Ladenstein R, Lasset C, Hartmann O, Klingebiel T, Bouffet E, Gadner H, Paolucci P, Burdach S, Chauvin F, Pinkerton R
Coordinating Center of the European Bone Marrow Transplantation Solid Tumor Working Party (EBMT-STWP), Centre Léon Bérard, Lyon, France.
Bone Marrow Transplant. 1994 Jul;14(1):37-46.
A case control study was performed to investigate the potential advantage of allogeneic bone marrow transplantation in advanced or poorly responding neuroblastoma first remission patients using the European BMT Solid Tumor Registry. Seventeen allogeneic and 34 autologous bone marrow transplantation (BMT) cases were matched based on a number of prognostic factors including age, sex, prior treatment duration, pre-graft response status and bone and BM involvement before BMT. Only single BMT procedures are included. The median age at diagnosis was 47 months (range 18-113 months). The median follow-up time since BMT is 58 months (range 13-133 months). The only significant prognostic factor within the allogeneic BMT (p = 0.012) and autologous BMT groups (p = 0.025) was residual skeletal disease before BMT, detected by mIBG in 86% of the cases. However, the progression-free survival was not significantly different: 35% and 41% at 2 years, respectively. Only half of the allogeneic BMT patients had developed graft-versus-host disease (GVHD): 7 of 9 grade I-II and only 2 of 9 grade IV. The median donor age was very young with 74 months (range 20-240 months) and 10 of 17 were sex matched. Thus absence of GVHD risk factors in young children could be the major obstacle in achieving an anti-tumor effect with allogeneic BMT in neuroblastoma.
利用欧洲骨髓移植实体瘤登记处，开展了一项病例对照研究，以调查异基因骨髓移植在晚期或反应不佳的神经母细胞瘤首次缓解患者中的潜在优势。根据年龄、性别、既往治疗时长、移植前反应状态以及骨髓移植前骨骼和骨髓受累情况等多项预后因素，对17例异基因和34例自体骨髓移植（BMT）病例进行了匹配。仅纳入单次BMT手术。诊断时的中位年龄为47个月（范围18 - 113个月）。自骨髓移植后的中位随访时间为58个月（范围13 - 133个月）。异基因BMT组（p = 0.012）和自体BMT组（p = 0.025）中唯一显著的预后因素是骨髓移植前残留的骨骼疾病，86%的病例通过间碘苄胍（mIBG）检测到。然而，无进展生存期无显著差异：2年时分别为35%和41%。仅一半异基因BMT患者发生了移植物抗宿主病（GVHD）：9例中有7例为I - II级，9例中仅2例为IV级。供者的中位年龄非常年轻，为74个月（范围20 - 240个月），17例中有10例性别匹配。因此，幼儿缺乏GVHD风险因素可能是异基因BMT在神经母细胞瘤中实现抗肿瘤效果的主要障碍。
