Eculizumab monotherapy for NMOSD: Data from PREVENT and its open-label extension.
Sean J PittockKazuo FujiharaJacqueline PalaceAchim BertheleHo Jin KimCelia Oreja-GuevaraIchiro NakashimaMichael LevyShulian ShangMarcus YountzLarisa MillerRóisín ArmstrongDean M Wingerchuknull nullPublished in: Multiple sclerosis (Houndmills, Basingstoke, England) (2021)
During PREVENT (a phase 3, randomized, double-blind, placebo-controlled, time-to-event study) and its open-label extension (interim analysis), 33 adults with aquaporin-4 immunoglobulin G-positive neuromyelitis optica spectrum disorder (AQP4-IgG + NMOSD) received eculizumab monotherapy for a median of 2.8 years (range, 14 weeks-5.2 years). At 192 weeks (~4 years), 96% of these patients were free from adjudicated relapses (Kaplan-Meier analysis; 95% confidence interval, 75.7-99.4). During PREVENT, 95% (20/21) of patients receiving eculizumab monotherapy had no disability worsening. Eculizumab monotherapy provides effective long-term relapse prevention, relieving the chronic immunosuppression burden in patients with AQP4-IgG + NMOSD. ClinicalTrials.gov; PREVENT: NCT01892345; open-label extension: NCT02003144.
Keyphrases
- open label
- phase iii
- phase ii
- double blind
- placebo controlled
- clinical trial
- study protocol
- phase ii study
- end stage renal disease
- combination therapy
- newly diagnosed
- squamous cell carcinoma
- multiple sclerosis
- prognostic factors
- risk factors
- ejection fraction
- big data
- artificial intelligence
- gestational age
- patient reported outcomes