Disease-Modifying Therapy of Pediatric Multiple Sclerosis

Neurotherapeutics

Volumn 10, Issue 1, 2013, Pages 89-96

  Chitnis, Tanuja ^a,b  

^a HARVARD MEDICAL SCHOOL   (United States)

^b MASSACHUSETTS GENERAL HOSPITAL   (United States)

Author keywords

adolescent; children; disease modifying therapy; multiple sclerosis; Pediatric

Indexed keywords

BETA1A INTERFERON; CYCLOPHOSPHAMIDE; DACLIZUMAB; FINGOLIMOD; GLATIRAMER; INTERFERON BETA SERINE; NATALIZUMAB; RITUXIMAB; TERIFLUNOMIDE;

ALOPECIA; AMENORRHEA; AMINOTRANSFERASE BLOOD LEVEL; ANEMIA; DIFFUSION TENSOR IMAGING; DRUG EFFICACY; DRUG HYPERSENSITIVITY; DRUG SAFETY; FLU LIKE SYNDROME; HUMAN; INFECTION; INJECTION SITE ABSCESS; INJECTION SITE NECROSIS; LEUKOPENIA; LIVER FUNCTION TEST; MOUTH ULCER; MULTIPLE SCLEROSIS; NEUROIMAGING; NUCLEAR MAGNETIC RESONANCE IMAGING; OSTEOPOROSIS; PRIORITY JOURNAL; PSORIASIS; REVIEW; SIDE EFFECT; THROMBOCYTOPENIA; TREATMENT RESPONSE; VOMITING;

EID: 84873124102     PISSN: 19337213     EISSN: 18787479     Source Type: Journal    
DOI: 10.1007/s13311-012-0158-1     Document Type: Review

References (91)

