Prodromal Marker Progression in Idiopathic Rapid Eye Movement Sleep Behavior Disorder: Sample Size for Clinical Trials
| Autor: | Ronald B. Postuma, Amélie Pelletier, Fatimah Alotaibi, Jean-François Gagnon, Jacques Montplaisir |
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| Rok vydání: | 2019 |
| Předmět: |
Male
0301 basic medicine medicine.medical_specialty Endpoint Determination Polysomnography Rapid eye movement sleep Prodromal Symptoms REM Sleep Behavior Disorder Disease Autonomic Nervous System Cohort Studies 03 medical and health sciences Behavior disorder Cognition 0302 clinical medicine Physical medicine and rehabilitation medicine Humans Dementia Prospective Studies Cognitive decline Aged Clinical Trials as Topic business.industry Parkinson Disease Middle Aged medicine.disease 3. Good health Clinical trial 030104 developmental biology Neurology Sample size determination Sample Size Disease Progression Female Neurology (clinical) business Psychomotor Performance 030217 neurology & neurosurgery |
| Zdroj: | Movement Disorders. 34:1914-1919 |
| ISSN: | 1531-8257 0885-3185 |
| Popis: | Objective To estimate prodromal marker progression in idiopathic rapid eye movement sleep behavior disorder patients with prodromal Parkinson's disease (PD) to calculate sample size for neuroprotective trials. Methods Patients with polysomnogram-proven idiopathic rapid eye movement sleep behavior disorder were assessed for prodromal PD using Movement Disorder Society criteria. We prospectively measured progression rates of numerous clinical variables, including motor, cognitive, special sensory, and autonomic variables and calculated the sample size required to demonstrate slowing of progression under 3 effectiveness assumptions (30%, 50%, and 70% slowing). Results Overall, the variables that progressed with lowest sample size requirements were motor variables (234 participants required per group for 50% efficacy over 2 years). By contrast, cognitive, special sensory, and autonomic variables showed modest progression with high variability, resulting in high sample sizes. The most efficient design was a time-to-event analysis using milestones of motor and cognitive decline (126 per group). Conclusion In idiopathic rapid eye movement sleep behavior disorder, time-to-event analysis assessing milestones of decline is the most efficient trial design for neuroprotective therapy. © 2019 International Parkinson and Movement Disorder Society. |
| Databáze: | OpenAIRE |
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