Effectiveness of nusinersen among adults with Type II & III 5q-spinal muscular atrophy: a multicenter disease registry in China

Topic:

Clinical Trials

Poster Number: P1

Author(s):

Yi Dai, Chinese Academy of Medical Sciences & Peking Union Medical College Hospital, Xiaoli Yao, The First Affiliated Hospital, Sun Yat-sen University, Wenhua Zhu, Huashan Hospital, Fudan University, Yuying Zhao, Qilu Hospital of Shandong University, Huifang Shang, West China Hospital, Sichuan University, Juanjuan Chen, Peking University Shenzhen Hospital, Qiang Meng, The First People’s Hospital of Yunnan Province, Yaling Liu, The Second Hospital of Hebei Medical University, Wanjin Chen, The First Affiliated Hospital of Fujian Medical University, Liqiang Yu, The First Affiliated Hospital of Soochow University, Lu Shen, Xiangya Hospital Central South University, Daojun Hong, The First Affiliated Hospital of Nanchang University, Ken Chen, IQVIA Solutions Enterprise Management Consulting (Shanghai) Co., Ltd, Li Zhang, Biogen Biotechnology (Shanghai) Co., Ltd, Maerhaba Mai, Biogen Biotechnology (Shanghai) Co., Ltd, Liying Cui, Chinese Academy of Medical Sciences & Peking Union Medical College Hospital

Liying Cui is corresponding author.
Objectives
To evaluate the effectiveness of nusinersen among Chinese adults with Type II & III 5q-spinal muscular atrophy (SMA).

Methods
This analysis leveraged a longitudinal, multicenter disease registry (prospective and retrospective) including data from adults with 5q-SMA in China. Responses to treatment regarding motor measures were assessed at 6 and 14 months among adults with Type II & III 5q-SMA receiving nusinersen as their first disease modifying therapy.

Results
Among 168 adults with Type II (n=37) and III (n=131) 5q-SMA, 59.5% (100/168) were male. The median age at nusinersen initiation was 23 years for Type II and 26 years for Type III and did not correlate with baseline motor measures.
For Hammersmith Functional Motor Scale Expanded (HFMSE), the percentage of improvement (≥3 points) was 51.7% (15/29) and 58.3% (14/24) among Type III walkers, 52.0% (13/25) and 40.0% (6/15) among Type III sitters, and 0.0% (0/2) and 0.0% (0/2) among Type II sitters at 6 and 14 months, respectively.
For Revised Upper Limb Module (RULM), the percentage of improvement (≥2 points) was 15.0% (3/20) and 30.8% (4/13) among Type III walkers, 33.3% (8/24) and 40.0% (6/15) among Type III sitters, 33.3% (1/3) and 0.0% (0/3) among Type III non-sitters, 83.3% (5/6) and 60.0% (3/5) among Type II sitters, and 44.4% (4/9) and 62.5% (5/8) among Type II non-sitters at 6 and 14 months, respectively.
For 6-Minute Walk Test (6-MWT), the percentage of improvement (≥30 meters) was 53.8% (7/13) and 38.5% (5/13) among Type III walkers at 6 and 14 months, respectively.
The overall responder (improvement of at least 1 scale) rate of Type III walkers, Type III sitters and Type II sitters was 73.1%, 68.8% and 60.0% at 14 months, respectively.

Conclusions
The findings indicate improvements in motor measures among Chinese adults with Type II & III 5q-SMA treated with nusinersen.

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. LZ and MM are employees of Biogen and may hold stock. KC is an employee of IQVIA.

Key words: Spinal muscular atrophy; Nusinersen; Adult; Registry