Toward European harmonization of national myasthenia gravis registries: modified Delphi procedure-based expert consensus on collectable data
Por:
Slioui, A, Tammam, G, Vanoli, F, Della Marina, A, Vohanka, S, Gilhus, NE, Moroni, I, Leite, MI, Piehl, F, Antozzi, C, Pini, J, Stascheit, F, Attarian, S, Santos, E, Verschuuren, J, Canonge, L, Garcia, J, Perriard, C, Cortés-Vicente, E, Mantegazza, R, Meisel, A, Sacconi, S
Publicada:
11 mar 2025
Resumen:
BackgroundMyasthenia gravis (MG) is a rare autoimmune disorder. Several new treatment concepts have emerged in recent years, but access to these treatments varies due to differing national reimbursement regulations, leading to disparities across Europe. This highlights the need for high-quality data collection by stakeholders to establish MG registries. A European MG registry could help bridge the treatment access gap across different countries, offering critical data to support regulatory decisions, foster international collaborations, and enhance clinical and epidemiological research. Several national MG registries already exist or are in development. To avoid duplication and ensure harmonization in data collection, a modified Delphi procedure was implemented to identify essential data elements for inclusion in national registries.ResultsFollowing a literature review, consultations with patient associations and pharmaceutical companies, and input from multiple European MG experts, 100 data elements were identified. Of these, 62 reached consensus for inclusion and classification, while only 1 item was agreed for exclusion. 30 items failed to reach the >= 80% agreement threshold and were excluded. Among the 62 accepted items, 21 were classified as mandatory data elements, 32 optional, and 9 items pertained to the informed consent form.ConclusionsThrough a modified Delphi procedure, consensus was successfully achieved. This consensus-based approach represents a crucial step toward harmonizing MG registries across Europe. The resulting dataset will facilitate the sharing of knowledge and enhance European collaborations. Furthermore, the harmonized data may assist in regulatory or reimbursement decisions regarding novel therapies, as well as address treatment access disparities between European countries.
Filiaciones:
Slioui, A:
Univ NICE, Nice Univ Hosp, Hop Pasteur 2, Pasteur Hosp 2,Reference Ctr Neuromuscular Disorde, 30 Voie Romaine, F-06000 Nice, France
Tammam, G:
Univ NICE, Nice Univ Hosp, Hop Pasteur 2, Pasteur Hosp 2,Reference Ctr Neuromuscular Disorde, 30 Voie Romaine, F-06000 Nice, France
Pavia Univ, Dept Brain & Behav Sci, IRCCS Fdn Mondino, Pavia, Italy
Vanoli, F:
Fdn IRCCS Ist Neurol Carlo Besta, Neuroimmunol & Neuromuscular Dis Unit, Milan, Italy
Sapienza Univ Rome, Dept Human Neurosci, Rome, Italy
Della Marina, A:
Univ Hosp Essen, Dept Pediat Neurol, Ctr Neuromuscular Disorders, C TNBS, Essen, Germany
Vohanka, S:
Masaryk Univ, Univ Hosp Brno, Dept Pediat Anesthesiol & Intens Care Med, Czechia, Brno, Czech Republic
Gilhus, NE:
Univ Bergen, Dept Clin Med, Bergen, Norway
Moroni, I:
Fdn IRCCS Ist Neurol Carlo Besta, Dept Pediat Neurosci, Milan, Italy
Leite, MI:
Univ Oxford, John Radcliffe Hosp, Nuffield Dept Clin Neurosci, Level 3,West Wing,Headley Way, Oxford OX3 9DU, England
Piehl, F:
Karolinska Univ Hosp, Karolinska Inst, Dept Clin Neurosci, Stockholm, Sweden
Karolinska Univ Hosp, Dept Neurol, Stockholm, Sweden
Antozzi, C:
Fdn IRCCS Ist Neurol Carlo Besta, Neuroimmunol & Neuromuscular Dis Unit, Milan, Italy
Fdn IRCCS Ist Neurol Carlo Besta, Dept Neurol, Milan, Italy
Pini, J:
Univ NICE, Nice Univ Hosp, Hop Pasteur 2, Pasteur Hosp 2,Reference Ctr Neuromuscular Disorde, 30 Voie Romaine, F-06000 Nice, France
Stascheit, F:
Free Univ Berlin, Charitepl 1, D-10117 Berlin, Germany
Univ Med Berlin, Humboldt Univ Berlin, Neurosci Clin Res Ctr, Dept Neurol Expt Neurol, Charitepl 1, D-10117 Berlin, Germany
Attarian, S:
Aix Marseille Univ, Timone Univ Hosp, Reference Ctr Neuromuscular Disorders & ALS, Marseille, France
Santos, E:
Univ Porto, CHU Santo Antonio, Unit Multidisciplinary Res Biomed UMIB, Unit Multidisciplinary Res Biomed,Inst Ciencias Bi, Porto, Portugal
Verschuuren, J:
Leiden Univ, Med Ctr, Dept Neurol, Leiden, Netherlands
Canonge, L:
Univ NICE, Nice Univ Hosp, Hop Pasteur 2, Pasteur Hosp 2,Reference Ctr Neuromuscular Disorde, 30 Voie Romaine, F-06000 Nice, France
Univ Gustave Eiffel, ESIEE Paris, Paris, France
Garcia, J:
Univ NICE, Nice Univ Hosp, Hop Pasteur 2, Pasteur Hosp 2,Reference Ctr Neuromuscular Disorde, 30 Voie Romaine, F-06000 Nice, France
Perriard, C:
Nice Univ Hosp, Lenval Pediat Hosp, Reference Ctr Neuromuscular Disorders, Nice, France
Cortés-Vicente, E:
Hosp Santa Creu & Sant Pau, Radiat Oncol, Barcelona, Spain
Mantegazza, R:
Fdn IRCCS Ist Neurol Carlo Besta, Neuroimmunol & Neuromuscular Dis Unit, Milan, Italy
Meisel, A:
Free Univ Berlin, Charitepl 1, D-10117 Berlin, Germany
Univ Med Berlin, Humboldt Univ Berlin, Neurosci Clin Res Ctr, Dept Neurol Expt Neurol, Charitepl 1, D-10117 Berlin, Germany
Sacconi, S:
Univ NICE, Nice Univ Hosp, Hop Pasteur 2, Pasteur Hosp 2,Reference Ctr Neuromuscular Disorde, 30 Voie Romaine, F-06000 Nice, France
Univ Cote Azur, Hop Pasteur 2, IULS, CNRS,INSERM,SNPM Hop Pasteur 2, 30 Voie Romaine, F-06000 Nice, France
Green Submitted, gold
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