原发性胆汁性胆管炎预后模型的演进与应用
DOI: 10.12449/JCH250730
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摘要: 原发性胆汁性胆管炎(PBC)是一种自身免疫性疾病,好发于中年女性。PBC早期临床症状不明显,最终可发展至肝硬化并出现各种并发症。熊去氧胆酸是治疗PBC最有效的药物,但仍有40%的PBC患者对熊去氧胆酸应答不敏感,导致病情缓慢进展。准确评估PBC患者病情及预后有助于治疗方案的优化。本文就PBC预后模型的研究进展进行综述,以期为临床治疗提供参考。Abstract: Primary biliary cholangitis (PBC) is an autoimmune liver disease often observed in middle-aged women. The early clinical symptoms of PBC are not obvious, and it can progress to liver cirrhosis and lead to various complications. Ursodeoxycholic acid (UDCA) is the most effective drug for the treatment of PBC, but about 40% of the patients are still insensitive to UDCA, which can lead to slow progression of the disease. Accurate assessment of disease conditions and patient prognosis can help to optimize treatment regimens. This article reviews the research advances in prognostic models for PBC, in order to provide a reference for clinical treatment.
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Key words:
- Primary Biliary Cholangitis /
- Prognosis /
- Model
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表 1 PBC预后模型/评分系统
Table 1. Primary biliary cholangitis prognostic models and scoring systems
预后模型/评分系统 模型组成/评分定义 Yale模型 0.037×1.037×年龄+0.74×2.1×肝肿大(否=0,是=1)-1.34×0.26×门静脉纤维化(否=0,是=1)+0.82×2.26×[胆
红素≥5 mg/dL(否=0,是=1)]-0.73×0.48×[胆红素<1.5 mg/dL(否=0,是=1)]简化版Mayo模型 0.871×lnTBil(mg/dL)-2.53×lnAlb(mg/dL)+0.039×年龄+2.38×ln凝血酶原时间(s)+0.859×水肿积分1) European模型 0.04×(年龄-55)+1.39×腹水(否=0,是=1)+0.65×静脉曲张破裂出血(否=0,是=1)-0.085×[Alb(g/L)-34.3]+
2.53×[log胆红素(µmol/L)-1.53]Rochester标准 ALP<2×ULN和Mayo模型<4.5分 巴塞罗那标准 ALP水平下降>40%或降至正常(UDCA治疗1年) ParisⅠ标准 ALP≤3×ULN,AST≤2×ULN,胆红素正常(UDCA治疗1年) 鹿特丹标准 胆红素和/或Alb降至正常(UDCA治疗1年) 爱媛标准 GGT水平下降≥70%或降至正常(UDCA治疗6个月) 多伦多标准 ALP<1.67×ULN(UDCA治疗2年) Paris Ⅱ标准 ALP≤1.5×ULN,AST≤1.5×ULN,胆红素正常(UDCA治疗1年) APRI指数 APRI≤0.54(UDCA治疗前或治疗1年) ALBI评分 -0.085×Alb(g/L)+0.66×log胆红素(µmol/L) GLOBE评分 0.044 378×年龄+0.939 82×ln(胆红素×ULN)+0.335 648×ln(ALP×ULN)-2.266 708×(Alb× LLN)-0.002 581×
血小板计数(109/L)+1.216 8652)UK-PBC评分 1-基线幸存者功能ˆexp{0.028 785 43×(治疗1年时ALP×ULN-1.722 136 304)-0.042 287 3×[(治疗1年时ALT
或AST×ULN/10)-1-8.675 729 006]+1.419 9×[ln(治疗1年时胆红素×ULN/10)+2.709 607 778]-1.960 303×(基
线Alb×LLN-1.176 730 01)-0.416 195 4×(基线血小板×LLN-1.873 564 875)}3)注:1)水肿积分(0,无水肿;0.5,利尿剂可控制的水肿;1,利尿剂不能控制的水肿);2)年龄为开始UCDA治疗时,胆红素、ALP、Alb及血小板计数为治疗1年后;ULN,正常值上限,LLN,正常值下限;3)基线幸存者功能即0.982(在5年时)、0.941(在10年时)、0.893(在15年时)。
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[1] LU M, ZHOU YR, HALLER IV, et al. Increasing prevalence of primary biliary cholangitis and reduced mortality with treatment[J]. Clin Gastroenterol Hepatol, 2018, 16( 8): 1342- 1350. e 1. DOI: 10.1016/j.cgh.2017.12.033. [2] ZENG N, DUAN WJ, CHEN S, et al. Epidemiology and clinical course of primary biliary cholangitis in the Asia-Pacific region: A systematic review and meta-analysis[J]. Hepatol Int, 2019, 13( 6): 788- 799. DOI: 10.1007/s12072-019-09984-x. [3] TRIVELLA J, JOHN BV, LEVY C. Primary biliary cholangitis: Epidemiology, prognosis, and treatment[J]. Hepatol