Results of treatment of patients with posterior mediastinal neuroblastoma in the observation group

Background. Neuroblastoma (NB) with primary tumor located in the posterior mediastinum is a unique subgroup characterized by a higher incidence of life-threatening complications, such as epidural compression (EC) and respiratory failure, yet it is associated with favorable long-term outcomes.

Materials and methods. The study included patients with low-risk posterior mediastinal NB treated at the Dmitry Rogachev National Medical Research Center of Pediatric Hematology, Oncology and Immunology between January 2012 and September 2024 (152 months). Patients were stratified into risk groups and managed according to the GPOH-NB2004 protocol. We evaluated relapse/progression detection methods, disease characteristics, treatment approaches, and long-term outcomes.

Results. The study included 146 patients with NB with primary tumor located in the posterior mediastinum, which is 21.6 % of all patients in the observation (low-risk) group registered in the research database of Dmitry Rogachev National Medical Research Center of Pediatric Hematology, Oncology and Immunology. The median age at diagnosis was 19.6 months (range 0.5–132.5 months) with a female predominance (male-to-female ratio 1:1.2). Most patients (85/146; 58.2 %) presented with stage 2 disease. 38/146 (26.0 %) patients received chemotherapy due to life threatening symptoms, primarily EC, while 87/146 (59.6 %) patients underwent best surgery in the volume of a macroscopic residual tumor. Relapse/progression occurred in 16.4 % (24/146), predominantly detected during routine follow-up imaging (18/24; 75,0 %), with a median time to relapse of 7.8 months (range 3.0–25.3 months). The majority of relapses/progressions were local (18/24; 75.0 %). In 93 % (14/15) of relapsed/progressed cases analyzed retrospectively, MLPA revealed segmental chromosomal aberrations (median 3 aberrations/ patient; range 0–6). The most frequent alterations included 17q gain (n = 11), 2p gain (n = 8), and deletions in 3p (n = 6), 11q (n = 5), and 4q (n = 4). The 5-year event-free survival in patients with mediastinal NB was 79,1 ± 4,0 %, patients with stage 1 showing the highest survival rates (90 ± 6 %) (p = 0.1932). The 5-year overall survival was 95,0 ± 2,1 %. The cumulative incidence of relapse/progression was 18.7 ± 3.5 %, while treatment-related mortality was 2.8 ± 1.4 % (one death from disease progression; five from therapy toxicity).

Conclusions. Mediastinal NB is a prognostically favorable group, with excellent survival rates independent of age or stage. Most patients in the low-risk group achieved favorable outcomes, even after relapse/progression. For the majority of patients in the low-risk group, surgery only is sufficient, and this strategy allows to reduce the risk of mortality from the toxicity of therapy without reducing overall survival.

However, chemotherapy remains critical for patients with life-threatening complications, such as EC and respiratory failure, which once again highlighting the challenges in managing this subgroup. Strict adherence to the entire follow-up plan during the period of catamnesis is essential for early relapse detection. The molecular profile of the tumor may predict relapses and treatment response, which underscores the need to integrate molecular diagnostic methods (MLPA) to all patients with NB in the low-risk group into routine diagnostics.

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