Answers are grounded in this paper and cite their sources.
Background Ravulizumab is a long-acting terminal complement C5 inhibitor approved for acetylcholine receptor antibody-positive generalized myasthenia gravis (AChR-Ab-positive gMG). However, real-world evidence from the Gulf region remains limited. This study describes clinical outcomes and safety of ravulizumab in a real-world cohort of patients with AChR-Ab-positive gMG in the UAE. Methods We conducted a hybrid retrospective-prospective, noninterventional, hospital-based case series of 12 consecutive adults treated with ravulizumab. Data available through September 30, 2025, were collected retrospectively, whereas data from October 1, 2025, onward were collected prospectively. Outcomes included Myasthenia Gravis Activities of Daily Living (MG-ADL), Myasthenia Gravis Composite (MGC), Myasthenia Gravis Foundation of America class (MGFA), MGFA Post-Intervention Status (MGFA-PIS), MG-ADL-defined minimal symptom expression (MSE), prednisone exposure, rescue therapy use, relapse/worsening, treatment persistence, and adverse events. Results Nine of the 12 patients were female, and the median age was 41.5 years. Median disease duration before ravulizumab initiation was 6.5 years, and median follow-up was 549 days. At final follow-up, median MG-ADL improved from 8 to 3 (median change −4.5, p < 0.001), MGC from 15 to 3.5 (change −9.5, p < 0.001), and MGFA class from 3 to 2 (change −1, p < 0.001). Clinically meaningful MG-ADL, MGC, and MGFA responses each occurred in 11/12 patients. Final MGFA-PIS was Minimal Manifestations (MM) in 5/12, Improved in 5/12, and Unchanged in 2/12; no patient was classified as Worse. Separately, 3/12 patients (25.0%) achieved MSE, defined as a final MG-ADL score of 0–1. Four patients experienced clinical worsening events; three of whom received a single outpatient IVIG infusion. None developed myasthenic crisis or required hospitalization or PLEX. No serious adverse event or meningococcal infection was recorded. Conclusion In this UAE case series, ravulizumab was associated with rapid and durable clinically meaningful improvement in both patient-reported and clinician-rated measures of gMG, with no serious safety concerns. Although limited by the small sample size and hybrid retrospective-prospective design, the findings add important real-world evidence from a region underrepresented in major clinical trials.
No takes yet. Share an insight, caveat, or question.
Aurangzeb et al. (2026) studied this question.
Synapse has enriched 10 related papers. Consider them for comparative context: