Is patient-reported outcome improved by nalfurafine hydrochloride in patients with primary biliary cholangitis and refractory pruritus? A post-marketing, single-arm, prospective study
- 8 Downloads
Patients with primary biliary cholangitis (PBC) frequently suffer from pruritus, which can severely impair their health-related quality of life (HRQOL). Nalfurafine hydrochloride, a selective κ-opioid receptor agonist, was recently approved in Japan for refractory pruritus in patients with chronic liver diseases, but it still remains unclear whether this treatment improves the patient-reported outcome (PRO) in PBC patients with refractory pruritus. Herein, we conducted a multicenter, post-marketing, single-arm prospective study to investigate the efficacy of nalfurafine in terms of PRO, and the associations of the efficacy with any clinical characteristics.
After screening for pruritus in 496 patients with PBC using PBC-40 and the visual analog scale (VAS), we identified 141 patients with moderate to severe pruritus; these were invited to participate in the study. The participants received 2.5 μg nalfurafine once daily for 12 weeks, and pruritus and HRQOL were assessed in week 12 of this treatment. Generic HRQOL, short form 36, blood chemistries, and serum autotaxin levels were also measured at baseline and at week 12.
Forty-four patients participated in this study. The mean PBC-40 itch domain scores and VAS declined during the study period, from 8.56 to 7.63 (P = 0.041) and from 42.9 to 29.3 (P = 0.001) at baseline and at week 12, respectively, indicating a significant effect of nalfurafine. The other domains of PBC-40 and all domains of SF-36 were not significantly altered by this treatment. We failed to find any association between the change in VAS and PBC-40 itch scores and any clinical variable. Serum autotaxin levels were significantly increased during the study period.
This study demonstrated that nalfurafine improved pruritus in patients with PBC, independent of their clinical characteristics, but had a limited effect on the PRO.
KeywordsPBC-40 SF-36 Opioid Autotaxin Health-related quality of life
Primary biliary cholangitis
Health-related quality of life
Visual analog scale
Short form 36
We are sincerely grateful to all of the patients who participated in this study. Also, we sincerely appreciate the secretarial assistance of Ms. Kayono Unno and Ms. Kanako Iwai.
A.T. and M.Y. designed the study. T.N., A.T., M.A., A.H., Y.M., H.O., H.Y., and H.T. invited participants and collected clinical data. A.T., Y.N., and H.T. analyzed and interpreted the data. A.T. and M.Y. drafted the paper; all authors critically reviewed the manuscript.
Compliance with ethical standards
Conflict of interest
AT received consultant fees from EA Pharma and GlaxoSmithKline. YM received lecture fees from Mitsubishi Tanabe Pharma, MSD K.K., AbbVie GK, Gilead Science, Janssen Pharmaceutical K.K. and commercial research findings from MSD K.K., AbbVie GK, Gilead Science, and Nobelpharma, Eisai Co.
- 10.Working Subgroup for Clinical Practice Guidelines for Primary Biliary Cirrhosis. Guidelines for the management of primary biliary cirrhosis: the intractable hepatobiliary disease Study Group supported by the Ministry of Health, Labour and Welfare of Japan. Hepatol Res. 2014;44(Suppl S1):71–90.CrossRefGoogle Scholar
- 17.Yagi M, Tanaka A, Abe M, et al. Symptoms and health-related quality of life in Japanese patients with primary biliary cholangitis. Submitted.Google Scholar
- 20.Ware JEJ. The SF-36 health survey. In: Spilker B, editor. Quality of life and pharmacoeconomics in clinical trials. 2nd ed. Philadelphia: Lippincott-Raven; 1996.Google Scholar