Allogeneic bone marrow transplantation following a busulfan-based conditioning regimen in young children with acute lymphoblastic leukemia: a Cooperative Study of the Société Française de Greffe de Moelle.
Allogeneic bone marrow transplantation following a busulfan-based conditioning regimen in young children with acute lymphoblastic leukemia: a Cooperative Study of the Société Française de Greffe de Moelle.
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对患有急性淋巴细胞白血病的幼儿进行以白消安为基础的调理方案后的同种异体骨髓移植：法国 Société Française de Greffe de Moelle 的合作研究。
DOI：
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发表时间：
1995
影响因子：
4.8
通讯作者：
J. Donadieu
中科院分区：
文献类型：
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作者：
A. von Bueltzingsloewen;H. Espérou;G. Souillet;F. Demeocq;F. Mechinaud;G. Michel;A. Sadoun;F. Bernaudin;G. Cornu;J. Donadieu
尽管实施了强化化疗方案，一个ALL儿童亚组仍有较高的复发风险，并可能从同种异体BMT中获益。最常用的细胞减灭方案是TBI与环磷酰胺联合。然而，各种各样的长期后遗症已随之放射治疗，特别是在年幼的儿童。本回顾性多中心研究分析了4岁以下ALL患儿在接受无辐射预处理方案后HLA基因一致BMT的结局。21名儿童（中位年龄：28个月，范围6-48）接受了以白消安为基础的环磷酰胺或美法仑+/-依托泊苷+/-阿糖胞苷方案。16例具有初始不良预后因素的患者在第一次完全缓解（CR）时接受移植，5例患者在复发或第二次CR时接受移植。中位随访47个月，结果显示总体4年DFS为61.1%。在8例患者中观察到白血病复发。预处理方案耐受性良好，无移植相关死亡。基于白消安的BMT制备方案可能是幼儿含TBI方案的治疗替代方案。目前的努力旨在通过优化化疗的杀肿瘤潜力和异基因BMT的移植物抗白血病效应来降低这些儿童的复发率。
A subgroup of children with ALL remains at high risk of relapse despite the administration of intensive chemotherapeutic protocols and may benefit from allogeneic BMT. The cytoreductive regimen used most often combines TBI with cyclophosphamide. Nevertheless, miscellaneous long-term sequelae have been consequent upon radiotherapy, especially in young children. This retrospective multicentric study analyzes the outcome of children with ALL under 4 years of age receiving an HLA-genoidentical BMT following a radiation-free preparative regimen. A busulfan-based regimen with cyclophosphamide or melphalan +/- etoposide +/- cytarabine was given to 21 children (median age: 28 months, range 6-48). Sixteen patients with initial poor prognostic factors were transplanted in first complete response (CR) and five patients in relapse or second CR. With a median follow-up of 47 months, the results show an overall 4-year DFS of 61.1%. Leukemic recurrence was observed in eight patients. The preparative regimen was well-tolerated and there were no transplant-related deaths. A busulfan-based BMT preparative regimen may be a therapeutic alternative to TBI-containing regimens in young children. Efforts are currently aimed at reducing the relapse rate in these children by optimizing the tumoricidal potential of chemotherapy and the graft-versus-leukemia effect of allogeneic BMT.