Clinical staging in amyotrophic lateral sclerosis: analysis of Edaravone Study 19
| Autor: | Adriano Chiò, Rebecca Bornheimer, Wendy Agnese, Gerry Oster, Stephen Apple, Charlotte Merrill, Ammar Al-Chalabi |
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| Rok vydání: | 2020 |
| Předmět: |
Male
medicine.medical_specialty Time Factors education Kaplan-Meier Estimate Placebo law.invention 03 medical and health sciences chemistry.chemical_compound 0302 clinical medicine Randomized controlled trial Double-Blind Method law Internal medicine Post-hoc analysis Edaravone medicine Humans 030212 general & internal medicine Amyotrophic lateral sclerosis Stage (cooking) business.industry Disease progression Amyotrophic Lateral Sclerosis Middle Aged medicine.disease Clinical trial Psychiatry and Mental health Neuroprotective Agents chemistry Neuromuscular Disease Progression Surgery Female Neurology (clinical) business 030217 neurology & neurosurgery |
| Zdroj: | Journal of Neurology, Neurosurgery, and Psychiatry |
| ISSN: | 1468-330X |
| Popis: | ObjectiveThis was a post hoc analysis of the Edaravone Phase III Study MCI186-19 (‘Study 19’) to examine the utility of clinical staging systems as end points in clinical trials in amyotrophic lateral sclerosis (ALS).MethodsAmyotrophic Lateral Sclerosis Functional Rating Scale—Revised item scores from Study 19 were retrospectively mapped to King’s stage and Milano-Torino staging (MiToS) stage. We assessed the percentage of patients who experienced progression in King’s and MiToS stages during Study 19. We also assessed disease progression in subgroups of patients according to baseline King’s stage.ResultsDuring double-blind treatment, the percentage of patients who experienced a progression in King’s stage was lower for edaravone (42.0%, 95% CI 30.4% to 53.6%) than placebo (55.9%, 95% CI 44.1% to 67.6%). The most pronounced effect was noted among patients who were in stage 1 and was maintained throughout open-label treatment. An analysis of a ≥2-stage progression in MiToS stage showed no difference between treatment arms during double-blind treatment, but during the open-label period, more rapid progression was noted among patients in the placebo–edaravone arm than among those in the edaravone–edaravone arm (log-rank test, pConclusionsThe King’s and MiToS staging systems provided utility in assessing clinical progression in Edaravone Study 19. These findings may support the use of staging systems as end points in ALS clinical trials and to understand the timing of benefit as measured by these scales. |
| Databáze: | OpenAIRE |
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