Stratification of amyotrophic lateral sclerosis patients: a crowdsourcing approach

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  • dc.contributor.author Kueffner, Robert
  • dc.contributor.author García-García, Javier, 1982-
  • dc.contributor.author Stolovitzky, Gustavo
  • dc.date.accessioned 2020-07-21T06:32:08Z
  • dc.date.available 2020-07-21T06:32:08Z
  • dc.date.issued 2019
  • dc.description.abstract Amyotrophic lateral sclerosis (ALS) is a fatal neurodegenerative disease where substantial heterogeneity in clinical presentation urgently requires a better stratification of patients for the development of drug trials and clinical care. In this study we explored stratification through a crowdsourcing approach, the DREAM Prize4Life ALS Stratification Challenge. Using data from >10,000 patients from ALS clinical trials and 1479 patients from community-based patient registers, more than 30 teams developed new approaches for machine learning and clustering, outperforming the best current predictions of disease outcome. We propose a new method to integrate and analyze patient clusters across methods, showing a clear pattern of consistent and clinically relevant sub-groups of patients that also enabled the reliable classification of new patients. Our analyses reveal novel insights in ALS and describe for the first time the potential of a crowdsourcing to uncover hidden patient sub-populations, and to accelerate disease understanding and therapeutic development.
  • dc.format.mimetype application/pdf
  • dc.identifier.citation Kueffner R, Zach N, Bronfeld M, Norel R, Atassi N, Balagurusamy V et al. Stratification of amyotrophic lateral sclerosis patients: a crowdsourcing approach. Sci Rep. 2019; 9(1):690. DOI: 10.1038/s41598-018-36873-4
  • dc.identifier.doi http://dx.doi.org/10.1038/s41598-018-36873-4
  • dc.identifier.issn 2045-2322
  • dc.identifier.uri http://hdl.handle.net/10230/45136
  • dc.language.iso eng
  • dc.publisher Nature Research
  • dc.relation.ispartof Sci Rep. 2019; 9(1):690
  • dc.rights © The Author(s) 2019. Open Access This article is licensed under a Creative Commons Attribution 4.0 International License, which permits use, sharing, adaptation, distribution and reproduction in any medium or format, as long as you give appropriate credit to the original author(s) and the source, provide a link to the Creative Commons license, and indicate if changes were made. The images or other third party material in this article are included in the article’s Creative Commons license, unless indicated otherwise in a credit line to the material. If material is not included in the article’s Creative Commons license and your intended use is not permitted by statutory regulation or exceeds the permitted use, you will need to obtain permission directly from the copyright holder. To view a copy of this license, visit http://creativecommons.org/licenses/by/4.0/.
  • dc.rights.accessRights info:eu-repo/semantics/openAccess
  • dc.rights.uri http://creativecommons.org/licenses/by/4.0/
  • dc.subject.keyword Amyotrophic lateral sclerosis
  • dc.subject.keyword Machine learning
  • dc.subject.keyword Predictive medicine
  • dc.title Stratification of amyotrophic lateral sclerosis patients: a crowdsourcing approach
  • dc.type info:eu-repo/semantics/article
  • dc.type.version info:eu-repo/semantics/publishedVersion