Classification and Surgical Management of Anorectal Malformations: A Systematic Review and Evidence-based Guideline From the APSA Outcomes and Evidence-based Practice Committee.

OBJECTIVE: Treatment of neonates with anorectal malformations (ARMs) can be challenging due to variability in anatomic definitions, multiple approaches to surgical management, and heterogeneity of reported outcomes. The purpose of this systematic review is to summarize existing evidence, identify treatment controversies, and provide guidelines for perioperative care. METHODS: The American Pediatric Surgical Association Outcomes and Evidence Based Practice Committee (OEBP) drafted five consensus-based questions regarding management of children with ARMs. These questions were related to categorization of ARMs and optimal methods and timing of surgical management. A comprehensive search strategy was performed, and the Preferred Reporting Items for Systematic Reviews and Meta-Analyses (PRISMA) guidelines were used to perform the systematic review to attempt to answer five questions related to surgical care of ARM. RESULTS: A total of 10,843 publications were reviewed, of which 90 were included in final recommendations, and some publications addressed more than one question (question: 1 n = 6, 2 n = 63, n = 15, 4 n = 44). Studies contained largely heterogenous groups of ARMs, making direct comparison for each subtype challenging and therefore, no specific recommendation for optimal surgical approach based on outcomes can be made. Both loop and divided colostomy may be acceptable methods of fecal diversion for patients with a diagnosis of anorectal malformation, however, loop colostomies have higher rates of prolapse in the literature reviewed. In terms of timing of repair, there did not appear to be significant differences in outcomes between early and late repair groups. Clear and uniform definitions are needed in order to ensure similar populations of patients are compared moving forward. Recommendations are provided based primarily on A-D levels of evidence. CONCLUSIONS: Evidence-based best practices for ARMs are lacking for many aspects of care. Multi-institutional registries have made progress to address some of these gaps. Further prospective and comparative studies are needed to improve care and provide consensus guidelines for this complex patient population.

Screening and Prophylaxis for Venous Thromboembolism in Pediatric Surgery: A Systematic Review.

OBJECTIVE: The American Pediatric Surgical Association Outcomes and Evidence-Based Practice Committee conducted a systematic review to describe the epidemiology of venous thromboembolism (VTE) in pediatric surgical and trauma patients and develop recommendations for screening and prophylaxis. METHODS: The Medline (Ovid), Embase, Cochrane, and Web of Science databases were queried from January 2000 through December 2021. Search terms addressed the following topics: incidence, ultrasound screening, and mechanical and pharmacologic prophylaxis. The Preferred Reporting Items for Systematic reviews and Meta-Analyses (PRISMA) guidelines were followed. Consensus recommendations were derived based on the best available literature. RESULTS: One hundred twenty-four studies were included. The incidence of VTE in pediatric surgical populations is 0.29% (Range = 0.1%-0.48%) and directly correlates with surgery type, transfusion, prolonged anesthesia, malignancy, congenital heart disease, inflammatory bowel disease, infection, and female sex. The incidence of VTE in pediatric trauma populations is 0.25% (Range = 0.1%-0.8%) and directly correlates with injury severity, major surgery, central line placement, body mass index, spinal cord injury, and length-of-stay. Routine ultrasound screening for VTE is not recommended. Consider sequential compression devices in at-risk nonmobile, pediatric surgical patients when an appropriate sized device is available. Consider mechanical prophylaxis alone or with pharmacologic prophylaxis in adolescents >15 y and post-pubertal children <15 y with injury severity scores >25. When utilizing pharmacologic prophylaxis, low molecular weight heparin is superior to unfractionated heparin. CONCLUSIONS: While VTE remains an infrequent complication in children, consideration of mechanical and pharmacologic prophylaxis is appropriate in certain populations. TYPE OF STUDY: Systematic Review of level 2-4 studies. LEVEL OF EVIDENCE: Level 3-4.

Rare tumors II: Adrenocortical tumors, neuroendocrine tumors of the appendix, pheochromocytoma and paraganglioma, pancreatoblastoma and solid pseudopapillary tumors of the pancreas.

Very rare tumors are usually divided into two groups: One includes tumors that are rare among both children and adults; the other one encompasses tumors that frequently occur in adults but are rarely observed in children. In this review, we focus on adrenocortical tumors, neuroendocrine tumors of the appendix, pheochromocytoma and paraganglioma, pancreatoblastoma and solid pseudopapillary tumors of the pancreas, with special attention to the role of surgery as main curative intervention or as part of the multimodal treatment.

