Current surgical management of children with osteosarcoma and pulmonary metastatic disease: A survey of the American Pediatric Surgical Association.

BACKGROUND: Rates of long-term survival for children with pulmonary metastatic osteosarcoma are low, and complete surgical resection of all visible pulmonary metastases is necessary for long term survival. Surgical approaches for metastasectomy include thoracotomy and thoracoscopy, with the approach chosen influenced by training and institutional bias. Thoracotomy with manual palpation of lung surfaces can identify nodules not seen on preoperative imaging, but no clear survival benefit has been demonstrated compared to complete thoracoscopic resection of all visible nodules. METHODS: All member of the American Pediatric Surgical Association were surveyed, and 204 members responded. RESULTS: Thoracoscopy was the preferred approach of 34% of surgeons for patients with 3 unilateral nodules but only 21% for those with 5 unilateral nodules. Hospital volume did not correlate with operative approach. Localization strategies are used by 37% of surgeons who prefer thoracotomy and 64% who prefer thoracoscopy. Importantly, the vast majority of responding surgeons (84%) expressed a willingness to participate in a randomized controlled trial of thoracotomy versus thoracoscopy. CONCLUSION: Findings of this survey of North American pediatric surgeons confirm both the need for, and interest in, a prospective trial to define optimal surgical management of children with osteosarcoma with limited pulmonary metastasis. LEVEL OF EVIDENCE: V.

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.

Prospective Evaluation of Radiation Dose Escalation in Patients With High-Risk Neuroblastoma and Gross Residual Disease After Surgery: A Report From the Children's Oncology Group ANBL0532 Study.

PURPOSE: A primary objective of the Children's Oncology Group (COG) ANBL0532 phase III study was to assess the effect of increasing local dose of radiation to a residual primary tumor on the cumulative incidence of local progression (CILP) in patients with high-risk neuroblastoma. PATIENTS AND METHODS: Newly diagnosed patients with high-risk neuroblastoma were randomly assigned or assigned to receive single or tandem autologous stem-cell transplantation (SCT) after induction chemotherapy. Local control consisted of surgical resection during induction chemotherapy and radiotherapy after last SCT. Patients received 21.6 Gy to the preoperative primary tumor volume. For patients with incomplete surgical resection, an additional boost of 14.4 Gy was delivered to the gross residual tumor, for a total dose of 36 Gy. CILP (primary end point) and event-free (EFS) and overall survival (OS; secondary end points) were compared with the COG A3973 historical cohort, in which all patients received single SCT and 21.6 Gy without a boost. RESULTS: For all patients in ANBL0532 receiving radiotherapy (n = 323), 5-year CILP, EFS, and OS rates were 11.2% ± 1.8%, 56.2% ± 3.4%, and 68.4% ± 3.2% compared with 7.1% ± 1.4% (P = .0590), 47.0% ± 3.5% (P = .0090), and 57.4% ± 3.5% (P = .0088) for all patients in A3973 receiving radiotherapy (n = 328), respectively. Five-year CILP, EFS, and OS rates for patients in A3973 with incomplete resection and radiotherapy (n = 47) were 10.6% ± 4.6%, 48.9% ± 10.1%, and 56.9% ± 10.0%, respectively. In comparison, 5-year CILP, EFS, and OS rates for patients in ANBL0532 who were randomly assigned or assigned to single SCT and received boost radiotherapy (n = 74) were 16.3% ± 4.3% (P = .4126), 50.9% ± 7.0% (P = .5084), and 68.1% ± 6.7% (P = .2835), respectively. CONCLUSION: Boost radiotherapy to gross residual tumor present at the end of induction did not significantly improve 5-year CILP. These results highlight the need for new strategies to decrease the risk of locoregional failure.

Effect of Tandem Autologous Stem Cell Transplant vs Single Transplant on Event-Free Survival in Patients With High-Risk Neuroblastoma: A Randomized Clinical Trial.

