Gasparini Alessandro, Humphreys Keith
Department of Medical Epidemiology and Biostatistics, 27106Karolinska Institutet, Stockholm, Sweden.
Stat Methods Med Res. 2022 May;31(5):862-881. doi: 10.1177/09622802211072496. Epub 2022 Feb 1.
We propose a framework for jointly modelling tumour size at diagnosis and time to distant metastatic spread, from diagnosis, based on latent dynamic sub-models of growth of the primary tumour and of distant metastatic detection. The framework also includes a sub-model for screening sensitivity as a function of latent tumour size. Our approach connects post-diagnosis events to the natural history of cancer and, once refined, may prove useful for evaluating new interventions, such as personalised screening regimes. We evaluate our model-fitting procedure using Monte Carlo simulation, showing that the estimation algorithm can retrieve the correct model parameters, that key patterns in the data can be captured by the model even with misspecification of some structural assumptions, and that, still, with enough data it should be possible to detect strong misspecifications. Furthermore, we fit our model to observational data from an extension of a case-control study of post-menopausal breast cancer in Sweden, providing model-based estimates of the probability of being free from detected distant metastasis as a function of tumour size, mode of detection (of the primary tumour), and screening history. For women with screen-detected cancer and two previous negative screens, the probabilities of being free from detected distant metastases 5 years after detection and removal of the primary tumour are 0.97, 0.89 and 0.59 for tumours of diameter 5, 15 and 35 mm, respectively. We also study the probability of having latent/dormant metastases at detection of the primary tumour, estimating that 33% of patients in our study had such metastases.
我们提出了一个框架,用于基于原发性肿瘤生长和远处转移检测的潜在动态子模型,对诊断时的肿瘤大小以及从诊断开始到远处转移扩散的时间进行联合建模。该框架还包括一个将筛查敏感性作为潜在肿瘤大小函数的子模型。我们的方法将诊断后的事件与癌症的自然史联系起来,一旦完善,可能对评估新的干预措施(如个性化筛查方案)有用。我们使用蒙特卡罗模拟评估了模型拟合过程,结果表明估计算法能够检索到正确的模型参数,即使在一些结构假设设定错误的情况下,模型也能捕捉到数据中的关键模式,而且,只要有足够的数据,就应该能够检测到严重的设定错误。此外,我们将模型应用于瑞典绝经后乳腺癌病例对照研究扩展的观察数据,提供了基于模型的估计,即无远处转移检测的概率是肿瘤大小、(原发性肿瘤的)检测方式和筛查史的函数。对于经筛查发现癌症且之前有两次阴性筛查结果的女性,在原发性肿瘤检测和切除后5年无远处转移检测的概率,直径为5毫米、15毫米和35毫米的肿瘤分别为0.97、0.89和0.59。我们还研究了原发性肿瘤检测时存在潜在/休眠转移的概率,估计我们研究中的33%的患者有此类转移。