‘Number to treat’ with UDCA may improve survival rates in PBC

While the absolute clinical efficacy of ursodeoxycholic acid for primary biliary cholangitis varied by baseline prognostic characteristics, study results showed generally high rates of liver transplant-free survival and overall survival.

“These results provide a clear understanding of the clinical importance of optimized UDCA therapy for patients and doctors, thereby stimulating compliance and treatment uptake,” Maren H. Harms, PhD student, from Erasmus University Medical Center in the Netherlands, and colleagues wrote.

Harms and colleagues designed this study to determine the number needed to treat (NNT) or how many patients with PBC should be treated with UDCA to prevent one event of LT or death with clear interpretation for physicians, patients and policymakers.

The study comprised 3,529 patients treated with UDCA and 373 patients not treated. During a median follow-up of 7.8 years (range, 4.1-12.1), 306 patients were lost to follow-up, 299 underwent LT, and 567 patients died.

The 5-year cumulative LT-free survival without UDCA therapy was 81% (95% CI, 79.3-82.7) and the overall adjusted HR for LT or death was 0.46 (95% CI, 0.4-0.52). Therefore, the NNT to prevent LT or death within 5 years in one patient was 11 (95% CI, 9-13).

“The HR of UDCA for LT or death was stable over the baseline characteristics and only differed statistically significantly among patients stratified according to their baseline age and [alkaline phosphatase] and albumin levels,” the researchers wrote.

Among patients with an ALP less than 1.67 times the upper limit of normal, the median GLOBE score prior to UDCA treatment was 0.0266. This translated to a 5-year LT-free survival rate of 94% and 10-year LT-free survival rate of 84.7%. As a result, the NNT to prevent one LT or death was 26 (95% CI, 24-29) for 5 years and 11 (95% CI, 10-12) for 10 years.

In contrast, the median GLOBE score prior to UDCA treatment among patients with a suboptimal biochemical response was 0.6978 for estimated LT-free survival rates of 88% for 5 years and 70.9% for 10 years. The NNT to prevent LT or death within 5 years among this patient group was 15 (95% CI, 14-18), and 7 (95% CI, 6-8) for 10 years.

“While policymakers may be interested in long-term effects of therapy, patients are more likely to prioritize short-term benefits,” Harms and colleagues wrote. “Moreover, physicians’ willingness to treat is reported to be dependent of the measure in which treatment benefit is presented. Providing information on both relative and absolute clinical efficacy may therefore prevent misinterpretation and aid well-informed decision making in daily clinical practice.” – by Talitha Bennett

Disclosures: Harms reports a speaker fee from Zambon Nederland. Please see the full report for all other authors’ relevant financial disclosures.