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26 results on '"Fazio R."'

1. Unclassified clinical presentations of chronic inflammatory demyelinating polyradiculoneuropathy

Author

Doneddu, P, Akyil, H, Manganelli, F, Briani, C, Cocito, D, Benedetti, L, Mazzeo, A, Fazio, R, Filosto, M, Cosentino, G, Di Stefano, V, Antonini, G, Marfia, G, Inghilleri, M, Siciliano, G, Clerici, A, Carpo, M, Schenone, A, Luigetti, M, Lauria, G, Mata, S, Rosso, T, Minicuci, G, Lucchetta, M, Cavaletti, G, Liberatore, G, Spina, E, Campagnolo, M, Peci, E, Germano, F, Gentile, L, Strano, C, Cotti Piccinelli, S, Vegezzi, E, Leonardi, L, Mataluni, G, Ceccanti, M, Schirinzi, E, Romozzi, M, Nobile-Orazio, E, Doneddu P. E., Akyil H., Manganelli F., Briani C., Cocito D., Benedetti L., Mazzeo A., Fazio R., Filosto M., Cosentino G., Di Stefano V., Antonini G., Marfia G. A., Inghilleri M., Siciliano G., Clerici A. M., Carpo M., Schenone A., Luigetti M., Lauria G., Mata S., Rosso T., Minicuci G. M., Lucchetta M., Cavaletti G., Liberatore G., Spina E., Campagnolo M., Peci E., Germano F., Gentile L., Strano C., Cotti Piccinelli S., Vegezzi E., Leonardi L., Mataluni G., Ceccanti M., Schirinzi E., Romozzi M., Nobile-Orazio E., Doneddu, P, Akyil, H, Manganelli, F, Briani, C, Cocito, D, Benedetti, L, Mazzeo, A, Fazio, R, Filosto, M, Cosentino, G, Di Stefano, V, Antonini, G, Marfia, G, Inghilleri, M, Siciliano, G, Clerici, A, Carpo, M, Schenone, A, Luigetti, M, Lauria, G, Mata, S, Rosso, T, Minicuci, G, Lucchetta, M, Cavaletti, G, Liberatore, G, Spina, E, Campagnolo, M, Peci, E, Germano, F, Gentile, L, Strano, C, Cotti Piccinelli, S, Vegezzi, E, Leonardi, L, Mataluni, G, Ceccanti, M, Schirinzi, E, Romozzi, M, Nobile-Orazio, E, Doneddu P. E., Akyil H., Manganelli F., Briani C., Cocito D., Benedetti L., Mazzeo A., Fazio R., Filosto M., Cosentino G., Di Stefano V., Antonini G., Marfia G. A., Inghilleri M., Siciliano G., Clerici A. M., Carpo M., Schenone A., Luigetti M., Lauria G., Mata S., Rosso T., Minicuci G. M., Lucchetta M., Cavaletti G., Liberatore G., Spina E., Campagnolo M., Peci E., Germano F., Gentile L., Strano C., Cotti Piccinelli S., Vegezzi E., Leonardi L., Mataluni G., Ceccanti M., Schirinzi E., Romozzi M., and Nobile-Orazio E.

Abstract

Background To assess the ability of the 2021 European Academy of Neurology/Peripheral Nerve Society (EAN/PNS) clinical criteria for chronic inflammatory demyelinating polyradiculoneuropathy (CIDP) to include within their classification the whole spectrum of clinical heterogeneity of the disease and to define the clinical characteristics of the unclassifiable clinical forms. Methods The 2021 EAN/PNS clinical criteria for CIDP were applied to 329 patients fulfilling the electrodiagnostic (and in some cases also the supportive) criteria for the diagnosis of CIDP. Clinical characteristics were reviewed for each patient not strictly fulfilling the clinical criteria ('unclassifiable'). Results At study inclusion, 124 (37.5%) patients had an unclassifiable clinical presentation, including 110 (89%) with a typical CIDP-like clinical phenotype in whom some segments of the four limbs were unaffected by weakness ('incomplete typical CIDP'), 10 (8%) with a mild distal, symmetric, sensory or sensorimotor polyneuropathy confined to the lower limbs with cranial nerve involvement ('cranial nerve predominant CIDP') and 4 (1%) with a symmetric sensorimotor polyneuropathy limited to the proximal and distal areas of the lower limbs ('paraparetic CIDP'). Eighty-one (65%) patients maintained an unclassifiable presentation during the entire disease follow-up while 13 patients progressed to typical CIDP. Patients with the unclassifiable clinical forms compared with patients with typical CIDP had a milder form of CIDP, while there was no difference in the distribution patterns of demyelination. Conclusions A proportion of patients with CIDP do not strictly fulfil the 2021 EAN/PNS clinical criteria for diagnosis. These unclassifiable clinical phenotypes may pose diagnostic challenges and thus deserve more attention in clinical practice and research.

Published
2023

2. Frequency of diabetes and other comorbidities in chronic inflammatory demyelinating polyradiculoneuropathy and their impact on clinical presentation and response to therapy

Author

Doneddu, P, Cocito, D, Manganelli, F, Fazio, R, Briani, C, Filosto, M, Benedetti, L, Bianchi, E, Jann, S, Mazzeo, A, Antonini, G, Cosentino, G, Marfia, G, Cortese, A, Clerici, A, Carpo, M, Schenone, A, Siciliano, G, Luigetti, M, Lauria, G, Rosso, T, Cavaletti, G, Beghi, E, Liberatore, G, Santoro, L, Spina, E, Peci, E, Tronci, S, Ruiz, M, Cotti Piccinelli, S, Verrengia, E, Gentile, L, Leonardi, L, Mataluni, G, Piccolo, L, Nobile-Orazio, E, Doneddu P. E., Cocito D., Manganelli F., Fazio R., Briani C., Filosto M., Benedetti L., Bianchi E., Jann S., Mazzeo A., Antonini G., Cosentino G., Marfia G. A., Cortese A., Clerici A. M., Carpo M., Schenone A., Siciliano G., Luigetti M., Lauria G., Rosso T., Cavaletti G., Beghi E., Liberatore G., Santoro L., Spina E., Peci E., Tronci S., Ruiz M., Cotti Piccinelli S., Verrengia E. P., Gentile L., Leonardi L., Mataluni G., Piccolo L., Nobile-Orazio E., Doneddu, P, Cocito, D, Manganelli, F, Fazio, R, Briani, C, Filosto, M, Benedetti, L, Bianchi, E, Jann, S, Mazzeo, A, Antonini, G, Cosentino, G, Marfia, G, Cortese, A, Clerici, A, Carpo, M, Schenone, A, Siciliano, G, Luigetti, M, Lauria, G, Rosso, T, Cavaletti, G, Beghi, E, Liberatore, G, Santoro, L, Spina, E, Peci, E, Tronci, S, Ruiz, M, Cotti Piccinelli, S, Verrengia, E, Gentile, L, Leonardi, L, Mataluni, G, Piccolo, L, Nobile-Orazio, E, Doneddu P. E., Cocito D., Manganelli F., Fazio R., Briani C., Filosto M., Benedetti L., Bianchi E., Jann S., Mazzeo A., Antonini G., Cosentino G., Marfia G. A., Cortese A., Clerici A. M., Carpo M., Schenone A., Siciliano G., Luigetti M., Lauria G., Rosso T., Cavaletti G., Beghi E., Liberatore G., Santoro L., Spina E., Peci E., Tronci S., Ruiz M., Cotti Piccinelli S., Verrengia E. P., Gentile L., Leonardi L., Mataluni G., Piccolo L., and Nobile-Orazio E.

Abstract

Objectives To determine the prevalence of different comorbidities in chronic inflammatory demyelinating polyradiculoneuropathy (CIDP), and their impact on outcome, treatment choice and response. Methods Using a structured questionnaire, we collected information on comorbidities from 393 patients with CIDP fulfilling the European Federation of Neurological Societies and Peripheral Nerve Society criteria included in the Italian CIDP database. Results One or more comorbidities were reported by 294 patients (75%) and potentially influenced treatment choice in 192 (49%) leading to a less frequent use of corticosteroids. Response to treatment did not differ, however, from that in patients without comorbidities. Diabetes (14%), monoclonal gammopathy of undetermined significance (MGUS) (12%) and other immune disorders (16%) were significantly more frequent in patients with CIDP than expected in the general European population. Patients with diabetes had higher disability scores, worse quality of life and a less frequent treatment response compared with patients without diabetes. Patients with IgG-IgA or IgM MGUS had an older age at CIDP onset while patients with other immune disorders had a younger age at onset and were more frequently females. IgM MGUS was more frequent in patients with motor CIDP than in patients with typical CIDP. Conclusions Comorbidities are frequent in patients with CIDP and in almost 50% of them have an impact on treatment choice. Diabetes, MGUS and other immune diseases are more frequent in patients with CIDP than in the general population. Only diabetes seems, however, to have an impact on disease severity and treatment response possibly reflecting in some patients a coexisting diabetic neuropathy.

