Leczenie pediatrycznego stwardnienia rozsianego – praktyczne wytyczne Artykuł przeglądowy
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Opublikowane:
cze 30, 2024
Słowa kluczowe:
pediatryczne stwardnienie rozsiane, terapia, leczenie
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Abstrakt
Stwardnienie rozsiane to rzadko występująca w populacji pediatrycznej choroba zapalno-zwyrodnieniowa, mająca znaczny wpływ na stan neurologiczny pacjenta i jakość jego życia. Agresywny przebieg u młodych chorych determinuje konieczność szybkiego i skutecznego leczenia. Podstawą terapii pacjentów pediatrycznych w Polsce jest „Program Terapeutyczny Leczenia Stwardnienia Rozsianego”. Istnieje możliwość wyboru spośród kilku dostępnych leków: interferonu β, octanu glatirameru, teryflunomidu, fumaranu dimetylu, a w przypadku szybko postępującego przebiegu – fingolimodu i natalizumabu. Coraz częściej bierze się pod uwagę stosowanie terapii o wysokiej skuteczności zamiast eskalacyjnego modelu leczenia. Zawsze jednak do powodzenia leczenia konieczna jest dobra współpraca z pacjentem i jego opiekunami.
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Bibliografia
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2. Renoux C, Vukusic S, Convavreux C et al. Natural history of multiple sclerosis with childhood onset. N Engl J Med. 2007; 356(25): 2603-13.
3. Trojano M, Paolicelli D, Bellacosa A et al. Atypical forms of multiple sclerosis or diffrent phases of a same disease? Neurol Sci. 2004; 25(S4): 323-5.
4. Gorman MP, Healy BC, Polgar-Turscanyi M et al. Increased relapse rate in pediatric-onset compared with adult-onset multiple sclerosis. Arch Neurol. 2009; 66 (1): 54-9.
5. Yeh EA, Weinstock-Guttman B, Ramanathan M et al. Magnetic resonance imaging characteristics of children and adults with paediatric-onset multiple sclerosis. Brain. 2009; 132(12): 3392-400.
6. Ruggieri M, Ianetti P, Polizzi A et al. Multiple sclerosis in children under 10 years of age. Neurol Sci. 2004; 25(Suppl 4): S326-35.
7. Wallach AI, Waltz M, Casper TC et al. Cognitive processing speed in pediatric-onset multiple sclerosis: Baseline characteristics of impairment and prediction of decline. Mult Scler. 2020; 26(14): 1938-47.
8. Brola W, Steinborn B, Niewiada M et al. Pediatric-onset multiple sclerosis in Poland: A registry-based retrospective cohort study. Mult Scler Relat Disord. 2022; 57: 103344.
9. Chitnis T, Tenembaum S, Banwell B et al. Consensus statement: evaluation of new and existing therapeutics for pediatric multiple sclerosi. Mult Scler J. 2012; 18(1):116-27.
10. Chitnis T, Tardieu M, Amato MP et al. International pediatric MS study group clinical trials summit: meeting report. Neurology. 2013; 80(12): 1161-8.
11. Colligan E, Metzler A, Tiryaki E et al. Shared decision-making in multiple sclerosis. Mult Scler. 2017; 23(2): 185-90.
12. Leczenie chorych na stwardnienie rozsiane (ICD-10: G35) – Załącznik B.29.
13. Wong YYM, de Mol CL, van der Vuurst de Vries RM et al. Real-world validation of the 2017 McDonald criteria for diagnosis pediatric MS. Neurol Neuroimmunol Neuroinflamm. 2019; 6(2): e528.
14. Banwell B. Pediatric multiple sclerosis: The 2022 ECTRIMS lecture Mult Scler J. 2023; 29(7): 772-8.
15. Chitnis T, Banwell B, Kappos L et al. Safety and efficacyof teriflunomide in paediatric multiple sclerosis (TERIKIDS): a multicentre, double-blind, phase 3, randomised, placebo-controlled trial. Lancet Neurol. 2021; 20(12): 1001-11.
16. Alroughani R, Das R, Penner R et al. Safety and efficacy of delayed-release dimethyl fumarate in pediatric patients with relapsing multiple-sclerosis (FOCUS). Pediatr Neurol. 2018; 83: 19-24.
17. Alroughani R, Huppke P, Mazurkiewicz-Beldzinska M et al. Delayed-release dimethyl fumarate safety and efficacy in pediatric patients with relapsin-remitting multiple sclerosis. Front Neurol. 2020; 11: 606418.
18. Chitnis T, Arnold DL, Banwell B et al. Trial of fingolimod versus interferon beta-1a in pediatric multiple sclerosis. N Engl J Med. 2018; 379(11): 1017-27.
19. Ghezzi A, Maiola L, Pozzilli C et al. Natalizumab in the pediatric MS population: results of the Italian registry. BMC Neurol. 2015; 15: 174.
20. Otero-Romero S, Lebrun-Frenay C, Reyes S et al. ECTRIMS/EAN consensus on vaccination in people with multiple sclerosis: Improving immunization strategies in the era of highly active immunotherapeutic drugs Mult Scler J. 2023; 29(8): 904-25.
