Nusinersen effectiveness and safety in pediatric patients with 5q-spinal muscular atrophy: a multicenter disease registry in China

Topic:

Clinical Trials

Poster Number: T388

Author(s):

Xiaoli Yao, The First Affiliated Hospital, Sun Yat-sen University, Jing Peng, Xiangya Hospital Central South University, Rong Luo, West China Second University Hospital, Sichuan University, Xiuxia Wang, The Second Hospital of Hebei Medical University, Xinguo Lu, Shenzhen Children's Hospital, Liwen Wu, Hunan Children's Hospital, Ruifeng Jin, Children's Hospital Affiliated to Shandong University, Jianmin Zhong, Jiangxi Provincial Children's Hospital, Jianmin Liang, The First Hospital of Jilin University, Siqi Hong, MD, Children's Hospital of Chongqing Medical University, Chong Qing, China, Lin Yang, The Second Affiliated Hospital of Xi’An Jiaotong University, Xiaoli Zhang, The Third Affiliated Hospital of Zhengzhou University, Shanshan Mao, Children's Hospital Zhejiang University School of Medicine, Jun Hu, Fujian Medical University Union Hospital, Zhe Tao, Dalian Women and Children's Medical Group, Dan Sun, Wuhan Children’s Hospital, Tongji Medical College Huazhong University of Science & Technology, Hua Wang, Shengjing Hospital of China Medical University, Li Zhang, MD, Biogen, Yanyan Xia, Biogen, Ken Chen, IQVIA Solutions Enterprise Management Consulting (Shanghai) Co., Ltd, Yi Wang, Children’s Hospital of Fudan University

Yi Wang is corresponding author.
Background and Objectives: To evaluate the effectiveness and safety of nusinersen for the treatment of 5q-spinal muscular atrophy (SMA) among Chinese pediatric patients in a real-world setting.
Methods: Using a longitudinal, multicenter registry, both prospective and retrospective data were collected from pediatric patients with 5q-SMA who received nusinersen treatment across 18 centers in China. Response to treatment regarding motor function measures was assessed at 6, 10, and 14 months post-treatment initiation by SMA type. Safety profile was evaluated among patients starting nusinersen treatment after enrollment into the registry.
Results: As of March 2nd, 2023, a total of 385 patients with nusinersen treatment were included in the registry. Patients predominantly demonstrated improvement or stability in motor function across all SMA types. For type I, the mean change from baseline at months 6, 10, and 14 was 6.7, 13.5, 14.3 in Children’s Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP-INTEND) and 0.6, 1.3, 4.0 in Hammersmith Infant Neurological Exam–Part 2 (HINE-2) scores. Type II patients demonstrated mean changes of 4.4, 4.1, 5.1 in Hammersmith Functional Motor Scale–Expanded (HFMSE) and 2.4, 2.3, 4.2 in Revised Upper Limb Module (RULM) scores at months 6, 10, and 14. Type III patients exhibited mean changes of 3.9, 4.3, 4.2 in HFMSE and 2.1, 1.5, -0.7 in RULM scores at months 6, 10, and 14. Two out of 132 patients experienced mild adverse events (aseptic meningitis and myalgia) considered to be related to nusinersen, resulting in no sequelae. One patient died from asphyxia unrelated to nusinersen.
Conclusion: Aligning with previous findings, these data demonstrate that nusinersen may lead to improvement or stability of motor function in Chinese pediatric patients with SMA in types I, II and III in a relatively short time in contrast to the natural course of the disease.
Disclaimer: This study was sponsored by Biogen. Writing and editorial support for the preparation of this abstract was provided by IQVIA; funding was provided by Biogen.
Key words: Spinal muscular atrophy; Nusinersen; Pediatric; Registry