Data were available for 40 adult patients in Japan [62.5% (25/40) female; mean age at
eculizumab initiation, 51.0 years]. Fifteen patients had a history of
thymoma. Six patients were excluded from the effectiveness analysis set due to participation in the open-label extension part of the phase III, randomized, double-blind, placebo-controlled REGAIN study [ClinicalTrials.gov identifier: NCT02301624]. After 26 weeks' follow up, 32 patients (80%) were continuing
eculizumab treatment.
Adverse drug reactions were reported by seven patients [most frequently
headache (n = 3)]. One death was reported during
eculizumab treatment (relationship unclear as determined by the treating physician) and there was one death 45 days after the last dose (considered unrelated). No
meningococcal infections were reported. Mean (standard deviation) changes from baseline in
Myasthenia Gravis-
Activities of Daily Living (MG-
ADL) and Quantitative
Myasthenia Gravis (QMG) scores were -3.7 (2.61) (n = 27) and -5.6 (3.50) (n = 26), respectively, at 12 weeks, and -4.3 (2.72) (n = 26) and -5.6 (4.02) (n = 24), respectively, at 26 weeks. Improvements in MG-
ADL and QMG scores were generally similar in patients with/without a history of
thymoma. Frequency of
IVIg use decreased following
eculizumab initiation.
CONCLUSION: In a real-world setting,
eculizumab was effective and well tolerated for the treatment of AChR+
gMG in adult Japanese patients whose disease was refractory to
IVIg or
plasmapheresis. These findings are consistent with the efficacy and safety results from the global phase III REGAIN study of
eculizumab.