ABSTRACT
BACKGROUND: Suboptimal response to ursodeoxycholic acid occurs in 40% of primary biliary cholangitis (PBC) patients, affecting survival. Achieving a deep response (normalisation of alkaline phosphatase [ALP] and bilirubin ≤0.6 upper limit of normal) improves survival. Yet, the long-term effectiveness of second-line treatments remains uncertain. AIMS: To evaluate the long-term effectiveness of obeticholic acid (OCA) ± fibrates. Focusing on biochemical response (ALP ≤1.67 times the upper limit of normal, with a decrease of at least 15% from baseline and normal bilirubin levels), normalisation of ALP, deep response and biochemical remission (deep response plus aminotransferase normalisation). METHODS: We conducted a longitudinal, observational, multicentre study involving ursodeoxyccholic acid non-responsive PBC patients (Paris-II criteria) from Spain and Portugal who received OCA ± fibrates. RESULTS: Of 255 patients, median follow-up was 35.1 months (IQR: 20.2-53). The biochemical response in the whole cohort was 47.2%, 61.4% and 68.6% at 12, 24 and 36 months. GLOBE-PBC and 5-year UK-PBC scores improved (p < 0.001). Triple therapy (ursodeoxycholic acid plus OCA plus fibrates) had significantly higher response rates than dual therapy (p = 0.001), including ALP normalisation, deep response and biochemical remission (p < 0.001). In multivariate analysis, triple therapy remained independently associated with biochemical response (p = 0.024), alkaline phosphatase normalisation, deep response and biochemical remission (p < 0.001). Adverse effects occurred in 41.2% of cases, leading to 18.8% discontinuing OCA. Out of 55 patients with cirrhosis, 12 developed decompensation. All with baseline portal hypertension. CONCLUSION: Triple therapy was superior in achieving therapeutic goals in UDCA-nonresponsive PBC. Decompensation was linked to pre-existing portal hypertension.
Subject(s)
Alkaline Phosphatase , Chenodeoxycholic Acid , Cholagogues and Choleretics , Drug Therapy, Combination , Liver Cirrhosis, Biliary , Ursodeoxycholic Acid , Humans , Chenodeoxycholic Acid/analogs & derivatives , Chenodeoxycholic Acid/therapeutic use , Male , Female , Middle Aged , Ursodeoxycholic Acid/therapeutic use , Longitudinal Studies , Liver Cirrhosis, Biliary/drug therapy , Aged , Treatment Outcome , Alkaline Phosphatase/blood , Cholagogues and Choleretics/therapeutic use , Fibric Acids/therapeutic use , Spain , Bilirubin/blood , AdultABSTRACT
Hemangiomas are benign vascular gastrointestinal tumours. They are infrequent, located in the submucosa, and can be asymptomatic or cause intestinal obstruction, anaemia or ferropenia. Their initial diagnosis is usually endoscopic despite the absence of pathognomonic features. Traditionally their treatment has been surgical although endoscopic resection is now possible. We present a case of ferropenia resolved after endoscopic resection of a capillary duodenal hemangioma.