The impact of clinical heterogeneity on conducting network meta-analyses in transthyretin amyloidosis with polyneuropathy.

Conclusions: An NMA of ATTR-PN treatments was not feasible, given the observed cross-trial heterogeneity. This decision highlights the importance of careful consideration for clinical heterogeneity that may threaten the validity of indirect comparisons. PMID: 32011182 [PubMed - as supplied by publisher]
Source: Current Medical Research and Opinion - Category: Research Tags: Curr Med Res Opin Source Type: research

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European Journal of Heart Failure, EarlyView.
Source: European Journal of Heart Failure - Category: Cardiology Authors: Tags: Editorial Comment Source Type: research
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Source: Amyloid: The Journal of Protein Folding Disorders - Category: Biochemistry Authors: Source Type: research
Systemic amyloidosis is a clinical manifestation of the accumulation of amyloid fibrils in tissues because of persistent acute phase elevation and chronic inflammation. Its most common causes are inflammatory diseases and malignancies. Here, we present a 12-year-old girl diagnosed with systemic amyloidosis and Hodgkin lymphoma (HL) who was also previously diagnosed with familial Mediterranean fever (FMF). Despite colchicine treatment for FMF, the patient had a persistent elevation of acute phase reactants and AA-type amyloid deposits were observed in a kidney biopsy. Anakinra, an interleukin-1 antagonist, was added to the ...
Source: Journal of Pediatric Hematology Oncology - Category: Hematology Tags: Clinical and Laboratory Observations Source Type: research
Abstract Alzheimer's disease (AD) is one of the most common neurodegenerative diseases and characterized by cognitive and memory impairments. Emerging evidence suggests that the extracellular matrix (ECM) biomacromolecules in brain plays an important role in etiology of AD. It has been detected that the levels of ECM proteins have been changed in the brains of AD patients and animal models. Some ECM components, e.g., elastin and heparan sulfate proteoglycans, are considered to promote the upregulation of extracellular amyloid-β (Aβ) proteins. In addition, collagen VI and laminin are shown to have interac...
Source: Chemistry - Category: Chemistry Authors: Tags: Chemistry Source Type: research
In this report, we describe an AL patient who had suffered from recurrent infection during previous treatment, but who responded to and tolerated well single-agent venetoclax for more than 1 year. The present report indicates that venetoclax monotherapy may be active and safe for refractory AL amyloidosis. PMID: 32207051 [PubMed - as supplied by publisher]
Source: International Journal of Hematology - Category: Hematology Authors: Tags: Int J Hematol Source Type: research
AbstractTafamidis, a non ‐nonsteroidal anti‐inflammatory benzoxazole derivative, acts as a transthyretin (TTR) stabilizer to slow progression of TTR amyloidosis (ATTR). Tafamidis meglumine, available as 20‐mg capsules, is approved in more than 40 countries worldwide for the treatment of adults with early‐stage sympt omatic ATTR polyneuropathy. This agent, administered as an 80‐mg, once‐daily dose (4 × 20‐mg capsules), is approved in the United States, Japan, Canada, and Brazil for the treatment of hereditary and wild‐type ATTR cardiomyopathy in adults. An alternative single solid oral dosage formulati...
Source: Clinical Pharmacology in Drug Development - Category: Drugs & Pharmacology Authors: Tags: Original Manuscript Source Type: research
AbstractAlthough the benefit of updated therapeutic regimens, including bortezomib, on the survival of immunoglobulin light chain (AL) amyloidosis patients with heart failure (HF) has been reported, predictors of mortality in the patients treated with the updated therapy remain unclear. We retrospectively enrolled AL amyloidosis patients who had severe HF at the time of diagnosis and received the updated therapy, including bortezomib (n = 19, 61 ± 6 years old, 68% male). Severe HF was defined as the presence of both NYHA functional class III or IV and BNP >  200 ...
Source: Heart and Vessels - Category: Cardiology Source Type: research
Pathogenic tau triggers tauopathy, which serves as a focal point for many other neurodegenerative diseases. Nanogold polyethylene glycol(Au ‐PEG) alters tau as a nanochaperon and lowers protopathic tau burden while also acting as kinetic stabilizer preventing tau oligomerization. Au‐PEG inhibits amyloidosis in human AD serum by remodeling tau‐like pro‐dementia factors. Editing tau function by Au‐PEG brings optimism to managing Alzheimer's dementia. AbstractTauopathy is a complex disorder associated at the junction of several other pathologies. Intrinsically disordered tau protein remains therapeutically challengi...
Source: Small - Category: Nanotechnology Authors: Tags: Communication Source Type: research
Condition:   Light Chain (AL) Amyloidosis Intervention:   Biological: STI-6129 Sponsor:   Sorrento Therapeutics, Inc. Not yet recruiting
Source: ClinicalTrials.gov - Category: Research Source Type: clinical trials
Publication date: Available online 19 March 2020Source: Clinical Lymphoma Myeloma and LeukemiaAuthor(s): Yuanyuan Yu, Zhongxia Huang, Wanli Hu, Xin Li, Man Shen, Jiajia Zhang, Ran Tang, Shilun Chen, Wenming Chen
Source: Clinical Lymphoma Myeloma and Leukemia - Category: Cancer & Oncology Source Type: research
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