115P - BrainStorm-NSE: Serum neuron-specific enolase as a biomarker for central nervous system metastases: A prospective cohort study

Background

Improving clinical outcomes of patients with central nervous system (CNS) metastases remains an unmet medical need. Serum neuron-specific enolase (NSE) has been investigated as a non-invasive biomarker of brain damage, and we believe it may contribute to the early detection of CNS metastases.

Methods

We aim to assess the predictive value of NSE for the development of CNS metastases in patients recruited for BrainStorm study, an international, multicentre prospective cohort study. BrainStorm will recruit around 600 patients with newly diagnosed non-CNS metastatic solid tumors with high risk of developing CNS metastases (Part A) to obtain 280 patients at diagnosis of CNS metastases (Part B). NSE assessment was added in a protocol amendment approved on 28 Oct 2021. All patients underwent blood collection for NSE assessment and magnetic resonance imaging for screening (Part A) or confirming (Part B) CNS metastases, at baseline and at regular intervals. Here we report an exploratory comparison of the baseline NSE from the first patients tested in Part A vs B. We applied Mann-Whitney test for comparing NSE levels (ng/ml) and Fisher’s exact test for proportion of patients with positive NSE (above upper limit of normal), using a two-sided statistical significance of 5%.

Results

We included 34 patients from Dec21 to Jun23, 26 in part A and 8 in part B - Table 115P. Median NSE was numerically higher in Part B (16.6, IQR 13.1-27.7 vs 12.4, 11.0-16.0) (p=0.26). NSE was positive for 62.5% of patients in Part B vs 34.6% in Part A (p=0.23). Median NSE levels were numerically lower in patients with breast cancer (Part B: 14.2 vs Part A: 12.7) and larger in patients with lung cancer (Part B: 19.0 vs Part A: 7.6). Table: 115P

Baseline characteristics at inclusion

Conclusions

Our preliminary results suggest a numerical trend for higher serum NSE levels in patients with established brain metastases supporting the need to formally investigate serum NSE as a circulating biomarker of CNS metastases once study accrual is completed and follow-up mature.

Editorial acknowledgement

Clinical trial identification

NCT04109131.

Legal entity responsible for the study

Clinical Trials Support Unit, Institut Jules Bordet.

Funding

NSE analyses: Fund iris-Research grant from King Baudouin Foundation. BrainStorm platform: La Fondation contre le cancer, Belgique, Les Amis de l'Institut Bordet, Bristol Myers Squibb, Fondation Cancer, Luxembourg.

