Pruritus Experience in Patients with PBC Treated with Obeticholic Acid Through 6 Years: Patient-Reported Quality of Life

Question Obeticholic acid (OCA) is approved as second-line treatment for PBC in patients with an incomplete response or intolerance to ursodeoxycholic acid. We evaluated the impact of OCA on patient-reported outcome measures (PROs) of pruritus through 6 years.

Methods POISE was a randomized, double-blind (DB) phase 3 study evaluating OCA 5–10 mg or 10 mg vs placebo over 12 months. A 5-year open-label extension (OLE) followed during which patients received OCA 5‒10 mg, or up-titrated to OCA 25 mg. PROs of pruritus were assessed every 3 months: pruritus severity visual analogue scale (VAS; 0 [none]–100 [severe]), 5-D pruritus questionnaire (5-D; 5 [none]–25 [severe]) and PBC-40 itch domain (0 [none]–15 [severe]). Pruritus was managed with concomitant medications, temporary OCA dose interruption or OCA dose reduction.

Results Median (Q1, Q3) scores on the pruritus severity VAS were not significantly different in the OCA 5‒10 mg group vs placebo and remained in the mild-to-moderate range in the DB phase. VAS scores in the OCA 10 mg groups were higher than placebo at DB month 3 but similar across groups by DB month 12 (Figure). Similar findings were observed during the DB phase for the 5-D and PBC-40 itch median scores. Median VAS (Figure), 5-D, and PBC-40 itch scores remained stable through the 5-year OLE phase. All VAS scores remained in the mild-to-moderate category.

Conclusions In POISE, with effective management and correct dose titration, a subset of patients experienced consistently mild pruritus with little impact on quality of life, which was sustained through the OLE phase.

Publication History

Article published online:
04 January 2021