Effect of scheduled endoscopic dilatation of dominant strictures on outcome in patients with primary sclerosing cholangitis

Abstract

ObjectiveScheduled endoscopic dilatation of dominant strictures (DS) in primary sclerosing cholangitis (PSC) might improve outcome relative to endoscopic treatment on demand, but evidence is limited. Since randomisation is difficult in clinical practice, we present a large retrospective study comparing scheduled versus on-demand endoscopic retrograde cholangiopancreatography (ERCP) based on patient preferences.DesignBetween 1987 and 2017, all new patients with PSC had been offered scheduled ERCP with dilatation of a DS if diagnosed; the latter was repeated at defined intervals until morphological resolution, independent of clinical symptoms (treatment group). Patients who refused participation were clinically evaluated annually and received endoscopic treatment only on demand (control group). The primary clinical endpoint was transplantation-free survival. Secondary outcomes were overall survival, bacterial cholangitis episodes, hepatic decompensation of liver cirrhosis and endoscopy-related adverse events.ResultsThe final study included 286 patients, 133 (46.5%) receiving scheduled ERCP and 153 (53.5%) receiving on-demand ERCP. After a mean follow-up of 9.9 years, the rate of transplantation-free survival was higher in patients receiving scheduled ERCP (51% vs 29.3%; p<0.001), as was transplantation-free survival time (median: 17.9 vs 15.2 years; log-rank: p=0.008). However, the benefit of scheduled ERCP was significant only in patients with the initial (17.1%) or later (45.5%) diagnosis of a DS (17.8 vs 11.1 years; log-rank: p<0.001). IBD (p=0.03), DS (p=0.006), higher Mayo Risk Score (p=0.02) and non-adherence to scheduled endoscopy (p=0.005) were independently associated with transplantation-free survival.ConclusionIn our large retrospective study, regular ERCP with endoscopic balloon dilatation significantly benefits patients with PSC with DS, diagnosed both at initial presentation and during surveillance, even if asymptomatic. Further studies have to find out how to best identify stricture patients non-invasively.

Precision medicine in primary sclerosing cholangitis

“…Interestingly, they found that the scheduled treatment group (n = 133) had improved TFS compared with the standard care group (n = 153) (5‐y survival rate: 84% vs 67%; 10‐y survival rate: 69% vs 50%; 20‐y survival rate: 40% vs 25%). However, in a subgroup analysis this benefit was found only in patients with DS, providing good evidence for the prognostic relevance of DS and the potential benefit of proactive endoscopic therapy …”

Role of Endoscopy in Primary Sclerosing Cholangitis

“…In this study, 51% of scheduled ERCP patients were transplant-free at 5 years, compared to 29% in the clinically indicated ERCP group. 43 Nevertheless, most centers do not offer scheduled procedures at this stage.…”

The future of autoimmune liver diseases – Understanding pathogenesis and improving morbidity and mortality

“…A phase III study with NorUDCA for PSC is ongoing. Rupp et al showed that endoscopic bile duct dilatation was beneficial in patients with PSC in a large retrospective study . PSC patients with a dominant bile duct stricture received either endoscopy with or without bile duct dilation at defined intervals or in case of clinical symptoms, an approach that does not follow current recommendations from European and American guidelines.…”

“…Rupp et al showed that endoscopic bile duct dilatation was beneficial in patients with PSC in a large retrospective study. 22…”