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首页> 外文期刊>Pediatric blood & cancer >Double‐conditioning regimen consisting of high‐dose thiotepa and melphalan with autologous stem cell rescue for high‐risk pediatric solid tumors: A second report
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Double‐conditioning regimen consisting of high‐dose thiotepa and melphalan with autologous stem cell rescue for high‐risk pediatric solid tumors: A second report

机译:由高剂量Thiotepa和Melphalan组成的双调方案,具有自体干细胞的高危小儿实体瘤:第二次报告

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Abstract Background Pediatric patients with high‐risk, relapsed, or refractory solid tumors have a poor prognosis. We have previously reported a dose‐finding experience of high‐dose chemotherapy consisting of thiotepa and melphalan (“double‐conditioning regimen”). Using doses derived from that study, we have treated patients since 2005. We now report a retrospective review of patients treated by this fixed dose. Procedure We reviewed 50 patients (median 4?years; range 0–15?years) with high‐risk or relapsed/refractory solid tumors treated by this dose‐fixed, double‐conditioning regimen from April 2005 to May 2014. Doses were thiotepa 800?mg/m 2 and melphalan 280?mg/m 2 for children ≥2?years of age, and 32?mg/kg and 6?mg/kg, respectively, for children 2?years of age. Further, doses were reduced according to creatinine clearance with poor renal function. Results Nonhematological toxicity was mainly gastrointestinal—grade 3 mucositis ( n? =?41) and grade 3–4 diarrhea ( n? =?10). Neurological, renal, and endothelial cell toxicity and sinusoidal obstruction syndrome were not observed. There were two toxic deaths (interstitial viral pneumonia). This regimen demonstrated antitumor activity against several types of tumors. Although the frequency of gastrointestinal toxicity was high, other severe toxicity was not observed. Conclusions Our double‐conditioning regimen was very well tolerated and demonstrated antitumor activity. We are moving forward with multi‐institutional trials now.
机译:摘要背景患者具有高风险,复发或难治性实体肿瘤的预后差。我们此前报道了由Thiotepa和Melphalan(“双调方案”)组成的高剂量化疗的剂量发现经验。使用来自该研究的剂量,我们自2005年以来已经过治疗患者。我们现在举报了对由该固定剂量治疗的患者的回顾性审查。手术程序我们审查了50名患者(中位数4岁; 0-15岁的范围),由2005年4月至2014年5月从2005年4月到2014年5月治疗的高风险或复发/难治性固体肿瘤。剂量是Thiotepa 800 ?Mg / m 2和Melphalan 280?mg / m 2,儿童≥2岁,分别为32毫克/ kg和6?mg / kg,儿童&lt 2岁。此外,根据肾功能差的肌酐清除,减少剂量。结果鼻咽学毒性主要是胃肠道3型粘液炎(N?= 41)和3-4级腹泻(N?=?10)。未观察到神经系统,肾病和内皮细胞毒性和正弦梗阻综合征。有两种有毒死亡(间质病毒肺炎)。该方案展示了针对几种类型的肿瘤的抗肿瘤活性。虽然胃肠道毒性的频率高,但未观察到其他严重的毒性。结论我们的双调方案非常耐受性和展示抗肿瘤活性。我们现在正在向前迈进多机构试验。

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