Changes in Management of Pediatric-onset Multiple Sclerosis: Are We Really Modifying the Disease Course? Data from the Italian MS Registry

NEUROLOGY(2020)

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摘要
Objective: to evaluate if disease course of pediatric-onset multiple sclerosis (POMS) has changed over time, with the availability of disease modifying therapies (DMTs). Background: with the exception of fingolimod, all DMTs are currently used off label to treat POMS patients. Many observational studies have shown the clinical efficacy of injectable DMTs, however we lack long-term results after new drugs became available. Design/Methods: we conducted a retrospective observational study using data from the Italian MS registry. Primary endpoints were time to reach a moderate (EDSS ≥4) and severe (EDSS ≥6) disability in patients with POMS across four different diagnostic periods (D1: Results: we included 3198 POMS patients: demographics and baseline disease activity were similar across all diagnostic periods. The risk reduction of achieving a moderate (D2: HR 0.69; D3: HR 0.47; D4: HR 0.43; p Conclusions: disease course of POMS has improved in recent years, probably due to the introduction of new and highly effective DMTs. Having more approved drugs available for children with MS will probably further improve the natural history of the disease. Disclosure: Dr. Baroncini has received personal compensation for consulting, serving on a scientific advisory board, speaking, or other activities with Travel funding by TEVA and Roche. Honoraria from NOVARTIS, MERCK, ALMIRALL. Dr. Iaffaldano has received personal compensation for consulting, serving on a scientific advisory board, speaking, or other activities with Biogen Idec, Bayer, Teva, Roche, Merck Serono, Novartis, Genzyme, and Sanofi Aventis.Dr. Simone has nothing to disclose. Dr. Brescia Morra has received personal compensation for consulting, serving on a scientific advisory board, speaking, or other activities with Almirall, Bayer, Biogen Idec, Merk, Novartis, Roche, Teva. Dr. Filippi has received personal compensation for consulting, serving on a scientific advisory board, speaking, or other activities with Bayer, Biogen Idec, Merck-Serono, Novartis, Roche, Sanofi Genzyme, Takeda, and Teva Pharmaceutical Industries. Dr. Filippi has received personal compensation in an editorial capacity for Journal of Neurology. Dr. Filippi has received research support from Biogen Idec, Merck-Serono, Novartis, Roche,Teva Pharmaceutical Industries.Dr. Romeo has received personal compensation for consulting, serving on a scientific advisory board, speaking, or other activities with Genzyme and Merck-Serono. Dr. Patti has received personal compensation for consulting, serving on a scientific advisory board, speaking, or other activities with received personal compensation for speaking and/or advisor board activities by Almirall, Bayer, Biogen, Celgene, Merck, Myalin, Novartis, Roche, Sanofi-Genzyme and TEVA.. Dr. Patti has received research support from received grant research by MIUR (Ministero Italiano della Universita e della Ricerca Scientifica), FISM (Fondazione Italiana Sclerosi Multipla), Biogen and Merck.. Dr. Chisari has received personal compensation for consulting, serving on a scientific advisory board, speaking, or other activities with Honoraria from Sanofi Genzyme. Travel funding from Bayer, TEVA, Sanofi Genzyme, Almirall, Novartis, Roche, Biogen, and Merck.. Dr. Cocco has received personal compensation for consulting, serving on a scientific advisory board, speaking, or other activities with Almirall, Bayer, Biogen, Merck, Novartis, Genzyme and Teva.. Dr. Fenu has received personal compensation for consulting, serving on a scientific advisory board, speaking, or other activities with Novartis, Merck Serono, Teva, and Biogen. Dr. Salemi has nothing to disclose. Dr. Ragonese has received personal compensation for consulting, serving on a scientific advisory board, speaking, or other activities with consulting fees and speaker honoraria from Biogen, Merck, Novartis, Genzyme, and Roche, and Teva; travel grants from Biogen, Merck, Genzyme, Novartis and Teva; research grants from Almirall.. Dr. Inglese has received personal compensation for consulting, serving on a scientific advisory board, speaking, or other activities with Novartis Pharmaceuticals, National Multiple Sclerosis Society, Noto Foundation, NIH, and Teva Neuroscience.Dr. Cellerino has nothing to disclose. Dr. Comi has received personal compensation for consulting, serving on a scientific advisory board, speaking, or other activities with honoraria for consultancy and/or speaking activities from Almirall, Biogen, Bayer, Chugai, Genzyme, Merck Serono, Novartis, Roche, Receptos, Sanofi, Serono Symposia International Foundation, and Teva.. Dr. Comi has received personal compensation in an editorial capacity for Clinical Investigation; European Journal of Neurology and Multiple Sclerosis; Neurological Sciences. Dr. Zaffaroni has received personal compensation for consulting, serving on a scientific advisory board, speaking, or other activities with Almirall, Biogen, Genzyme, Merck, Novartis, and Teva. Dr. Ghezzi has received personal compensation for consulting, serving on a scientific advisory board, speaking, or other activities with Merck, Novartis, Biogen, Genzyme, Teva, Roche, Almirall, Mylan, and Serono Symposia International.Dr. Italian MS Registry has nothing to disclose.
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