Aprepitant is a neurokinin-1 receptor antagonist (NK1R) approved for the treatment of chemotherapy-induced nausea. We aimed to investigate the safety and efficacy of aprepitant in patients with subacute sclerosing panencephalitis (SSPE).
A randomized, double-blind, placebo-controlled study was conducted in patients with SSPE assigned to receive two courses of aprepitant 250 mg/day p.o. or placebo for 15 days with an interval of 2 months between courses. Primary endpoints were safety and tolerability and secondary endpoint, clinical improvement/stabilization assessed by SSPE Scoring System. Electroencephalography (EEG), brain magnetic resonance imaging (MRI) and cerebrospinal fluid measles-specific IgG index were evaluated before and after treatment.
Sixty-two SSPE patients were allocated to aprepitant (n=31, median age 18 years) or placebo (n=31, median age 22 years) groups. Fifteen patients left the study within the first 6 months and 12 patients left between 6-12 months. Aprepitant was well tolerated and treatment–associated adverse events were similar to those described in the treatment of nausea. Clinical status at 6 and 12 months follow-up did not differ between aprepitant and placebo groups. Post-treatment EEG scores at 12 months were better in the aprepitant group (p=0.015). Cerebral atrophy on MRI increased in both groups while measles-specific IgG index decreased in the placebo group.
In this first clinical trial of aprepitant treatment in SSPE patients, the drug was safe and well tolerated. No clinical effect was observed. A modest improvement in EEG findings might justify trials for longer periods because EEG changes can precede clinical findings in SSPE.