Importance: Induction chemotherapy followed by high-dose therapy with autologous stem cell transplant and subsequent antidisialoganglioside antibody immunotherapy is standard of care for patients with high-risk neuroblastoma, but survival rate among these patients remains low. Objective: To determine if tandem autologous transplant improves event-free survival (EFS) compared with single transplant. Design, Setting, and Participants: Patients were enrolled in this randomized clinical trial from November 2007 to February 2012 at 142 Children's Oncology Group centers in the United States, Canada, Switzerland, Australia, and New Zealand. A total of 652 eligible patients aged 30 years or younger with protocol-defined high-risk neuroblastoma were enrolled and 355 were randomized. The final date of follow-up was June 29, 2017, and the data analyses cut-off date was June 30, 2017. Interventions: Patients were randomized to receive tandem transplant with thiotepa/cyclophosphamide followed by dose-reduced carboplatin/etoposide/melphalan (n = 176) or single transplant with carboplatin/etoposide/melphalan (n = 179). Main Outcomes and Measures: The primary outcome was EFS from randomization to the occurrence of the first event (relapse, progression, secondary malignancy, or death from any cause). The study was designed to test the 1-sided hypothesis of superiority of tandem transplant compared with single transplant. Results: Among the 652 eligible patients enrolled, 297 did not undergo randomization because they were nonrandomly assigned (n = 27), ineligible for randomization (n = 62), had no therapy (n = 1), or because of physician/parent preference (n = 207). Among 355 patients randomized (median diagnosis age, 36.1 months; 152 [42.8%] female), 297 patients (83.7%) completed the study and 21 (5.9%) were lost to follow-up after completing protocol therapy. Three-year EFS from the time of randomization was 61.6% (95% CI, 54.3%-68.9%) in the tandem transplant group and 48.4% (95% CI, 41.0%-55.7%) in the single transplant group (1-sided log-rank P=.006). The median (range) duration of follow-up after randomization for 181 patients without an event was 5.6 (0.6-8.9) years. The most common significant toxicities following tandem vs single transplant were mucosal (11.7% vs 15.4%) and infectious (17.9% vs 18.3%). Conclusions and Relevance: Among patients aged 30 years or younger with high-risk neuroblastoma, tandem transplant resulted in a significantly better EFS than single transplant. However, because of the low randomization rate, the findings may not be representative of all patients with high-risk neuroblastoma. Trial Registration: ClinicalTrials.gov Identifier: NCT00567567.

Pancreatic islet cell tumors in adolescents and young adults.

BACKGROUND: Pancreatic islet cell tumors are rare in adolescents, and most studies published to date focus on older patients. We utilized a national database to describe the histology and clinical pattern of pancreatic islet cell tumors in adolescent and young adult (AYA) patients, and to compare AYAs to older adults. We hypothesized that AYAs with pancreatic islet cell tumors would have better overall survival. METHODS: The National Cancer Data Base (NCDB, 1998-2012) was queried for AYA patients (15-39 years) with a pancreatic islet cell tumor diagnosis. Demographics, tumor characteristics, treatment modalities, and outcomes were abstracted and compared to adults (≥40 years). RESULTS: 383 patients (56.4% female, 65% non-Hispanic Whites) were identified, with a median age of 27 (IQR 16-34) years. Islet cell carcinoma was the most common histology. Of patients with known stage of disease, 49% presented with early stage (I or II). Seventy percent of patients underwent surgical resection, including local excision 44%, Whipple procedure 37.5%, or total pancreatectomy 19%. Chemotherapy was utilized in 27% and radiotherapy in 7%. All-cause mortality was 36%. AYA patients underwent more extensive resections (p = 0.001) and had lower mortality rates (p < 0.001), with no differences in tumor stage or use of adjuvant therapies, when compared to adults. CONCLUSIONS: AYA patients with pancreatic islet cell tumors had comparable utilization of adjuvant therapies but underwent more extensive resections and demonstrated a higher overall survival rate than adult counterparts. Further investigation into approaches to earlier diagnosis and tailoring of multimodality therapy of these neoplasms in the AYA population is needed. LEVELS OF EVIDENCE: Prognostic Study, Level II - retrospective study.

Pediatric gastric adenocarcinoma: A National Cancer Data Base review.