1. Chitnis T. Pediatric multiple sclerosis. Neurologist 2006; 12: 299-310.
2. Duquette P, Murray TJ, Pleines J, et al. Multiple sclerosis in childhood: clinical profile in 125 patients. J Pediatr 1987; 111: 359-363.
3. Boiko A, Vorobeychik G, Paty D, Devonshire V, Sadovnick D. Early onset multiple sclerosis: a longitudinal study. Neurology 2002; 59: 1006-1010.
4. Ghezzi A, Deplano V, Faroni J, et al. Multiple sclerosis in childhood: clinical features of 149 cases. Mult Scler 1997; 3: 43-46.
5. Sindern E, Haas J, Stark E, Wurster U. Early onset MS under the age of 16: clinical and paraclinical features. Acta Neurol Scand 1992; 86: 280-284.
6. Chitnis T, Glanz B, Jaffin S, Healy B. Demographics of pediatric-onset multiple sclerosis in an MS center population from the Northeastern United States. Mult Scler 2009; 15: 627-631.
7. Simone IL, Carrara D, Tortorella C, et al. Course and prognosis in early-onset MS: comparison with adult-onset forms. Neurology 2002; 59: 1922-1928.
8. Langer-Gould A, Zhang JL, Chung J, Yeung Y, Waubant E, Yao J. Incidence of acquired CNS demyelinating syndromes in a multiethnic cohort of children. Neurology 2011; 77: 1143-1148.
9. Banwell B, Kennedy J, Sadovnick D, et al. Incidence of acquired demyelination of the CNS in Canadian children. Neurology 2009; 72: 232-239.
10. Pohl D, Hennemuth I, von Kries R, Hanefeld F. Paediatric multiple sclerosis and acute disseminated encephalomyelitis in Germany: results of a nationwide survey. Eur J Pediatr 2007; 166: 405-412.
11. Renoux C, Vukusic S, Mikaeloff Y, et al. Natural history of multiple sclerosis with childhood onset. N Engl J Med 2007; 356: 2603-2613.
12. Tenembaum S, Chamoles N, Fejerman N. Acute disseminated encephalomyelitis: a long-term follow-up study of 84 pediatric patients. Neurology 2002; 59: 1224-1231.
13. Banwell B, Ghezzi A, Bar-Or A, Mikaeloff Y, Tardieu M. Multiple sclerosis in children: clinical diagnosis, therapeutic strategies, and future directions. Lancet Neurol 2007; 6: 887-902.
14. Chabas D, Castillo-Trivino T, Mowry EM, Strober JB, Glenn OA, Waubant E. Vanishing MS T2-bright lesions before puberty: a distinct MRI phenotype? Neurology 2008; 71: 1090-1093.
15. Atzori M, Battistella PA, Perini P, et al. Clinical and diagnostic aspects of multiple sclerosis and acute monophasic encephalomyelitis in pediatric patients: a single centre prospective study. Mult Scler 2009; 15: 363-370.
16. Gorman MP, Healy BC, Polgar-Turcsanyi M, Chitnis T. Increased relapse rate in pediatric-onset compared with adult-onset multiple sclerosis. Arch Neurol 2009; 66: 54-59.
17. Banwell B, Bar-Or A, Arnold DL, et al. Clinical, environmental, and genetic determinants of multiple sclerosis in children with acute demyelination: a prospective national cohort study. Lancet Neurol 2011; 10: 436-445.
18. Mikaeloff Y, Suissa S, Vallee L, et al. First episode of acute CNS inflammatory demyelination in childhood: prognostic factors for multiple sclerosis and disability. J Pediatr 2004; 144: 246-252.
19. Ghezzi A, Pozzilli C, Liguori M, et al. Prospective study of multiple sclerosis with early onset. Mult Scler 2002; 8: 115-118.
20. Ruggieri M, Iannetti P, Polizzi A, Pavone L, Grimaldi LM. Multiple sclerosis in children under 10 years of age. Neurol Sci 2004; 25(suppl 4): S326-S335.
21. Fay AJ, Mowry EM, Strober J, Waubant E. Relapse severity and recovery in early pediatric multiple sclerosis. Mult Scler 2012; 18: 1008-1012.
22. Banwell BL, Anderson PE. The cognitive burden of multiple sclerosis in children. Neurology 2005; 64: 891-894.
23. MacAllister WS, Belman AL, Milazzo M, et al. Cognitive functioning in children and adolescents with multiple sclerosis. Neurology 2005; 64: 1422-1425.
24. MacAllister WS, Christodoulou C, Milazzo M, Krupp LB. Longitudinal neuropsychological assessment in pediatric multiple sclerosis. Dev Neuropsychol 2007; 32: 625-644.