Published
2020

3. Second IVIg course in Guillain-Barré syndrome with poor prognosis. The non-randomised ISID study

Author

Verboon, C., Van Den Berg, B., Cornblath, D. R., Venema, E., Gorson, K. C., Lunn, M. P., Lingsma, H., Van Den Bergh, P., Harbo, T., Bateman, K., Pereon, Y., Sindrup, So. H., Kusunoki, S., Miller, J., Islam, Z., Hartung, H. -P., Chavada, G., Jacobs, B. C., Hughes, R. A. C., Addington, Van Doorn P. A. J. M., MD (University of Virginia, Charlottesville, USA), on October 7, S. Consortia. Protected by copyright., Downloaded from 8 Verboon C, 2019 at Uppsala Universitet BIBSAM http://jnnp. bmj. com/ J Neurol Neurosurg Psychiatry: first published as 10. 1136/jnnp-2019-321496 on 5 October 2019., J Neurol Neurosurg Psychiatry 2019, et al., 1136/jnnp-2019-321496 Neuromuscular Ajroud-Driss, 0:1–9. doi:10., MD (Northwestern University Feinberg, Chicago, USA), Antonini, G., MD (Mental Health and Sensory Organs (NESMOS), Sapienza, University, Sant’Andrea, Hospital, Rome, Italy), Attarian, S., PhD (CHU Timone, Marseille, France), Barroso, F. A., MD (Instituto de Investigaciones Neurológicas Raúl Carrea, Fleni, Buenos, Aires, Argentina), Benedetti, L., PhD (Ospedale Sant’ Andrea La Spezia, Spezia, La, Italy), Bertorini, T. E., MD (The University of Tennessee Health Science Center (UTHSC), Memphis, USA), Brannagan, T. H., MD (Columbia University, New York City, USA), Briani, C., MD (University of Padova, Padova, Italy), Bhavaraju-Sanka, R., MD (University Hospital/University of Texas Health Science Center, San Antonio Texas, Butterworth, S., MD (Pinderfields Hospital, Wakefield, UK), Casasnovas, C., Ciberer, PhD (Bellvitge University Hospital – IDIBELL Neurometabolic Diseases Group., Barcelona, Spain), Cavaletti, G., MD (University Milano-Bicocca, Monza, Italy), Chen, S., Phd, (Rutgers, Robert Wood Johnson University Hospital, New, Brunswick, Claeys, K. G., University Hospitals Leuven, PhD (1., Leuven, Belgium, KU Leuven, 2., Leuven, Belgium), Cosgrove, J. S., MD (Leeds General Infirmary, Leeds, UK), Davidson, A., MD (University of Glasgow, Glasgow, UK), Dardiotis, E., MD (University of Thessaly, Hospital of Larissa, Larissa, Greece), Dornonville de la Cour, C., MD (National Hospital Copenhagen, Copenhagen, Denmark), Faber, C. G., PhD (Maastricht University Medical Centre, Maastricht, The, Netherlands), Feasby, T. E., MD (University of Calgary, Calgary, Canada), Fujioka, T., MD (Toho University Medical Center, Tokyo, Japan), Galassi, G., MD (University Hospital of Modena, Modena, Italy), Gilchrist, J. M., MD (Soulthern Illinois University School of Medicine, Springfield, USA), Goyal, N. A., MD (University of California, Irvine, USA), Granit, V., MD (Montefiore Medical, Center, New, York, Gutiérrez-Gutiérrez, G., MD (Hospital Universitario Infanta Sofia, San, Sebastian, Spain), Hadden, R. D. M., PhD (King’s College Hospital, London, UK), Holt, J. K. L., Phd, FRCP (The Walton Centre, Liverpool, UK), Htut, M., George’s Hospital, MD (St., Jericó Pascual, I., PhD (Complejo Hospitalario de Navarra, Pamplona, Spain), Karafiath, S., MD (University of Utah School of Medicine, Salt Lake City, Katzberg, H. D., MD (University of Toronto, Toronto, Canada), Kiers, L., MD (University of Melbourne, Royal Melbourne Hospital, Parkville, Australia), Kieseier, B. C., MD (Heinrich Heine University, Düsseldorf, Germany), Kimpinski, K., MD (University Hospital, Lhsc, London-Ontario, Canada), Kuwabara, S., PhD (Chiba University, Chiba, Japan), Kwan, J. Y., MD (University of Maryland School of Medicine, Baltimore, USA), Ladha, S. S., MD (Barrow Neurology Clinics, Phoenix, Arizona, Lawson, V., MD (Wexner Medical Center at The Ohio State University, Columbus, USA), Lehmann, H., PhD (University Hospital of Cologne, Universitätsklinikum, Köln, Cologne, Germany), Manji, H., FRCP (Ipswich Hospital, Ipswich, UK), Marfia, G. A., MD (Neurological Clinic, Policlinico Tor Vergata, Márquez Infante, C., MD (Hospital Universitario Virgen del Rocio, Seville, Spain), Mattiazzi, M. G., MD (Hospital Militar Central, Mcdermott, C. J., MD (Royal Hallamshire Hospital, Nihr, Clinical, Sheffield, UK), Monges, M. S., Garrahan, MD (Hospital de Pediatría J. P., Morís de la Tassa, G., MD (Hospital Universitario Central de Asturias, Asturias, Spain), Nascimbene, C., PhD (Luigi Sacco Hospital, Milan, Italy), Nobile Orazio, E., PhD (Milan University, Humanitas Clinicala and Research Institute Milan, Nowak, R. J., MD (Yale University School of Medicine, New, Haven, Osei-Bonsu (James Cook University Hospital, M., Middlesbrough, UK), Pardo Fernandez (Hospital Clínico de Santiago, J., Santiago de Compostela (A Coruña), Querol Gutierrez, L., PhD (Hospital de la Santa Creu, i Sant Pau, Universitat Autònoma de Barcelona, Reisin (Hospital Britanico, R., Rinaldi, S., Mbchb, Roberts, PhD (University of Oxford R. C., MD (Addenbrooke’s Hospital Cambridge, Cambridge, UK), Rojas-Marcos, I., MD (Hospital Univesitario Reina Sofia, Cordoba, Spain), Rudnicki, S. A., MD (University of Arkansas, Fayetteville, USA), Schenone, A., Department of Neurosciences, PhD (1., Rehabilitation, Ophthalmology, Genetics and Maternal and Infantile Sciences (DINOGMI), University of Genova, Genova, IRCCS Policlinico San Martino, Italy 2., Genova, Italy), Sedano Tous, M. J., MD (Hospital Universitario Marques de Valdecilla, Santander, Cantabria, Shahrizaila, N., MD (Neurology Unit, Department of Medicine, Faculty of Medicine, University of Malaya, Malaya), Sheikh, K., PhD (The University of Texas Health Science Center at Houston, Houston, USA), Silvestri, N. J., MD (Buffalo General Medical Center, Buffalo, Ny, Sommer, C. L., MD (Universitätsklinikum Würzburg, Würzburg, Germany), Varrato, J. D., DO (Lehigh Valley Health Network, Allentown, USA), Verschuuren, J., PhD (Leiden University Medical Centre, Leiden, Vytopil, M. V., Waheed, PhD (Tufts University School of Medicine Lahey Hospital W., MD (University of Vermont Medical Center, Burlington, USA), Zhou, L., PhD (Icahn School of Medicine at Mount Sinai, Badrising, USA). Other collaborators were:U. A., Bella, I. R., MD (University of Mass Medical School, Worcester, USA), Bunschoten, C., PhD candidate (Erasmus University Medical Centre, Rotterdam, Bürmann, J., Universitätsklinikum des Saarlandes, Homburg, Germany), Busby, M., Bradford, UK), Chao, C. C., PhD (National Taiwan University Hospital, Taipei, Taiwan), Conti, M. E., MD (University Hospital Clinicas, Dalakas, M. C., Thomas Jefferson University, MD (1., Philadelphia, Usa, National and Kapodistrian University of Athens, 2., Athens, Greece), Van Damme, P., PhD (University Hospital Leuven, Doets, A., van Dijk, G. W., MD (Canisius Wilhelmina Hospital, Nijmegen, Dimachkie, M. M., MD (University of Kansas Medical Center, Kansas, City, Doppler, K., Echaniz-Laguna, A., MD (Hopital de Hautepierre, Strasbourgh, France), Eftimov, F., PhD (Amsterdam University Medical Centre, Amsterdam, Fazio, R., MD (Scientific Institute San Raffaele, Fokke, C., MD (Gelre Hospital, Zutphen and Apeldoorn, Fulgenzi, E. A., MD (Hospital Cesar Milstein Buenos Aires, Garssen, M. P. J., PhD (Jeroen Bosch Hospital, Hertogenbosch, ’S, Zaltbommel and Drunen, Gijsbers, C. J., MD (Vlietland Hospital, Schiedam, Gilhuis, J., PhD (Reinier de Graaf Gasthuis, Delft, Grapperon, A., MD (CHU Timone, Hsieh, S. T., Illa, I., Islam, B., PhD (International Centre for Diarrhoeal Disease Research, Bangladesh, (icddr, Dhaka, b), Bangladesh), Jellema, K., PhD (Haaglanden Medisch Centrum, The, Hague, Kaida, K., PhD (National Defense Medical College, Saitama, Japan), Kokubun, N., MD (Dokkyo Medical University, Tochigi, Japan), Kolb, N., MD (University of Vermont, Burlington, Vt, van Koningsveld, R., PhD (Elkerliek Hospital, Helmond and Deurne, van der Kooi, A. J., Kuitwaard, K., PhD (Albert Schweitzer Hospital, Dordrecht, Landschoff Lassen, L., MD (Glostrup Hospital, Glostrup, Denmark), Leonhard, S. E., Mandarakas, M., PhD (Erasmus University Medical Centre, Martinez Hernandez, E., MD (Institut d’Investigacions Biomèdiques August Pi, i Sunyer (IDIBAPS), Hospital, Clinic, Mohammad, Q. D., PhD (National Institute of Neurosciences and Hospital, Dhaka, Bangladesh), Pulley, M., MD (University of Florida, Jacksonville, USA), Rajabally, Y. A., PhD (Queen Elizabeth Hospital, Birmingham, UK), Reddel, S. W., PhD (Concord Repatriation General Hospital, Sydney, Australia), van der Ree, T., (Westfriesgasthuis, Md, Hoorn, Roodbol, J., Sachs, G. M., MD (University of Rhode Island, Providence, USA), Samijn, J. P. A., PhD (Maasstad Hospital, Santoro, L., PhD (University Federico II, Napels, Italy), Stein, B., Joseph’s Regional Medical Center, MD (St., Paterson, USA), Vermeij, F. H., MD (Franciscus Gasthuis, Visser, L. H., PhD (Elisabeth-TweeSteden Hospital, Tilburg and Waalwijk, Willison, H. J., PhD (University of Glasgow, Wirtz, P., Phd, (HagaZiekenhuis, Zivkovich, S. A., PhD (University of Pittsburgh Medical Center, and Pittsburgh, USA).

