FRINK Linked Data Fragments server

Matches in SemOpenAlex for { <https://semopenalex.org/work/W3089505857> ?p ?o ?g. }

• W3089505857 abstract "Abstract Background Immunoglobulin light chain (AL) amyloidosis is a rare, multi-systemic disorder characterized by two disease processes: an underlying plasma cell dyscrasia that provides the source of pathologic light chains, and the resulting organ dysfunction caused by deposition of amyloid light chain fibrils. There are no FDA approved treatments for AL amyloidosis; regimens developed for multiple myeloma are used off-label to treat the plasma cell disorder and no therapies are directed at organ deposition. Thus, an unmet medical need persists despite advances in disease management. A public-private partnership was recently formed between the Amyloidosis Research Consortium (ARC) and the US Food and Drug Administration (FDA) to bridge scientific gaps in drug development for the treatment of AL amyloidosis. Main Body The inaugural Amyloidosis Forum was convened at FDA on 12 November 2019 and led by a multidisciplinary panel of physicians, health outcomes professionals, and representatives from the FDA, ARC, and pharmaceutical companies. Patients provided important perspectives on the pathway to diagnosis, challenges of rigorous treatment, and the burden of disease. The panel reviewed the epidemiology, pathobiology, and clinical features of AL amyloidosis. Hematologic characteristics, staging systems, and response criteria were examined with clear consensus that a “deep response” to plasma cell-directed treatments was critical to overall survival. Emphasis was placed on the heterogeneous clinical phenotypes of AL amyloidosis, including cardiovascular, renal, neurological, and gastrointestinal system manifestations that contribute to morbidity and/or mortality, but render challenges to clinical trial endpoint selection. FDA representatives discussed regulatory perspectives regarding demonstration of clinical benefits of investigational therapies in the context of a rare disease with multi-systemic manifestations. The panel also highlighted the potential importance of well-designed health-related quality of life instruments, quantification of system organ effects, the potential of advanced imaging technologies, and survival prediction models. Conclusions The Amyloidosis Forum identified a clear need for novel trial designs that are scientifically rigorous, feasible, and incorporate clinically meaningful endpoints based on an understanding of the natural history of the disease in an evolving therapeutic landscape. Future forums will delve into these issues and seek to include participation from additional stakeholders." @default.
• W3089505857 created "2020-10-08" @default.
• W3089505857 creator A5044024465 @default.
• W3089505857 date "2020-09-29" @default.
• W3089505857 modified "2023-09-30" @default.
• W3089505857 title "The Amyloidosis Forum: a public private partnership to advance drug development in AL amyloidosis" @default.
• W3089505857 cites W1892471285 @default.
• W3089505857 cites W1976601231 @default.
• W3089505857 cites W1984636918 @default.
• W3089505857 cites W2003181078 @default.
• W3089505857 cites W2063772107 @default.
• W3089505857 cites W2098093182 @default.
• W3089505857 cites W2107104642 @default.
• W3089505857 cites W2111080788 @default.
• W3089505857 cites W2115902267 @default.
• W3089505857 cites W2126673903 @default.
• W3089505857 cites W2141145512 @default.
• W3089505857 cites W2171033052 @default.
• W3089505857 cites W2194865217 @default.
• W3089505857 cites W2217385909 @default.
• W3089505857 cites W2330120407 @default.
• W3089505857 cites W2341446173 @default.
• W3089505857 cites W2461719883 @default.
• W3089505857 cites W2582134011 @default.
