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Impact of baseline polyneuropathy severity on patisiran treatment outcomes in the APOLLO trial.
Quan, Dianna; Obici, Laura; Berk, John L; Ando, Yukio; Aldinc, Emre; White, Matthew T; Adams, David.
Affiliation
  • Quan D; Department of Neurology, University of Colorado Anschutz, Aurora, CO, USA.
  • Obici L; Amyloidosis Research and Treatment Centre, Fondazione IRCCS Policlinico San Matteo, Pavia, Italy.
  • Berk JL; Amyloidosis Center, Boston Medical Center, Boston, MA, USA.
  • Ando Y; Department of Neurology, Graduate School of Medical Sciences, Kumamoto University, Kumamoto, Japan.
  • Aldinc E; Alnylam Pharmaceuticals, Cambridge, MA, USA.
  • White MT; Alnylam Pharmaceuticals, Cambridge, MA, USA.
  • Adams D; Neurology Department, APHP, CHU Bicêtre, Université Paris-Saclay, U1195, INSERM, Neurology Department, AP-HP, CHU Bicêtre, Le Kremlin Bicêtre, France.
Amyloid ; 30(1): 49-58, 2023 Mar.
Article in En | MEDLINE | ID: mdl-36120830
ABSTRACT

OBJECTIVE:

Assess how baseline polyneuropathy severity impacts response to patisiran regarding neurologic impairment and quality of life (QOL) in patients with hereditary transthyretin-mediated amyloidosis (ATTRv amyloidosis).

METHODS:

This post hoc analysis grouped patients from the Phase 3 APOLLO study (n = 225) by baseline Neuropathy Impairment Score (NIS) into quartiles 6-<31; 31-<57; 57-<85.5; 85.5-141.6. Neurologic impairment (modified NIS+7 [mNIS+7], NIS total score), disability (Rasch-built Overall Disability Scale [R-ODS]), gait speed (10-meter walk test [10-MWT]), grip strength, and QOL (Norfolk Quality of Life-Diabetic Neuropathy [Norfolk QOL-DN] questionnaire) were assessed.

RESULTS:

Across all baseline NIS quartiles, patisiran improved several clinical markers of disease compared with placebo at 18 months. Patients in lower NIS quartiles, treated with patisiran earlier in the disease course, maintained better scores in mNIS+7, NIS total score, R-ODS, 10-MWT, grip strength, and Norfolk QOL-DN versus those in higher NIS quartiles, while placebo-treated patients experienced worsening of all functional measures after 18 months across all quartiles.

CONCLUSIONS:

Patisiran treatment improved neurologic function and QOL across a wide range of baseline polyneuropathy severities versus placebo. Timing of treatment initiation in patients with ATTRv amyloidosis remains critical for the preservation of function.(ClinicalTrials.gov number, NCT01960348).
Subject(s)
Key words

Full text: 1 Collection: 01-internacional Database: MEDLINE Main subject: Polyneuropathies / Amyloid Neuropathies, Familial Type of study: Clinical_trials Aspects: Patient_preference Limits: Humans Language: En Journal: Amyloid Journal subject: BIOQUIMICA Year: 2023 Document type: Article Affiliation country:

Full text: 1 Collection: 01-internacional Database: MEDLINE Main subject: Polyneuropathies / Amyloid Neuropathies, Familial Type of study: Clinical_trials Aspects: Patient_preference Limits: Humans Language: En Journal: Amyloid Journal subject: BIOQUIMICA Year: 2023 Document type: Article Affiliation country: