVE: To investigate how consensus is operationalized in Delphi studies and to explore the role of consensus in determining the results of these studies. STUDY DESIGN AND SETTINGS: Systematic review of a random sample of 100 English language Delphi studies, from two large multidisciplinary databases [ISI Web of Science (Thompson Reuters, New York, NY) and Scopus (Elsevier, Amsterdam, NL)], published between 2000 and 2009. RESULTS: About 98 of the Delphi studies purported to assess consensus, although a definition for consensus was only provided in 72 of the studies (64 a priori). The most common definition for consensus was percent agreement (25 studies), with 75% being the median threshold to define consensus. Although the authors concluded in 86 of the studies that consensus was achieved, consensus was only specified a priori (with a threshold value) in 42 of these studies. Achievement of consensus was related to the decision to stop the Delphi study in only 23 studies, with 70 studies terminating after a specified number of rounds. CONCLUSION: Although consensus generally is felt to be of primary importance to the Delphi process, definitions of consensus vary widely and are poorly reported. Improved criteria for reporting of methods of Delphi studies are required.

Expert beliefs regarding novel lipid-based approaches to pediatric intestinal failure-associated liver disease.

OBJECTIVE: To determine expert beliefs regarding the probability of intestinal failure-associated liver disease (IFALD) with novel lipid-based approaches (lipid minimization/ω-3 lipids) in managing IFALD to facilitate Bayesian analyses of clinical trials of these therapies. STUDY DESIGN: Structured interviews were conducted using a validated approach to belief elicitation with 60 intestinal failure (IF) experts from across North America. Participants were asked to estimate, in an average population of infants referred for management of IF with early IFALD, the probability of advanced IFALD at 3 months following referral in each of 3 scenarios: (1) conventional lipid, (2) ω-3 lipids, and (3) lipid minimization. Probability distributions of the risk of advanced IFALD with each strategy were developed. Distributions of the elicited treatment effect for the novel approaches, relative to conventional lipid, were calculated. RESULTS: Median duration of experience of participants managing patients with IF was 8.5 (range, 2-35) years. The median probability of advanced IFALD using conventional lipid was 32.5%; ω-3 lipids, 17.5%; and lipid minimization, 13%. The median of the elicited treatment effects relative to conventional lipid was a relative risk of 0.53 for the ω-3 lipid and 0.45 for lipid minimization. CONCLUSIONS: There was consistent expert opinion that the novel lipid-based approaches are superior to conventional therapy, with similar estimates of treatment efficacy for the 2 approaches. The distributions of the elicited treatment effects can be used as prior distributions in Bayesian analyses of clinical trials of these novel strategies.

Novel lipid-based approaches to pediatric intestinal failure-associated liver disease.

Historically, intestinal failure-associated liver disease (IFALD) has been the greatest contributor to the morbidity experienced by children with intestinal failure. Although the cause of IFALD is multifactorial, recently much attention has been devoted to the critical role that intravenous lipid emulsions play in the development of IFALD. This attention has prompted an interest in alternate approaches to the provision of intravenous lipid in children with IFALD. The 2 approaches that have been advanced are that of lipid minimization and alternate intravenous lipid emulsions, including those containing ω-3 fatty acids. This article examines the rationale and current evidence for these approaches in children with intestinal failure. Our overall finding is that although these alternate approaches show significant promise, they have primarily been studied in uncontrolled settings, mainly in children with advanced IFALD. As such, we believe that there remains a lack of definitive evidence for their efficacy. Furthermore, important safety parameters remain to be evaluated, including the effect of these therapies on growth and development. Therefore, there is currently insufficient evidence to support the use of these novel therapies as standard of care in children with no or early IFALD with the goal of preventing the progression of liver disease.

Graft type in pediatric liver transplantation.

PURPOSE OF REVIEW: To review the literature on graft type in pediatric liver transplantation, with a specific focus on publications since 2010. RECENT FINDINGS: Due to the limited availability of whole livers for transplantation, the majority of pediatric patients will receive a technical variant graft (live donor, reduced, split). Although the outcomes of these grafts may be inferior to whole organs, the detrimental impact needs to be balanced with the impact of technical variants on improved access and survival to transplantation. Vigilance in detecting and managing posttransplant complications is critical in ensuring the optimal outcome. Infants under 5 kg pose a particular challenge in terms of graft selection with hyperreduced and monosegment grafts proposed for this population. Grafts from donors after cardiac death show promise in expanding the donor pool. However, experience in pediatric patients with these grafts is quite limited, particularly in young children who form the majority of pediatric transplant recipients. Auxiliary transplantation is proposed as a strategy for children presenting with fulminant hepatic failure and for children with metabolic diseases. SUMMARY: The majority of children will receive a technical variant graft, with graft choice being largely determined by organ availability.

