RM Gorbacheva Research Institute, Pavlov University, St. Petersburg, Russian Federation.
V.F. Voyno-Yasenetsky Scientific and Practical Center of Specialized Medical Care for Children, Moscow, Russian Federation.
Bone Marrow Transplant. 2022 Apr;57(4):607-612. doi: 10.1038/s41409-022-01593-x. Epub 2022 Feb 3.
Withdrawal of radiotherapy in patients with brain tumors under four years decreases chance for cure. AutoHSCT in a series of pilot studies demonstrated a potential to improve outcomes in these patients. The study included 50 patients with median age of 39 months (7-53). Medulloblastoma (n = 28, 56%), ETMR (n = 9, 18%) and other histological types (n = 13, 26%) were most commonly diagnosed. Forty two patients (84%) received tandem autoHSCT by HIT-MED protocol, and single autoHSCT was performed in eight children (16%). Adjuvant radiotherapy was administered in 25 (50%) children and treatment of relapse included radiotherapy in 6 (12%). Median follow-up was 39.6 months (6-121). Long-term CIR was 37%, and TRM - 6%. Five-year OS was 71% in medulloblastoma, 37% in ETMR and in other tumors - 51% (p = 0.07). Irradiation-free OS at 5 years for children with medulloblastoma was 24%. For the whole cohort of CNS tumors, independently of histology, OS and PFS at five years were 60% and 46%, respectively Young children with medulloblastoma, following tandem autoHSCT, demonstrate OS comparable to older children. Patients with other histological types demonstrate suboptimal long-term survival rates after autoHSCT and one should assess whether these patients benefit from autoHSCT.
在四岁以下的脑瘤患者中停止放疗会降低治愈机会。在一系列试点研究中，自体造血干细胞移植（AutoHSCT）显示出改善这些患者预后的潜力。该研究纳入了 50 名中位年龄为 39 个月（7-53 岁）的患者。最常见的诊断是髓母细胞瘤（n=28，56%）、胚胎性肿瘤（ETMR，n=9，18%）和其他组织学类型（n=13，26%）。42 名患者（84%）接受了 HIT-MED 方案的串联自体 HSCT，8 名儿童（16%）接受了单次自体 HSCT。25 名儿童（50%）接受了辅助放疗，6 名儿童（12%）接受了放疗治疗复发。中位随访时间为 39.6 个月（6-121 个月）。长期 CIR 为 37%，TRM 为 6%。5 年 OS 分别为髓母细胞瘤 71%、ETMR 37%和其他肿瘤 51%（p=0.07）。髓母细胞瘤患儿无放疗 5 年 OS 为 24%。对于整个 CNS 肿瘤队列，无论组织学如何，5 年 OS 和 PFS 分别为 60%和 46%。接受串联自体 HSCT 的小龄髓母细胞瘤患儿的 OS 与大龄患儿相当。接受自体 HSCT 的其他组织学类型患儿的长期生存结果不佳，应评估这些患儿是否从自体 HSCT 中获益。