PURPOSE: We sought to compare the presentation, management, and outcomes in gastric adenocarcinoma cancer for pediatric and adult patients. METHODS: Using the 2004 to 2014 National Cancer Database (NCDB), patients ≤21 years (pediatric) were retrospectively compared to >21 years (adult). Chi-squared tests were used to compare categorical variables, and Cox regression was used to estimate hazard ratios (HR) for survival differences. RESULTS: Of the 129,024 gastric adenocarcinoma cases identified, 129 (0.10%) occurred in pediatric patients. Pediatric cases presented with more advanced disease, including poorly differentiated tumors (81% vs 65%, p = 0.006) and stage 4 disease (56% vs 41%, p = 0.002). Signet ring adenocarcinoma comprised 45% of cases in the pediatric group as compared to 20% of cases in the adults (P < 0.001). Similar proportions in both groups underwent surgery. However, near-total gastrectomy was more common in the pediatric group (16% vs 6%, p < 0.001). The proportions of patients with negative margins, nodal examination, and presence of positive nodes were similar. There was no overall survival difference between the two age groups (HR 0.92, 95% Confidence interval 0.73-1.15). CONCLUSION: While gastric adenocarcinoma in pediatric patients present with a more advanced stage and poorly differentiated tumors compared to adults, survival appears to be comparable. TYPE OF STUDY: Retrospective cohort study. LEVEL OF EVIDENCE: III.

Factors influencing survival after recurrence in osteosarcoma: A report from the Children's Oncology Group.

BACKGROUND: Despite drastic improvement in overall survival for pediatric patients with cancer, those with osteosarcoma have stable rates of survival since the 1980s. This project evaluates the effect of several variables on survival after first recurrence in patients with osteosarcoma. METHODS: Data from three prospective North American cooperative group trials for newly diagnosed osteosarcoma are included: INT-0133, POG-9754, and AOST0121. The analytic population for this study is all enrolled patients with first event-free survival (EFS) event of relapse. The primary outcome measure for this retrospective analysis was survival after recurrence (SAR). RESULTS: The analytic population consisted of N = 431 patients. SAR was statistically significantly associated with age at enrollment (<10 years, P = 0.027), presence of metastatic disease at diagnosis (localized, P < 0.0001), site of relapse (combination lung + bone, unfavorable, P = 0.005), and time to first relapse (2+ years, favorable, P < 0.0001) in multivariate analysis. Ethnicity, primary site of tumor, race, and sex were not significantly related to SAR. CONCLUSIONS: Prolonged SAR in patients with relapsed osteosarcoma is associated with age, extent of disease at diagnosis, site of and time to relapse. Adolescent and young adult patients with osteosarcoma have shorter SAR than younger patients, consistent with studies showing decreased overall survival in this group. Although patients with primary metastatic disease have shorter SAR, there is a subset of patients who relapse greater than 2 years from initial diagnosis that will become survivors. Histological response was significantly associated with time to relapse, but was not predictive of SAR.

A phase II study of eribulin in recurrent or refractory osteosarcoma: A report from the Children's Oncology Group.

BACKGROUND: Patients with recurrent or refractory osteosarcoma have a poor prognosis with less than 30% surviving two years. Eribulin is a synthetic analog of halichondrin B, has a novel mechanism of action when compared with other microtubule inhibitors, and may have antitumor activity in osteosarcoma. METHODS: A prospective study was designed to assess the disease control success at four months and objective response rates in patients with recurrent or refractory osteosarcoma treated with eribulin. Eligible patients were between 12 and 50 years of age, had measurable tumor, and met standard organ function requirements. Patients were given eribulin 1.4 mg/m2 /dose on days 1 and 8 of each 3-week cycle for up to 24 months if there was no progressive disease. Response to therapy was assessed using RECIST 1.1 criteria after cycles 2 and 5 and every fourth cycle thereafter. RESULTS: Nineteen patients enrolled on the AOST1322 study. The median age of enrollment was 16 years (range, 12-25 years). Twelve patients were male and seven female. Eribulin was well tolerated, with neutropenia identified as the most common toxicity. The median progression-free survival was 38 days and no patients reached the four-month time point without progression. No objective responses were seen in any patient. CONCLUSION: This study rapidly assessed the clinical activity of a novel agent in this patient population. Eribulin was well tolerated, but there were no patients who demonstrated objective response, and all patients had progression prior to four months.

