Liver and Bile

Eur J Gastroenterol Hepatol. 2024;36(5):628–35

Grossi Lopes Cançado G, Mota de Faria Gomes N, Alves Couto C, Cançado ELR, Terrabuio DRB, Alves Villela-Nogueira C, Harriz Braga M, Nardelli MJ, Costa Faria L, Gomes Oliveira EM, Rotman V, Oliveira MB, Muniz Carvalho Fernandes da Cunha S, Ferraz de Campos Mazo D, Sampaio Costa Mendes L, Pontes Ivantes CA, Codes L, Ferreira de Almeida e Borges V, de Lima Pace FH, Guimarães Pessôa M, Venturini Signorelli I, Perdomo Coral G, Lisboa Bittencourt P, Fucuta P, de Carvalho Filho RJ, Gomes Ferraz ML

A new and simple score to predict adequate and deep response to ursodeoxycholic acid in patients with primary biliary cholangitis: The ALP-A score

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 pretreatment 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 [ALP] at diagnosis/upper limit of normal). ALP-A score accuracy was evaluated using the area under the receiver-operating characteristic 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: The ALP-A score had good predictive power for adequate (area under the curve [AUC], 0.794; 95% confidence interval [CI]: 0.737–0.852) and deep (AUC, 0.76; 95% CI: 0.69–0.83) UDCA response at 1 year of treatment. A cut-off 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: The ALP-A score performs comparably to the ursodeoxycholic acid (UDCA) response score but offers the great advantage of simplicity for routine clinical use. It serves as a valuable tool to identify primary biliary cholangitis patients less likely to respond to UDCA treatment, facilitating early consideration of alternative therapeutic approaches

DOI: 10.1097/meg.0000000000002744