Application of hematopoietic stem cell transplantation in the treatment of neuroblastoma
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摘要: 高危神经母细胞瘤(neuroblastoma,NB)恶性程度高且预后差,需要综合治疗。造血干细胞移植(hematopoietic stem cell transplantation,HSCT)是诱导缓解后巩固治疗中的重要组成部分,目前以自体造血干细胞移植(autologous HSCT,AHSCT)为主,取得较好的客观缓解,也有复发/难治性患儿接受异基因造血干细胞移植(allogeneic HSCT,allo-HSCT)。作为超大剂量化疗下的支持治疗,双次HSCT比单次HSCT更能改善患儿无事件生存。HSCT预处理方案以白消安/马法兰(Bu/Mel)和卡铂/依托泊苷/马法兰(CEM)为主。Bu/Mel在无事件生存率上优于CEM方案。尽管免疫治疗明显改善了高危患儿的生存期,但是HSCT目前仍在一线综合治疗中处于重要地位。本文拟就HSCT在NB中的应用进行综述。Abstract: High-risk neuroblastoma (HRNBL) is an aggressive solid tumor with a poor prognosis. Treatment is based on multidisciplinary collaboration. Hematopoietic stem cell transplantation (HSCT) plays an important role in the consolidation phase. Autologous stem cell transplantation is frequently used in clinical practice. Some relapsed/refractory patients achieve a good response through the use of allogeneic HSCT (allo-HSCT). Tandem transplantation results in a significantly better event-free survival (EFS) than single transplantation. Conditioning regimens include busulfan/melphalan (Bu/Mel) and carboplatin/etoposide/melphalan (CEM) for advanced-stage neuroblastoma. Bu/Mel is associated with better EFS than CEM. HSCT is the standard of care in the treatment of HRNBL, although immunotherapy has significantly improved survival. This article reviews the use of HSCT in neuroblastoma.
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[1] Yan P, Qi F, Bian LZ, et al. Comparison of incidence and outcomes of neuroblastoma in children, adolescents, and adults in the United States: a surveillance, epidemiology, and end results (SEER) program population study[J]. Med Sci Monit, 2020, 26:e927218. [2] Berthold F, Boos J, Burdach S, et al. Myeloablative megatherapy with autologous stem-cell rescue versus oral maintenance chemotherapy as consolidation treatment in patients with high-risk neuroblastoma: a randomised controlled trial[J]. Lancet Oncol, 2005, 6(9):649-658. doi: 10.1016/S1470-2045(05)70291-6 [3] Pritchard J, Cotterill SJ, Germond SM, et al. High dose melphalan in the treatment of advanced neuroblastoma: results of a randomised trial (ENSG-1) by the European Neuroblastoma Study Group[J]. Pediatr Blood Cancer, 2005, 44(4):348-357. [4] Jain R, Hans R, Totadri S, et al. Autologous stem cell transplant for high-risk neuroblastoma: achieving cure with low-cost adaptations[J]. Pediatr Blood Cancer, 2020, 67(6):e28273. [5] Berthold F, Ernst A, Hero B, et al. Long-term outcomes of the GPOH NB97 trial for children with high-risk neuroblastoma comparing high-dose chemotherapy with autologous stem cell transplantation and oral chemotherapy as consolidation[J]. Br J Cancer, 2018, 119(3):282-290. doi: 10.1038/s41416-018-0169-8 [6] Matthay KK, Reynolds CP, Seeger RC, et al. Long-term results for children with high-risk neuroblastoma treated on a randomized trial of myeloablative therapy followed by 13-cis-retinoic acid: a children's oncology group study[J]. J Clin Oncol, 2009, 27(7):1007-1013. doi: 10.1200/JCO.2007.13.8925 [7] Yu AL, Gilman AL, Ozkaynak MF, et al. Long-term follow-up of a phase III study of ch14.18 (dinutuximab) + cytokine immunotherapy in children with high-risk neuroblastoma: COG study ANBL0032[J]. Clin Cancer Res, 2021, 27(8):2179-2189. [8] Zhao ZS, Shao W, Liu JK. Autologous or allogeneic hematopoietic stem cells transplantation combined with high-dose chemotherapy for refractory neuroblastoma: a protocol for systematic review and meta-analysis[J]. Medicine, 2021, 100(49):e28096. doi: 10.1097/MD.0000000000028096 [9] Kreissman SG, Seeger RC, Matthay KK, et al. Purged versus non-purged peripheral blood stem-cell transplantation for high-risk neuroblastoma (COG A3973): a randomised phase 3 trial[J]. Lancet Oncol, 2013, 14(10):999-1008. doi: 10.1016/S1470-2045(13)70309-7 [10] Bird N, Scobie N, Palmer A, et al. To transplant, or not to transplant? That is the question. A patient advocate evaluation of autologous stem cell transplant in neuroblastoma[J]. Pediatr Blood Cancer, 2022, 69(8):e29663. [11] Elborai Y, Hafez H, Moussa EA, et al. Comparison of toxicity following different conditioning regimens (busulfan/melphalan and carboplatin/etoposide/melphalan) for advanced stage neuroblastoma: experience of two transplant centers[J]. Pediatr Transplant, 2016, 20(2):284-289. doi: 10.1111/petr.12638 [12] Ladenstein R, Pötschger 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. [13] Granger MM, Naranjo A, Bagatell