Commun, 2023, 7( 6): e0179. DOI: 10.1097/HC9.0000000000000179. [4] TANAKA A, LEUNG PS, GERSHWIN ME. Environmental basis of primary biliary cholangitis[J]. Exp Biol Med(Maywood), 2018, 243( 2): 184- 189. DOI: 10.1177/1535370217748893. [5] HOURI I, HIRSCHFIELD GM. Primary biliary cholangitis: Pathophysiology[J]. Clin Liver Dis, 2024, 28( 1): 79- 92. DOI: 10.1016/j.cld.2023.06.006. [6] CHALIFOUX SL, KONYN PG, CHOI G, et al. Extrahepatic manifestations of primary biliary cholangitis[J]. Gut Liver, 2017, 11( 6): 771- 780. DOI: 10.5009/gnl16365. [7] EFE C, TORGUTALP M, HENRIKSSON I, et al. Extrahepatic autoimmune diseases in primary biliary cholangitis: Prevalence and significance for clinical presentation and disease outcome[J]. J Gastroenterol Hepatol, 2021, 36( 4): 936- 942. DOI: 10.1111/jgh.15214. [8] ROLL J, BOYER JL, BARRY D, et al. The prognostic importance of clinical and histologic features in asymptomatic and symptomatic primary biliary cirrhosis[J]. N Engl J Med, 1983, 308( 1): 1- 7. DOI: 10.1056/NEJM198301063080101. [9] DICKSON ER, GRAMBSCH PM, FLEMING TR, et al. Prognosis in primary biliary cirrhosis: Model for decision making[J]. Hepatology, 1989, 10( 1): 1- 7. DOI: 10.1002/hep.1840100102. [10] KIM WR, WIESNER RH, POTERUCHA JJ, et al. Adaptation of the Mayo primary biliary cirrhosis natural history model for application in liver transplant candidates[J]. Liver Transpl, 2000, 6( 4): 489- 494. DOI: 10.1053/jlts.2000.6503. [11] MIJIC M, SARIC I, DELIJA B, et al. Pretransplant evaluation and liver transplantation outcome in PBC patients[J]. Can J Gastroenterol Hepatol, 2022, 2022: 7831165. DOI: 10.1155/2022/7831165. [12] GOET JC, MURILLO PEREZ CF, HARMS MH, et al. A comparison of prognostic scores(mayo, UK-PBC, and GLOBE) in primary biliary cholangitis[J]. Am J Gastroenterol, 2021, 116( 7): 1514- 1522. DOI: 10.14309/ajg.0000000000001285. [13] FENG J, XU JM, FU HY, et al. Prognostic scores in primary biliary cholangitis patients with advanced disease[J]. World J Gastrointest Surg, 2023, 15( 8): 1774- 1783. DOI: 10.4240/wjgs.v15.i8.1774. [14] CHRISTENSEN E, NEUBERGER J, CROWE J, et al. Beneficial effect of azathioprine and prediction of prognosis in primary biliary cirrhosis. Final results of an international trial[J]. Gastroenterology, 1985, 89( 5): 1084- 1091. DOI: 10.1016/0016-5085(85)90213-6. [15] European Association for the Study of the Liver. EASL Clinical Practice Guidelines: The diagnosis and management of patients with primary biliary cholangitis[J]. J Hepatol, 2017, 67( 1): 145- 172. DOI: 10.1016/j.jhep.2017.03.022. [16] ANGULO P, LINDOR KD, THERNEAU TM, et al. Utilization of the Mayo risk score in patients with primary biliary cirrhosis receiving ursodeoxycholic acid[J]. Liver, 1999, 19( 2): 115- 121. DOI: 10.1111/j.1478-3231.1999.tb00020.x. [17] PARÉS A, CABALLERÍA L, RODÉS J. Excellent long-term survival in patients with primary biliary cirrhosis and biochemical response to ursodeoxycholic Acid[J]. Gastroenterology, 2006, 130( 3): 715- 720. DOI: 