Partial Nephrectomy for Nephroblastoma: A National Cancer Data Base Review.

Standard of care for unilateral nephroblastoma includes total nephrectomy (TN) with nodal sampling. We sought to compare the outcomes of TN and partial nephrectomy (PN). We performed a retrospective cohort study of TN and PN for nephroblastoma using the National Cancer Data Base. The outcomes included nodal sampling frequency, margin status, and survival. Categorical and continuous data were evaluated with χ2 and t tests, respectively (P < 0.05). Generalized linear models evaluated nodal sampling and margin status. Cox regression compared survival. In total, 235 patients underwent PN and 3572 had TN. TN patients were 50 per cent more likely to undergo nodal sampling (RR: 1.47, 95% CI 1.30-1.66). There was no difference in margin status (RR: 0.91, 95% CI 0.65-1.28) or overall survival (HR 1.57; 95% CI 0.78-3.19). This study reports the largest review of patients with PN for unilateral nephroblastoma. PN patients had less nodal sampling but similar margin involvement and overall survival.

Disparities in fertility-sparing surgery in adolescent and young women with stage I ovarian dysgerminoma.

BACKGROUND: In many cancers, racial and socioeconomic disparities exist regarding the extent of surgery. For ovarian dysgerminoma, fertility-sparing (FS) surgery is recommended whenever possible. The aim of this study was to investigate rates of FS versus non-fertility-sparing (NFS) procedures for stage I ovarian dysgerminoma in adolescents and young adults (AYAs) by ethnicity/race and socioeconomic status. MATERIALS AND METHODS: The National Cancer Data Base was queried for patients with ovarian dysgerminoma from 1998 to 2012. After selecting patients aged 15-39 y with stage I disease, a multivariate regression analysis was performed, and rates of FS and NFS procedures were compared, first according to ethnicity/race, and then by socioeconomic surrogate variables. RESULTS: Among the 687 AYAs with stage I ovarian dysgerminoma, there was no significant difference in rates of FS and NFS procedures based on ethnicity/race alone (P = 0.17), but there was a significant difference in procedure type for all three socioeconomic surrogates. The uninsured had higher NFS rates (30%) than those with government (21%) or private (19%) insurance (P = 0.036). Those in the poorest ZIP codes had almost twice the rate of NFS procedures (31%) compared with those in the most affluent ZIP codes (17%). For those in the least-educated regions, 24% underwent NFS procedures compared to 14% in the most-educated areas (P = 0.027). CONCLUSIONS: AYAs with stage I ovarian dysgerminoma in lower socioeconomic groups were more likely to undergo NFS procedures than those in higher socioeconomic groups, but there was no difference in rates of FS versus NFS procedures by ethnicity/race. Approaches aimed at reducing socioeconomic disparities require further examination.

Pediatric-protocol of multimodal therapy is associated with improved survival in AYAs and adults with rhabdomyosarcoma.