21. McGinley M, Rossman I. Bringing the HEET: The Argument for High-Efficacy Early Treatment for Pediatric-Onset Multiple Sclerosis Neurotherap. 2017; 14: 985-98.
22. Sandesjo F, Wassmer E, Deiva K et al. Current international trends in the treatment of multiple sclerosis in children – Impact od the COVID-19 pandemic. Mult Scler Relat Disord. 2021; 56: 103277.
23. Duffy L, Sarill K, Forbes P et al. Shared Decision Making and Disease Modyfying Therapy in Families of Children and Adolescents with Pediatric Onset Multiple Sclerosis. J Ped Nurs. 2021; 61: P404-9.
2. Renoux C, Vukusic S, Convavreux C et al. Natural history of multiple sclerosis with childhood onset. N Engl J Med. 2007; 356(25): 2603-13.
3. Trojano M, Paolicelli D, Bellacosa A et al. Atypical forms of multiple sclerosis or diffrent phases of a same disease? Neurol Sci. 2004; 25(S4): 323-5.
4. Gorman MP, Healy BC, Polgar-Turscanyi M et al. Increased relapse rate in pediatric-onset compared with adult-onset multiple sclerosis. Arch Neurol. 2009; 66 (1): 54-9.
5. Yeh EA, Weinstock-Guttman B, Ramanathan M et al. Magnetic resonance imaging characteristics of children and adults with paediatric-onset multiple sclerosis. Brain. 2009; 132(12): 3392-400.
6. Ruggieri M, Ianetti P, Polizzi A et al. Multiple sclerosis in children under 10 years of age. Neurol Sci. 2004; 25(Suppl 4): S326-35.
7. Wallach AI, Waltz M, Casper TC et al. Cognitive processing speed in pediatric-onset multiple sclerosis: Baseline characteristics of impairment and prediction of decline. Mult Scler. 2020; 26(14): 1938-47.
8. Brola W, Steinborn B, Niewiada M et al. Pediatric-onset multiple sclerosis in Poland: A registry-based retrospective cohort study. Mult Scler Relat Disord. 2022; 57: 103344.
9. Chitnis T, Tenembaum S, Banwell B et al. Consensus statement: evaluation of new and existing therapeutics for pediatric multiple sclerosi. Mult Scler J. 2012; 18(1):116-27.
10. Chitnis T, Tardieu M, Amato MP et al. International pediatric MS study group clinical trials summit: meeting report. Neurology. 2013; 80(12): 1161-8.
11. Colligan E, Metzler A, Tiryaki E et al. Shared decision-making in multiple sclerosis. Mult Scler. 2017; 23(2): 185-90.
12. Leczenie chorych na stwardnienie rozsiane (ICD-10: G35) – Załącznik B.29.
13. Wong YYM, de Mol CL, van der Vuurst de Vries RM et al. Real-world validation of the 2017 McDonald criteria for diagnosis pediatric MS. Neurol Neuroimmunol Neuroinflamm. 2019; 6(2): e528.
14. Banwell B. Pediatric multiple sclerosis: The 2022 ECTRIMS lecture Mult Scler J. 2023; 29(7): 772-8.
15. Chitnis T, Banwell B, Kappos L et al. Safety and efficacyof teriflunomide in paediatric multiple sclerosis (TERIKIDS): a multicentre, double-blind, phase 3, randomised, placebo-controlled trial. Lancet Neurol. 2021; 20(12): 1001-11.
16. Alroughani R, Das R, Penner R et al. Safety and efficacy of delayed-release dimethyl fumarate in pediatric patients with relapsing multiple-sclerosis (FOCUS). Pediatr Neurol. 2018; 83: 19-24.
17. Alroughani R, Huppke P, Mazurkiewicz-Beldzinska M et al. Delayed-release dimethyl fumarate safety and efficacy in pediatric patients with relapsin-remitting multiple sclerosis. Front Neurol. 2020; 11: 606418.
18. Chitnis T, Arnold DL, Banwell B et al. Trial of fingolimod versus interferon beta-1a in pediatric multiple sclerosis. N Engl J Med. 2018; 379(11): 1017-27.
19. Ghezzi A, Maiola L, Pozzilli C et al. Natalizumab in the pediatric MS population: results of the Italian registry. BMC Neurol. 2015; 15: 174.
20. Otero-Romero S, Lebrun-Frenay C, Reyes S et al. ECTRIMS/EAN consensus on vaccination in people with multiple sclerosis: Improving immunization strategies in the era of highly active immunotherapeutic drugs Mult Scler J. 2023; 29(8): 904-25.
21. McGinley M, Rossman I. Bringing the HEET: The Argument for High-Efficacy Early Treatment for Pediatric-Onset Multiple Sclerosis Neurotherap. 2017; 14: 985-98.
22. Sandesjo F, Wassmer E, Deiva K et al. Current international trends in the treatment of multiple sclerosis in children – Impact od the COVID-19 pandemic. Mult Scler Relat Disord. 2021; 56: 103277.
23. Duffy L, Sarill K, Forbes P et al. Shared Decision Making and Disease Modyfying Therapy in Families of Children and Adolescents with Pediatric Onset Multiple Sclerosis. J Ped Nurs. 2021; 61: P404-9.