Myocardial Effects in Patients With ATTRv With Polyneuropathy Treated With Patisiran or Vutrisiran (MyocardON-TTR)

Rennes University Hospital

Status

Active, not recruiting

Conditions

Transthyretin Amyloidosis

Amyloidosis, Hereditary

Treatments

Other: COMPASS31 self questionnaire

Other: Kansas City questionnaire

Other: Six minutes walk test

Study type

Observational

Funder types

Other

Identifiers

NCT05873868

35RC22_9817_MyocardON-TTR

Details and patient eligibility

About

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

Full description

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.

During their therapeutic management, including the prescription of Patisiran or vutrisiran, the routine examinations carried out at the inclusion, one and two year later will allow us to observe the consequences on myocardial activity during the routine consultation after 1 and 2 years of treatment.

Examinations are : clinical and biological exams, EKC, echocardiography, cardiac MRI and scintigraphy.

Data at the start of treatment and at 1 and 2 years will be collected, especially cardiac function assesment In addition, during these two consultations, a life quality questionnaire, a dysautonomia questionnaire and a functional walking test will be carried out specifically for the study.

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.

Enrollment

20 estimated patients

Sex

All

Ages

18+ years old

Volunteers

No Healthy Volunteers

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