Autonomic symptoms: 1,2,a
- COMPASS 31 is a 31-item questionnaire that evaluates autonomic function across 6 domains: orthostatic intolerance, vasomotor, secretomotor, gastrointestinal, bladder, and pupillomotor (score range 0 to 100)2,3
- Patients receiving ONPATTRO® (patisiran) experienced a significant benefit relative to placebo in reduction of autonomic symptoms
—In ONPATTRO-treated patients, improvement compared to baseline was seen for the orthostatic intolerance and gastrointestinal domains, the 2 domains that contribute most to the overall COMPASS 31 score4
Change in COMPASS 31 score from baseline at 18 monthsb-d
aCOMPASS 31 scores at baseline were 30.6 with ONPATTRO and 30.3 with placebo.1bONPATTRO-placebo treatment difference (LS mean): -7.53.5 c95% CI: -11.9, -3.2.5 dNumber of patients assessed at 18 months was 136 with ONPATTRO and 53 with placebo.1
CI=confidence interval; COMPASS 31=Composite Autonomic Symptom Score 31; LS=least squares.
Activities of daily living: 1,2,e
- R-ODS is a 24-item scale that evaluates limitations on everyday activity (score range 0 to 48)
- Patients receiving ONPATTRO saw a significant benefit relative to placebo in the ability to perform activities of daily living and everyday function
Change in R-ODS score from baseline at 18 monthsf-h
eR-ODS scores at baseline were 29.7 with ONPATTRO and 29.8 with placebo.1fONPATTRO-placebo treatment difference (LS mean): 9.0.5 g95% CI: 7.0, 10.9.5 hNumber of patients assessed at 18 months was 138 with ONPATTRO and 54 with placebo.1
CI=confidence interval; R-ODS=Rasch-built Overall Disability Scale; LS=least squares.
ONPATTRO improved other key
measures of disease burden1,5,6
- Gait speed measured by 10-meter walk test (10MWT)
- 53% of patients treated with ONPATTRO showed an improvement in gait speed (change >0) at 18 months, compared to 13% of patients treated with placebo
- Separation from placebo was seen in the ONPATTRO-treated patients as early as 3 months
- ONPATTRO-treated patients showed improvement at 18 months in these domains of the Euro Quality of Life 5 Dimensions 5 Levels (EQ-5D-5L) instrument compared to placebo-treated patients
- EQ-5D-5L was an exploratory endpoint in the APOLLO study
aONPATTRO-placebo treatment difference (LS mean): 0.31.6 b95% CI: 0.23, 0.39.6 cONPATTRO-placebo treatment difference (LS mean): 116.6 d95% CI: 82, 149.6 eONPATTRO-placebo treatment difference (LS mean): -17.9.5 f95% CI: -22.3, -13.4.5
CI=confidence interval; LS=least squares.