Effectiveness of autologous hematopoietic stem cell transplantation in the treatment of high-risk neuroblastoma in children: a single-center clinical study

WANG Li-Hui; CHEN Kai; ZHANG Na; YANG Jing-Wei; ZHANG Ting; SHAO Jing-Bo

doi:10.7499/j.issn.1008-8830.2301005

PDF(580 KB)

Chinese Journal of Contemporary Pediatrics ›› 2023, Vol. 25 ›› Issue (5) : 476-482. DOI: 10.7499/j.issn.1008-8830.2301005

CLINICAL RESEARCH

Effectiveness of autologous hematopoietic stem cell transplantation in the treatment of high-risk neuroblastoma in children: a single-center clinical study

WANG Li-Hui, CHEN Kai, ZHANG Na, YANG Jing-Wei, ZHANG Ting, SHAO Jing-Bo

Author information +

History +

Abstract

Objective To investigate the effectiveness of high-dose chemotherapy combined with autologous hematopoietic stem cell transplantation (ASCT) in the treatment of children with high-risk neuroblastoma (NB). Methods A retrospective analysis was performed on 29 children with high-risk NB who were admitted to Shanghai Children's Hospital and were treated with high-dose chemotherapy combined with ASCT from January 2013 to December 2021, and their clinical features and prognosis were analyzed. Results Among the 29 children treated by high-dose chemotherapy combined with ASCT, there were 18 boys (62%) and 11 girls (38%), with a median age of onset of 36 (27, 59) months. According to the International Neuroblastoma Staging System, 6 children (21%) had stage III NB and 23 children (79%) had stage IV NB, and the common metastatic sites at initial diagnosis were bone in 22 children (76%), bone marrow in 21 children (72%), and intracalvarium in 4 children (14%). All 29 children achieved reconstruction of hematopoietic function after ASCT. After being followed up for a median time of 25 (17, 45) months, 21 children (72%) had continuous complete remission and 8 (28%) experienced recurrence. The 3-year overall survival rate and event-free survival rate were 68.9%±16.1% and 61.4%±14.4%, respectively. Presence of bone marrow metastasis, neuron-specific enolase ≥370 ng/mL and positive bone marrow immunophenotyping might reduce the 3-year event-free survival rate (P<0.05). Conclusions Children with high-risk NB who have bone marrow metastasis at initial diagnosis tend to have a poor prognosis. ASCT combined with high-dose chemotherapy can effectively improve the prognosis of children with NB with a favorable safety profile.

Key words

Neuroblastoma / Autologous hematopoietic stem cell transplantation / Conditioning regimen / Prognosis / Child

Cite this article

EndNote

Ris (Procite)

Bibtex

Download Citations

WANG Li-Hui, CHEN Kai, ZHANG Na, YANG Jing-Wei, ZHANG Ting, SHAO Jing-Bo. Effectiveness of autologous hematopoietic stem cell transplantation in the treatment of high-risk neuroblastoma in children: a single-center clinical study[J]. Chinese Journal of Contemporary Pediatrics. 2023, 25(5): 476-482 https://doi.org/10.7499/j.issn.1008-8830.2301005

