GEN-O-MA project: an Italian network studying clinical course and pathogenic pathways of moyamoya disease-study protocol and preliminary results.


Journal

Neurological sciences : official journal of the Italian Neurological Society and of the Italian Society of Clinical Neurophysiology
ISSN: 1590-3478
Titre abrégé: Neurol Sci
Pays: Italy
ID NLM: 100959175

Informations de publication

Date de publication:
Mar 2019
Historique:
received: 27 02 2018
accepted: 28 11 2018
pubmed: 4 1 2019
medline: 17 4 2019
entrez: 4 1 2019
Statut: ppublish

Résumé

GENetics of mOyaMoyA (GEN-O-MA) project is a multicenter observational study implemented in Italy aimed at creating a network of centers involved in moyamoya angiopathy (MA) care and research and at collecting a large series and bio-repository of MA patients, finally aimed at describing the disease phenotype and clinical course as well as at identifying biological or cellular markers for disease progression. The present paper resumes the most important study methodological issues and preliminary results. Nineteen centers are participating to the study. Patients with both bilateral and unilateral radiologically defined MA are included in the study. For each patient, detailed demographic and clinical as well as neuroimaging data are being collected. When available, biological samples (blood, DNA, CSF, middle cerebral artery samples) are being also collected for biological and cellular studies. Ninety-eight patients (age of onset mean ± SD 35.5 ± 19.6 years; 68.4% females) have been collected so far. 65.3% of patients presented ischemic (50%) and haemorrhagic (15.3%) stroke. A higher female predominance concomitantly with a similar age of onset and clinical features to what was reported in previous studies on Western patients has been confirmed. An accurate and detailed clinical and neuroimaging classification represents the best strategy to provide the characterization of the disease phenotype and clinical course. The collection of a large number of biological samples will permit the identification of biological markers and genetic factors associated with the disease susceptibility in Italy.

Sections du résumé

BACKGROUND BACKGROUND
GENetics of mOyaMoyA (GEN-O-MA) project is a multicenter observational study implemented in Italy aimed at creating a network of centers involved in moyamoya angiopathy (MA) care and research and at collecting a large series and bio-repository of MA patients, finally aimed at describing the disease phenotype and clinical course as well as at identifying biological or cellular markers for disease progression. The present paper resumes the most important study methodological issues and preliminary results.
METHODS METHODS
Nineteen centers are participating to the study. Patients with both bilateral and unilateral radiologically defined MA are included in the study. For each patient, detailed demographic and clinical as well as neuroimaging data are being collected. When available, biological samples (blood, DNA, CSF, middle cerebral artery samples) are being also collected for biological and cellular studies.
RESULTS RESULTS
Ninety-eight patients (age of onset mean ± SD 35.5 ± 19.6 years; 68.4% females) have been collected so far. 65.3% of patients presented ischemic (50%) and haemorrhagic (15.3%) stroke. A higher female predominance concomitantly with a similar age of onset and clinical features to what was reported in previous studies on Western patients has been confirmed.
CONCLUSION CONCLUSIONS
An accurate and detailed clinical and neuroimaging classification represents the best strategy to provide the characterization of the disease phenotype and clinical course. The collection of a large number of biological samples will permit the identification of biological markers and genetic factors associated with the disease susceptibility in Italy.

Identifiants

pubmed: 30604336
doi: 10.1007/s10072-018-3664-z
pii: 10.1007/s10072-018-3664-z
doi:

Types de publication

Journal Article Multicenter Study Observational Study

Langues

eng

Sous-ensembles de citation

IM

Pagination

561-570

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Auteurs

Anna Bersano (A)

Cerebrovascular Unit, Neurological Institute "C. Besta" IRCCS Foundation, Milan, Italy. anna.bersano@gmail.com.

Gloria Bedini (G)

Laboratory of Cellular Neurobiology, Fondazione IRCCS Istituto Neurologico Carlo Besta, Milan, Italy.

Sara Nava (S)

Laboratory of Cellular Neurobiology, Fondazione IRCCS Istituto Neurologico Carlo Besta, Milan, Italy.

