Letter to the Editor: Oral Vancomycin Versus No Therapy for Pediatric Primary Sclerosing Cholangitis

Abstract

In their recent study addressing efficacy of oral vancomycin (OVT) and ursodeoxycholic acid (UDCA) compared with no therapy in pediatric primary sclerosing cholangitis (PSC), Deneau et al. state that "OVT and UDCA are not effective treatments for PSC in children." (1) Their study shows that the null hypothesis of no effect has not been rejected; a real effect might have been present but missed. Complications take up to 10 years to develop, but the authors' median followup was just 4.2 years. The subsequent editorial (2) noted the limitations of a retrospective, propensitymatched study and argued cogently for the need to replace empiric practice with data from well-planned randomized controlled trials (RCTs).The authors' findings are discrepant with the experience of others. A prospective study in 45 pediatric patients over a median of 2.7 years showed improvement not only in biochemical findings, histologic findings, and symptoms of ulcerative colitis (UC) but also in magnetic resonance cholangiopancreatography in 26 of 34 patients. (3) A systematic review supports clinical efficacy in patients with PSC who are 16 years and older. (4) Although Deneau et al. attempted to construct a control group through retrospective matching, that method cannot correct for hidden covariates, which include the OVT brand, (5) adjustments of the dose to the level of disease activity, adherence, or genetic and microbiome differences among patients.The effects of OVT on colitis are often dramatic. The relief from diarrhea and fatigue have transformed the lives of many of our patients, with substantial improvement of UC symptoms within days or weeks being shown. Deneau et al. apparently recommend against vancomycin therapy without regard to these effects.We do not agree that the study by Deneau et al. justifies the blanket statement that OVT is not effective for PSC/UC in children. The editorial authors recommend an individualized, informed-consent process, and we agree completely. OVT should remain an empiric option in the individualized management of children with this heterogeneous liver and bowel disease that results in significant morbidity and mortality and has a paucity of treatment options. Good RCTs are urgently needed.