hATTR-PN often starts with small-fiber neuropathy, which may result in dysautonomia and rapid deterioration of patients' quality of life1-4

Up to 83% of patients can experience dysautonomia*†
—debilitating diarrhea, GI distress, and orthostatic hypotension are common manifestations that can compromise daily living5-7
In hATTR, dysautonomia often precedes sensorimotor neuropathy. hATTR-PN can lead to‡1,5,6,8:
LOSS OF INDEPENDENCE
Dressing
Bathing
Using the bathroom
LOSS OF PHYSICAL FUNCTION
Standing
Walking
Gripping objects

I had diarrhea—sometimes I didn't know if I was going to make it to the bathroom. I didn't even want to leave the house.

Explore the disease manifestations of hATTR
See how WAINUA targets TTR at the source
FOOTNOTES AND ABBREVIATIONS
*Based on an ongoing, global, longitudinal, observational survey (THAOS) in patients with hATTR, ATTRwt, and asymptomatic gene carriers with TTR mutations (N=2922). Autonomic dysfunction was present in 40.4% of the overall patient population and 83% in patients with hATTR once unknown patients were accounted for.6
†Based on a study (n=124) of hATTR patients who reported, 27% of patients had autonomic dysfunction symptoms at disease onset, and 78% had autonomic dysfunction symptoms at the time of first assessment (mean disease duration at time of first assessment was 4.5 years).7
‡Data from semistructured interviews in a qualitative, noninterventional study of 14 adults with hATTR.8
ATTRwt, wild-type transthyretin-mediated amyloidosis; GI, gastrointestinal; hATTR, hereditary transthyretin-mediated amyloidosis; hATTR-PN, polyneuropathy of hereditary transthyretin-mediated amyloidosis.
REFERENCES
- 1. Nativi-Nicolau JN, Karam C, Khella S, Maurer MS. Screening for ATTR amyloidosis in the clinic: overlapping disorders, misdiagnosis, and multiorgan awareness. Heart Fail Rev. 2022;27(3):785-793.
- 2. Adams D, Ando Y, Beirão JM, et al. Expert consensus recommendations to improve diagnosis of ATTR amyloidosis with polyneuropathy. J Neurol. 2021;268(6):2109-2122.
- 3. Hawkins PN, Ando Y, Dispenzeri A, Gonzalez-Duarte A, Adams D, Suhr OB. Evolving landscape in the management of transthyretin amyloidosis. Ann Med. 2015;47(8):625-638.
- 4. Carroll A, Dyck PJ, de Carvalho M, et al. Novel approaches to diagnosis and management of hereditary transthyretin amyloidosis. J Neurol Neurosurg Psychiatry. 2022;93(6):668-678.
- 5. Sander L, Chiaro G, Abelardo D, et al. Early cardiovascular autonomic failure in ATTRv predicts poor prognosis and may respond to disease-modifying therapy. Amyloid. 2025;32(3):246-254.
- 6. Barroso FA, Coelho T, Dispenzieri A, et al; THAOS Investigators. Characteristics of patients with autonomic dysfunction in the Transthyretin Amyloidosis Outcomes Survey (THAOS). Amyloid. 2022;29(3):175-183.
- 7. Chiaro G, Stancanelli C, Koay S, et al. Cardiovascular autonomic failure in hereditary transthyretin amyloidosis and TTR carriers is an early and progressive disease marker. Clin Auton Res. 2024;34(3):341-352.
- 8. Lovley A, Raymond K, Guthrie SD, Pollock M, Sanchorawala V, White MK. Patient-reported burden of hereditary transthyretin amyloidosis on functioning and well-being. J Patient Rep Outcomes. 2021;5(1):3.


