{Reference Type}: Journal Article
{Title}: A new and simple score to predict adequate and deep response to ursodeoxycholic acid in patients with primary biliary cholangitis: the ALP-A score.
{Author}: Cançado GGL;Gomes NMF;Couto CA;Cançado ELR;Terrabuio DRB;Villela-Nogueira CA;Braga MH;Nardelli MJ;Faria LC;Oliveira EMG;Rotman V;Oliveira MB;Cunha SMCFD;Mazo DFC;Mendes LSC;Ivantes CAP;Codes L;Borges VFAE;Pace FHL;Pessôa MG;Signorelli IV;Coral GP;Bittencourt PL;Fucuta P;Filho RJC;Ferraz MLG;
{Journal}: Eur J Gastroenterol Hepatol
{Volume}: 36
{Issue}: 5
{Year}: 2024 May 1
{Factor}: 2.586
{DOI}: 10.1097/MEG.0000000000002744
{Abstract}: BACKGROUND: 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.
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).
CONCLUSIONS: 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.