The Right Therapy for Neurological Disorders: From Randomized Trials to Clinical Practice
Most neurological disorders are chronic and aging-related. With the increase of life expectancy their incidence and prevalence will grow in the decades to come, which in turn will increase the load on medical and social systems worldwide. There is thus a desperate need for successful preventive and therapeutic measures based on randomized clinical trials (RTCs) conducted by independent organizations.
This book provides a compendium relating most of the principles of reliable RTCs to specific neurological diseases. Contributed by specialized neurologists, the articles touch on important aspects of RCTs with a clear critical approach, highlighting their limitations as well as giving recommendations for their planning and conducting to address the variable genotypic and phenotypic aspects of neurological conditions. Consideration is also given to combining the clinical impact of the study results with patients’ values and the interests of pharmaceutical companies.
Neurologists involved in clinical trials will certainly benefit from this book, which should become a basic text for all neurological courses dealing with evidence-based neurology.
Download citation file:
Digital Version
Print Version
Table of Contents
-
1 - 7: The Basic Structure of a Randomized Clinical TrialByEttore BeghiEttore BeghiLaboratory of Neurological Disorders, IRCCS-Mario Negri Institute for Pharmacological Research, Milan, ItalySearch for other works by this author on:
-
8 - 23: Peculiarities of Neurological Disorders and Study DesignsByEttore Beghi;Ettore BeghiLaboratory of Neurological Disorders, IRCCS-Mario Negri Institute for Pharmacological Research, Milan, ItalySearch for other works by this author on:Elisabetta Pupillo;Elisabetta PupilloLaboratory of Neurological Disorders, IRCCS-Mario Negri Institute for Pharmacological Research, Milan, ItalySearch for other works by this author on:Giorgia GiussaniGiorgia GiussaniLaboratory of Neurological Disorders, IRCCS-Mario Negri Institute for Pharmacological Research, Milan, ItalySearch for other works by this author on:
-
24 - 36: Current Issues in Randomized Clinical Trials of Neurodegenerative Disorders at Enrolment and Reporting: Diagnosis, Recruitment, Representativeness of Patients, Ethnicity, and Quality of ReportingByGiancarlo Logroscino;Giancarlo LogroscinoaDepartment of Basic Medical Sciences, Neuroscience and Sense Organs, University of Bari ‘Aldo Moro', Bari, andbUnit of Neurodegenerative Diseases, Department of Clinical Research in Neurology, University of Bari ‘Aldo Moro', ‘Pia Fondazione Cardinale G. Panico', Tricase, Italy;Search for other works by this author on:Rosa Capozzo;Rosa CapozzoaDepartment of Basic Medical Sciences, Neuroscience and Sense Organs, University of Bari ‘Aldo Moro', Bari, andbUnit of Neurodegenerative Diseases, Department of Clinical Research in Neurology, University of Bari ‘Aldo Moro', ‘Pia Fondazione Cardinale G. Panico', Tricase, Italy;Search for other works by this author on:Rosanna Tortelli;Rosanna TortelliaDepartment of Basic Medical Sciences, Neuroscience and Sense Organs, University of Bari ‘Aldo Moro', Bari, andbUnit of Neurodegenerative Diseases, Department of Clinical Research in Neurology, University of Bari ‘Aldo Moro', ‘Pia Fondazione Cardinale G. Panico', Tricase, Italy;Search for other works by this author on:Benoît MarinBenoît MarinaDepartment of Basic Medical Sciences, Neuroscience and Sense Organs, University of Bari ‘Aldo Moro', Bari, andbUnit of Neurodegenerative Diseases, Department of Clinical Research in Neurology, University of Bari ‘Aldo Moro', ‘Pia Fondazione Cardinale G. Panico', Tricase, Italy;cINSERM, U1094, Tropical Neuroepidemiology,dUMR_S 1094, Tropical Neuroepidemiology, Institute of Neuroepidemiology and Tropical Neurology, CNRS FR 3503 GEIST, University of Limoges, andeCentre d'Epidémiologie de Biostatistique et de Méthodologie de la Recherche, CHU Limoges, Limoges, FranceSearch for other works by this author on:
-
37 - 49: How to Distinguish between Statistically Significant Results and Clinically Relevant ResultsByDerrick A. BennettDerrick A. BennettClinical Trial Service Unit and Epidemiological Studies Unit, Nuffield Department of Population Health, University of Oxford, Oxford, UKSearch for other works by this author on:
