Section of Pediatrics, National Kyusyu Cancer Center, Fukuoka, Japan.
Bone Marrow Transplant. 2011 Aug;46(8):1057-62. doi: 10.1038/bmt.2010.253. Epub 2010 Nov 1.
We evaluated the efficacy and safety of the conditioning regimen that consisted of TBI and melphalan (L-PAM), followed by hematopoietic SCT (HSCT) in 23 children with advanced hematological malignancies. The median age at HSCT was 9 (range, 2-15) years. The underlying diseases were ALL in 16 patients (5 in CR2, 3 in CR3, 6 in relapse (RP) and 2 in induction failure (IF)), AML in 4 patients (3 in RP and 1 in IF) and non-Hodgkin's lymphoma in 3 patients (1 in CR3, 1 in CR4 and 1 in RP). The stem cell sources were BM for 19 patients and cord blood for 4 patients. All patients received the conditioning regimen that consisted of TBI 12 or 13.2 Gy and L-PAM 210 mg/m(2). In all, 22 patients engrafted on the median of day 16 (range, 10-23). The regimen was well tolerated and common regimen-related toxicities (RRTs) included grade II stomatitis and grade I hepatic toxicity. The cumulative incidences of RP and TRM were 47.6 and 21.5%, respectively. At a median follow-up of 24.4 months, the probability of disease-free survival was 41.0%. The regimen may provide sufficient anti-leukemic effect without increased RRT for advanced pediatric hematological malignancies.
我们评估了包含 TBI 和马法兰（L-PAM）的预处理方案在 23 例晚期血液系统恶性肿瘤患儿中的疗效和安全性，这些患儿随后接受了造血干细胞移植（HSCT）。HSCT 时的中位年龄为 9 岁（范围：2-15 岁）。基础疾病包括 16 例 ALL（2 例在 CR2，3 例在 CR3，6 例在复发（RP），2 例在诱导失败（IF）），4 例 AML（3 例在 RP，1 例在 IF）和 3 例非霍奇金淋巴瘤（1 例在 CR3，1 例在 CR4，1 例在 RP）。干细胞来源为 19 例患者的骨髓和 4 例患者的脐带血。所有患者均接受了包含 TBI 12 或 13.2 Gy 和 L-PAM 210 mg/m2 的预处理方案。共有 22 例患者在中位第 16 天（范围：10-23 天）植入。该方案耐受性良好，常见的预处理相关毒性（RRTs）包括 II 度口腔炎和 I 度肝毒性。RP 和 TRM 的累积发生率分别为 47.6%和 21.5%。中位随访 24.4 个月时，无病生存率为 41.0%。对于晚期儿科血液系统恶性肿瘤，该方案可能提供足够的抗白血病效果，而不会增加 RRT。
