Efficacy and Safety of Efgartigimod in the Treatment of Amyotrophic Lateral Sclerosis (ALS): A Retrospective Cohort Study
Xiaoli Yao1, Youna Xie1, Sen Huang1, Mingying Zheng1, Xinyu Yang1, Zhanhua Zhong1, Yuxin Wang1
1Department of Neurology, The First Affiliated Hospital, Sun Yat-sen University; Guangdong Provincial Key Laboratory of Diagnosis and Treatment of Major Neurological Diseases; National Key Clinical Department and Key Discipline of Neurology, No.58 Zhongshan Road 2, Guangzhou, 510080, China
Objective:
To evaluate the efficacy and safety of efgartigimod in the treatment of ALS in China.
Background:
Growing evidence indicates the presence of pathogenic antibodies in the serum or cerebrospinal fluid of ALS patients, although the precise mechanism remains unknown. Antibody clearance therapy could potentially improve symptoms in ALS patient
Design/Methods:
In this retrospective study, we analyzed data from 78 patients diagnosed as ALS who were admitted to the Department of Neurology at the First Affiliated Hospital of Sun Yat-sen University from January to August 2024. Each patients received at least one injection of efgartigimod (10 mg/kg) and their ALSFRS-R scores before and after treatments were recorded.
Results:
A total of 58 patients had their ALSFRS-R scores recorded both at baseline and after treatment, including 19 with bulbar-onset ALS and 39 with spinal-onset ALS. The baseline ALSFRS-R scores ranged from 15 to 45. A majority (70.6%) of the patients received a minimum of two injections of efgartigimod. The mean follow-up period was 8 weeks. Improvements in ALSFRS-R scores were observed in 46.5% (27/58) of the patients, while 24.1% (14/58) exhibited stable scores. The overall response rate was 70.6%, with comparable rates between bulbar-onset and spinal-onset ALS, although the improvement rate was notably higher in spinal-onset ALS. Long-term treatment with efgartigimod appears to slow the progression of ALS without additional adverse events. Further detailed data will be presented at the conference.
Conclusions:
In this study, we observed the certain efficacy and safety of efgartigimod in treatment of ALS. The study is still in progress, with ongoing collection of efficacy and safety data from a larger cohort. We intend to further investigate the relationship between patients' antibody test results and the outcomes.
Disclaimer: Abstracts were not reviewed by Neurology® and do not reflect the views of Neurology® editors or staff.