Department of Pediatric Hematology, Oncology and Transplantology, Medical University of Lublin, Lublin, Poland.
Bone Marrow Transplant. 2012 Dec;47(12):1530-4. doi: 10.1038/bmt.2012.78. Epub 2012 May 21.
According to the published report on current practice of hematopoietic SCT in Europe, high-dose therapy (HDT) with autologous stem cell support is a standard of care in paediatric patients with high risk (HR) or relapsed Ewing's sarcoma (ES). Randomized trials, however, have not confirmed the value of this procedure yet. In this retrospective analysis we intended to evaluate the role of HDT as a consolidation therapy in first remission of ES. A total of 102 patients were included in the analysis and divided according to the following risk factors: metastatic disease at presentation, feasibility of surgery and histological response after induction. Forty-one patients were classified as standard risk (SR) patients, while the remaining 61 children, with at least one risk factor, were classified as HR patients. HR group patients were non-randomized and qualified according to the decision of the local clinician to give a conventional consolidation (CC) or to perform high-dose chemotherapy and radiotherapy in selected patients. Twenty-six children were given CC while 35 patients were treated with HDT. The HDT consisted of oral BU 4 mg/kg p.o. in divided doses daily for 4 days (total dose 16 mg/kg) followed by melphalan 140 mg/m(2) i.v. on day -2. Probability of relapse-free survival (RFS) in median observation time was significantly worse in HR patients who were given CC therapy as compared with children with HR features receiving high-dose chemotherapy (0.27 vs 0.66 (P = 0.008); OS 0.31 vs 0.71 (P = 0.007), respectively). Patients from the SR group had a probability of RFS of 0.72 and OS of 0.75, and the difference between SR and HR patients after HDT was NS (P = 0.37). Our observation confirms that the consolidation of the first-line treatment with BU and melphalan improves the outcome in ES patients with HR features.
根据发表的关于欧洲造血干细胞移植现状的报告，高危（HR）或复发性尤文肉瘤（ES）患儿采用自体干细胞支持的大剂量化疗（HDT）是标准治疗方法。然而，随机试验尚未证实该程序的价值。在这项回顾性分析中，我们旨在评估 HDT 作为 ES 首次缓解后的巩固治疗的作用。共有 102 名患者纳入分析，并根据以下危险因素进行分组：就诊时的转移性疾病、手术可行性和诱导后组织学反应。41 名患者被归类为标准风险（SR）患者，而其余 61 名儿童至少有一个危险因素，被归类为 HR 患者。HR 组患者为非随机分组，根据当地临床医生的决定，符合条件的患者接受常规巩固治疗（CC）或在选定患者中进行大剂量化疗和放疗。26 名儿童接受 CC 治疗，35 名儿童接受 HDT 治疗。HDT 包括口服 BU 4mg/kg 每日分 4 次口服（总剂量 16mg/kg），随后在第-2 天给予美法仑 140mg/m²iv。在中位观察时间内，接受 CC 治疗的 HR 患者的无复发生存率（RFS）明显低于接受大剂量化疗的 HR 特征儿童（0.27 比 0.66（P=0.008）；OS 0.31 比 0.71（P=0.007））。SR 组患者的 RFS 概率为 0.72，OS 概率为 0.75，SR 和 HR 患者接受 HDT 后差异无统计学意义（P=0.37）。我们的观察结果证实，用 BU 和美法仑巩固一线治疗可改善 HR 特征 ES 患者的预后。
