Damiano Baroncini1, Mauro Zaffaroni1, Lucia Moiola2, Lorena Lorefice3, Giuseppe Fenu3, Pietro Iaffaldano4, Marta Simone5, Fulvia Fanelli6, Francesco Patti7, Emanuele D'Amico7, Marco Capobianco8, Antonio Bertolotto8, Paolo Gallo9, Monica Margoni9, Silvia Miante9, Nicoletta Milani10, Maria Pia Amato11, Isabella Righini11, Paolo Bellantonio12, Cinzia Scandellari13, Gianfranco Costantino14, Elio Scarpini15, Roberto Bergamaschi16, Giulia Mallucci16, Giancarlo Comi17, Angelo Ghezzi1. 1. Multiple Sclerosis Study Center, Gallarate Hospital, ASST Valle Olona, Via Eusebio Pastori 4, 21013 Gallarate, Italy. 2. Department of Neurology, San Raffaele Hospital, Milan, Italy. 3. Multiple Sclerosis Center, Binaghi Hospital, ATS Sardegna, Cagliari, Italy/Department of Medical Sciences and Public Health, University of Cagliari, Cagliari, Italy. 4. Department of Basic Medical Sciences, Neurosciences and Sense Organs, University of Bari Aldo Moro, Bari, Italy. 5. Child and Adolescence Neuropsychiatry Unit, Department of Basic Medical Sciences, Neurosciences and Sense Organs, University of Bari Aldo Moro, Bari, Italy. 6. Centro Sclerosi Multipla, Azienda Ospedaliera Sant Andrea, Università degli studi di Roma La Sapienza, Rome, Italy. 7. Centro Sclerosi Multipla, Azienda Ospedaliera Sant Andrea, Università degli studi di Roma La Sapienza,Rome, Italy/Department of Medical and Surgical Science and Advanced Technologies, GF Ingrassia, Neurosciences Section, Multiple Sclerosis Center, University of Catania, Catania, Italy. 8. Neurologia & CRESM (Centro Riferimento Regionale SM), AOU San Luigi, Orbassano, Italy. 9. Multiple Sclerosis Centre, Department of Neurosciences DNS, University Hospital, University of Padua, Padua, Italy. 10. Child Neuropsychiatry Unit, Fondazione IRCCS Istituto Neurologico Carlo Besta, Milan, Italy. 11. Department NEUROFARBA, University of Florence, Florence, Italy. 12. Multiple Sclerosis Center, IRCCS Neuromed, Isernia, Italy. 13. UOSD Riabilitazione e Sclerosi Multipla, Villa Mazzacorati, IRCCS Istituto delle Scienze Neurologiche di Bologna, Bologna, Italy. 14. Simple Unit Multiple Sclerosis, University Hospital of Ospedali Riuniti, Foggia, Italy. 15. Multiple Sclerosis Center "Dino Ferrari," University of Milan IRCCS Fondazione Ca' Granda, Ospedale Maggiore Policlinico, Milan, Italy. 16. Department of Neurology, Neurological Institute C. Mondino, Pavia, Italy. 17. Department of Neurology, San Raffaele Hospital, Milan, Italy/Department of Neurology, Institute of Experimental Neurology, Division of Neuroscience, San Raffaele Hospital, Milan, Italy/.
Abstract
BACKGROUND: Few data are available on very long-term follow-up of pediatric multiple sclerosis (MS) patients treated with disease modifying treatments (DMTs). OBJECTIVES: To present a long-term follow-up of a cohort of Pediatric-MS patients starting injectable first-line agents. METHODS: Data regarding treatments, annualized relapse rate (ARR), Expanded Disability Status Scale (EDSS) score, and serious adverse event were collected. Baseline characteristics were tested in multivariate analysis to identify predictors of disease evolution. RESULTS: In total, 97 patients were followed for 12.5 ± 3.3 years. They started therapy at 13.9 ± 2.1 years, 88 with interferons and 9 with copaxone. During the whole follow-up, 82 patients changed therapy, switching to immunosuppressors/second-line treatment in 58% of cases. Compared to pre-treatment phase, the ARR was significantly reduced during the first treatment (from 3.2 ± 2.6 to 0.7 ± 1.5, p < 0.001), and it remained low during the whole follow-up (0.3 ± 0.2, p < 0.001). At last observation, 40% had disability worsening, but EDSS score remained <4 in 89%. One patient died at age of 23 years due to MS. One case of natalizumab-related progressive multifocal encephalopathy (PML) was recorded. Starting therapy before 12 years of age resulted in a better course of disease in multivariate analysis. CONCLUSION: Pediatric-MS patients benefited from interferons/copaxone, but the majority had to switch to more powerful drugs. Starting therapy before 12 years of age could lead to a more favorable outcome.
BACKGROUND: Few data are available on very long-term follow-up of pediatric multiple sclerosis (MS) patients treated with disease modifying treatments (DMTs). OBJECTIVES: To present a long-term follow-up of a cohort of Pediatric-MS patients starting injectable first-line agents. METHODS: Data regarding treatments, annualized relapse rate (ARR), Expanded Disability Status Scale (EDSS) score, and serious adverse event were collected. Baseline characteristics were tested in multivariate analysis to identify predictors of disease evolution. RESULTS: In total, 97 patients were followed for 12.5 ± 3.3 years. They started therapy at 13.9 ± 2.1 years, 88 with interferons and 9 with copaxone. During the whole follow-up, 82 patients changed therapy, switching to immunosuppressors/second-line treatment in 58% of cases. Compared to pre-treatment phase, the ARR was significantly reduced during the first treatment (from 3.2 ± 2.6 to 0.7 ± 1.5, p < 0.001), and it remained low during the whole follow-up (0.3 ± 0.2, p < 0.001). At last observation, 40% had disability worsening, but EDSS score remained <4 in 89%. One patient died at age of 23 years due to MS. One case of natalizumab-related progressive multifocal encephalopathy (PML) was recorded. Starting therapy before 12 years of age resulted in a better course of disease in multivariate analysis. CONCLUSION: Pediatric-MS patients benefited from interferons/copaxone, but the majority had to switch to more powerful drugs. Starting therapy before 12 years of age could lead to a more favorable outcome.
Authors: Kristen M Krysko; Jennifer Graves; Mary Rensel; Bianca Weinstock-Guttman; Gregory Aaen; Leslie Benson; Tanuja Chitnis; Mark Gorman; Manu Goyal; Lauren Krupp; Timothy Lotze; Soe Mar; Moses Rodriguez; John Rose; Michael Waltz; T Charles Casper; Emmanuelle Waubant Journal: Neurology Date: 2018-10-17 Impact factor: 9.910