-
50 - 59: Modeling and Prediction in Neurological Disorders: The Biostatistical PerspectiveByMassimiliano Copetti;Massimiliano CopettiaUnit of Biostatistics, IRCSS Casa Sollievo della Sofferenza, San Giovanni Rotondo, Italy;Search for other works by this author on:Andrea Fontana;Andrea FontanaaUnit of Biostatistics, IRCSS Casa Sollievo della Sofferenza, San Giovanni Rotondo, Italy;Search for other works by this author on:Fabio PellegriniFabio PellegrinibWorldwide Medical, Biogen Idec Inc., Cambridge, Mass., USASearch for other works by this author on:
-
60 - 70: Composite Scores and Other Outcome Measures in Stroke TrialsByFrancesca Pistoia;Francesca PistoiaNeurological Institute, Department of Biotechnological and Applied Clinical Sciences, University of L'Aquila, L'Aquila, ItalySearch for other works by this author on:Simona Sacco;Simona SaccoNeurological Institute, Department of Biotechnological and Applied Clinical Sciences, University of L'Aquila, L'Aquila, ItalySearch for other works by this author on:Raffaele Ornello;Raffaele OrnelloNeurological Institute, Department of Biotechnological and Applied Clinical Sciences, University of L'Aquila, L'Aquila, ItalySearch for other works by this author on:Diana Degan;Diana DeganNeurological Institute, Department of Biotechnological and Applied Clinical Sciences, University of L'Aquila, L'Aquila, ItalySearch for other works by this author on:Cindy Tiseo;Cindy TiseoNeurological Institute, Department of Biotechnological and Applied Clinical Sciences, University of L'Aquila, L'Aquila, ItalySearch for other works by this author on:Antonio CaroleiAntonio CaroleiNeurological Institute, Department of Biotechnological and Applied Clinical Sciences, University of L'Aquila, L'Aquila, ItalySearch for other works by this author on:
-
71 - 80: Age, Comorbidity, Frailty in Observational and Analytic Studies of Neurological DiseasesByJan Novy;Jan NovyaDepartment of Clinical Neurosciences, Centre Hospitalier Universitaire Vaudois (CHUV) and University of Lausanne, Lausanne, Switzerland;Search for other works by this author on:Josemir W. SanderJosemir W. SanderbNIHR University College London Hospitals Biomedical Research Centre, Department of Clinical and Experimental Epilepsy, UCL Institute of Neurology, London, andcEpilepsy Society, Chalfont St. Peter, UK;dStichting Epilepsie Instellingen Nederland (SEIN), Heemstede, The NetherlandsSearch for other works by this author on:
-
81 - 92: Disease Course, Outcome Measures, and Prognostic Predictors in Epilepsy: Opportunities for Improving Outcome of Drug TrialsByDieter SchmidtDieter SchmidtEpilepsy Research Group, Berlin, GermanySearch for other works by this author on:
-
93 - 100: Assessing Functional Decline in Neurological Diseases Clinical Trials: Duration of Follow-Up - The Case of Multiple SclerosisByFilippo Martinelli Boneschi;Filippo Martinelli BoneschiaDepartment of Neurology and Neurorehabilitation andbLaboratory of Human Genetics of Neurological Disorders, Division of Neuroscience, Institute of Experimental Neurology (INSPE), San Raffaele Scientific Institute, Milan, ItalySearch for other works by this author on:Giancarlo ComiGiancarlo ComiaDepartment of Neurology and Neurorehabilitation andSearch for other works by this author on:
-
101 - 108: Biomarkers in Randomized Clinical Trials: Magnetic Resonance ImagingByJennifer L. WhitwellJennifer L. WhitwellMayo Clinic, Rochester, Minn., USASearch for other works by this author on:
-
109 - 116: Biomarkers in Randomized Clinical Trials: Positron Emission Tomography and Nuclear Medicine TechniquesByEmily SternEmily SternbRadiology, andcPsychiatry, Brigham and Women's Hospital, Harvard Medical School, Boston, Mass., USASearch for other works by this author on:
-
