Nusinersen effectiveness in children <5 and ≥5 years with Type II/III 5q-spinal muscular atrophy: 3rd interim analysis from a Chinese registry

Topic:

Clinical Trials

Poster Number: 201 M

Author(s):

Hanbing Lu, Xuzhou Central Hospital, Xiuxia Wang, The Second Hospital of Hebei Medical University, 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, Jianmin Liang, The First Hospital of Jilin University, Jianmin Zhong, Jiangxi Provincial Children’s Hospital, Lin Yang, The Second Affiliated Hospital of Xi’An Jiaotong University, Ruifeng Jin, Children’s Hospital Affiliated to Shandong University, Yiyan Ruan, Maternity and Child Health Care of Guangxi Zhuang Autonomous Region, Xiaoli Zhang, The Third Affiliated Hospital of Zhengzhou University, Xufeng Luo, West China Second University Hospital, Siqi Hong, MD, Children’s Hospital of Chongqing Medical University, Shanshan Mao, Children’s Hospital Zhejiang University School of Medicine, Jihua Wu, Anhui Children’s Hospital, Liwen Wu, Hunan Children’s Hospital, Hua Wang, Shengjing Hospital of China Medical University, Dan Sun, Wuhan Children’s Hospital, Tongji Medical College Huazhong University of Science & Technology, Jun Hu, Fujian Medical University Union Hospital, Zhe Tao, Dalian Women and Children’s Medical Group, Guanglu Yang, The Affiliated Hospital of Inner Mongolia Medical University, Hui Xiong, MD, PhD, Children’s Medical Center, Peking University First Hospital, Lanlan Meng, Biogen Biotechnology (Shanghai) Co., Ltd, Li Zhang, MD, Biogen Biotechnology (Shanghai) Co., Ltd, Junli Zhu, IQVIA Solutions Enterprise Management Consulting (Shanghai) Co., Ltd, Yi Wang, Children’s Hospital of Fudan University

Yi Wang is corresponding author.

Background and objective: To assess the effectiveness of nusinersen in children with Type II/III 5q-spinal muscular atrophy (SMA) across age group <5 and ≥5 years. Methods: A multicenter, longitudinal registry was established to collect data retrospectively and prospectively from children with 5q-SMA across 25 sites in China. Motor function was assessed at baseline and at 14, 26, and 38 months using the Hammersmith Functional Motor Scale Expanded (HFMSE) and Revised Upper Limb Module (RULM). Analyses were stratified by age (<5 and ≥5 years) at treatment initiation. Results: As of June 11th, 2025, a total of 511 participants with Type II/III SMA were analyzed, of whom 189 initiated nusinersen at age <5 and 322 at age ≥5 years. Among <5-year participants with available data, for HFMSE, 72%, 21%, and 7% showed improvement, stability, and worsening, respectively, from 14 to 26 months, and 40%, 50%, and 10% from 26 to 38 months. For RULM, 82%, 14%, and 4% showed improvement, stability, and worsening from 14 to 26 months, and 50%, 25%, and 25% from 26 to 38 months. At 38 months, the mean (standard deviation, SD) score changes in HFMSE and RULM from baseline were 10.3 (6.68) and 14.3 (10.94), respectively. As for ≥5-year, for HFMSE, 24%, 68%, and 8% showed improvement, stability, and worsening, respectively, from 14 to 26 months, and 17%, 60%, and 23% from 26 to 38 months. For RULM, 28%, 52%, and 20% showed improvement, stability, and worsening from 14 to 26 months, and 30%, 52%, and 18% from 26 to 38 months. At 38 months, the mean (SD) score changes in HFMSE and RULM from baseline were 2.9 (5.78) and 4.0 (4.16), respectively. Conclusion: Advancing beyond previous interim analysis, the findings indicate nusinersen continues showing improvements in motor function among children of both <5- and ≥5-year groups. 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. Lanlan MENG and Li ZHANG are employees of Biogen and may hold stock in Biogen. Junli ZHU is an employee of IQVIA. Key words: Spinal muscular atrophy; Nusinersen; Pediatric; Registry