Incidence and Outcomes of Non-High Risk Neuroblastoma Patients – With and Without Liver Disease at Initial Presentation
Presenter Status
Medical Student
Abstract Type
Clinical Research
Primary Mentor or Principal Investigator
Kevin Campbell, MD
Presentation Type
Poster
Start Date
21-5-2026 11:00 AM
End Date
21-5-2026 12:00 PM
Abstract Text
Background: Neuroblastoma is the most common solid tumor in childhood. Treatment is stratified based on patient age, stage of tumor, tumor biology, MYCN amplification, and tumor resectability. Patients with non-high-risk neuroblastoma generally have an excellent prognosis, with 5-year overall survival exceeding 90%. However, the presence of hepatic disease at diagnosis in non-high-risk cases can result in significant morbidity and may necessitate escalation of therapy from observation alone to urgent chemotherapy aimed at reducing tumor burden and alleviating organ compromise.
Objectives: We aimed to determine the proportion of patients with non-high-risk neuroblastoma with hepatic disease at initial presentation and compare their clinical course and outcomes with non-high-risk neuroblastoma patients without hepatic involvement at diagnosis.
Methods: A retrospective review was conducted of patients presenting to Children’s Mercy Hospital (CMH) with newly diagnosed non-high-risk neuroblastoma between 1999 and 2024. Patients were excluded if chart review identified them as high-risk patients or if they were treated on high-risk treatment protocols. For patients included, a comprehensive chart review was performed to assess hepatic disease at presentation and to further document treatment and outcomes for these patients.
Results: Among 63 eligible patients, 13 (21%) presented with hepatic involvement at diagnosis. Patients with hepatic disease at initial presentation were younger (median age 2 months) compared to those without hepatic disease (9 months). Adrenal gland was the most common primary tumor site among all the patients (44%), though much higher in the hepatic subgroup at 85% compared to those without hepatic disease (34%). Additionally, all hepatic involvement patients all had metastatic disease at presentation compared to non-hepatic involvement group (26%).
Patients with hepatic disease were more likely to require chemotherapy upfront with 54% treated with chemotherapy alone compared to 23% of chemotherapy alone in the patients without hepatic involvement. Patients with hepatic disease were less likely to undergo surgery alone for treatment (8%) compared to the non-hepatic group (28%). Observation-only management was slightly more common in hepatic disease patients (15%) compared to patients without hepatic disease (8%).
Five-year event-free survival (EFS) and overall survival (OFS) were 86% and 97% for all patients. Patients with hepatic disease at presentation had slightly higher EFS and OFS than patients without hepatic disease, with EFS of 92% vs 84% and OFS of 100% and 96%, respectively. Of the 13 patients who presented with hepatic disease, 15% (n=3) were observed, while 85% (n=10) received chemotherapy. Of 10 patients with response data post two cycles of therapy, 4 saw reduction between 0-25%, 3 had reduction between 25-50%, and 1 with >50% reduction.
Conclusion: Patients that presented to CMH with newly diagnosed non-high-risk neuroblastoma with hepatic involvement, contrary to national trends, did not have increased morbidity and mortality. Conclusions for this finding are likely multifactorial. Further, outcomes for patients without hepatic involvement were still excellent. Last, our cohort did reflect national trends in which these patients are younger and commonly have adrenal primary tumors.
Incidence and Outcomes of Non-High Risk Neuroblastoma Patients – With and Without Liver Disease at Initial Presentation
Background: Neuroblastoma is the most common solid tumor in childhood. Treatment is stratified based on patient age, stage of tumor, tumor biology, MYCN amplification, and tumor resectability. Patients with non-high-risk neuroblastoma generally have an excellent prognosis, with 5-year overall survival exceeding 90%. However, the presence of hepatic disease at diagnosis in non-high-risk cases can result in significant morbidity and may necessitate escalation of therapy from observation alone to urgent chemotherapy aimed at reducing tumor burden and alleviating organ compromise.
Objectives: We aimed to determine the proportion of patients with non-high-risk neuroblastoma with hepatic disease at initial presentation and compare their clinical course and outcomes with non-high-risk neuroblastoma patients without hepatic involvement at diagnosis.
Methods: A retrospective review was conducted of patients presenting to Children’s Mercy Hospital (CMH) with newly diagnosed non-high-risk neuroblastoma between 1999 and 2024. Patients were excluded if chart review identified them as high-risk patients or if they were treated on high-risk treatment protocols. For patients included, a comprehensive chart review was performed to assess hepatic disease at presentation and to further document treatment and outcomes for these patients.
Results: Among 63 eligible patients, 13 (21%) presented with hepatic involvement at diagnosis. Patients with hepatic disease at initial presentation were younger (median age 2 months) compared to those without hepatic disease (9 months). Adrenal gland was the most common primary tumor site among all the patients (44%), though much higher in the hepatic subgroup at 85% compared to those without hepatic disease (34%). Additionally, all hepatic involvement patients all had metastatic disease at presentation compared to non-hepatic involvement group (26%).
Patients with hepatic disease were more likely to require chemotherapy upfront with 54% treated with chemotherapy alone compared to 23% of chemotherapy alone in the patients without hepatic involvement. Patients with hepatic disease were less likely to undergo surgery alone for treatment (8%) compared to the non-hepatic group (28%). Observation-only management was slightly more common in hepatic disease patients (15%) compared to patients without hepatic disease (8%).
Five-year event-free survival (EFS) and overall survival (OFS) were 86% and 97% for all patients. Patients with hepatic disease at presentation had slightly higher EFS and OFS than patients without hepatic disease, with EFS of 92% vs 84% and OFS of 100% and 96%, respectively. Of the 13 patients who presented with hepatic disease, 15% (n=3) were observed, while 85% (n=10) received chemotherapy. Of 10 patients with response data post two cycles of therapy, 4 saw reduction between 0-25%, 3 had reduction between 25-50%, and 1 with >50% reduction.
Conclusion: Patients that presented to CMH with newly diagnosed non-high-risk neuroblastoma with hepatic involvement, contrary to national trends, did not have increased morbidity and mortality. Conclusions for this finding are likely multifactorial. Further, outcomes for patients without hepatic involvement were still excellent. Last, our cohort did reflect national trends in which these patients are younger and commonly have adrenal primary tumors.
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Poster Board Number: 17