Real-World Effectiveness of Initial Disease-Modifying Therapies in Pediatric Multiple Sclerosis.

Item request has been placed! ×
Item request cannot be made. ×
loading   Processing Request
  • Additional Information
    • Affiliation:
      UCSF Weill Institute for Neurosciences, Department of Neurology, University of California, San Francisco, San Francisco CA
      Department of Neurology, University of California, San Diego, La Jolla CA
      Department of Neurology, Cleveland Clinic, Cleveland OH
      Department of Neurology, State University of New York at Buffalo, Buffalo NY
      Department of Pediatrics, Loma Linda University, San Bernardino CA
      Department of Neurology, New York University Langone Medical Center, New York NY
      Department of Neurology, Boston Children's Hospital, Boston MA
      Department of Pediatric Neurology, Massachusetts General Hospital, Boston MA
      Mallinckrodt Institute of Radiology, Washington University in Saint Louis, St Louis MO
      Department of Nursing, University of Alabama at Birmingham, Birmingham AL
      Department of Neurology, Texas Children's Hospital, Houston TX
      Department of Neurology, Washington University in Saint Louis, St Louis MO
      Department of Pediatrics and Neurology, Dell Children's Hospital, University of Texas, Austin TX
      Department of Pediatrics, University of Alabama at Birmingham, Birmingham AL
      Department of Neurology, Mayo Clinic, Rochester MN
      Department of Neurology, University of Utah, Salt Lake City UT
      Departments of Neurology and Pediatrics, University of Colorado, Aurora CO
    • Corporate Authors:
    • Abstract:
      Objective: To assess real-world effectiveness of initial treatment with newer compared to injectable disease-modifying therapies (DMTs) on disease activity in pediatric multiple sclerosis (MS) and clinically isolated syndrome (CIS).Methods: This is a cohort study of children with MS/CIS followed at 12 clinics in the US Network of Pediatric MS Centers, who received initial therapy with newer (fingolimod, dimethyl fumarate, teriflunomide, natalizumab, rituximab, ocrelizumab) or injectable (interferon-β, glatiramer acetate) DMTs. Propensity scores (PSs) were computed, including preidentified confounders. Relapse rate while on initial DMT was modeled with negative binomial regression, adjusted for PS-quintile. Time to new/enlarging T2-hyperintense and gadolinium-enhancing lesions on brain magnetic resonance imaging were modeled with midpoint survival analyses, adjusted for PS-quintile.Results: A total of 741 children began therapy before 18 years, 197 with newer and 544 with injectable DMTs. Those started on newer DMTs were older (15.2 vs injectable 14.4 years, p = 0.001) and less likely to have a monofocal presentation. In PS-quintile-adjusted analysis, those on newer DMTs had a lower relapse rate than those on injectables (rate ratio = 0.45, 95% confidence interval (CI) = 0.29-0.70, p < 0.001; rate difference = 0.27, 95% CI = 0.14-0.40, p = 0.004). One would need to treat with newer rather than injectable DMTs for 3.7 person-years to prevent 1 relapse. Those started on newer DMTs had a lower rate of new/enlarging T2 (hazard ratio [HR] = 0.51, 95% CI = 0.36-0.72, p < 0.001) and gadolinium-enhancing lesions (HR = 0.38, 95% CI = 0.23-0.63, p < 0.001) than those on injectables.Interpretation: Initial treatment of pediatric MS/CIS with newer DMTs led to better disease activity control compared to injectables, supporting greater effectiveness of newer therapies. Long-term safety data for newer DMTs are required. ANN NEUROL 2020 ANN NEUROL 2020;88:42-55.
