ADDITIONAL DATA

Prespecified exploratory endpoint in the NEURO-TTRansform trial

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Improvement in autonomic neuropathy symptom score, as measured by COMPASS-311,2

COMPASS-31 is a patient-reported questionnaire used to evaluate symptoms of autonomic dysfunction, including GI and orthostatic intolerance*†‡3

Mean (+/- SE) change from baseline with WAINUA in COMPASS-31 total score at Week 81*†‡1,2

WAINUA (eplontersen) Compass-31 dataWAINUA (eplontersen) Compass-31 data

See study design.

Autonomic symptoms in hATTR-PN may emerge early and can greatly reduce quality of life4-6

  1. *COMPASS-31 total score at baseline in patients treated with WAINUA (n=141) was 19.4 (SD, 11.3).2

  2. †COMPASS-31 is a 31-item questionnaire that evaluates autonomic function across 6 domains. Final scores range from 0 to 100, with higher scores indicating worse autonomic dysfunction.1

  3. ‡This analysis did not include the external placebo group, as COMPASS-31 scores were not collected in the NEURO-TTR trial.2

The COMPASS-31 questionnaire is recommended for broad assessment of the severity and extent of a range of specific autonomic symptoms4

COMPASS-31 is a 31-item, self-administered questionnaire that evaluates autonomic function across 6 domains3:

Gastrointestinal

Covers symptoms like early satiety, bloating, vomiting, and bowel movement irregularities

Orthostatic intolerance

Evaluates symptoms like dizziness or lightheadedness upon standing

Vasomotor

Assesses symptoms related to blood vessel function like skin color changes

Secretomotor

Assesses problems related to sweating and dryness of eyes or mouth

Pupillomotor

Assesses light sensitivity and focusing difficulties

Bladder

Evaluates urinary symptoms, including incontinence and difficulty in voiding

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FOOTNOTES AND ABBREVIATIONS

COMPASS-31, Composite Autonomic Symptom Score-31; GI, gastrointestinal; hATTR-PN, polyneuropathy of hereditary transthyretin-mediated amyloidosis; SD, standard deviation; SE, standard error.

REFERENCES

  1. 1. Coelho T, Marques W Jr, Dasgupta NR, et al. Eplontersen for hereditary transthyretin amyloidosis with polyneuropathy [article and supplementary online content]. JAMA. 2023;330(15):1448-1458.
  2. 2. Wixner J, Berk J, Adams D, et al. Eplontersen improves autonomic neuropathy symptoms in hereditary ATTR: an analysis from NEURO-TTRansform. Presented at: 4th International ATTR Amyloidosis Meeting; November 2-3, 2023; Madrid, Spain.
  3. 3. Sletten DM, Suarez GA, Low PA, Mandrekar J, Singer W. COMPASS 31: a refined and abbreviated composite autonomic symptom score [article and supplementary appendix]. Mayo Clin Proc. 2012;87(12):1196-1201.
  4. 4. Wixner J, Berk JL, Adams D, et al. Effects of eplontersen on symptoms of autonomic neuropathy in hereditary transthyretin-mediated amyloidosis: secondary analysis from the NEURO-TTRansform trial. Amyloid. 2025;32(1):29-38.
  5. 5. Gonzalez-Duarte A, Valdés-Ferrer SI, Cantú-Brito C. Characteristics and natural history of autonomic involvement in hereditary ATTR amyloidosis: a systematic review. Clin Auton Res. 2019;29(suppl 1):1-9.
  6. 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.