Mayo Clinic Neuro-informatics Laboratory, Mayo Clinic, Rochester, Minnesota.
Department of Neurologic Surgery, Mayo Clinic, Rochester, Minnesota.
JAMA Netw Open. 2022 Jan 4;5(1):e2141927. doi: 10.1001/jamanetworkopen.2021.41927.
Maximal resection is the preferred management for sacral chordomas but can be associated with unacceptable morbidity. Outcomes with radiotherapy are poor. Carbon ion radiotherapy (CIRT) is being explored as an alternative when surgery is not preferred.
To compare oncologic outcomes and treatment-related toxicity of CIRT and en bloc resection for sacral chordoma.
DESIGN, SETTING, AND PARTICIPANTS: Univariable logistic regression was performed to evaluate the association between treatment type and oncologic and toxicity outcomes in this retrospective cohort study. Nearest-neighbor propensity score matching was used to match the CIRT cohort with the en bloc resection cohort and 10 National Cancer Database (NCDB) cohorts separately, with the objective of obtaining more homogeneous cohorts when comparing treatments. Patient- and tumor-related characteristics from 2 institutional cohorts were collected for patients diagnosed with sacral chordomas between April 1, 1994, and July 31, 2017. The NCDB was queried for data on patients with sacral chordoma from January 1, 2004, to December 31, 2016, as a comparator in overall survival (OS) analyses. Data analysis was conducted from February 24, 2020, to January 16, 2021.
En bloc resection, incomplete resection, photon radiotherapy, proton radiotherapy, and CIRT.
Overall survival was estimated using the Kaplan-Meier method and compared using the Cox proportional hazards model. Peripheral motor nerve toxic effects were scored using Common Terminology Criteria for Adverse Events, version 4.03.
A total of 911 patients were included in the study (NCDB: n = 669; median age, 64 [IQR, 52-74] years; 410 [61.3%] men; CIRT: n = 188; median age, 66 [IQR, 58-71] years; 128 [68.1%] men; en bloc surgical resection: n = 54; median age, 53.5 [IQR 49-64] years, 36 [66.7%] men). Comparison of the propensity score-matched institutional en bloc resection and CIRT cohorts revealed no statistically significant difference in OS (CIRT: median OS, 68.1 [95% CI, 44.0-102.6] months; en bloc resection: median OS, 58.6 [95% CI, 25.6-123.5] months; P = .57; hazard ratio, 0.71 [95% CI, 0.25-2.06]; P = .53). The CIRT cohort experienced lower rates of peripheral motor neuropathy (odds ratio, 0.13 [95% CI, 0.04-0.40]; P < .001). On comparison of the propensity score-matched NCDB cohorts with the CIRT cohort, significantly higher OS was found for CIRT compared with margin-positive surgery without adjuvant radiotherapy (CIRT: median OS, 64.7 [95% CI, 57.8-69.7] months; margin-positive surgery without adjuvant radiotherapy: median OS, 60.6 [95% CI, 44.2-69.7] months, P = .03) and primary radiotherapy alone (CIRT: median OS, 64.9 [95% CI 57.0-70.5] months; primary radiotherapy alone: 31.8 [95% CI, 27.9-40.6] months; P < .001).
These findings suggest that CIRT can be used as treatment for older patients with high performance status and sacral chordoma in whom surgery is not preferred. CIRT might provide additional benefit for patients who undergo margin-positive resection or who are candidates for primary photon radiotherapy.
重要性：最大程度的切除是治疗骶骨脊索瘤的首选方法，但可能会带来无法接受的发病率。放疗的效果很差。当手术不被首选时，碳离子放疗（CIRT）正被探索作为一种替代方法。
目的：比较 CIRT 和整块切除术治疗骶骨脊索瘤的肿瘤学结果和治疗相关毒性。
设计、设置和参与者：在这项回顾性队列研究中，采用单变量逻辑回归来评估治疗类型与肿瘤学和毒性结果之间的关联。为了在比较治疗方法时获得更同质的队列，使用最近邻倾向评分匹配将 CIRT 队列与整块切除术队列和 10 个国家癌症数据库（NCDB）队列分别进行匹配，每组匹配 10 个队列。从 1994 年 4 月 1 日至 2017 年 7 月 31 日期间，收集了 2 个机构队列中诊断为骶骨脊索瘤患者的患者和肿瘤相关特征。从 2004 年 1 月 1 日至 2016 年 12 月 31 日，NCDB 检索了骶骨脊索瘤患者的数据，作为总生存（OS）分析的比较。数据分析于 2020 年 2 月 24 日至 2021 年 1 月 16 日进行。
暴露：整块切除术、不完全切除术、光子放疗、质子放疗和 CIRT。
主要结果和测量：使用 Kaplan-Meier 方法估计总生存率，并使用 Cox 比例风险模型进行比较。使用常见不良事件术语标准，版本 4.03 对周围运动神经毒性作用进行评分。
结果：共纳入 911 名患者（NCDB：n=669；中位年龄 64 [IQR，52-74] 岁；410 [61.3%] 名男性；CIRT：n=188；中位年龄 66 [IQR，58-71] 岁；128 [68.1%] 名男性；整块切除术：n=54；中位年龄 53.5 [IQR 49-64] 岁，36 [66.7%] 名男性）。对机构整块切除术和 CIRT 队列的倾向评分匹配进行比较，发现 OS 无统计学显著差异（CIRT：中位 OS 68.1 [95%CI，44.0-102.6] 个月；整块切除术：中位 OS 58.6 [95%CI，25.6-123.5] 个月；P=0.57；危险比 0.71 [95%CI，0.25-2.06]；P=0.53）。CIRT 队列周围运动神经病的发生率较低（优势比，0.13 [95%CI，0.04-0.40]；P<0.001）。与 CIRT 队列的 NCDB 队列的倾向评分匹配比较，与边缘阳性手术且无辅助放疗相比，CIRT 显著提高了 OS（CIRT：中位 OS 64.7 [95%CI，57.8-69.7] 个月；边缘阳性手术且无辅助放疗：中位 OS 60.6 [95%CI，44.2-69.7] 个月，P=0.03）和单纯原发放疗（CIRT：中位 OS 64.9 [95%CI 57.0-70.5] 个月；单纯原发放疗：31.8 [95%CI，27.9-40.6] 个月；P<0.001）。
结论和相关性：这些发现表明，对于不首选手术且有较高表现状态的老年患者，CIRT 可作为治疗骶骨脊索瘤的方法。对于边缘阳性切除或适合原发性光子放疗的患者，CIRT 可能会提供额外的益处。
