Esophageal Surveillance Practices in Esophageal Atresia Patients: A Survey by the Eastern Pediatric Surgery Network.

INTRODUCTION: Endoscopic surveillance guidelines for patients with repaired esophageal atresia (EA) rely primarily on expert opinion. Prior to embarking on a prospective EA surveillance registry, we sought to understand EA surveillance practices within the Eastern Pediatric Surgery Network (EPSN). METHODS: An anonymous, 23-question Qualtrics survey was emailed to 181 physicians (surgeons and gastroenterologists) at 19 member institutions. Likert scale questions gauged agreement with international EA surveillance guideline-derived statements. Multiple-choice questions assessed individual and institutional practices. RESULTS: The response rate was 77%. Most respondents (80%) strongly agree or agree that EA surveillance endoscopy should follow a set schedule, while only 36% claimed to perform routine upper GI endoscopy regardless of symptoms. Many institutions (77%) have an aerodigestive clinic, even if some lack a multi-disciplinary EA team. Most physicians (72%) expressed strong interest in helping develop evidence-based guidelines. CONCLUSIONS: Our survey reveals physician agreement with current guidelines but weak adherence. Surveillance methods vary greatly, underscoring the lack of evidence-based data to guide EA care. Aerodigestive clinics may help implement surveillance schedules. Respondents support evidence-based protocols, which bodes well for care standardization. Results will inform the first multi-institutional EA databases in the United States (US), which will be essential for evidence-based care. LEVEL OF EVIDENCE: This is a prognosis study with level 4 evidence.

Dollars and Sense: The Business of Pediatric Surgery.

INTRODUCTION: This study evaluated North American pediatric surgeons' opinions and knowledge of business and economics in medicine and their perceptions of trends in their healthcare delivery environment. METHODS: We conducted an elective online survey of 1119 American Pediatric Surgical Association members. Over 8 mo, we iteratively developed the survey focused on four areas: opinion, knowledge, current practice environment, and trends in practice environment over the past 5 y. RESULTS: We received 227 (20.3%) complete surveys from pediatric surgeons. One hundred ninety four (85.5%) perceive healthcare as a business and most (85.9%) believe healthcare decisions may affect patients' out-of-pocket expenses. More than half (51.1%) of surgeons believe it has become more challenging to perform emergent cases and most believe staff quality has decreased for elective (56.4%) and emergent (63.0%) cases over the past 5 y. CONCLUSIONS: Pediatric surgeons recognize that medicine is a business and have concerns regarding the decreasing quality of operating room staff and the increasing difficulty providing surgical care over the last 5 y.

Congenital Diaphragmatic Hernia Repair at the Bedside or Operating Theater.

BACKGROUND: For critically ill congenital diaphragmatic hernia (CDH) patients on high frequency oscillatory ventilation (HFOV), extracorporeal membrane oxygenation (ECMO), and/or inhaled nitric oxide (iNO), operative repair in the neonatal intensive care unit (NICU) has been proposed to avoid complications during transport to an operating room (OR). This study compared neonates with CDH who received herniorrhaphy in the NICU or OR, with a subgroup analysis considering only patients supported with ECMO. METHODS: Patients admitted to the NICU in the first 2 weeks of life at a free-standing children's hospital between July 2004 and September 2021 were examined. Patients were categorized according to location of CDH repair, and impact on operative complications and survival was compared. RESULTS: 185 patients were admitted to the NICU with posterolateral CDH and received operative repair. 48 cases were operated on at the bedside in the NICU and 137 in the OR. Patients repaired in the NICU had higher use of HFOV, pulmonary vasodilators, and ECMO (all P < .001). Children repaired in the NICU experienced significantly higher in-hospital death and overall mortality (P < .001). However, in multivariate analysis, repair location was not a significant predictor of survival to discharge in patients receiving ECMO. No significant difference in surgical site infection was detected for operative location (P = .773). DISCUSSION: Congenital diaphragmatic hernia repair in the NICU occurred more frequently among higher risk patients who experienced worse survival. The rate of surgical site infection appeared similar overall and across subgroups suggesting adequate sterility and technique for bedside procedures, when necessary, despite restricted access to advanced operative equipment.