BACKGROUND: Multimodal therapy is the standard treatment for pediatric rhabdomyosarcoma, but for adolescents and young adults (AYAs: ages 15-39) and older adults with rhabdomyosarcoma, the use of adjuvant therapy is variable, and survival is greatly decreased compared with younger patients. METHODS: All patients with rhabdomyosarcoma who had a curative operative were identified from the 1998-2012 National Cancer Database. Regression analyses identified independent factors relating to receipt of multimodal therapy (resection + chemotherapy + radiation) and the influence of multimodal therapy on 5-year overall survival. RESULTS: Of 2,312 patients, 44% were pediatric (age < 15 years), 22% AYA (ages 15-39), and 34% adult (age ≥ 40 years). Adults received multimodal therapy least often (pediatric: 62%, AYA: 46%, adults: 24%; P < .001), even after controlling for demographic characteristics, tumor features, and stage. In the entire cohort, multimodal therapy was associated with a decreased risk of death within 5 years (hazard ratio [HR] 0.72, 95% confidence interval [CI] 0.62-0.84), with similar findings after stratification by age (pediatric: HR 0.64, 95% CI 0.48-0.85; AYA: HR 0.72, 95% CI 0.55-0.95; adult: HR 0.74, 95% CI 0.58-0.93). In AYAs only, black and Hispanic patients had an increased risk of death within 5 years (black patients: HR 1.64, 95% CI 1.14-2.37; Hispanic patients: HR 1.62, 95% CI 1.11-2.36). CONCLUSION: This first large national study suggests that multimodal therapy is independently associated with improved survival for both AYAs and adults with rhabdomyosarcoma, similar to pediatric patients, but multimodal therapy is appreciably underused. Implementation of multimodal therapy for all patients could potentially improve overall outcomes of patients with rhabdomyosarcoma.

Testicular tumors in prepubescent patients.

BACKGROUND/PURPOSE: Pediatric testicular tumors are rare, constituting only 1% of all pediatric solid tumors. Single-institution studies addressing pediatric testicular tumors published to date have been limited in the number of patients. METHODS: We utilized the National Cancer Data Base (1998-2012) to review all prepubescent patients (≤12 years old) with testicular neoplasms. Demographics, tumor characteristics, treatment modalities, and outcomes were abstracted. RESULTS: A total of 479 patients were identified, with a median age of 3 years (IQR 0-4) at diagnosis. 67% of cases were diagnosed by 3 years of age. Yolk sac tumors were the most common histology (202 patients, 42.2%). Most tumors were diagnosed at a low stage. Resection was performed in 465 boys, with 75% having undergone radical orchiectomies. Chemotherapy was utilized in 28% of cases and radiotherapy in 7%. With mean follow-up of 5.6 years, mortality rate was 3%. No difference in mortality was noted based on histology or extent of surgical resection. CONCLUSIONS: This series of prepubertal testicular tumors is the largest yet reported and highlights the patient demographics, tumor characteristics, treatment modalities and outcomes for these tumors. TYPE OF STUDY: Prognosis study LEVEL OF EVIDENCE: II.

Socioeconomic disparities affect survival in malignant ovarian germ cell tumors in AYA population.

BACKGROUND: Malignant ovarian germ cell tumors (MOGCTs) are a rare form of ovarian malignancy. Socioeconomic status (SES) has been shown to affect survival in several gynecologic cancers. We examined whether SES impacted survival in adolescent and young adults (AYAs) with MOGCT. MATERIALS AND METHODS: The National Cancer Data Base was used to identify AYAs (aged 15-39 years) with MOGCT from 1998 to 2012. Three SES surrogate variables identified were as follows: insurance type, income quartile, and education quartile. Pooled variance t-tests and chi-square tests were used to compare tumor characteristics, the time from diagnosis to staging/treatment, and clinical outcome variables for each SES surrogate variable, while controlling for age and race/ethnicity in a multivariate model. Kaplan-Meier survival estimates were calculated using the log-rank test. RESULTS: A total of 3125 AYAs with MOGCT were identified. Subjects with lower SES measures had higher overall stage and T-stage MOGCTs at presentation. There was no significant difference in the time to staging/treatment, extent of surgery, or use of chemotherapy by SES. Subjects from a lower education background, from a lower income quartile, and without insurance had decreased survival (P ≤ 0.02 for all). Controlling for overall stage and T-stage, the difference in survival was no longer significant. CONCLUSIONS: AYAs with MOGCT from lower SES backgrounds presented with more advanced stage disease. Further studies that focus on the underlying reasons for this difference are needed to address these disparities.

Effects of socioeconomic status on children with well-differentiated thyroid cancer.

