Effectiveness and Safety of Ravulizumab in Generalized Myasthenia Gravis (gMG): Updated Analysis from a Global Registry
Pushpa Narayanaswami1, Michael Pulley2, Samir Macwan3, James Winkley4, Lida Zeinali5, Chuang Liu5, Vern Juel6, Rup Tandan7, Francesco Sacca8, Andrew Gordon9, James Howard10
1Beth Israel Deaconess Medical Center/Harvard Medical School, 2University of Florida, 3Eisenhower Health Center, 4Baptist Health Medical Group Neurology, 5Alexion, AstraZeneca Rare Disease, 6Duke University, 7University of Vermont Medical Center, 8NSRO Department, University of Naples Federico II, 9Northwest Neurology, Ltd., 10The University of North Carolina at Chapel Hill School of Medicine
Objective:
To assess updated effectiveness and safety data for the complement protein C5 inhibitor therapy (C5IT) ravulizumab among patients with gMG in routine clinical practice.
Background:
In prior analyses from the ongoing, global MG SPOTLIGHT Registry (NCT04202341), ravulizumab was well tolerated and effective in clinical practice.
Design/Methods:
MG-ADL total scores and MGFA classification were assessed in patients enrolled in the MG SPOTLIGHT Registry who received ravulizumab, including those who received ravulizumab only (ravu-only subgroup) or transitioned from eculizumab to ravulizumab (ecu-to-ravu subgroup) with data available prior to C5IT initiation (“pre-C5IT”) and ≥1 assessment post-ravulizumab initiation (“post-ravu”). This descriptive study had no adjustment for covariates. Safety was assessed in all patients.
Results:
114/249 patients enrolled as of 1/1/2024 met inclusion criteria (male: 62%; mean±SD age at MG diagnosis: 57.9±18.6 years). Among patients with 1 pre-C5IT and 2 post-ravu assessments (n=52), average ravulizumab treatment duration was 4.1 months at first post-ravu assessment (FA) and 10.4 months at last post-ravu assessment (LA). Mean±SD MG-ADL scores improved (pre-C5IT: 7.6±3.6; FA: 3.9±3.6; LA: 3.4±3.3), as did the proportions of patients with minimal symptom expression (MSE, MG-ADL≤1) (pre-C5IT: 1/52 [2%]; FA: 18/52 [35%]; LA: 17/52 [33%]) and MGFA classification 0-II (pre-C5IT: 18/45 [40%]; FA: 41/45 [91%]; LA: 40/45 [89%]). In the ecu-to-ravu subgroup, improvements were largest post-eculizumab initiation (pre-C5IT vs last eculizumab assessment: mean±SD MG-ADL, 8.0±4.0 vs 4.4±4.2; MSE, 1/24 [4%] vs 7/24 [29%]; MGFA 0-II, 5/21 [24%] vs 19/21 [90%]) with sustained/gradual improvements through LA (3.0±2.8; 9/24 [38%]; 20/21 [95%]). In the ravu-only subgroup, outcomes improved post-ravu (pre-C5IT vs LA: mean±SD MG-ADL, 6.3±3.0 vs 4.0±3.4; MGFA 0-II, 9/14 [64%] vs 12/14 [86%]). Ravulizumab was well tolerated; no meningococcal infections were reported. Updated data will be presented.
Conclusions:
Consistent with prior findings, these results demonstrate the long-term effectiveness and safety of ravulizumab in patients with gMG in routine clinical practice.
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