Multivariate Survival Analysis of a Comprehensive Clinical Trial of Rapamycin in Amyotrophic Lateral Sclerosis Explores Prognostic Factors and Survival Patterns in C9orf72 Carriers
What this study shows
Secondary, observational survival analysis of the RAP-ALS rapamycin trial (n=63) finds a protective association between rapamycin treatment and survival in C9orf72 mutation carriers (log-rank p=0.026), consistent with a comparison against an independent C9orf72+ cohort (n=40; RMST p=0.04); confounding by indication is not excluded, and the authors flag this as exploratory given the small treated-carrier subgroup (n=6).
Abstract
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Amyotrophic lateral sclerosis (ALS) is a progressive neurodegenerative disease marked by considerable variability in survival times. This retrospective study aims to evaluate the prognostic value of a broad range of variables by conducting a comprehensive survival analysis on longitudinal data from RAP-ALS, a clinical trial investigating the effects of rapamycin in ALS patients (n = 63). Covariates were classified as risk or protective factors according to their hazard ratios. Regularized Cox regression was utilized to select the best-performing multivariate models in cross-validation.
Read the full abstract on PubMed →At a glance
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| Evidence type | H Human study Marked H because it is direct evidence from a human clinical trial or human cohort; the code names the kind of study, not its quality -- a small, well-run trial is still H. |
| Study type | 3 - Human Observational |
| Model system | Human (ALS patients, RAP-ALS trial, n=63) |
| Journal | European Journal of Neurology |
| Year | 2026 |
| Peer reviewed | Yes |
| Record last updated | 2026-10-01 |
| Source | DOI 10.1111/ene.70756 · PMID 42760861 |
Where this study fits
How this study connects to the pathway map, open questions, people and other studies in the Atlas.
People in the Atlas
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Mathematical modeller in Trento who applies survival and epidemic models to medical data, and first author of the 2026 re-analysis of the RAP-ALS rapamycin trial in ALS
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Computational biologist building quantitative systems-pharmacology and disease models; senior author on a 2026 survival analysis of the RAP-ALS rapamycin trial
More: 6 other authors
No profile yet: Paris A, Lauria M, Martinelli I, Zucchi E, Simonini C, Mandrioli J
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Topics
Extracted findings
These fields were extracted from the paper with AI assistance and have not been checked against the source. Read them as a pointer into the paper, not as curated claims; where they disagree with the curated summary above, the curated summary stands.
| Intervention | Rapamycin (RAP-ALS trial), post hoc survival analysis |
| Target | mTOR |
| Model | Human (ALS patients, n=63; treated C9orf72 carriers n=6) |
| Effect | Exploratory association between rapamycin treatment and longer survival in C9orf72 carriers; confounding not excluded. |
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