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一项多机构立体定向体部放疗治疗肉瘤儿童和青年患者骨转移的 2 期临床试验。

A multi-institutional phase 2 trial of stereotactic body radiotherapy in the treatment of bone metastases in pediatric and young adult patients with sarcoma.

机构信息

Department of Radiation Oncology and Molecular Radiation Sciences, Johns Hopkins University School of Medicine, Baltimore, Maryland.

Department of Radiation Oncology, St. Jude Children's Research Hospital, Memphis, Tennessee.

出版信息

Cancer. 2021 Mar 1;127(5):739-747. doi: 10.1002/cncr.33306. Epub 2020 Nov 10.

DOI:10.1002/cncr.33306
PMID:33170960
Abstract

BACKGROUND

Metastasectomy is standard of care for pediatric patients with metastatic sarcoma with limited disease. For patients with unresectable disease, stereotactic body radiotherapy (SBRT) may serve as an alternative. Herein, the authors report the results of a prospective, multi-institutional phase 2 trial of SBRT in children and young adults with metastatic sarcoma.

METHODS

Patients aged >3 years and ≤40 years with unresected, osseous metastatic nonrhabdomyosarcoma sarcomas of soft tissue and bone were eligible. Patients received SBRT to a dose of 40 Gray (Gy) in 5 fractions. Local control (LC), progression-free survival (PFS), and overall survival (OS) were calculated using the Kaplan-Meier method.

RESULTS

Fourteen patients with a median age of 17 years (range, 4-25 years) were treated to 37 distinct metastatic lesions. With a median follow-up of 6.8 months (30.5 months in surviving patients), the Kaplan-Meier patient-specific and lesion-specific LC rates at 6 months were 89% and 95%, respectively. The median PFS was 6 months and the median OS was 24 months. In a post hoc analysis, PFS (median, 9.3 months vs 3.7 months; log-rank P = .03) and OS (median not reached vs 12.7 months; log-rank P = .02) were improved when all known sites of metastatic disease were consolidated with SBRT compared with partial consolidation. SBRT was well tolerated, with 2 patients experiencing grade 3 toxicities.

CONCLUSIONS

SBRT achieved high rates of LC in pediatric patients with inoperable metastatic nonrhabdomyosarcoma sarcomas of soft tissue and bone. These results suggest that the ability to achieve total consolidation of metastatic disease with SBRT is associated with improved PFS and OS.

摘要

背景

对于转移性肉瘤且疾病有限的儿科患者,转移瘤切除术是标准治疗方法。对于无法切除的疾病患者,立体定向体放射治疗(SBRT)可能是一种替代方法。在此,作者报告了一项针对患有转移性肉瘤的儿童和年轻成人的 SBRT 的前瞻性、多机构 2 期试验结果。

方法

符合条件的患者为年龄大于 3 岁且≤40 岁、患有无法切除的骨转移非横纹肌肉瘤肉瘤的软组织和骨。患者接受 40 格雷(Gy)剂量的 SBRT,共 5 个剂量。使用 Kaplan-Meier 方法计算局部控制(LC)、无进展生存期(PFS)和总生存期(OS)。

结果

14 名中位年龄为 17 岁(范围 4-25 岁)的患者接受了 37 个不同转移性病变的治疗。在中位随访 6.8 个月(存活患者的中位随访时间为 30.5 个月)后,患者特异性和病变特异性的 6 个月 Kaplan-Meier LC 率分别为 89%和 95%。中位 PFS 为 6 个月,中位 OS 为 24 个月。在事后分析中,与部分巩固治疗相比,所有已知的转移性疾病部位均用 SBRT 巩固治疗时,PFS(中位数 9.3 个月比 3.7 个月;对数秩 P =.03)和 OS(中位数未达到比 12.7 个月;对数秩 P =.02)均得到改善。SBRT 耐受性良好,有 2 名患者发生 3 级毒性。

结论

SBRT 可实现无法切除的软组织和骨非横纹肌肉瘤肉瘤的儿童患者高 LC 率。这些结果表明,SBRT 实现转移性疾病完全巩固的能力与改善 PFS 和 OS 相关。

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