Effectiveness of early cycles of fast-acting treatment in generalised myasthenia gravis

BackgroundEarly fast-acting treatment (EFT) is the aggressive use of fast-acting therapies such as plasmapheresis, intravenous immunoglobulin and/or intravenous high-dose methylprednisolone (IVMP) from the early phases of treatment. EFT is reportedly beneficial for early achievement of minimal manifestations (MM) or better status with <= 5 mg/day prednisolone (MM5mg), a practical therapeutic target for myasthenia gravis (MG). ObjectiveThe current study aimed to clarify which specific EFT regimen is efficacious and the patient characteristics that confer sensitivity to EFT. MethodsWe recruited a total of 1710 consecutive patients with MG who enrolled in the Japan MG Registry for this large-cohort study. Among them, 1066 with generalised MG who had received immunotherapy were analysed. Prognostic background factors were matched in a 1:1 ratio using propensity score matching analysis between patients treated with EFT (n=350) and those treated without EFT (n=350). The clinical course and time to first achieve MM5mg after starting immunotherapy was analysed in relation to treatment combinations and patient characteristics. ResultsKaplan-Meier analyses showed that EFT had a significant effect on the achievement of MM5mg (p<0.0001, log-rank test; HR 1.82, p<0.0001). Notably, EFT was efficacious for any type of MG, and the inclusion of IVMP resulted in earlier and more frequent achievement of MM5mg (p=0.0352, log-rank test; HR 1.46, p=0.0380). In addition, early administration of calcineurin inhibitors also promoted MM5mg achievement. ConclusionEarly cycles of intervention with EFT and early use of calcineurin inhibitors provides long-term benefits in terms of achieving therapeutic targets for generalised MG, regardless of clinical subtype.

Automated summary

Early fast-acting treatment (EFT), including plasmapheresis, intravenous immunoglobulin and/or intravenous high-dose methylprednisolone (IVMP), is beneficial for achieving minimal manifestations (MM) or <= 5 mg/day prednisolone (MM5mg) in patients with myasthenia gravis (MG). This study aimed to identify the effective EFT regimen and patient characteristics. The results showed that EFT was effective for all types of MG, and the inclusion of IVMP and early use of calcineurin inhibitors promoted the achievement of MM5mg.