Trial reportJournal of clinical gastroenterology2017
Combination Therapy of All-Trans Retinoic Acid With Ursodeoxycholic Acid in Patients With Primary Sclerosing Cholangitis: A Human Pilot Study.
Trial report in Journal of clinical gastroenterology, 2017. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 25 papers.
What it found
Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.
The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.
Who cites it
25 citing papers in PubMed, 49 citations in OpenAlex.
- Pediatric Autoimmune or Primary Sclerosing Cholangitis: Metronidazole Effectiveness on Biochemical Data, Bile Acid Profile, and Gut Microbiota: A Pilot Study.JPGN reports · 2023Trial
- Cholecystectomy Is Linked to Worse Clinical Outcomes in Primary Sclerosing Cholangitis.Cureus · 2025Article
- Hepatic Nuclear Receptors in Cholestasis-to-Cholangiocarcinoma Pathology.The American journal of pathology · 2025Review
- Current status of pharmacotherapy for primary sclerosing cholangitis.Frontiers in medicine · 2025Review
- Fat malabsorption in short bowel syndrome: A review of pathophysiology and management.Nutrition in clinical practice : official publication of the American Society for Parenteral and Enteral Nutrition · 2024Review
- Current Therapeutics in Primary Sclerosing Cholangitis.Journal of clinical and translational hepatology · 2023Review
- Treatment of primary sclerosing cholangitis combined with inflammatory bowel disease.Intestinal research · 2023Review
- The gut-liver axis in cholangiopathies: focus on bile acid based pharmacological treatment.Current opinion in gastroenterology · 2022Review
- Therapeutic targets, novel drugs, and delivery systems for diabetes associated NAFLD and liver fibrosis.Advanced drug delivery reviews · 2021Review
- Review
- The role of the retinoid receptor, RAR/RXR heterodimer, in liver physiology.Biochimica et biophysica acta. Molecular basis of disease · 2021Review
- The Management of Cholestatic Liver Diseases: Current Therapies and Emerging New Possibilities.Journal of clinical medicine · 2021Review
- Review: Pathogenesis of cholestatic liver diseases.World journal of hepatology · 2020Review
- Gut microbiome in primary sclerosing cholangitis: A review.World journal of gastroenterology · 2020Review
- Emerging therapies in primary sclerosing cholangitis: pathophysiological basis and clinical opportunities.Journal of gastroenterology · 2020Review
- Novel Therapies for Managing Cholestasis.Clinical liver disease · 2020Review
- Treatment of primary sclerosing cholangitis in children.World journal of hepatology · 2019Review
- Review of pharmacotherapeutic treatments for primary sclerosing cholangitis.Canadian liver journal · 2019Review
- Altered Expression of Retinol Metabolism-Related Genes in an ANIT-Induced Cholestasis Rat Model.International journal of molecular sciences · 2018Article
- Update in the Care and Management of Patients with Primary Sclerosing Cholangitis.Current gastroenterology reports · 2018Review
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
11 authors at 5 institutions in 1 country.
Funding
Abstract
goalsTo perform an exploratory pilot study of all-trans retinoic acid (ATRA) combined with ursodeoxycholic acid (UDCA) in patients with primary sclerosing cholangitis (PSC).
backgroundPSC is a progressive disorder for which there is no accepted therapy. Studies in human hepatocyte cultures and in animal models of cholestasis indicate that ATRA might have beneficial effects in cholestatic disorders. STUDY: ATRA (45 mg/m/d, divided and given twice daily) was combined with moderate-dose UDCA in patients with PSC who had incomplete response to UDCA monotherapy. The combination was administered for 12 weeks, followed by a 12-week washout in which patients returned to UDCA monotherapy. We measured alkaline phosphatase (ALP), alanine aminotransferase (ALT), bilirubin, cholesterol, bile acids, and the bile acid intermediate 7α-hydroxy-4-cholesten-3-one (C4) at baseline, week 12, and after washout.
resultsFifteen patients completed 12 weeks of therapy. The addition of ATRA to UDCA reduced the median serum ALP levels (277±211 to 243±225 U/L, P=0.09) although this, the primary endpoint, did not reach significance. In contrast, median serum ALT (76±55 to 46±32 U/L, P=0.001) and C4 (9.8±19 to 7.9±11 ng/mL, P=0.03) levels significantly decreased. After washout, ALP and C4 levels nonsignificantly increased, whereas ALT levels significantly increased (46±32 to 74±74, P=0.0006), returning to baseline.
conclusionsIn this human pilot study, the combination of ATRA and UDCA did not achieve the primary endpoint (ALP); however, it significantly reduced ALT and the bile acid intermediate C4. ATRA appears to inhibit bile acid synthesis and reduce markers of inflammation, making it a potential candidate for further study in PSC (NCT 01456468).
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Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.