Development of an enhanced recovery after surgery program for pediatric solid tumors.

Introduction: Enhanced recovery after surgery (ERAS) is an evidence-based, multi-modal approach to decrease surgical stress, expedite recovery, and improve postoperative outcomes. ERAS is increasingly being utilized in pediatric surgery. Its applicability to pediatric patients undergoing abdominal tumor resections remains unknown. Methods and Analysis: A group of key stakeholders adopted ERAS principles and developed a protocol suitable for the variable complexity of pediatric abdominal solid tumor resections. A multi-center, prospective, propensity-matched case control study was then developed to evaluate the feasibility of the protocol. A pilot-phase was utilized prior to enrollment of all patients older than one month of age undergoing any abdominal, retroperitoneal, or pelvic tumor resections. The primary outcome was 90-day complications per patient. Additional secondary outcomes included: ERAS protocol adherence, length of stay, time to administration of adjuvant chemotherapy, readmissions, reoperations, emergency room visits, pain scores, opioid usage, and differences in Quality of Recovery 9 scores. Ethics and Dissemination: Institutional review board approval was obtained at all participating centers. Informed consent was obtained from each participating patient. The results of this study will be presented at pertinent society meetings and published in peer-reviewed journals. We expect the results will inform peri-operative care for pediatric surgical oncology patients and provide guidance on initiation of ERAS programs. We anticipate this study will take four years to meet accrual targets and complete follow-up. Trial Registration Number: NCT04344899.

Management and Outcomes of Pediatric Lymphatic Malformations: A Systematic Review From the APSA Outcomes and Evidence-Based Practice Committee.

BACKGROUND: Significant variation in management strategies for lymphatic malformations (LMs) in children persists. The goal of this systematic review is to summarize outcomes for medical therapy, sclerotherapy, and surgery, and to provide evidence-based recommendations regarding the treatment. METHODS: Three questions regarding LM management were generated according to the Preferred Reporting Items for Systematic Reviews and Meta-Analyses (PRISMA). Publicly available databases were queried to identify articles published from January 1, 1990, to December 31, 2021. A consensus statement of recommendations was generated in response to each question. RESULTS: The initial search identified 9326 abstracts, each reviewed by two authors. A total of 600 abstracts met selection criteria for full manuscript review with 202 subsequently utilized for extraction of data. Medical therapy, such as sirolimus, can be used as an adjunct with percutaneous treatments or surgery, or for extensive LM. Sclerotherapy can achieve partial or complete response in over 90% of patients and is most effective for macrocystic lesions. Depending on the size, extent, and location of the malformation, surgery can be considered. CONCLUSION: Evidence supporting best practices for the safety and effectiveness of management for LMs is currently of moderate quality. Many patients benefit from multi-modal treatment determined by the extent and type of LM. A multidisciplinary approach is recommended to determine the optimal individualized treatment for each patient.

Hepatic Metastasectomy in Pediatric Patients: An Observational Study.

BACKGROUND: The role of hepatectomy for metastatic disease in children is controversial. Rationales include potential cure, obtaining a diagnosis, and guiding chemotherapy decisions. This study examines the safety and utility of hepatic metastasectomy for children at a single institution. METHODS: After IRB approval (#22-1258), medical records were reviewed from 1995 to 2022 for children undergoing hepatic metastasectomy. En-bloc hepatectomies during primary tumor resection were excluded. RESULTS: Hepatic metastasectomy was performed in 16 patients for a variety of histologies. Median patient age was 12.2 years [IQR 6.9-22.6], and 13/16 patients were female (81 %). Number of hepatic metastases ranged from 1 to 23 and involved between 1 and 8 Couinaud segments. Anatomic resections included 4 hemihepatectomies and 1 sectionectomy. All other resections were nonanatomic. 3/6 resections for germ cell tumor (GCT) revealed only mature teratoma, driving adjuvant therapy decisions. When indicated, median time to adjuvant chemotherapy was 19 days [IQR 11-22]. No patients had Clavien-Dindo Class III or higher perioperative morbidity. Three patients (1 GCT, 1 adrenocortical carcinoma (ACC), and 1 gastric neuroendocrine tumor (GNET) experienced hepatic relapse. The patients with relapsed GCT and GNET are alive with disease at 17 and 135 months, respectively. The patient with ACC died of disease progression and liver failure. One patient with Wilms tumor experienced extrahepatic, retroperitoneal recurrence and died. With a median follow-up of 38 months, 10-year disease-specific and disease-free survival were 77 % and 61 %, respectively. CONCLUSIONS: Hepatic metastasectomy can be accomplished safely in children, may guide adjuvant therapy decisions, and is associated with long-term survival in selected patients. LEVEL OF EVIDENCE: Level IV. TYPE OF STUDY: Treatment Study, Case series with no comparison group.