The role of parenteral lipids in the development of advanced intestinal failure-associated liver disease in infants: a multiple-variable analysis.

BACKGROUND: Given the recent interest in the role of ω-6 lipids in the development of intestinal failure-associated liver disease (IFALD), the authors sought to examine the role of parenteral lipids in the development of a serum conjugated bilirubin >100 µmol/L (5.9 mg/dL; CB100) in infants. METHOD: Between 2003 and 2004, data were collected prospectively on infants undergoing an abdominal surgical procedure. Univariate logistic regression models for the prediction of CB100 by 1 year postoperatively were developed. Predictors significant at the 0.2 level on univariate analysis were entered into a backward stepwise multiple variable logistic regression. RESULTS: Of 152 infants who received parenteral nutrition (PN) postoperatively, 22 developed CB100. Predictors that met criteria for consideration in the multiple-variable model were age, weight, small bowel length, presence of a stoma, proportion of enteral feeds postoperatively, septic episodes, days of maximal PN amino acid (>2.5 g/kg/d), days of maximal lipid (>2.5 g/kg/d), and PN duration. The final model included septic episodes (odds ratio, 3.23; 95% confidence interval, 1.8-5.9) and days of lipid >2.5 g/kg/d (1.04; 1.003-1.06). At 60 days of maximal lipid, the odds of advanced IFALD were increased 10-fold. CONCLUSIONS: This model suggests a key role of parenteral lipids and septic events in the development of CB100 from IFALD. These data may provide targets, such as careful line care, reduction in maximal lipid dose, or alternate lipids such as ω-3 fatty acids, to prevent CB100, an identified marker of subsequent liver failure from IFALD.

Complete vs partial fundoplication in children with esophageal atresia.

PURPOSE: The purpose of the study was to compare outcomes after partial vs complete fundoplication in patients with prior esophageal atresia repair. METHODS: All patients undergoing fundoplication following esophageal atresia repair at a tertiary care pediatric hospital from 1987 to 2006 were retrospectively reviewed. All children had at least 1 year of follow-up postfundoplication. RESULTS: Of 47 children, 31 (66%) had a partial fundoplication and 16 (34%) had complete fundoplication. Demographics, presence of tracheoesophageal fistula, early complications of esophageal atresia repair, gastroesophageal reflux symptoms before fundoplication, and operative details of fundoplication were statistically similar between groups, except for the frequency of hiatus repair during fundoplication (23% vs 69%, P = .004). Patients were followed for a median of 4.98 years (range, 1-17.8 years). Postfundoplication symptoms of vomiting (39% vs 31%), dysphagia (45% vs 38%), retching (10% vs 25%), abnormal findings on barium study, and need for reoperation (19% vs 13%) were not statistically different between groups. However, a greater proportion of children undergoing partial fundoplication achieved long-term symptom- and medication-free recovery (52% vs 13%, P = .012). CONCLUSIONS: Our data suggest that partial fundoplication is associated with a greater likelihood of symptom- and medication-free recovery than complete fundoplication in children with previously repaired esophageal atresia.

Does the colon play a role in intestinal adaptation in infants with short bowel syndrome? A multiple variable analysis.

PURPOSE: We sought to examine in a multiple variable model the impact of residual colonic length on time to intestinal adaptation in a cohort of infants with short bowel syndrome. METHOD: Infants with a surgical diagnosis of short bowel syndrome who underwent operation 90 days or younger were included in this analysis. Univariate Cox proportional hazards models for time to full-enteral feeds were developed. Predictors significant at the .2 level were entered into a stepwise multiple variable Cox proportional hazards model. RESULTS: A total of 106 infants were included in the cohort (70 adapted). Predictors meeting the criteria for the multiple variable model were as follows: multidisciplinary management (P = .045), Serial Transverse Enteroplasty Procedure (P = .057), percent small bowel (P < .001), percent large bowel (P < .001), preserved ileocecal valve (P = .001), number of septic (P < .001), and central line complications (P < .001). The final model included the following: multidisciplinary management (hazard ratio [HR], 1.932; 95% confidence interval [CI], 1.137-3.281), percent small bowel (HR, 1.028; 95% CI, 1.02-1.04), and septic events (HR, 0.695; 95% CI, 0.6-0.805). CONCLUSIONS: The colon does not seem to play a significant role in intestinal adaptation. However, in addition to highlighting the importance of residual small bowel length, our model highlights the benefit of multidisciplinary intestinal rehabilitation and reduction of septic complications in achieving intestinal adaptation.

