Children's Oncology Group's 2023 blueprint for research: Neuroblastoma

Abstract

Neuroblastoma is the most common extra‐cranial solid tumor in children and is known for its clinical heterogeneity. A greater understanding of the biology of this disease has led to both improved risk stratification and new approaches to therapy. Outcomes for children with low and intermediate risk disease are excellent overall, and efforts to decrease therapy for such patients have been largely successful. Although survival has improved over time for patients with high‐risk disease and treatments evaluated in the relapse setting are now being moved into earlier phases of treatment, much work remains to improve survival and decrease therapy‐related toxicities. Studies of highly annotated biobanked samples continue to lead to important insights regarding neuroblastoma biology. Such studies, along with correlative biology studies incorporated into therapeutic trials, are expected to continue to provide insights that lead to new and more effective therapies. A focus on translational science is accompanied by an emphasis on new agent development, optimized risk stratification, and international collaboration to address questions relevant to molecularly defined subsets of patients. In addition, the COG Neuroblastoma Committee is committed to addressing the patient/family experience, mitigating late effects of therapy, and studying social determinants of health in patients with neuroblastoma.

Percutaneous biopsy for the diagnosis, risk stratification, and molecular profiling of neuroblastoma: A single‐center retrospective study

“…In the era of precision medicine, evolving treatment guidelines for neuroblastoma may lead to new prognostic and therapeutic biologic markers. 24 This highlights the need to acquire sufficient tissue to evaluate current biomarkers through current techniques, but also to have further tissue to develop and validate new techniques. The high proportion of biopsies that resulted in specimens for biobanking (94%) is notable.…”

“…Standardizing biopsy techniques, as previously recommended by investigators, may enhance molecular testing success rates and facilitate integration into future multi-institutional COG protocols. 3,24 A prospective, multicenter study employing a 20-core approach with an 18-or 16-gauge needle, coupled with CEUS and real-time pathologic assessment may allow for robust evaluation of predictors for successful PCNB for initial biopsy in patients with suspected neuroblastoma. Finally, we did not compare features of those who underwent PCNB to surgical biopsy in this single-center study.…”

BPTF cooperates with MYCN and MYC to link neuroblastoma cell cycle control to epigenetic cellular states

“…Neuroblastoma is an extra-cranial solid tumor originating from undifferentiated neural crest-derived cells that is widely metastatic in 50% of patients at diagnosis and continues to show one of the least favorable outcomes among pediatric cancers 15, 16, 17 . MYCN amplification is strongly associated with poor outcome and is integrated into risk stratification and treatment planning 18 .…”

Integrated machine learning-driven disulfidptosis profiling: CYFIP1 and EMILIN1 as therapeutic nodes in neuroblastoma

“…NB is a malignant tumor of the sympathetic nervous system, originating in the developing sympathetic nervous system and commonly found in bilateral adrenal and sympathetic ganglia (Marshall et al 2014 ). There are various staging systems for NB tumors based on size and metastasis, such as The International Neuroblastoma Staging System (INSS) (Brodeur et al 1988 ; Castel et al 1999 ) and the Children’s Oncology Group (COG) staging system (Bagatell et al 2023 ; Pinto et al 2019 ). NB is categorized as low, medium, or high risk, with favorable outcomes for low and medium risk, often curable through surgery or experiencing spontaneous regression.…”