Pediatric-onset multiple sclerosis (POMS) is a chronic autoimmune disease that affects the central nervous system in children and adolescents. Unfortunately, there are few approved therapies for POMS, making it challenging to manage the symptoms effectively. However, a clinical trial has found that dimethyl fumarate (DMF) is more efficacious than interferon β-1a (IFNβ-1a) in treating POMS.
The CONNECT study was a randomized clinical trial that compared the efficacy, safety, and tolerability of DMF and IFNβ-1a in patients with POMS aged 10 to less than 18 years. The study was conducted between August 2014 and November 2020, and the data were analyzed from January through October 2021.
The primary endpoint of the study was the proportion of patients free of new or newly enlarging T2 hyperintense lesions at week 96 among trial completers. The secondary endpoints included the number of T2 lesions, the proportion of patients free of relapse, annualized relapse rate (ARR), and safety.
This study looked at 150 kids with a type of multiple sclerosis called POMS. 78 of them received DMF, and 72 received IFNβ-1a. After 96 weeks, a smaller proportion of patients who completed the study without dropping out had new or bigger brain lesions with DMF (16.1%) compared to IFNβ-1a (4.9%). When they looked at all the patients, including those who dropped out, the numbers were still better for DMF (12.8%) compared to IFNβ-1a (2.8%).
More patients who took DMF were free of relapse after 96 weeks compared to IFNβ-1a (66.2% vs 52.3%). The adjusted ARR (annualized relapse rate) was also lower for DMF (0.24) compared to IFNβ-1a (0.53). The number of side effects, serious side effects, and patients who stopped the medicine because of side effects were similar for both DMF and IFNβ-1a.
These results indicate that DMF is more effective than IFNβ-1a in treating POMS. The study found that more pediatric patients with POMS treated with DMF were free of new or newly enlarging T2 lesions, and the adjusted ARR was lower among these patients compared with those treated with IFNβ-1a. Moreover, the safety profiles of both drugs were similar.
DMF is an FDA-approved therapy for adults with multiple sclerosis, but its use in children and adolescents is limited. The findings of this study provide strong evidence for the use of DMF in treating POMS. The study's results can help clinicians make informed decisions regarding the best treatment options for their patients with POMS.
The CONNECT study provides evidence that DMF is more efficacious than IFNβ-1a in treating POMS, and both drugs have similar safety profiles. These findings are a significant step forward in the management of POMS, and DMF should be considered a viable treatment option for pediatric patients with POMS. Further studies are needed to assess the long-term effects of DMF treatment in children and adolescents with POMS.__________