Email this article The results of therapy in patients with high-risk neuroblastoma: the experience of the Dmitry Rogachev National Medical Research Center of Pediatric Hematology, Oncology and Immunology
- Authors: Shamanskaya T.V.1, Varfolomeeva S.R.2, Kachanov D.Y.1, Moiseenko R.A.1, Teleshova M.V.1, Konovalov D.M.1, Roshchin V.Y.1, Kazakova A.N.1, Zemtsova L.V.1, Drui A.E.1, Yadgarov M.Y.1, Tereshchenko G.V.1, Shcherbakov A.P.1, Likar Y.N.1, Sukhov M.N.3, Grachev N.S.1, Akhaladze D.G.1, Maschan M.A.1, Nechesnyuk A.V.1, Pshonkin A.V.1, Kurnikova E.E.1, Skorobogatova E.V.3, Khachatryan L.A.1, Fomin D.K.4, Maschan A.A.1, Rumyantsev A.G.1, Novichkova G.A.1
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Affiliations:
- The Dmitry Rogachev National Medical Research Center of Pediatric Hematology, Oncology and Immunology of Ministry of Healthcare of the Russian Federation
- Research Institute of Pediatric Oncology and Hematology of the N. N. Blokhin National Medical Research Center of Oncology of Ministry of Healthcare of the Russian Federation
- Russian Children's Clinical Hospital of the N. I. Pirogov Russian National Research Medical University of Ministry of Healthcare of the Russian Federation
- Russian Scientific Center of Roentgenology and Radiology of Ministry of Healthcare of the Russian Federation
- Issue: Vol 22, No 2 (2023)
- Pages: 65-91
- Section: ORIGINAL ARTICLES
- Submitted: 04.04.2023
- Published: 08.07.2025
- URL: https://hemoncim.com/jour/article/view/726
- DOI: https://doi.org/10.24287/1726-1708-2023-22-2-65-91
- ID: 726
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Abstract
Treatment of patients with high-risk neuroblastoma (NB) is a complex challenge, and it is based on response to certain elements of therapy. The development and introduction of new treatment approaches, such as GD2-targeted immunotherapy (IT), leads to improved survival in this cohort of patients.
The aim of the study was to retrospectively assess the effectiveness of therapy in patients with high-risk NB before the introduction of IT into clinical practice.
We retrospectively analyzed the data of 151 NB patients stratified into a high-risk group who had received treatment in accordance with the modified NB2004 protocol of the German Society for Pediatric Oncology and Hematology (GPOH) at the Dmitry Rogachev National Medical Research Center of Pediatric Hematology, Oncology and Immunology from 01.2012 to 12.2017. This study was approved by the Independent Ethics Committee and the Academic Council of the Dmitry Rogachev National Medical Research Center of Pediatric Hematology, Oncology and Immunology. All the study subjects (or their legal representatives) signed a voluntary informed consent form indicating their agreement to treatment and use of their data for research purposes. Overall survival (OS), event-free survival (EFS), and risk factors were analyzed in the patients with high-risk NB including those who had completed multimodal therapy with autologous hematopoietic stem cell transplantation and post-consolidation therapy with isotretinoin and had achieved a satisfactory response to induction therapy (complete response (CR), very good partial response (VGPR), partial response (PR)) (population of special interest). The main unfavorable prognostic clinical and molecular genetic factors affecting survival in the high-risk NB patients were older age, MYCN gene amplification, and stage 4 of the disease. The use of the modified GPOH NB2004 protocol resulted in a satisfactory response (CR/VGPR/PR) to the induction therapy in most patients: 124/151 (82.1 %). Surgery (other than primary tumor biopsy) led to improved survival, with no statistical difference between macroscopic radical surgery and macroscopic residual tumor. At the same time, radiation therapy (RT), as the second element of local control, had a significant impact on EFS in the group of the patients with stage 4 disease: the 3-year EFS was 39.4 % (95 % confidence interval (CI) 23.1–55.4) in the patients with RT versus 25.7 % (95 % CI 17.5–34.7) in the patients without RT (p = 0.0295). The introduction of a new high-dose TreoMel chemotherapy regimen did not result in worse survival rates but led to a decrease in transplant-related toxicity. The 5-year OS and 5-year EFS were 49.4 % (95 % CI 40.9–57.3 %) and 33.3 % (95 % CI 25.9–40.9) respectively for all the study subjects, and 81.6 % (95 % CI 70.3–88.9) and 55.1 % (95 % CI 43.1–65.5) respectively for the patients from the population of special interest. The analysis of the results of therapy in the high-risk NB patients who had received treatment at the Dmitry Rogachev National Medical Research Center of Pediatric Hematology, Oncology and Immunology, yielded results comparable to those of the original GPOH NB2004 protocol. The patients with CR/VGPR/PR to the induction therapy who had completed the protocol treatment with autologous hematopoietic stem cell transplantation and isotretinoin post-consolidation therapy demonstrated higher 5-year EFS rates. However, there remains a need to develop more effective treatment regimens for high-risk NB.
