Altshuler C, Haley K, Dhall G, Vasquez L, Gardner S L, Stanek J, Finlay J L
Department of Pediatrics, Keck School of Medicine, University of Southern California, Los Angeles, CA, USA.
Children's Center for Cancer and Blood Diseases, Children's Hospital Los Angeles, Los Angeles, CA, USA.
Bone Marrow Transplant. 2016 Jul;51(7):945-8. doi: 10.1038/bmt.2016.45. Epub 2016 Mar 7.
Since 1991, three sequential prospective clinical trials have been conducted by the 'Head Start' (HS) Consortium in which young children with newly-diagnosed malignant central nervous system (CNS) tumors were treated with induction chemotherapy followed by single-cycle marrow-ablative chemotherapy and autologous hematopoietic rescue as a means of improving disease cure rate and quality of survival through avoidance (<6 years old at diagnosis) or reduction (6-10 years old) of brain irradiation. Bone Marrow (HS I) or filgrastim-mobilized peripheral hematopoietic cells (HS II and III) were obtained following recovery from the first and/or second induction cycles. Radiotherapy was administered following all chemotherapy only for patients with residual tumor following completion of induction or with age greater than 6 years at diagnosis. Two hundred and twenty-six children were enrolled on three consecutive HS trials with primary malignant CNS tumors and underwent marrow-ablative chemotherapy. The 100-day treatment-related mortality (TRM) steadily declined as did grade IV transplant-related oropharyngeal mucositis. Factors most likely associated with the decrease in TRM and morbidity are increasing experience with the marrow-ablative chemotherapy regimen combined with improved leukapheresis and post-reinfusion supportive care techniques, contributing toward improved overall survival.
自1991年以来，“启智计划”（HS）联盟开展了三项连续的前瞻性临床试验，其中新诊断为恶性中枢神经系统（CNS）肿瘤的幼儿先接受诱导化疗，随后进行单周期骨髓清除化疗和自体造血挽救，以此作为通过避免（诊断时年龄<6岁）或减少（6 - 10岁）脑部放疗来提高疾病治愈率和生存质量的一种手段。在从第一个和/或第二个诱导周期恢复后获取骨髓（HS I）或非格司亭动员的外周造血细胞（HS II和III）。仅对诱导结束后有残留肿瘤或诊断时年龄大于6岁的患者，在所有化疗后给予放疗。226名患有原发性恶性CNS肿瘤的儿童连续参加了三项HS试验并接受了骨髓清除化疗。100天治疗相关死亡率（TRM）稳步下降，IV级移植相关口咽粘膜炎也有所下降。最有可能与TRM和发病率降低相关的因素是骨髓清除化疗方案经验的增加，以及白细胞分离术和再输注后支持治疗技术的改进，这有助于提高总体生存率。
