Scoping review of the availability and uptake of disease modifying therapies in children and adolescents with multiple sclerosis.
Lauren Strasser, Beyza Ciftci, Joley Johnstone, Jessie Cunningham, Helen Tremlett, E Ann Yeh
Author Information
Lauren Strasser: Division of Neurology, Department of Pediatrics, The Hospital for Sick Children, Program in Neuroscience and Mental Health-SickKids Research Institute, University of Toronto, Toronto, ON, Canada. ORCID
Beyza Ciftci: Department of Clinical Neurological Sciences, Western University, London, ON, Canada. ORCID
Joley Johnstone: Division of Neurology, Department of Pediatrics, The Hospital for Sick Children, Program in Neuroscience and Mental Health-SickKids Research Institute, University of Toronto, Toronto, ON, Canada.
Jessie Cunningham: SickKids Health Sciences Library, The Hospital for Sick Children, University of Toronto, Toronto, ON, Canada. ORCID
Helen Tremlett: Division of Neurology, Djavad Mowafaghian Centre for Brain Health, University of British Columbia, Vancouver, BC, Canada. ORCID
E Ann Yeh: Division of Neurology, Department of Pediatrics, The Hospital for Sick Children, Program in Neuroscience and Mental Health-SickKids Research Institute, University of Toronto, Toronto, ON, Canada. ORCID
INTRODUCTION: Approximately 10% of individuals with multiple sclerosis (MS) have pediatric-onset (<18-years-old). Pediatric-specific barriers to accessing disease modifying therapies (DMT) exist. Issues include few pediatric-based randomized controlled trials (RCT), often required for formal regulatory approval, and resultant challenges with cost/coverage. This review assessed real-world DMT uptake in pediatric-MS to better understand potential barriers. AREAS COVERED: We performed a scoping review of observational studies examining DMTs in patients with pediatric-MS published between 07/1993 and 06/2024. PRISMA guidelines were used. Databases searched included: Cochrane Library, Ovid MEDLINE/Embase, Scopus, and Web of Science. Studies must include >10 DMT exposed pediatric-MS patients with full-text available in English. RCTs/pharmaceutical-industry funded studies were excluded. Of 2114 abstracts screened, 88 studies were included. A total of 21,591 patients (13,411 females) were included. DMTs were used in 68.7% ( = 14,833). Most studies were from Europe (53.4%), North America (22.7%), or the Middle East (10%). Regional variabilities were found in DMT uptake between continents. Only 13 (14.8%) studies included information on DMT funding source. EXPERT OPINION: Pediatric-MS patients showed low DMT uptake with variability in DMT use based on region. Limited data was found regarding specific barriers to DMT access. Further research is needed to better understand regional barriers to access.
