Lee Chee Khoon, Goldstein David, Gibbs Emma, Joensuu Heikki, Zalcberg John, Verweij Jaap, Casali Paolo G, Maki Robert G, Cioffi Angela, Mcarthur Grant, Lord Sarah J, Yip Desmond, Kanjanapan Yada, Rutkowski Piotr
National Health and Medical Research Council Clinical Trials Centre, The University of Sydney, Sydney, Australia.
Department of Medical Oncology, Prince of Wales Hospital, Sydney, Australia; Prince of Wales Clinical School, University of New South Wales, Sydney, Australia.
Eur J Cancer. 2015 May;51(7):852-60. doi: 10.1016/j.ejca.2015.02.015. Epub 2015 Mar 19.
Metastatic gastrointestinal stromal tumour (GIST) is generally an incurable disease with variable response to imatinib. We aimed to develop prognostic nomograms to predict overall survival (OS) and progression-free survival (PFS) for patients treated with imatinib.
Nomograms were developed in a training cohort (n=330) of patients treated in a randomised trial (EORTC-ISG-AGITG 62005 phase III study) using Cox regression models, and validated in patients (n=236) treated in routine clinical care from six referral centres. Nomogram performance was assessed by calculating the c statistic. A classification based on the nomograms' scores was generated to group patients according to risk.
Nomogram risk factors for OS and PFS were size of the largest metastasis, tumour genotype, primary tumour mitotic count, haemoglobin and blood neutrophil count at commencement of imatinib. The nomograms predicted survival with a c statistic of 0.75 (training) and 0.62 (validation) for OS, and 0.69 (training) and 0.62 (validation) for PFS. When tested in the validation cohort, the nomograms discriminated well the high and intermediate risk from low risk patients (hazard ratio [HR] for OS 3.83, 95% confidence interval [CI] 1.71-8.56; and 2.48, 95% CI 1.12-5.50; for PFS 2.84, 95% CI 1.66-4.87; and 1.45, 95% CI 0.87-2.41, respectively).
The nomograms predicted the risk of GIST progression and death with good discrimination of risk groups, and may be of value for patient counselling and risk stratification.
转移性胃肠道间质瘤(GIST)通常是一种无法治愈的疾病,对伊马替尼的反应各异。我们旨在开发预后列线图,以预测接受伊马替尼治疗患者的总生存期(OS)和无进展生存期(PFS)。
使用Cox回归模型在一项随机试验(EORTC - ISG - AGITG 62005 III期研究)治疗的患者训练队列(n = 330)中开发列线图,并在来自六个转诊中心接受常规临床护理的患者(n = 236)中进行验证。通过计算c统计量评估列线图性能。根据列线图得分生成分类,以便根据风险对患者进行分组。
OS和PFS的列线图风险因素包括最大转移灶大小、肿瘤基因型、原发肿瘤有丝分裂计数、伊马替尼开始治疗时的血红蛋白和血液中性粒细胞计数。列线图预测生存期时,OS的c统计量在训练组为0.75,验证组为0.62;PFS的c统计量在训练组为0.69,验证组为0.62。在验证队列中进行测试时,列线图能很好地区分高风险和中风险患者与低风险患者(OS的风险比[HR]为3.83,95%置信区间[CI]为1.71 - 8.56;中风险为2.48,95%CI为1.12 - 5.50;PFS的HR为2.84,95%CI为1.66 - 4.87;中风险为1.45,95%CI为0.87 - 2.41)。
列线图能较好地区分风险组,预测GIST进展和死亡风险,可能对患者咨询和风险分层有价值。