Treatment of high-risk neuroblastoma with intensive chemotherapy, radiotherapy, autologous bone marrow transplantation, and 13-cis-retinoic acid
Treatment of high-risk neuroblastoma with intensive chemotherapy, radiotherapy, autologous bone marrow transplantation, and 13-cis-retinoic acid
复制标题
DOI：
10.1056/nejm199910143411601
复制
发表时间：
1999-10-14
影响因子：
158.5
通讯作者：
Reynolds, CP
中科院分区：
文献类型：
--
作者：
Matthay, KK;Villablanca, JG;Reynolds, CP
背景 高危神经母细胞瘤患儿预后不良。在本研究中，我们评估了清髓性治疗联合自体骨髓移植相较于单纯化疗是否能提高无事件生存率，以及后续使用13 - 顺式维甲酸（异维甲酸）治疗是否能进一步提高无事件生存率。
方法 所有患者均接受相同的初始化疗方案，无疾病进展的患者随后被随机分配接受清髓性化疗、全身照射以及净化神经母细胞瘤细胞后的自体骨髓移植治疗，或者仅接受三个周期的强化化疗。所有完成细胞毒性治疗且无疾病进展的患者随后再次被随机分配接受不再进一步治疗或接受13 - 顺式维甲酸治疗6个月。
结果 在首次随机分组3年后，被分配接受移植的189名患者的平均（±标准误）无事件生存率显著优于被分配接受继续化疗的190名患者（34±4%对22±4%，P = 0.034）。在第二次随机分组3年后，被分配接受13 - 顺式维甲酸治疗的130名患者的无事件生存率显著优于被分配不再接受进一步治疗的128名患者（46±6%对29±5%，P = 0.027）。
结论 清髓性治疗和自体骨髓移植可提高高危神经母细胞瘤患儿的无事件生存率。此外，对于化疗或移植后无疾病进展的患者，13 - 顺式维甲酸治疗是有益的。（《新英格兰医学杂志》1999年；341卷：1165 - 1173页）（1999年，马萨诸塞州医学协会）
Background Children with high-risk neuroblastoma have a poor outcome. In this study, we assessed whether myeloablative therapy in conjunction with transplantation of autologous bone marrow improved event-free survival as compared with chemotherapy alone, and whether subsequent treatment with 13-cis-retinoic acid (isotretinoin) further improves event-free survival.Methods All patients were treated with the same initial regimen of chemotherapy, and those without disease progression were then randomly assigned to receive continued treatment with myeloablative chemotherapy, total-body irradiation, and transplantation of autologous bone marrow purged of neuroblastoma cells or to receive three cycles of intensive chemotherapy alone. All patients who completed cytotoxic therapy without disease progression were then randomly assigned to receive no further therapy or treatment with 13-cis-retinoic acid for six months.Results The mean (+/-SE) event-free survival rate three years after the first randomization was significantly better among the 189 patients who were assigned to undergo transplantation than among the 190 patients assigned to receive continuation chemotherapy (34+/-4 percent vs. 22+/-4 percent, P = 0.034). The event-free survival rate three years after the second randomization was significantly better among the 130 patients who were assigned to receive 13-cis-retinoic acid than among the 128 patients assigned to receive no further therapy (46+/-6 percent vs. 29+/-5 percent, P = 0.027).Conclusions Treatment with myeloablative therapy and autologous bone marrow transplantation improved event-free survival among children with highrisk neuroblastoma. In addition, treatment with 13-cis-retinoic acid was beneficial for patients without progressive disease when it was administered after chemotherapy or transplantation. (N Engl J Med 1999; 341:1165-73.) (C)1999, Massachusetts Medical Society.