Sections of Pediatric Hematology-Oncology-BMT and Neuro-oncology, Department of Pediatrics, HIMA San Pablo Oncology Hospital, Caguas, Puerto Rico.
The Neuro-oncology Program, Children's Center for Cancer and Blood Diseases, Children's Hospital Los Angeles and Keck School of Medicine, University of Southern California, Los Angeles, CA, USA.
Bone Marrow Transplant. 2017 Nov;52(11):1543-1548. doi: 10.1038/bmt.2017.166. Epub 2017 Aug 7.
To improve survival in young children with malignant brain tumors, irradiation-avoiding or -minimizing marrow-ablative chemotherapy (HDCx) with autologous hematopoietic cell transplantation (AuHCT) has been investigated. We evaluated the outcome of 44 children with malignant brain tumors treated with HDCx and tandem AuHCT at Children's Hospital Los Angeles between June 1999 and July 2012. Forty-four children with malignant brain tumors were studied. Twenty-one had medulloblastoma/primitive neuro-ectodermal tumor, eight atypical teratoid/rhabdoid tumor (ATRT), five high-grade glioma, four malignant germ cell tumor, three ependymoma and three choroid plexus carcinoma. Twenty-nine patients received three tandem transplants and 15 received two tandem transplants, respectively. The 5-year PFS and overall survivals (OS) for all patients were 46.3±8.2% and 51.7±8.5%, respectively. The PFS and OS for 27 newly diagnosed patients were 68.9±9.9% and 73.5±9.3%, respectively, compared with 17 transplanted at relapse 11.8±9.8% (P<0.001) and 15.1±12.3% (P=0.0231), respectively. The 5-year PFS and OS in 13 previously unirradiated patients were 74±13% and 74±13% versus 33.2±9.8% and 40.2±10.6% in 31 irradiated patients (P=0.11 and P=0.239), respectively. One patient died of transplant-related toxicity. HDCx with tandem AuHCT is feasible and safe in children with malignant brain tumors with encouraging irradiation-free survival in newly diagnosed children.
为了提高患有恶性脑肿瘤的幼儿的生存率，已经研究了避免或减少照射的骨髓清除性化疗（HDCx）联合自体造血细胞移植（AuHCT）。我们评估了 1999 年 6 月至 2012 年 7 月期间在洛杉矶儿童医院接受 HDCx 和串联 AuHCT 治疗的 44 例恶性脑肿瘤患儿的结果。
研究了 44 例患有恶性脑肿瘤的儿童。21 例为髓母细胞瘤/原始神经外胚层肿瘤，8 例为非典型畸胎瘤/横纹肌样瘤（ATRT），5 例为高级别胶质瘤，4 例为恶性生殖细胞瘤，3 例为室管膜瘤和 3 例脉络丛癌。29 例患者接受了 3 次串联移植，15 例患者接受了 2 次串联移植。所有患者的 5 年 PFS 和总生存率（OS）分别为 46.3±8.2%和 51.7±8.5%。27 例初诊患者的 PFS 和 OS 分别为 68.9±9.9%和 73.5±9.3%，而 17 例复发患者的 PFS 和 OS 分别为 11.8±9.8%（P<0.001）和 15.1±12.3%（P=0.0231）。13 例未接受放疗的患者的 5 年 PFS 和 OS 分别为 74±13%和 74±13%，而 31 例接受放疗的患者分别为 33.2±9.8%和 40.2±10.6%（P=0.11 和 P=0.239）。1 例患者死于移植相关毒性。HDCx 联合串联 AuHCT 对患有恶性脑肿瘤的儿童是可行且安全的，新诊断的儿童可实现无放疗的生存。
