Ronsley Rebecca, Bradford Miranda C, Crotty Erin E, Vitanza Nicholas A, Runco Daniel V, Stevens Jeffrey, Hoeppner Corinne, Holtzclaw Susan L, Wein Amy R, Lee Amy, Cole Bonnie L, Ermoian Ralph, Leary Sarah E S
Fred Hutch Cancer Center, Seattle, Washington, USA.
Ben Towne Center for Childhood Cancer Research, Seattle Children's Research Institute, Seattle, Washington, USA.
Neurooncol Pract. 2024 Nov 18;12(3):489-497. doi: 10.1093/nop/npae114. eCollection 2025 Jun.
Effective therapy for medulloblastoma at the time of relapse is limited. The objective of this study is to review outcomes from the Seattle Children's Hospital (SCH) institutional standard therapy for relapsed medulloblastoma, modified from the published ACNS0821 regimen.
Retrospective review of patients treated for relapsed medulloblastoma from 2012-2024 treated with modified ACNS0821 therapy, including combination bevacizumab, irinotecan, and temozolomide, referred to as "." Each cycle includes oral temozolomide (200 mg/m/day) for the first 5 days, intravenous (IV) bevacizumab (10 mg/kg/dose), and IV irinotecan (125 mg/m/dose or 340 mg/m) on days 1 and 15 of each cycle. Patient medical history, prior treatment, therapy toxicity, response, and outcome were collected. The analysis included Kaplan-Meier estimates of 3-year overall survival (OS) and 3-year progression-free survival.
Fifteen patients were treated with TIB for relapsed medulloblastoma at SCH (median age 5.81 (0.21-23.6) years, 60% male). Twelve patients completed planned therapy. Therapy was discontinued for toxicity ( = 1) and family preference ( = 1). The most common toxicities were thrombocytopenia ( = 7), neutropenia ( = 4), nausea ( = 5), vomiting ( = 5), and diarrhea ( = 3). Five patients required dose modification of one agent for toxicity. Median follow-up from TIB therapy start was 1.61 (0.47-7.66) years. Three-year OS was 48% (95% CI: 18%-74%) and 3-year event-free survival was 16% (95% CI: 1%-49%).
TIB was well-tolerated in pediatric patients with relapsed medulloblastoma, and outcomes were similar to those published in clinical trials. TIB therapy should be considered for patients with relapsed medulloblastoma, especially patients with limited access to care due to travel barriers.
髓母细胞瘤复发时的有效治疗方法有限。本研究的目的是回顾西雅图儿童医院（SCH）对复发髓母细胞瘤的机构标准治疗方案的疗效，该方案是在已发表的ACNS0821方案基础上修改而来。
回顾性分析2012年至2024年接受改良ACNS0821治疗的复发髓母细胞瘤患者，该治疗包括联合使用贝伐单抗、伊立替康和替莫唑胺，简称为“TIB”。每个周期包括前5天口服替莫唑胺（200mg/m²/天），每个周期的第1天和第15天静脉注射（IV）贝伐单抗（10mg/kg/剂量）和IV伊立替康（125mg/m²/剂量或340mg/m²）。收集患者的病史、既往治疗、治疗毒性、反应和结局。分析包括3年总生存期（OS）和3年无进展生存期的Kaplan-Meier估计值。
15例复发髓母细胞瘤患者在SCH接受了TIB治疗（中位年龄5.81（0.21 - 23.6）岁，60%为男性）。12例患者完成了计划治疗。治疗因毒性（n = 1）和家属意愿（n = 1）而中断。最常见的毒性反应为血小板减少（n = 7）、中性粒细胞减少（n = 4）、恶心（n = 5）、呕吐（n = 5）和腹泻（n = 3）。5例患者因毒性反应需要对一种药物进行剂量调整。从TIB治疗开始的中位随访时间为1.61（0.47 - 7.66）年。3年总生存率为48%（95%CI：18% - 74%），3年无事件生存率为16%（95%CI：1% - 49%）。
TIB在复发髓母细胞瘤的儿科患者中耐受性良好，其疗效与临床试验发表的结果相似。对于复发髓母细胞瘤患者，尤其是因交通障碍而获得治疗机会有限的患者，应考虑使用TIB治疗。
