Expert review of gastroenterology & hepatology · 2026
Oral vancomycin for primary sclerosing cholangitis and associated inflammatory bowel disease - paving a path forward.
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Johnson Kevin M, Grady John, Kellermayer Richard, Winter Harland S, Tabibian James H, Buness Cynthia W, Fischer Ryan, Shah Ayesha, Dulai Parambir S, Rangnekar Amol S, Alrabadi Leina, Shah Shamita B, Holtmann Gerald, Räisänen Laura, Lewindon Peter, Ali Ahmad H
Аннотация
INTRODUCTION: Primary sclerosing cholangitis (PSC) is a fibro-inflammatory cholangiopathy strongly associated with inflammatory bowel disease (PSC-IBD). With no approved PSC therapy, clinicians face uncertainty about oral vancomycin (OV) as a therapeutic option. This review synthesizes clinical effectiveness evidence alongside mechanistic data.
AREAS COVERED: We searched PubMed/Google Scholar for studies of vancomycin in PSC from 1998 through November, 2025. OV shows consistent IBD benefits and variable liver responses. In PSC-IBD, clinical and endoscopic remission occurred in 60% at 6 months and in 71% at 12 months in a pediatric cohort; in an adult single-arm study (n = 15), 80% achieved endoscopic remission at 4 weeks with universal mucosal healing, reductions in fecal calprotectin and Mayo scores, and relapse after withdrawal. For liver disease, a pediatric open-label cohort (n = 45) reported ≥50% declines in gamma-glutamyl transferase in 82%; in an adult pilot randomized controlled trial, alkaline phosphatase fell 46% at 12 weeks. Imaging/histology improved as evidenced by MRCP in 26/34 large-duct PSC and reduced portal/periportal inflammation in 11/12 small-duct PSC. No vancomycin-resistant enterococci development has been reported.
EXPERT OPINION: OV appears effective for colitis control in PSC-IBD. Differences in observed liver outcomes across studies likely reflect variation in treatment duration, dose, and endpoint selection. Liver responses may depend on higher doses. Cross-specialty guidance and pragmatic, integrated trials are needed.
AREAS COVERED: We searched PubMed/Google Scholar for studies of vancomycin in PSC from 1998 through November, 2025. OV shows consistent IBD benefits and variable liver responses. In PSC-IBD, clinical and endoscopic remission occurred in 60% at 6 months and in 71% at 12 months in a pediatric cohort; in an adult single-arm study (n = 15), 80% achieved endoscopic remission at 4 weeks with universal mucosal healing, reductions in fecal calprotectin and Mayo scores, and relapse after withdrawal. For liver disease, a pediatric open-label cohort (n = 45) reported ≥50% declines in gamma-glutamyl transferase in 82%; in an adult pilot randomized controlled trial, alkaline phosphatase fell 46% at 12 weeks. Imaging/histology improved as evidenced by MRCP in 26/34 large-duct PSC and reduced portal/periportal inflammation in 11/12 small-duct PSC. No vancomycin-resistant enterococci development has been reported.
EXPERT OPINION: OV appears effective for colitis control in PSC-IBD. Differences in observed liver outcomes across studies likely reflect variation in treatment duration, dose, and endpoint selection. Liver responses may depend on higher doses. Cross-specialty guidance and pragmatic, integrated trials are needed.