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Background Cholangiocarcinoma is an epithelial malignancy of the intrahepatic or extrahepatic biliary tree, primarily driven by chronic inflammation and fibrosis. Fibrosis has been shown to correlate with malignancy, and the aminotransferase-platelet ratio index (APRI) score, a marker for hepatic fibrosis, has proved useful in prognosticating hepatocellular carcinoma. This study aimed to assess the utility of APRI score in predicting post-surgical outcomes in cholangiocarcinoma patients. Methods Clinical data from a total of 152 cholangiocarcinoma patients who underwent surgical resection at the Mayo Clinic were collected. The data were subsequently analyzed to determine if there was a relationship between APRI score and the demographic, laboratory, pathologic and outcome data, including overall survival. To determine the relationship between quantitative and qualitative data and the APRI score, a P-value <0.05 was considered as statistically significant. Results No relationship between APRI score and demographic factors was identified. There were correlations between APRI score and alanine transaminase, albumin and bilirubin, but the remaining laboratory parameters showed no correlation. APRI score did not prove to be useful as a prognostic tool, as it did not correlate with tumor pathology features (tumor grade t-test P=0.86, N stage ANOVA P=0.94, vascular invasion t-test P=0.59, and perineural invasion t-test P=0.14), or with post-surgical recurrence (t-test P=0.22) and mortality (t-test P=0.39). Conclusion APRI score is not a prognostic tool for post-surgical outcomes in patients with cholangiocarcinoma. [ABSTRACT FROM AUTHOR]

Myotonic dystrophy type 1 (DM1) is a CTG microsatellite expansion (CTGexp) disorder caused by expression of CUGexp RNAs. These mutant RNAs alter the activities of RNA processing factors, including MBNL proteins, leading to re-expression of fetal isoforms in adult tissues and DM1 pathology. While this pathogenesis model accounts for adult-onset disease, the molecular basis of congenital DM (CDM) is unknown. Here, we test the hypothesis that disruption of developmentally regulated RNA alternative processing pathways contributes to CDM disease. We identify prominent alternative splicing and polyadenylation abnormalities in infant CDM muscle, and, although most are also misregulated in adult-onset DM1, dysregulation is significantly more severe in CDM. Furthermore, analysis of alternative splicing during human myogenesis reveals that CDM-relevant exons undergo prenatal RNA isoform transitions and are predicted to be disrupted by CUGexp-associated mechanisms in utero. To test this possibility and the contribution of MBNLs to CDM pathogenesis, we generated mouse Mbnl double (Mbnl1; Mbnl2) and triple (Mbnl1; Mbnl2; Mbnl3) muscle-specific knockout models that recapitulate the congenital myopathy, gene expression, and spliceopathy defects characteristic of CDM. This study demonstrates that RNA misprocessing is a major pathogenic factor in CDM and provides novel mouse models to further examine roles for cotranscriptional/post-transcriptional gene regulation during development. [ABSTRACT FROM AUTHOR]