Science News: Initial Clinical Experience and Outcomes of Ravulizumab in AChR+ gMG

Submitted by: Nakul Katyal, MD

Edited by: Rebecca O'Bryan, MD

Citation: Crawford TO, Day JW, De Vivo DC, et al. Long-term efficacy, safety, and patient-reported outcomes of apitegromab in patients with spinal muscular atrophy: results from the 36-month TOPAZ study. Front Neurol. 2024;15:1419791. Published 2024 Jul 22. doi:10.3389/fneur.2024.1419791

Summary: Ravulizumab, a longer-acting C5 monoclonal antibody, has been FDA-approved for patients with acetylcholine receptor antibody-positive generalized myasthenia gravis (AChR+ve gMG). This study detailed the initial clinical experience of using ravulizumab in AChR+ve gMG.

The cohort included 18 patients with a mean age of 61.83 years (±16.08) and a mean Myasthenia Gravis Activities of Daily Living (MG-ADL) score of 5.05 (±3.84). Among 10 complement inhibitor-naive patients, there was a significant reduction in the mean MG-ADL score from 6.6 (±3.58) at baseline to 4.4 (±2.28) post-ravulizumab. Clinically meaningful reductions were observed in 60% of these patients, with 2 achieving minimal symptom expression (MSE).

In the 8 patients who switched from eculizumab to ravulizumab, the mean MG-ADL score decreased from 3.25 (±3.34) at baseline to 1.5 (±2.34) post-ravulizumab. There were 4 patients with baseline MSE status that maintained MSE status post ravulizumab. Out of the 4 patients without baseline MSE status, 2 patients achieved MSE status post ravulizumab therapy whereas the MG-ADL score remained unchanged for the remaining 2 patients. 

Comments: This study demonstrates the clinical effectiveness of ravulizumab in diverse populations of AChR+ve MG patients. Among complement inhibitor-naive patients, 60% experienced a clinically meaningful improvement in MG-ADL scores with ravulizumab. Patients who switched from eculizumab to ravulizumab showed further improvement in their mean MG-ADL scores. Additionally, the majority of patients on prednisone therapy were able to reduce their prednisone dosage. A notable limitation of the study is the variable timing of patient evaluations post-infusion, making it challenging to determine the exact onset of ravulizumab's benefits. Further long-term analysis is required to evaluate the persistence of clinical benefits and the need for ongoing complement inhibition therapy.