Aggressive Pursuit of No Evidence of Disease Status in Hepatoblastoma Improves Survival: An Observational Study.

BACKGROUND: The utility of repeated surgical interventions in hepatoblastoma to achieve no evidence of disease (NED) is not well-defined. We examined the effect of aggressive pursuit of NED status on event-free (EFS) and overall survival (OS) in hepatoblastoma with subgroup analysis of high-risk patients. METHODS: Hospital records were queried for patients with hepatoblastoma from 2005 to 2021. Primary outcomes were OS and EFS stratified by risk and NED status. Group comparisons were performed using univariate analysis and simple logistic regression. Survival differences were compared with log-rank tests. RESULTS: Fifty consecutive patients with hepatoblastoma were treated. Forty-one (82%) were rendered NED. NED was inversely correlated with 5-year mortality (OR 0.006; CI 0.001-0.056; P < .01). Ten-year OS (P < .01) and EFS (P < .01) were improved by achieving NED. Ten-year OS was similar between 24 high-risk and 26 not high-risk patients when NED was attained (P = .83). Fourteen high-risk patients underwent a median of 2.5 pulmonary metastasectomies, 7 for unilateral disease, and 7 for bilateral, with a median of 4.5 nodules resected. Five high-risk patients relapsed, and three were salvaged. CONCLUSIONS: NED status is necessary for survival in hepatoblastoma. Repeated pulmonary metastasectomy and/or complex local control strategies to obtain NED can achieve long-term survival in high-risk patients. LEVEL OF EVIDENCE: Level III - Treatment Study - Retrospective Comparative Study.

Impact of insertion site on complications in central venous access devices.

PURPOSE: There is still debate over the safest route for the placement of long-term central venous access devices. The aim of this study was to review a large, single-institution experience to determine the impact of access location on peri-operative complications. METHODS: The records of patients undergoing subcutaneous port (SQP) and tunneled catheter insertion over a seven-year period were reviewed. Vein cannulated (subclavian (SCV) versus internal jugular (IJ) vein), and 30-day complications were assessed. Surgical complications included pneumothorax, hemothorax, infections, arrhythmia or malpositioning requiring intervention. RESULTS: A total of 1,309 patients were included (618 SQP, 691 tunneled catheters). The location for insertion was SCV (909, 69.4%) and IJ (400, 30.6%). There were 69 complications (5.2%) (41, 4.5% SCV, 28, 7.0% IJV) including: malpositioning/malfunctioning (SCV 13, 1.4% and IJV 14, 3.0%), pneumothorax (SCV 4, 0.4% and IJV 1, 0.3%), hemothorax (SCV 0 and IJV 1, 0.3%), arrhythmia (SCV 1, 0.1%, and IJV 0), and infection within 30 days of placement (SCV 20, 2.2% and IJ 11, 2.8%). The complication rates were not significantly different based on site (p = 0.080). CONCLUSION: There was no significant difference in complication rates when using the subclavian versus the internal jugular vein as the site for long-term central venous access. LEVEL OF EVIDENCE: III, retrospective comparative study.

Histologic type predicts disparate outcomes in pediatric hepatocellular neoplasms: A Pediatric Surgical Oncology Research Collaborative study.

