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 at 1, 3, and 6 months. The primary outcome was maximum improvement on the Myasthenia Gravis Activities of Daily Living (MG-ADL) scale.

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.

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 is logical in the earlier stages of MG.

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 2025, Helsinki, Finland.

Medical writing support was provided by Michiel Tent.

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Posted on 24 June 2025