Characteristics, treatments, and outcomes of adolescents and adults with neuroblastoma: a retrospective study in China.

Background: Neuroblastoma (NB) is rare in adolescents and adults, resulting in limited availability of data.

Objectives: We comprehensively investigated the characteristics, treatments, and outcomes of adolescent and adult patients with NB, aiming to provide a more in-depth insight into this disease.

Design: A retrospective, single-center study.

Methods: We retrieved and analyzed the medical data of patients with NB aged 10 years or older at diagnosis who were treated at Sun Yat-sen University Cancer Center between June 2005 and January 2024.

Results: Sixty-five patients (30 males and 35 females) were enrolled, with a median age of 20 years (interquartile range, 14-26 years), including 27 patients aged 10-18 years and 38 patients aged >18 years. Most patients were classified as M-stage disease (n = 40, 61.5%), high-risk (n = 42, 64.6%), and poorly differentiated NB (n = 27, 41.5%). Additionally, 3 (6.7%) patients had MYCN amplification, and 5 (25%) had ALK mutations. The genomic landscape revealed that mutations in the cell cycle and DNA repair pathways are related to chemotherapy sensitivity. After induction therapy, 34 (52.3%) patients achieved complete response (CR). The 5-year progression-free survival (PFS) and overall survival (OS) rates were 33.1% ± 6.9% and 55.1% ± 7.6%, respectively. Patients who achieved CR after induction therapy had superior PFS (p = 0.009), with 5-year PFS rates of 44.0% ± 10.6% compared to 18.5% ± 8.5% in non-CR patients.

Conclusion: Adolescent and adult patients with NB exhibit distinct characteristics, less chemotherapy sensitivity, and poorer outcomes compared to pediatric patients. Achieving CR after induction therapy is associated with better outcomes. Further investigation for new therapies is required.