Administration Practice and Adherence of Nusinersen in Pediatric Patients with 5q-Spinal Muscular Atrophy in China


Clinical Trials

Poster Number: T387


Jing Peng, Xiangya Hospital Central South University, Xiaoli Yao, The First Affiliated Hospital, Sun Yat-sen University, Rong Luo, West China Second University Hospital, Sichuan University, Xiuxia Wang, The Second Hospital of Hebei Medical University, Liwen Wu, Hunan Children's Hospital, Jianmin Zhong, Jiangxi Provincial Children's Hospital, Ruifeng Jin, Children's Hospital Affiliated to Shandong University, Xinguo Lu, Shenzhen 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, Zhe Tao, Dalian Women and Children's Medical Group, Jun Hu, Fujian Medical University Union Hospital, 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 investigate the administration practice and adherence of nusinersen in Chinese pediatric patients with 5q-SMA.
Methods: The present cohort study utilized data from a longitudinal, multicenter registry enrolling pediatric patients with 5q-SMA in China. Nusinersen administration data, including administration date, care setting, use of sedation and general anesthesia, method of administration, and use of imaging guide before administration, were collected both retrospectively and prospectively. Nusinersen was scheduled to be administered on Day 0, 14, 28, 63, and every 4 months thereafter. Adherence rate was calculated at dose level. A dose was considered adherent if the inter-dose interval between the current and previous dose followed the standard dosing regimen, with a grace period of ±7 days for Dose 2 to 4 and ±28 days for doses thereafter.
Results: A total of 385 patients receiving nusinersen with a total of 2,415 doses were included in the study. The median (range) number of doses administered per patient was 6 (3-14). The majority of injections (>99%) were given in inpatient setting. Few injections (n=3, 0.1%) required general anesthesia, while limited doses (n=217, 9.0%) were administered with sedation use. Interlaminar lumbar puncture (n=2,407, 99.7%) was the most common method of administration, followed by cervical puncture (n=5, 0.2%) and transforaminal lumbar puncture (n=3, 0.1%). Over 90% of injections did not utilize any imaging guide prior to administration. Among all imaging guides, ultrasound (n=142, 5.9%) was most commonly used. The adherence rate was 95.7% (1,943/2,030). The median inter-dose intervals aligned well with dosing schedule, with 14 days for Dose 2 and 3, 35 days for Dose 4, and 114-124 days for maintenance doses thereafter.
Conclusions: Findings from our study characterized the patterns of nusinersen administration practice and demonstrated high real-world adherence to nusinersen administration in Chinese pediatric patients with 5q-SMA.
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: Nusinersen; Pediatric; Registry; Adherence; Administration practice