10.1053/j.gastro.2005.12.029. [18] CORPECHOT C, ABENAVOLI L, RABAHI N, et al. Biochemical response to ursodeoxycholic acid and long-term prognosis in primary biliary cirrhosis[J]. Hepatology, 2008, 48( 3): 871- 877. DOI: 10.1002/hep.22428. [19] LAMMERS WJ, van BUUREN HR, HIRSCHFIELD GM, et al. Levels of alkaline phosphatase and bilirubin are surrogate end points of outcomes of patients with primary biliary cirrhosis: An international follow-up study[J]. Gastroenterology, 2014, 147( 6): 1338- 1349. e5; quize 15. DOI: 10.1053/j.gastro.2014.08.029. [20] CARBONE M, SHARP SJ, FLACK S, et al. The UK-PBC risk scores: Derivation and validation of a scoring system for long-term prediction of end-stage liver disease in primary biliary cholangitis[J]. Hepatology, 2016, 63( 3): 930- 950. DOI: 10.1002/hep.28017. [21] KUIPER EMM, HANSEN BE, de VRIES RA, et al. Improved prognosis of patients with primary biliary cirrhosis that have a biochemical response to ursodeoxycholic acid[J]. Gastroenterology, 2009, 136( 4): 1281- 1287. DOI: 10.1053/j.gastro.2009.01.003. [22] BALASUBRAMANIAM K, GRAMBSCH PM, WIESNER RH, et al. Diminished survival in asymptomatic primary biliary cirrhosis. A prospective study[J]. Gastroenterology, 1990, 98( 6): 1567- 1571. DOI: 10.1016/0016-5085(90)91091-J. [23] AZEMOTO N, ABE M, MURATA Y, et al. Early biochemical response to ursodeoxycholic acid predicts symptom development in patients with asymptomatic primary biliary cirrhosis[J]. J Gastroenterol, 2009, 44( 6): 630- 634. DOI: 10.1007/s00535-009-0051-9. [24] AZEMOTO N, KUMAGI T, ABE M, et al. Biochemical response to ursodeoxycholic acid predicts long-term outcome in Japanese patients with primary biliary cirrhosis[J]. Hepatol Res, 2011, 41( 4): 310- 317. DOI: 10.1111/j.1872-034X.2011.00782.x. [25] KUMAGI T, GUINDI M, FISCHER SE, et al. Baseline ductopenia and treatment response predict long-term histological progression in primary biliary cirrhosis[J]. Am J Gastroenterol, 2010, 105( 10): 2186- 2194. DOI: 10.1038/ajg.2010.216. [26] ZHANG LN, SHI TY, SHI XH, et al. Early biochemical response to ursodeoxycholic acid and long-term prognosis of primary biliary cirrhosis: Results of a 14-year cohort study[J]. Hepatology, 2013, 58( 1): 264- 272. DOI: 10.1002/hep.26322. [27] TABABI R, MRABET S, AKKARI I, et al. Prognostic scores in primary biliary cholangitis[J]. Future Sci OA, 2024, 10( 1): FSO975. DOI: 10.2144/fsoa-2023-0203. [28] TRIVEDI PJ, BRUNS T, CHEUNG A, et al. Optimising risk stratification in primary biliary cirrhosis: AST/platelet ratio index predicts outcome independent of ursodeoxycholic acid response[J]. J Hepatol, 2014, 60( 6): 1249- 1258. DOI: 10.1016/j.jhep.2014.01.029. [29] JOHNSON PJ, BERHANE S, KAGEBAYASHI C, et al. Assessment of liver function in patients with hepatocellular carcinoma: A new evidence-based approach-the ALBI grade[J]. J Clin Oncol, 2015, 33( 6): 550- 558. DOI: 10.1200/JCO.2014.57.9151. [30] CHAN AWH, CHAN RCK, WONG GLH, et al. New simple prognostic score for primary biliary cirrhosis: