A new and simple score to predict adequate and deep response to ursodeoxycholic acid in patients with primary biliary cholangitis: the ALP-A score.
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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
Аннотация
METHODS: 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.
RESULTS: 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).
CONCLUSION: 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.