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Table 4 Changes in clinical assessments at 24 months with patisiran treatment

From: A phase II, open-label, extension study of long-term patisiran treatment in patients with hereditary transthyretin-mediated (hATTR) amyloidosis

Assessment Change from baseline to Month 24
n Mean (SEM)
EQ-5D (max. impairment: 0) 26 −0.01 (0.02)
R-ODS (no limitations: 48) 25 −1.8 (0.8)
10-m walk (m/s) 21 0.03 (0.04)
Hand grip strength (kg) 26 1.5 (1.2)
mBMI (kg/m2 × g/L) 22 −60.8 (34.9)
COMPASS-31 (max. impairment: 100) 26 1.3 (1.8)
Cardiac subgroup (n = 11)
 NT-proBNP (pg/mL) 8 −49.6 (170.8)
 Troponin I (ng/mL) 8 −0.1 (0.1)
 LV mass (g) 10 −16.7 (11.7)
 LV wall thickness (cm) 10 −0.08 (0.1)
 Ejection fraction (%) 10 −0.6 (1.5)
 Peak longitudinal strain (%) 10 0.9 (0.9)
 10-m walk (m/s) 7 0.03 (0.05)
  1. Abbreviations: EQ-5D EuroQoL 5-dimensions questionnaire, COMPASS Composite Autonomic Symptom Score, LV left ventricular, m meter, mBMI modified body mass index, NT-proBNP N-terminal pro-brain natriuretic peptide, QOL quality of life, R-ODS Rasch-built Overall Disability Scale