• W3089505857 cites W2604327713 @default.
• W3089505857 cites W2618289397 @default.
• W3089505857 cites W2619452966 @default.
• W3089505857 cites W2729564033 @default.
• W3089505857 cites W2749536631 @default.
• W3089505857 cites W2751712131 @default.
• W3089505857 cites W2755913072 @default.
• W3089505857 cites W2758037713 @default.
• W3089505857 cites W2772440046 @default.
• W3089505857 cites W2781851390 @default.
• W3089505857 cites W2790071297 @default.
• W3089505857 cites W2796384138 @default.
• W3089505857 cites W2804360768 @default.
• W3089505857 cites W2810534146 @default.
• W3089505857 cites W2886155385 @default.
• W3089505857 cites W2898144881 @default.
• W3089505857 cites W2912723176 @default.
• W3089505857 cites W2940107318 @default.
• W3089505857 cites W2977735554 @default.
• W3089505857 cites W4205498725 @default.
• W3089505857 cites W4292024189 @default.
• W3089505857 doi "https://doi.org/10.1186/s13023-020-01525-2" @default.
• W3089505857 hasPubMedCentralId "https://www.ncbi.nlm.nih.gov/pmc/articles/7523334" @default.
• W3089505857 hasPubMedId "https://pubmed.ncbi.nlm.nih.gov/32993758" @default.
• W3089505857 hasPublicationYear "2020" @default.
• W3089505857 type Work @default.
• W3089505857 sameAs 3089505857 @default.
• W3089505857 citedByCount "8" @default.
• W3089505857 countsByYear W30895058572020 @default.
• W3089505857 countsByYear W30895058572021 @default.
• W3089505857 countsByYear W30895058572022 @default.
• W3089505857 countsByYear W30895058572023 @default.
• W3089505857 crossrefType "journal-article" @default.
• W3089505857 hasAuthorship W3089505857A5044024465 @default.
• W3089505857 hasBestOaLocation W30895058571 @default.
• W3089505857 hasConcept C126322002 @default.
• W3089505857 hasConcept C159654299 @default.
• W3089505857 hasConcept C177713679 @default.
• W3089505857 hasConcept C203014093 @default.
• W3089505857 hasConcept C2779551797 @default.
• W3089505857 hasConcept C2779951007 @default.
• W3089505857 hasConcept C2780411336 @default.
• W3089505857 hasConcept C36394416 @default.
• W3089505857 hasConcept C535046627 @default.
• W3089505857 hasConcept C71924100 @default.
• W3089505857 hasConceptScore W3089505857C126322002 @default.
• W3089505857 hasConceptScore W3089505857C159654299 @default.
• W3089505857 hasConceptScore W3089505857C177713679 @default.
• W3089505857 hasConceptScore W3089505857C203014093 @default.
• W3089505857 hasConceptScore W3089505857C2779551797 @default.
• W3089505857 hasConceptScore W3089505857C2779951007 @default.
• W3089505857 hasConceptScore W3089505857C2780411336 @default.
• W3089505857 hasConceptScore W3089505857C36394416 @default.
• W3089505857 hasConceptScore W3089505857C535046627 @default.
• W3089505857 hasConceptScore W3089505857C71924100 @default.
• W3089505857 hasIssue "1" @default.
• W3089505857 hasLocation W30895058571 @default.
• W3089505857 hasLocation W30895058572 @default.
• W3089505857 hasLocation W30895058573 @default.
• W3089505857 hasLocation W30895058574 @default.
• W3089505857 hasOpenAccess W3089505857 @default.
• W3089505857 hasPrimaryLocation W30895058571 @default.
• W3089505857 hasRelatedWork W1980048113 @default.
• W3089505857 hasRelatedWork W2027051530 @default.
• W3089505857 hasRelatedWork W2103595935 @default.
• W3089505857 hasRelatedWork W2120884623 @default.
• W3089505857 hasRelatedWork W2739204548 @default.
• W3089505857 hasRelatedWork W2980556910 @default.
• W3089505857 hasRelatedWork W3040975616 @default.
• W3089505857 hasRelatedWork W4290698720 @default.
• W3089505857 hasRelatedWork W4386021633 @default.
• W3089505857 hasRelatedWork W2183019163 @default.
• W3089505857 hasVolume "15" @default.
• W3089505857 isParatext "false" @default.
• W3089505857 isRetracted "false" @default.
• W3089505857 magId "3089505857" @default.

• next