25. Amato MP, Goretti B, Ghezzi A, et al. Cognitive and psychosocial features of childhood and juvenile MS. Neurology 2008; 70: 1891-1897.
26. Amato MP, Goretti B, Ghezzi A, et al. Cognitive and psychosocial features in childhood and juvenile MS: two-year follow-up. Neurology 2010; 75: 1134-1140.
27. Portaccio E, Goretti B, Zipoli V, et al. Cognitive rehabilitation in children and adolescents with multiple sclerosis. Neurol Sci 2011; 32(6): 1043-1046.
28. Waubant E, Chabas D, Okuda DT, et al. Difference in disease burden and activity in pediatric patients on brain magnetic resonance imaging at time of multiple sclerosis onset vs adults. Arch Neurol 2009; 66: 967-971.
29. 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(pt 12): 3392-3400.
30. Absinta M, Rocca MA, Moiola L, et al. Cortical lesions in children with multiple sclerosis. Neurology 2011; 76: 910-913.
31. Vishwas MS, Chitnis T, Pienaar R, Healy BC, Grant PE. Tract-based analysis of callosal, projection, and association pathways in pediatric patients with multiple sclerosis: a preliminary study. AJNR Am J Neuroradiol 2010; 31: 121-128.
32. Vishwas MS, Healy BC, Pienaar R, Gorman MP, Grant PE, Chitnis T. Diffusion tensor analysis of pediatric multiple sclerosis and clinically isolated syndromes. AJNR Am J Neuroradiol 2012. doi: 10. 3174/ajnr. A3216.
33. Hahn CD, Shroff MM, Blaser SI, Banwell BL. MRI criteria for multiple sclerosis: Evaluation in a pediatric cohort. Neurology 2004; 62: 806-808.
34. Sadaka Y, Verhey LH, Shroff MM, et al. 2010 McDonald criteria for diagnosing pediatric multiple sclerosis. Ann Neurol 2012; 72: 211-223.
35. Chitnis T, Tenembaum S, Banwell B, et al. Consensus statement: evaluation of new and existing therapeutics for pediatric multiple sclerosis. Mult Scler 2012; 18: 116-127.
36. Waldman AT, Gorman MP, Rensel MR, Austin TE, Hertz DP, Kuntz NL. Management of pediatric central nervous system demyelinating disorders: consensus of United States neurologists. Journal of child neurology. 2011; 26: 675-682.
37. Dhib-Jalbut S, Marks S. Interferon-beta mechanisms of action in multiple sclerosis. Neurology 2010; 74(suppl 1): S17-S24.
38. Group IMSS. Interferon beta-1b is effective in relapsing-remitting multiple sclerosis. I Clinical results of a multicenter, randomized, double blind, placebo-controlled trial. Neurology 1993: 655-661.
39. Jacobs L, Cookfair D, Rudick R, et al. Intramuscular interferon beta-1a for disease progression in relapsing multiple sclerosis. Ann Neurol 1996; 39: 285-294.
40. Group PRISMS. Randomised, double-blind, placebo-controlled study of interferon beta-1a in relapsing/remitting multiple sclerosis. Lancet 1998; 352: 1498-1504.
41. Mikaeloff Y, Moreau T, Debouverie M, et al. Interferon-beta treatment in patients with childhood-onset multiple sclerosis. J Pediatr 2001; 139: 443-446.
42. Banwell B, Reder AT, Krupp L, et al. Safety and tolerability of interferon beta-1b in pediatric multiple sclerosis. Neurology 2006; 66: 472-476.
43. Bykova OV, Kuzenkova LM, Maslova OI. [The use of beta-interferon-1b in children and adolescents with multiple sclerosis]. Zh Nevrol Psikhiatr Im S S Korsakova 2006; 106(9): 29-33.
44. Pohl D, Rostasy K, Gartner J, Hanefeld F. Treatment of early onset multiple sclerosis with subcutaneous interferon beta-1a. Neurology 2005; 64: 888-890.
45. Bergamaschi R, Rezzani C, Minguzzi S, et al. Validation of the DYMUS questionnaire for the assessment of dysphagia in multiple sclerosis. Funct Neurol 2009; 24: 159-162.
46. Ghezzi A, Amato MP, Capobianco M, et al. Disease-modifying drugs in childhood-juvenile multiple sclerosis: results of an Italian co-operative study. Mult Scler 2005; 11: 420-424.
47. Ghezzi A, Amato MP, Capobianco M, et al. Treatment of early-onset multiple sclerosis with intramuscular interferonbeta-1a: long-term results. Neurol Sci 2007; 28: 127-132.
