Biagini M R, McCormick P A, Guardascione M, Surrenti C, Burroughs A K
Hepato-Biliary and Liver Transplantation Unit, Royal Free Hospital and School of Medicine, London, U.K.
Ital J Gastroenterol. 1991 May;23(4):222-6.
Recent progress has been made in estimating prognosis in primary biliary cirrhosis using Cox models. These models have also demonstrated the therapeutic value of liver transplantation by comparing the observed survival for a group after transplantation with the expected survival without transplantation calculated from the Cox prognostic model. However, good risk patients and those not transplanted principally for hepatocellular failure may not have a survival advantage for many years. Cox models have several limitations: the selection criteria for the patient populations used to derive the models, the selection of the time at which the patients are evaluated, the poor prognostic accuracy for individual patients rather than patient groups and lastly the fact that they use variables derived at only one time point-time independent Cox models. Thus new statistical tools must be used to improve prediction of survival in individual patients with PBC in order to optimize timing of liver transplantation. In addition a more precise definition of the natural history of both symptomatic and asymptomatic forms of this disease is needed to evaluate the efficacy of therapeutic agents in randomized clinical trials. However, although use and timing of therapeutic intervention, including liver transplantation, still requires good clinical experience and judgement, statistical modelling does give some objective measurement of prognosis, which is useful for the clinician treating patients with PBC. At the same time that new treatments are being evaluated, there is an obvious need to improve prognostic tools for application to individual patients with PBC. This may be achieved by using serial data in a different form of modelling-time dependent Cox models.
在使用Cox模型评估原发性胆汁性肝硬化的预后方面已取得了最新进展。通过将移植后一组患者的观察生存率与根据Cox预后模型计算出的未移植预期生存率进行比较，这些模型还证明了肝移植的治疗价值。然而，病情较轻的患者以及那些并非主要因肝细胞衰竭而接受移植的患者，可能在多年内都没有生存优势。Cox模型有几个局限性：用于推导模型的患者群体的选择标准、患者评估时间的选择、对个体患者而非患者群体的预后预测准确性较差，以及最后一点，即它们使用仅在一个时间点得出的变量——时间独立Cox模型。因此，必须使用新的统计工具来改善对原发性胆汁性肝硬化个体患者生存情况的预测，以优化肝移植的时机。此外，需要对这种疾病有症状和无症状形式的自然史进行更精确的定义，以便在随机临床试验中评估治疗药物的疗效。然而，尽管治疗干预（包括肝移植）的使用和时机仍需要良好的临床经验和判断，但统计建模确实能对预后进行一些客观测量，这对治疗原发性胆汁性肝硬化患者的临床医生很有用。在评估新治疗方法的同时，显然需要改进适用于原发性胆汁性肝硬化个体患者的预后工具。这可以通过在不同形式的建模——时间依赖Cox模型中使用系列数据来实现。