Subjects
treatment, disability evaluation, drug administration schedule, adult, guillain-barré syndrome, poor prognosis, second ivig course, aged, female, guillain-barre syndrome, humans, immunoglobulin g, immunoglobulins, intravenous, immunologic factors, male, middle aged, prognosis, time factors, treatment outcome
Published
2020

4. Frequency of diabetes and other comorbidities in chronic inflammatory demyelinating polyradiculoneuropathy and their impact on clinical presentation and response to therapy

Author

Doneddu, P. E., Cocito, D., Manganelli, F., Fazio, R., Briani, C., Filosto, M., Benedetti, L., Bianchi, E., Jann, S., Mazzeo, A., Antonini, Gabriele, Cosentino, G., Marfia, G. A., Cortese, A., Clerici, Anna Marina, Carpo, M., Schenone, A., Siciliano, Giovanni, Luigetti, Marco, Lauria, G., Rosso, T., Cavaletti, G., Beghi, E., Liberatore, G., Santoro, Luca, Spina, E., Peci, E., Tronci, S., Ruiz, M., Cotti Piccinelli, S., Verrengia, E. P., Gentile, L., Leonardi, L., Mataluni, G., Piccolo, L., Nobile-Orazio, E., Antonini G., Clerici A. M., Siciliano G., Luigetti M. (ORCID:0000-0001-7539-505X), Santoro L., Doneddu, P. E., Cocito, D., Manganelli, F., Fazio, R., Briani, C., Filosto, M., Benedetti, L., Bianchi, E., Jann, S., Mazzeo, A., Antonini, Gabriele, Cosentino, G., Marfia, G. A., Cortese, A., Clerici, Anna Marina, Carpo, M., Schenone, A., Siciliano, Giovanni, Luigetti, Marco, Lauria, G., Rosso, T., Cavaletti, G., Beghi, E., Liberatore, G., Santoro, Luca, Spina, E., Peci, E., Tronci, S., Ruiz, M., Cotti Piccinelli, S., Verrengia, E. P., Gentile, L., Leonardi, L., Mataluni, G., Piccolo, L., Nobile-Orazio, E., Antonini G., Clerici A. M., Siciliano G., Luigetti M. (ORCID:0000-0001-7539-505X), and Santoro L.

Abstract

Objectives To determine the prevalence of different comorbidities in chronic inflammatory demyelinating polyradiculoneuropathy (CIDP), and their impact on outcome, treatment choice and response. Methods Using a structured questionnaire, we collected information on comorbidities from 393 patients with CIDP fulfilling the European Federation of Neurological Societies and Peripheral Nerve Society criteria included in the Italian CIDP database. Results One or more comorbidities were reported by 294 patients (75%) and potentially influenced treatment choice in 192 (49%) leading to a less frequent use of corticosteroids. Response to treatment did not differ, however, from that in patients without comorbidities. Diabetes (14%), monoclonal gammopathy of undetermined significance (MGUS) (12%) and other immune disorders (16%) were significantly more frequent in patients with CIDP than expected in the general European population. Patients with diabetes had higher disability scores, worse quality of life and a less frequent treatment response compared with patients without diabetes. Patients with IgG-IgA or IgM MGUS had an older age at CIDP onset while patients with other immune disorders had a younger age at onset and were more frequently females. IgM MGUS was more frequent in patients with motor CIDP than in patients with typical CIDP. Conclusions Comorbidities are frequent in patients with CIDP and in almost 50% of them have an impact on treatment choice. Diabetes, MGUS and other immune diseases are more frequent in patients with CIDP than in the general population. Only diabetes seems, however, to have an impact on disease severity and treatment response possibly reflecting in some patients a coexisting diabetic neuropathy.

Published
2020

5. Atypical CIDP: Diagnostic criteria, progression and treatment response. Data from the Italian CIDP Database

Author

Doneddu, P, Cocito, D, Manganelli, F, Fazio, R, Briani, C, Filosto, M, Benedetti, L, Mazzeo, A, Marfia, G, Cortese, A, Fierro, B, Jann, S, Beghi, E, Clerici, A, Carpo, M, Schenone, A, Luigetti, M, Lauria, G, Antonini, G, Rosso, T, Siciliano, G, Cavaletti, G, Liberatore, G, Santoro, L, Peci, E, Tronci, S, Ruiz, M, Cotti Piccinelli, S, Toscano, A, Mataluni, G, Piccolo, L, Cosentino, G, Sabatelli, M, Nobile-Orazio, E, Doneddu, PE, Marfia, GA, Clerici, AM, Doneddu, P, Cocito, D, Manganelli, F, Fazio, R, Briani, C, Filosto, M, Benedetti, L, Mazzeo, A, Marfia, G, Cortese, A, Fierro, B, Jann, S, Beghi, E, Clerici, A, Carpo, M, Schenone, A, Luigetti, M, Lauria, G, Antonini, G, Rosso, T, Siciliano, G, Cavaletti, G, Liberatore, G, Santoro, L, Peci, E, Tronci, S, Ruiz, M, Cotti Piccinelli, S, Toscano, A, Mataluni, G, Piccolo, L, Cosentino, G, Sabatelli, M, Nobile-Orazio, E, Doneddu, PE, Marfia, GA, and Clerici, AM

Abstract

Objectives A few variants of chronic inflammatory demyelinating polyradiculoneuropathy (CIDP) have been described, but their frequency and evolution to typical CIDP remain unclear. To determine the frequency and characteristics of the CIDP variants, their possible evolution to typical CIDP, and treatment response. Methods We applied a set of diagnostic criteria to 460 patients included in a database of Italian patients with CIDP. Clinical characteristics and treatment response were reviewed for each patient. The Kaplan-Meier curve was used to estimate the progression rate from atypical to typical CIDP. Results At the time of inclusion, 376 (82%) patients had a diagnosis of typical CIDP while 84 (18%) had atypical CIDP, including 34 (7%) with distal acquired demyelinating symmetric neuropathy (DADS), 17 (4%) with purely motor, 17 (4%) with Lewis-Sumner syndrome (LSS) and 16 (3.5%) with purely sensory CIDP. Based on retrospective review of the symptoms and signs present at onset and for at least 1 year, 180 (39%) patients had an initial diagnosis compatible with atypical CIDP that in 96 (53%) patients evolved to typical CIDP. Mean disease duration was longer in patients evolving to typical CIDP than in those not evolving (p=0.0016). Patients with DADS and LSS had a less frequent response to immunoglobulin than those with typical CIDP, while patients with purely motor and sensory CIDP had a similar treatment response. Conclusions The proportion of patients with atypical CIDP varies during the disease course. DADS and LSS have a less frequent response to intravenous immunoglobulin compared with typical CIDP, raising the possibility of a different underlying pathogenetic mechanism.

Published
2019

6. Frequency and time to relapse after discontinuing 6-month therapy with IVIg or pulsed methylprednisolone in CIDP

Author

Nobile orazio, E., Cocito, D., Jann, S., Uncini, A., Messina, P., Antonini, G., Fazio, R., Gallia, F., Schenone, A., Francia, A., Pareyson, D., SANTORO, LUCIO, Tamburin, S., Cavaletti, G., Giannini, F., Sabatelli, M., Beghi, E., Paolasso, I., De Toni Franceschini, L., Notturno, F., Clemenzi, A., Bianchi, F., Fiorina, E., Pontecorvo, S., Piscosquito, G., MANGANELLI, FIORE, Praitano, M. L., Piatti, M., Torzini, A., Luigetti, M., R. Macchia, Nobile Orazio, E, Cocito, D, Jann, S, Uncini, A, Messina, P, Antonini, G, Fazio, R, Gallia, F, Schenone, A, Francia, A, Pareyson, D, Santoro, L, Tamburin, S, Cavaletti, G, Giannini, F, Sabatelli, M, Beghi, E, Nobile orazio, E., Cocito, D., Jann, S., Uncini, A., Messina, P., Antonini, G., Fazio, R., Gallia, F., Schenone, A., Francia, A., Pareyson, D., Santoro, Lucio, Tamburin, S., Cavaletti, G., Giannini, F., Sabatelli, M., Beghi, E., Paolasso, I., De Toni Franceschini, L., Notturno, F., Clemenzi, A., Bianchi, F., Fiorina, E., Pontecorvo, S., Piscosquito, G., Manganelli, Fiore, Praitano, M. L., Piatti, M., Torzini, A., Luigetti, M., and R., Macchia

Subjects
medicine.medical_specialty, Neuromuscular disease, Time Factors, NEUROIMMUNOLOGY, NEUROPATHY, STEROIDS, Anti-Inflammatory Agents, Humans, Immunoglobulins, Intravenous, Immunologic Factors, Methylprednisolone, Polyradiculoneuropathy, Chronic Inflammatory Demyelinating, Recurrence, Retrospective Studies, Treatment Outcome, Polyradiculoneuropathy, Immunoglobulins, Time to relapse, Arts and Humanities (miscellaneous), Internal medicine, medicine, In patient, Chronic Inflammatory Demyelinating, neuroimmunology,neuropathy,steroids, business.industry, Multiple sclerosis, Medicine (all), Retrospective cohort study, Neurology (clinical), Psychiatry and Mental Health, Surgery, medicine.disease, Discontinuation, Settore MED/26 - NEUROLOGIA, business, Intravenous, medicine.drug
Abstract

Background: We reported that 6-month therapy with intravenous immunoglobulin (IVIg) was more frequently effective or tolerated than intravenous methylprednisolone (IVMP) in patients with chronic inflammatory demyelinating polyradiculoneuropathy (CIDP). We now retrospectively compared the proportion of patients who eventually worsened after discontinuing therapy and the median time to clinical worsening. Methods: By March 2013, data were available from 41 of the 45 patients completing the trial with a median follow-up after therapy discontinuation of 42 months (range 1-60). Three patients withdrew during the original study and one failed to respond to either of the therapies. No patient received a diagnosis alternative to CIDP during the follow-up. Results: Twenty-eight of the 32 patients treated with IVIg (as primary or secondary therapy after failing to respond to IVMP) improved after therapy (87.5%) as compared with 13 of the 24 patients treated with IVMP as primary or secondary therapy (54.2%). After a median follow-up of 42 months (range 1-57), 24 out of 28 patients responsive to IVIg (85.7%) worsened after therapy discontinuation. The same occurred in 10 out of 13 patients (76.9%) responsive to IVMP (p=0.659) after a median follow-up of 43 months (range 7-60). Worsening occurred 1-24 months (median 4.5) after IVIg discontinuation and 1-31 months (median 14) after IVMP discontinuation (p=0.0126). Conclusions: A similarly high proportion of patients treated with IVIg or IVMP eventually relapse after therapy discontinuation but the median time to relapse was significantly longer after IVMP than IVIg. This difference may help to balance the more frequent response to IVIg than to IVMP in patients with CIDP.

