Mo1446 - Initial Results of the Wuppsc Study – Prospective Multicenter Withdrawal of Ursodeoxycholic Acid in Pediatric Primary Sclerosing Cholangitis (PSC)

Black, Dennis D.; Mack, Cara L.; Kerkar, Nanda; Miloh, Tamir; Sundaram, Shikha S.; Anand, Ravinder; Gupta, Ashutosh; Alonso, Estella M.; Arnon, Ronen; Bulut, Pinar; Karpen, Saul J.; Lin, Chuan-Hao; Rosenthal, Philip; Ryan, Matthew D.; Squires, Robert H.; Valentino, Pamela L.; Shneider, Benjamin L.

doi:10.1016/s0016-5085(18)33994-5

Cited by 3 publications

(2 citation statements)

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“…Upon complete withdrawal of the medication for 12 weeks, one-third of patients maintained serum GGT levels <29 IU/L. 22 Whether the one-third of patients who may have spontaneously normalized GGT and the one-third of patients who maintained a normal GGT on complete withdrawal of UDCA represent the same group is speculative, but it is likely that a large proportion of children on chronic UDCA are receiving no clinical benefit. More data on the natural history of GGT in patients exposed and not exposed to UDCA within the first 3-6 months after diagnosis are needed to determine the optimal length of a trial of UDCA therapy.…”

Section: Discussionmentioning

confidence: 99%

Ursodeoxycholic Acid Therapy in Pediatric Primary Sclerosing Cholangitis: Predictors of Gamma Glutamyltransferase Normalization and Favorable Clinical Course

Deneau

Perito

Ricciuto

et al. 2019

The Journal of Pediatrics

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Objective To investigate patient factors predictive of gamma glutamyltransferase (GGT) normalization following ursodeoxycholic acid (UDCA) therapy in children with primary sclerosing cholangitis. Study design We retrospectively reviewed patient records at 46 centers. We included patients with a baseline serum GGT level ≥50 IU/L at diagnosis of primary sclerosing cholangitis who initiated UDCA therapy within 1 month and continued therapy for at least 1 year. We defined "normalization" as a GGT level <50 IU/L without experiencing portal hypertensive or dominant stricture events, liver transplantation, or death during the first year. Results We identified 263 patients, median age 12.1 years at diagnosis, treated with UDCA at a median dose of 15 mg/kg/d. Normalization occurred in 46%. Patients with normalization had a lower prevalence of Crohn's disease, lower total bilirubin level, lower aspartate aminotransferase to platelet ratio index, greater platelet count, and greater serum albumin level at diagnosis. The 5-year survival with native liver was 99% in those patients who achieved normalization vs 77% in those who did not.

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Section: Discussionmentioning

confidence: 99%

Ursodeoxycholic Acid Therapy in Pediatric Primary Sclerosing Cholangitis: Predictors of Gamma Glutamyltransferase Normalization and Favorable Clinical Course

Deneau

Perito

Ricciuto

et al. 2019

The Journal of Pediatrics

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“…A recent clinical trial evaluated UDCA withdrawal from children with PSC who had been on chronic therapy with normal biochemistry. Upon complete withdrawal of the medication for 12 wk, 15/22 patients (68%) did not have a flare (GGT > 100) including 7/22 (32%) who maintained GGT < 29[32]. To prevent unnecessary chronic medication use, it is reasonable to attempt therapeutic withdrawal with regular monitoring of serum biochemistry to ensure each child truly needs chronic UDCA.…”

Section: Ursodeoxycholic Acidmentioning

confidence: 99%

Treatment of primary sclerosing cholangitis in children

Laborda

Jensen

Kavan

et al. 2019

WJH

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Primary sclerosing cholangitis (PSC) is a rare disease of stricturing and destruction of the biliary tree with a complex genetic and environmental etiology. Most patients have co-occurring inflammatory bowel disease. Children generally present with uncomplicated disease, but undergo a variable progression to end-stage liver disease. Within ten years of diagnosis, 50% of children will develop clinical complications including 30% requiring liver transplantation. Cholangiocarcinoma is a rare but serious complication affecting 1% of children. Ursodeoxycholic acid and oral vancomycin therapy used widely in children as medical therapy, and may be effective in a subset of patients. Gamma glutamyltransferase is a potential surrogate endpoint for disease activity, with improved survival in patients who achieve a normal value. Endoscopic retrograde cholangiopancreatography is a necessary adjunct to medical therapy to evaluate mass lesions or dominant strictures for malignancy, and also to relieve biliary obstruction. Liver transplantation remains the only option for patients who progress to end-stage liver disease. We review special considerations for patients before and after transplant, and in patients with inflammatory bowel disease. There is presently no published treatment algorithm or guideline for the management of children with PSC. We review the evidence for drug efficacy, dosing, duration of therapy, and treatment targets in PSC, and provide a framework for endoscopic and medical management of this complex problem.

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Willingness to Participate in Home Screening for Urologic Cancers in the General Population: An Online Survey of Over 1400 Adults

Tsai

Hudnall

Weiner

et al. 2020

Urology

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Mo1446 - Initial Results of the Wuppsc Study – Prospective Multicenter Withdrawal of Ursodeoxycholic Acid in Pediatric Primary Sclerosing Cholangitis (PSC)

Cited by 3 publications

References 0 publications

Ursodeoxycholic Acid Therapy in Pediatric Primary Sclerosing Cholangitis: Predictors of Gamma Glutamyltransferase Normalization and Favorable Clinical Course

Ursodeoxycholic Acid Therapy in Pediatric Primary Sclerosing Cholangitis: Predictors of Gamma Glutamyltransferase Normalization and Favorable Clinical Course

Treatment of primary sclerosing cholangitis in children

Willingness to Participate in Home Screening for Urologic Cancers in the General Population: An Online Survey of Over 1400 Adults

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