Multiple Sclerosis Journal | January 12, 2023

Jens Kuhle, Tanuja Chitnis, Brenda Banwell, Marc Tardieu, Douglas L Arnold, Andreea M Rawlings, Svend S Geertsen, Alex L Lublin, Stephane Saubadu, Philippe Truffinet, Ludwig Kappos

Multiple Sclerosis Journal. 2023;0(0).

https://doi.org/10.1177/13524585221144742

Abstract

Background:

The phase 3 TERIKIDS study demonstrated efficacy and manageable safety for teriflunomide versus placebo in children with relapsing multiple sclerosis (RMS).

Objective:

Evaluate plasma neurofilament light chain (pNfL) concentrations in TERIKIDS.

Methods:

Patients received placebo or teriflunomide (14 mg adult equivalent) for up to 96 weeks in the double-blind (DB) period. In the open-label extension (OLE), all patients received teriflunomide until up to 192 weeks after randomization. pNfL was measured using single-molecule array assay (Simoa^® NF-light^™).

Results:

Baseline mean age was 14.5 years; 69.4% were female. Baseline geometric least square mean pNfL levels were similar for teriflunomide (n = 78) and placebo (n = 33) patients (19.83 vs 18.30 pg/mL). Over the combined DB and OLE periods, pNfL values were lower for teriflunomide versus placebo (analysis of variance p < 0.01; Week 192: 10.61 vs 17.32 pg/mL). Observed between-group pNfL differences were attenuated upon adjustment for gadolinium (Gd)-enhancing or new/enlarged T2 lesion counts at DB Week 24. Higher baseline pNfL levels were associated with shorter time since first MS symptom onset, higher baseline Gd-enhancing lesion counts and T2 lesion volume, and increased hazard of high magnetic resonance imaging activity or clinical relapse during the DB period.

Conclusion:

Teriflunomide treatment was associated with significantly reduced pNfL levels in children with RMS.