BACKGROUND: Hepatocellular carcinoma (HCC) is a rare cancer in children, with various histologic subtypes and a paucity of data to guide clinical management and predict prognosis. METHODS: A multi-institutional review of children with hepatocellular neoplasms was performed, including demographic, staging, treatment, and outcomes data. Patients were categorized as having conventional HCC (cHCC) with or without underlying liver disease, fibrolamellar carcinoma (FLC), and hepatoblastoma with HCC features (HB-HCC). Univariate and multivariate analyses identified predictors of mortality and relapse. RESULTS: In total, 262 children were identified; and an institutional histologic review revealed 110 cHCCs (42%; 69 normal background liver, 34 inflammatory/cirrhotic, 7 unknown), 119 FLCs (45%), and 33 HB-HCCs (12%). The authors observed notable differences in presentation and behavior among tumor subtypes, including increased lymph node involvement in FLC and higher stage in cHCC. Factors associated with mortality included cHCC (hazard ratio [HR], 1.63; P = .038), elevated α-fetoprotein (HR, 3.1; P = .014), multifocality (HR, 2.4; P < .001), and PRETEXT (pretreatment extent of disease) stage IV (HR, 5.76; P < .001). Multivariate analysis identified increased mortality in cHCC versus FLC (HR, 2.2; P = .004) and in unresectable tumors (HR, 3.4; P < .001). Disease-free status at any point predicted survival. CONCLUSIONS: This multi-institutional, detailed data set allowed a comprehensive analysis of outcomes for children with these rare hepatocellular neoplasms. The current data demonstrated that pediatric HCC subtypes are not equivalent entities because FLC and cHCC have distinct anatomic patterns and outcomes in concert with their known molecular differences. This data set will be further used to elucidate the impact of histology on specific treatment responses, with the goal of designing risk-stratified algorithms for children with HCC. LAY SUMMARY: This is the largest reported granular data set on children with hepatocellular carcinoma. The study evaluates different subtypes of hepatocellular carcinoma and identifies key differences between subtypes. This information is pivotal in improving understanding of these rare cancers and may be used to improve clinical management and subsequent outcome in children with these rare malignancies.

Improving pathways to eliminate underrepresentation in the pediatric surgery workforce: A call to action.

Despite progress, diversity and minority representation within the pediatric surgery workforce still does not match the expansive backgrounds of the patients we treat. The problem stems from underrepresentation of minority populations at every step along the pediatric surgery training pathway. Strategies aimed at improving diversity and representation in medical school, general surgery residencies, and ultimately pediatric surgery fellowship are necessary to assemble a more diverse pool of pediatric surgeons. The aim of this paper is to review the current demographic make-up of medical and surgical specialties, highlight the value of diversity, and provide evidence-based strategies for increasing minority representation throughout the pediatric surgery pathway. Future patients will be better served with a more representative pediatric surgery workforce.

Impact of Neoadjuvant Chemotherapy on Image-Defined Risk Factors in High-Risk Neuroblastoma.

PURPOSE: Image-defined risk factors (IDRFs) are associated with surgical risks in neuroblastoma. We sought to evaluate the impact of neoadjuvant therapy on IDRFs and associated ability to achieve gross total resection (GTR) of locoregional disease in patients with high-risk neuroblastoma. METHODS: We retrospectively reviewed charts of patients treated on four consecutive high-risk neuroblastoma protocols over a 20-year period at a single institution. The number of IDRFs at diagnosis and just prior to surgery, and the percent decrease of tumor volume from just prior to surgery to the end of induction were determined. RESULTS: Eighty-eight patients were included. There were 438 IDRFs (average 5.0 ± 3.1 per patient) at diagnosis and 198 (average 2.3 ± 1.9 per patient) after neoadjuvant chemotherapy (p < 0.01). A reduction in IDRFs was seen in 81.8% of patients with average decrease of 2.9 ± 2.5 per patient. The average percent reduction in tumor volume was 89.8 ± 18.9% and correlated with the number of IDRFs present after chemotherapy (p < 0.01). Three or fewer IDRFs prior to surgery was associated with the highest odds ratio for > 90% GTR at 9.33 [95% confidence interval 3.14-31.5]. CONCLUSION: Neoadjuvant chemotherapy reduced the number of IDRFs in the majority of patients with high-risk neuroblastoma. The number of IDRFs present after neoadjuvant therapy correlated with the extent of resection.

Implications of Tumor Characteristics and Treatment Modality on Local Recurrence and Functional Outcomes in Children With Chest Wall Sarcoma: A Pediatric Surgical Oncology Research Collaborative Study.

