Eculizumab treatment for myasthenia gravis subgroups: 2021 update
作者
Jiao, L; Li, HH; Guo, SG
作者单位
[Jiao, Li; Li, Honghao; Guo, Shougang] Shandong First Med Univ, Shandong Prov Hosp, Dept Neurol, Jing Wu Rd, Jinan 250021, Shandong, Peoples R China
摘要
Eculizumab is a recombinant humanized monoclonal antibody that targets the complement protein C5, inhibiting its cleavage into C5a and C5b and ultimately preventing the formation of C5b-9 membrane attack complex (MACs), thereby protecting the neuromuscular junction from the damage of complement activation. In 2017, eculizumab became the second FDA-approved medication for AchR-positive generalized myasthenia gravis (gMG) patients based on the successful results of a randomized, double-blinded, placebo-controlled, phase 2, phase 3 study (the REGAIN trial) and its open-label extension study. Despite the efficacy of eculizumab in treating AchR antibody-positive refractory gMG was demonstrated in the REGAIN study, there is few information on its efficacy in other subgroup of MG patients including seronegative MG, thymoma-associated MG and MG crisis. This narrative review summarizes current clinical studies of eculizumab in these refractory gMG patients, with a focus on the therapeutic efficacy and tolerability in different subgroup of MG.
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