Ergenlikten Erişkinliğe Geçişte Multipl Skleroz
Özet
Pediatrik Multipl Skleroz (MS) ve diğer edinilmiş demiyelinizan hastalıklar, çocukluk ve ergenlik döneminde nadir görülen ancak karmaşık bir klinik tablo sunan otoimmün süreçlerdir. Tanıda McDonald kriterleri ve radyolojik görüntüleme büyük önem taşırken, hastalığın seyri ve prognozu genetik, çevresel faktörler ve biyobelirteçlerle yakından ilişkilidir. Özellikle erken dönemde teşhis ve kişiselleştirilmiş immünmodülatör tedaviler, hastalığın aktivitesini kontrol altına almak ve kalıcı sakatlıkları minimize etmek için kritik rol oynamaktadır. Ergenlik dönemi, bu hastalıkların en sık ortaya çıktığı süreç olduğundan, bu dönemdeki hastaların klinik takibi ve tedavi stratejilerinin belirlenmesi uzun dönem başarısı için hayati önemdedir.
Pediatric Multiple Sclerosis (MS) and other acquired demyelinating diseases are autoimmune processes that present a rare but complex clinical picture during childhood and adolescence. While McDonald criteria and radiological imaging are essential for diagnosis, the course and prognosis of the disease are closely related to genetic factors, environmental influences, and biomarkers. Early diagnosis and personalized immunomodulatory treatments play a critical role in controlling disease activity and minimizing permanent disability. Since adolescence is the peak period for the onset of these conditions, careful clinical monitoring and the determination of appropriate treatment strategies for patients in this age group are vital for long-term outcomes.
Referanslar
Wells E, Hacohen Y, Waldman A, Tillema JM, Soldatos A, Ances B, et al. Neuroimmune disorders of the central nervous system in children in the molecular era. Nature reviews Neurology. 2018;14(7):433-45.
Duignan S, Brownlee W, Wassmer E, Hemingway C, Lim M, Ciccarelli O, et al. Paediatric multiple sclerosis: a new era in diagnosis and treatment. Dev Med Child Neurol. 2019;61(9):1039-49.
Fadda G, Armangue T, Hacohen Y, Chitnis T, Banwell B. Paediatric multiple sclerosis and antibody-associated demyelination: clinical, imaging, and biological considerations for diagnosis and care. The Lancet Neurology. 2021;20(2):136-49.
Waldman A, Ghezzi A, Bar-Or A, Mikaeloff Y, Tardieu M, Banwell B. Multiple sclerosis in children: an update on clinical diagnosis, therapeutic strategies, and research. The Lancet Neurology. 2014;13(9):936-48.
Ochi H. Clinical features, diagnosis and therapeutic strategies in pediatric multiple sclerosis. Clinical and Experimental Neuroimmunology. 2017;8:33-9.
Banwell B, Bar-Or A, Arnold DL, Sadovnick D, Narayanan S, McGowan M, et al. Clinical, environmental, and genetic determinants of multiple sclerosis in children with acute demyelination: a prospective national cohort study. The Lancet Neurology. 2011;10(5):436-45.
Boiko A, Vorobeychik G, Paty D, Devonshire V, Sadovnick D. Early onset multiple sclerosis: a longitudinal study. Neurology. 2002;59(7):1006-10.
Otallah S, Banwell B. Pediatric Multiple Sclerosis: an Update. Curr Neurol Neurosci Rep. 2018;18(11):76.
Yılmaz Ü, Anlar B, Gücüyener K. Characteristics of pediatric multiple sclerosis: The Turkish pediatric multiple sclerosis database. Eur J Paediatr Neurol. 2017;21(6):864-72.
Garg N, Smith TW. An update on immunopathogenesis, diagnosis, and treatment of multiple sclerosis. Brain Behav. 2015;5(9):e00362.
Lazibat I, Rubinić Majdak M, Županić S. Multiple Sclerosis: New Aspects of Immunopathogenesis. Acta Clin Croat. 2018;57(2):352-61.
Gianfrancesco MA, Stridh P, Shao X, Rhead B, Graves JS, Chitnis T, et al. Genetic risk factors for pediatric-onset multiple sclerosis. Mult Scler. 2018;24(14):1825-34.