R, et al. Myeloablative busulfan/melphalan consolidation following induction chemotherapy for patients with newly diagnosed high-risk neuroblastoma: children’s oncology group trial ANBL12P1[J]. Transplant Cell Ther, 2021, 27(6):490. [14] George RE, Li SL, Medeiros-Nancarrow C, et al. High-risk neuroblastoma treated with tandem autologous peripheral-blood stem cell-supported transplantation: long-term survival update[J]. J Clin Oncol, 2006, 24(18):2891-2896. doi: 10.1200/JCO.2006.05.6986 [15] Granger M, Grupp SA, Kletzel M, et al. Feasibility of a tandem autologous peripheral blood stem cell transplant regimen for high risk neuroblastoma in a cooperative group setting: a Pediatric Oncology Group study: a report from the Children’s Oncology Group[J]. Pediatr Blood Cancer, 2012, 59(5):902-907. [16] Seif AE, Naranjo A, Baker DL, et al. A pilot study of tandem high-dose chemotherapy with stem cell rescue as consolidation for high-risk neuroblastoma: children’s Oncology Group study ANBL00P1[J].Bone Marrow Transplant, 2013, 48(7):947-952. [17] 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. doi: 10.1001/jama.2019.11642 [18] Hara J, Nitani C, Shichino H, et al. Outcome of children with relapsed high-risk neuroblastoma in Japan and analysis of the role of allogeneic hematopoietic stem cell transplantation[J]. Jpn J Clin Oncol, 2022, 52(5):486-492. doi: 10.1093/jjco/hyac007 [19] Matthay KK, Seeger RC, Reynolds CP, et al. Allogeneic versus autologous purged bone marrow transplantation for neuroblastoma: a report from the Childrens Cancer Group[J]. J Clin Oncol, 1994, 12(11):2382-2389. doi: 10.1200/JCO.1994.12.11.2382 [20] Inoue M, Nakano T, Yoneda A, et al. Graft-versus-tumor effect in a patient with advanced neuroblastoma who received HLA haplo-identical bone marrow transplantation[J]. Bone Marrow Transplant, 2003, 32(1):103-106. doi: 10.1038/sj.bmt.1704070 [21] Yi ES, Son MH, Hyun JK, et al. Predictors of survival in patients with high-risk neuroblastoma who failed tandem high-dose chemotherapy and autologous stem cell transplantation[J]. Pediatr Blood Cancer, 2020, 67(2):e28066. [22] Liu APY, Lee PPW, Kwok JSY, et al. Selective T cell-depleted haploidentical hematopoietic stem cell transplantation for relapsed/refractory neuroblastoma[J]. Pediatr Transplant, 2018, 19:e13240. [23] Wawrzyniak-Dzierżek E, Gajek K, Rybka B, et al. Feasibility and safety of treosulfan, melphalan, and thiotepa-based megachemotherapy with autologous or allogeneic stem cell transplantation in heavily pretreated children with relapsed or refractory neuroblastoma[J]. Biol Blood Marrow Transplant, 2019, 25(9):1792-1797. doi: 10.1016/j.bbmt.2019.05.006 [24] Illhardt T, Toporski J, Feuchtinger T, et al. Haploidentical stem cell transplantation for refractory/relapsed neuroblastoma[J]. Biol Blood Marrow Transplant, 2018, 24(5):1005-1012. [25] Seitz CM, Flaadt T, Mezger M, et al. Immunomonitoring of stage Ⅳ relapsed neuroblastoma patients undergoing haploidentical hematopoietic stem cell transplantation and subsequent GD2 (ch14.18/CHO) antibody treatment[J]. Front Immunol, 2021, 12:690467. [26] Ladenstein R, Pötschger U, Valteau-Couanet D, et al. Investigation of the role of dinutuximab beta-based immunotherapy in the SIOPEN high-risk neuroblastoma 1 trial (HR-NBL1)[J]. Cancers (Basel),2020, 12(2):309. [27] Voeller J, Sondel PM. Advances in anti-GD2 immunotherapy for treatment of high-risk neuroblastoma[J]. J Pediatr Hematol Oncol, 2019, 41(3):163-169. doi: 10.1097/MPH.0000000000001369 [28] Kushner BH, Ostrovnaya I, Cheung IY, et al. Lack of survival advantage with autologous stem-cell transplantation in high-risk neuroblastoma consolidated by anti-GD2 immunotherapy and isotretinoin[J]. Oncotarget, 2016, 7(4):4155-4166. doi: 10.18632/oncotarget.6393 [29] Mora J, Castañeda A, Gorostegui M, et al. Naxitamab combined with granulocyte-macrophage colony-stimulating factor as consolidation for high-risk neuroblastoma patients in complete remission[J]. Pediatr Blood Cancer, 2021, 68(10):e29121. [30] Mora J, Castañeda A, Flores MA, et al. The role of autologous stem-cell transplantation in high-risk neuroblastoma consolidated by anti-GD2 immunotherapy. results of two consecutive studies[J]. Front Pharmacol, 2020, 11:575009. doi: 10.3389/fphar.2020.575009 [31] Mora J. Autologous stem-cell transplantation for high-risk neuroblastoma: historical and critical review[J]. Cancers (Basel), 2022, 14(11):2572. doi: 10.3390/cancers14112572
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