Mugishima H, Harada K, Suzuki T, Chin M, Shimada T, Takamura M, Shichino H, Fujisawa T, Ichikawa M, Iwata M
Department of Pediatrics, Nihon University, Tokyo, Japan.
Acta Paediatr Jpn. 1995 Aug;37(4):493-9. doi: 10.1111/j.1442-200x.1995.tb03361.x.
Encouraging results are reported with high-dose chemotherapy and total body irradiation followed by autologous bone marrow transplantation in the treatment of advanced neuroblastoma. However, relapse remains a significant problem. We used high-dose chemotherapy, surgery, intraoperative radiation and an autologous bone marrow transplant treated in vitro to remove tumor cells followed by 13-cis-retinoic acid to treat 36 children with advanced neuroblastoma. This comprehensive treatment appears to improve the survival rate of patients with advanced neuroblastoma, including those with N-myc amplification and bony involvement. The disease-free survival rate was 66% (95% confidence interval, 49-84%) at 3 years. All patients who received 13-cis-retinoic acid developed cheilitis, but no bone marrow depression occurred in these patients. Five patients developed hemolytic uremic syndrome (HUS) post-transplant. This may have been related to the procedure used for total body irradiation. Patients who had their kidneys shielded during this procedure did not develop this syndrome. Patients who received local irradiation at the primary site showed no evidence of relapse in this region, indicating that such therapy may help to prevent a relapse. These data suggest a high rate of 3 year disease-free survival with this treatment strategy. The nonrandomized nature of the study and use of multiple modalities precludes analysis of the specific contribution of each.
据报道，大剂量化疗和全身照射后进行自体骨髓移植治疗晚期神经母细胞瘤取得了令人鼓舞的结果。然而，复发仍然是一个重大问题。我们采用大剂量化疗、手术、术中放疗以及体外处理以去除肿瘤细胞的自体骨髓移植，随后使用13-顺式维甲酸治疗36例晚期神经母细胞瘤患儿。这种综合治疗似乎提高了晚期神经母细胞瘤患者的生存率，包括那些N-myc扩增和骨受累的患者。3年时无病生存率为66%（95%置信区间，49 - 84%）。所有接受13-顺式维甲酸治疗的患者均出现唇炎，但这些患者均未发生骨髓抑制。5例患者移植后发生溶血尿毒综合征（HUS）。这可能与全身照射所采用的程序有关。在此程序中肾脏得到屏蔽的患者未发生该综合征。在原发部位接受局部照射的患者在该区域未显示复发迹象，表明这种治疗可能有助于预防复发。这些数据表明这种治疗策略具有较高的3年无病生存率。该研究的非随机性质以及多种治疗方式的使用妨碍了对每种方式具体贡献的分析。
