Myasthenie.com

Neurology. 2025 Apr 8;104(7_Supplement_1):1835. doi: 10.1212/WNL.0000000000208774. Epub 2025 Apr 7.

ABSTRACT

OBJECTIVE: To report the third-year analysis from the postmarketing surveillance (PMS) evaluating real-world effectiveness and safety of eculizumab in adults with anti-acetylcholine receptor antibody-positive (AChR-Ab+) gMG.

BACKGROUND: Eculizumab was approved in Japan for treatment of gMG in December 2017. Regulatory-mandated PMS of its real-world use is ongoing.

DESIGN/METHODS: This real-world analysis included patients with gMG receiving eculizumab who had eligible data from approval to data cutoff (April 2023). Changes in Myasthenia Gravis Activities of Daily Living (MG-ADL) total score, Quantitative Myasthenia Gravis (QMG), number of crises, intravenous immunoglobulin (IVIg)/plasma exchange (PLEX) use, concomitant oral corticosteroid doses, and safety were assessed.

RESULTS: The effectiveness analysis included 254 patients (female: 66.1%; mean [SD] age: 53.1 [15.6] years; average [SD] duration of gMG: 7.5 [8.0] years; Myasthenia Gravis Foundation of America [MGFA] classification at first dose: IIa, 20.5%; IIb, 16.9%; IIIa, 21.3%; IIIb, 15.0%; IVa, 7.9%; IVb, 7.1%; and V, 10.2%). Cumulative response rates at 3 years: 179/232 (77.2%) patients had a 2-point change in MG-ADL; 146/224 (65.2%) patients had a 3-point change in QMG. Mean (SD) changes in MG-ADL and QMG scores were -5.5 (3.7) and -7.7 (5.9), respectively. Mean (SD) changes in MG-ADL scores were -4.8 (3.0) and -11.7 (4.2) for patients with MGFA class IIa-IVb and MGFA class V, respectively. Similar reductions were seen for QMG. Crises decreased from 20.4/100 patient-years to 2.0/100 patient-years. Proportion of patients receiving IVIg and PLEX decreased from 65.4% to 12.8% and 24.8% to 2.1%, respectively. Concomitant oral corticosteroid use of ≤5 mg/day increased from 11.3% at baseline to 34.2% at cutoff. Among 264 patients in the safety analysis, 40.5% experienced adverse drug reactions (ADRs), and 22.7% had serious ADRs. No meningococcal infections were reported.

CONCLUSIONS: Consistent with previous real-world data, eculizumab treatment demonstrated sustained effectiveness regardless of MGFA classification and was well tolerated. No new safety signals were observed. Disclosure: Dr. Murai has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Alexion Pharmaceuticals. Dr. Murai has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for argenx. Dr. Murai has received personal compensation in the range of $500-$4,999 for serving as a Consultant for UCB. Dr. Murai has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Chugai Pharmaceutical. Dr. Murai has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Novartis. The institution of Dr. Murai has received research support from Japan Blood Products Organization. Dr. Mizuno has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Alexion pharma. Prof. Kondo has nothing to disclose. Mitsuru Watanabe has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Alexion Pharma, Mitsubishi Tanabe Pharma, UCB Japan. Mitsuru Watanabe has received personal compensation in the range of $5,000-$9,999 for serving on a Speakers Bureau for Alexion Pharma, Argenx, UCB Japan, Novartis Pharma, Biogen Japan, Mitsubishi Tanabe Pharma, Chugai Pharmaceutical, Viatris, and Daiichi Sankyo. The institution of Mitsuru Watanabe has received research support from Japan. Mitsuru Watanabe has received publishing royalties from a publication relating to health care. Masayuki Masuda has received personal compensation in the range of $0-$499 for serving on a Speakers Bureau for Alexion pharma, Argenix pharma, Aasahi kasei pharma, UCB, Takeda pharma. Dr. Takahashi has received personal compensation in the range of $500-$4,999 for serving as a Consultant for argenx. Dr. Takahashi has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Dyne Therapeutics. Dr. Takahashi has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Japan Clinical Research Operations. Dr. Takahashi has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Nippon Shinyaku. Dr. Takahashi has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Ono Pharmaceutical. Dr. Takahashi has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Alexion Pharmaceuticals. Dr. Takahashi has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Alexion Pharmaceuticals. Dr. Takahashi has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for argenx. Dr. Takahashi has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for UCB Japan. Dr. Takahashi has received personal compensation in the range of $0-$499 for serving on a Speakers Bureau for Myotonic Dystrophy Patients' Group of Japan. Dr. Shigeaki has nothing to disclose. Mr. Okuda has received personal compensation for serving as an employee of Alexion Pharma GK. Mr. Okuda has stock in Alexion Pharmaceuticals. Dr. Tezuka has received personal compensation for serving as an employee of Alexion pharma GK. Dr. Utsugisawa has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for argenx. Dr. Utsugisawa has received personal compensation in the range of $10,000-$49,999 for serving on a Scientific Advisory or Data Safety Monitoring board for UCB pharma. Dr. Utsugisawa has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Janssen pharma. Dr. Utsugisawa has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Viela Bio. Dr. Utsugisawa 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. Utsugisawa has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Mitsubisi Tanabe pharma. Dr. Utsugisawa has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Merck. Dr. Utsugisawa has received personal compensation in the range of $5,000-$9,999 for serving on a Speakers Bureau for argenx. Dr. Utsugisawa has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Alexion phama. Dr. Utsugisawa has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Japan Blood Products Organization. Dr. Utsugisawa has received personal compensation in the range of $5,000-$9,999 for serving on a Speakers Bureau for UCB. Disclaimer: Abstracts were not reviewed by Neurology® and do not reflect the views of Neurology® editors or staff.

PMID:40194101 | DOI:10.1212/WNL.0000000000208774


Source: https://pubmed.ncbi.nlm.nih.gov/4019410 ... t9+e462414