117 - 123: Cerebrospinal Fluid Biomarkers for Target Engagement and Efficacy in Clinical Trials for Alzheimer's and Parkinson's DiseasesByLucilla Parnetti;Lucilla ParnettiaSection of Neurology, Centre for Memory Disturbances, Ospedale S. Maria della Misericordia, Department of Medicine, University of Perugia, Perugia, Italy;Search for other works by this author on:Paolo Eusebi;Paolo EusebiaSection of Neurology, Centre for Memory Disturbances, Ospedale S. Maria della Misericordia, Department of Medicine, University of Perugia, Perugia, Italy;Search for other works by this author on:Alberto LleóAlberto LleóbMemory Unit, Department of Neurology, Hospital de Sant Pau, Barcelona, andcCentro de Investigación Biomédica en Red en enfermedades Neurodegenerativas (CIBERNED), Madrid, SpainSearch for other works by this author on:
-
124 - 135: Pharmacogenetics in Neurodegenerative Diseases: Implications for Clinical TrialsByRosanna Tortelli;Rosanna TortelliaNeurodegenerative Disease Unit, Department of Basic Medicine, Neuroscience and Sense Organs, University of Bari ‘Aldo Moro', Bari,bDepartment of Clinical Research in Neurology, University of Bari ‘Aldo Moro', ‘Pia Fondazione Cardinale G. Panico', Tricase,Search for other works by this author on:Davide Seripa;Davide SeripacGeriatric Unit and Laboratory of Gerontology and Geriatrics, Department of Medical Sciences, IRCCS ‘Casa Sollievo della Sofferenza', San Giovanni Rotondo, andSearch for other works by this author on:Francesco Panza;Francesco PanzaaNeurodegenerative Disease Unit, Department of Basic Medicine, Neuroscience and Sense Organs, University of Bari ‘Aldo Moro', Bari,bDepartment of Clinical Research in Neurology, University of Bari ‘Aldo Moro', ‘Pia Fondazione Cardinale G. Panico', Tricase,cGeriatric Unit and Laboratory of Gerontology and Geriatrics, Department of Medical Sciences, IRCCS ‘Casa Sollievo della Sofferenza', San Giovanni Rotondo, andSearch for other works by this author on:Vincenzo Solfrizzi;Vincenzo SolfrizzidGeriatric Medicine-Memory Unit and Rare Disease Centre, University of Bari ‘Aldo Moro', Bari, ItalySearch for other works by this author on:Giancarlo LogroscinoGiancarlo LogroscinoaNeurodegenerative Disease Unit, Department of Basic Medicine, Neuroscience and Sense Organs, University of Bari ‘Aldo Moro', Bari,bDepartment of Clinical Research in Neurology, University of Bari ‘Aldo Moro', ‘Pia Fondazione Cardinale G. Panico', Tricase,Search for other works by this author on:
-
136 - 146: Randomized Trials in Developing Countries: Different Priorities and Study DesignByBenoît Marin;Benoît MarinaInserm U1094, Tropical Neuroepidemiology, andbUniversity of Limoges, UMR_S 1094, Tropical Neuroepidemiology, Institute of Neuroepidemiology and Tropical Neurology, CNRS FR 3503 GEIST, Limoges, FranceSearch for other works by this author on:Gino Cédric Agbota;Gino Cédric AgbotaaInserm U1094, Tropical Neuroepidemiology, andbUniversity of Limoges, UMR_S 1094, Tropical Neuroepidemiology, Institute of Neuroepidemiology and Tropical Neurology, CNRS FR 3503 GEIST, Limoges, FranceSearch for other works by this author on:Pierre-Marie Preux;Pierre-Marie PreuxaInserm U1094, Tropical Neuroepidemiology, andbUniversity of Limoges, UMR_S 1094, Tropical Neuroepidemiology, Institute of Neuroepidemiology and Tropical Neurology, CNRS FR 3503 GEIST, Limoges, FranceSearch for other works by this author on:Farid BoumédieneFarid BoumédieneaInserm U1094, Tropical Neuroepidemiology, andbUniversity of Limoges, UMR_S 1094, Tropical Neuroepidemiology, Institute of Neuroepidemiology and Tropical Neurology, CNRS FR 3503 GEIST, Limoges, FranceSearch for other works by this author on:
-
147 - 153: The Right Therapy for Neurological Disorders: From Randomized Trials to Clinical Practice - Patients versus Investigator Expectations and NeedsByLucie I. Bruijn;Lucie I. BruijnaALS Association, Washington, D.C., andSearch for other works by this author on:Steve KolbSteve KolbbPatient Advocat, Norfolk, Va., USASearch for other works by this author on:
-
154 - 162: General Overview, Conclusions, and Future DirectionsByEttore Beghi;Ettore BeghiaLaboratory of Neurological Disorders, IRCCS-Mario Negri Institute for Pharmacological Research, Milan,Search for other works by this author on:Giancarlo LogroscinoGiancarlo LogroscinobDepartment of Basic Medical Sciences, Neuroscience and Sense Organs, University of Bari ‘Aldo Moro', Bari, andcUnit of Neurodegenerative Diseases, Department of Clinical Research in Neurology, University of Bari ‘Aldo Moro', ‘Pia Fondazione Cardinale G. Panico', Tricase, ItalySearch for other works by this author on: