Nusinersen effectiveness among adults with Type II & III 5q-spinal muscular atrophy after 26-month treatment: a multicenter disease registry in China

Topic:

Clinical Trials

Poster Number: 202 M

Author(s):

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

Wenhua Zhu and Yi Dai contributed equally as co-first authors. Liying Cui and Yuying Zhao contributed equally as co-corresponding authors.

Objectives
To evaluate the nusinersen effectiveness among Chinese adults with Type II & III 5q-spinal muscular atrophy (SMA) after 26-month treatment.

Methods
This analysis leveraged a longitudinal, multicenter disease registry (prospective and retrospective) of adults with 5q-SMA in China. Responses to treatment were assessed at 6, 14 and 26 months from nusinersen initiation (baseline) among adults with Type II & III 5q-SMA receiving nusinersen as their first disease modifying therapy.

Results
In total, 167 adults with Type II (n=34) and III (n=133) 5q-SMA were included. Type II adults exhibited mean (standard deviation (SD)) changes of 0.50 (0.837) (n=6), 0.40 (1.517) (n=5), 0.00 (0.000) (n=2) in Hammersmith Functional Motor Scale–Expanded (HFMSE) and 3.50 (4.338) (n=12), 3.58 (3.502) (n=12), 2.50 (4.764) (n=6) in Revised Upper Limb Module (RULM) from baseline at months 6, 14 and 26, respectively. Type III adults exhibited mean (SD) changes of 3.25 (4.436) (n=59), 3.51 (4.320) (n=45), 5.00 (3.893) (n=27) in HFMSE, 0.59 (3.272) (n=49), 1.11 (3.438) (n=37), 1.65 (3.638) (n=23) in RULM and 43.01 (38.579) (n=14), 22.98 (38.341) (n=18) and 42.67 (42.939) (n=6) meters in 6-minute Walk Test (6-MWT) from baseline at months 6, 14 and 26, respectively. The absolute mean (SD) Clinical Global Impression of Improvement (CGI-I) scores at 6, 14 and 26 months were 4.00 (0.000) (n=3), 3.88 (0.641) (n=8) and 3.44 (0.726) (n=9) for Type II, and 3.67 (0.985) (n=12), 3.33 (0.734) (n=27) and 3.50 (0.577) (n=52) for Type III, respectively. The overall responder (improvement of HFSME or RULM for sitters, and HFMSE, RULM or 6-MWT for walkers) rates of Type III walkers, Type III sitters and Type II sitters were 87.5% (14/16), 60.0% (9/15), and 75.0% (3/4) at month 26, respectively.

Conclusions
The findings indicate long-term benefits 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