Primary biliary cholangitis-response criteria of first-line treatment and perspectives of second-line therapy
© 2024. The Author(s), under exclusive licence to Springer Medizin Verlag GmbH, ein Teil von Springer Nature..
BACKGROUND: Primary biliary cholangitis (PBC) is a chronic cholestatic liver disease that can progress to liver cirrhosis if left untreated. Early diagnosis, initiation of therapy and, if necessary, adjustment of treatment are essential to prevent disease progression. The timing and thresholds for assessing adequate treatment response are inconsistently defined in the literature and can pose a challenge in clinical practice.
OBJECTIVE: In addition to providing a concise overview of the guideline-based diagnostic work-up and first-line therapy, this study offers practical guidance for the evaluation of treatment response and options for second-line treatment in PBC.
MATERIALS AND METHODS: This article is based on the current European Association for the Study of the Liver (EASL) clinical practice guidelines for the management of PBC from 2017 as well as a literature review of studies from 2017 to 2023, focusing on defining treatment response, assessing disease progression risk, and the approved and investigational agents for second-line therapy.
RESULTS: There are varying definitions for a sufficient response to ursodeoxycholic acid (UDCA). Therapeutic goals are tailored to the individual risk of disease progression. The lowest risk appears to be associated with normalization of alkaline phosphatase (AP) and serum bilirubin below 0.6 the upper limit of normal. Established second-line therapies include obeticholic acid and bezafibrate (off-label use), while other peroxisome proliferator-activated receptor (PPAR) agonists and combination therapies are under clinical investigation.
DISCUSSION: Early evaluation of treatment response to UDCA is mandatory. In the case of insufficient treatment response, second-line therapy should be initiated according to the individual's risk profile.
| Medienart: |
E-Artikel |
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| Erscheinungsjahr: |
2024 |
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| Erschienen: |
2024 |
| Enthalten in: |
Zur Gesamtaufnahme - volume:65 |
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| Enthalten in: |
Innere Medizin (Heidelberg, Germany) - 65(2024), 4 vom: 07. März, Seite 340-346 |
| Sprache: |
Deutsch |
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| Weiterer Titel: |
Primär biliäre Cholangitis – Response-Kriterien der Erstlinientherapie und Perspektiven der Zweitlinientherapie |
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| Beteiligte Personen: |
Steinmann, Silja [VerfasserIn] |
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| Links: |
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| Themen: |
724L30Y2QR |
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| Anmerkungen: |
Date Completed 25.03.2024 Date Revised 25.03.2024 published: Print-Electronic Citation Status MEDLINE |
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| doi: |
10.1007/s00108-024-01674-7 |
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| funding: |
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| Förderinstitution / Projekttitel: |
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| 245 | 1 | 0 | |a Primary biliary cholangitis-response criteria of first-line treatment and perspectives of second-line therapy |
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| 520 | |a © 2024. The Author(s), under exclusive licence to Springer Medizin Verlag GmbH, ein Teil von Springer Nature. | ||
| 520 | |a BACKGROUND: Primary biliary cholangitis (PBC) is a chronic cholestatic liver disease that can progress to liver cirrhosis if left untreated. Early diagnosis, initiation of therapy and, if necessary, adjustment of treatment are essential to prevent disease progression. The timing and thresholds for assessing adequate treatment response are inconsistently defined in the literature and can pose a challenge in clinical practice | ||
| 520 | |a OBJECTIVE: In addition to providing a concise overview of the guideline-based diagnostic work-up and first-line therapy, this study offers practical guidance for the evaluation of treatment response and options for second-line treatment in PBC | ||
| 520 | |a MATERIALS AND METHODS: This article is based on the current European Association for the Study of the Liver (EASL) clinical practice guidelines for the management of PBC from 2017 as well as a literature review of studies from 2017 to 2023, focusing on defining treatment response, assessing disease progression risk, and the approved and investigational agents for second-line therapy | ||
| 520 | |a RESULTS: There are varying definitions for a sufficient response to ursodeoxycholic acid (UDCA). Therapeutic goals are tailored to the individual risk of disease progression. The lowest risk appears to be associated with normalization of alkaline phosphatase (AP) and serum bilirubin below 0.6 the upper limit of normal. Established second-line therapies include obeticholic acid and bezafibrate (off-label use), while other peroxisome proliferator-activated receptor (PPAR) agonists and combination therapies are under clinical investigation | ||
| 520 | |a DISCUSSION: Early evaluation of treatment response to UDCA is mandatory. In the case of insufficient treatment response, second-line therapy should be initiated according to the individual's risk profile | ||
| 650 | 4 | |a English Abstract | |
| 650 | 4 | |a Journal Article | |
| 650 | 4 | |a Review | |
| 650 | 4 | |a Alkaline phosphatase | |
| 650 | 4 | |a Obeticholic acid | |
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