Disclosure

D. Martins-Branco: Financial Interests, Personal, Advisory Board, (14/01/2021): Janssen; Financial Interests, Personal, Advisory Board, (22/03/2021): Merck Sharp & Dohme; Financial Interests, Personal, Invited Speaker, (03/02/2022 and 23/09/2022): AstraZeneca, Daiichi Sankyo; Financial Interests, Personal, Invited Speaker, (18/06/2021): Novartis; Financial Interests, Personal, Other, Meeting/travel grant (11/02/2021): Merck Sharp & Dohme; Financial Interests, Personal, Other, Meeting/travel grant (07/12/2021): Novartis; Financial Interests, Institutional, Research Grant, Institutional funding for an observational research project: Novartis; Financial Interests, Institutional, Research Grant, Institutional funding for an investigator-initiated clinical trial (NCT05075538): F. Hoffmann-La Roche Ltd.; Financial Interests, Institutional, Other, Work for Institut Jules Bordet as academic partner medical advisor in two industry-sponsored clinical trials (NCT01358877 and NCT03498716): F. Hoffmann-La Roche Ltd.; Financial Interests, Institutional, Other, Institutional funding for an investigator-initiated clinical trial (NCT03339843): Eli Lilly; Non-Financial Interests, Personal, Member of Board of Directors: Associação de Investigação e Cuidados de Suporte em Oncologia - Portuguese MASCC affiliate; Non-Financial Interests, Personal, Leadership Role, Portuguese Young Oncologists Committee Chair: November 2020 - May 2022: Sociedade Portuguesa de Oncologia; Non-Financial Interests, Personal, Leadership Role, Oncology Committee Chair: January 2020 - January 2021: Health Parliament Portugal; Other, Personal, Other, Employment of non-household immediate family member: F. Hoffmann-La Roche Ltd.M.A. Franzoi: Financial Interests, Institutional, Funding, Research Funding: Resilience Care; Financial Interests, Institutional, Invited Speaker: Novartis. G. Nader-Marta: Financial Interests, Personal, Other, meeting/travel grant: AstraZeneca, Roche, Bayer. A. Gombos: Financial Interests, Personal, Advisory Board: AstraZeneca, Daiichi Sankyo, Seattle Genetics; Financial Interests, Personal, Other, travel grant: Pfizer, AstraZeneca; Financial Interests, Personal, Other, educational fees: Eli Lilly. A. Goncalves: Financial Interests, Institutional, Advisory Board: AstraZeneca, Novartis, MSD, Innate Pharma, Parexel; Financial Interests, Personal, Other, meeting/travel grant: Roche, AstraZeneca, Mylan. F. Clatot: Financial Interests, Personal, Other, honoraria or travel grant: AstraZeneca, BMS, Merck, MSD, Lilly, Roche, Novartis, Pfizer, Gilead. S. Holbrechts: Financial Interests, Personal, Other, honoraria: Bristol Myers Squibb; Financial Interests, Personal, Other, travel grant: Roche. C. Cheymol: Financial Interests, Personal, Other, honoraria and travel grant: Lilly Oncology; Financial Interests, Personal, Other, travel grant: Pfizer. E. Borcoman: Financial Interests, Personal, Other, honoraria: Eisai, Merck Sharp & Dohme, Sandoz, Amgen; Financial Interests, Personal, Other, meeting/travel grant: Daiichi Sankyo, Eisai, Amgen, Sandoz, Merck Sharp & Dohme, Bristol Myers Squibb, Novartis, Pfizer, Roche; Financial Interests, Personal, Other, consulting: Egle Tx. H. Denys: Financial Interests, Institutional, Invited Speaker, consulting fees (advisory role): Pfizer, Roche, PharmaMar, AstraZeneca, Eli Lilly, Novartis, Amgen, GSK, MSD, Seagen, Gilead; Financial Interests, Institutional, Invited Speaker, travel, accommodations, expenses: Pfizer, Roche, PharmaMar, Teva, AstraZeneca, MSD, GSK, Gilead. J. Gligorov: Financial Interests, Personal, Other, honoraria and travel grant: AstraZeneca, Daiichi Sankyo, Eisai, Eva Pharm, Lilly, Merck Sharp & Dohme, Novartis, Pfizer, Roche Genentech, Seattle Genetics, Sothema; Financial Interests, Personal, Other, honoraria: Exact Sciences, Menarini, Onxeo; Financial Interests, Personal, Other, research support: Eisai, Exact Sciences, Roche Genentech. A.H. Awada: Financial Interests, Personal, Advisory Board: Amgen, AstraZeneca, Bayer, Daiichi Sankyo, Eisai, Genomic Health, Hengrui, Innate, Ipsen, Leo Pharma, Lilly, MSD, Menarini, Merck, Novartis, Pfizer, Seattle Genetics; Financial Interests, Personal, Invited Speaker: Amgen, AstraZeneca, Bayer, Daiichi Sankyo, Eisai, Genomic Health, Ipsen, Leo Pharma, Lilly, MSD, Merck, Novartis, Pfizer, Seattle Genetics; Financial Interests, Institutional, Research Grant: BMS, Roche; Non-Financial Interests, Personal, Other, Co-Chair: Oncodistinct network for clinical research; Non-Financial Interests, Personal, Other, Scientific Committee member: Fondation Contre le Cancer (Belgium; Non-Financial Interests, Personal, Other, Member: BSMO; Non-Financial Interests, Personal, Other, Chair: AllCan Belgium. N. Kotecki: Financial Interests, Personal, Other, meeting/travel grant: OSE Immunotherapeutics. All other authors have declared no conflicts of interest.