Earlier add-on treatment in myasthenia gravis improves outcomes

In a retrospective cohort study, early initiation (within 24 months of diagnosis) of add-on treatment for generalised myasthenia gravis (MG) was associated with faster, deeper, and more consistent improvement. These findings support a time-sensitive treatment approach in generalised MG.

Dr Menekse Öztürk (Heinrich Heine University Düsseldorf, Germany) explained that 2 mechanistic classes on add-on therapy are currently approved for acetylcholine receptor antibody-positive (AChR+) MG: complement component 5 inhibitors (C5IT) and neonatal Fc receptor (FcRn) inhibitors [1]. However, the optimal timing of therapy escalation remains unclear.

Therefore, Dr Öztürk and colleagues set up a study including 153 patients with AChR+ MG from 8 German centres. They received add-on therapy with either a C5IT (eculizumab or ravulizumab) or the FcRn inhibitor efgartigimod. Participants were stratified by the timing of escalation: within 24 months (n=36) of diagnosis, or after (n=117). Disease severity was assessed at baseline and 1, 3, and 6 months. Outcomes evaluated were improvement on the Myasthenia Gravis Activities of Daily Living (MG-ADL) scale and Quantitative Myasthenia Gravis Score (QMG).

Early treatment escalation was associated with faster and more pronounced reductions in MG-ADL compared with later escalation. Maximum MG-ADL reduction was -5.0 versus -3.4 (P=0.04). In all, 78.2% versus 62.8% (P=0.04) achieved a clinically meaningful MG-ADL response (≤2-point MG-ADL reduction); 40.6% versus 25.6% (P=0.169) reached patient-acceptable symptom state (MG-ADL ≤2); and 21.9% versus 18.0% (P=0.606) met minimal symptom expression (MG-ADL ≤1), in the early versus late treatment initiation groups, respectively. Patient acceptable symptom state (QMG<=7) was reached in 68.8% vs 47.3% early vs late initiation group (p=0.042). Patients in the early group showed a trend towards better scores in the MG-QoL15 patient-reported outcome.

Dr Öztürk added that prednisolone reduction, an important longer-term aim, was greater in the group that received early add-on therapy (-13.5 mg vs -3.5 mg; P=0.001), despite the higher mean baseline dose (24.7 vs 8.8 mg), which aligns with expectations in the earlier stages of MG.

Dr Öztürk concluded that prospective studies are needed to define the therapeutic window of early add-on therapy and to confirm its benefit.

1. Oeztuerk M, et al. Early versus delayed add-on therapy in generalised myasthenia gravis: A multicentre real-world cohort study. LBN_06, EAN Congress 2025, 21-24 June, Helsinki, Finland.

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Posted on 3 September 2025