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The British Journal of Dermatology Sep 1953
Topics: Amyloidosis; Immunoglobulin Light-chain Amyloidosis
PubMed: 13081917
DOI: No ID Found -
Blood Reviews May 2023Amyloidosis 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... (Review)
Review
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 million/year. The core purpose of the present literature review is to shed light on the academic and clinical knowledge on the condition, encompassing its i) epidemiology, ii) economic burden, and iii) quality of life consequences. The areas of interest are Europe and North America. Literature search was primarily performed on Embase® and finally integrated with additional, deemed eligible, sources. Pre-defined PICOS criteria were employed for the inclusion and exclusion processes. A total of 64 studies were comprehensively included in the current literature review as compliant with the inclusion criteria. The results were presented according to the outcomes of interest and eventually triangulated and compared to available literature studies. A broad picture on the main aspects of AL amyloidosis is delivered.
Topics: Humans; Immunoglobulin Light-chain Amyloidosis; Quality of Life; Rare Diseases; Amyloidosis; Risk Assessment
PubMed: 36697295
DOI: 10.1016/j.blre.2023.101040 -
California Medicine Jul 1949
Topics: Amyloidosis; Humans; Immunoglobulin Light-chain Amyloidosis; Multiple Myeloma
PubMed: 18153468
DOI: No ID Found -
Hematology. American Society of... Dec 2017Systemic amyloidosis is caused by misfolding and extracellular deposition of circulating proteins as amyloid fibrils, resulting in the dysfunction of vital organs. The... (Review)
Review
Systemic amyloidosis is caused by misfolding and extracellular deposition of circulating proteins as amyloid fibrils, resulting in the dysfunction of vital organs. The most common systemic amyloidosis, light-chain (AL) amyloidosis, is caused by misfolded light chains produced by a small, dangerous B-cell clone. The process of amyloid formation, organ targeting, and damage is multifaceted and, after disease initiation, the complexity of the downstream pathogenic cascade increases, rendering its control a challenge. Because of the progressive nature of the disease, early diagnosis to prevent end-stage organ damage is vital. Improving awareness and systematic use of biomarkers of organ damage in screening populations at risk may improve the still unsatisfactory diagnostic process. Amyloid imaging is now emerging as an important companion of biomarkers in formulating the diagnosis and prognosis and monitoring the effects of therapy. An accurate diagnosis is the basis for appropriate therapy that is risk-adapted and response-tailored. Effective treatments targeting the clone and rapidly and profoundly reducing the amyloid light chains have produced marked improvements in overall survival, making AL amyloidosis the most successful model of all amyloidoses. New therapies targeting the amyloid deposits are now under development, together with novel agents modulating light chain aggregation and proteotoxicity. The future of AL amyloidosis treatment is combination therapy and will require an innovative collaborative model for a rapid translation from bench to bedside with the ultimate aim of achieving a cure for this complex disease.
Topics: B-Lymphocytes; Biomarkers; Disease-Free Survival; Humans; Immunoglobulin Light-chain Amyloidosis; Protein Folding; Survival Rate
PubMed: 29222231
DOI: 10.1182/asheducation-2017.1.1 -
Journal of the National Medical... Sep 1963
Topics: Amyloidosis; Electrocardiography; Geriatrics; Humans; Immunoglobulin Light-chain Amyloidosis; Nephrotic Syndrome; Pathology; Radiography, Thoracic
PubMed: 14049554
DOI: No ID Found -
Hematology/oncology Clinics of North... Dec 2020In systemic light-chain amyloidosis, monoclonal antibodies target antigens that are either membrane-bound or circulating or deposited in the organs. CD38 holds high... (Review)
Review
In systemic light-chain amyloidosis, monoclonal antibodies target antigens that are either membrane-bound or circulating or deposited in the organs. CD38 holds high promise as a target against clonal plasma cells. Multiple anti-CD38 antibodies are either approved for use or being investigated in clinical trials. Daratumumab has been investigated and has clinical efficacy in upfront or refractory settings. High rates of hematologic response are seen with daratumumab, which translates to high organ response rates. Rituximab is usually integrated into the treatment regimen for IgM amyloidosis. Anti-amyloid therapies have shown preclinical proof of principle, but lack confirmation of improvement.