References

1 Gatta G, Botta L, Rossi S, et al. Childhood cancer survival in Europe 1999-2007: results of EUROCARE-5—a population-based study[J]. Lancet Oncol, 2014, 15(1): 35-47. PMID: 24314616. DOI: 10.1016/S1470-2045(13)70548-5.
2 Ward E, DeSantis C, Robbins A, et al. Childhood and adolescent cancer statistics, 2014[J]. CA Cancer J Clin, 2014, 64(2): 83-103. PMID: 24488779. DOI: 10.3322/caac.21219.
3 梁伟玲, 叶小帆, 钟共, 等. 儿童4期神经母细胞瘤联合治疗的临床疗效观察[J]. 中国当代儿科杂志, 2022, 24(7): 759-764. PMID: 35894190. PMCID: PMC9336616. DOI: 10.7499/j.issn.1008-8830.2203049.
4 任海龙, 何国倩, 郭霞. 神经母细胞瘤骨和骨髓转移机制的研究进展[J]. 中华儿科杂志, 2022, 60(3): 258-262. PMID: 35240752. DOI: 10.3760/cma.j.cn112140-20210704-00550.
5 刘涛, 盛庆丰, 刘江斌, 等. INSS 4期神经母细胞瘤患儿3年无事件生存率的相关因素分析[J]. 临床小儿外科杂志, 2022, 21(2): 121-127. DOI: 10.3760/cma.j.cn.101785-202011036-005.
6 张婷, 李艳华, 李珊珊, 等. 单中心儿童神经母细胞瘤4期患者临床疗效及预后分析[J]. 中国小儿血液与肿瘤杂志, 2020, 25(3): 153-158. DOI: 10.3969/j.issn.1673-5323.2020.03.007.
7 Bearman SI, Appelbaum FR, Buckner CD, et al. Regimen-related toxicity in patients undergoing bone marrow transplantation[J]. J Clin Oncol, 1988, 6(10): 1562-1568. PMID: 3049951. DOI: 10.1200/JCO.1988.6.10.1562.
8 Brodeur GM, Pritchard J, Berthold F, et al. Revisions of the international criteria for neuroblastoma diagnosis, staging, and response to treatment[J]. J Clin Oncol, 1993, 11(8):1466-1477. PMID: 8336186. DOI: 10.1200/JCO.1993.11.8.1466.
9 Shao JB, Lu ZH, Huang WY, et al. A single center clinical analysis of children with neuroblastoma[J]. Oncol Lett, 2015, 10(4): 2311-2318. PMID: 26622841. PMCID: PMC4579968. DOI: 10.3892/ol.2015.3588.
10 汤梦婕, 袁晓军, 安霞, 等. 转移性神经母细胞瘤伴骨和骨髓转移的治疗疗效及预后分析[J]. 中华转移性肿瘤杂志, 2019, 2(3): 18-24. DOI: 10.3760/cma.j.issn.2096-5400.2019.03.004.
11 Samim A, Tytgat GAM, Bleeker G, et al. Nuclear medicine imaging in neuroblastoma: current status and new developments[J]. J Pers Med, 2021, 11(4): 270. PMID: 33916640. PMCID: PMC8066332. DOI: 10.3390/jpm11040270.
12 马越. 472例儿童神经母细胞瘤的临床特征及预后的单中心回顾性分析[D]. 重庆: 重庆医科大学, 2021. DOI: 10.27674/d.cnki.gcyku.2021.000758.
13 Moreno L, Guo D, Irwin MS, et al. A nomogram of clinical and biologic factors to predict survival in children newly diagnosed with high-risk neuroblastoma: an International Neuroblastoma Risk Group project[J]. Pediatr Blood Cancer, 2021, 68(3): e28794. PMID: 33205902. DOI: 10.1002/pbc.28794.
14 苏雁, 马晓莉, 王焕民, 等. 单中心458例高危神经母细胞瘤患儿临床特征及预后分析[J]. 中华儿科杂志, 2020, 58(10): 796-801. DOI: 10.3760/cma.j.cn112140-20200525-00540.
15 Moroz V, Machin D, Hero B, et al. The prognostic strength of serum LDH and serum ferritin in children with neuroblastoma: a report from the International Neuroblastoma Risk Group (INRG) project[J]. Pediatr Blood Cancer, 2020, 67(8): e28359. PMID: 32472746. DOI: 10.1002/pbc.28359.
16 Katzenstein HM, Cohn SL, Shore RM, et al. Scintigraphic response by 123I-metaiodobenzylguanidine scan correlates with event-free survival in high-risk neuroblastoma[J]. J Clin Oncol, 2004, 22(19): 3909-3915. PMID: 15459212. DOI: 10.1200/JCO.2004.07.144.
17 Ladenstein R, P?tschger U, Pearson ADJ, et al. Busulfan and melphalan versus carboplatin, etoposide, and melphalan as high-dose chemotherapy for high-risk neuroblastoma (HR-NBL1/SIOPEN): an international, randomised, multi-arm, open-label, phase 3 trial[J]. Lancet Oncol, 2017, 18(4): 500-514. PMID: 28259608. DOI: 10.1016/S1470-2045(17)30070-0.
18 Park JR, Kreissman SG, London WB, et al. Effect of tandem autologous stem cell transplant vs single transplant on event-free survival in patients with high-risk neuroblastoma: a randomized clinical trial[J]. JAMA, 2019, 322(8): 746-755. PMID: 31454045. PMCID: PMC6714031. DOI: 10.1001/jama.2019.11642.
19 GD2单抗治疗神经母细胞瘤临床应用协作组. GD2抗体达妥昔单抗β治疗神经母细胞瘤的临床应用专家共识(2021年版)[J]. 临床儿科杂志, 2022, 40(1): 14-20. DOI: 10.12372/jcp.2022.21e1685.