During the past 5 years the number of trials in Myasthenia Gravis, and subsequent FDA/EMA approvals, has shown a huge increase. Soon after the availability of these therapies, the first problem that many specialists face, is how to make the right choice for the right patient with MG. This is a complex call for all of us due to the lack of comparative data on efficacy, safety, and tolerability of new therapies. We recently published a meta-analysis comparing placebo-controlled trials in MG and found a comparable effect of all innovative approaches, except for Rituximab, on the MG-ADL, and a higher effect of anti-FcRNs on the QMG (1). A second risk-benefit analysis of the same innovative treatments confirmed how easy it is in clinical practice to see an improvement in MG symptoms with new treatments. Efgartigimod showed the lowest NNT and CPIO in the studied treatment interval (2)..
While meta-analyses offer the opportunity of comparing different trials without the need of running direct comparisons, their limitations in MG trials are the difference in administration schemes (continuous for complement inhibitors vs cyclical for anti-FcRns) that does not allow for fair long-term comparison, and the differences in the inclusion/exclusion criteria of the underlying trials. Although randomized trials, where eligible patients are allocated by chance to different treatments, may not be feasible for refractory MG, we should aim at collecting good quality RWE data in an international effort to provide definitive evidence for treatment comparisons.
Key Points:
- Efgartigimod IV had the lowest Number Needed to Treat (NNT) versus placebo for achieving a ≥ 3- and ≥ 5-point reduction in QMG, as well as a ≥ 5-point reduction in MG-ADL
- All studied therapies had a similar Number Needed to Harm (NNH) versus placebo
- Efgartigimod has the lowest Cost Per Improved Outcome (CPIO) for all assessed efficacy outcomes.
References:
- Saccà F, Pane C, Espinosa PE, Sormani MP, Signori A. Efficacy of innovative therapies in myasthenia gravis: A systematic review, meta-analysis and network meta-analysis. Eur J Neurol. 2023 Dec;30(12):3854-3867. doi: 10.1111/ene.15872.
- Smith AG, Wolfe GI, Habib AA, Qi CZ, Yang H, Du M, Chen X, Gelinas D, Brauer E, Phillips G, Saccà F. Risk-Benefit Analysis of Novel Treatments for Patients with Generalized Myasthenia Gravis. Adv Ther. 2024 Dec;41(12):4628-4647. doi:10.1007/s12325-024-03014-5.
Publish on behalf of the Scientific Panel on Muscle and NMJ disorders