Christian Lunetta1, Cristina Moglia2,3, Andrea Lizio1, Claudia Caponnetto4, Raffaele Dubbioso5, Fabio Giannini6, Sabrina Matà7, Letizia Mazzini8, Mario Sabatelli9,10, Gabriele Siciliano11, Isabella Laura Simone12, Gianni Sorarù13, Antonella Toriello14, Francesca Trojsi15, Marcella Vedovello16, Fabrizio D'Ovidio2, Massimo Filippi17,18, Andrea Calvo19,20. 1. NEMO (NeuroMuscular Omnicentre) Clinical Center Milano, Fondazione Serena Onlus, Milan, Italy. 2. "Rita Levi Montalcini" Department of Neuroscience, ALS Centre, University of Turin, Via Cherasco 15, 10126, Turin, Italy. 3. Azienda Ospedaliero-Universitaria Città della Salute e della Scienza di Torino, Turin, Italy. 4. Department of Neurosciences, Rehabilitation Ophtalmology, Genetics, Mother and Child Disease, IRCCS Ospedale Policlinico San Martino, Genova, Italy. 5. Department of Neurosciences, Reproductive Sciences and Odontostomatology, University of Naples Federico II, Naples, Italy. 6. Department of Medical and Surgery Sciences and Neurosciences, University of Siena, Siena, Italy. 7. Dipartimento Neuromuscoloscheletrico e degli Organi di Senso of Neurological and Psychiatric Sciences, AOU of Careggi, Florence, Italy. 8. ALS Centre, Neurologic Clinic, University Hospital "Maggiore della Carità", Novara, Italy. 9. IRCCS Foundation Policlinico A. Gemelli, Rome, Italy. 10. NEMO Clinical Center Roma, Fondazione Serena Onlus-Pol. A. Gemelli, Rome, Italy. 11. Department of Clinical and Experimental Medicine, Neurological Clinic, University Hospital of Pisa, Pisa, Italy. 12. Department of Medical Sciences and Neuroscience, Policlinic, University "Aldo Moro" of Bari, Bari, Italy. 13. Department of Neurosciences, University of Padua, Padua, Italy. 14. Operative Unit of Neurology, ALS Center, AOU "San Giovanni di Dio e Ruggi d'Aragona", Salerno, Italy. 15. Department of Advanced Medical and Surgical Sciences, University of Campania "Luigi Vanvitelli", Naples, Italy. 16. Neurology Unit, ASST Papa Giovanni XXIII, Bergamo, Italy. 17. Department of Neurology and Clinical Neurophysiology, IRCCS "San Raffaele Scientific Institute", Milan, Italy. 18. Neuroimaging Research Unit, Division of Neuroscience, San Raffaele Scientific Institute, Vita-Salute San Raffaele University, Milan, Italy. 19. "Rita Levi Montalcini" Department of Neuroscience, ALS Centre, University of Turin, Via Cherasco 15, 10126, Turin, Italy. andrea.calvo@unito.it. 20. Azienda Ospedaliero-Universitaria Città della Salute e della Scienza di Torino, Turin, Italy. andrea.calvo@unito.it.
Abstract
OBJECTIVES: The aim of the study is to analyze the ALS disease progression and respiratory function of Italian patients treated with edaravone (EVN), as well as the adherence to, and the effects of, the therapy. METHODS: We performed an observational study of patients treated with EVN from May 2017 to May 2019, in 39 Italian ALS Centers. Taking into account ALS patients with at least 12 months of EVN treatment, we compared the decline of ALSFRS-R and FVC with a group of matched historical controls from the Pooled Resource Open-Access ALS Clinical Trials (PRO-ACT) database, using both descriptive and survival analysis approaches. RESULTS: A total of 331 ALS Italian patients treated with EVN and 290 matched historical controls were recruited in this study. No significant differences on disease progression or respiratory function were found comparing the two cohorts in both descriptive and survival analyses. The EVN treatment was overall well tolerated. CONCLUSIONS: The study showed that EVN treatment was well tolerated. No significant differences were reported in ALS patients treated and not treated with EVN, in terms of both disease progression and respiratory function. These findings prove that further studies are required to better clarify whether EVN could be considered an effective treatment for ALS disease.
OBJECTIVES: The aim of the study is to analyze the ALS disease progression and respiratory function of Italian patients treated with edaravone (EVN), as well as the adherence to, and the effects of, the therapy. METHODS: We performed an observational study of patients treated with EVN from May 2017 to May 2019, in 39 Italian ALS Centers. Taking into account ALSpatients with at least 12 months of EVN treatment, we compared the decline of ALSFRS-R and FVC with a group of matched historical controls from the Pooled Resource Open-Access ALS Clinical Trials (PRO-ACT) database, using both descriptive and survival analysis approaches. RESULTS: A total of 331 ALS Italian patients treated with EVN and 290 matched historical controls were recruited in this study. No significant differences on disease progression or respiratory function were found comparing the two cohorts in both descriptive and survival analyses. The EVN treatment was overall well tolerated. CONCLUSIONS: The study showed that EVN treatment was well tolerated. No significant differences were reported in ALSpatients treated and not treated with EVN, in terms of both disease progression and respiratory function. These findings prove that further studies are required to better clarify whether EVN could be considered an effective treatment for ALS disease.
Entities:
Keywords:
Amyotrophic lateral sclerosis; Edaravone; Multicenter study; Observational study
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