Liver transplant in patients with primary sclerosing cholangitis: A retrospective cohort from Northeastern Brazil
Surrogate endpoints are needed to estimate clinical outcomes in primary sclerosing cholangitis (PSC). Serum alkaline phosphatase was among the first markers studied, but there is substantial variability in alkaline phosphatase levels during the natural history of PSC without intervention. The Mayo r...
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| Published in: | World journal of hepatology Vol. 15; no. 9; pp. 1033 - 1042 |
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| Main Authors: | , , , , , , , , , , |
| Format: | Journal Article |
| Language: | English |
| Published: |
Baishideng Publishing Group Inc
27-09-2023
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| Subjects: | |
| Online Access: | Get full text |
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| Summary: | Surrogate endpoints are needed to estimate clinical outcomes in primary sclerosing cholangitis (PSC). Serum alkaline phosphatase was among the first markers studied, but there is substantial variability in alkaline phosphatase levels during the natural history of PSC without intervention. The Mayo risk score incorporates noninvasive variables and has served as a surrogate endpoint for survival for more than two decades. Newer models have better test performance than the Mayo risk score, including the primary sclerosing risk estimate tool (PREsTo) model and UK-PSC score that estimate hepatic decompensation and transplant free survival, respectively. The c-statistics for transplant-free survival for the Mayo risk model and the long-term UK-PSC model are 0.68 and 0.85, respectively. The c-statistics for hepatic decompensation for the Mayo risk model and PREsTo model are 0.85 and 0.90, respectively. The Amsterdam-Oxford model included patients with large duct and small duct PSC and patients with PSC-autoimmune hepatitis overlap and had a c-statistic of 0.68 for transplant-free survival. Other noninvasive tests that warrant further validation include magnetic resonance imaging, elastography and the enhanced liver fibrosis score. Prognostic models, noninvasive tests or a combination of these surrogate endpoints may not only serve to be useful in clinical trials of investigational agents, but also serve to inform our patients about their prognosis. |
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| Bibliography: | ObjectType-Article-1 SourceType-Scholarly Journals-1 ObjectType-Feature-2 content type line 23 Author contributions: Freitas LTS and Hyppolito EB designed the study; Freitas LTS, Hyppolito EB, Barreto VL, Júnior LHJC, Jorge BCM, Háteras FCTSB and Marzola MB reviewed the literature, collected data and drafted the original manuscript; Hyppolito EB performed statistical analyses and reviewed the manuscript; Hyppolito EB, Coelho GR, Garcia JHP, Lima CA and Celedonio RM reviewed the intellectual content of the manuscript; all authors read and approved the final version of the manuscript. Corresponding author: Louyse Teixeira de Souza Freitas, BSc, Nurse, Liver Transplant Department, Walter Cantídio Teaching Hospital, 1142 Coronel Nunes de Melo Street, Fortaleza 60430270, Ceará, Brazil. louyseteixeira.s@gmail.com |
| ISSN: | 1948-5182 1948-5182 |
| DOI: | 10.4254/wjh.v15.i9.1033 |