48. Tenembaum SN, Segura MJ. Interferon beta-1a treatment in childhood and juvenile-onset multiple sclerosis. Neurology 2006; 67: 511-513.
49. Waubant E, Hietpas J, Stewart T, et al. Interferon beta-1a in children with multiple sclerosis is well tolerated. Neuropediatrics 2001; 32: 211-213.
50. Mikaeloff Y, Caridade G, Tardieu M, Suissa S. Effectiveness of early beta interferon on the first attack after confirmed multiple sclerosis: a comparative cohort study. Eur J Paediatr Neurol 2008; 12: 205-209.
51. Yeh E, Heim R, Karpinski M, Ray J, Weinstock-Guttman B. Neutralizing antibodies to interferon in children with MS on interferon treatment. Presented at the Annual Meeting of the Child Neurology Society; October 14-17, 2009; Louisville, KY.
52. Dhib-Jalbut S. Sustained immunological effects of Glatiramer acetate in patients with multiple sclerosis treated for over 6 years. J Neurol Sci 2002; 201: 71-77.
53. Johnson K, Brooks B, Cohen J, et al. Copolymer 1 reduces relapse rate and improves disability in relapsing-remitting multiple sclerosis: results of a phase III multicenter, double-blind, placebo-controlled trial. Neurology 1995; 45: 1268-1276.
54. Kornek B, Bernert G, Balassy C, Geldner J, Prayer D, Feucht M. Glatiramer acetate treatment in patients with childhood and juvenile onset multiple sclerosis. Neuropediatrics 2003; 34: 120-126.
55. Cohen BA, Khan O, Jeffery DR, et al. Identifying and treating patients with suboptimal responses. Neurology 2004; 63(12 suppl 6): S33-S40.
56. Freedman MS, Cohen B, Dhib-Jalbut S, et al. Recognizing and treating suboptimally controlled multiple sclerosis: steps toward regaining command. Curr Med Res Opin 2009;(10): 2459-2470.
57. Rio J, Nos C, Tintore M, et al. Assessment of different treatment failure criteria in a cohort of relapsing-remitting multiple sclerosis patients treated with interferon beta: implications for clinical trials. Ann Neurol 2002; 52: 400-406.
58. Ghezzi A, Pozzilli C, Grimaldi LM, et al. Safety and efficacy of natalizumab in children with multiple sclerosis. Neurology 2010; 75(10): 912-917.
59. Yeh EA, Waubant E, Krupp LB, et al. Multiple Sclerosis therapies in pediatric patients with refractory multiple sclerosis. Arch Neurol 2010; 68(4): 437-444.
60. Polman CH, O'Connor PW, Havrdova E, et al. A randomized, placebo-controlled trial of natalizumab for relapsing multiple sclerosis. N Engl J Med 2006; 354: 899-910.
61. Huppke P, Stark W, Zurcher C, Huppke B, Bruck W, Gartner J. Natalizumab use in pediatric multiple sclerosis. Arch Neurol 2008; 65: 1655-1658.
62. Borriello G, Prosperini L, Luchetti A, Pozzilli C. Natalizumab treatment in pediatric multiple sclerosis: a case report. Eur J Paediatr Neurol 2009; 13: 67-71.
63. Ghezzi A, Pozzilli C, Grimaldi LM, et al. Safety and efficacy of natalizumab in children with multiple sclerosis. Neurology 2010; 75: 912-917.
64. Hyams JS, Wilson DC, Thomas A, et al. Natalizumab therapy for moderate to severe Crohn disease in adolescents. J Pediatr Gastroenterol Nutr 2007; 44: 185-191.
65. Gorelik L, Lerner M, Bixler S, et al. Anti-JC virus antibodies: implications for PML risk stratification. Ann Neurol 2010; 68: 295-303.
66. Bloomgren G, Richman S, Hotermans C, et al. Risk of natalizumab-associated progressive multifocal leukoencephalopathy. N Engl J Med 2012; 366: 1870-1880.
67. Sorensen PS, Bertolotto A, Edan G, et al. Risk stratification for progressive multifocal leukoencephalopathy in patients treated with natalizumab. Mult Scler 2012; 18: 143-152.
68. Kean JM, Rao S, Wang M, Garcea RL. Seroepidemiology of human polyomaviruses. PLoS Pathog 2009; 5: e1000363.
69. Knowles WA, Pipkin P, Andrews N, et al. Population-based study of antibody to the human polyomaviruses BKV and JCV and the simian polyomavirus SV40. J Med Virol 2003; 71: 115-123.
70. Comi G, O'Connor P, Montalban X, Antel J, et al. Phase II study of oral fingolimod (FTY720) in multiple sclerosis: 3-year results. Mult Scler 2010; 16(2): 197-207.