Surgeon perceptions of volume threshold and essential practices for pediatric thyroidectomy✰.

INTRODUCTION: The topics of sub-specialization and regionalization of care have garnered increased attention among pediatric surgeons. Thyroid surgeries are one such sub-specialty and are commonly concentrated within practices. A national survey was conducted examining current surgeon practices and beliefs surrounding pediatric thyroid surgery. METHODS: Non-resident members of the American Pediatric Surgical Association (APSA) were surveyed in October 2020. Respondents were stratified based on self-reported thyroid surgical experience. Those who performed thyroid surgery were asked about surgical technique and operative practices; those who did not were asked about referral patterns. All respondents were asked about perceptions surrounding the volume-outcome relationship for pediatric thyroid surgery. RESULTS: Among 1015 APSA members, 405 (40%) responded, with 79% (317/400) practicing at academic hospitals, 58% (232/401) practicing in major metropolitan area, and 41% (161/392) with over 10 years of attending pediatric surgery experience. Most respondents (88%, n = 356) agreed that thyroid surgery volume affects outcome, though wide variation was reported in the annual case threshold for "high volume" surgery. Eighty-four respondents (21%) reported performing ≥ 1 pediatric thyroid surgery in the past year. Of these, 82% routinely use recurrent laryngeal nerve monitoring, 32% routinely send hemithyroidectomy patients home the same day, and there was little consensus surrounding postoperative hypocalcemia management. The majority of respondents endorse performing thyroid procedures with a colleague. CONCLUSIONS: Pediatric thyroid surgery appears to be performed by a subset of active pediatric surgeons, most of whom endorse the use of a dual operating team. More evidence is needed to build consensus around additional perioperative practices.

Rare and de novo variants in 827 congenital diaphragmatic hernia probands implicate LONP1 as candidate risk gene.

Congenital diaphragmatic hernia (CDH) is a severe congenital anomaly that is often accompanied by other anomalies. Although the role of genetics in the pathogenesis of CDH has been established, only a small number of disease-associated genes have been identified. To further investigate the genetics of CDH, we analyzed de novo coding variants in 827 proband-parent trios and confirmed an overall significant enrichment of damaging de novo variants, especially in constrained genes. We identified LONP1 (lon peptidase 1, mitochondrial) and ALYREF (Aly/REF export factor) as candidate CDH-associated genes on the basis of de novo variants at a false discovery rate below 0.05. We also performed ultra-rare variant association analyses in 748 affected individuals and 11,220 ancestry-matched population control individuals and identified LONP1 as a risk gene contributing to CDH through both de novo and ultra-rare inherited largely heterozygous variants clustered in the core of the domains and segregating with CDH in affected familial individuals. Approximately 3% of our CDH cohort who are heterozygous with ultra-rare predicted damaging variants in LONP1 have a range of clinical phenotypes, including other anomalies in some individuals and higher mortality and requirement for extracorporeal membrane oxygenation. Mice with lung epithelium-specific deletion of Lonp1 die immediately after birth, most likely because of the observed severe reduction of lung growth, a known contributor to the high mortality in humans. Our findings of both de novo and inherited rare variants in the same gene may have implications in the design and analysis for other genetic studies of congenital anomalies.

Mucinous Adenocarcinoma With Intrapulmonary Metastasis Harboring KRAS and GNAS Mutations Arising in Congenital Pulmonary Airway Malformation.

