{"id":"doi:10.1016/j.mayocpiqo.2026.100739","kind":"article","title":"Tofersen Treatment in <i>SOD1</i>-ALS: Real-World Evidence from a Retrospective Multicenter Study in France (FORSLA Study)","authors":["Erazo D","Hesters A","Bruneteau G","De La Cruz E","Bernard E","Grapperon AM","Le Masson G","Cintas P","Soriani MH","Guy N","Cassereau J","Camdessanché JP","Genestet S","Corcia P","Pittion S","Danel V","Filliatre M","Choumert A","Jacquin-Piques A","Salachas F","Esselin F","Bohic A","Lautrette G","Attarian S","Catteau J"],"author_count":31,"year":2026,"published":"2026-07-27","venue":"Mayo Clinic proceedings. Innovations, quality & outcomes","cited_by":1,"doi":"10.1016/j.mayocpiqo.2026.100739","arxiv_id":null,"nct_id":null,"source_url":"https://doi.org/10.1016/j.mayocpiqo.2026.100739","archive_url":"https://space-kitty.com/papers/doi:10.1016/j.mayocpiqo.2026.100739","updated":null,"pmid":"42548788","pmcid":"PMC13429897","openalex_id":null,"pdf_url":"https://europepmc.org/articles/PMC13429897/pdf","abstract":"Objective To evaluate the effectiveness of Tofersen in patients with superoxide dismutase 1 gene ( SOD1-ALS ) patients in France in a real-world setting, using disease progression within patient comparisons and with a historical cohort. Patients and methods Patients with SOD1 -ALS were included from across 19 French FILSLAN network centers. Baseline was defined as the treatment initiation date. Main endpoints were the ALSFRS-R progression rate and plasmatic neurofilament light chain (NfL) levels at baseline and 12 months after baseline. Results In the Tofersen Cohort (N=46), within-group comparisons showed that the mean ALS functional rating scale revised (ALSFRS-R) progression rate slowed from 0.53 ± 0.5 at baseline to 0.22 ± 0.3 point/month at 12 months ( P =.006). NfL levels significantly decreased from 89.0 ± 9.0 pg/ml at baseline to 29.2 ± 19.5.5 at 12 months ( P =.004). Exploratory comparisons with a propensity score (PS) matched historical cohort (39 matched pairs) using a mixed-effects model, ALSFRS-R progression rate at baseline, 6 months, and 12 months after baseline, showed no statistically significant differences between groups P =.30, whereas longitudinal ALSFRS-R scores differed significantly between groups (time-treatment interaction P =.006). The mean survival of the PS matched population was longer in the Tofersen Cohort (42.6 months) than the Historical Cohort (31.8 months) P =.004. Time-dependent adjusted cox analysis showed that Tofersen was associated with a reduction in mortality risk (adjusted HR=0.34; 95% CI, 0.12-0.91; P =.03). Conclusion Tofersen seems to be associated with slower functional decline and reduced NfL levels. While limitations of retrospective design and ALSFRS-R sensitivity must be acknowledged, these findings provide real-world evidence suggesting a clinical benefit of Tofersen.","abstract_license":"CC-BY (article license as recorded by Europe PMC; abstract reproduced unmodified apart from markup removal)","license":"CC-BY","is_open_access":true,"retracted":false,"peer_reviewed":null,"categories":[],"details":null,"provenance":"europepmc","attribution":"Abstract of \"Tofersen Treatment in <i>SOD1</i>-ALS: Real-World Evidence from a Retrospective Multicenter Study in France (FORSLA Study)\" by Erazo D, Hesters A, Bruneteau G et al., https://doi.org/10.1016/j.mayocpiqo.2026.100739, used under its Creative Commons Attribution license (CC-BY); markup removed, otherwise unmodified.","topics":[{"slug":"neurodegeneration","name":"Alzheimer's, Parkinson's & Neurodegeneration","domain_slug":"medicine"}],"cited_in_dossiers":[]}