Six minutes walk testKansas City questionnaireCOMPASS31 self questionnaire
Six minutes walk test: Six minutes walk test just before first intake of treatment and after 1 and 2 years
Kansas City questionnaire: Kansas City questionnaire just before first intake of treatment and after 1 and 2 years
COMPASS31 self questionnaire: Compass31 self questionnaire assessing dysautonomia just before first intake of treatment and after 1 and 2 years
Study summary
ATTRv amyloidosis is a systemic disease with two clinical forms, neurological and cardiological, which are sometimes combined (so-called mixed forms).
Patisiran and vutrisiran have shown protective effects on the progression of neurological damage.
The effects of Patisiran or vutrisiran on the heart remain incompletely understood. The aim of this study is to better understand the morphological and functional cardiac consequences in ATTRv patients with stage 1 or 2 polyneuropathy with a mixed form treated with Patisiran or vutrisiran
Eligibility
Sex
ALL
Min age
18 Years
Max age
—
Healthy volunteers
No
Inclusion Criteria:
* Patients aged 18 years or older
* Patients with hereditary transthyretin amyloidosis (ATTRv) with stage 1 or 2 polyneuropathy
* Patient not previously treated for ATTRv
* Patients for whom treatment with patisiran or vutrisiran has been initiated by a hospital neurologist in accordance with recommendations for a minimum of 24 months.
* Patients with NYHA stage 1 and 2 cardiac disease.
* Beneficiary of a social security scheme
* Person who does not object to his/her participation in the research
Exclusion Criteria:
* Patients treated with Tafamidis simultaneously with patisiran or vutrisiran
* Adults under legal protection (legal guardianship, curatorship, guardianship), persons deprived of liberty.
* Contraindications to the explorations provided for in the protocol: claustrophobia, metallic implant contraindicating MRI, woman of childbearing age
Primary outcome measure(s)
Difference in longitudinal relaxation time (T1) per mapping between M0 and M24 — 24 months Longitudinal relaxation time (T1) is the process by which the net magnetization (M) grows/returns to its initial maximum value (Mo) parallel to Bo in a MRI.
Myocardial T1 depends on the pulse sequence, cardiac cycle as well as other factors and increases at higher magnetic field strength. T1-mapping can detect a variety of myocardial pathologies, where it shows increased values. Because of variations between scanners the primary use of a local reference range is recommended and if a local reference range is not available quantitative results should not be clinically reported. An intermediate analysis will be done at 12 months
This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.
We use cookies to analyse site traffic and improve your experience. With your consent, we may also use cookies for advertising. You can change your choice at any time.
