
about family history and red-flag symptoms
Hereditary transthyretin-mediated (hATTR) amyloidosis is a debilitating disease with a median survival of 4.7 years after diagnosis. Obtaining a complete family history is an integral step toward recognizing and accelerating the diagnosis of this inherited disease.1-3
To enable a timely diagnosis of hATTR amyloidosis, it is critical to ask patients about their complete clinical history, including symptoms of the most common manifestations of the disease.2-4

Sensory-motor neuropathy
Typically length-dependent, symmetric, small-fiber neuropathy symptoms that progress over time
- Neuropathic pain
- Paresthesia
- Bilateral carpal tunnel syndrome
- Weakness
- Loss of fine motor control
- Difficulty walking

Autonomic neuropathy
May affect a variety of organ systems including cardiovascular, gastrointestinal, and genitourinary systems
- Orthostatic hypotension
- Abnormal sweating
- Nausea and vomiting
- Constipation and/or diarrhea
- Urinary and/or fecal incontinence
- Early satiety and wasting
- Sexual dysfunction

Cardiac manifestations
May include conduction abnormalities, arrhythmias, heart failure
Rapid worsening of these signs and symptoms should also alert you to the possibility of hATTR amyloidosis.2

the evidence of disease through diagnostic tools

IDENTIFY
When you identify a patient who may have hATTR amyloidosis, you can choose from a number of different tests that can help assist in the assessment and diagnosis of hATTR amyloidosis2,4-6

CONFIRM
Several tests are available to help confirm the presence of amyloid deposits and to help make a diagnosis of hATTR amyloidosis, including genetic testing, scintigraphy, and tissue biopsy3,4,6
IDENTIFY4-8 | |
---|---|
Electromyography (EMG) |
Electromyography (EMG)
|
Nerve conduction study (NCS) |
Nerve conduction study (NCS)
|
Quantitative sudomotor axon reflex test (QSART) |
Quantitative sudomotor axon reflex test (QSART)
|
Sympathetic skin response (SSR) |
Sympathetic skin response (SSR)
|
Heart rate deep breathing |
Heart rate deep breathing
|
Tilt table |
Tilt table
|
Valsalva maneuver |
Valsalva maneuver
|
Echocardiography |
Echocardiography
|
Electrocardiography (ECG) |
Electrocardiography (ECG)
|
Cardiac magnetic resonance imaging (CMRI) |
Cardiac magnetic resonance imaging (CMRI)
|
CONFIRM3,4,6 | |
---|---|
Genetic testing |
Genetic testing
|
Scintigraphy |
Scintigraphy
|
Tissue biopsy |
Tissue biopsy
|

that ONPATTRO® (patisiran) can reverse the
polyneuropathy manifestations of the disease9,10

Novel RNAi-based approach that targets the pathogenic protein at its source9

Reversal in neuropathy impairment from baseline9
- Mean change from baseline at 18 months in mNIS+7 of -6.0 points vs 28.0 with placebo, a treatment difference of -34 points (95% CI: -39.9, -28.1; p<0.001)
- 56% of patients treated with ONPATTRO experienced reversal in neuropathy impairment from baseline at 18 months vs 4% of patients treated with placebo

Improvement in quality of life9,10
- Mean change from baseline at 18 months in Norfolk QoL-DN of -6.7 points vs 14.4 with placebo, a treatment difference of -21.1 points (95% CI: -27.2, -15.0; p<0.001)
ONPATTRO demonstrated a benefit versus placebo in the treatment of the polyneuropathy of hATTR amyloidosis at 18 months.9,10
Read more about the efficacy of ONPATTRO »Study design9
The efficacy of ONPATTRO was demonstrated in a randomized, double-blind, placebo-controlled, multicenter clinical trial in adults with hATTR amyloidosis with polyneuropathy. Patients were randomized to receive ONPATTRO 0.3 mg/kg (N=148) or placebo (N=77) via intravenous infusion once every 3 weeks for 18 months.
Primary endpoint
The modified Neuropathy Impairment Score + 7 (mNIS+7) is an objective 304-point assessment of neuropathy that measures cranial nerve function, muscle strength, reflexes, postural blood pressure, quantitative sensory testing, and peripheral nerve electrophysiology, with higher scores representing worsening neuropathy.
Key secondary endpoint
Norfolk Quality of Life-Diabetic Neuropathy (QoL-DN) scale is a patient-reported assessment that evaluates neuropathy in the following domains: physical functioning/large fiber neuropathy, activities of daily living, symptoms, small fiber neuropathy, and autonomic neuropathy (score range -4 to 136), with higher scores representing worsening quality of life.
Now is the time to act.
hATTR amyloidosis is a rapidly progressive disease that can lead to significant disability and reduced quality of life for patients.6,11,12
Use the ASKnow guide to learn about how you can diagnose and treat the polyneuropathy of hATTR amyloidosis in adults