Published
2015

7. Frequency and time to relapse after discontinuing 6-month therapy with IVIg or pulsed methylprednisolone in CIDP

Author

Nobile Orazio, E, Cocito, D, Jann, S, Uncini, A, Messina, P, Antonini, G, Fazio, R, Gallia, F, Schenone, A, Francia, A, Pareyson, D, Santoro, L, Tamburin, S, Cavaletti, G, Giannini, F, Sabatelli, M, Beghi, E, CAVALETTI, GUIDO ANGELO, Beghi, E., Nobile Orazio, E, Cocito, D, Jann, S, Uncini, A, Messina, P, Antonini, G, Fazio, R, Gallia, F, Schenone, A, Francia, A, Pareyson, D, Santoro, L, Tamburin, S, Cavaletti, G, Giannini, F, Sabatelli, M, Beghi, E, CAVALETTI, GUIDO ANGELO, and Beghi, E.

Abstract

Background: We reported that 6-month therapy with intravenous immunoglobulin (IVIg) was more frequently effective or tolerated than intravenous methylprednisolone (IVMP) in patients with chronic inflammatory demyelinating polyradiculoneuropathy (CIDP). We now retrospectively compared the proportion of patients who eventually worsened after discontinuing therapy and the median time to clinical worsening. Methods: By March 2013, data were available from 41 of the 45 patients completing the trial with a median follow-up after therapy discontinuation of 42 months (range 1-60). Three patients withdrew during the original study and one failed to respond to either of the therapies. No patient received a diagnosis alternative to CIDP during the follow-up. Results: Twenty-eight of the 32 patients treated with IVIg (as primary or secondary therapy after failing to respond to IVMP) improved after therapy (87.5%) as compared with 13 of the 24 patients treated with IVMP as primary or secondary therapy (54.2%). After a median follow-up of 42 months (range 1-57), 24 out of 28 patients responsive to IVIg (85.7%) worsened after therapy discontinuation. The same occurred in 10 out of 13 patients (76.9%) responsive to IVMP (p=0.659) after a median follow-up of 43 months (range 7-60). Worsening occurred 1-24 months (median 4.5) after IVIg discontinuation and 1-31 months (median 14) after IVMP discontinuation (p=0.0126). Conclusions: A similarly high proportion of patients treated with IVIg or IVMP eventually relapse after therapy discontinuation but the median time to relapse was significantly longer after IVMP than IVIg. This difference may help to balance the more frequent response to IVIg than to IVMP in patients with CIDP.

Published
2015

8. Frequency and time to relapse after discontinuing 6-month therapy with IVIg or pulsed methylprednisolone in CIDP

Author

Nobile-Orazio, E., Cocito, D., Jann, S., Uncini, A., Messina, P., Antonini, G., Fazio, R., Gallia, F., Schenone, A., Francia, A., Pareyson, D., Santoro, Luca, Tamburin, S., Cavaletti, G., Giannini, F., Sabatelli, Mario, Beghi, E., Paolasso, Ilaria, De Toni Franceschini, L., Notturno, F., Clemenzi, A., Bianchi, F., Fiorina, E., Pontecorvo, S., Piscosquito, G., Manganelli, F., Praitano, M. L., Piatti, M., Torzini, A., Luigetti, Marco, Macchia, R., Santoro L., Sabatelli M. (ORCID:0000-0001-6635-4985), Paolasso I., Luigetti M. (ORCID:0000-0001-7539-505X), Nobile-Orazio, E., Cocito, D., Jann, S., Uncini, A., Messina, P., Antonini, G., Fazio, R., Gallia, F., Schenone, A., Francia, A., Pareyson, D., Santoro, Luca, Tamburin, S., Cavaletti, G., Giannini, F., Sabatelli, Mario, Beghi, E., Paolasso, Ilaria, De Toni Franceschini, L., Notturno, F., Clemenzi, A., Bianchi, F., Fiorina, E., Pontecorvo, S., Piscosquito, G., Manganelli, F., Praitano, M. L., Piatti, M., Torzini, A., Luigetti, Marco, Macchia, R., Santoro L., Sabatelli M. (ORCID:0000-0001-6635-4985), Paolasso I., and Luigetti M. (ORCID:0000-0001-7539-505X)

Abstract

Background: We reported that 6-month therapy with intravenous immunoglobulin (IVIg) was more frequently effective or tolerated than intravenous methylprednisolone (IVMP) in patients with chronic inflammatory demyelinating polyradiculoneuropathy (CIDP). We now retrospectively compared the proportion of patients who eventually worsened after discontinuing therapy and the median time to clinical worsening. Methods: By March 2013, data were available from 41 of the 45 patients completing the trial with a median follow-up after therapy discontinuation of 42 months (range 1-60). Three patients withdrew during the original study and one failed to respond to either of the therapies. No patient received a diagnosis alternative to CIDP during the follow-up. Results: Twenty-eight of the 32 patients treated with IVIg (as primary or secondary therapy after failing to respond to IVMP) improved after therapy (87.5%) as compared with 13 of the 24 patients treated with IVMP as primary or secondary therapy (54.2%). After a median follow-up of 42 months (range 1-57), 24 out of 28 patients responsive to IVIg (85.7%) worsened after therapy discontinuation. The same occurred in 10 out of 13 patients (76.9%) responsive to IVMP (p=0.659) after a median follow-up of 43 months (range 7-60). Worsening occurred 1-24 months (median 4.5) after IVIg discontinuation and 1-31 months (median 14) after IVMP discontinuation (p=0.0126). Conclusions: A similarly high proportion of patients treated with IVIg or IVMP eventually relapse after therapy discontinuation but the median time to relapse was significantly longer after IVMP than IVIg. This difference may help to balance the more frequent response to IVIg than to IVMP in patients with CIDP.

Published
2015

9. Rituximab in patients with chronic inflammatory demyelinating polyradiculoneuropathy: a report of 13 cases and review of the literature.

Author

Benedetti, L, Briani, C, Sabatelli, Mario, Luigetti, Marco, Cocito, D, Franciotta, D, Fazio, R, Paolasso, I, Comi, C, Giannini, F, Mancardi, Gl, Schenone, A, Nobile Orazio, E., Sabatelli, Mario (ORCID:0000-0001-6635-4985), Luigetti, Marco (ORCID:0000-0001-7539-505X), Benedetti, L, Briani, C, Sabatelli, Mario, Luigetti, Marco, Cocito, D, Franciotta, D, Fazio, R, Paolasso, I, Comi, C, Giannini, F, Mancardi, Gl, Schenone, A, Nobile Orazio, E., Sabatelli, Mario (ORCID:0000-0001-6635-4985), and Luigetti, Marco (ORCID:0000-0001-7539-505X)

Abstract

Background A few case reports have shown controversial results of rituximab efficacy in patients with chronic inflammatory demyelinating polyradiculoneuropathy (CIDP). Objective To analyse the efficacy of rituximab in a large CIDP cohort. Methods A retrospective, observational and multicentre study on the use of rituximab in CIDP. 13 Italian CIDP patients were treated with rituximab after the partial or complete lack of efficacy of conventional therapies. Eight patients had co-occurring haematological diseases. Patients who improved by at least two points in standard clinical scales, or who reduced or discontinued the pre-rituximab therapies, were considered as responders. Results Nine patients (seven with haematological diseases) responded to rituximab: six of them, who were non-responders to conventional therapies, improved clinically, and the other three maintained the improvement that they usually achieved with intravenous immunoglobulin or plasma exchange. Significantly associated with shorter disease duration, rituximab responses started after a median period of 2.0 months (range, 1-6) and lasted for a median period of 1 year (range, 1-5). Conclusions Rituximab seems to be a promising therapeutic choice when it targets both CIDP and co-occurring haematological diseases. Timely post-onset administration of rituximab seems to be associated with better responses.

Published
2011
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10. Point mutations and a large intragenic deletion in SPG11 in complicated spastic paraplegia without thin corpus callosum.

Author

Crimella, C., Arnoldi, A., Crippa, F., Mostacciuolo, M. L., Boaretto, F., Sironi, M., D'Angelo, M. Grazia, Manzoni, S., Piccinini, L., Turconi, A. C., Toscano, A., Musumeci, O., Benedetti, S., Fazio, R., Bresolin, N., Daga, A., Martinuzzi, A., and Bassi, M. T.

Subjects
PARAPLEGIA, PARALYSIS, GENETIC disorders, CORPUS callosum, LOCUS (Genetics), GENETIC mutation, MEDICAL genetics, MEDICAL research
Abstract

Background: Hereditary spastic paraplegia (HSP) with thin corpus callosum (HSP-TCC) is a frequent subtype of complicated HSP clinically characterised by slowly progressive spastic paraparesis with cognitive impairment and thin corpus callosum (TCC). SPG11, the gene associated with the major locus involved, encodes spatacsin, a protein of unknown function. Methods: Different types of mutations were identified in patients with the complex form of HSP (cHSP) including TCC. We screened a series of 45 index patients with different types of cHSP with (n = 10) and without (n = 35) TCC. Results: Ten mutations, of which five are novel, were detected in seven patients. Of importance, three out of seven mutated patients present with cHSP without TCC. Among the novel mutations identified, we characterised a large intragenic rearrangement deleting 2.6 kb of the SPG11 gene. The rearrangement is due to non-allelic homologous recombination between Alu sequences flanking the breakpoints. Conclusions: These findings expand the mutation spectrum of SPG11 and suggest that SPG11 mutations may occur more frequently in familial than sporadic forms of cHSP without TCC. This helps to define further clinical and molecular criteria for a correct diagnosis of the SPG11 related form of cHSP. In addition, the intragenic deletion detected here, and the mechanism involved, both provide clues to address the issue of SPG11 missing mutant alleles previously reported. [ABSTRACT FROM AUTHOR]

Published
2009
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11. Polyneuropathy in hypothyroidism: clinical, electrophysiological and morphological findings in four cases.

Author

Nemni, R, Bottacchi, E, Fazio, R, Mamoli, A, Corbo, M, Camerlingo, M, Galardi, G, Erenbourg, L, and Canal, N

Subjects
ACTION potentials, COMPARATIVE studies, HYPOTHYROIDISM, RESEARCH methodology, MEDICAL cooperation, MUSCLES, PERIPHERAL neuropathy, NEURAL conduction, RESEARCH, EVALUATION research
Abstract

Clinical, neurophysiological and morphological studies of four patients with polyneuropathy and secondary hypothyroidism are reported. Neurophysiological studies revealed signs of muscle denervation and reduction of conduction velocity in all the patients. Sural nerve biopsies showed axonal degeneration in all cases but one. All the patients were treated with replacement therapy and clinical symptomatology and neurophysiological parameters improved in all patients. [ABSTRACT FROM AUTHOR]

Published
1987

12. Intravenous immunoglobulin treatment in patients with chronic inflammatory demyelinating neuropathy not responsive to other treatments.