Is the use of parenteral omega-3 lipid emulsions justified in surgical neonates with mild parenteral nutrition-associated liver dysfunction?

PURPOSE: Although evidence suggests that parenteral omega-3 lipid emulsions (O-3LEs) may be beneficial in treating advanced parenteral nutrition (PN)-associated liver disease, our objective was to determine if O-3LEs are justified in those with early liver disease. METHODS: This is a retrospective analysis of prospectively collected data on all surgical neonates, who received more than 1 day of PN postoperatively between 2001 and 2004 with observation through 2005 (era before O-3LE introduction). We examined the proportion of those who developed mild and advanced liver dysfunction. RESULTS: Of the 292 infants in the cohort, 104 (36%) developed mild liver dysfunction (conjugated bilirubin, 34 micromol/L [cBili34]) after a mean of 22 days. Thirty-one (30%) of the cBili34 patients reached a serum conjugated bilirubin of 100 micromol/L, and 13 (13%) developed liver failure. Of these, 4 underwent transplantation, and 5 died of hepatic disease. Overall, 86 of the cBili34 patients (83%) were weaned off PN. CONCLUSION: With more than 80% of cBili34 patients being weaned from PN without adverse hepatic sequelae, it is difficult, in the absence of definitive evidence of efficacy and safety for O-3LEs together with increased costs, to justify the routine use of O-3LEs in this low-risk population outside formal research protocols.

Outcomes after laparoscopic surgery in children with inflammatory bowel disease.

BACKGROUND: The utility and efficacy of the laparoscopic approach to the management of inflammatory bowel disease (IBD) in children are not clearly known. METHODS: We conducted a retrospective descriptive cohort study of children with a diagnosis of IBD who underwent a laparoscopic or laparoscopy-assisted procedure at a quaternary pediatric referral center between 1999 and 2007. RESULTS: One-hundred thirty-six children underwent 154 operations (85 small bowel/ileocolic and 69 colorectal) over the 8 years of the study. Median age was 14.8 years (range = 1.8-18.8). The diagnosis was Crohn's disease in 83, ulcerative colitis in 50, and indeterminate colitis in 3. Median time to regular diet was 5 days (range = 1-19), and median postoperative stay was 7 days (range = 1-70). Seven patients undergoing a small bowel/ileocolic resection (8.2%) were converted to an open procedure. Overall morbidity for the small bowel/ileocolic procedures was 27.1%. The conversion rate during subtotal colectomy (STC) was 7.1% (3/42), and it was 0% for the 22 patients who underwent ileal pouch-anal anastomosis (IPAA) procedures. Overall morbidity associated with STC was 62.8%, and following IPAA it was 63.6%. Sixteen percent (7/69) of those who underwent a colorectal procedure developed a late postoperative bowel obstruction with three patients requiring operative intervention. CONCLUSION: A laparoscopic approach is feasible with a low conversion rate in most children with IBD. Despite superior cosmesis, perioperative morbidity is similar to that seen with open procedures. Laparoscopic colorectal IBD procedures are associated with an unexpectedly high incidence of postoperative bowel obstruction, although the rates are comparable to those seen with open surgery.

Omega-3 lipids for intestinal failure associated liver disease.

Intestinal failure associated liver disease (IFALD) is one of the most common and devastating complications in infants with intestinal failure. Although multifactorial, its pathophysiology is clearly related to the administration of parenteral nutrition (PN), with a recent focus on the role of PN lipid emulsions. This paper will review the evidence for the use of omega-3 fatty acid PN lipid emulsions, which are proposed to have efficacy in the treatment of IFALD. Mechanisms explaining their effects will be considered as will future research directions.

Establishing norms for intestinal length in children.

BACKGROUND: Existing data on pediatric intestinal length (IL) are limited because most studies report postmortem values. Using prospective data, appropriate norms for IL were developed. METHOD: The IL measurements, using a silk suture on the antimesenteric border, were prospectively made on patients between 24 weeks of gestational age and 5 years of age undergoing laparotomy. Patients with gastrointestinal malformations or those above or below 2 SDs for growth parameters were excluded. A curve fitting process was applied to determine the best model for IL (small bowel and colon separately) from among postconception age, weight, and height at surgery. RESULTS: One hundred eight patients participated in this study. Highly predictive (R(2) > 0.8) models for IL were determined for all predictor variables (postconception age, weight, and height) examined suggesting that all of these variables are excellent predictors determinants of IL. Although all models had statistically similar properties, the model using height had the best performance across the full range of the variable. CONCLUSION: Although age, weight, nor height was definitely superior for the prediction of IL, we propose that until external validations of our models occur, height at surgery be used for the prediction of expected small intestinal and colon length in infants.

