PATTERNS OF CARE IN TREATING CHILDHOOD NEUROBLASTOMA: EVIDENCE FROM A POPULATION LEVEL STUDY IN THE UNITED STATES

Purpose: To describe the treatment of childhood neuroblastoma in real-world settings using population level data in the United States. Neuroblastoma describes a heterogeneous group of extracranial solid tumors with different outcomes associated with different risk factors. This heterogeneity is also reflected in the wide span of treatment modalities with higher risk group patients undergo more aggressive treatment. 

Method:  The National Cancer Institute (NCI)’s Patterns of Care initiative focused on the treatment of newly diagnosed neuroblastoma patients in 2010 and 2011. Patient sociodemographic and clinical characteristics along with provider characteristics were abstracted from case notes. Treatments were ascertained from a physician verification form. This data was analyzed, stratifying by the Children’s Oncology Group (COG)’s risk schema. Survival analysis based on up to 48 months of follow-up was also conducted.

Result: The treatment patterns of 252 patients from 14 cancer registries found that majority (75%) of patients were registered in a treatment protocol in larger, non-government, not-for-profit hospitals with residency training programs. Stratifying by risk showed that 100% of low-risk patients (N=36) received surgery. The combination of carboplatin and etoposide was the most practiced first-line regimen (35.7%) in patients with an intermediate-risk profile (N=57). High-risk neuroblastoma patients (N=100) received extensive, multimodal pattern of treatment with cyclophosphamide as the most prevalently used chemotherapeutic agent (90%). Many combinations of chemotherapy were used along with other systemic therapies such as immunotherapy and isotretinoin. Multivariate regression analysis showed that receipt of transplant was based on neeed only and not insurance or other socioeconomic factors. Kaplan-Meier survival model showed that high-risk patients had the highest mortality risk in the 48-months follow-up period. Only 60% of  high-risk patients were alive at the end of the follow-up period. Sensitivity analysis reconciling the "unknown risk" patients (N=59) did not provide any other trend information.

Conclusion: The treatment of childhood neuroblastoma is most often under protocol and is broadly in adherence to risk stratification. It is high-risk patients that receive most treatment modalities and regimens but are most likely to have the poorest survival rates in the United States.