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Real-World Effectiveness of Initial Disease-Modifying Therapies in Pediatric Multiple Sclerosis.
Ann Neurol. 2020 Jul; 88(1):42-55.AN

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.

Authors+Show Affiliations

UCSF Weill Institute for Neurosciences, Department of Neurology, University of California, San Francisco, San Francisco, CA.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.UCSF Weill Institute for Neurosciences, Department of Neurology, University of California, San Francisco, San Francisco, CA.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.Department of Neurology, Boston Children's 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, New York University Langone Medical Center, New York, NY.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.Department of Neurology, Mayo Clinic, Rochester, MN.Department of Pediatrics, University of Utah, Salt Lake City, UT.Department of Pediatrics, University of Utah, Salt Lake City, UT.UCSF Weill Institute for Neurosciences, Department of Neurology, University of California, San Francisco, San Francisco, CA.No affiliation info available

Pub Type(s)

Journal Article

Language

eng

PubMed ID

32267005

Citation

Krysko, Kristen M., et al. "Real-World Effectiveness of Initial Disease-Modifying Therapies in Pediatric Multiple Sclerosis." Annals of Neurology, vol. 88, no. 1, 2020, pp. 42-55.
Krysko KM, Graves JS, Rensel M, et al. Real-World Effectiveness of Initial Disease-Modifying Therapies in Pediatric Multiple Sclerosis. Ann Neurol. 2020;88(1):42-55.
Krysko, K. M., Graves, J. S., Rensel, M., Weinstock-Guttman, 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., ... Waubant, E. (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
Krysko KM, et al. Real-World Effectiveness of Initial Disease-Modifying Therapies in Pediatric Multiple Sclerosis. Ann Neurol. 2020;88(1):42-55. PubMed PMID: 32267005.
* Article titles in AMA citation format should be in sentence-case
TY - JOUR T1 - Real-World Effectiveness of Initial Disease-Modifying Therapies in Pediatric Multiple Sclerosis. AU - Krysko,Kristen M, AU - Graves,Jennifer S, AU - Rensel,Mary, AU - Weinstock-Guttman,Bianca, AU - Rutatangwa,Alice, AU - Aaen,Gregory, AU - Belman,Anita, AU - Benson,Leslie, AU - Chitnis,Tanuja, AU - Gorman,Mark, AU - Goyal,Manu S, AU - Harris,Yolanda, AU - Krupp,Lauren, AU - Lotze,Timothy, AU - Mar,Soe, AU - Moodley,Manikum, AU - Ness,Jayne, AU - Rodriguez,Moses, AU - Rose,John, AU - Schreiner,Teri, AU - Tillema,Jan-Mendelt, AU - Waltz,Michael, AU - Casper,T Charles, AU - Waubant,Emmanuelle, AU - ,, Y1 - 2020/05/14/ PY - 2019/12/21/received PY - 2020/03/31/revised PY - 2020/04/02/accepted PY - 2020/4/9/pubmed PY - 2020/4/9/medline PY - 2020/4/9/entrez SP - 42 EP - 55 JF - Annals of neurology JO - Ann. Neurol. VL - 88 IS - 1 N2 - 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. SN - 1531-8249 UR - https://www.unboundmedicine.com/medline/citation/32267005/Real-World_Effectiveness_of_Initial_Disease-Modifying_Therapies_in_Pediatric_Multiple_Sclerosis L2 - https://doi.org/10.1002/ana.25737 DB - PRIME DP - Unbound Medicine ER -
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