Author

Nemni, R, Amadio, S, Fazio, R, Galardi, G, Previtali, S, and Comi, G

Subjects
THERAPEUTIC use of immunoglobulins, PREDNISONE, COMPARATIVE studies, DEMYELINATION, RESEARCH methodology, MEDICAL cooperation, NEURAL conduction, PLASMA exchange (Therapeutics), POLYNEUROPATHIES, RESEARCH, EVALUATION research, THERAPEUTICS
Abstract

Nine patients with chronic inflammatory demyelinating poliradiculoneuropathy (CIDP) were treated with intravenous immunoglobulin. All patients had been previously treated with prednisone and/or plasma exchange without effect. Objective improvement in clinical condition occurred in six patients. One patient became refractory after two treatment courses, two patients had no response. The results indicate that intravenous immunoglobulin has beneficial effects in a high percentage of patients with CIDP who are unresponsive to other treatments. [ABSTRACT FROM AUTHOR]

Published
1994

13. Clinical and neurophysiological assessment of immunoglobulin therapy in five patients with multifocal motor neuropathy.

Author

Comi, G, Amadio, S, Galardi, G, Fazio, R, and Nemni, R

Subjects
THERAPEUTIC use of immunoglobulins, CLINICAL trials, COMPARATIVE studies, ELECTROMYOGRAPHY, RESEARCH methodology, MEDICAL cooperation, MOTOR neuron diseases, NERVE block, NEURAL conduction, RESEARCH, EVALUATION research, THERAPEUTICS
Abstract

High dose intravenous immunoglobulin (IVIg) is an effective treatment for demyelinating neuropathies. IVIg was given to five patients with multifocal motor neuropathy, a motor neuropathy showing a clinical syndrome of asymmetrical weakness and amyotrophy, electrophysiological evidence of motor conduction block and, in many cases, high titres of serum anti-GM1 antibodies. Muscle strength was evaluated by a conventional score before and after each IVIg course. In all patients there was relevant improvement on muscle strength after each immunoglobulin course, but in most cases the clinical benefits partially declined after three to eight weeks. At the eight month follow up, however, the pretreatment examination showed a significant improvement compared with the initial evaluation. The effects of each IVIg course were still present after a number of courses. Electrophysiological study revealed a decrease in conduction block in one or more nerves in all patients. However, conduction block was unchanged or increased in other sites. IVIg treatment did not affect anti-GM1 antibody titres. [ABSTRACT FROM AUTHOR]

Published
1994

14. Prospective open-label trial with rituximab in patients with chronic inflammatory demyelinating polyradiculoneuropathy not responding to conventional immune therapies.

Author

Doneddu PE, Cocito D, Fazio R, Benedetti L, Peci E, Liberatore G, Falzone YM, Germano F, Gallia F, Giannotta C, Lleixà C, Bianchi E, and Nobile-Orazio E

Subjects
Humans, Male, Female, Middle Aged, Prospective Studies, Adult, Aged, Treatment Outcome, Neural Conduction drug effects, Polyradiculoneuropathy, Chronic Inflammatory Demyelinating drug therapy, Rituximab therapeutic use, Immunologic Factors therapeutic use
Abstract

Background: To evaluate the efficacy of rituximab in chronic inflammatory demyelinating polyradiculoneuropathy (CIDP) patients not responding to conventional immune therapies., Methods: An open-label, prospective exploratory study was conducted with intravenous rituximab on 17 CIDP patients who had not responded to at least two first-line therapies. The primary endpoint was to determine the proportion of patients who showed improvement 6 months after rituximab therapy. The percentage of responders to rituximab, along with a 95% CI, was reported and compared with the 30% response rate after other immunosuppressive drugs previously documented in the literature., Results: 13 of the 17 treated patients (76.5%) showed improvement at 6 months (95% CI 50.1 to 93.2). Among the 14 patients who completed the 12-month follow-up (2 were lost to follow-up after showing improvement at months 8 and 10, and 1 deteriorated at 6 months), 13 (92.9%) demonstrated improvement at 12 months (95% CI 66.1 to 99.8). Nerve conduction parameters improved by at least 20% in two nerves in 6 out of 15 (40%) patients at 6 months and in 7 out of 13 (53.9%) at 12 months. None of the treated patients withdrew from the study due to side effects. There was a significant reduction of circulating CD19+ cells 15 days, 2, 6 and 12 months after treatment., Conclusion: Rituximab seems to be a safe therapy in most patients with CIDP not responding to conventional immune therapies. The high percentage of patients who improved in this study suggests a possible positive effect of rituximab which is worth investigating in future randomised controlled clinical trials., Trial Registration Number: NCT05877040., Competing Interests: Competing interests: PED received travel grants to attend scientific meetings from CSL Behring—Italy and Kedrion—Italy. DC received honoraria for lecturing from CSL Behring—Italy, Kedrion—Italy and Shire/Takeda—Italy and received travel grants to attend scientific meetings from CSL Behring—Italy, Kedrion—Italy and Shire/Takeda. RF has served on scientific advisory boards for CSL Behring—Italy and received travel grants to attend scientific meetings from CSL Behring—Italy and Kedrion—Italy. EP has received travel grants to attend scientific meetings from CSL Behring—Italy. GL has received travel grants to attend scientific meetings from CSL Behring—Italy and Kedrion—Italy. EN-O reports personal fees for Advisory or Scientific Board from ArgenX—Belgium, CSL-Behring—USA, Dianthus—USA; Janssen—USA, Kedrion—Italy, LFB—France, Longboard Pharma—USA, Roche—Switzerland, Sanofi—USA and received travel grants to attend scientific meetings from CSL Behring—Italy and Kedrion—Italy. The other authors declare no conflict of interest., (© Author(s) (or their employer(s)) 2024. No commercial re-use. See rights and permissions. Published by BMJ.)

Published
2024
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15. Unclassified clinical presentations of chronic inflammatory demyelinating polyradiculoneuropathy.

Author

Doneddu PE, Akyil H, Manganelli F, Briani C, Cocito D, Benedetti L, Mazzeo A, Fazio R, Filosto M, Cosentino G, Di Stefano V, Antonini G, Marfia GA, Inghilleri M, Siciliano G, Clerici AM, Carpo M, Schenone A, Luigetti M, Lauria G, Matà S, Rosso T, Minicuci GM, Lucchetta M, Cavaletti G, Liberatore G, Spina E, Campagnolo M, Peci E, Germano F, Gentile L, Strano C, Cotti Piccinelli S, Vegezzi E, Leonardi L, Mataluni G, Ceccanti M, Schirinzi E, Romozzi M, and Nobile-Orazio E

Subjects
Humans, Peripheral Nerves, Cranial Nerves, Phenotype, Neural Conduction physiology, Polyradiculoneuropathy, Chronic Inflammatory Demyelinating diagnosis, Polyneuropathies
Abstract

Background: To assess the ability of the 2021 European Academy of Neurology/Peripheral Nerve Society (EAN/PNS) clinical criteria for chronic inflammatory demyelinating polyradiculoneuropathy (CIDP) to include within their classification the whole spectrum of clinical heterogeneity of the disease and to define the clinical characteristics of the unclassifiable clinical forms., Methods: The 2021 EAN/PNS clinical criteria for CIDP were applied to 329 patients fulfilling the electrodiagnostic (and in some cases also the supportive) criteria for the diagnosis of CIDP. Clinical characteristics were reviewed for each patient not strictly fulfilling the clinical criteria ('unclassifiable')., Results: At study inclusion, 124 (37.5%) patients had an unclassifiable clinical presentation, including 110 (89%) with a typical CIDP-like clinical phenotype in whom some segments of the four limbs were unaffected by weakness ('incomplete typical CIDP'), 10 (8%) with a mild distal, symmetric, sensory or sensorimotor polyneuropathy confined to the lower limbs with cranial nerve involvement ('cranial nerve predominant CIDP') and 4 (1%) with a symmetric sensorimotor polyneuropathy limited to the proximal and distal areas of the lower limbs ('paraparetic CIDP'). Eighty-one (65%) patients maintained an unclassifiable presentation during the entire disease follow-up while 13 patients progressed to typical CIDP. Patients with the unclassifiable clinical forms compared with patients with typical CIDP had a milder form of CIDP, while there was no difference in the distribution patterns of demyelination., Conclusions: A proportion of patients with CIDP do not strictly fulfil the 2021 EAN/PNS clinical criteria for diagnosis. These unclassifiable clinical phenotypes may pose diagnostic challenges and thus deserve more attention in clinical practice and research., Competing Interests: Competing interests: PED has received travel grants to attend scientific meetings from CSL Behring and Kedrion. Fiore Manganelli reports personal fees for scientific events from CSL Behring and has received travel grants to attend scientific meetings from CSL Behring and Kedrion. Dario Cocito has received honoraria for lecturing from Shire, CSL Behring, and Kedrion and travel grants to attend scientific meeting from Shire, Kedrion and CSL Behring. RF has served on scientific advisory boards for CSL Behring and has received travel grants from Kedrion and CSL Behring to attend scientific meeting. CB has served on scientific advisory boards for Pfizer, Alnylam, and Akcea, and has received travel grants from Kedrion and CSL Behring to attend scientific meeting. AM has received travel grants from Kedrion and CSL Behring to attend scientific meeting. MF has served on scientific advisory boards for CSL Behring, Sanofi and Amicus and has received travel grants from Sanofi, Biogen, Kedrion and CSL Behring to attend scientific meeting. GC has received travel grants to attend scientific meetings from CSL Behring and Kedrion. ML has received travel grants to attend scientific meetings from Kedrion. GAM has received travel grants to attend scientific meetings from CSL Behring and Kedrion. GL has received travel grants to attend scientific meetings from CSL Behring and Kedrion. EP has received travel grants to attend scientific meetings from CSL Behring. TR has received travel grants to attend scientific meetings from CSL Behring. EN-O reports personal fees for Advisory or Scientific Board from ArgenX—Belgium, Takeda—Italy and USA, CSL-Behring—Italy and USA, Janssen—USA, Kedrion—Italy, LFB—France, Roche—Switzerland, Sanofi—USA. The other authors declare no conflict of interest., (© Author(s) (or their employer(s)) 2023. No commercial re-use. See rights and permissions. Published by BMJ.)

