Incidence of Adrenal Insufficiency in Patients with High-Risk
Neuroblastoma: A Single Institution Analysis
Abstract
Background: Neuroblastoma is the most common extracranial solid
tumor in children, with about half of cases classified as high risk.
Treatment varies by risk level, with high-risk patients undergoing
aggressive multimodal therapy. Long-term survival has improved, but
survivors face significant risks of late treatment effects, including
adrenal insufficiency. This study investigates the incidence of adrenal
insufficiency among neuroblastoma patients, focusing on high-risk versus
non-high-risk cases. Procedure: This retrospective cohort study
at a single tertiary children’s hospital reviewed records from 1998 to
2021, identifying 370 neuroblastoma patients, of which 137 had complete
risk stratification. The primary outcome was the incidence of adrenal
insufficiency, diagnosed based on clinical evaluation and response to
hydrocortisone therapy. Demographic and clinical data were collected,
and statistical analyses were performed to compare high-risk and
non-high-risk groups. Results: Among 137 neuroblastoma
patients, 9 (12.0%) high-risk and 3 (4.9%) non-high-risk patients were
diagnosed with adrenal insufficiency. The cumulative incidence of
adrenal insufficiency was 16.6% in high-risk and 3.5% in non-high-risk
patients. High-risk patients with adrenal insufficiency had a median
time of 10.2 months from neuroblastoma diagnosis to adrenal
insufficiency diagnosis, with all cases occurring in patients with
adrenal primary tumors. There were no significant differences in
demographic or clinical characteristics between high-risk patients with
and without adrenal insufficiency. Conclusions: The study found
a higher cumulative incidence of adrenal insufficiency in high-risk
neuroblastoma patients, particularly those with adrenal primary tumors.
Despite the lack of significant prevalence difference between risk
groups, the findings underscore the need for vigilant monitoring and
screening for adrenal insufficiency in neuroblastoma patients during and
after treatment. Future research should include larger,
multi-institutional cohorts to better understand risk factors and
optimize screening protocols.