AL Amyloidosis for Cardiologists: Awareness, Diagnosis, and Future Prospects: JACC: CardioOncology State-of-the-Art Review

AD Wechalekar, M Fontana, CC Quarta, M Liedtke - Cardio Oncology, 2022 - jacc.org
Amyloid light chain (AL) amyloidosis is a rare, debilitating, often fatal disease. Symptoms of
cardiomyopathy are common presenting features, and patients often are referred to …

[HTML][HTML] State-of-the-art review on AL amyloidosis in Western countries: epidemiology, health economics, risk assessment and therapeutic management of a rare …

A Sabinot, G Ghetti, L Pradelli, S Bellucci, A Lausi… - Blood Reviews, 2023 - Elsevier
Amyloidosis is the term to define a broad array of rare protein misfolding syndromes. Among
them, light chain (AL) amyloidosis is the most common, affecting roughly 10 people per …

[PDF][PDF] Birtamimab plus standard of care in light-chain amyloidosis: the phase 3 randomized placebo-controlled VITAL trial

MA Gertz, AD Cohen, RL Comenzo, E Kastritis… - Blood, 2023 - ashpublications.org
Amyloid light-chain (AL) amyloidosis is a rare, typically fatal disease characterized by the
accumulation of misfolded immunoglobulin light chains (LCs). Birtamimab is an …

Health-related quality of life instruments for clinical trials in AL amyloidosis: report from the Amyloidosis Forum HRQOL Working Group

AA Rizio, MK White, A D'Souza, K Hsu… - Patient Related …, 2023 - Taylor & Francis
Systemic AL (light chain) amyloidosis is a rare protein misfolding disorder associated with
plasma cell dyscrasia affecting various organs leading to organ dysfunction and failure. The …

Changing paradigm in the treatment of amyloidosis: from disease-modifying drugs to anti-fibril therapy

CC Quarta, M Fontana, T Damy, J Catini… - Frontiers in …, 2022 - frontiersin.org
Cardiac amyloidosis is a rare, debilitating, and usually fatal disease increasingly recognized
in clinical practice despite patients presenting with non-specific symptoms of …

A cross-sectional study of patient-reported outcomes and symptom burden using PROMIS and PRO-CTCAE measures in light chain amyloidosis

A D'Souza, A Szabo, I Akinola, M Finkel… - Quality of Life Research, 2023 - Springer
Background We conducted a cross-sectional study to characterize health-related quality of
life and symptom burden in individuals living with light chain (AL) amyloidosis. Methods …

Development of a conceptual model of patient-reported outcomes in light chain amyloidosis: a qualitative study

A D'Souza, J Myers, R Cusatis, A Dispenzieri… - Quality of Life …, 2022 - Springer
Background Light chain (AL) amyloidosis is a plasma cell neoplasm associated with high
early mortality and severe morbidity that can cause severe disability. We explored the impact …

[HTML][HTML] Pooled analysis of safety from birtamimab phase 1-3 studies in patients with light chain (AL) amyloidosis

V Sanchorawala, AD Wechalekar, E Kastritis… - Blood, 2023 - Elsevier
Introduction: SystemicAL amyloidosis is a progressive and often fatal disease caused by
misfolded light chains that aggregate into amyloid fibrils and deposit in vital organs, leading …

Health‐related quality of life in patients with light chain amyloidosis treated with bortezomib, cyclophosphamide, and dexamethasone±daratumumab: Results from the …

V Sanchorawala, G Palladini… - American journal of …, 2022 - Wiley Online Library
In the phase 3 ANDROMEDA trial, patients treated with daratumumab, bortezomib,
cyclophosphamide, and dexamethasone (D‐VCd) had significantly higher rates of organ …

Systematic literature review of evidence in amyloid light-chain amyloidosis

C Lee, A Lam, T Kangappaden… - Journal of …, 2022 - becarispublishing.com
Introduction: Treatment of amyloid light-chain (AL) amyloidosis, a rare disease with a< 5-
year lifespan, remains challenging. This systematic literature review (SLR) aimed to …