Published
2023
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16. Comparison of the diagnostic accuracy of the 2021 EAN/PNS and 2010 EFNS/PNS diagnostic criteria for chronic inflammatory demyelinating polyradiculoneuropathy.

Author

Doneddu PE, De Lorenzo A, Manganelli F, Cocito D, Fazio R, Briani C, Mazzeo A, Filosto M, Cosentino G, Benedetti L, Schenone A, Marfia GA, Antonini G, Matà S, Luigetti M, Liberatore G, Spina E, Peci E, Strano C, Cacciavillani M, Gentile L, Cotti Piccinelli S, Cortese A, Bianchi E, and Nobile-Orazio E

Subjects
Humans, Retrospective Studies, Peripheral Nerves, Sensitivity and Specificity, Neural Conduction physiology, Polyradiculoneuropathy, Chronic Inflammatory Demyelinating diagnosis, Neurology
Abstract

Objectives: To compare the sensitivity and specificity of the 2021 European Academy of Neurology/Peripheral Nerve Society (EAN/PNS) diagnostic criteria for chronic inflammatory demyelinating polyradiculoneuropathy (CIDP) with those of the 2010 European Federation of Neurological Societies/Peripheral Nerve Society (EFNS/PNS)., Methods: Sensitivity and specificity of the two sets of criteria were evaluated in 330 patients with CIDP and 166 axonal peripheral neuropathy controls. Comparison of the utility of nerve conduction studies with different number of nerves examined and of the sensitivity and specificity of the two criteria in typical CIDP and its variants were assessed., Results: EFNS/PNS criteria had a sensitivity of 92% for possible CIDP and 85% for probable/definite CIDP, while the EAN/PNS criteria had a sensitivity of 83% for possible CIDP and 74% for CIDP. Using supportive criteria, the sensitivity of the EAN/PNS criteria for possible CIDP increased to 85% and that of CIDP to 77%, remaining lower than that of the EFNS/PNS criteria. Specificity of the EFNS/PNS criteria was 68% for possible CIDP and 84% for probable/definite CIDP, while the EAN/PNS criteria had a specificity of 88% for possible CIDP and 98% for CIDP. More extended studies increased the sensitivity of both sets of criteria by 4%-7% but reduced their specificity by 2%-3%. The EFNS/PNS criteria were more sensitive for the diagnosis of typical CIDP while the EAN/PNS criteria were more specific for the diagnosis of distal and sensory CIDP., Conclusions: In our population, the EAN/PNS criteria were more specific but less sensitive than the EFNS/PNS criteria. With the EAN/PNS criteria, more extended nerve conduction studies are recommended to obtain an acceptable sensitivity while maintaining a high specificity., Competing Interests: Competing interests: Pietro Emiliano Doneddu has received travel grants to attend scientific meetings from CSL Behring and Kedrion. Fiore Manganelli reports personal fees for scientific events from CSL Behring and has received travel grants to attend scientific meetings from CSL Behring and Kedrion. Dario Cocito has received honoraria for lecturing from Shire, CSL Behring, and Kedrion and travel grants to attend scientific meeting from Shire, Kedrion, and CSL Behring. Raffaella Fazio has served on scientific advisory boards for CSL Behring and has received travel grants from Kedrion and CSL Behring to attend scientific meeting. Chiara Briani has served on scientific advisory boards for Pfizer, Alnylam, and Akcea, and has received travel grants from Kedrion and CSL Behring to attend scientific meeting. Anna Mazzeo has received travel grants from Kedrion and CSL Behring to attend scientific meeting. Massimiliano Filosto has served on scientific advisory boards for CSL Behring, Sanofi and Amicus and has received travel grants from Sanofi, Biogen, Kedrion and CSL Behring to attend scientific meeting. Giuseppe Cosentino has received travel grants to attend scientific meetings from CSL Behring and Kedrion. Marco Luigetti has received travel grants to attend scientific meetings from Kedrion. Girolama Alessandra Marfia has received travel grants to attend scientific meetings from CSL Behring and Kedrion. Giuseppe Liberatore has received travel grants to attend scientific meetings from CSL Behring and Kedrion. Erdita Peci has received travel grants to attend scientific meetings from CSL Behring. Eduardo Nobile-Orazio reports personal fees for Advisory or Scientific Board from ArgenX—Belgium, Takeda—Italy and USA, CSL-Behring - Italy and USA, Janssen—USA, Kedrion—Italy, LFB—France, Roche—Switzerland, Sanofi - USA. The other authors declare no conflict of interest., (© Author(s) (or their employer(s)) 2022. No commercial re-use. See rights and permissions. Published by BMJ.)

Published
2022
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17. Guillain-Barré syndrome and COVID-19: an observational multicentre study from two Italian hotspot regions.

Author

Filosto M, Cotti Piccinelli S, Gazzina S, Foresti C, Frigeni B, Servalli MC, Sessa M, Cosentino G, Marchioni E, Ravaglia S, Briani C, Castellani F, Zara G, Bianchi F, Del Carro U, Fazio R, Filippi M, Magni E, Natalini G, Palmerini F, Perotti AM, Bellomo A, Osio M, Scopelliti G, Carpo M, Rasera A, Squintani G, Doneddu PE, Bertasi V, Cotelli MS, Bertolasi L, Fabrizi GM, Ferrari S, Ranieri F, Caprioli F, Grappa E, Broglio L, De Maria G, Leggio U, Poli L, Rasulo F, Latronico N, Nobile-Orazio E, Padovani A, and Uncini A

Subjects
Adult, Aged, COVID-19 diagnosis, COVID-19 therapy, Female, Guillain-Barre Syndrome diagnosis, Guillain-Barre Syndrome therapy, Hospitalization, Humans, Incidence, Italy, Male, Middle Aged, Referral and Consultation, Retrospective Studies, COVID-19 complications, Guillain-Barre Syndrome epidemiology
Abstract

Objective: Single cases and small series of Guillain-Barré syndrome (GBS) have been reported during the SARS-CoV-2 outbreak worldwide. We evaluated incidence and clinical features of GBS in a cohort of patients from two regions of northern Italy with the highest number of patients with COVID-19., Methods: GBS cases diagnosed in 12 referral hospitals from Lombardy and Veneto in March and April 2020 were retrospectively collected. As a control population, GBS diagnosed in March and April 2019 in the same hospitals were considered., Results: Incidence of GBS in March and April 2020 was 0.202/100 000/month (estimated rate 2.43/100 000/year) vs 0.077/100 000/month (estimated rate 0.93/100 000/year) in the same months of 2019 with a 2.6-fold increase. Estimated incidence of GBS in COVID-19-positive patients was 47.9/100 000 and in the COVID-19-positive hospitalised patients was 236/100 000. COVID-19-positive patients with GBS, when compared with COVID-19-negative subjects, showed lower MRC sum score (26.3±18.3 vs 41.4±14.8, p=0.006), higher frequency of demyelinating subtype (76.6% vs 35.3%, p=0.011), more frequent low blood pressure (50% vs 11.8%, p=0.017) and higher rate of admission to intensive care unit (66.6% vs 17.6%, p=0.002)., Conclusions: This study shows an increased incidence of GBS during the COVID-19 outbreak in northern Italy, supporting a pathogenic link. COVID-19-associated GBS is predominantly demyelinating and seems to be more severe than non-COVID-19 GBS, although it is likely that in some patients the systemic impairment due to COVID-19 might have contributed to the severity of the whole clinical picture., Competing Interests: Competing interests: None declared., (© Author(s) (or their employer(s)) 2021. No commercial re-use. See rights and permissions. Published by BMJ.)

Published
2021
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18. Frequency of diabetes and other comorbidities in chronic inflammatory demyelinating polyradiculoneuropathy and their impact on clinical presentation and response to therapy.

Author

Doneddu PE, Cocito D, Manganelli F, Fazio R, Briani C, Filosto M, Benedetti L, Bianchi E, Jann S, Mazzeo A, Antonini G, Cosentino G, Marfia GA, Cortese A, Clerici AM, Carpo M, Schenone A, Siciliano G, Luigetti M, Lauria G, Rosso T, Cavaletti G, Beghi E, Liberatore G, Santoro L, Spina E, Peci E, Tronci S, Ruiz M, Cotti Piccinelli S, Verrengia EP, Gentile L, Leonardi L, Mataluni G, Piccolo L, and Nobile-Orazio E

Subjects
Adolescent, Adrenal Cortex Hormones therapeutic use, Adult, Age of Onset, Aged, Aged, 80 and over, Autoimmune Diseases epidemiology, Child, Comorbidity, Female, Humans, Immunoglobulins, Intravenous therapeutic use, Immunologic Factors therapeutic use, Italy epidemiology, Male, Middle Aged, Plasma Exchange, Polyradiculoneuropathy, Chronic Inflammatory Demyelinating therapy, Quality of Life, Sex Distribution, Treatment Outcome, Young Adult, Diabetes Mellitus epidemiology, Diabetic Neuropathies epidemiology, Monoclonal Gammopathy of Undetermined Significance epidemiology, Polyradiculoneuropathy, Chronic Inflammatory Demyelinating epidemiology
Abstract