Albumin-bilirubin score[J]. J Gastroenterol Hepatol, 2015, 30( 9): 1391- 1396. DOI: 10.1111/jgh.12938. [31] CHEN QL, ZHONG R, WANG Y, et al. The albumin-bilirubin score as a predictor of liver-related mortality in primary biliary cholangitis with compensated cirrhosis[J]. Dig Dis, 2023, 41( 6): 946- 956. DOI: 10.1159/000531557. [32] ITO T, ISHIGAMI M, MOROOKA H, et al. The albumin-bilirubin score as a predictor of outcomes in Japanese patients with PBC: An analysis using time-dependent ROC[J]. Sci Rep, 2020, 10( 1): 17812. DOI: 10.1038/s41598-020-74732-3. [33] LAMMERS WJ, HIRSCHFIELD GM, CORPECHOT C, et al. Development and validation of a scoring system to predict outcomes of patients with primary biliary cirrhosis receiving ursodeoxycholic acid therapy[J]. Gastroenterology, 2015, 149( 7): 1804- 1812. e 4. DOI: 10.1053/j.gastro.2015.07.061. [34] CARBONE M, HARMS MH, LAMMERS WJ, et al. Clinical application of the GLOBE and United Kingdom-primary biliary cholangitis risk scores in a trial cohort of patients with primary biliary cholangitis[J]. Hepatol Commun, 2018, 2( 6): 683- 692. DOI: 10.1002/hep4.1180. [35] SOHAL A, KOWDLEY KV. Primary biliary cholangitis: Promising emerging innovative therapies and their impact on GLOBE scores[J]. Hepat Med, 2023, 15: 63- 77. DOI: 10.2147/HMER.S361077. [36] CHEUNG AC, GULAMHUSEIN AF, JURAN BD, et al. External validation of the United Kingdom-primary biliary cholangitis risk scores of patients with primary biliary cholangitis treated with ursodeoxycholic acid[J]. Hepatol Commun, 2018, 2( 6): 676- 682. DOI: 10.1002/hep4.1186. [37] YANG F, YANG Y, WANG Q, et al. The risk predictive values of UK-PBC and GLOBE scoring system in Chinese patients with primary biliary cholangitis: The additional effect of anti-gp210[J]. Aliment Pharmacol Ther, 2017, 45( 5): 733- 743. DOI: 10.1111/apt.13927. [38] ALOMARI M, COVUT F, MOMANI L AL, et al. Evaluation of the United Kingdom-primary biliary cholangitis and global primary biliary cholangitis group prognostic models for primary biliary cholangitis patients treated with ursodeoxycholic acid in the U.S. population[J]. JGH Open, 2020, 4( 2): 132- 139. DOI: 10.1002/jgh3.12223. [39] CHEUNG KS, SETO WK, FUNG J, et al. Prognostic factors for transplant-free survival and validation of prognostic models in Chinese patients with primary biliary cholangitis receiving ursodeoxycholic acid[J]. Clin Transl Gastroenterol, 2017, 8( 6): e100. DOI: 10.1038/ctg.2017.23. [40] MARENCO-FLORES A, ROJAS AMARIS N, KAHAN T, et al. The external validation of GLOBE and UK-PBC risk scores for predicting ursodeoxycholic acid treatment response in a large U.S. cohort of primary biliary cholangitis patients[J]. J Clin Med, 2024, 13( 15): 4497. DOI: 10.3390/jcm13154497. [41] WANG R, MA JL, ZHANG FK, et al. Prediction of decompensation in patients with primary biliary cirrhosis[J]. Chin Hepatol, 2012, 17( 7): 460- 464.王蕊, 马佳丽, 张福奎, 等. 原发性胆汁性肝硬化发生失代偿的预测[J]. 肝脏, 2012, 17( 7): 460- 464. -
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