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布加综合征的预后指标:对临床研究有效,但对个体治疗不足。

Prognostic indices for Budd-Chiari syndrome: valid for clinical studies but insufficient for individual management.

作者信息

Rautou Pierre-Emmanuel, Moucari Rami, Escolano Sylvie, Cazals-Hatem Dominique, Denié Cécile, Chagneau-Derrode Carine, Charpignon Claire, Lédinghen Victor de, Grenouillet-Delacre Marieke, Habersetzer François, Nousbaum Jean-Baptiste, Denninger Marie-Hélène, Valla Dominique C, Plessier Aurélie

机构信息

Pôle des Maladies de l'Appareil Digestif, Service d'Hépatologie, Hôpital Beaujon, AP-HP and INSERM U773, Centre de Recherche Biomédicale Bichat Beaujon CRB3, Université Paris 7-Denis-Diderot, Clichy, France.

出版信息

Am J Gastroenterol. 2009 May;104(5):1140-6. doi: 10.1038/ajg.2009.63. Epub 2009 Mar 31.

DOI:10.1038/ajg.2009.63
PMID:19337247
Abstract

OBJECTIVES

Several prognostic indices (PIs) have been proposed for Budd-Chiari syndrome (BCS). However, patient characteristics, causal factors, and treatment outcomes have changed since these indices have been elaborated. Validation in a recent patient population and comparison of predictive accuracy between these PIs are needed.

METHODS

A database of 96 BCS patients diagnosed between 1995 and 2005 was analyzed. Cox survival models were fitted with time to liver transplantation or death, and time to invasive therapy or death, as end points. The prognostic values of known indices (Child-Pugh score, model for end-stage liver disease (MELD), Clichy, Rotterdam BCS index, New Clichy, and BCS-TIPS (transjugular intrahepatic portosystemic shunt)) at diagnosis were assessed in Cox models using the chi-square test, the Kent and O'Quigley measure of dependence, and unrestricted bootstrapping analysis. Areas under receiver operating characteristic curves (AUROCs) were built for both end points and compared.

RESULTS

All prognostic indices, except BCS-TIPS, were significant predictors of transplant-free and invasive therapy-free survival. However, only 31 and 37% of the variance in transplant-free and invasive therapy-free survival, respectively, were explained by the best performing indices. For transplant-free survival, AUROCs were < 0.70. For invasive therapy-free survival, AUROCs were < 0.80. For both end points, BCS-TIPS PI AUROCs were significantly lower than others.

CONCLUSIONS

Most PIs are valid for transplant-free survival and invasive therapy-free survival in a population of current BCS patients, and thus can be used for stratification in clinical studies. However, predictive accuracy is insufficient to be used for individual patients.

摘要

目的

已针对布加综合征(BCS)提出了多种预后指数(PI)。然而,自这些指数制定以来,患者特征、病因以及治疗结果都已发生变化。因此需要在近期患者群体中进行验证,并比较这些PI之间的预测准确性。

方法

分析了一个包含1995年至2005年间确诊的96例BCS患者的数据库。以肝移植或死亡时间以及侵入性治疗或死亡时间作为终点,拟合Cox生存模型。使用卡方检验、肯特和奥奎利依赖度量以及无限制自助分析,在Cox模型中评估诊断时已知指数(Child-Pugh评分、终末期肝病模型(MELD)、克利希、鹿特丹BCS指数、新克利希和BCS-TIPS(经颈静脉肝内门体分流术))的预后价值。针对两个终点构建受试者操作特征曲线下面积(AUROC)并进行比较。

结果

除BCS-TIPS外,所有预后指数都是无移植生存和无侵入性治疗生存的显著预测指标。然而,分别仅有31%和37%的无移植生存和无侵入性治疗生存差异可由表现最佳的指数解释。对于无移植生存,AUROC<0.70。对于无侵入性治疗生存,AUROC<0.80。对于两个终点,BCS-TIPS PI的AUROC均显著低于其他指数。

结论

大多数PI对于当前BCS患者群体的无移植生存和无侵入性治疗生存是有效的,因此可用于临床研究中的分层。然而,预测准确性不足以用于个体患者。

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