Open-label prospective therapeutic clinical trials: oral vancomycin in children and adults with primary sclerosing cholangitis

Ahmad Hassan Ali, Jennifer Damman, Shamita B. Shah, Yinka Davies, Melissa Hurwitz, Mariam Stephen, Leta M. Lemos, Elizabeth J. Carey, Keith D. Lindor, Cynthia W. Buness, Leina Alrabadi, William E. Berquist, Kenneth L. Cox

Research output: Contribution to journalArticlepeer-review

32 Scopus citations


Background: Oral vancomycin (OV) in primary sclerosing cholangitis (PSC) has been evaluated as a potential therapeutic agent. We report the long-term biochemical course and outcomes of patients with PSC treated with OV. Methods: Patients were enrolled in 2 open-label clinical trials ( Identifier: NCT01802073 and NCT01322386) and offered OV at 50 mg/kg/day in 3 divided doses if weight <30kg, and 500 mg 3 times/day if weight ≥30kg. Patients with biliary strictures requiring stenting or awaiting liver transplant were excluded. Liver biochemistry, MRCP and histology were documented at baseline and while on OV. The primary outcome was a decrease in elevated gamma glutamyl transferase (GGT), alkaline phosphatase (ALP), and/or alanine aminotransferase (ALT) from baseline. Results: 30 subjects were enrolled, and 29 additional subjects who learned of the clinical trial requested OV (total n = 59; median age was 13.5 years [range, 1.5–44 years]; 64.4% were male; and 94.9% had inflammatory bowel disease [IBD]). The median treatment duration was 2.7 years (range, 0.2–14 years). Ninety-six percent (57/59), 81.3% (48/59), and 94.9% (56/59) experienced reduction of GGT, ALP, and ALT, respectively. Furthermore, 39% (23/59), 22% (13/59), and 55.9% (33/59) experienced normalization of GGT, ALP, and ALT, respectively, within the first 6 months of OV treatment. One patient underwent liver transplantation 8 years after beginning OV treatment, and one developed biliary strictures requiring endoscopic intervention. OV was well-tolerated by patients, and no patient developed treatment-related adverse events. Conclusion: In PSC, OV was well-tolerated and was associated with improvement in liver chemistry. A randomized placebo-controlled clinical trial is warranted.

Original languageEnglish (US)
Pages (from-to)1-10
Number of pages10
JournalScandinavian Journal of Gastroenterology
StatePublished - 2020


  • Primary sclerosing cholangitis
  • and ulcerative colitis
  • cholangiography
  • cirrhosis
  • inflammatory bowel disease
  • liver biochemistry

ASJC Scopus subject areas

  • Gastroenterology


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