OBJECTIVES: Mucinous adenocarcinoma arising in unresected congenital pulmonary airway malformation (CPAM) is rare. Underlying driver mutations in addition to KRAS gain-of-function mutations in this setting and the long-term outcomes of these patients are unknown. METHODS: We report a case of metastatic mucinous adenocarcinoma harboring both KRAS and GNAS mutations arising in a type 1 CPAM of a 14-year-old male. A literature review was performed. RESULTS: Next-generation sequencing revealed identical KRAS (G12V) mutations in both the CPAM and metastatic adenocarcinoma and a missense mutation in the GNAS (R201C) gene in the metastatic adenocarcinoma only. Median survival was 23 and 4 years for patients with localized (no or limited spread within the same lobe of CPAM) and distant involvement (spread to any different lobe of CPAM) of mucinous cells, respectively (95% confidence interval, 23-23 and 1.5-22 years, respectively; P = .017). CONCLUSIONS: Mucinous cell proliferation associated with type 1 CPAM has exceptionally good long-term outcomes if confined within the same lobe of CPAM. A second oncogenic mutation in the GNAS gene may be necessary for progression to malignancy and distant spread.

Short Bowel Syndrome in an Infant.

Short bowel syndrome (SBS) is a malabsorptive state that may occur either after surgical bowel resection or as the result of congenital bowel anomalies. SBS can incur significant morbidity and mortality including intestinal failure, cholestasis, sepsis, and death. For patients with SBS, management involves a multidisciplinary approach that begins with neonatology, pediatric surgery, nutritionists, pharmacists, and nurses in the NICU and also includes the transition to an intestinal rehabilitation program. The aim of this review is to provide the neonatologist with an overview of the common causes of neonatal SBS, anticipated nutritional deficiencies, complications associated with SBS, and the surgical and medical management of SBS to assist in counseling affected families.

Comparative outcomes of right versus left congenital diaphragmatic hernia: A multicenter analysis.

BACKGROUND: Congenital diaphragmatic hernia (CDH) occurs in 1 out of 2500-3000 live births. Right-sided CDHs (R-CDHs) comprise 25% of all CDH cases, and data are conflicting on outcomes of these patients. The aim of our study was to compare outcomes in patients with right versus left CDH (L-CDH). METHODS: We analyzed a multicenter prospectively enrolled database to compare baseline characteristics and outcomes of neonates enrolled from January 2005 to January 2019 with R-CDH vs. L-CDH. RESULTS: A total of 588, 495 L-CDH, and 93 R-CDH patients with CDH were analyzed. L-CDHs were more frequently diagnosed prenatally (p=0.011). Lung-to-head ratio was similar in both cohorts. R-CDHs had a lower frequency of primary repair (p=0.022) and a higher frequency of need for oxygen at discharge (p=0.013). However, in a multivariate analysis, need for oxygen at discharge was no longer significantly different. There were no differences in long-term neurodevelopmental outcomes assessed at two year follow up. There was no difference in mortality, need for ECMO, pulmonary hypertension, or hernia recurrence. CONCLUSION: In this large series comparing R to L-CDH patients, we found no significant difference in mortality, use of ECMO, or pulmonary complications. Our study supports prior studies that R-CDHs are relatively larger and more often require a patch or muscle flap for repair. TYPE OF STUDY: Prognosis study LEVEL OF EVIDENCE: Level II.

Improving the value of care for appendectomy through an individual surgeon-specific approach.

