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颅脊髓照射联合每日顺铂治疗高危中枢神经系统肿瘤的儿童和成人的可行性及疗效

Feasibility and Outcomes of Craniospinal Irradiation and Concurrent Daily Carboplatin in Children and Adults With High-risk Central Nervous System Tumours.

作者信息

Singer R, Lorimer C, Welsh L, Carceller F, Vaidya S, Marshall L V, Stone J, Evans R, O'Leary B, Zacharoulis S, Saran F, Mandeville H

机构信息

Weston Park Cancer Centre, Sheffield Teaching Hospitals NHS Foundation Trust, Sheffield, UK.

The Royal Marsden NHS Foundation Trust, London, UK; University Hospitals Sussex NHS Foundation Trust.

出版信息

Clin Oncol (R Coll Radiol). 2025 Aug;44:103866. doi: 10.1016/j.clon.2025.103866. Epub 2025 May 10.

Abstract

AIMS

High-risk medulloblastoma, pineoblastoma, and other embryonal central nervous system (CNS) tumours are associated with a poor prognosis. Children's Oncology Group trial CCG-99701 demonstrated concomitant daily carboplatin during radiotherapy, followed by adjuvant chemotherapy, to be a promising strategy for treating these malignancies in children. In this case series, we describe treatment feasibility, toxicities, and outcomes for children and adults from a heterogeneous real-world population, treated with this regimen for high-risk CNS tumours.

MATERIALS AND METHODS

Data from clinical records were collected on surgery, radiotherapy, chemotherapy, acute toxicities, supportive treatment, and survival for all patients treated with the regimen between January 2012 and June 2018.

RESULTS

Twenty-seven patients (13 children and 14 adults) received chemoradiotherapy. Seven patients (26%) were rested for one or more radiotherapy fractions due to medical or anaesthetic issues, and 4 patients (15%) had an extended overall treatment time. Grade 4 haematological toxicity was common, but no grade 4 non-haematological toxicities were observed. Twenty-two patients (81%) required haematological support. Two patients discontinued adjuvant chemotherapy early due to toxicity. Two-year overall survival (OS) in paediatric and adult patients was 85% and 46%, respectively, and 5-year OS was 69% and 38%, respectively. Progression-free survival (PFS) was significantly longer in paediatric patients than in adults (median PFS not reached and 0.9 years, respectively; hazard ratio: 0.26; 95% confidence interval [CI]: 0.09 to 0.75; P = 0.01). There was no significant difference in OS between paediatric and adult patients (median OS not reached and 1.3 years, respectively; hazard ratio: 0.43; 95% CI: 0.14 to 1.29; P = 0.12).

CONCLUSION

The treatment regimen was feasible for paediatric and adult patients but was associated with significant toxicity. The survival outcomes in paediatric patients were favourable, consistent with published data. Due to the close clinical supervision required, this regimen should ideally be delivered in centres with experience in managing paediatric or teenage and young adult high-risk CNS tumours.

摘要

目的

高危髓母细胞瘤、松果体母细胞瘤及其他胚胎性中枢神经系统(CNS)肿瘤预后较差。儿童肿瘤学组试验CCG - 99701表明,放疗期间每日同步使用卡铂,随后进行辅助化疗,是治疗儿童这些恶性肿瘤的一种有前景的策略。在本病例系列中,我们描述了来自异质性现实世界人群的儿童和成人采用该方案治疗高危CNS肿瘤的治疗可行性、毒性及预后。

材料与方法

收集了2012年1月至2018年6月期间所有接受该方案治疗患者的临床记录数据,包括手术、放疗、化疗、急性毒性、支持治疗及生存情况。

结果

27例患者(13例儿童和14例成人)接受了放化疗。7例患者(26%)因医疗或麻醉问题放疗中断一个或多个分次,4例患者(15%)总治疗时间延长。4级血液学毒性常见,但未观察到4级非血液学毒性。22例患者(81%)需要血液学支持。2例患者因毒性提前终止辅助化疗。儿童和成人患者的2年总生存率(OS)分别为85%和46%,5年OS分别为69%和38%。儿童患者的无进展生存期(PFS)显著长于成人(中位PFS未达到和0.9年,分别;风险比:0.26;95%置信区间[CI]:0.09至0.75;P = 0.01)。儿童和成人患者的OS无显著差异(中位OS未达到和1.3年,分别;风险比:0.43;95% CI:0.14至1.29;P = 0.12)。

结论

该治疗方案对儿童和成人患者可行,但有显著毒性。儿童患者的生存结果良好,与已发表数据一致。由于需要密切临床监测,该方案理想情况下应在有治疗儿童或青少年及年轻成人高危CNS肿瘤经验的中心实施。

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