OBJECTIVE: To determine the impact of tumor characteristics and treatment approach on (1) local recurrence, (2) scoliosis development, and (3) patient-reported quality of life in children with sarcoma of the chest wall. SUMMARY OF BACKGROUND DATA: Children with chest wall sarcoma require multimodal therapy including chemotherapy, surgery, and/or radiation. Despite aggressive therapy which places them at risk for functional impairment and scoliosis, these patients are also at significant risk for local recurrence. METHODS: A multi-institutional review of 175 children (median age 13 years) with chest wall sarcoma treated at seventeen Pediatric Surgical Oncology Research Collaborative institutions between 2008 and 2017 was performed. Patient-reported quality of life was assessed prospectively using PROMIS surveys. RESULTS: The most common diagnoses were Ewing sarcoma (67%) and osteosarcoma (9%). Surgical resection was performed in 85% and radiation in 55%. A median of 2 ribs were resected (interquartile range = 1-3), and number of ribs resected did not correlate with margin status ( P = 0.36). Local recurrence occurred in 23% and margin status was the only predictive factor(HR 2.24, P = 0.039). With a median follow-up of 5 years, 13% developed scoliosis (median Cobb angle 26) and 5% required corrective spine surgery. Scoliosis was associated with posteriorrib resection (HR 8.43; P= 0.003) and increased number of ribs resected (HR 1.78; P = 0.02). Overall, patient-reported quality of life is not impaired after chest wall tumor resection. CONCLUSIONS: Local recurrence occurs in one-quarter of children with chest wall sarcoma and is independent of tumor type. Scoliosis occurs in 13% of patients, but patient-reported quality of life is excellent.

Does epidural analgesia really enhance recovery in pediatric surgery patients?

PURPOSE: We sought to determine the benefits of epidural anesthesia (EA) in pediatric surgical patients. METHODS: This study is a single-institution retrospective review of EA for pediatric patients undergoing thoracotomy or laparotomy from 2015 to 2020. Patients with recent or chronic opioid use were excluded. Urgent or emergent cases, patients with hemodynamic instability, or those with surgical complications that significantly impacted their post-operative course were also excluded. The primary objectives were comparison of pain scores and systemic opioid use between those patients with EA and those without EA. RESULTS: Epidural anesthesia was used in 151 (81.6%) laparotomies and 58 (77.3%) thoracotomies. EA use was associated with lower mean systemic opioid administration during the early post-operative period for laparotomy (POD#0-0.33 ± 0.3 oral morphine equivalents per kilogram (OME/Kg) with EA vs 0.93 ± 1.53, p < 0.001, POD#1-1.34 ± 1.79 OME/Kg with EA vs 2.61 ± 2.60, p < 0.001) and thoracotomy (POD#0-0.40 ± 0.37 OME/Kg with EA vs 0.68 ± 0.41, p = 0.008, POD#1-0.89 ± 0.86 OME/Kg with EA vs 2.02 ± 1.92, p < 0.001). There were no differences seen by POD#2. Average pain scores were significantly lower in patients with EA following laparotomy (POD#0-1.22 ± 0.99 with EA vs 1.75 ± 1.33, p = 0.008) and thoracotomy (POD#0-1.71 ± 1.13 with EA vs 2.40 ± 1.52, p = 0.04). CONCLUSIONS: The use of EA in pediatric surgery patients was associated with lower pain scores despite lower systemic opioid requirements in the early post-operative period.

Thoracoscopy vs thoracotomy for the management of metastatic osteosarcoma: A Pediatric Surgical Oncology Research Collaborative Study.

Complete surgical resection of pulmonary metastatic disease in patients with osteosarcoma is crucial to long-term survival. Open thoracotomy allows palpation of nodules not identified on imaging but the impact on survival is unknown. The objective of this study was to compare overall survival (OS) and pulmonary disease-free survival (DFS) in children who underwent thoracotomy vs thoracoscopic surgery for pulmonary metastasectomy. A multi-institutional collaborative group retrospectively reviewed 202 pediatric patients with osteosarcoma who underwent pulmonary metastasectomy by thoracotomy (n = 154) or thoracoscopy (n = 48). Results were analyzed by Kaplan-Meier survival estimates and multivariate Cox proportional hazard regression models. With median follow-up of 45 months, 135 (67.5%) patients had a pulmonary relapse and 95 (47%) patients were deceased. Kaplan-Meier analysis showed no significant difference in 5-year pulmonary DFS (25% vs 38%; P = .18) or OS (49% vs 42%, P = .37) between the surgical approaches of thoracotomy and thoracoscopy. In Cox regression analysis controlling for other factors impacting outcome, there was a significantly increased risk of mortality (HR 2.11; P = .027; 95% CI 1.09-4.09) but not pulmonary recurrence (HR 0.96; P = .90; 95% CI 0.52-1.79) with a thoracoscopic approach. However, in the subset analysis limited to patients with oligometastatic disease, thoracoscopy had no increased risk of mortality (HR 1.16; P = .62; 0.64-2.11). In conclusion, patients with metastatic osteosarcoma and limited pulmonary disease burden demonstrate comparable outcomes after thoracotomy and thoracoscopy for metastasectomy. While significant selection bias in these surgical cohorts limits the generalizability of the conclusions, clinical equipoise for a randomized clinical trial in patients with oligometastatic disease is supported.