Francesco Acerbi (F)

Neurosurgical Unit, Fondazione IRCCS Istituto Neurologico Carlo Besta, Milan, Italy.

Davide Rossi Sebastiano (DR)

Neurophysiopathology Department and Epilepsy Centre, Fondazione IRCCS Istituto Neurologico Carlo Besta, Milan, Italy.

Simona Binelli (S)

Neurophysiopathology Department and Epilepsy Centre, Fondazione IRCCS Istituto Neurologico Carlo Besta, Milan, Italy.

Silvana Franceschetti (S)

Neurophysiopathology Department and Epilepsy Centre, Fondazione IRCCS Istituto Neurologico Carlo Besta, Milan, Italy.

Giuseppe Faragò (G)

Diagnostic Imaging Department & Interventional Neuroradiology, Fondazione IRCCS Istituto Neurologico Carlo Besta, Milan, Italy.

Marina Grisoli (M)

Neuroradiological Unit, Fondazione IRCCS Istituto Neurologico Carlo Besta, Milan, Italy.

Andrea Gioppo (A)

Diagnostic Imaging Department & Interventional Neuroradiology, Fondazione IRCCS Istituto Neurologico Carlo Besta, Milan, Italy.

Paolo Ferroli (P)

Neurosurgical Unit, Fondazione IRCCS Istituto Neurologico Carlo Besta, Milan, Italy.

Maria Grazia Bruzzone (MG)

Neuroradiological Unit, Fondazione IRCCS Istituto Neurologico Carlo Besta, Milan, Italy.

Daria Riva (D)

Developmental Neurology Division, Fondazione IRCCS Istituto Neurologico Carlo Besta, Milan, Italy.

Elisa Ciceri (E)

Neuroradiological Unit, Fondazione IRCCS Istituto Neurologico Carlo Besta, Milan, Italy.

Chiara Pantaleoni (C)

Developmental Neurology Division, Fondazione IRCCS Istituto Neurologico Carlo Besta, Milan, Italy.

Veronica Saletti (V)

Developmental Neurology Division, Fondazione IRCCS Istituto Neurologico Carlo Besta, Milan, Italy.

Silvia Esposito (S)

Developmental Neurology Division, Fondazione IRCCS Istituto Neurologico Carlo Besta, Milan, Italy.

Nardo Nardocci (N)

Department of Child Neurology, Fondazione IRCCS Istituto Neurologico Carlo Besta, Milan, Italy.

Federica Zibordi (F)

Department of Child Neurology, Fondazione IRCCS Istituto Neurologico Carlo Besta, Milan, Italy.

Luigi Caputi (L)

Cerebrovascular Unit, Neurological Institute "C. Besta" IRCCS Foundation, Milan, Italy.

Stefania Bianchi Marzoli (SB)

Neuropthalmologic Unit, IRCCS Istituto Auxologico, Milan, Italy.

Maria Luisa Zedde (ML)

Neurology Unit, Stroke Unit, Azienda Unità Sanitaria Locale-IRCCS di Reggio Emilia, Reggio Emilia, Italy.

Marco Pavanello (M)

Neurosurgery Unit, Istituto Giannina Gaslini, Genoa, Italy.

Alessandro Raso (A)

Neurosurgery Unit, Istituto Giannina Gaslini, Genoa, Italy.

Valeria Capra (V)

Neurosurgery Unit, Istituto Giannina Gaslini, Genoa, Italy.

Leonardo Pantoni (L)

L.Sacco Department of Biomedical and Clinical Science, University of Milan, Milan, Italy.

Cristina Sarti (C)

NEUROFARBA Department Neuroscience Section, University of Florence, Florence, Italy.

Alessandro Pezzini (A)

Department of Clinical and Experimental Sciences, Neurology Clinic, University of Brescia, Brescia, Italy.

Filomena Caria (F)

Department of Clinical and Experimental Sciences, Neurology Clinic, University of Brescia, Brescia, Italy.

Maria Luisa Dell' Acqua (ML)

Stroke Unit, Nuovo Ospedale Civile S Agostino Estense, University Hospital of Modena, Modena, Italy.

