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

Full Text via DOI: 10.1080/00365521.2020.1787501 PMID: 32633158 Web of Science: 000547465400001

Cited authors

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


  • 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 (totaln = 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.

Publication date

  • 2020


International Standard Serial Number (ISSN)

  • 0036-5521

Start page

  • 941

End page

  • 950


  • 55


  • 8