PMID- 37649429
OWN - NLM
STAT- MEDLINE
DCOM- 20241209
LR  - 20241209
IS  - 1563-5279 (Electronic)
IS  - 0020-7454 (Linking)
VI  - 134
IP  - 11
DP  - 2024 Nov
TI  - Safety and effectiveness of nusinersen, a treatment for spinal muscular atrophy, 
      in 524 patients: results from an interim analysis of post-marketing surveillance 
      in Japan.
PG  - 1185-1197
LID - 10.1080/00207454.2023.2251662 [doi]
AB  - PURPOSE: Nusinersen is an antisense oligonucleotide approved for the treatment of 
      spinal muscular atrophy (SMA). A post-marketing surveillance (PMS) has been 
      ongoing (August 2017-August 2025) in all patients in Japan who were administered 
      nusinersen intrathecally in real-world clinical settings. We report the interim 
      analysis results for safety and effectiveness. METHODS: This interim analysis was 
      conducted using data collected from 524 patients whose case report forms were 
      obtained at least once by May 30, 2022. Collected data included patient 
      demographics and adverse events (AEs) for safety, and motor function assessments 
      and Clinical Global Impressions of Improvement (CGI-I) for effectiveness. 
      RESULTS: Of the 524 patients in the safety analysis set, 522 patients who were 
      diagnosed with SMA were included in the effectiveness analysis (infantile-onset 
      SMA [n = 153, 29.3%], later-onset SMA [n = 369, 70.7%]). The median duration of 
      treatment was 785.0 (range 1-1549) days. AEs occurred in 35.9% of patients (49.0% 
      in infantile-onset SMA and 30.6% in later-onset SMA). Nusinersen treatment 
      significantly improved Hammersmith Infant Neurological Examination scores in 
      patients with infantile-onset SMA and Hammersmith Functional Motor Scale-Expanded 
      scores in patients with later-onset SMA for up to nearly 3 years. Based on CGI-I 
      assessments, 98.5-100% of patients receiving nusinersen 'improved' or remain 
      'unchanged'. CONCLUSIONS: This interim analysis of the large-scale, all-case PMS 
      in patients who were administered nusinersen in Japan supports the safety and 
      effectiveness of nusinersen. The benefit-risk balance of nusinersen treatment 
      remains favorable.
FAU - Tachibana, Yosuke
AU  - Tachibana Y
AUID- ORCID: 0000-0001-5910-2296
AD  - Biogen Japan, Tokyo, Japan.
FAU - Sato, Ryusuke
AU  - Sato R
AUID- ORCID: 0000-0002-7826-1978
AD  - Biogen Japan, Tokyo, Japan.
FAU - Makioka, Haruki
AU  - Makioka H
AUID- ORCID: 0000-0002-8295-3723
AD  - Biogen Japan, Tokyo, Japan.
FAU - Hoshino, Misuzu
AU  - Hoshino M
AUID- ORCID: 0000-0003-1429-9923
AD  - Biogen Japan, Tokyo, Japan.
FAU - Jin, Mingshou
AU  - Jin M
AUID- ORCID: 0000-0002-6966-5989
AD  - Biogen Japan, Tokyo, Japan.
LA  - eng
PT  - Journal Article
DEP - 20230907
PL  - England
TA  - Int J Neurosci
JT  - The International journal of neuroscience
JID - 0270707
RN  - 5Z9SP3X666 (nusinersen)
RN  - 0 (Oligonucleotides)
SB  - IM
MH  - Humans
MH  - *Oligonucleotides/administration & dosage/pharmacology/adverse effects
MH  - Japan
MH  - *Product Surveillance, Postmarketing
MH  - Male
MH  - Female
MH  - Infant
MH  - *Muscular Atrophy, Spinal/drug therapy
MH  - Child, Preschool
MH  - Child
MH  - Adolescent
MH  - Adult
MH  - Young Adult
MH  - Treatment Outcome
MH  - Middle Aged
MH  - Injections, Spinal
OTO - NOTNLM
OT  - Spinal muscular atrophy
OT  - effectiveness
OT  - nusinersen
OT  - post-marketing surveillance
OT  - safety
EDAT- 2023/08/31 06:41
MHDA- 2024/12/09 17:33
CRDT- 2023/08/31 03:48
PHST- 2024/12/09 17:33 [medline]
PHST- 2023/08/31 06:41 [pubmed]
PHST- 2023/08/31 03:48 [entrez]
AID - 10.1080/00207454.2023.2251662 [doi]
PST - ppublish
SO  - Int J Neurosci. 2024 Nov;134(11):1185-1197. doi: 10.1080/00207454.2023.2251662. 
      Epub 2023 Sep 7.