Natural history study of adults with primary central nervous system tumors: Initial results for first 1000 participants.

Abstract

Background: Primary central nervous system tumors (PCNST) are rare, understudied tumors for which knowledge of their clinical course, biology, physical, and psychological impact is limited. The National Cancer Institute's Neuro-Oncology Branch Natural History Study was developed to better understand the long-term disease course and outcomes experienced by these patients.

Methods: Past medical histories, tumor tissue (if available), and patient-reported outcomes assessing symptom burden, general health status, anxiety, depression, and perceived cognitive deficits were collected at study enrollment. Sociodemographic, clinical, molecular, and patient-reported outcome data were analyzed descriptively.

Results: Among the first 1000 participants, 796 had primary brain tumors (PBT) and 92 had primary spine tumors (PST); 88 were excluded. The cohort was predominantly male (56%), White (81%), and non-Hispanic (89%). Based on advanced molecular testing at enrollment, 190 (21%) patients had a change in diagnosis or subclassification since their initial diagnosis. Meningioma cases reported family brain tumor history more frequently than others. Most PBTs were diagnosed within 6 months of symptoms, while most PSTs were diagnosed ≥1 year after. Patients with PBT and PST reported an average of 10 symptoms, with 4 and 5 symptoms rated as moderate/severe, respectively. Nearly half of PBT participants reported anxiety or depression (46%) and difficulty with usual activities (48%). PST participants reported higher functional impairment and worse general health status.

Conclusion: These findings underscore the substantial burden of PCNSTs and highlight the value of longitudinal, tumor-agnostic data in generating real-world insights into the disease trajectories of these rare and heterogeneous tumors to inform clinical research.