Abstract
Objective NA.
Background The terminal complement C5 inhibitor eculizumab is approved in Japan for the prevention of aquaporin-4 antibody-positive (AQP4+) neuromyelitis optica spectrum disorder (NMOSD) relapse and undergoing mandatory post-marketing surveillance (PMS) of real-world use.
Design/Methods This PMS interim analysis assessed eculizumab's safety and effectiveness in Japanese patients AQP4+ NMOSD patients from approval (November 2019) to interim data cut-off (1 April 2021).
Results At data cut-off, 79 patients treated with eculizumab were registered under PMS. Data were available for 20 patients who provided consent for publication and constituted the safety data set; three patients from the phase 3 PREVENT study were excluded from the effectiveness data set. Two patients discontinued before data cut-off owing to physician or patient decision (one each). In the effectiveness data set, 16/17 (94%) patients were female, mean illness duration was 9.2 years (standard deviation [SD] 7.5 years, range 0.3–23.8 years) and mean age at eculizumab initiation was 48.6 years (SD 11.9 years, range 27–77 years). In the 2 years before eculizumab, 12/17 patients experienced relapse, and 6/17 experienced =2 relapses. Importantly, no relapses were reported in the effectiveness data set (10.7 patient-years [PY] of treatment); this compared favorably with a 0.65/PY relapse rate in the 2 years before treatment (34.0 PY). Three adverse events (malaise, eyelid oedema, erythema) and one serious adverse event (urinary tract infection [UTI]) were observed in three patients in the safety data set: eyelid oedema and erythema (one patient) were deemed treatment-related; malaise and UTI were deemed unrelated to treatment. No meningococcal infections were reported, and the safety results were consistent with those from PREVENT.
Conclusions For the first time in a real-world setting, eculizumab was effective in preventing relapses and well tolerated in Japanese patients with AQP4+ NMOSD, consistent with its efficacy and safety profile in the global phase 3 PREVENT study.
Footnotes
Disclosure: Dr. Nakashima has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Alexion. Dr. Nakashima has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Biogen. Dr. Nakashima has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Novartis. Dr. Nakashima has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Chugai. Dr. Nakashima has received personal compensation in the range of $10,000-$49,999 for serving on a Speakers Bureau for Alexion. Dr. Nakashima has received personal compensation in the range of $10,000-$49,999 for serving on a Speakers Bureau for Biogen. Dr. Nakashima has received personal compensation in the range of $10,000-$49,999 for serving on a Speakers Bureau for Novartis. Dr. Nakashima has received personal compensation in the range of $10,000-$49,999 for serving on a Speakers Bureau for Chugai. The institution of Dr. Nakashima has received research support from LSI Medience. Dr. Hoshino has nothing to disclose. Ms. Okamura has received personal compensation for serving as an employee of Alexion Pharma GK. Ms. Okamura has received stock or an ownership interest from Astra Zeneca Plc. Mr. Kikui has received personal compensation for serving as an employee of Alexion Pharma GK. Dr. Fujihara has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Abbvie. Dr. Fujihara has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Merck Boipharma. Dr. Fujihara has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Japan Tobacco. Dr. Fujihara has received personal compensation in the range of $5,000-$9,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Novartis. Dr. Fujihara has received personal compensation in the range of $5,000-$9,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Mitsubishi-Tanabe. Dr. Fujihara has received personal compensation in the range of $5,000-$9,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Chugai. Dr. Fujihara has received personal compensation in the range of $5,000-$9,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Alexion. Dr. Fujihara has received personal compensation in the range of $5,000-$9,999 for serving on a Scientific Advisory or Data Safety Monitoring board for UCB. Dr. Fujihara has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Biogen. The institution of Dr. Fujihara has received research support from Ministry of Health, Welfare and Labor of Japan. Dr. Fujihara has received personal compensation in the range of $10,000-$49,999 for serving as a speaker, chair, etc with Novartis. Dr. Fujihara has received personal compensation in the range of $5,000-$9,999 for serving as a speaker, chair, etc with Biogen. Dr. Fujihara has received personal compensation in the range of $10,000-$49,999 for serving as a speaker, chair, etc with Mitsubishi-Tanabe. Dr. Fujihara has received personal compensation in the range of $10,000-$49,999 for serving as a speaker, chair, etc with Chugai. Dr. Fujihara has received personal compensation in the range of $10,000-$49,999 for serving as a speaker, chair, etc with Alexion. Dr. Fujihara has received personal compensation in the range of $500-$4,999 for serving as a chair, speaker with Asahi Kasei Medical. Dr. Fujihara has received personal compensation in the range of $500-$4,999 for serving as a speaker with Eisai. Dr. Fujihara has received personal compensation in the range of $500-$4,999 for serving as a speaker, chair with Teijin. Dr. Fujihara has received personal compensation in the range of $5,000-$9,999 for serving as a speaker with Roche.
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