Topics: Antibodies, Monoclonal; Humans; Immunoglobulin Light-chain Amyloidosis; Rituximab
PubMed: 33099430
DOI: 10.1016/j.hoc.2020.08.005 -
Amyloid : the International Journal of... Mar 2022
Topics: Humans; Immunoglobulin Light-chain Amyloidosis
PubMed: 34514911
DOI: 10.1080/13506129.2021.1974831 -
European Journal of Haematology Nov 2023This study evaluated data from six Swedish national registries to fill current evidence gaps on the epidemiology, clinical burden, and overall survival (OS) associated...
OBJECTIVES
This study evaluated data from six Swedish national registries to fill current evidence gaps on the epidemiology, clinical burden, and overall survival (OS) associated with light-chain (AL) amyloidosis.
METHODS
Patients newly diagnosed with AL amyloidosis were identified using six linked Swedish nationwide population-based registers. For each case, individuals from the general population were selected and matched with a maximum ratio of 1:5 based on age, sex, calendar year, and county.
RESULTS
846 patients newly diagnosed with AL amyloidosis and 4227 demographically matched individuals were identified. From 2011 to 2019, annual AL amyloidosis incidence increased from 10.5 to 15.1 cases per million. At baseline, patients with AL amyloidosis had a significantly higher disease burden including higher rates of cardiac and renal failure relative to the comparison group. Among patients with AL amyloidosis, 21.5% had incident heart failure and 17.1% had incident renal failure after initial diagnosis. Median OS for patients with AL amyloidosis was 56 months versus not reached in the matched general population comparison group.
CONCLUSION
The incidence of newly diagnosed AL amyloidosis in Sweden increased over time with AL amyloidosis being associated with a higher risk of cardiac/renal failure and all-cause mortality compared with the general population.
Topics: Humans; Immunoglobulin Light-chain Amyloidosis; Sweden; Amyloidosis; Heart Failure; Renal Insufficiency; Retrospective Studies
PubMed: 37533343
DOI: 10.1111/ejh.14063 -
International Journal of Cardiology Nov 2018Light-chain (AL) amyloidosis is the most common type of systemic amyloidosis, affecting around 10 people per million per year. This serious disorder is characterized by... (Review)
Review
Light-chain (AL) amyloidosis is the most common type of systemic amyloidosis, affecting around 10 people per million per year. This serious disorder is characterized by the presence of a clone of bone marrow plasma cells that produces monoclonal light chains (LCs) of the κ or predominantly λ type. These amyloidogenic LCs undergo extracellular misfolding and aggregation into proteotoxic soluble oligomers and amyloid fibrils that deposit within tissues. The lethal consequences of AL amyloidosis are due to the toxic products (the LCs) and not to the malignant behaviour of the plasma cell clone. Almost 80% of patients with AL amyloidosis have some degree of cardiac involvement, manifesting as heart failure (HF), and carrying a particularly poor prognosis. The past decade has seen major advances in the treatment of AL amyloidosis, and a rapidly fatal disease has become a treatable and possibly curable condition. The number of therapeutic options is rapidly expanding, offering hope to address currently unmet needs (most notably, the treatment of frail patients). The treatment of AL amyloidosis consists in a combination of agents targeting multiple steps of the amyloid cascade, associated with effective HF management, and there is ground for hope for dramatically improving the outcome in the near future. In the present review we will summarize our current knowledge on therapy for cardiac AL amyloidosis, targeting clinical cardiologists involved in the care of this serious disorder.
Topics: Animals; Cell- and Tissue-Based Therapy; Humans; Immunoglobulin Light-chain Amyloidosis; Plasma Cells
PubMed: 30223349
DOI: 10.1016/j.ijcard.2018.05.018 -
Ugeskrift For Laeger Jul 2018Localised laryngeal amyloidosis is a rare tumour of the upper respiratory tract, which is characterised by extra-cellular accumulation of proteinaceous material in the...
Localised laryngeal amyloidosis is a rare tumour of the upper respiratory tract, which is characterised by extra-cellular accumulation of proteinaceous material in the submucosa. The aetiology is still unclear. This is a case report of localised multifocal amyloidosis located to larynx and rhinopharynx. A 50-year-old women with a history of progressive dysphonia and dyspnoea underwent ear-nose-throat and haematological investigation with no signs of systemic involvement. The amyloid deposits in larynx were effectively treated with laser resection in general anaesthesia and regular follow-up.
Topics: Female; Humans; Immunoglobulin Light-chain Amyloidosis; Laryngeal Diseases; Middle Aged
PubMed: 30020074
DOI: No ID Found