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Epidemiology of transthyretin (ATTR) amyloidosis: a systematic literature review.

Orphanet J Rare Dis

January 2025

Cardiovascular, Renal and Metabolism (CVRM) Evidence, BioPharmaceuticals Medical, AstraZeneca, Gothenburg, Sweden.

Introduction: Significant advances in the treatment of transthyretin (ATTR) amyloidosis has led to an evolving understanding of the epidemiology of this condition. This systematic literature review (SLR) aims to synthesize current evidence on epidemiology and mortality outcomes in ATTR amyloidosis, addressing the need for a comprehensive understanding of its current global impact.

Methods: An SLR of the literature from January 2018 to April 2023 was conducted using the Medline and Embase databases.

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Objectives: To describe the clinical presentation and clinicopathological findings of dogs with nodular splenic lesions composed of heterogeneous cell components associated with systemic inflammation and to provide information on the outcome after surgical resection.

Materials And Methods: Medical records were searched for dogs with histologically and immunohistochemically characterised nodular splenic lesions with mixed stromal, histiocytic and lymphoid cells and the presence of systemic inflammatory markers at the time of diagnosis.

Results: Four dogs were included, of which three had an undifferentiated splenic stromal sarcoma and one had a splenic leiomyosarcoma.

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There is no established treatment for late or chronic antibody-mediated rejection of a kidney graft. Rituximab-based treatment is not effective, since long-lived high-affinity plasma cells do not express CD20 and do not depend on previous maturation steps to generate donor-specific antibodies. Conversely, daratumumab, an anti-CD38 monoclonal antibody, directly targets plasma cells, with proven efficacy in multiple myeloma.

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Purpose Of Review: Familial Mediterranean Fever (FMF) is a hereditary autoinflammatory disorder predominantly affecting individuals of Mediterranean and Middle Eastern descent, including those with certain heritages including Sephardic Jewish, Armenian, Turkish, and Arab. The disorder affects up to 1 in 200 people making it a very common etiology for pain states worldwide, including serositis mediated painful states of the chest, joint, and abdomen.

Recent Findings: Defined by recurrent episodes of fever and inflammation, FMF can lead to not only severe pain, but complications such as renal amyloidosis, if untreated.

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Cardiovascular-kidney-metabolic (CKM) syndrome is the association between obesity, diabetes, CKD (chronic kidney disease), and cardiovascular disease. GDF-15 mainly acts through the GFRAL (Glial cell line-derived neurotrophic factor Family Receptor Alpha-Like) receptor. GDF-15 and GDFRAL complex act mainly through RET co-receptors, further activating Ras and phosphatidylinositol-3-kinase (PI3K)/Akt pathways through downstream signaling.

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