Evidence map›Paper›PMID 41974001›Full record

ArticleAmyotrophic lateral sclerosis & frontotemporal degeneration2026

Development of a machine learning-based survival prediction model for ALS inclusive of the advanced-stage population.

Danielle Beaulieu, Kelly Smith, Campbell Ross, Sylvia Yip, Tania C Felizardo, Christina Fournier, Jonathan D Glass, James D Berry, Daniel H Fowler, David L Ennist and 1 more

Abstract read
In one paragraph

Article in Amyotrophic lateral sclerosis & frontotemporal degeneration, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Not yet cited in PubMed.

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1 · What the graph read from it

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2 · The registry

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3 · Its place in the literature

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4 · The record

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5 · Who and what money

Authors and funding

11 authors.

Danielle BeaulieuOrigent Data Sciences, Washington, DC, USA.
Kelly SmithOrigent Data Sciences, Washington, DC, USA.
Campbell RossRapa Therapeutics, Rockville, MD, USA.
Sylvia YipRapa Therapeutics, Rockville, MD, USA.
Tania C FelizardoRapa Therapeutics, Rockville, MD, USA.
Christina FournierEmory University, Atlanta, GA, USA, and.
Jonathan D GlassEmory University, Atlanta, GA, USA, and.
James D BerryMass General Hospital, Boston, MA, USA.
Daniel H FowlerRapa Therapeutics, Rockville, MD, USA.
David L EnnistOrigent Data Sciences, Washington, DC, USA.
Pooled Resource Open-Access ALS Clinical Trials Consortium

Funding

Intermediate-Size Expanded Access Trial of Autologous Hybrid TREG/Th2 Cell Therapy (RAPA-501) of Amyotrophic Lateral SclerosisU01NS136020 · NINDS · MASSACHUSETTS GENERAL HOSPITAL · PI BABU, SUMA, BERRY, JAMES DALE · 2023 to 2025
$33.0M
NINDS NIH HHS U01 NS136020
6 · The paper itself

Abstract

objectiveDevelop a machine learning-based model for survival prediction in ALS, including advanced-stage patients (≤50% predicted normal vital capacity [VC

methodsTraining data from the PRO-ACT Database (

resultsBaseline characteristics with the highest RI for driving survival predictions included: VC% slope (20.2%); age (12.4%); VC% (9.9%); VC(L) (7.5%); ALSFRS-R (6.6%); and ALSFRS-R slope (5.1%). Model performance upon external validation was satisfactory for both discrimination (C-index, 0.709 [95% CI, 0.671-0.746]) and calibration (calibration-in-the-large, 0.083 [95% CI, -0.073-0.232]; calibration slope, 0.992 [95% CI, 0.789-1.198]). At 8-months from baseline, the model successfully stratified patients by survival prognosis, with low-, average-, and high-risk population tertiles having observed median survival probabilities of 85, 69, and 43%, respectively.

conclusionsThis model accurately predicts survival prognosis in ALS, including patients with severely impaired respiratory function. This new understanding of patient-specific factors that drive survival prognostication will be invaluable for reducing patient heterogeneity in clinical trials evaluating novel therapeutic modalities in early- and advanced-stage ALS.

Indexed as

Amyotrophic Lateral SclerosisMachine LearningAgedBoosting Machine Learning AlgorithmsClassification AlgorithmsFemaleHumansKaplan-Meier EstimateMaleMiddle AgedPrediction AlgorithmsPredictive Learning ModelsPrognosisRandom ForestAmyotrophic lateral sclerosisgradient boosting machine modelrespiratory insufficiencysurvivalvital capacity

Identifiers

PMID41974001
PMCPMC13138003

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Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.