Kang H J, Shin H Y, Choi H S, Han K S, Ahn H S
Pediatric Oncology Branch, National Cancer Center, Gyeonggi-do, Republic of Korea.
Bone Marrow Transplant. 2004 Mar;33(5):471-6. doi: 10.1038/sj.bmt.1704389.
Autologous peripheral blood stem cell transplantation (APBSCT) after intensifying conditioning is one of the post-remission therapeutic options in childhood acute myeloid leukemia (AML) patients without a matched family donor, but the optimal conditioning regimen has not been defined. This study was performed to evaluate the efficacy of a novel conditioning regimen without busulfan or total body irradiation. In total, 28 children with AML underwent APBSCT with BCVAC (BCNU, etoposide, cytosine arabinoside and cyclophosphamide) conditioning regimen during first remission. The event-free survival rate was 71.43% for all patients and the only cause of treatment failure was relapse. Eight male patients recurred at 1-11 months (median 5 months) after APBSCT. One patient remains alive with salvage therapy after relapse. With the exception of fever, mucositis and diarrhea, no serious complications occurred during APBSCT, including veno-occlusive disease (VOD), and there was no transplantation-related mortality. One patient developed secondary MDS after APBSCT but recovered hematologically on medication. APBSCT with BCVAC conditioning was found to be a safe and effective alternative option for patients with childhood AML in first remission, without a matched family donor.
强化预处理后的自体外周血干细胞移植（APBSCT）是没有匹配的家族供体的儿童急性髓系白血病（AML）患者缓解后治疗选择之一，但最佳预处理方案尚未确定。本研究旨在评估一种不含白消安或全身照射的新型预处理方案的疗效。共有28例AML患儿在首次缓解期接受了采用BCVAC（卡莫司汀、依托泊苷、阿糖胞苷和环磷酰胺）预处理方案的APBSCT。所有患者的无事件生存率为71.43%，治疗失败的唯一原因是复发。8例男性患者在APBSCT后1 - 11个月（中位时间5个月）复发。1例患者复发后经挽救治疗仍存活。除发热、黏膜炎和腹泻外，APBSCT期间未发生包括静脉闭塞性疾病（VOD）在内的严重并发症，也没有移植相关死亡。1例患者在APBSCT后发生继发性骨髓增生异常综合征，但经药物治疗血液学恢复。对于首次缓解期、没有匹配家族供体的儿童AML患者，采用BCVAC预处理的APBSCT是一种安全有效的替代选择。
