Impact of Baseline Polyneuropathy Severity on Eplontersen Efficacy in the NEURO-TTRansform Clinical Trial.
Thomas H Brannagan, Markus Weiler, Márcia Waddington Cruz et al.
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In 30 seconds
This post hoc analysis of the NEURO-TTRansform trial evaluated the effect of baseline severity of hereditary transthyretin amyloidosis with polyneuropathy (ATTRv-PN) on the efficacy of eplontersen. Results showed that mean modified Neuropathy Impairment Score (mNIS+7) changes from baseline were -4.5 for the least impaired group, -1.3 for moderate impairment, and -2.6 for the most impaired group, indicating sustained benefits across severity levels.
Key findings
- Mean mNIS+7 changes from baseline were -4.5, -1.3, and -2.6 for T1, T2, and T3, respectively.
- Patients receiving placebo experienced worsening across all assessed outcomes.
- Eplontersen provided consistent benefits regardless of baseline ATTRv-PN severity.
- Early treatment initiation is emphasized for patients across the disease spectrum.
Why it matters
Understanding the impact of baseline severity on treatment response is crucial for optimizing management strategies in patients with ATTRv-PN. This study highlights the potential for eplontersen to benefit patients at various stages of the disease.