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首页> 外文期刊>Pediatric Hematology and Oncology >High-dose busulfan and melphalan as conditioning regimen for autologous peripheral blood progenitor cell transplantation in high-risk ewing sarcoma patients: A long-term follow-up single-center study
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High-dose busulfan and melphalan as conditioning regimen for autologous peripheral blood progenitor cell transplantation in high-risk ewing sarcoma patients: A long-term follow-up single-center study

机译:大剂量白消安和美法仑作为高危尤因肉瘤患者自体外周血祖细胞移植的调节方案:一项长期随访的单中心研究

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The aim of this retrospective study was to analyze the outcome and identify risk factors associated to progression-free survival (PFS) in 47 children with high-risk Ewing sarcoma who underwent autologous peripheral blood stem cell (PBSC) transplantation in the authors' institution between 1995 and 2009. The conditioning regimen used in all patients consisted of high dose of busulfan and melphalan. Median age was 13 years (range: 4-21 years). Forty-three percent of patients had metastases at diagnosis. The probability of transplant-related mortality (TRM) was 6 ± 3. Recurrence/progressive disease was observed in 17 patients. The probability of recurrence/progression was 39 ± 7. With a median follow-up of 92 months (range: 6-168 months), the PFS was 56 ± 4 for the whole group. In multivariate analysis, localized disease at diagnosis and obtaining complete remission (CR) by 3 months after transplantation were variables associated to better outcomes. The probability of PFS was 78 ± 8 and 27 ± 10 for patients with localized and metastatic disease at diagnosis, respectively (P .0001). This retrospective study shows a high long-term survival using high dose of busulfan and melphalan as conditioning regimen in children with high-risk Ewing tumors. Patients with localized disease at diagnosis and those with good response to treatment before or after transplant would benefit most.
机译:这项回顾性研究的目的是分析作者所在机构之间47例接受自体外周血干细胞(PBSC)移植的高危尤因肉瘤儿童的结局并确定与无进展生存(PFS)相关的危险因素。 1995年和2009年。在所有患者中使用的调理方案包括高剂量的白消安和美法仑。中位年龄为13岁(范围:4-21岁)。百分之四十三的患者在诊断时有转移。移植相关死亡率(TRM)的可能性为6±3。在17例患者中观察到了复发/进行性疾病。复发/进展的可能性为39±7。中位随访时间为92个月(范围:6-168个月),整个组的PFS为56±4。在多变量分析中,诊断时的局部疾病以及移植后3个月之前获得完全缓解(CR)是与更好预后相关的变量。诊断时患有局灶性和转移性疾病的患者的PFS概率分别为78±8和27±10(P .0001)。这项回顾性研究显示,在患有高危尤因肿瘤的儿童中,使用大剂量的白消安和美法仑作为调理方案可实现较高的长期存活率。诊断时具有局部疾病的患者以及移植前后对治疗反应良好的患者将受益最大。

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