Keri Streby

and 13 more

BACKGROUND: Diagnostic mIBG (meta-iodobenzylguanidine) scans are an integral component of response assessment in children with high-risk neuroblastoma. The role of end of induction (EOI) Curie Scores (CS) was previously described in patients undergoing a single autologous hematopoietic cell transplant (AHCT) as consolidation therapy. OBJECTIVE: We now examine the prognostic significance of CS in patients randomized to tandem AHCT on the Children’s Oncology Group (COG) trial ANBL0532. STUDY DESIGN: A retrospective analysis of mIBG scans obtained from patients enrolled in COG ANBL0532 was performed. Evaluable patients had mIBG-avid, International Neuroblastoma Staging System (INSS) stage 4 disease, did not progress during induction therapy, consented to consolidation randomization, and received a tandem AHCT (n=80). Optimal CS cut points maximized the outcome difference (≤ vs >CS cut-off) according to the Youden index. RESULTS: For recipients of tandem AHCT, the optimal cut point at diagnosis was CS=12, with superior EFS from study enrollment for patients with CS<12 (3-year EFS 74.2±7.9%) vs CS>12 (59.2±7.1%) (p=0.002). At EOI, the optimal cut point was CS=0, with superior end-induction EFS for patients with CS=0 (72.9±6.4%) vs CS>0 (46.5±9.1%) (p=0.002). CONCLUSION: In the setting of tandem transplantation for children with high-risk neuroblastoma, Curie scores at diagnosis and end-induction may identify a more favorable patient group. Patients treated with tandem AHCT who exhibited a CS<12 at diagnosis or CS=0 at EOI had superior EFS compared to those with CS above these cut points.

Daniel Zheng

and 6 more

Background: Modern therapeutic advances in high-risk neuroblastoma have improved overall survival (OS), but it is unclear whether these survival gains have been equitable. This study sought to examine the relationship between socioeconomic status (SES) and OS in children with high-risk neuroblastoma, and to investigate whether SES-associated disparities have changed over time. Procedure: In this population-based cohort study, children <18 years diagnosed with high-risk neuroblastoma (diagnosis at age ≥12 months with metastatic disease) from 1991-2015 were identified through the National Cancer Institute’s Surveillance, Epidemiology, and End Results database. Associations of county-level SES variables and OS were tested with univariate Cox proportional hazards regression. For a sub-cohort diagnosed after 2007, insurance status was examined as an individual-level SES variable. Multivariable regression analyses with treatment era and interaction terms were performed when SES variables reached near-significance (p≤0.1) in univariate and bivariate modeling with treatment era. Results: Among 1,217 children, 2-year OS improved from 53.0±3.4% in 1991-1998 to 76.9±2.9% in 2011-2015 (p<0.001). In univariate analyses, children with Medicaid (hazard ratio [HR]=1.40, 95% confidence interval [CI]=1.05-1.86, p=0.02) and those in high-poverty counties (HR=1.74, CI=1.17-2.60, p=0.007) experienced an increased hazard of death. No interactions between treatment era and SES variables were statistically significant in multivariable analyses, indicating that changes in OS over time did not differ between groups. Conclusions: Low SES is associated with inferior survival in children with high-risk neuroblastoma. Survival disparities have not widened over time, suggesting equitable access to and benefit from therapeutic advances. Interventions to narrow existing disparities are paramount.