PURPOSE: Standardized care via a unified surgeon preference card for pediatric appendectomy can result in significant cost reduction. The purpose of this study was to evaluate the impact of cost and outcome feedback to surgeons on value of care in an environment reluctant to adopt a standardized surgeon preference card. METHODS: Prospective observational study comparing operating room (OR) supply costs and patient outcomes for appendectomy in children with 6-month observation periods both before and after intervention. The intervention was real-time feedback of OR supply cost data to individual surgeons via automated dashboards and monthly reports. RESULTS: Two hundred sixteen children underwent laparoscopic appendectomy for non-perforated appendicitis (110 pre-intervention and 106 post-intervention). Median supply cost significantly decreased after intervention: $884 (IQR $705-$1025) to $388 (IQR $182-$776), p<0.001. No significant change was detected in median OR duration (47min [IQR 36-63] to 50min [IQR 38-64], p=0.520) or adverse events (1 [0.9%] to 6 [4.7%], p=0.062). OR supply costs for individual surgeons significantly decreased during the intervention period for 6 of 8 surgeons (87.5%). CONCLUSION: Approaching value measurement with a surgeon-specific (rather than group-wide) approach can reduce OR supply costs while maintaining excellent clinical outcomes. LEVEL OF EVIDENCE: Level II.

Review of 1,000 consecutive extracorporeal membrane oxygenation runs as a quality initiative.

BACKGROUND: Extracorporeal membrane oxygenation is a resource-intensive mode of life-support potentially applicable when conventional therapies fail. Given the initial success of extracorporeal membrane oxygenation to support neonates and infants in the 1980s, indications have expanded to include adolescents, adults, and selected moribund patients during cardiopulmonary resuscitation. This single-institution analysis was conducted to evaluate programmatic growth, outcomes, and risk for death despite extracorporeal membrane oxygenation across all ages and diseases. METHODS: Beginning in 1989, we registered prospectively all extracorporeal membrane oxygenation patient data with the Extracorporeal Life Support Organization. We queried this registry for our institution-specific data to compare the parameter of "discharge alive" between age groups (neonatal, pediatric, adult), disease groups (respiratory, cardiac, cardiopulmonary resuscitation), and modes of extracorporeal membrane oxygenation (veno-venous; veno-arterial). Extracorporeal membrane oxygenation-specific complications (mechanical, hemorrhagic, neurologic, renal, cardiovascular, pulmonary, infectious, metabolic) were analyzed similarly. Descriptive statistics, Kaplan-Meier, and linear regression analyses were conducted. RESULTS: After 1,052 extracorporeal membrane oxygenation runs, indications have expanded to include adults, to supplement cardiopulmonary resuscitation, to support hemodialysis in neonates and plasmapheresis in children, and to bridge all age patients to heart and lung transplant. Overall survival to discharge was 52% and was better for respiratory diseases (P < .001). Probability of individual survival decreased to <50% if pre-extracorporeal membrane oxygenation mechanical ventilation exceeded respectively 123 hours for cardiac, 166 hours for cardiopulmonary resuscitation, and 183 hours for respiratory diseases (P = .013). Complications occurred most commonly among cardiac and cardiopulmonary resuscitation runs (P < .001), the veno-arterial mode (P < .001), and in adults (P = .044). CONCLUSION: Our extracorporeal membrane oxygenation program, an Extracorporeal Life Support Organization-designated Center of Excellence, has experienced substantial growth in volume and indications, including increasing age and disease severity. Considering the entire cohort, pre-extracorporeal membrane oxygenation ventilation exceeding 7 days was associated with an increased probability of death.

Tumor proximity to the recurrent laryngeal nerve in patients with primary hyperparathyroidism undergoing parathyroidectomy.

BACKGROUND: Recurrent laryngeal nerve (RLN) injury is a rare complication for patients undergoing neck exploration for primary hyperparathyroidism (pHPT). Distances between RLNs and parathyroid adenomas have not been previously published. In this study we used a RLN monitor to identify the RLN and to measure the proximity to parathyroid tumors. METHODS: Patients with pHPT (n = 136) underwent neck exploration and had the clinical data recorded prospectively. Adenomas were recorded in 1 of 4 locations (right upper, right lower, left upper, left lower). Measurement of RLN to adenoma distances were recorded intraoperatively with the gland in situ. The RLN location was confirmed with a RLN monitor. RESULTS: The average RLN to adenoma distance was 0.52 ± 0.52 cm. Adenomas in the right upper position were significantly closer to the nerve (0.25 ± 0.39 cm) compared with adenomas in the left upper (0.48 ± 0.61 cm, p = .03), left lower (0.70 ± 0.53 cm, p < .001), and right lower position (1.02 ± 0.56 cm, p < .001). Left upper adenomas were also significantly closer to the nerve compared with right lower adenomas (p < .001). Adenomas in the right upper position abutted the nerve more often (47 %) compared with adenomas in other positions (p = .001). There were no perioperative characteristics that predicted tumor abutment. There were no permanent RLN injuries. CONCLUSION: In patients with sporadic pHPT, parathyroid adenomas in the right upper location have, on average, greater proximity to the RLN and are more often directly abutting compared with adenomas in other locations.

