Real-world treatment management in hereditary transthyretin amyloidosis – an experience report and proposal for therapy switch decision criteria

Abstract Background Hereditary transthyretin amyloidosis is a rapidly progressive and lethal disease. Thanks to the increasing number of disease-modifying treatments, prognosis has improved significantly. However, new challenges regarding treatment response and when to change treatment remain unansw...

書目詳細資料
發表在:	Neurological Research and Practice
Main Authors:	Duc Chu Dieu, Helena F. Pernice, Harisa Muratovic, Paul J. Wetzel, Gina Barzen, Nicolas W. Wieder, Stefanie M. Werhahn, Bettina Heidecker, Sebastian Spethmann, Katrin Hahn
格式:	Article
語言:	英语
出版:	BMC 2025-09-01
主題:	Hereditary transthyretin amyloidosis Patisiran Vutrisiran Tafamidis Inotersen SiRNA
在線閱讀:	https://doi.org/10.1186/s42466-025-00428-6

相似書籍

Treatment characteristics of patients with hereditary transthyretin amyloidosis: a cohort study
由: Taha N. Qarni, et al.
出版: (2024-05-01)

A real-world pharmacovigilance analysis for transthyretin inhibitors: findings from the FDA adverse event reporting database
由: Yuan Liu, et al.
出版: (2024-05-01)

Comparative Study on Cardiac Findings in Patients with Transthyretin Amyloidosis Before and After Treatment with a Transthyretin Silencer
由: Priya Arivalagan, et al.
出版: (2025-09-01)

Assessing the effectiveness and safety of Patisiran and Vutrisiran in ATTRv amyloidosis with polyneuropathy: a systematic review
由: Mohammad Amin Karimi, et al.
出版: (2024-09-01)

A mutation in transthyretin gene in a Mexican patient leading to hereditary amyloidosis: a case report
由: Muhammad Reebal Malik, et al.
出版: (2025-08-01)

Cardiac Amyloidosis Treatment
由: Lily K. Stern, et al.
出版: (2022-03-01)

Practical Guidance for the Use of Patisiran in the Management of Polyneuropathy in Hereditary Transthyretin-Mediated Amyloidosis
由: Dixon S, et al.
出版: (2023-11-01)

Outcomes in Cardiac Transthyretin Amyloidosis and Association With New York Heart Association Class: Real‐World Data
由: Maximilian Leo Müller, et al.
出版: (2024-07-01)

Vutrisiran in Transthyretin Amyloidosis
由: Ronald M. Witteles, MD, et al.
出版: (2025-09-01)

Italian Real-Life Experience of Patients with Hereditary Transthyretin Amyloidosis Treated with Patisiran
由: Di Stefano V, et al.
出版: (2022-05-01)

Hereditary Transthyretin Amyloidosis (ATTRv) with Symmetric Paresthesia in All Limbs: A Case Report and Literature Review
由: Lilia Böckels, et al.
出版: (2025-09-01)

A Comparison of Currently Approved Small Interfering RNA (siRNA) Medications to Alternative Treatments by Costs, Indications, and Medicaid Coverage
由: Inder Sehgal, et al.
出版: (2024-03-01)

Real‐world characteristics and treatment of cardiac transthyretin amyloidosis: A multicentre, observational study
由: Richard J. Nies, et al.
出版: (2025-04-01)

Transthyretin Stabilizers and Seeding Inhibitors as Therapies for Amyloid Transthyretin Cardiomyopathy
由: Paolo Morfino, et al.
出版: (2023-04-01)

Updated Evaluation of the Safety, Efficacy and Tolerability of Tafamidis in the Treatment of Hereditary Transthyretin Amyloid Polyneuropathy
由: Falcão de Campos C, et al.
出版: (2023-02-01)

Real-World Utilization Patterns, Safety, and Efficacy of Tafamidis in Patients With Hereditary Transthyretin Amyloidosis in Japan
由: Hiroaki Konishi, PharmD, et al.
出版: (2025-01-01)

