Quality of Life

ONPATTRO^® (patisiran) significantly improved quality of life¹

Key Secondary Endpoint: ^1,2

Change from baseline at 18 months vs placebo in Norfolk Quality of Life-Diabetic Neuropathy (QoL-DN)
Norfolk QoL-DN score is a patient-reported assessment that evaluates neuropathy in domains such as physical function, activities of daily living, symptoms, and autonomic neuropathy (score ranges from -4 to 136)

Change in Norfolk QoL-DN score^1,a-c

Change in Norfolk QoL-DN score from baseline at 18 months

^aNorfolk QoL-DN scores at baseline were 59.6 with ONPATTRO and 55.5 with placebo.¹ ^bBars represent SEM. ^cN=number of evaluable patients. ^d95% CI: -19.8, -10.2.¹ ^e95% CI: -27.2, -15.0.¹

CI=confidence interval; LS=least squares; SEM=standard error of the mean.

Improvement in quality of life from baseline at 18 months^3,f,g

Improvement in quality of life from baseline^3,f,g

51% ONPATTRO®-treated patients experienced improvement in QoL from baseline at 18 months vs 10% of placebo-treated patients

From New England Journal of Medicine, Adams D, Gonzalez-Duarte A, O’Riordan WD, et al, "Patisiran, an RNAi Therapeutic, for Hereditary Transthyretin Amyloidosis," 379(1). Copyright © 2018 Massachusetts Medical Society. Reprinted with permission from Massachusetts Medical Society.

^fOdds ratio: 10.0 (4.4-22.5).³
^gN=number of evaluable patients.
^h95% CI: 43, 59.³
ⁱ95% CI: 4, 17.³

More than half of patients treated with ONPATTRO
experienced improvement in quality of life³

51% of patients treated with ONPATTRO experienced improvement
in quality of life compared to 10% of patients treated with placebo
Improvements relative to placebo were observed across all domains
of Norfolk QoL-DN

The benefit of ONPATTRO on quality of life was observed across all subgroups, including age, sex, mutation status, previous tetramer stabilizer use, and disease stage.

ONPATTRO demonstrated significant improvement in quality of life as measured by physical, functional, and social assessments.^1,4

Get started with ONPATTRO now »

NEXT: View the benefit in
measures of disease burden

References:

ONPATTRO [package insert]. Cambridge, MA: Alnylam Pharmaceuticals, Inc; 2018.
Vinik EJ, Vinik AI, Paulson JF, et al. J Peripher Nerv Syst. 2014;19:104-119.
Adams D, Gonzalez-Duarte A, O’Riordan WD, et al. N Engl J Med. 2018;279(1):11-21.
Vinik EJ, Hayes RP, Oglesby A, et al. Diabetes Technol Ther. 2005;7(3):497-508.

Infusion-Related Reactions

Infusion-related reactions (IRRs) have been observed in patients treated with ONPATTRO^® (patisiran). In a controlled clinical study, 19% of ONPATTRO-treated patients experienced IRRs, compared to 9% of placebo-treated patients. The most common symptoms of IRRs with ONPATTRO were flushing, back pain, nausea, abdominal pain, dyspnea, and headache.

To reduce the risk of IRRs, patients should receive premedication with a corticosteroid, acetaminophen, and antihistamines (H1 and H2 blockers) at least 60 minutes prior to ONPATTRO infusion. Monitor patients during the infusion for signs and symptoms of IRRs. If an IRR occurs, consider slowing or interrupting the infusion and instituting medical management as clinically indicated. If the infusion is interrupted, consider resuming at a slower infusion rate only if symptoms have resolved. In the case of a serious or life-threatening IRR, the infusion should be discontinued and not resumed.

Reduced Serum Vitamin A Levels and Recommended Supplementation

ONPATTRO treatment leads to a decrease in serum vitamin A levels. Supplementation at the recommended daily allowance (RDA) of vitamin A is advised for patients taking ONPATTRO. Higher doses than the RDA should not be given to try to achieve normal serum vitamin A levels during treatment with ONPATTRO, as serum levels do not reflect the total vitamin A in the body.

Patients should be referred to an ophthalmologist if they develop ocular symptoms suggestive of vitamin A deficiency (e.g. night blindness).

Infusion-Related Reactions

Reduced Serum Vitamin A Levels and Recommended Supplementation

Patients should be referred to an ophthalmologist if they develop ocular symptoms suggestive of vitamin A deficiency (e.g. night blindness).

Questions?
Call 1-833-256-2748

ONPATTRO^® (patisiran) significantly improved quality of life¹

Key Secondary Endpoint: ^1,2

Change in Norfolk QoL-DN score^1,a-c

Improvement in quality of life from baseline at 18 months^3,f,g

Improvement in quality of life from baseline^3,f,g

More than half of patients treated with ONPATTRO
experienced improvement in quality of life³

Sample questions from the questionnaire⁴:

Important Safety Information

Infusion-Related Reactions

Reduced Serum Vitamin A Levels and Recommended Supplementation

Adverse Reactions

Indication

Important Safety Information

Infusion-Related Reactions

Reduced Serum Vitamin A Levels and Recommended Supplementation

Adverse Reactions

Indication

ONPATTRO® (patisiran) significantly improved quality of life1

Key Secondary Endpoint: Norfolk QoL-DN score1,2

Change in Norfolk QoL-DN score1,a-c

Improvement in quality of life from baseline at 18 months3,f,g

Improvement in quality of life from baseline3,f,g

More than half of patients treated with ONPATTRO experienced improvement in quality of life3

Important Safety Information

Infusion-Related Reactions

Reduced Serum Vitamin A Levels and Recommended Supplementation

Adverse Reactions

Indication

Important Safety Information

Infusion-Related Reactions

Reduced Serum Vitamin A Levels and Recommended Supplementation

Adverse Reactions

Indication

ONPATTRO^® (patisiran) significantly improved quality of life¹

Key Secondary Endpoint: ^1,2

Change in Norfolk QoL-DN score^1,a-c

Improvement in quality of life from baseline at 18 months^3,f,g

Improvement in quality of life from baseline^3,f,g

More than half of patients treated with ONPATTRO
experienced improvement in quality of life³