Data di Pubblicazione:
2019
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 subpopulations, and to accelerate disease understanding and therapeutic development.
Tipologia CRIS:
03A-Articolo su Rivista
Elenco autori:
Kueffner R.; Zach N.; Bronfeld M.; Norel R.; Atassi N.; Balagurusamy V.; Di Camillo B.; Chio A.; Cudkowicz M.; Dillenberger D.; Garcia-Garcia J.; Hardiman O.; Hoff B.; Knight J.; Leitner M.L.; Li G.; Mangravite L.; Norman T.; Wang L.; Xiao J.; Fang W.-C.; Peng J.; Yang C.; Chang H.-J.; Stolovitzky G.; Alkallas R.; Anghel C.; Avril J.; Bacardit J.; Balser B.; Balser J.; Bar-Sinai Y.; Ben-David N.; Ben-Zion E.; Bliss R.; Cai J.; Chernyshev A.; Chiang J.-H.; Chicco D.; Corriveau B.A.N.; Dai J.; Deshpande Y.; Desplats E.; Durgin J.S.; Espiritu S.M.G.; Fan F.; Fevrier P.; Fridley B.L.; Godzik A.; Golinska A.; Gordon J.; Graw S.; Guo Y.; Herpelinck T.; Hopkins J.; Huang B.; Jacobsen J.; Jahandideh S.; Jeon J.; Ji W.; Jung K.; Karanevich A.; Koestler D.C.; Kozak M.; Kurz C.; Lalansingh C.; Larrieu T.; Lazzarini N.; Lerner B.; Lesinski W.; Liang X.; Lin X.; Lowe J.; Mackey L.; Meier R.; Min W.; Mnich K.; Nahmias V.; Noel-Macdonnell J.; O'donnell A.; Paadre S.; Park J.; Polewko-Klim A.; Raghavan R.; Rudnicki W.; Saghapour E.; Salomond J.-B.; Sankaran K.; Sendorek D.; Sharan V.; Shiah Y.-J.; Sirois J.-K.; Sumanaweera D.N.; Usset J.; Vang Y.S.; Vens C.; Wadden D.; Wang D.; Wong W.C.; Xie X.; Xu Z.; Yang H.-T.; Yu X.; Zhang H.; Zhang L.; Zhang S.; Zhu S.
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