Fingolimod Therapy for Pediatric Relapsing-Remitting Multiple Sclerosis: A Real-Life Study

Abstract

Objective: Clinical studies in childhood multiple sclerosis (MS) are very limited compared with adults. Although first-line injection therapies are well tolerated in this patient group, there are difficulties in the long-term treatment of patients due to the difficulty and adverse effects of injections for children. Oral therapies are better tolerated for use and adverse effects compared with injection therapies. This study aimed to evaluate the clinical features and treatment outcomes of patients aged under 18 years with relapsing-remitting MS (RRMS) who received fingolimod treatment. Materials and Methods: In this study, the clinical records of 22 patients with RRMS who received fingolimod treatment were examined. The patients were prospectively followed up between February 2015 and December 2018. The patients were evaluated in terms of relapse rate, Expanded Disability Status Scale (EDSS) scores, brain and cervical magnetic resonance imaging, and adverse effects before and after fingolimod treatment. Results: The median age of 13 (59%) female and 9 (41%) male patients was 18 (range: 10-21) years. The median fingolimod treatment duration was 2.5 (range: 1.3-3.9) years. The median age of disease onset was 13 (range: 8-17) years. Fifteen patients (68%) received a first-line treatment prior to fingolimod. The reason for switching to fingolimod was inefficacy in 33%, adverse effects in 40%, and both in %27 of patients. In 32% of patients, fingolimod was started as a first-line treatment. A total of three patients had recurrent relapses under treatment with fingolimod. The median annualized relapse rate (ARR) before fingolimod was 1.9 (range: 0.3-8.0), and the ARR with fingolimod was 0 (range: 0-2.0) (Wilcoxon’s signed-rank test; p<0.001). The median EDSS was 1.5 (range: 1.0-4.0) before fingolimod, and the EDSS after treatment was 1.5 (range: 1.0 to 5.0) (Wilcoxon’s signed rank test; p=0.3). None of the patients had any adverse effects during the first dose monitoring. Conclusion: Our study shows that fingolimod is an effective and safe treatment alternative in childhood MS.

Keywords

• child and adolescent multiple sclerosis
• fingolimod
• real-world evidence