Indirect treatment comparison (ITC) of the efficacy of vutrisiran and tafamidis for hereditary transthyretin-mediated amyloidosis with polyneuropathy.

Abstract

BACKGROUND:

Vutrisiran and tafamidis are approved therapies for treating hereditary transthyretin-mediated (ATTRv/hATTR) amyloidosis with polyneuropathy, a rapidly progressive and fatal disease. To assist healthcare decision-makers, an indirect treatment comparison (ITC) was undertaken to explore the comparative efficacy of vutrisiran and tafamidis.

RESEARCH DESIGN AND METHODS:

Individual patient data (vutrisiran vs. placebo) and published results (tafamidis vs. placebo) from phase 3 randomized controlled trials were used in a Bucher analysis to assess differences in treatment effects between vutrisiran and tafamidis on: Neuropathy Impairment Score-Lower Limbs (NIS-LL), Norfolk Quality of Life-Diabetic Neuropathy (Norfolk QOL-DN) score, NIS-LL Response, and modified Body Mass Index (mBMI).

RESULTS:

Greater treatment effects were observed at 18 months with vutrisiran vs. tafamidis for all endpoints, with statistically significant improvements in polyneuropathy (relative mean change in NIS-LL: -5.3 [95% confidence interval (CI): -9.4, -1.2; p = 0.011]), health-related quality of life (HRQOL, relative mean change in Norfolk QOL-DN: -18.3 [95% CI: -28.6, -8.0; p < 0.001]), and nutritional status (relative mean change in mBMI: 63.9 [95% CI: 10.1, 117.7; p = 0.020]).

CONCLUSIONS:

This analysis suggests vutrisiran has greater efficacy on multiple measures of polyneuropathy impairment and HRQOL compared to tafamidis in patients with ATTRv amyloidosis with polyneuropathy.

• Authors: Madeline Merkel, David Danese, Chongshu Chen, Jessie Wang, Aozhou Wu, Hongbo Yang, Hollis Lin
• Journal: Expert opinion on pharmacotherapy (Expert Opin Pharmacother) 2023 May-Aug Vol. 24 Issue 10 Pg. 1205-1214 ISSN: 1744-7666 [Electronic] England
• PMID: 37219406 (Publication Type: Journal Article)

Chemical References

• tafamidis
• Prealbumin

Topics

• Humans
• Quality of Life
• Prealbumin (therapeutic use)
• Amyloid Neuropathies, Familial (complications, drug therapy)
• Polyneuropathies (drug therapy)