Saarinen U M, Wikström S, Mäkipernaa A, Lanning M, Perkkiö M, Hovi L, Rapola J, Sariola H
Division of Pediatric Hematology/Oncology, Children's Hospital, University of Helsinki, Finland.
J Clin Oncol. 1996 Oct;14(10):2791-802. doi: 10.1200/JCO.1996.14.10.2791.
To evaluate the following prospectively in poor-risk neuroblastoma (NBL) patients: (1) the feasibility and efficacy of in vivo purging of bone marrow; and (2) the outcome after autologous bone marrow transplantation (ABMT) when immunologically tumor-free, unpurged autografts were used.
Twenty-three children with poor-risk NBL were evaluated during induction chemotherapy by repeat bone marrow examinations, including aspirate, biopsy, and an immunofluorescence method using the anti-GD2 monoclonal antibody 3A7. Nineteen patients completed the program with surgery with or without local irradiation followed by ABMT.
Autologous bone marrow grafts, both immunologically and cytologically clean, were obtained and used in 19 of 23 children. The overall 4-year disease-free survival of the 19 grafted children was 53%, with a toxic death rate of 16% and a posttransplant relapse rate of 37%. According to the in vivo purging efficacy of the 18 children with initial marrow disease, the following three groups were formed: patients with (1) perfect in vivo purging (n = 5); (2) eventually successful in vivo purging (n = 8); and (3) unsuccesful in vivo purging (n = 5). The 4-year DFS was 100%, 67%, and 0%, respectively (P < 0.001). The five patients with unsuccessful in vivo purging failed because of resistant/progressive bulky disease.
In patients with poor-risk NBL, in vivo purging of bone marrow by conventional chemotherapy is feasible, can be monitored, and the purging efficacy during the first 3 months after diagnosis is a strong prognostic factor reflecting tumor responsiveness to therapy. Autografting with immunologically clean, unpurged marrows gives a DFS well comparable to previous studies using ex vivo purging.
前瞻性评估高危神经母细胞瘤（NBL）患者的以下情况：（1）骨髓体内净化的可行性和疗效；（2）使用免疫肿瘤学上无肿瘤、未净化的自体移植物进行自体骨髓移植（ABMT）后的结局。
对23例高危NBL患儿在诱导化疗期间进行评估，通过重复骨髓检查，包括抽吸、活检以及使用抗GD2单克隆抗体3A7的免疫荧光法。19例患者完成了该方案，接受了手术（有或无局部放疗），随后进行ABMT。
23例患儿中有19例获得了免疫和细胞学上均清洁的自体骨髓移植物并用于移植。19例接受移植患儿的4年无病生存率为53%，毒性死亡率为16%，移植后复发率为37%。根据18例初始骨髓有病变患儿的体内净化疗效，分为以下三组：（1）体内净化完美（n = 5）；（2）体内净化最终成功（n = 8）；（3）体内净化未成功（n = 5）。4年无病生存率分别为100%、67%和0%（P < 0.001）。5例体内净化未成功的患者因耐药/进行性大块病变而失败。
在高危NBL患者中，通过传统化疗进行骨髓体内净化是可行的，可进行监测，诊断后前3个月的净化疗效是反映肿瘤对治疗反应性的有力预后因素。使用免疫清洁、未净化的骨髓进行自体移植的无病生存率与先前使用体外净化的研究相当。
