Pathway for Development and Validation of Multi-domain Endpoints for Amyloid Light Chain (AL) Amyloidosis Article Swipe
James Signorovitch
,
Jialü Zhang
,
David T. Brown
,
Preston Dunnmon
,
Liang Xiu
,
Nicolae Done
,
Kristen Hsu
,
Yolanda Barbáchano
,
Isabelle Lousada
·
YOU?
·
· 2024
· Open Access
·
· DOI: https://doi.org/10.1007/s43441-024-00641-6
YOU?
·
· 2024
· Open Access
·
· DOI: https://doi.org/10.1007/s43441-024-00641-6
Immunoglobin light chain (AL) amyloidosis is a rare disease in which a plasma cell dyscrasia leads to deposition of insoluble amyloid fibrils in multiple organs. To facilitate development of new therapies for this heterogenous disease, a public–private partnership was formed between the nonprofit Amyloidosis Research Consortium and the US Food and Drug Administration Center for Drug Evaluation and Research. In 2020, the Amyloidosis Forum launched an initiative to identify clinical trial endpoints and analytic strategies across affected organ systems and life impacts via specialized working groups. This review summarizes the proceedings of the Statistical Group and proposes a pathway for development and validation of multi-domain endpoints (MDEs) for potential use in AL amyloidosis clinical trials. Specifically, drawing on candidate domain-specific endpoints recommended by each organ-specific working group, different approaches to constructing MDEs were considered. Future studies were identified to assess the validity, meaningfulness and performance of MDEs through use of natural history and clinical trial data. Ultimately, for drug development, the context of use in a regulatory evaluation, the specific patient population, and the investigational therapeutic mechanism should drive selection of appropriate endpoints. MDEs for AL amyloidosis, once developed and validated, will provide important options for advancing patient-focused drug development in this multi-system disease.
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Metadata
- Type
- article
- Language
- en
- Landing Page
- https://doi.org/10.1007/s43441-024-00641-6
- https://link.springer.com/content/pdf/10.1007/s43441-024-00641-6.pdf
- OA Status
- hybrid
- Cited By
- 2
- References
- 46
- Related Works
- 10
- OpenAlex ID
- https://openalex.org/W4394872227
All OpenAlex metadata
Raw OpenAlex JSON
- OpenAlex ID
-
https://openalex.org/W4394872227Canonical identifier for this work in OpenAlex
- DOI
-
https://doi.org/10.1007/s43441-024-00641-6Digital Object Identifier
- Title
-
Pathway for Development and Validation of Multi-domain Endpoints for Amyloid Light Chain (AL) AmyloidosisWork title
- Type
-
articleOpenAlex work type
- Language
-
enPrimary language
- Publication year
-
2024Year of publication
- Publication date
-
2024-04-17Full publication date if available
- Authors
-
James Signorovitch, Jialü Zhang, David T. Brown, Preston Dunnmon, Liang Xiu, Nicolae Done, Kristen Hsu, Yolanda Barbáchano, Isabelle LousadaList of authors in order
- Landing page
-
https://doi.org/10.1007/s43441-024-00641-6Publisher landing page
- PDF URL
-
https://link.springer.com/content/pdf/10.1007/s43441-024-00641-6.pdfDirect link to full text PDF
- Open access
-
YesWhether a free full text is available
- OA status
-
hybridOpen access status per OpenAlex
- OA URL
-
https://link.springer.com/content/pdf/10.1007/s43441-024-00641-6.pdfDirect OA link when available
- Concepts
-
Amyloidosis, Clinical trial, Context (archaeology), Drug development, Medicine, Clinical endpoint, Pharmacy, AL amyloidosis, Drug, Pharmacology, Intensive care medicine, Internal medicine, Immunology, Immunoglobulin light chain, Family medicine, Antibody, Biology, PaleontologyTop concepts (fields/topics) attached by OpenAlex
- Cited by
-
2Total citation count in OpenAlex
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2024: 2Per-year citation counts (last 5 years)
- References (count)
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46Number of works referenced by this work
- Related works (count)
-
10Other works algorithmically related by OpenAlex
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