Severe obesity is associated with symptomatic presentation, higher parathyroid hormone levels, and increased gland weight in primary hyperparathyroidism.

CONTEXT: A relationship between primary hyperparathyroidism (PHPT) and obesity has been observed but is incompletely understood. Furthermore, obesity has been associated with vitamin D deficiency, suggesting that the three conditions may be linked. OBJECTIVE: We hypothesized that PHPT in morbidly obese patients is more severe and that the difference may be explained by vitamin D deficiency. DESIGN AND SETTING, PARTICIPANTS, AND OUTCOME MEASURES: Records of 196 patients with surgically treated PHPT and known body mass index (BMI) were examined. Patients were stratified into three BMI groups: group I (nonobese), BMI < 25 kg/m(2) (n = 54); group II (non-severely obese), BMI 25-34 kg/m(2) (n = 102); and group III (severely obese), BMI 35 kg/m(2) or greater (n = 40). RESULTS: Preoperative PTH levels were higher in group ΙΙΙ compared with group Ι (181 ± 153 vs. 140 ± 80 pg/ml, p = 0.04). Group III patients had larger tumors on average compared with group I (1.8 ± 1.5 vs. 1.04 ± 1.5 g, P = 0.0002). In group III, BMI positively correlated with parathyroid tumor weight (r = 0.5, P = 0.002). Postoperative PTH was higher in group III compared with group Ι (61 ± 41 vs. 44 ± 28 pg/ml, P = 0.02). There was higher frequency of depression, musculoskeletal symptoms, weakness, and gastroesophageal reflux disease in group III patients. CONCLUSIONS: BMI positively correlated with parathyroid tumor weight independent of vitamin D. Severely obese patients have larger parathyroid tumor weight, higher pre- and postoperative PTH, and greater symptoms.

Protocolized approach to the management of congenital diaphragmatic hernia: benefits of reducing variability in care.

PURPOSE: Variable approaches to the care of infants with congenital diaphragmatic hernia (CDH) by multiple providers may contribute to inconsistent care. Our institution developed a comprehensive evidence-based protocol to standardize the management of CDH infants. This report reviews patient outcomes before and after the implementation of the protocol. METHODS: Retrospective chart review of CDH infants managed with individualized care (preprotocol group, January 1997-December 2001, n = 22) or on the protocol (Protocol group, January 2002-July 2009, n = 47). Survival and other categorical variables were compared by chi(2) analysis, and continuous variables were compared using 1-sided analysis of variance analysis, with significance defined as P < .05. RESULTS: Survival to discharge was significantly greater in the Protocol group (40/47; 85%) than the preprotocol group (12/22; 52%; P = .006), although mean gestational age, mean birth weight, and expected survival were not statistically different between the 2 groups. The use of supportive therapies, including high-frequency jet ventilation, inhaled nitric oxide, and extracorporeal life support, was similar between groups as well. CONCLUSIONS: Since the implementation of a management protocol for infants with CDH, survival has improved significantly compared with expected survival and preprotocol controls. Reduction in the variability of care through use of an evidence-based protocol may improve the survival of CDH infants.

Improved staging in node-positive breast cancer patients using lymph node ratio: results in 1,788 patients with long-term follow-up.