Objectives: To determine the prevalence of different comorbidities in chronic inflammatory demyelinating polyradiculoneuropathy (CIDP), and their impact on outcome, treatment choice and response., Methods: Using a structured questionnaire, we collected information on comorbidities from 393 patients with CIDP fulfilling the European Federation of Neurological Societies and Peripheral Nerve Society criteria included in the Italian CIDP database., Results: One or more comorbidities were reported by 294 patients (75%) and potentially influenced treatment choice in 192 (49%) leading to a less frequent use of corticosteroids. Response to treatment did not differ, however, from that in patients without comorbidities. Diabetes (14%), monoclonal gammopathy of undetermined significance (MGUS) (12%) and other immune disorders (16%) were significantly more frequent in patients with CIDP than expected in the general European population. Patients with diabetes had higher disability scores, worse quality of life and a less frequent treatment response compared with patients without diabetes. Patients with IgG-IgA or IgM MGUS had an older age at CIDP onset while patients with other immune disorders had a younger age at onset and were more frequently females. IgM MGUS was more frequent in patients with motor CIDP than in patients with typical CIDP., Conclusions: Comorbidities are frequent in patients with CIDP and in almost 50% of them have an impact on treatment choice. Diabetes, MGUS and other immune diseases are more frequent in patients with CIDP than in the general population. Only diabetes seems, however, to have an impact on disease severity and treatment response possibly reflecting in some patients a coexisting diabetic neuropathy., Competing Interests: Competing interests: PED has received travel grants to attend scientific meetings from CSL Behring and Kedrion. DC has received honoraria for lecturing from Shire, CSL Behring and Kedrion and travel grants to attend scientific meeting from Shire, Kedrion, and CSL Behring. FM reports personal fees for scientific events from CSL Behring and has received travel grants to attend scientific meetings from CSL Behring and Kedrion. RF has served on scientific advisory boards for CSL Behring and has received travel grants from Kedrion and CSL Behring to attend scientific meeting. Chiara Briani has served on scientific advisory boards for Pfizer, Alnylam, and Akcea, and has received travel grants from Kedrion and CSL Behring to attend scientific meeting. MF has served on scientific advisory boards for CSL Behring and has received travel grants from Kedrion, Baxter and CSL Behring to attend scientific meeting. SJ has received research grants from Grifols, outside this work, and travel grants from Grifols and Kedrion. Anna Mazzeo has received travel grants from Kedrion and CSL Behring to attend scientific meeting. GC has received travel grants to attend scientific meetings from CSL Behring and Kedrion. AC has received travel grants to attend scientific meetings from Kedrion. MC has received travel grants to attend scientific meetings from Kedrion. ML has received travel grants to attend scientific meetings from Kedrion. GC has received honoraria for lecturing and travel grants to attend scientific meetings from Kedrion. E Beghi reports grants from UCB-Pharma, grants from Shire, grants from EISAI, personal fees from Viropharma, grants from Italian Ministry of Health, grants from Fondazione Borgonovo, grants from Associazione IDIC 15, grants from European Union, outside the submitted work. Giuseppe Liberatore has received travel grants to attend scientific meetings from CSL Behring and Kedrion. Lucio Santoro reports personal fees for scientific events from CSL Behring and has received travel grants to attend scientific meetings from CSL Behring and Kedrion. EP has received travel grants to attend scientific meetings from CSL Behring. Eduardo Nobile Orazio reports personal fees for Advisory or Scientific Board from Kedrion, Italy, Baxter, Italy, Novartis, Switzerland, CSL-Behring, Italy, Astellas, the Netherlands, outside the submitted work and travel grants to attend Scientific Meeting from Baxter, Grifols, Kedrion, and Novartis, Italy. The other authors declare no conflict of interest., (© Author(s) (or their employer(s)) 2020. No commercial re-use. See rights and permissions. Published by BMJ.)

Published
2020
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19. Clinical features and outcomes of the flail arm and flail leg and pure lower motor neuron MND variants: a multicentre Italian study.

Author

Schito P, Ceccardi G, Calvo A, Falzone YM, Moglia C, Lunetta C, Marinou K, Ticozzi N, Scialo C, Sorarù G, Trojsi F, Conte A, Tortelli R, Russo M, Zucchi E, Pozzi L, Domi T, Carrera P, Agosta F, Quattrini A, Fazio R, Chiò A, Sansone VA, Mora G, Silani V, Volanti P, Caponnetto C, Querin G, Tedeschi G, Sabatelli M, Logroscino G, Messina S, Mandrioli J, Riva N, and Filippi M

Subjects
Age of Onset, Aged, Female, Humans, Italy, Male, Middle Aged, Motor Neuron Disease pathology, Phenotype, Retrospective Studies, Sex Factors, Arm physiopathology, Leg physiopathology, Motor Neuron Disease diagnosis, Motor Neuron Disease physiopathology, Motor Neurons pathology
Abstract

Competing Interests: Competing interests: None declared.

Published
2020
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20. Atypical CIDP: diagnostic criteria, progression and treatment response. Data from the Italian CIDP Database.

Author

Doneddu PE, Cocito D, Manganelli F, Fazio R, Briani C, Filosto M, Benedetti L, Mazzeo A, Marfia GA, Cortese A, Fierro B, Jann S, Beghi E, Clerici AM, Carpo M, Schenone A, Luigetti M, Lauria G, Antonini G, Rosso T, Siciliano G, Cavaletti G, Liberatore G, Santoro L, Peci E, Tronci S, Ruiz M, Cotti Piccinelli S, Toscano A, Mataluni G, Piccolo L, Cosentino G, Sabatelli M, and Nobile-Orazio E

Subjects
Adolescent, Adult, Aged, Aged, 80 and over, Child, Databases, Factual, Disease Progression, Female, Humans, Italy, Kaplan-Meier Estimate, Male, Middle Aged, Polyradiculoneuropathy, Chronic Inflammatory Demyelinating therapy, Retrospective Studies, Young Adult, Polyradiculoneuropathy, Chronic Inflammatory Demyelinating complications, Polyradiculoneuropathy, Chronic Inflammatory Demyelinating diagnosis
Abstract

Objectives: A few variants of chronic inflammatory demyelinating polyradiculoneuropathy (CIDP) have been described, but their frequency and evolution to typical CIDP remain unclear. To determine the frequency and characteristics of the CIDP variants, their possible evolution to typical CIDP, and treatment response., Methods: We applied a set of diagnostic criteria to 460 patients included in a database of Italian patients with CIDP. Clinical characteristics and treatment response were reviewed for each patient. The Kaplan-Meier curve was used to estimate the progression rate from atypical to typical CIDP., Results: At the time of inclusion, 376 (82%) patients had a diagnosis of typical CIDP while 84 (18%) had atypical CIDP, including 34 (7%) with distal acquired demyelinating symmetric neuropathy (DADS), 17 (4%) with purely motor, 17 (4%) with Lewis-Sumner syndrome (LSS) and 16 (3.5%) with purely sensory CIDP. Based on retrospective review of the symptoms and signs present at onset and for at least 1 year, 180 (39%) patients had an initial diagnosis compatible with atypical CIDP that in 96 (53%) patients evolved to typical CIDP. Mean disease duration was longer in patients evolving to typical CIDP than in those not evolving (p=0.0016). Patients with DADS and LSS had a less frequent response to immunoglobulin than those with typical CIDP, while patients with purely motor and sensory CIDP had a similar treatment response., Conclusions: The proportion of patients with atypical CIDP varies during the disease course. DADS and LSS have a less frequent response to intravenous immunoglobulin compared with typical CIDP, raising the possibility of a different underlying pathogenetic mechanism., Competing Interests: Competing interests: EN-O reports personal fees for Advisory or Scientific Board from Kedrion, Italy, Baxter, Italy, Novartis, Switzerland, CSL Behring, Italy, Astellas, the Netherlands, outside the submitted work and travel grants to attend scientific meeting from Baxter, Grifols, Kedrion and Novartis, Italy. PED has received travel grants to attend scientific meetings from CSL Behring and Kedrion. GLib has received travel grants to attend scientific meetings from CSL Behring and Kedrion. DC has received honoraria for lecturing from Shire, CSL Behring and Kedrion and travel grants to attend scientific meeting from Shire, Kedrion and CSL Behring. EP has received travel grants to attend scientific meetings from CSL Behring. RF has served on scientific advisory boards for CSL Behring and has received travel grants from Kedrion and CSL Behring to attend scientific meeting. MC has received travel grants to attend scientific meetings from Kedrion. AM has received travel grants from Kedrion and CSL Behring to attend scientific meeting. CB has served on scientific advisory boards for Pfizer and has received travel grants from Kedrion and CSL Behring to attend scientific meeting. GC has received travel grants to attend scientific meetings from CSL Behring and Kedrion. BF has received travel grants to attend scientific meetings from CSL Behring and liberal contribution from CSL Behring for the neuromuscular diseases centre, outside the submitted work. EB reports grants from UCB-Pharma, grants from Shire, grants from EISAI, personal fees from Viropharma, grants from Italian Ministry of Health, grants from Fondazione Borgonovo, grants from Associazione IDIC 15, grants from European Union, outside the submitted work. AC has received travel grants to attend scientific meetings from Kedrion. ML has received honoraria for scientific board from Pfeizer and Alnylam and travel grants from Grifols and Kedrion to attend scientific meeting. LS reports personal fees for scientific events from CSL Behring and has received travel grants to attend scientific meetings from CSL Behring and Kedrion. FM reports personal fees for scientific events from CSL Behring and has received travel grants to attend scientific meetings from CSL Behring and Kedrion. GC has received honoraria for lecturing and travel grants to attend scientific meetings from Kedrion. MF has served on scientific advisory boards for CSL Behring and has received travel grants from Kedrion, Baxter and CSL Behring to attend scientific meeting. SJ has received research grants from Grifols, outside this work, and travel grants from Grifols and Kedrion., (© Author(s) (or their employer(s)) 2019. No commercial re-use. See rights and permissions. Published by BMJ.)

Published
2019
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21. IgM MGUS and Waldenstrom-associated anti-MAG neuropathies display similar response to rituximab therapy.

Author

Campagnolo M, Zambello R, Nobile-Orazio E, Benedetti L, Marfia GA, Riva N, Castellani F, Bianco M, Salvalaggio A, Garnero M, Ruiz M, Mataluni G, Fazio R, Ermani M, and Briani C

Subjects
Adult, Aged, Aged, 80 and over, Autoantibodies blood, Female, Humans, Male, Middle Aged, Peripheral Nervous System Diseases blood, Retrospective Studies, Waldenstrom Macroglobulinemia blood, Waldenstrom Macroglobulinemia complications, Immunoglobulin M immunology, Immunologic Factors therapeutic use, Peripheral Nervous System Diseases etiology, Rituximab therapeutic use, Waldenstrom Macroglobulinemia drug therapy
Abstract

Competing Interests: Competing interests: None declared.

Published
2017
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22. TBK1 mutations in Italian patients with amyotrophic lateral sclerosis: genetic and functional characterisation.

