title: Patient-led research in amyotrophic lateral sclerosis: Quo vadis?
abstract: Patient-led trials are steadily claiming their place in modern neurological research. A popular paper on the effects of lithium in amyotrophic lateral sclerosis included a report on a clinical trial in humans. Subsequently, a follow-up study using patient-reported data did not confirm the original result, opening a scientific debate on how patient-reported data could contribute to neurological research. This review critically illustrates the major benefits and caveats of the current patient-led trials and presents recent U.S. and UK guidelines, which aim to the optimal incorporation of patient-reported outcomes in a reinvigorated research perspective.
authors: a Department of Clinical Neuroscience , Institute of Psychiatry, King's College London , London , UK.
authors: Maria-Ioanna
authors: MI
authors: Stefanou
authors: b University Hospital Zurich , Zurich , Switzerland.
authors: Iakovos
authors: I
authors: Amygdalos
mesh_terms: D000690:Amyotrophic Lateral Sclerosis / Q000628:therapy*; D035843:Biomedical Research / Q000379:methods*; D002986:Clinical Trials as Topic / Q000379:methods*; D006801:Humans; D063868:Patient Outcome Assessment
keywords: ALS; PROs; lithium; patient-led; patient-reported outcomes