BACKGROUND: Well-differentiated thyroid cancer is the most common endocrine malignancy in children. Adult literature has demonstrated socioeconomic disparities in patients undergoing thyroidectomy, but the effects of socioeconomic status on the management of pediatric well-differentiated thyroid cancer remains poorly understood. METHODS: Patients ≤21 years of age with well-differentiated thyroid cancer remains were reviewed from the National Cancer Data Base. Three socioeconomic surrogate variables were identified: insurance type, median income, and educational quartile. Tumor characteristics, diagnostic intervals, and clinical outcomes were compared within each socioeconomic surrogate variable. RESULTS: A total of 9,585 children with well-differentiated thyroid cancer remains were reviewed. In multivariate analysis, lower income, lower educational quartile, and insurance status were associated with higher stage at diagnosis. Furthermore, lower income quartile was associated with a longer time from diagnosis to treatment (P < .002). Similarly, uninsured children had a longer time from diagnosis to treatment (28 days) compared with those with government (19 days) or private (18 days) insurance (P < .001). Despite being diagnosed at a higher stage and having a longer time interval between diagnosis and treatment, there was no significant difference in either overall survival or rates of unplanned readmissions based on any of the socioeconomic surrogate variables. CONCLUSION: Children from lower income families and those lacking insurance experienced a longer period from diagnosis to treatment of their well-differentiated thyroid cancer remains. These patients also presented with higher stage disease. These data suggest a delay in care for children from low-income families. Although these findings did not translate into worse outcomes for well-differentiated thyroid cancer remains, future efforts should focus on reducing these differences.

Breast Malignancies in Children: Presentation, Management, and Survival.

PURPOSE: Pediatric breast malignancies are rare, and descriptions in the literature are limited. The purpose of our study was to compare pediatric and adult breast malignancy. METHODS: We performed a retrospective cohort study using the National Cancer Data Base comparing patients ≤21 years to those >21 years at diagnosis (1998-2012). Generalized linear models estimated differences in demographic, tumor, and treatment characteristics. Cox regression was used to compare overall survival. RESULTS: Of 1,999,181 cases of invasive breast malignancies, 477 (0.02%) occurred in patients ≤21 years. Ninety-nine percent of adult patients had invasive carcinoma compared with 64.8% of pediatric patients with the remaining patients having sarcoma, malignant phyllodes, or malignancy not otherwise specified (p < 0.001). Pediatric patients were twice as likely to have an undifferentiated malignancy [relative risk (RR) 2.19; 95% confidence interval (CI) 1.72-3.79]. Half of adults presented with Stage I disease compared with only 22.7% of pediatric patients (p < 0.001). Pediatric patients were 40% more likely to have positive axillary nodes (RR 1.42; 95% CI 1.10-1.84). Among patients with invasive carcinoma, pediatric patients were more than four times as likely to receive a bilateral than a unilateral mastectomy compared with adults (RR 4.56; 95% CI 3.19-6.53). There was no difference in overall survival between children and adults. CONCLUSIONS: Pediatric breast malignancies are more advanced at presentation, and there is variability in treatment practices. Adult and pediatric patients with invasive carcinoma have similar overall survival.

Characteristics and outcomes in children with undifferentiated embryonal sarcoma of the liver: A report from the National Cancer Database.

OBJECTIVE: To examine patient characteristics and outcomes in children with undifferentiated embryonal sarcoma of the liver (UESL) using a multi-institutional database. SUMMARY BACKGROUND DATA: UESL is a rare disease (incidence is one per million). Therefore, the current literature is mostly limited to small case series. METHODS: The National Cancer Database was queried for primary UESL diagnosed between 1998 and 2012. RESULTS: A total of 103 patients (<18 years) were identified. The 5-year overall survival of the entire group was 86%. The best outcomes were seen in children who had tumors smaller than 15 cm and were able to undergo surgical resection with or without chemotherapy. Margin status did not appear to significantly affect survival. The most common type of resection was hemihepatectomy (37%), followed by sectionectomy (10%) and trisectionectomy (10%). Orthotopic liver transplant was performed in 10 children, all of whom survived to 5 years. CONCLUSION: Surgical resection with or without chemotherapy should be the mainstay of treatment in children with UESL, and is associated with very favorable outcomes. Negative surgical margins were not associated with improved survival. Orthotopic liver transplantation may be a viable method of attaining local control in tumors, which would otherwise be unresectable.