Impact of RNAi therapeutics on cardiac parameters in patients with hereditary transthyretin amyloidosis initially treated with stabilizers: A French real-world study - PubMed
5 hours ago
- #real-world study
- #ATTRv cardiomyopathy
- #RNAi therapeutics
- Study focuses on the impact of RNAi therapeutics on cardiac parameters in hereditary transthyretin amyloidosis (ATTRv) patients initially treated with stabilizers.
- Data from 50 patients (median age 63, 66% male) from 10 French centers were analyzed retrospectively.
- Patients switched from tafamidis monotherapy to RNAi therapeutics (patisiran or vutrisiran) or added RNAi to their treatment.
- Primary reason for switching/adding RNAi was neurological progression (80% of cases).
- Under tafamidis monotherapy, 24% of patients worsened, while 76% remained stable.
- After switching/adding RNAi, 90% of patients were stable, 10% improved, and none worsened.
- Study highlights potential benefits of early RNAi intervention in patients with insufficient cardiac response to tafamidis.
- First real-world switch/add-on study in mixed ATTRv phenotypes.