About the authors
T. V. Shamanskaya
The Dmitry Rogachev National Medical Research Center of Pediatric Hematology, Oncology and Immunology of Ministry of Healthcare of the Russian Federation
Author for correspondence.
Email: shamanskaya.tatyana@gmail.com
ORCID iD: 0000-0002-3767-4477
Tatyana V. Shamanskaya, Cand. Med. Sci., Head of the Department
Institute of Oncology, Radiology and Nuclear Medicine
Department for Embryonal Tumor Research
117997
1 Samory Mashela St.
Moscow
Russian FederationS. R. Varfolomeeva
Research Institute of Pediatric Oncology and Hematology of the N. N. Blokhin National Medical Research Center of Oncology of Ministry of Healthcare of the Russian Federation
ORCID iD: 0000-0001-6131-1783
Moscow
Russian FederationD. Yu. Kachanov
The Dmitry Rogachev National Medical Research Center of Pediatric Hematology, Oncology and Immunology of Ministry of Healthcare of the Russian Federation
ORCID iD: 0000-0002-3704-8783
Moscow
Russian FederationR. A. Moiseenko
The Dmitry Rogachev National Medical Research Center of Pediatric Hematology, Oncology and Immunology of Ministry of Healthcare of the Russian Federation
ORCID iD: 0000-0002-9654-084X
Moscow
Russian FederationM. V. Teleshova
The Dmitry Rogachev National Medical Research Center of Pediatric Hematology, Oncology and Immunology of Ministry of Healthcare of the Russian Federation
ORCID iD: 0000-0003-4042-0125
Moscow
Russian FederationD. M. Konovalov
The Dmitry Rogachev National Medical Research Center of Pediatric Hematology, Oncology and Immunology of Ministry of Healthcare of the Russian Federation
ORCID iD: 0000-0001-7732-8184
Moscow
Russian FederationV. Yu. Roshchin
The Dmitry Rogachev National Medical Research Center of Pediatric Hematology, Oncology and Immunology of Ministry of Healthcare of the Russian Federation
ORCID iD: 0000-0002-9375-7517
Moscow
Russian FederationA. N. Kazakova
The Dmitry Rogachev National Medical Research Center of Pediatric Hematology, Oncology and Immunology of Ministry of Healthcare of the Russian Federation
ORCID iD: 0000-0002-1085-4646
Moscow
Russian FederationL. V. Zemtsova
The Dmitry Rogachev National Medical Research Center of Pediatric Hematology, Oncology and Immunology of Ministry of Healthcare of the Russian Federation
ORCID iD: 0000-0002-0993-9183
Moscow
Russian FederationA. E. Drui
The Dmitry Rogachev National Medical Research Center of Pediatric Hematology, Oncology and Immunology of Ministry of Healthcare of the Russian Federation
ORCID iD: 0000-0003-1308-8622
Moscow
Russian FederationM. Ya. Yadgarov
The Dmitry Rogachev National Medical Research Center of Pediatric Hematology, Oncology and Immunology of Ministry of Healthcare of the Russian Federation
ORCID iD: 0000-0003-3792-1682
Moscow
Russian FederationG. V. Tereshchenko
The Dmitry Rogachev National Medical Research Center of Pediatric Hematology, Oncology and Immunology of Ministry of Healthcare of the Russian Federation
ORCID iD: 0000-0001-7317-7104
Moscow
Russian FederationA. P. Shcherbakov
The Dmitry Rogachev National Medical Research Center of Pediatric Hematology, Oncology and Immunology of Ministry of Healthcare of the Russian Federation
ORCID iD: 0000-0001-8129-0545
