Journal of neurology · 2026
Early versus late add-on therapy in generalized myasthenia gravis: a multicenter real-world cohort study.
СтатьяМногоцентровое исследованиеНаблюдательное исследованиеНазвание и аннотация: Перевод в очереди
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Oeztuerk Menekse, Huntemann Niklas, Gerischer Lea, Herdick Meret, Nelke Christopher, Stascheit Frauke, Hoffmann Sarah, Lehnerer Sophie, Stein Maike, Schubert Charlotte, Schneider-Gold Christiane, Pfeuffer Steffen, Krämer Heidrun H, Konen Franz Felix, Skripuletz Thomas, Pawlitzki Marc, Rehmann Robert, Arat Ercan, Menskes Christina B, Glaubitz Stefanie, Zschüntzsch Jana, Scherwietes Valerie, Totzeck Andreas, Hagenacker Tim, Meisel Andreas, Meuth Sven G, Ruck Tobias
Аннотация
BACKGROUND: This study examined whether the timing of targeted add-on therapy initiation influences clinical outcomes in acetylcholine receptor (AChR) antibody-positive generalized myasthenia gravis (gMG), based on the hypothesis that earlier escalation may improve treatment response by intervening before structural or immunological consolidation occurs.
METHODS: In this multicenter, retrospective real-world cohort study, 153 patients with AChR antibody-positive gMG were included from eight German tertiary centers. All received either complement C5 inhibitors (eculizumab, ravulizumab) or the FcRn antagonist efgartigimod as add-on therapy. Patients were grouped by treatment initiation within 24 months of diagnosis (Early Intensified Treatment; EIT) or later (Late Intensified Treatment; LIT). MG-ADL, QMG, and MG-QoL15 scores, as well as daily corticosteroid and pyridostigmine doses, were assessed at baseline and at 1, 3, and 6 months.
RESULTS: The EIT group (n = 36) showed more pronounced and consistent clinical improvement. Significant differences emerged in maximum MG-ADL (p = 0.013) and QMG (p = 0.002) reductions. Patient-acceptable symptom states (MG-ADL ≤ 2, QMG ≤ 7) were more often reached with EIT (p = 0.038, p = 0.006). QMG worsening occurred only in the LIT group (n = 117) (p = 0.021). Prednisone declined more steeply in EIT patients (p = 0.001).
CONCLUSION: Initiating add-on therapy within two years of diagnosis was associated with stronger and more consistent clinical responses, fewer deteriorations, and a steeper reduction of treatment burden. These findings support timely escalation as a strategy to enhance both efficacy and tolerability in gMG care.
METHODS: In this multicenter, retrospective real-world cohort study, 153 patients with AChR antibody-positive gMG were included from eight German tertiary centers. All received either complement C5 inhibitors (eculizumab, ravulizumab) or the FcRn antagonist efgartigimod as add-on therapy. Patients were grouped by treatment initiation within 24 months of diagnosis (Early Intensified Treatment; EIT) or later (Late Intensified Treatment; LIT). MG-ADL, QMG, and MG-QoL15 scores, as well as daily corticosteroid and pyridostigmine doses, were assessed at baseline and at 1, 3, and 6 months.
RESULTS: The EIT group (n = 36) showed more pronounced and consistent clinical improvement. Significant differences emerged in maximum MG-ADL (p = 0.013) and QMG (p = 0.002) reductions. Patient-acceptable symptom states (MG-ADL ≤ 2, QMG ≤ 7) were more often reached with EIT (p = 0.038, p = 0.006). QMG worsening occurred only in the LIT group (n = 117) (p = 0.021). Prednisone declined more steeply in EIT patients (p = 0.001).
CONCLUSION: Initiating add-on therapy within two years of diagnosis was associated with stronger and more consistent clinical responses, fewer deteriorations, and a steeper reduction of treatment burden. These findings support timely escalation as a strategy to enhance both efficacy and tolerability in gMG care.