Author

Pozzi L, Valenza F, Mosca L, Dal Mas A, Domi T, Romano A, Tarlarini C, Falzone YM, Tremolizzo L, Sorarù G, Cerri F, Ferraro PM, Basaia S, Agosta F, Fazio R, Comola M, Comi G, Ferrari M, Quattrini A, Lunetta C, Penco S, Bonanomi D, Carrera P, and Riva N

Subjects
Adult, Aged, Cohort Studies, Female, Humans, Italy, Male, Middle Aged, Pedigree, Amyotrophic Lateral Sclerosis genetics, Mutation, Protein Serine-Threonine Kinases genetics
Abstract

Background: TANK-binding kinase 1 ( TBK1 ) gene has been recently identified as a causative gene of amyotrophic lateral sclerosis (ALS)., Methods: We sequenced the TBK1 gene in a cohort of 154 Italian patients with ALS with unclear genetic aetiology. We subsequently assessed the pathogenic potential of novel identified TBK1 variants using functional in vitro studies: expression, targeting and activity were evaluated in patient-derived fibroblasts and in cells transfected with mutated- TBK1 plasmids., Results: We identified novel genomic TBK1 variants including two loss-of-function (LoF) (p.Leu59Phefs*16 and c.358+5G>A), two missense (p.Asp118Asn and p.Ile397Thr) and one intronic variant (c.1644-5_1644-2delAATA), in addition to two previously reported pathogenetic missense variants (p.Lys291Glu and p.Arg357Gln). Functional studies in patient-derived fibroblasts revealed that the c.358+5G>A causes aberrant pre-mRNA processing leading TBK1 haploinsufficiency. Biochemical studies in cellular models showed that the truncating variant p.Leu59Phefs*16 abolishes TBK1 protein expression, whereas the p.Asp118Asn variant severely impairs TBK1 phosphorylation activity. Conversely, the p.Ile397Thr variant displayed enhanced phosphorylation activity, whose biological relevance is not clear., Conclusion: The observed frequency of TBK1 LoF variants was 1.3% (2/154), increasing up to 3.2% (5/154) by taking into account also the functional missense variants that we were able to classify as potentially pathogenic, supporting the relevance of TBK1 in the Italian population with ALS., Competing Interests: Competing interests: None declared., (© Article author(s) (or their employer(s) unless otherwise stated in the text of the article) 2017. All rights reserved. No commercial use is permitted unless otherwise expressly granted.)

Published
2017
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23. Subcutaneous immunoglobulin in CIDP and MMN: a different long-term clinical response?

Author

Cocito D, Merola A, Romagnolo A, Peci E, Toscano A, Mazzeo A, Gentile L, Russo M, Fazio R, Filosto M, Siciliano G, Schirinzi E, Nobile-Orazio E, and Lopiano L

Subjects
Humans, Immunoglobulins, Intravenous administration & dosage, Injections, Subcutaneous, Italy, Middle Aged, Retrospective Studies, Immunoglobulins administration & dosage, Motor Neuron Disease drug therapy, Polyradiculoneuropathy, Chronic Inflammatory Demyelinating drug therapy
Published
2016
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24. Erythropoietin in amyotrophic lateral sclerosis: a multicentre, randomised, double blind, placebo controlled, phase III study.

Author

Lauria G, Dalla Bella E, Antonini G, Borghero G, Capasso M, Caponnetto C, Chiò A, Corbo M, Eleopra R, Fazio R, Filosto M, Giannini F, Granieri E, La Bella V, Logroscino G, Mandrioli J, Mazzini L, Monsurrò MR, Mora G, Pietrini V, Quatrale R, Rizzi R, Salvi F, Siciliano G, Sorarù G, Volanti P, Tramacere I, and Filippini G

Subjects
Adult, Aged, Amyotrophic Lateral Sclerosis mortality, Double-Blind Method, Epoetin Alfa, Erythropoietin adverse effects, Female, Humans, Male, Middle Aged, Recombinant Proteins adverse effects, Recombinant Proteins therapeutic use, Treatment Outcome, Amyotrophic Lateral Sclerosis drug therapy, Erythropoietin therapeutic use
Abstract

Objective: To assess the efficacy of recombinant human erythropoietin (rhEPO) in amyotrophic lateral sclerosis (ALS)., Methods: Patients with probable laboratory-supported, probable or definite ALS were enrolled by 25 Italian centres and randomly assigned (1:1) to receive intravenous rhEPO 40,000 IU or placebo fortnightly as add-on treatment to riluzole 100 mg daily for 12 months. The primary composite outcome was survival, tracheotomy or >23 h non-invasive ventilation (NIV). Secondary outcomes were ALSFRS-R, slow vital capacity (sVC) and quality of life (ALSAQ-40) decline. Tolerability was evaluated analysing adverse events (AEs) causing withdrawal. The randomisation sequence was computer-generated by blocks, stratified by centre, disease severity (ALSFRS-R cut-off score of 33) and onset (spinal or bulbar). The main outcome analysis was performed in all randomised patients and by intention-to-treat for the entire population and patients stratified by severity and onset. The study is registered, EudraCT 2009-016066-91., Results: We randomly assigned 208 patients, of whom 5 (1 rhEPO and 4 placebo) withdrew consent and 3 (placebo) became ineligible (retinal thrombosis, respiratory insufficiency, SOD1 mutation) before receiving treatment; 103 receiving rhEPO and 97 placebo were eligible for analysis. At 12 months, the annualised rate of death (rhEPO 0.11, 95% CI 0.06 to 0.20; placebo: 0.08, CI 0.04 to 0.17), tracheotomy or >23 h NIV (rhEPO 0.16, CI 0.10 to 0.27; placebo 0.18, CI 0.11 to 0.30) did not differ between groups, also after stratification by onset and ALSFRS-R at baseline. Withdrawal due to AE was 16.5% in rhEPO and 8.3% in placebo. No differences were found for secondary outcomes., Conclusions: RhEPO 40,000 IU fortnightly did not change the course of ALS., (Published by the BMJ Publishing Group Limited. For permission to use (where not already granted under a licence) please go to http://group.bmj.com/group/rights-licensing/permissions.)

Published
2015
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25. Frequency and time to relapse after discontinuing 6-month therapy with IVIg or pulsed methylprednisolone in CIDP.

Author

Nobile-Orazio E, Cocito D, Jann S, Uncini A, Messina P, Antonini G, Fazio R, Gallia F, Schenone A, Francia A, Pareyson D, Santoro L, Tamburin S, Cavaletti G, Giannini F, Sabatelli M, and Beghi E

Subjects
Anti-Inflammatory Agents administration & dosage, Humans, Immunoglobulins, Intravenous administration & dosage, Immunologic Factors administration & dosage, Methylprednisolone administration & dosage, Polyradiculoneuropathy, Chronic Inflammatory Demyelinating epidemiology, Polyradiculoneuropathy, Chronic Inflammatory Demyelinating prevention & control, Recurrence, Retrospective Studies, Time Factors, Treatment Outcome, Anti-Inflammatory Agents therapeutic use, Immunoglobulins, Intravenous therapeutic use, Immunologic Factors therapeutic use, Methylprednisolone therapeutic use, Polyradiculoneuropathy, Chronic Inflammatory Demyelinating drug therapy
Abstract

Background: We reported that 6-month therapy with intravenous immunoglobulin (IVIg) was more frequently effective or tolerated than intravenous methylprednisolone (IVMP) in patients with chronic inflammatory demyelinating polyradiculoneuropathy (CIDP). We now retrospectively compared the proportion of patients who eventually worsened after discontinuing therapy and the median time to clinical worsening., Methods: By March 2013, data were available from 41 of the 45 patients completing the trial with a median follow-up after therapy discontinuation of 42 months (range 1-60). Three patients withdrew during the original study and one failed to respond to either of the therapies. No patient received a diagnosis alternative to CIDP during the follow-up., Results: Twenty-eight of the 32 patients treated with IVIg (as primary or secondary therapy after failing to respond to IVMP) improved after therapy (87.5%) as compared with 13 of the 24 patients treated with IVMP as primary or secondary therapy (54.2%). After a median follow-up of 42 months (range 1-57), 24 out of 28 patients responsive to IVIg (85.7%) worsened after therapy discontinuation. The same occurred in 10 out of 13 patients (76.9%) responsive to IVMP (p=0.659) after a median follow-up of 43 months (range 7-60). Worsening occurred 1-24 months (median 4.5) after IVIg discontinuation and 1-31 months (median 14) after IVMP discontinuation (p=0.0126)., Conclusions: A similarly high proportion of patients treated with IVIg or IVMP eventually relapse after therapy discontinuation but the median time to relapse was significantly longer after IVMP than IVIg. This difference may help to balance the more frequent response to IVIg than to IVMP in patients with CIDP., (Published by the BMJ Publishing Group Limited. For permission to use (where not already granted under a licence) please go to http://group.bmj.com/group/rights-licensing/permissions.)

Published
2015
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26. Rituximab in patients with chronic inflammatory demyelinating polyradiculoneuropathy: a report of 13 cases and review of the literature.

Author

Benedetti L, Briani C, Franciotta D, Fazio R, Paolasso I, Comi C, Luigetti M, Sabatelli M, Giannini F, Mancardi GL, Schenone A, Nobile-Orazio E, and Cocito D

Subjects
Adult, Aged, Child, Female, Humans, Male, Middle Aged, Polyradiculoneuropathy, Chronic Inflammatory Demyelinating immunology, Polyradiculoneuropathy, Chronic Inflammatory Demyelinating physiopathology, Retrospective Studies, Rituximab, Treatment Outcome, Antibodies, Monoclonal, Murine-Derived therapeutic use, Immunologic Factors therapeutic use, Polyradiculoneuropathy, Chronic Inflammatory Demyelinating drug therapy
Abstract

Background: A few case reports have shown controversial results of rituximab efficacy in patients with chronic inflammatory demyelinating polyradiculoneuropathy (CIDP)., Objective: To analyse the efficacy of rituximab in a large CIDP cohort., Methods: A retrospective, observational and multicentre study on the use of rituximab in CIDP. 13 Italian CIDP patients were treated with rituximab after the partial or complete lack of efficacy of conventional therapies. Eight patients had co-occurring haematological diseases. Patients who improved by at least two points in standard clinical scales, or who reduced or discontinued the pre-rituximab therapies, were considered as responders., Results: Nine patients (seven with haematological diseases) responded to rituximab: six of them, who were non-responders to conventional therapies, improved clinically, and the other three maintained the improvement that they usually achieved with intravenous immunoglobulin or plasma exchange. Significantly associated with shorter disease duration, rituximab responses started after a median period of 2.0 months (range, 1-6) and lasted for a median period of 1 year (range, 1-5)., Conclusions: Rituximab seems to be a promising therapeutic choice when it targets both CIDP and co-occurring haematological diseases. Timely post-onset administration of rituximab seems to be associated with better responses.

Published
2011
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