Is patient-reported outcome improved by nalfurafine hydrochloride in patients with primary biliary cholangitis and refractory pruritus? A post-marketing, single-arm, prospective study.

Abstract

BACKGROUND:

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.

METHODS:

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.

RESULTS:

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.

CONCLUSIONS:

This study demonstrated that nalfurafine improved pruritus in patients with PBC, independent of their clinical characteristics, but had a limited effect on the PRO.

• Authors: Minami Yagi, Atsushi Tanaka, Tadashi Namisaki, Atsushi Takahashi, Masanori Abe, Akira Honda, Yasushi Matsuzaki, Hiromasa Ohira, Hitoshi Yoshiji, Hajime Takikawa, Japan PBC Study Group (JPBCSG)
• Journal: Journal of gastroenterology (J Gastroenterol) Vol. 53 Issue 10 Pg. 1151-1158 (Oct 2018) ISSN: 1435-5922 [Electronic] Japan
• PMID: 29663077 (Publication Type: Journal Article, Multicenter Study)

Chemical References

• Analgesics, Opioid
• Biomarkers
• Morphinans
• Receptors, Opioid, kappa
• Spiro Compounds
• TRK 820
• Phosphoric Diester Hydrolases
• alkylglycerophosphoethanolamine phosphodiesterase

Topics

• Aged
• Analgesics, Opioid (administration & dosage, therapeutic use)
• Biomarkers (blood)
• Female
• Follow-Up Studies
• Humans
• Japan
• Liver Cirrhosis, Biliary (complications)
• Male
• Middle Aged
• Morphinans (administration & dosage, therapeutic use)
• Patient Reported Outcome Measures
• Phosphoric Diester Hydrolases (blood)
• Prospective Studies
• Pruritus (blood, drug therapy, etiology)
• Quality of Life
• Receptors, Opioid, kappa (agonists)
• Spiro Compounds (administration & dosage, therapeutic use)
• Statistics, Nonparametric
• Treatment Outcome