Novel Targets in Rare Liver Diseases in Childhood DOI Open Access

Stefan Bittmann

Applied Medical Research, Journal Year: 2024, Volume and Issue: unknown, P. 1 - 11

Published: April 29, 2024

Rare liver diseases are highly diverse and driven by multiple mechanisms that not well understood. Classifying these is challenging, but the European Reference Network (ERN) on rare has proposed categorizing them as autoimmune, infectious, genetic/hereditary, vascular, neoplastic, others with unknown causes.

Language: Английский

Causal associations between gut microbiota and primary biliary cholangitis: a bidirectional two-sample Mendelian randomization study DOI Creative Commons
Jiahao Zhang,

Gefeng Wu,

Yuhong Tang

et al.

Frontiers in Microbiology, Journal Year: 2023, Volume and Issue: 14

Published: Nov. 15, 2023

Background Previous studies have suggested an association between gut microbiota and primary biliary cholangitis (PBC). Nonetheless, the causal relationship PBC risk remains unclear. Methods A bidirectional two-sample Mendelian Randomization (MR) study was employed using summary statistical data for from MiBioGen consortium Genome-Wide Association Studies (GWAS) database to investigate relationships 211 risk. Inverse variance weighted (IVW) method analytical approach assess causality, pleiotropy heterogeneity tests were verify robustness of findings. Additionally, we performed reverse MR analyses possibility association. Results The IVW identified five that demonstrated associations with PBC. Order Selenomonadales [odds ratio (OR) 2.13, 95% confidence interval (CI) 1.10–4.14, p = 0.03], Bifidobacteriales (OR 1.58, CI 1.07–2.33, 0.02), Genus Lachnospiraceae_UCG_004 1.64, 95%CI 1.06–2.55, 0.03) correlated a higher PBC, while Family Peptostreptococcaceae 0.65, 0.43–0.98, 0.04) Ruminococcaceae 0.33, 0.15–0.72, 0.01) had protective effect on analysis no statistically significant these specific microbial taxa. Conclusion This revealed there taxa which may provide novel perspectives theoretical basis clinical prevention, diagnosis, treatment

Language: Английский

Citations

7
The treatment of primary biliary cholangitis: from shadow to light DOI Creative Commons
Sylvia Dražilová,

Tomas Koky,

Marian Macej

et al.

Therapeutic Advances in Gastroenterology, Journal Year: 2024, Volume and Issue: 17

Published: Jan. 1, 2024

Primary biliary cholangitis (PBC) is a chronic autoimmune cholestatic disease characterized by the destruction of small intrahepatic bile ducts, which can progress to liver cirrhosis. The gold standard in treatment PBC ursodeoxycholic acid (UDCA), indicated all patients with because it improves not only biochemical parameters but also patients’ survival. An important milestone identification at risk assessment response UDCA. Patients who respond have lower incidence hepatic events and better prognosis than do not. Several scoring systems be used assess identify non-responders will benefit from second-line treatment. Obeticholic (OCA) currently approved for PBC, effective UDCA therapy or patients, tolerated therapy. However, OCA contraindicated advanced cirrhosis portal hypertension. Moreover, pruritus may limiting factor administration OCA. Fibrates shown promising data supporting their use they improve elastographic findings possible antipruritic effects. Therefore, idea triple seems interesting. Clinical research focusing on several other groups drugs: peroxisome proliferator-activated receptor (PPAR) δ- α/δ agonists, non-steroidal farnesoid X fibroblast growth 19 modulators, inhibitors nicotinamide adenine dinucleotide phosphate oxidase 1 4.

Language: Английский

Citations

2
ALKALINE PHOSPHATASE AND LIVER FIBROSIS AT DIAGNOSIS ARE ASSOCIATED WITH DEEP RESPONSE TO URSODEOXYCHOLIC ACID IN PRIMARY BILIARY CHOLANGITIS DOI
Guilherme Grossi Lopes Cançado, Patrícia da Silva Fucuta,

Nathália Mota de Faria Gomes

et al.

Clinics and Research in Hepatology and Gastroenterology, Journal Year: 2024, Volume and Issue: 48(8), P. 102453 - 102453

Published: Aug. 23, 2024

Language: Английский

Citations

2
Molecular Mechanisms of Fibrosis in Cholestatic Liver Diseases and Regenerative Medicine-Based Therapies DOI Creative Commons
Weilu Wang,

Huibin Lian,

Yingyu Liang

et al.

Cells, Journal Year: 2024, Volume and Issue: 13(23), P. 1997 - 1997

Published: Dec. 3, 2024

The aim of this review is to explore the potential new regenerative medicine approaches in treatment cholestatic liver fibrosis. Cholestatic diseases, such as primary biliary cholangitis (PBC), sclerosing (PSC), and atresia (BA), due accumulation bile, often progress fibrosis, cirrhosis, failure. When disease becomes severe enough require transplantation. Deeply understanding disease's progression fibrosis formation crucial for better diagnosis treatment. Current treatments mainly target root causes no direct method itself. Recent advances offer a approach that may help find ways directly, offering hope improved outcomes. We also summarize, analyze, discuss current state benefits therapies mesenchymal stem cell (MSC) therapy, induced pluripotent cells (iPSCs), organoid technology, which diseases. Focusing on latest research reveal targets enhance therapeutic efficacy, potentially leading more effective management even curative strategies

Language: Английский

Citations

2
Novel Targets in Rare Liver Diseases in Childhood DOI Open Access

Stefan Bittmann

Applied Medical Research, Journal Year: 2024, Volume and Issue: unknown, P. 1 - 11

Published: April 29, 2024

Rare liver diseases are highly diverse and driven by multiple mechanisms that not well understood. Classifying these is challenging, but the European Reference Network (ERN) on rare has proposed categorizing them as autoimmune, infectious, genetic/hereditary, vascular, neoplastic, others with unknown causes.

Language: Английский

Citations

1