Saarinen U M, Hovi L, Mäkipernaa A, Riikonen P
Children's Hospital, University of Helsinki, Finland.
Bone Marrow Transplant. 1991 Nov;8(5):369-76.
High-dose thiotepa was given as a single agent at a total dose of 1125 mg/m2 with autologous bone marrow rescue to nine patients with recurrent/refractory/poor risk pediatric malignancies (primitive neuroepithelial tumor (PNET), two; neuroblastoma, one; Wilms' tumor, one; osteosarcoma, one; Ewing's sarcoma one, Hodgkin's disease one, high-grade glioma, two). The response rate in these heavily pretreated patients was 71% (five out of seven evaluable patients) including two complete responses (Wilms', glioma), three partial responses (osteosarcoma, Ewing's sarcoma, Hodgkin's disease), and two with stable disease (PNET, glioma). The median duration of response was 2.5 months. The extramedullary toxicity was acceptable with symptoms mainly of skin and gastrointestinal tract. The data indicate that high-dose thiotepa is effective in several types of recurrent pediatric solid tumors, and merits further evaluation in combination regimens.
对9例复发/难治性/高危儿童恶性肿瘤（原始神经上皮肿瘤（PNET）2例；神经母细胞瘤1例；肾母细胞瘤1例；骨肉瘤1例；尤因肉瘤1例；霍奇金病1例；高级别胶质瘤2例）患者，给予大剂量噻替派单药治疗，总剂量为1125mg/m²，并进行自体骨髓解救。这些经过大量预处理的患者的缓解率为71%（7例可评估患者中的5例），包括2例完全缓解（肾母细胞瘤、胶质瘤）、3例部分缓解（骨肉瘤、尤因肉瘤、霍奇金病）和2例病情稳定（PNET、胶质瘤）。中位缓解持续时间为2.5个月。髓外毒性可以接受，主要症状为皮肤和胃肠道症状。数据表明，大剂量噻替派对几种类型的复发儿童实体瘤有效，值得在联合方案中进一步评估。
