Post marketing real-world experience of ravulizumab in NMOSD.

BACKGROUND

Ravulizumab is a long-acting complement C5 inhibitor recently approved for treatment of AQP4-IgG-positive neuromyelitis optica spectrum disorder (AQP4-NMOSD). No post-marketing real-world data is currently available.

OBJECTIVE

To assess whether the real-world clinical experience of ravulizumab in AQP4-NMOSD is similar to that seen in pivotal clinical trials.

DESIGN

A multicenter retrospective cohort study that uses standardized data extracted from medical records.

METHODS

We conducted a multi-center retrospective cohort study of AQP4-NMOSD patients treated with ravulizumab at two major neurological centers in the UAE. Data on demographics, disease characteristics, relapse rates, EDSS scores, and treatment-related adverse events were extracted from electronic medical records. Annualized relapse rates and EDSS changes were calculated, and adverse events were summarized using counts and frequencies.

RESULTS

Nine AQP4-NMOSD patients (female:male ratio 8:1) were treated with ravulizumab. Median disease duration was 6.6 years (IQR 6.5), and 44.4% were treatment-naïve. Over a median follow-up of 14.5 months (IQR 6.25), all patients remained relapse-free, with annualized relapse rate decreasing from 0.9 ± 0.5 to 0 (p = 0.0012). Mean EDSS improved from 3.9 ± 2.4 pre-treatment to 2.2 ± 3 post-treatment (p = 0.02). Two patients (22.2%) experienced mild infusion reactions, and two patients (22.2%) developed uncomplicated infections. No cases of meningitis were observed. One patient discontinued therapy due to insurance denial.

CONCLUSION

Early post-marketing real-world experience with ravulizumab in the UAE is consistent with pivotal trial results and aligns with the safety and efficacy profiles reported for other complement-targeted therapies; however, findings are limited by the small sample size and short follow-up.