High-dose carboplatin, thiotepa, and etoposide with autologous stem cell rescue for patients with previously irradiated recurrent medulloblastoma
High-dose carboplatin, thiotepa, and etoposide with autologous stem cell rescue for patients with previously irradiated recurrent medulloblastoma
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DOI：
10.1093/neuonc/nop031
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发表时间：
2010-03-01
期刊：
影响因子：
15.9
通讯作者：
Finlay, Jonathan L.
中科院分区：
文献类型：
--
作者：
Dunkel, Ira J.;Gardner, Sharon L.;Finlay, Jonathan L.
复发性髓母细胞瘤是高度致命的，在以前的辐射病人。方案入组前达到极轻微疾病状态的既往接受过放疗的M-0-M-3复发患者接受卡铂治疗（Calvert公式，曲线下面积= 7 mg/mL min，最大500 mg/m（2）天）油天-8至6，噻替派（300 mg/m2/天）和依托泊苷（250 mg/m2/天），然后在第0天进行自体干细胞拯救（ASCR）。25例患者在ASCR时年龄为7.6-44.7岁（中位数13.8岁），接受了治疗。3例（12%）在ASCR后30天内死于治疗相关毒性，提示多器官系统衰竭（n = 2）和曲霉菌感染伴静脉闭塞性疾病（n = 1）。肿瘤复发16例，中位时间8.5个月（范围2.3-58.5个月）。6例为ASCR后中位151.2个月（范围127.2-201.6个月）的无事件生存者。ASCR后10年时，Kaplan-Meier估计的中位总生存期为26.8个月（95% CI：11.9-51.1个月），无事件生存期（EFS）和总生存期均为24%（95% CI：9.8%-41.7%）。方案前M-0（vs M-1 +）复发、缺乏复发的组织确认和单独放疗（RT）的初始治疗（vs RT +化疗）与更好的EFS无显著相关性（分别为P = .33、.34和.27）。在接受额外RT作为其恢复治疗的一部分的患者（n = 5）中观察到了更好的EFS趋势（P = 0.07），其复发性疾病被证明对再诱导化疗敏感（P = 0.09）。这种回收策略为一些既往接受过放射治疗的复发性髓母细胞瘤患者提供了长期EFS。额外RT的使用可能与更好的结局相关。
Recurrent medulloblastoma is highly lethal in previously irradiated patients. Previously irradiated patients with M-0-M-3 recurrences who achieved a minimal disease state prior to protocol enrollment received carboplatin (Calvert formula with area under the curve = 7 mg/mL min, maximum 500 mg/m(2) day) oil days -8 to -6, and thiotepa (300 mg/m(2)/day) and etoposide (250 mg/m(2)/day) on days -5 to -3, followed by autologous stern cell rescue (ASCR) oil day 0. Twenty-five patients, aged 7.6-44.7 years (median 13.8 years) at ASCR, were treated. Three (12%,) died of treatment-related toxicities within 30 days of ASCR, clue to multi-organ system failure (n = 2) and aspergillus infection with veno-occlusive disease (n = 1). Tumor recurred in 16 at a median of 8.5 months (range 2.3-58.5 months). Six are event-free survivors at a median of 151.2 months post-ASCR (range 127.2-201.6 months). The Kaplan-Meier estimate of median overall survival is 26.8 months (95% Cl: 11.9-51.1 months) and of event-free survival (EFS) and overall survival are both 24% (95% Cl: 9.8%-41.7%) at 10 years post-ASCR. M-0 (vs M-1 +) recurrence prior to protocol, lack of tissue confirmation of relapse, and initial therapy of radiation therapy (RT) alone (vs RT + chemotherapy) were not significantly associated with better EFS (P = .33, .34, and .27, respectively). Trends toward better EFS were noted in patient, (n = 5) who received additional RT as part of their retrieval therapy (P = .07) and whose recurrent disease was demonstrated to be sensitive to reinduction chemotherapy (P = .09). This retrieval strategy provides long-term EFS for some patients with previously irradiated recurrent medulloblastoma. The use of ad additional RT may be associated with better outcome.