Yara Dadalti Fragoso1, Soniza Vieira Alves-Leon2, Amilton Antunes Barreira3, Dagoberto Callegaro4, Maria Lucia Brito Ferreira5, Alessandro Finkelsztejn6, Sidney Gomes7, Marcus Vinicius Magno Goncalves8, Maria Iris Moraes Machado5, Vanessa Daccach Marques3, Andre Palma Cunha Matta9, Regina Maria Papais-Alvarenga10, Samira Luisa Apostolos Pereira4, Carlos Bernardo Tauil11. 1. Department of Neurology and MS Unit, Universidade Metropolitana de Santos, Santos, SP, Brazil. Electronic address: yara@bsnet.com.br. 2. Department of Neurology and MS Unit, Universidade Federal do Rio de Janeiro, Rio de Janeiro, RJ, Brazil; Department of Neurology and MS Unit, Universidade Federal do Estado do Rio de Janeiro, Rio de Janeiro, RJ, Brazil. 3. Department of Neurology and MS Unit, Universidade de Sao Paulo, Ribeirao Preto, SP, Brazil. 4. Department of Neurology and MS Unit, Hospital das Clinicas, Universidade de Sao Paulo, Sao Paulo, SP, Brazil. 5. Department of Neurology and MS Unit, Hospital da Restauracao de Recife, Recife, PE, Brazil. 6. Department of Neurology and MS Unit, Hospital de Clinicas de Porto Alegre, Porto Alegre, RS, Brazil. 7. Department of Neurology and MS Unit, Hospital Beneficencia Portuguesa and Hospital Paulistano, Sao Paulo, SP, Brazil. 8. Department of Neurology and MS Unit, Hospital Regional Hans Dieter Schimidt, Joinville, SC, Brazil. 9. Department of Neurology and MS Unit, Universidade Federal Fluminense, Niteroi, RJ, Brazil. 10. Department of Neurology and MS Unit, Universidade Federal do Estado do Rio de Janeiro, Rio de Janeiro, RJ, Brazil. 11. Department of Neurology and MS Unit, Hospital de Base do Distrito Federal, Brasilia, DF, Brazil.
Abstract
BACKGROUND: There have been no clinical trials for approval of medications for treating multiple sclerosis in patients younger than age 18 years. All treatments are based on personal experience and data from open observational studies. Fingolimod is an oral drug for multiple sclerosis that has been shown to be efficient and safe in adults. The aim of our study is to describe patients with multiple sclerosis who started treatment with fingolimod before the age of 18 years. PARTICIPANTS AND METHODS: Seventeen patients treated with fingolimod were identified in the Brazilian database of children and adolescents with multiple sclerosis. The average time of use of the drug was 8.6 months. RESULTS: Fingolimod showed a good safety and efficacy profile in these patients, all of whom had very active multiple sclerosis. After starting treatment with fingolimod, only one patient had a relapse and a new lesion on magnetic resonance imaging. The patients' degree of disability did not progress. No major adverse events were reported in relation to the first dose of the drug, nor in the short- and medium-term treatment. No patient has been followed for longer than 18 months, thus limiting long-term conclusions. CONCLUSIONS: Off-label use of fingolimod in patients younger than age 18 years may be a good therapeutic option for multiple sclerosis control.
BACKGROUND: There have been no clinical trials for approval of medications for treating multiple sclerosis in patients younger than age 18 years. All treatments are based on personal experience and data from open observational studies. Fingolimod is an oral drug for multiple sclerosis that has been shown to be efficient and safe in adults. The aim of our study is to describe patients with multiple sclerosis who started treatment with fingolimod before the age of 18 years. PARTICIPANTS AND METHODS: Seventeen patients treated with fingolimod were identified in the Brazilian database of children and adolescents with multiple sclerosis. The average time of use of the drug was 8.6 months. RESULTS:Fingolimod showed a good safety and efficacy profile in these patients, all of whom had very active multiple sclerosis. After starting treatment with fingolimod, only one patient had a relapse and a new lesion on magnetic resonance imaging. The patients' degree of disability did not progress. No major adverse events were reported in relation to the first dose of the drug, nor in the short- and medium-term treatment. No patient has been followed for longer than 18 months, thus limiting long-term conclusions. CONCLUSIONS: Off-label use of fingolimod in patients younger than age 18 years may be a good therapeutic option for multiple sclerosis control.
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