Disability improvement as a clinically relevant outcome in clinical trials of relapsing forms of multiple sclerosis.
| Autor: | Cree BA; UCSF Weill Institute for Neurosciences, Department of Neurology, University of California San Francisco, San Francisco, CA, USA., Cohen JA; Department of Neurology, Mellen Center for Multiple Sclerosis Treatment and Research, Neurological Institute, Cleveland Clinic, Cleveland, OH, USA., Reder AT; Department of Neurology, University of Chicago, Chicago, IL, USA., Tomic D; Novartis Pharma AG, Basel, Switzerland., Silva D; Novartis Pharma AG, Basel, Switzerland., Piani Meier D; Novartis Pharma AG, Basel, Switzerland., Laflamme AK; Novartis Pharma AG, Basel, Switzerland., Ritter S; Novartis Pharmaceuticals Corporation, East Hanover, NJ, USA., Leppert D; Novartis Pharma AG, Basel, Switzerland., Kappos L; Research Center for clinical Neuroimmunology and Neuroscience Basel (RC2NB) and MS Center, Departments of Medicine, Clinical Research, Biomedicine, and Biomedical Engineering, University Hospital, University of Basel, Basel, Switzerland. |
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| Jazyk: | angličtina |
| Zdroj: | Multiple sclerosis (Houndmills, Basingstoke, England) [Mult Scler] 2021 Dec; Vol. 27 (14), pp. 2219-2231. Date of Electronic Publication: 2021 Mar 26. |
| DOI: | 10.1177/13524585211000280 |
| Abstrakt: | Background: Disease-modifying therapies (DMTs) can reduce the risk of disability worsening in patients with relapsing forms of multiple sclerosis (RMS). High-efficacy DMTs can lead to confirmed or sustained disability improvement (CDI and SDI). Objective and Methods: Post hoc analyses of data from the TRANSFORMS, FREEDOMS, and FREEDOMS II trials and their extensions assessed the effects of fingolimod (0.5-1.25 mg/day) on stabilizing or improving disability over ⩽8 years in participants with RMS. CDI and SDI rates were compared between participants initially randomized to fingolimod, interferon (IFNβ-1a), or placebo. Results: At 8 years' follow-up in TRANSFORMS, 35.1% (95% confidence interval [CI], 28.2%-43.1%) of assessed participants in the IFNβ-1a-fingolimod switch group and 41.9% (36.6%-47.6%) on continuous fingolimod experienced CDI; disability did not worsen in approximately 70%. Similar results were seen in the combined FREEDOMS population. Proportionally fewer TRANSFORMS participants achieved SDI in the IFNβ-1a-fingolimod switch group than on continuous fingolimod (5.4% [3.0%-9.5%] vs 14.2% [10.8%-18.4%], p = 0.01). Conclusion: CDI and SDI are outcomes of interest for clinical trials and for long-term follow-up of participants with RMS. Monitoring CDI and SDI in addition to disability worsening may facilitate understanding of the therapeutic benefit of RMS treatments. |
| Databáze: | MEDLINE |
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