INTERFERON BETA-1A TREATMENT IN EARLY-CHILDHOOD AND JUVENILE MULTIPLE SCLEROSIS

Objectives: To study long-term safety, tolerability and efficacy of subcutaneous (sc) interferon (IFN) beta-1a in paediatric patients with multiple sclerosis (MS).

Methods: Twenty seven children with relapsing/remitting MS (RRMS) (n=23) and secondary progressive MS (SPMS) (n=4), were included. Age at first injection ranged between 3.8–17.9 years. All patients had clinically defined MS according to Poser's and McDonald's criteria, and had experienced at least one relapse in the last 2 years. Neurological examination, EDSS score, formal questionnaire for adverse events, liver function and blood tests, were assessed regularly during treatment. Eighteen patients received the adult-dose of 22 mcg sc three times weekly (tiw) from the start and seven children after a dose-escalation phase. Differences between relapse rate and EDSS scores before and after treatment were assessed by the two-sided Wilcoxon signed-rank test.

Results: Treatment period ranged 6–89 months. Frequencies of clinical adverse events were: injection-site reaction 75%; flu-like symptoms 58%; menstrual disorder 11%. Transient elevation of liver enzymes was observed in 33% of patients. No patient discontinued treatment due to flu-like symptoms, skin reactions or laboratory abnormalities. Two possibly related serious adverse events occurred: a juvenile chronic arthritis-like illness and a suicide attempt. A significant relapse rate reduction was observed in RRMS patients at 22 mcg tiw. Median EDSS scores showed reductions during the seven years on therapy in early-childhood patients, and remain stable in juvenile patients with RRMS.

Conclusion: IFN beta-1a treatment was well tolerated in most children of this cohort. We recommend to routinely assess genetically susceptibility for JCA and to evaluate mood disorders before initiating therapy; and to regularly monitoring liver function during treatment. Further paediatric MS treatment trials are necessary to extend our results.