Real-world treatment management in hereditary transthyretin amyloidosis – an experience report and proposal for therapy switch decision criteria
In this study, reasons for treatment switches were manifold, encompassing disease progression, the occurrence of adverse events, patient preferences and/or the availability of newly approved drugs. Hence, multidimensional consideration of these reasons remains pivotal in guiding the subsequent choice of medication in particular and managing hereditary transthyretin amyloidosis in general.
Authors
- Nicolas Wieder (ORCID: https://orcid.org/0000-0001-5683-2891)
- Bettina Heidecker (ORCID: https://orcid.org/0000-0002-3811-7920)
- Sebastian Spethmann (ORCID: https://orcid.org/0000-0003-0987-8007)
- Stefanie Maria Werhahn (ORCID: https://orcid.org/0000-0002-0805-3129)
- Gina Barzen
- Paul J Wetzel (ORCID: https://orcid.org/0009-0001-9393-5512)
- Katrin Hahn (ORCID: https://orcid.org/0000-0002-6013-0072)
- Helena F Pernice (ORCID: https://orcid.org/0009-0001-8711-5526)
- Harisa Muratovic
- Duc Chu Dieu
Institutions
- Humboldt-Universität zu Berlin (DE)
- Deutsches Herzzentrum der Charité (DE)
- German Centre for Cardiovascular Research (DE)
- Berlin Institute of Health at Charité - Universitätsmedizin Berlin (DE)
- Freie Universität Berlin (DE)
- Charité - Universitätsmedizin Berlin (DE)
Publication Details
- Journal
- Neurological Research and Practice
- Published
- 2025-09-12
- DOI
- https://doi.org/10.1186/s42466-025-00428-6
- Citations
- 1
- Primary Topic
- Amyloidosis: Diagnosis, Treatment, Outcomes
- Type
- article
- Field-Weighted Citation Impact
- 0.81