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  • A new and simple score to p...
    Cançado, Guilherme Grossi Lopes; Gomes, Nathalia Mota de Faria; Couto, Cláudia Alves; Cançado, Eduardo Luiz Rachid; Terrabuio, Debora Raquel Benedita; Villela-Nogueira, Cristiane Alves; Braga, Michelle Harriz; Nardelli, Mateus Jorge; Faria, Luciana Costa; Oliveira, Elze Maria Gomes; Rotman, Vivian; Oliveira, Maria Beatriz; Cunha, Simone Muniz Carvalho Fernandes da; Mazo, Daniel Ferraz de Campos; Mendes, Liliana Sampaio Costa; Ivantes, Claudia Alexandra Pontes; Codes, Liana; Borges, Valéria Ferreira de Almeida E; Pace, Fabio Heleno de Lima; Pessôa, Mário Guimarães; Signorelli, Izabelle Venturini; Coral, Gabriela Perdomo; Bittencourt, Paulo Lisboa; Fucuta, Patrícia; Filho, Roberto José de Carvalho; Ferraz, Maria Lucia Gomes

    European journal of gastroenterology & hepatology, 2024-May-01, 2024-05-00, 20240501, Volume: 36, Issue: 5
    Journal Article

    Ursodeoxycholic acid (UDCA) is the standard treatment for primary biliary cholangitis (PBC), but a significant proportion of patients do not respond adequately, leading to increased risk of adverse outcomes. This study aims to develop a new and straightforward predictive score to identify PBC patients likely to achieve a complete response to UDCA. A logistic regression analysis was conducted using a derivation cohort of PBC patients to identify pre-treatment variables associated with response to UDCA. This analysis led to the development of the ALP-A score, calculated as: Age at diagnosis divided by (alkaline phosphatase at diagnosis/upper limit of normal). ALP-A score accuracy was evaluated using the area under the ROC curve, validated with a large external cohort from Brazil. Additionally, the correlation between the ALP-A score and the previously validated UDCA response score (URS) was assessed. ALP-A score had good predictive power for adequate (AUC 0.794; 95% CI, 0.737-0.852) and deep (0.76; 95% CI, 0.69-0.83) UDCA response at 1 year of treatment. A cutoff score of 17 and 23 points was determined to be the optimal threshold for distinguishing adequate and deep responders, respectively, from non-responders. ALP-A score demonstrated a sensitivity of 73%, specificity of 71%, positive predictive value of 65%, negative predictive value of 78%, and overall accuracy of 72% for biochemical response. The URS displayed similar discriminative ability (AUC 0.798; 95% CI, 0.741-0.855). ALP-A score performs comparably to URS but offers the great advantage of simplicity for routine clinical use. It serves as a valuable tool to identify PBC patients less likely to respond to UDCA treatment, facilitating early consideration of alternative therapeutic approaches.