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列线图的建立与验证:预测 T1 期黑素瘤患者局部、区域和远处复发风险

Development and Validation of Nomograms to Predict Local, Regional, and Distant Recurrence in Patients With Thin (T1) Melanomas.

机构信息

Melanoma Institute Australia, The University of Sydney, Sydney, New South Wales, Australia.

Department of Dermatology, University Medical Center Utrecht, Utrecht University, Utrecht, the Netherlands.

出版信息

J Clin Oncol. 2021 Apr 10;39(11):1243-1252. doi: 10.1200/JCO.20.02446. Epub 2021 Feb 18.

Abstract

PURPOSE

Although the prognosis of patients with thin primary cutaneous melanomas (T1, ≤ 1.0 mm) is generally excellent, some develop recurrence. We sought to develop and validate a model predicting recurrences in patients with thin melanomas.

METHODS

A Dutch population-based cohort (n = 25,930, development set) and a cohort from an Australian melanoma treatment center (n = 2,968, validation set) were analyzed (median follow-up 6.7 and 12.0 years, respectively). Multivariable Cox models were generated for local, regional, and distant recurrence-free survival (RFS). Discrimination was assessed using Harrell's C-statistic for each outcome. Each nomogram performance was evaluated using calibration plots defining low-risk and high-risk groups as the lowest and top 5% of the nomogram risk score, respectively. The nomograms' C-statistics were compared with those of a model including the current American Joint Committee on Cancer staging parameters (T-stage and sentinel node status).

RESULTS

Local, regional, and distant recurrences were found in 209 (0.8%), 503 (1.9%), and 203 (0.8%) Dutch patients, respectively, and 23 (0.8%), 61 (2.1%), and 75 (2.5%) Australian patients, respectively. C-statistics of 0.79 (95% CI, 0.75 to 0.82) for local RFS, 0.77 (95% CI, 0.75 to 0.78) for regional RFS, and 0.80 (95% CI, 0.77 to 0.83) for distant RFS were obtained for the development model. External validation showed C-statistics of 0.80 (95% CI, 0.69 to 0.90), 0.76 (95% CI, 0.70 to 0.82), and 0.74 (95% CI, 0.69 to 0.80), respectively. Calibration plots showed a good match between predicted and observed rates. Using the nomogram, the C-statistic was increased by 9%-12% for the development cohort and by 11%-15% for the validation cohort, compared with a model including only T-stage and sentinel node status.

CONCLUSION

Most patients with thin melanomas have an excellent prognosis, but some develop recurrence. The presented nomograms can accurately identify a subgroup at high risk. An online calculator is available at www.melanomarisk.org.au.

摘要

目的

尽管 T1 期(厚度≤1.0mm)原发性皮肤黑色素瘤患者的预后通常较好,但仍有部分患者会出现复发。我们旨在建立并验证预测 T1 期黑色素瘤患者复发的模型。

方法

分析了荷兰人群队列(n=25930,开发集)和澳大利亚黑色素瘤治疗中心队列(n=2968,验证集)(分别中位随访 6.7 年和 12.0 年)。使用多变量 Cox 模型对局部、区域和远处无复发生存率(RFS)进行了分析。使用 Harrell 的 C 统计量评估每种结局的区分度。使用校准图将风险评分最低的和前 5%的患者分别定义为低风险组和高风险组,评估每个列线图的性能。比较了列线图的 C 统计量与包括当前美国癌症联合委员会分期参数(T 分期和前哨淋巴结状态)的模型。

结果

荷兰队列分别有 209 例(0.8%)、503 例(1.9%)和 203 例(0.8%)患者发生局部、区域和远处复发,澳大利亚队列分别有 23 例(0.8%)、61 例(2.1%)和 75 例(2.5%)患者发生上述情况。开发模型中局部 RFS 的 C 统计量为 0.79(95%CI,0.75 至 0.82),区域 RFS 为 0.77(95%CI,0.75 至 0.78),远处 RFS 为 0.80(95%CI,0.77 至 0.83)。外部验证显示,C 统计量分别为 0.80(95%CI,0.69 至 0.90)、0.76(95%CI,0.70 至 0.82)和 0.74(95%CI,0.69 至 0.80)。校准图显示预测值与观察值之间具有良好的一致性。与仅包含 T 分期和前哨淋巴结状态的模型相比,使用列线图可使开发队列的 C 统计量增加 9%-12%,使验证队列的 C 统计量增加 11%-15%。

结论

大多数 T1 期黑色素瘤患者的预后较好,但仍有部分患者会出现复发。本研究提供的列线图可以准确识别高风险亚组。一个在线计算器可在 www.melanomarisk.org.au 上获取。

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