| [Introduction] Although ursodeoxycholic acid (UDCA) is a first-line treatment in patients with primary biliary cholangitis (PBC), 30% of patients with PBC exhibit incomplete response to UDCA. Recently Carbone et al.demonstrated the UDCA Response Score, which predict UDCA response using pre-treatment parameters of UK PBC patients (derivation cohort, n=2703; validation cohort, n=460). In the current study, we validated the UDCA Response Score in Japanese PBC patients. [Patients and Methods] We took advantage of PBC patients’ registry data in Japan, (n=890). Among this registry, we selected patients who provided all clinical parameters required for calculation of the UDCA Response Score. The endpoint was UDCA response, defined as ALP less than 1.67 times the upper limit of normal (ULN) after 12 months of treatment with UDCA. When bezafibrate was commenced within 12 months of UDCA treatment, we defined the endpoint was not met. [Results] Among 890 patients in the Japanese cohort, all parameters were available in 804 patients (male/female=120/684, age 58.9+/-11.0).The endpoint was achieved in 190 (1.67 xULN, 24%). When the endpoint was defined as 1.67 xULN, AUROC was 0.75 (95% CI 0.71-0.80), which was 0.83 (95%CI 0.79-0.87) in the original publication.[Conclusion] The validity of the UDCA Response Score was acceptable but slightly lower in Japanese cohort, presumably because of early intervention with bezafibrate before judgement of UDCA response in Japanese clinical practice. |