Disanto G, Magalhaes S, Handel AE, Morrison KM, Sadovnick AD, Ebers GC, et al. HLA-DRB1 confers increased risk of pediatric-onset MS in children with acquired demyelination. Neurology. 2011;76(9):781-6.
Mowry EM, Krupp LB, Milazzo M, Chabas D, Strober JB, Belman AL, et al. Vitamin D status is associated with relapse rate in pediatric-onset multiple sclerosis. Annals of neurology. 2010;67(5):618-24.
Tremlett H, Waubant E. Gut microbiome and pediatric multiple sclerosis. Mult Scler. 2018;24(1):64-8.
Tremlett H, Fadrosh DW, Faruqi AA, Hart J, Roalstad S, Graves J, et al. Gut microbiota composition and relapse risk in pediatric MS: A pilot study. Journal of the neurological sciences. 2016;363:153-7.
Cryan JF, O'Riordan KJ, Sandhu K, Peterson V, Dinan TG. The gut microbiome in neurological disorders. The Lancet Neurology. 2020;19(2):179-94.
Thompson AJ, Banwell BL, Barkhof F, Carroll WM, Coetzee T, Comi G, et al. Diagnosis of multiple sclerosis: 2017 revisions of the McDonald criteria. The Lancet Neurology. 2018;17(2):162-73.
Brownlee WJ, Hardy TA, Fazekas F, Miller DH. Diagnosis of multiple sclerosis: progress and challenges. Lancet (London, England). 2017;389(10076):1336-46.
Iaffaldano P, Simone M, Lucisano G, Ghezzi A, Coniglio G, Brescia Morra V, et al. Prognostic indicators in pediatric clinically isolated syndrome. Annals of neurology. 2017;81(5):729-39.
Padilha IG, Fonseca AP, Pettengill AL, Fragoso DC, Pacheco FT, Nunes RH, et al. Pediatric multiple sclerosis: from clinical basis to imaging spectrum and differential diagnosis. Pediatric radiology. 2020;50(6):776-92.
Hacohen Y, Absoud M, Woodhall M, Cummins C, De Goede CG, Hemingway C, et al. Autoantibody biomarkers in childhood-acquired demyelinating syndromes: results from a national surveillance cohort. J Neurol Neurosurg Psychiatry. 2014;85(4):456-61.
Huppke B, Ellenberger D, Rosewich H, Friede T, Gärtner J, Huppke P. Clinical presentation of pediatric multiple sclerosis before puberty. Eur J Neurol. 2014;21(3):441-6.
Chitnis T. Role of puberty in multiple sclerosis risk and course. Clin Immunol. 2013;149(2):192-200.
Gorman MP, Healy BC, Polgar-Turcsanyi M, Chitnis T. Increased relapse rate in pediatric-onset compared with adult-onset multiple sclerosis. Archives of neurology. 2009;66(1):54-9.
Okuda DT, Siva A, Kantarci O, Inglese M, Katz I, Tutuncu M, et al. Radiologically isolated syndrome: 5-year risk for an initial clinical event. PloS one. 2014;9(3):e90509.
Moore F, Okuda DT. Incidental MRI anomalies suggestive of multiple sclerosis: the radiologically isolated syndrome. Neurology. 2009;73(20):1714.
Simonsen CS, Flemmen H, Lauritzen T, Berg-Hansen P, Moen SM, Celius EG. The diagnostic value of IgG index versus oligoclonal bands in cerebrospinal fluid of patients with multiple sclerosis. Mult Scler J Exp Transl Clin. 2020;6(1):2055217319901291.
Reinert MC, Benkert P, Wuerfel J, Michalak Z, Ruberte E, Barro C, et al. Serum neurofilament light chain is a useful biomarker in pediatric multiple sclerosis. Neurol Neuroimmunol Neuroinflamm. 2020;7(4).
Duignan S, Wright S, Rossor T, Cazabon J, Gilmour K, Ciccarelli O, et al. Myelin oligodendrocyte glycoprotein and aquaporin-4 antibodies are highly specific in children with acquired demyelinating syndromes. Dev Med Child Neurol. 2018;60(9):958-62.
