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
Department of Hematology/Oncology, Shanghai Children's Hospital, School of Medicine, Shanghai Jiao Tong University, Shanghai 200040, China
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.
WANG Li-Hui,CHEN Kai,ZHANG Na et al. Effectiveness of autologous hematopoietic stem cell transplantation in the treatment of high-risk neuroblastoma in children: a single-center clinical study[J]. CJCP, 2023, 25(5): 476-482.
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.
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.
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.
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.
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.
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.
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.
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.
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.