BACKGROUND: Axillary lymph node status remains the most important prognostic factor in breast cancer. Established staging systems emphasize the absolute number of positive nodes, without regard for the total number of lymph nodes examined. We sought to confirm that a ratio of positive nodes to total nodes examined (LNR) has prognostic value beyond the current TNM classification for women with node-positive breast cancer. STUDY DESIGN: Using the Duke University Medical Center breast cancer tumor registry, we identified women diagnosed with node-positive breast cancer between 1985 and 2005 (n = 1,788). Variables analyzed for impact on disease-free survival (DFS) included the number of positive nodes, N classification, and the calculated LNR. Based on LNR, the patients were divided into low- (< or =0.2), intermediate- (>0.2 and < or =0.65), and high- (>0.65) risk groups. Kaplan-Meier survival analysis was performed with groups compared by the log-rank test. Values of p < 0.05 were considered significant. RESULTS: For all patients, the 10-year actuarial DFS rates for the low-, intermediate-, and high-risk LNR groups were 69%, 60%, and 45%, respectively. The DFS curves for the 3 LNR groups were significantly different (p < 0.0001). Furthermore, when patients were stratified by pN status, the DFS curves for the LNR groups remained significantly different. The LNR discerned groups of patients with divergent survival probabilities across all pN groups. CONCLUSIONS: Our data show that LNR has prognostic value in assessing breast cancer survival beyond the current TNM classification. This study supports the inclusion of LNR for enhanced risk stratification beyond traditional pN classification.

Low volume is associated with worse patient outcomes for pediatric liver transplant centers.

BACKGROUND: An inverse association between hospital procedure volume and postoperative mortality has been demonstrated for a variety of pediatric surgical procedures. The objective of our study was to determine whether such an association exists for pediatric liver transplantation. METHODS: We performed a retrospective analysis of pediatric liver transplant procedures included in the Scientific Registry of Transplant Recipients over a 7.5-year time period from July 1, 2000, through December 31, 2007. Pediatric liver transplant centers were divided into three volume categories (high, middle, low) based on absolute annual volume. Mean 1-year patient survival rates and aggregate 1-year observed-to-expected (O:E) patient death ratios were calculated for each hospital volume category and then compared using ordered logistic regression and chi square analyses. RESULTS: High-volume pediatric liver transplant centers achieved significantly lower aggregate 1-year O:E patient death ratios than low-volume centers. When freestanding children's hospitals (FCH), children's hospitals within adult hospitals (CAH), and other centers (OC) were considered separately, we found that a significant volume-outcomes association existed among OC centers but not among FCH or CAH centers. Low-volume OC centers, which represent 41.6% of all pediatric liver transplant centers and perform 10% of all pediatric liver transplantation, had the least favorable aggregate 1-year O:E patient death ratio of all groups. CONCLUSIONS: We demonstrate that a significant center volume-outcomes relationship exists among OC pediatric liver transplant centers but not among FCH or CAH centers. These findings support the possible institution of minimum annual procedure volume requirements for OC pediatric liver transplant centers.

Surgical intervention in children with multiple endocrine neoplasia type 2.

PURPOSE OF REVIEW: We provide a summary of the literature published in the past year addressing the surgical approach to multiple endocrine neoplasia type 2 in the pediatric population. RECENT FINDINGS: The review focuses first on medullary thyroid carcinoma and performing prophylactic thyroidectomy for the prevention or cure of this disease. The timing and extent of surgery as well as additional surgical intervention for persistent or recurrent disease is discussed. Then the surgical management of hereditary pheochromocytoma is reviewed. SUMMARY: Surgery is often the only treatment that can prevent or cure the endocrinopathies associated with multiple endocrine neoplasia type 2. Determining the proper timing and extent of surgical intervention in children affected with multiple endocrine neoplasia type 2 will lead to better outcomes and survival.