Amyloid : the international journal of experimental and clinical investigation : the official journal of the International Society of Amyloidosis
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The novel class of compounds represented by lipid nanoparticle (LNP)-encapsulated siRNA formulations has an enormous potential to target disease, notably of the liver. Endocytosis of LNPs is believed to be mediated by APOE, an important serum protein of lipoprotein homeostasis. APOE polymorphisms affect binding to hepatic receptors and have been associated with development of specific disease. ⋯ Analysis of APOE polymorphisms in ATTR amyloidosis patients revealed three most frequent genotypes E3/3, E3/4 and E3/2. APOE stratification of patients did not show significant differences of TTR plasma concentrations following treatment. Our results suggest that APOE is an important mediator of TTR silencing by Patisiran, however efficacy is independent of the APOE genotype.
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Amyloidosis is an extremely rare event in rats. In this study, we report that lipopolysaccharide binding protein (LBP) is the most likely amyloidogenic protein in rat mammary amyloidosis. Histologically, corpora amylacea (CA) and stromal amyloid (SA) were observed in rat mammary glands, and needle-shaped amyloid (NA) was also observed on the surface or gap of CA and SA. ⋯ In the same analysis, LBP was detected as a prime candidate protein in NA, and NA was positive following immunohistochemistry and immunoelectron microscopy with anti-LBP antibody. Furthermore, synthetic peptides derived from rat LBP formed amyloid fibrils in vitro. Overall, these results provide evidence that LBP is an amyloid precursor protein of NA in rat mammary glands.
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Letter Case Reports
Coexistence of wild type and hereditary ATTR amyloidosis in one family.
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Introduction: HHV8-negative Castleman disease (CD) is classified as hyaline vascular (HV) type, or mixed or plasma cell (PC) types. It may present as multicentric CD (MCD) or unicentric CD (UCD). CD is a rare cause of AA amyloidosis (AAA). ⋯ Clinical and biologic remission was achieved in six patients with MCD (43%), all of whom were treated with anti-interleukin-6 (IL-6) therapy. Conclusions: AAA is a rare complication of CD, namely idiopathic MCD and UCD presenting with the PC histologic subtype. Surgical excision of UCD should be the first-line treatment whenever possible, while anti-IL-6 therapies seem effective for MCD.
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Objective: To characterize the changing spectrum of amyloidosis classes, as well as patient demographics, at a major US referral centre. Patients and methods: A retrospective analysis was conducted of all referrals to the Amyloidosis Centre at Boston University and Boston Medical Centre over the last 3 decades. Results: A total of 3987 new patients with amyloidosis were evaluated between 1990 and 2018 with the average number of new cases per year increasing 2.5-fold during this period. ⋯ Conclusion: Amyloid diseases are more widely recognized and classes of amyloidosis, including ATTRwt and ATTRV122I, once considered rare are now increasingly diagnosed. These data likely reflect a national trend of increased amyloidosis awareness facilitated by accessible diagnostic approaches, emerging treatments, and coordinated educational initiatives. ClinicalTrials.gov identifier: NCT00898235.