Proposed Additions to the NCCN Guidelines for Adult Medulloblastoma

Abstract

Medulloblastoma is a rare brain tumor that occurs in both children and adults, with patients aged 15 to 39 years accounting for 30% of all cases. In adults, guidelines for diagnosis and treatment are often based on retrospective data and extrapolated from the pediatric experience due to limited availability of prospective trials or registries involving adults. Importantly, adult patients differ from pediatric patients in many aspects, including the molecular features of the tumor and tolerance to treatment. In 2017, the NCI was granted support from the Cancer Moonshot initiative to address the challenges and unmet needs of adults with rare central nervous system (CNS) tumors through the NCI Comprehensive Oncology Network for Evaluating Rare CNS Tumors (NCI-CONNECT). On November 25, 2019, NCI-CONNECT convened a multidisciplinary workshop on adult medulloblastoma. Working groups identified unmet needs in clinical care and research and developed specific action items, including a proposal for inclusion of new items in the NCCN Guidelines for Adult Medulloblastoma, delineated in this review along with the evidence supporting their incorporation. Recommendations included facilitating referral of patients to centers of excellence; promoting patient participation in clinical trials or registries; encouraging use of DNA methylation for confirmation of diagnosis and subgrouping; offering counseling on contraception and fertility preservation; evaluating patients for symptoms and medical management of endocrine, vision, hearing, and neurocognitive deficits; providing psychosocial support and referral to neurorehabilitation; minimizing delays in therapy; and incorporating imaging standards and criteria for progression.

Virtual multi-institutional tumor board: a strategy for personalized diagnoses and management of rare CNS tumors

“…Approximately half of the presented cases involved the diagnosis and management of an NCI-CONNECT rare CNS tumor and the median age was young. While many of the rare tumors presented at the MTB still lack well-defined standards of care [ 26 ], the NCI-CONNECT program is fostering progress by publishing guidelines [ 22 ] and hosting expert workshops [ 22 , 23 , 25 , 27 ]. Therefore, our virtual MTB offered providers guidance and possibly offered patients reassurance that their case was being reviewed by a diverse group of specialists with the knowledge and tools needed to provide informed treatment suggestions, a critical benefit worth exploring in future studies [ 42 ].…”

Prognostic factors related to overall survival in adolescent and young adults with medulloblastoma: A systematic review

“…There are already dedicated studies and platforms which aim to understand the unique molecular gap between pediatric and adult oncology; this will be crucial in order to improve the outcomes for AYA medulloblastoma research. 52 In recently published proposed additions to the NCCN adult medulloblastoma guidelines, 53 the promotion of participation in clinical trials and registries and referral to specialized centers was encouraged as a key step towards this goal.…”