Baumann M, Hennes EM, Schanda K, Karenfort M, Kornek B, Seidl R, et al. Children with multiphasic disseminated encephalomyelitis and antibodies to the myelin oligodendrocyte glycoprotein (MOG): Extending the spectrum of MOG antibody positive diseases. Mult Scler. 2016;22(14):1821-9.
Huppke P, Rostasy K, Karenfort M, Huppke B, Seidl R, Leiz S, et al. Acute disseminated encephalomyelitis followed by recurrent or monophasic optic neuritis in pediatric patients. Mult Scler. 2013;19(7):941-6.
Hacohen Y, Wong YY, Lechner C, Jurynczyk M, Wright S, Konuskan B, et al. Disease Course and Treatment Responses in Children With Relapsing Myelin Oligodendrocyte Glycoprotein Antibody-Associated Disease. JAMA Neurol. 2018;75(4):478-87.
Le Page E, Veillard D, Laplaud DA, Hamonic S, Wardi R, Lebrun C, et al. Oral versus intravenous high-dose methylprednisolone for treatment of relapses in patients with multiple sclerosis (COPOUSEP): a randomised, controlled, double-blind, non-inferiority trial. Lancet (London, England). 2015;386(9997):974-81.
Renoux C, Vukusic S, Mikaeloff Y, Edan G, Clanet M, Dubois B, et al. Natural history of multiple sclerosis with childhood onset. The New England journal of medicine. 2007;356(25):2603-13.
Macaron G, Feng J, Moodley M, Rensel M. Newer Treatment Approaches in Pediatric-Onset Multiple Sclerosis. Curr Treat Options Neurol. 2019;21(10):50.
Ziemssen T, Derfuss T, de Stefano N, Giovannoni G, Palavra F, Tomic D, et al. Optimizing treatment success in multiple sclerosis. Journal of neurology. 2016;263(6):1053-65.
Rieckmann P. Concepts of induction and escalation therapy in multiple sclerosis. Journal of the neurological sciences. 2009;277 Suppl 1:S42-5.
Spelman T, Magyari M, Piehl F, Svenningsson A, Rasmussen PV, Kant M, et al. Treatment Escalation vs Immediate Initiation of Highly Effective Treatment for Patients With Relapsing-Remitting Multiple Sclerosis: Data From 2 Different National Strategies. JAMA Neurol. 2021;78(10):1197-204.
Chitnis T, Tenembaum S, Banwell B, Krupp L, Pohl D, Rostasy K, et al. Consensus statement: evaluation of new and existing therapeutics for pediatric multiple sclerosis. Mult Scler. 2012;18(1):116-27.
Chitnis T, Arnold DL, Banwell B, Brück W, Ghezzi A, Giovannoni G, et al. Trial of Fingolimod versus Interferon Beta-1a in Pediatric Multiple Sclerosis. The New England journal of medicine. 2018;379(11):1017-27.
Alroughani R, Das R, Penner N, Pultz J, Taylor C, Eraly S. Safety and Efficacy of Delayed-Release Dimethyl Fumarate in Pediatric Patients With Relapsing Multiple Sclerosis (FOCUS). Pediatr Neurol. 2018;83:19-24.
Alroughani R, Huppke P, Mazurkiewicz-Beldzinska M, Blaschek A, Valis M, Aaen G, et al. Delayed-Release Dimethyl Fumarate Safety and Efficacy in Pediatric Patients With Relapsing-Remitting Multiple Sclerosis. Front Neurol. 2020;11:606418.
Chitnis T, Tardieu M, Banwell B, Gücüyener K, Deiva K, Skripchenko N, et al. Evaluation of teriflunomide in children and adolescents with relapsing MS: TERIKIDS phase 3 study design, enrollment update, and baseline data (P4. 354). AAN Enterprises; 2018.
Ghezzi A, Moiola L, Pozzilli C, Brescia-Morra V, Gallo P, Grimaldi LM, et al. Natalizumab in the pediatric MS population: results of the Italian registry. BMC Neurol. 2015;15:174.