Vascular tumors.

Vascular tumors are a rare subset of vascular anomalies. These are classified based on their malignant potential or local destruction potential. Classification has been historically difficult and treatment recommendations are based on case series. The purpose of this chapter is to review the presentation, pathologic and imaging characteristics. Treatment recommendations are summarized based on the current literature. Congenital and infantile hemangiomas are covered separately in a separate chapter in this issue.

Alternative approaches to retroperitoneal lymph node dissection for paratesticular rhabdomyosarcoma.

PURPOSE: The aim of this study was to evaluate outcomes based on surgical approach for retroperitoneal lymph node dissection (RPLND) in patients with paratesticular rhabdomyosarcoma (PT-RMS). METHODS: Patients undergoing RPLND for PT-RMS over 10 years at a single institution were retrospectively reviewed. Length of stay (LOS), complications, oral morphine equivalents per kilogram (OME/Kg), lymph node yield, and time to chemotherapy were assessed. The surgical approaches compared were: open transabdominal, open extraperitoneal, laparoscopic, and retroperitoneoscopic. For cases with lymphatic mapping, indocyanine green (ICG) was injected into the spermatic cord. RESULTS: Twenty patients were included: five open transabdominal, six open extraperitoneal, three laparoscopic, and six retroperitoneoscopic operations. LOS was shorter in the retroperitoneoscopic group than laparoscopic (p = 0.029) and both open groups (p < 0.001). Mean OME/kg used was lowest in the retroperitoneoscopic (0.13 ±â€¯0.15) group compared to laparoscopic (0.68 ±â€¯0.53, p = 0.043), open transabdominal (14.90 ±â€¯8.87, p = 0.003), and extraperitoneal (10.11 ±â€¯2.44, p < 0.001). Time to chemotherapy was shorter for retroperitoneoscopic patients (0.13 days ±â€¯0.15) compared to open transabdominal (15.6 days±6.5, p = 0.005). There was no difference in lymph node yield between groups. Spermatic cord ICG demonstrated iliac lymph node avidity on near-infrared spectroscopy. CONCLUSIONS: Minimally invasive RPLND appears to offer a faster recovery without compromising lymph node yield for patients with PT-RMS. LEVEL OF EVIDENCE: III.

Optimization of percutaneous biopsy for diagnosis and pretreatment risk assessment of neuroblastoma.

BACKGROUND: Image-guided percutaneous core needle biopsy (PCNB) is increasingly utilized to diagnose solid tumors. The objective of this study is to determine whether PCNB is adequate for modern biologic characterization of neuroblastoma. PROCEDURE: A multi-institutional retrospective study was performed by the Pediatric Surgical Oncology Research Collaborative on children with neuroblastoma at 12 institutions over a 3-year period. Data collected included demographics, clinical details, biopsy technique, complications, and adequacy of biopsies for cytogenetic markers utilized by the Children's Oncology Group for risk stratification. RESULTS: A total of 243 children were identified with a diagnosis of neuroblastoma: 79 (32.5%) tumor excision at diagnosis, 94 (38.7%) open incisional biopsy (IB), and 70 (28.8%) PCNB. Compared to IB, there was no significant difference in ability to accurately obtain a primary diagnosis by PCNB (95.7% vs 98.9%, P = .314) or determine MYCN copy number (92.4% vs 97.8%, P = .111). The yield for loss of heterozygosity and tumor ploidy was lower with PCNB versus IB (56.1% vs 90.9%, P < .05; and 58.0% vs. 88.5%, P < .05). Complications did not differ between groups (2.9 % vs 3.3%, P = 1.000), though the PCNB group had fewer blood transfusions and lower opioid usage. Efficacy of PCNB was improved for loss of heterozygosity when a pediatric pathologist evaluated the fresh specimen for adequacy. CONCLUSIONS: PCNB is a less invasive alternative to open biopsy for primary diagnosis and MYCN oncogene status in patients with neuroblastoma. Our data suggest that PCNB could be optimized for complete genetic analysis by standardized protocols and real-time pathology assessment of specimen quality.