    • Journal Subset:
      Biomedical; Peer Reviewed; USA
    • ISSN:
      0364-5134
    • MEDLINE Info:
      PMID: NLM32267005 NLM UID: 7707449
    • Grant Information:
      FP-1605-08753//National Multiple Sclerosis Society/; HC-1509-06233//National Multiple Sclerosis Society/
    • Publication Date:
      In Process
    • Publication Date:
      20200630
    • DOI:
      http://dx.doi.org/10.1002/ana.25737
    • Accession Number:
      144209097
  • Citations
    • ABNT:
      KRYSKO, K. M. et al. Real-World Effectiveness of Initial Disease-Modifying Therapies in Pediatric Multiple Sclerosis. Annals of Neurology, [s. l.], v. 88, n. 1, p. 42–55, 2020. DOI 10.1002/ana.25737. Disponível em: http://search.ebscohost.com/login.aspx?direct=true&site=eds-live&db=rzh&AN=144209097. Acesso em: 10 ago. 2020.
    • AMA:
      Krysko KM, Graves JS, Rensel M, et al. Real-World Effectiveness of Initial Disease-Modifying Therapies in Pediatric Multiple Sclerosis. Annals of Neurology. 2020;88(1):42-55. doi:10.1002/ana.25737
    • APA:
      Krysko, K. M., Graves, J. S., Rensel, M., Weinstock, G. B., Rutatangwa, A., Aaen, G., Belman, A., Benson, L., Chitnis, T., Gorman, M., Goyal, M. S., Harris, Y., Krupp, L., Lotze, T., Mar, S., Moodley, M., Ness, J., Rodriguez, M., Rose, J., & Schreiner, T. (2020). Real-World Effectiveness of Initial Disease-Modifying Therapies in Pediatric Multiple Sclerosis. Annals of Neurology, 88(1), 42–55. https://doi.org/10.1002/ana.25737
    • Chicago/Turabian: Author-Date:
      Krysko, Kristen M., Jennifer S. Graves, Mary Rensel, Guttman, Bianca Weinstock, Alice Rutatangwa, Gregory Aaen, Anita Belman, et al. 2020. “Real-World Effectiveness of Initial Disease-Modifying Therapies in Pediatric Multiple Sclerosis.” Annals of Neurology 88 (1): 42–55. doi:10.1002/ana.25737.
    • Harvard:
      Krysko, K. M. et al. (2020) ‘Real-World Effectiveness of Initial Disease-Modifying Therapies in Pediatric Multiple Sclerosis’, Annals of Neurology, 88(1), pp. 42–55. doi: 10.1002/ana.25737.
    • Harvard: Australian:
      Krysko, KM, Graves, JS, Rensel, M, Weinstock, GB, Rutatangwa, A, Aaen, G, Belman, A, Benson, L, Chitnis, T, Gorman, M, Goyal, MS, Harris, Y, Krupp, L, Lotze, T, Mar, S, Moodley, M, Ness, J, Rodriguez, M, Rose, J & Schreiner, T 2020, ‘Real-World Effectiveness of Initial Disease-Modifying Therapies in Pediatric Multiple Sclerosis’, Annals of Neurology, vol. 88, no. 1, pp. 42–55, viewed 10 August 2020, .
    • MLA:
      Krysko, Kristen M., et al. “Real-World Effectiveness of Initial Disease-Modifying Therapies in Pediatric Multiple Sclerosis.” Annals of Neurology, vol. 88, no. 1, July 2020, pp. 42–55. EBSCOhost, doi:10.1002/ana.25737.
    • Chicago/Turabian: Humanities:
      Krysko, Kristen M., Jennifer S. Graves, Mary Rensel, Guttman, Bianca Weinstock, Alice Rutatangwa, Gregory Aaen, Anita Belman, et al. “Real-World Effectiveness of Initial Disease-Modifying Therapies in Pediatric Multiple Sclerosis.” Annals of Neurology 88, no. 1 (July 2020): 42–55. doi:10.1002/ana.25737.
    • Vancouver/ICMJE:
      Krysko KM, Graves JS, Rensel M, Weinstock GB, Rutatangwa A, Aaen G, et al. Real-World Effectiveness of Initial Disease-Modifying Therapies in Pediatric Multiple Sclerosis. Annals of Neurology [Internet]. 2020 Jul [cited 2020 Aug 10];88(1):42–55. Available from: http://search.ebscohost.com/login.aspx?direct=true&site=eds-live&db=rzh&AN=144209097