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Comprehensive Autopsy Program for Individuals with Multiple Sclerosis
Published on: July 19, 2019
Use of newer disease-modifying therapies in pediatric multiple sclerosis in the US
Kristen M Krysko1, Jennifer Graves2, Mary Rensel2
1From the Department of Neurology (K.M.K., J.G., E.W.), University of California San Francisco; Department of Neurology (M. Rensel), Cleveland Clinic, OH; Department of Neurology (B.W.-G.), State University of New York at Buffalo; Department of Pediatrics (G.A.), Loma Linda University, CA; Department of Neurology (L.B., M. Gorman), Boston Children's Hospital, MA; Department of Neurology (T.C.), Massachusetts General Hospital, Boston; Department of Neurology (M. Goyal, S.M.), Washington University in Saint Louis, MO; Department of Neurology (L.K.), New York University Langone Medical Center, NY; Department of Neurology (T.L.), Texas Children's Hospital, Houston; Department of Neurology (M. Rodriguez), Mayo Clinic, Rochester, MN; and Department of Neurology (J.R.), Biostatistician II (M.W.), and Department of Pediatrics (T.C.C.), University of Utah, Salt Lake City. Kristen.krysko@ucsf.edu.
Insights
Newer disease-modifying therapies (DMTs) are increasingly used in pediatric multiple sclerosis (MS) and clinically isolated syndrome (CIS). These treatments show similar short-term safety and side effect profiles to those observed in adults, aiding pediatric MS management.
Area of Science:
- Pediatric Neurology
- Immunology
- Neuroscience
Background:
- Multiple sclerosis (MS) and clinically isolated syndrome (CIS) can affect children and adolescents.
- Disease-modifying therapies (DMTs) are crucial for managing MS, with newer agents emerging for adult use.
- Understanding the safety and efficacy of these newer DMTs in pediatric populations is essential.
Purpose of the Study:
- To characterize the utilization patterns of newer disease-modifying therapies (DMTs) in pediatric patients under 18 years of age diagnosed with MS or CIS.
- To evaluate the safety and tolerability profiles of these newer DMTs in this young patient cohort.
- To compare the observed side effects in children with those reported in adult MS patients.
Main Methods:
- A cohort study was conducted involving pediatric patients with MS or CIS across 12 US outpatient practices.
- Data on DMT use, including type, duration, dosage, and reported side effects, were collected and analyzed.
- Newer DMTs were defined as those approved by the FDA or with increased use in adults post-2005.
Main Results:
- Out of 1,019 pediatric patients (748 MS, 271 CIS), 78% with MS and 11% with CIS received DMT before age 18.
- 42% received at least one newer DMT, with dimethyl fumarate and natalizumab being most common.
- The use of newer DMTs has increased over the past decade, particularly in patients aged 12 and older.
- Short-term side effect profiles of newer DMTs in children were comparable to those seen in adults.
Conclusions:
- Newer DMTs are frequently employed in the management of pediatric MS and CIS.
- These agents demonstrate comparable short-term safety and tolerability profiles in pediatric patients versus adults.
- Findings support the informed use of newer DMTs for managing pediatric MS and CIS.
Objective:
To characterize the use and safety of newer disease-modifying therapies (DMTs) in children with multiple sclerosis (MS) and clinically isolated syndrome (CIS) treated under 18 years of age.
Methods:
This is a cohort study including children with MS or CIS followed at 12 outpatient practices participating in the US Network of Pediatric MS Centers. DMT use, including duration, dose, and side effects, was analyzed. Newer DMTs were defined as agents receiving Food and Drug Administration approval or with increased use in adult MS after 2005.
Results:
As of July 2017, 1,019 pediatric patients with MS (n = 748) or CIS (n = 271) were enrolled (65% female, mean onset 13.0 ± 3.9 years, mean follow-up 3.5 ± 3.1 years, median 1.6 visits per year). Of these, 78% (n = 587) with MS and 11% (n = 31) with CIS received DMT before 18 years of age. This consisted of at least one newer DMT in 42%, including dimethyl fumarate (n = 102), natalizumab (n = 101), rituximab (n = 57), fingolimod (n = 37), daclizumab (n = 5), and teriflunomide (n = 3). Among 17%, the initial DMT prescribed was a newer agent (36 dimethyl fumarate, 30 natalizumab, 22 rituximab, 14 fingolimod, 2 teriflunomide). Over the last 10 years, the use of newer agents has increased, particularly in those ≥12 years and to lesser extent in those <12 years. The short-term side effect profiles of newer DMTs did not differ from those reported in adults.
Conclusion:
Newer DMTs are often used in pediatric MS, and have similar short-term safety, tolerability, and side effect profiles as in adults. These findings may help inform pediatric MS management.
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