Stratification of amyotrophic lateral sclerosis patients: a crowdsourcing approach.

Autor: Kueffner R; Icahn School of Medicine at Mount Sinai, New York, NY, USA. r.m.kueffner@gmail.com., Zach N; Teva Pharmaceuticals, Netanyah, Israel. netazach@gmail.com., Bronfeld M; Prize4Life, Haifa, Israel., Norel R; IBM Research, Yorktown Heights, NY, USA., Atassi N; Massachusetts General Hospital, Boston, MA, USA., Balagurusamy V; IBM Research, Yorktown Heights, NY, USA., Di Camillo B; Information Engineering Department, University of Padova, Padova, Italy., Chio A; University of Turin, Turin, Italy., Cudkowicz M; Massachusetts General Hospital, Boston, MA, USA., Dillenberger D; IBM Research, Yorktown Heights, NY, USA., Garcia-Garcia J; Pompeu Fabra University, Barcelona, Spain., Hardiman O; Institute of Neuroscience, Trinity College, Dublin, Ireland., Hoff B; Sage Bionetworks, Seattle, WA, USA., Knight J; IBM Research, Yorktown Heights, NY, USA., Leitner ML; Accelerating NeuroVentures, Boston, MA, USA., Li G; Amazon, Seattle, WA, USA., Mangravite L; Sage Bionetworks, Seattle, WA, USA., Norman T; Sage Bionetworks, Seattle, WA, USA., Wang L; Zillow, Seattle, WA, USA., Xiao J; Department of Computer Science, University of Illinois at Urbana-Champaign, Champaign, IL, USA., Fang WC; Department of Information and Learning Technology, National University, Tainan City, Taiwan., Peng J; Department of Computer Science, University of Illinois at Urbana-Champaign, Champaign, IL, USA., Yang C; Department of Computer Science and Information Engineering, National University, Tainan City, Taiwan., Chang HJ; Faculty of Information Technology, Monash University, Clayton, Australia., Stolovitzky G; IBM Research, Yorktown Heights, NY, USA.
Jazyk: angličtina
Zdroj: Scientific reports [Sci Rep] 2019 Jan 24; Vol. 9 (1), pp. 690. Date of Electronic Publication: 2019 Jan 24.
DOI: 10.1038/s41598-018-36873-4
Abstrakt: 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.
Databáze: MEDLINE
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