Patisiran for the treatment of patients with p.Ile88Leu hereditary transthyretin amyloidosis: an Italian real-life experience
由: Giacomo Urbinati, et al.
出版: (2024-06-01)

Tafamidis decreased cardiac amyloidosis deposition in patients with Ala97Ser hereditary transthyretin cardiomyopathy: a 12-month follow-up cohort study
由: Cheng-Hsuan Tsai, et al.
出版: (2023-09-01)

Transthyretin Amyloidosis Therapies: Guiding the Future
由: Alejandra González-Duarte
出版: (2021-01-01)

Coprevalence of Amyloidosis and Aortic Stenosis: When to Screen and Who to Treat?
由: Brett W. Sperry
出版: (2025-01-01)

A clinical case of transthyretin amyloidosis with manifestations of seronegative arthritis
由: V. A. Mareeva, et al.
出版: (2024-02-01)

SGLT2 inhibitor therapy for transthyretin amyloid cardiomyopathy: early tolerance and clinical response to dapagliflozin
由: Stephan Dobner, et al.
出版: (2023-02-01)

Delays in diagnosis and treatment of ATTR cardiac amyloidosis: A real‐world data analysis
由: Julia Vogel, et al.
出版: (2025-08-01)

Kidney, the Cinderella Story of Transthyretin Amyloidosis
由: Nelson Leung
出版: (2025-06-01)

Chest pain in a patient with transthyretin cardiac amyloidosis: A case report
由: Linfeng Xie, et al.
出版: (2024-04-01)

Clinical case of hereditary transthyretin amyloidosis
由: N. Spasova, et al.
出版: (2024-12-01)

Treating amyloid transthyretin cardiomyopathy: lessons learned from clinical trials
由: Daniela Tomasoni, et al.
出版: (2023-05-01)

Spectrum of transthyretin gene mutations and clinical characteristics of Polish patients with cardiac transthyretin amyloidosis
由: Monika Gawor, et al.
出版: (2022-12-01)

Modern approaches to cardiovascular amyloidosis treatment
由: Svetlana N. Morozkina, et al.
出版: (2021-12-01)

A Clinical Case of the Hereditary Transthyretin Amyloidosis
由: E. V. Reznik, et al.
出版: (2021-05-01)

Hereditary transthyretin amyloidosis
由: T. A. Adyan, et al.
出版: (2020-01-01)

A phase II, open-label, extension study of long-term patisiran treatment in patients with hereditary transthyretin-mediated (hATTR) amyloidosis
由: Teresa Coelho, et al.
出版: (2020-07-01)

A new era in treatment of cardiac amyloidosis: an overview of the Congress of cardiology
由: I. E. Strelkova
出版: (2022-01-01)

Impact of patisiran on polyneuropathy of hereditary transthyretin amyloidosis in patients with a V122I or T60A variant: a phase IV multicenter study
由: Yessar Hussain, et al.
出版: (2025-12-01)

Hereditary systemic transthyretin amyloidosis: a clinical case and an opinion on the problem
由: A. S. Draganova, et al.
出版: (2019-07-01)

Myocardial work assessment to improve baseline risk stratification in patients with transthyretin amyloidosis
由: Ana Moya, et al.
出版: (2024-12-01)

Successful Treatment with Patisiran in Amyloid Polyneuropathy Harboring His90Asn Mutation in the <i>TTR</i> Gene
由: Vincenzo Di Stefano, et al.
出版: (2024-05-01)

Clinical and genetic profiles of patients with hereditary and wild-type transthyretin amyloidosis: the Transthyretin Cardiac Amyloidosis Registry in the state of São Paulo, Brazil (REACT-SP)
由: Fábio Fernandes, et al.
出版: (2024-07-01)

Hereditary cardiac amyloidosis associated with Pro24Ser transthyretin mutation: a case report
由: Hiroyuki Yamamoto, et al.
出版: (2018-12-01)

Proteomic Analysis of Serum in Cardiac Transthyretin Amyloidosis: Diagnostic and Prognostic Implications for Biomarker Discovery
由: Joanna Waś, et al.
出版: (2025-07-01)