Andrea Zini (A)

Stroke Unit, Nuovo Ospedale Civile S Agostino Estense, University Hospital of Modena, Modena, Italy.

Claudio Baracchini (C)

Stroke Unit and Neurosonology Laboratory, Department of Neurological Sciences, University of Padua School of Medicine, Padua, Italy.

Filippo Farina (F)

Stroke Unit and Neurosonology Laboratory, Department of Neurological Sciences, University of Padua School of Medicine, Padua, Italy.

Sandro Sanguigni (S)

Department of Neurology, General Hospital Madonna del Soccorso, San Benedetto del Tronto, Italy.

Maria Luisa De Lodovici (ML)

Stroke Unit Circolo Hospital and Macchi Foundation, Varese Hospital, Varese, Italy.

Giorgio Bono (G)

Stroke Unit Circolo Hospital and Macchi Foundation, Varese Hospital, Varese, Italy.

Fioravanti Capone (F)

Unit of Neurology, Neurophysiology, Neurobiology, Department of Medicine, Università Campus Bio-Medico di Roma, Via Alvaro del Portillo 200, 00128, Rome, Italy.

Vincenzo Di Lazzaro (V)

Unit of Neurology, Neurophysiology, Neurobiology, Department of Medicine, Università Campus Bio-Medico di Roma, Via Alvaro del Portillo 200, 00128, Rome, Italy.

Silvia Lanfranconi (S)

Department of Neuroscience and Sensory Organs, Neurology Unit, Maggiore Policlinico Hospital Foundation IRCCS Ca' Granda, Milan, Italy.

Massimiliano Toscano (M)

Department of Neurology and Psychiatry, Sapienza University of Rome, Rome, Italy.

Vittorio Di Piero (V)

Department of Neurology and Psychiatry, Sapienza University of Rome, Rome, Italy.

Simona Sacco (S)

Department of Neurology, Avezzano Hospital, University of L'Aquila, L'Aquila, Italy.

Antonio Carolei (A)

Department of Neurology, Avezzano Hospital, University of L'Aquila, L'Aquila, Italy.

Danilo Toni (D)

Department of Neurology and Psychiatry, Sapienza University of Rome, Rome, Italy.

Maurizio Paciaroni (M)

Stroke Unit and Division of Cardiovascular Medicine, University of Perugia, Perugia, Italy.

Valeria Caso (V)

Stroke Unit and Division of Cardiovascular Medicine, University of Perugia, Perugia, Italy.

Patrizia Perrone (P)

Stroke Unit Legnano Hospital ASST Ovest Milanese, Legnano, Italy.

Maria Vittoria Calloni (MV)

Stroke Unit Legnano Hospital ASST Ovest Milanese, Legnano, Italy.

Alfredo Romani (A)

IRCCS Foundation C. Mondino Neurological Institute, Pavia, Italy.

Marco Cenzato (M)

Department of Neurosurgery, Niguarda Cà Granda Hospital, Milan, Italy.

Alessia Fratianni (A)

Department of Neurosurgery, Niguarda Cà Granda Hospital, Milan, Italy.

Emilio Ciusani (E)

Laboratory of Clinical Investigations, Fondazione IRCCS Istituto Neurologico Carlo Besta, Milan, Italy.

Paolo Prontera (P)

Neonatology Unit and Prenatal Diagnosis (P.P.), Medical Genetic Unit, Ospedale S. Maria della Misericordia, Perugia, Italy.

Elisabeth Tournier Lasserve (ET)

Inserm UMR-S1161, Génétique et Physiopathologie des Maladies Cérébro-vasculaires, Université Paris Diderot, Sorbonne Paris Cité, Paris, France.

Kinga Blecharz (K)

Department of Neurosurgery, Charite Universitätsmedizin, Berlin, Germany.

Peter Vajkoczy (P)

Department of Neurosurgery, Charite Universitätsmedizin, Berlin, Germany.

Eugenio Agostino Parati (EA)

Cerebrovascular Unit, Neurological Institute "C. Besta" IRCCS Foundation, Milan, Italy.

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