Challenges and limitations of clinical trials in the adolescent and young adult CNS cancer population: A systematic review

Emma M Byrne; Maeve Pascoe; Diane Cooper; Terri S Armstrong; Mark R Gilbert

doi:10.1093/noajnl/vdad159

Challenges and limitations of clinical trials in the adolescent and young adult CNS cancer population: A systematic review

Neurooncol Adv. 2023 Dec 10;6(1):vdad159. doi: 10.1093/noajnl/vdad159. eCollection 2024 Jan-Dec.

Authors

Emma M Byrne¹, Maeve Pascoe¹, Diane Cooper², Terri S Armstrong¹, Mark R Gilbert¹

Affiliations

¹ Neuro-Oncology Branch, National Cancer Institute, National Institute of Health, Bethesda, Maryland, USA.
² National Institute of Health Library, National Institute of Health, Bethesda, Maryland, USA.

Abstract

Background: The adolescent and young adult (AYA) cancer population, aged 15-39, carries significant morbidity and mortality. Despite growing recognition of unique challenges with this age group, there has been little documentation of unmet needs in their care, trial participation, and quality of life, particularly in those with primary brain tumors.

Methods: A systematic literature review of 4 databases was conducted following Preferred Reporting Items for Systematic Reviews and Meta-Analyses (PRISMA) standards. Studies included editorials, reviews, and practice guidelines on the challenges and limitations faced by the AYA population. Papers had to address CNS tumors.

Results: Sixty-eight studies met the inclusion criteria. The challenges and limitations in clinical trials in the AYA population were synthesized into 11 categories: molecular heterogeneity, tumor biology, diagnostic delay, access to care, physician factors, patient factors, primary brain tumor (PBT) factors, accrual, limited trials, long term follow up, and trial design. The published papers' recommendations were categorized based on the target of the recommendation: providers, coordination of care, organizations, accrual, and trial design. The AYA cancer population was found to suffer from unique challenges and barriers to care and the construction of trials.

Conclusions: The AYA CNS cancer population suffers from unique challenges and barriers to care and construction of trials that make it critical to acknowledge AYAs as a distinct patient population. In addition, AYAs with primary brain tumors are underrecognized and underreported in current literature. More studies in the AYA primary brain tumor patient population are needed to improve their care and participation in trials.

Keywords: CNS neoplasms; access to care; adolescents and young adults; barriers to care; oncology.

Published by Oxford University Press on behalf of the Society for Neuro-Oncology and the European Association of Neuro-Oncology 2023. This work is written by (a) US Government employee(s) and is in the public domain in the US.