71. Cohen JA, Barkhof F, Comi G, et al. Oral fingolimod or intramuscular interferon for relapsing multiple sclerosis. N Engl J Med 2010; 362: 402-415.
72. Kappos L, Radue EW, O'Connor P, et al. A placebo-controlled trial of oral fingolimod in relapsing multiple sclerosis. N Engl J Med 2006; 355(11): 1124-1140.
73. Mehling M, Lindberg R, Raulf F, et al. Th17 central memory T cells are reduced by FTY720 in patients with multiple sclerosis. Neurology 2010; 75: 403-410.
74. Hauser SL, Dawson DM, Lehrich JR, et al. Intensive immunosuppression in progressive multiple sclerosis. A randomized, three-arm study of high-dose intravenous cyclophosphamide, plasma exchange, and ACTH. N Engl J Med 1983; 308: 173-180.
75. Weiner HL, Mackin GA, Orav EJ, et al. Intermittent cyclophosphamide pulse therapy in progressive multiple sclerosis: final report of the Northeast Cooperative Multiple Sclerosis Treatment Group. Neurology 1993; 43: 910-918.
76. Makhani N, Gorman MP, Branson HM, Stazzone L, Banwell BL, Chitnis T. Cyclophosphamide therapy in pediatric multiple sclerosis. Neurology 2009; 72: 2076-2082.
77. Baca V, Lavalle C, Garcia R, et al. Favorable response to intravenous methylprednisolone and cyclophosphamide in children with severe neuropsychiatric lupus. J Rheumatol 1999; 26: 432-439.
78. Yildirim-Toruner C, Diamond B. Current and novel therapeutics in the treatment of systemic lupus erythematosus. J Allergy Clin Immunol 2011; 127: 303-314.
79. Adams A, MacDermott EJ, Lehman TJ. Pharmacotherapy of lupus nephritis in children: a recommended treatment approach. Drugs 2006; 66: 1191-1207.
80. Hauser SL, Waubant E, Arnold DL, et al. B-cell depletion with rituximab in relapsing-remitting multiple sclerosis. N Engl J Med 2008; 358: 676-688.
81. Tzaribachev N, Koetter I, Kuemmerle-Deschner JB, Schedel J. Rituximab for the treatment of refractory pediatric autoimmune diseases: a case series. Cases J 2009; 2: 6609.
82. Carson KR, Focosi D, Major EO, et al. Monoclonal antibody-associated progressive multifocal leucoencephalopathy in patients treated with rituximab, natalizumab, and efalizumab: a Review from the Research on Adverse Drug Events and Reports (RADAR) Project. Lancet Oncol 2009; 10: 816-824.
83. Bielekova B, Catalfamo M, Reichert-Scrivner S, et al. Regulatory CD56(bright) natural killer cells mediate immunomodulatory effects of IL-2Ralpha-targeted therapy (daclizumab) in multiple sclerosis. Proc Natl Acad Sci U S A 2006; 103: 5941-5946.
84. Bielekova B, Richert N, Howard T, et al. Humanized anti-CD25 (daclizumab) inhibits disease activity in multiple sclerosis patients failing to respond to interferon beta. Proc Natl Acad Sci U S A 2004; 101: 8705-8708.
85. Rose JW, Watt HE, White AT, Carlson NG. Treatment of multiple sclerosis with an anti-interleukin-2 receptor monoclonal antibody. Ann Neurol 2004; 56: 864-867.
86. Ali EN, Healy BC, Stazzone LA, Brown BA, Weiner HL, Khoury SJ. Daclizumab in treatment of multiple sclerosis patients. Mult Scler 2009; 15: 272-274.
87. Sheridan C. Abbott outbids Biogen for Facet's multiple sclerosis antibody. Nat Biotechnol 2010; 28: 387-389.
88. Fernandez-Fernandez O, Alvarez-Cermeno JC, Arbizu-Urdiain T, et al. [Review of the novelties presented at the 26th Congress of the European Committee for Treatment and Research in Multiple Sclerosis (ECTRIMS) (II)]. Rev Neurol 2011; 52: 289-299.
89. Gorman MP, Tillema JM, Ciliax AM, Guttmann CR, Chitnis T. Daclizumab use in patients with pediatric multiple sclerosis. Arch Neurol 2012; 69: 78-81.
90. Di Filippo S. Anti-IL-2 receptor antibody vs. polyclonal anti-lymphocyte antibody as induction therapy in pediatric transplantation. Pediatr Transplant 2005; 9: 373-380.
91. Gallagher M, Quinones K, Cervantes-Castaneda RA, Yilmaz T, Foster CS. Biological response modifier therapy for refractory childhood uveitis. Br J Ophthalmol 2007; 91: 1341-1344.