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Experience of patisiran with transthyretin stabilizers in patients with hereditary transthyretin-mediated amyloidosis.
- Source :
-
Neurodegenerative disease management [Neurodegener Dis Manag] 2020 Oct; Vol. 10 (5), pp. 289-300. Date of Electronic Publication: 2020 Jun 10. - Publication Year :
- 2020
-
Abstract
- Aim: Examine safety and pharmacodynamics of patisiran alone or with concomitant transthyretin stabilizers from the Phase II open-label extension study and safety and efficacy of patisiran in patients with prior transthyretin stabilizer use from the Phase III APOLLO study. Patients & methods: Post hoc analyses in patients with hereditary transthyretin-mediated amyloidosis with polyneuropathy. Results: Patisiran safety was consistent regardless of concomitant or prior transthyretin stabilizers. In the Phase II open-label extension (n = 27), transthyretin reduction was similar over 24 months, regardless of concomitant transthyretin stabilizers. In APOLLO (n = 225), patisiran-treated groups showed stabilization or improvements in neurological function (modified Neuropathy Impairment Score +7) and quality of life (Norfolk Quality of Life-Diabetic Neuropathy questionnaire) at 18 months, regardless of prior transthyretin stabilizers. Conclusion: Patients benefit from patisiran regardless of transthyretin stabilizer use.
Details
- Language :
- English
- ISSN :
- 1758-2032
- Volume :
- 10
- Issue :
- 5
- Database :
- MEDLINE
- Journal :
- Neurodegenerative disease management
- Publication Type :
- Academic Journal
- Accession number :
- 32519928
- Full Text :
- https://doi.org/10.2217/nmt-2020-0020