Moscow
Russian FederationYu. N. Likar
The Dmitry Rogachev National Medical Research Center of Pediatric Hematology, Oncology and Immunology of Ministry of Healthcare of the Russian Federation
ORCID iD: 0000-0002-6158-2222
Moscow
Russian FederationM. N. Sukhov
Russian Children's Clinical Hospital of the N. I. Pirogov Russian National Research Medical University of Ministryof Healthcare of the Russian Federation
ORCID iD: 0000-0003-4412-3056
Moscow
Russian FederationN. S. Grachev
The Dmitry Rogachev National Medical Research Center of Pediatric Hematology, Oncology and Immunology of Ministry of Healthcare of the Russian Federation
ORCID iD: 0000-0002-4451-3233
Moscow
Russian FederationD. G. Akhaladze
The Dmitry Rogachev National Medical Research Center of Pediatric Hematology, Oncology and Immunology of Ministry of Healthcare of the Russian Federation
ORCID iD: 0000-0002-1387-209X
Moscow
Russian FederationM. A. Maschan
The Dmitry Rogachev National Medical Research Center of Pediatric Hematology, Oncology and Immunology of Ministry of Healthcare of the Russian Federation
ORCID iD: 0000-0003-1735-0093
Moscow
Russian FederationA. V. Nechesnyuk
The Dmitry Rogachev National Medical Research Center of Pediatric Hematology, Oncology and Immunology of Ministry of Healthcare of the Russian Federation
ORCID iD: 0000-0002-2537-6157
Moscow
Russian FederationA. V. Pshonkin
The Dmitry Rogachev National Medical Research Center of Pediatric Hematology, Oncology and Immunology of Ministry of Healthcare of the Russian Federation
ORCID iD: 0000-0002-2057-2036
Moscow
Russian FederationE. E. Kurnikova
The Dmitry Rogachev National Medical Research Center of Pediatric Hematology, Oncology and Immunology of Ministry of Healthcare of the Russian Federation
ORCID iD: 0000-0003-4767-5382
Moscow
Russian FederationE. V. Skorobogatova
Russian Children's Clinical Hospital of the N. I. Pirogov Russian National Research Medical University of Ministryof Healthcare of the Russian Federation
ORCID iD: 0000-0003-4431-1444
Moscow
Russian FederationL. A. Khachatryan
The Dmitry Rogachev National Medical Research Center of Pediatric Hematology, Oncology and Immunology of Ministry of Healthcare of the Russian Federation
ORCID iD: 0000-0001-7265-0414
Moscow
Russian FederationD. K. Fomin
Russian Scientific Center of Roentgenology and Radiology of Ministry of Healthcare of the Russian Federation
ORCID iD: 0000-0002-7316-3519
Moscow
Russian FederationA. A. Maschan
The Dmitry Rogachev National Medical Research Center of Pediatric Hematology, Oncology and Immunology of Ministry of Healthcare of the Russian Federation
ORCID iD: 0000-0002-0016-6698
Moscow
Russian FederationA. G. Rumyantsev
The Dmitry Rogachev National Medical Research Center of Pediatric Hematology, Oncology and Immunology of Ministry of Healthcare of the Russian Federation
ORCID iD: 0000-0002-1632-4822
Moscow
Russian FederationG. A. Novichkova
The Dmitry Rogachev National Medical Research Center of Pediatric Hematology, Oncology and Immunology of Ministry of Healthcare of the Russian Federation
ORCID iD: 0000-0002-2322-5734
Moscow
Russian FederationReferences
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