Pediatric blunt and penetrating trauma deaths in Ontario: a population-based study.

PURPOSE: The purpose of the study was to describe the mechanisms of injury and causes of death in children dying in a modern, integrated trauma system. METHOD: Records of all children (<16 years of age) who died in Ontario from 2001 through 2003 after blunt or penetrating trauma were obtained from the Chief Coroner. Demographics and the nature and causes of injury and the causes of death were recorded. Estimates of the mortality rate were determined using census data. RESULTS: There were 234 injury deaths (222 blunt, 12 penetrating) over the 3 years. Thirty (13%) resulted from intentional injury. The median age was 10 (range, 0-15.9) years; 62% were male. Sixty-eight percent resulted from incidents involving motor vehicles (passenger, pedestrian, or cyclist). Most (74%) died at the scene; only 5% survived for more than 24 hours. Devastating craniocervical injury (Abbreviated Injury Scale 5 or 6) was present in 84% and was the only life-threatening injury in 40%. The annual mortality rate averaged 3.2 per 100,000 children. CONCLUSIONS: In a modern, integrated trauma system, most pediatric injury deaths occur at the scene from severe head injuries. In this population, strategies to reduce the death rate from pediatric trauma must focus on primary and secondary injury prevention.

Extravasation of intravenous computed tomography scan contrast in blunt abdominal and pelvic trauma.

BACKGROUND: Intravenous contrast extravasation (CE) on computed tomography (CT) scan in blunt abdominal trauma is generally regarded as an indication for the need for invasive intervention (either angiography or laparotomy). More recently, improvements in CT scan technology have increased the sensitivity in detecting CE, and, thus, we postulate that not all patients with this finding require intervention. METHODS: This study is a retrospective review of all patients who underwent a CT scan for blunt abdominal trauma between January 1999 and September 2003. Patterns of injury, associated injuries, management, and outcomes were examined for patients with CE. RESULTS: Seventy of 1,435 patients (4.8%) demonstrated CE. Mean age was 44 years and mean Injury Severity Score was 39. The location of CE was intra-abdominal in 25, pelvis/retroperitoneum in 39, and both areas in 3 patients. Six patients received supportive treatment for nonsurvivable head injury and were excluded from further analysis. Overall, 30 (47%) patients underwent immediate intervention (angiography or laparotomy) and 34 (53%) were managed nonoperatively. Of those who had initial nonoperative management, overall seven (20.5%) underwent intervention, with the remainder being managed without intervention. The success for nonoperative management was greater for those with pelvic/retroperitoneal CE (4 of 7: 57%) than for intra-abdominal extravasation (23 of 27: 85%). CONCLUSION: Although evidence of CE may suggest significant vascular injury, our data suggest that not all patients require invasive intervention. Further studies are needed to better define criteria for nonoperative management in patients with CE identified on their initial CT scan.

Preventable pediatric trauma deaths in Ontario: a comparative population-based study.

INTRODUCTION: Previously, we demonstrated that 21% of pediatric (<16 years) trauma deaths in the Province of Ontario during the period 1985 to 1987 were potentially preventable. Since then many trauma system changes have occurred including field triage, designation of trauma centers, and improved injury prevention. This study aims to examine the current preventable trauma death rate in our system using identical methodology to our previous study. METHOD: The records of all children (<16 years) who died in Ontario from 2001 to 2003 after blunt or penetrating trauma were obtained from the Chief Coroner and compared with those in our previous report. In both series, we excluded cases where care was not sought and all deaths due to asphyxia. Deaths were considered unpreventable if the Injury Severity Score, based on Abbreviated Injury Scale 1985, was >59; or if there was a head injury that received an Abbreviated Injury Scale score of 5 with the exception of isolated extra-axial hematomas. RESULTS: Eleven preventable deaths were identified. The preventable death rate was 7%, a significant decline from the 21% previously identified (p < 0.001; relative risk reduction for preventable death, 68% [95% confidence interval, 42-83%]; number needed to treat, 7). CONCLUSION: There has been a threefold decline in the preventable death rate, which we believe is related to improvements in the trauma system. We estimated that, for every seven deaths from fatal injuries, system changes between the two study periods eliminated one preventable death.