Switching from inotersen to eplontersen in patients with hereditary transthyretin-mediated amyloidosis with polyneuropathy: analysis from NEURO-TTRansform
Switching from inotersen to eplontersen further reduced serum TTR, halted disease progression, stabilized QoL, restored platelet count, and improved tolerability, without deterioration in nutritional status. This supports a positive benefit-risk profile for patients with ATTRv-PN who switch from inotersen to eplontersen.
Authors
- Rosie Z. Yu (ORCID: https://orcid.org/0000-0001-9266-7280)
- T. Jesse Kwoh
- Morie A. Gertz (ORCID: https://orcid.org/0000-0002-3853-5196)
- John L. Berk (ORCID: https://orcid.org/0000-0002-1768-2373)
- Pedro André Kowacs (ORCID: https://orcid.org/0000-0001-7770-7475)
- Chi‐Chao Chao (ORCID: https://orcid.org/0000-0001-6499-5789)
- Márcia Waddington‐Cruz (ORCID: https://orcid.org/0000-0003-4853-2236)
- Markus Weiler (ORCID: https://orcid.org/0000-0002-8942-7662)
- Noel R. Dasgupta
- Ahmad Masri (ORCID: https://orcid.org/0000-0002-6390-6526)
- Isabel M. Conceição (ORCID: https://orcid.org/0000-0003-0934-9631)
- Shahram Attarian (ORCID: https://orcid.org/0000-0002-7211-4694)
- Sami Khella
- Teresa Coelho (ORCID: https://orcid.org/0000-0003-4678-3189)
- Jersey Chen
- Shiangtung W. Jung
- Nicholas J. Viney
Institutions
- Universidade Federal do Rio de Janeiro (BR)
- University of Lisbon (PT)
- Heidelberg University (DE)
- Ionis Pharmaceuticals (United States) (US)
- University Hospital Heidelberg (DE)
- Amyloidosis Foundation (US)
- Mayo Clinic in Arizona (US)
- Hospital de Santo António (PT)
- National Taiwan University Hospital (TW)
- Instituto de Neurologia de Curitiba (BR)
- AstraZeneca (United States) (US)
- Hôpital de la Timone (FR)
- Indiana University School of Medicine
- Indiana University – Purdue University Indianapolis (US)
- University of Pennsylvania (US)
Publication Details
- Journal
- Journal of Neurology
- Published
- 2024-08-13
- DOI
- https://doi.org/10.1007/s00415-024-12616-6
- Citations
- 9
- Primary Topic
- Amyloidosis: Diagnosis, Treatment, Outcomes
- Type
- article
- Field-Weighted Citation Impact
- 2.10
Funders
- Bristol-Myers Squibb
- Pfizer
- AstraZeneca
- Sanofi
- Alnylam Pharmaceuticals
- Celgene
- Ionis Pharmaceuticals
- Cytokinetics
- Prothena
- Aptitude Health