PMID- 35787224
OWN - NLM
STAT- MEDLINE
DCOM- 20240205
LR  - 20240206
IS  - 1563-5279 (Electronic)
IS  - 0020-7454 (Linking)
VI  - 134
IP  - 2
DP  - 2024 Jun
TI  - Real-world safety and effectiveness of nusinersen, a treatment for spinal 
      muscular atrophy, in 401 Japanese patients: results from an interim analysis of 
      post-marketing surveillance.
PG  - 153-162
LID - 10.1080/00207454.2022.2095270 [doi]
AB  - Purpose: Nusinersen is an antisense oligonucleotide 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 received intrathecal 
      nusinersen in real-world clinical settings. We report the interim analysis 
      results of safety and effectiveness.Methods: This interim analysis was conducted 
      using data collected from 401 patients whose case report forms were obtained at 
      least once by 30 May 2020. 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: All 401 
      patients were diagnosed with SMA and were included in the safety and 
      effectiveness analysis (infantile-onset SMA [n = 126, 31.4%], later-onset SMA 
      [n = 275, 68.6%]). The median duration of treatment was 330 days (range 
      1-823 days). The incidence proportion of AEs was 31.7% (37.3% in infantile-onset 
      SMA and 29.1% in later-onset SMA). The most common AEs were headache (4.5%), 
      pyrexia (4.2%), and pneumonia (3.7%). The incidence proportion of serious AEs was 
      11.5%. Nusinersen improved motor function scores and was assessed as 'effective' 
      based on CGI-I in 99.7-100% of patients.Conclusions: This interim analysis of the 
      PMS in Japanese patients treated with nusinersen found no new safety concerns, 
      with the type of AEs consistent with the expected safety profile. 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 - Takasaki, Sakura
AU  - Takasaki S
AUID- ORCID: 0000-0002-6393-7277
AD  - Biogen Japan, Tokyo, Japan.
FAU - Hoshino, Misuzu
AU  - Hoshino M
AUID- ORCID: 0000-0003-1429-9923
AD  - Biogen Japan, Tokyo, Japan.
FAU - Makioka, Haruki
AU  - Makioka H
AUID- ORCID: 0000-0002-8295-3723
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 - 20220727
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  - Japan
MH  - *Muscular Atrophy, Spinal/drug therapy
MH  - Oligonucleotides/adverse effects
MH  - *Spinal Muscular Atrophies of Childhood/drug therapy
MH  - Product Surveillance, Postmarketing
OTO - NOTNLM
OT  - Spinal muscular atrophy
OT  - effectiveness
OT  - nusinersen
OT  - post-marketing surveillance
OT  - safety
EDAT- 2022/07/06 06:00
MHDA- 2024/02/05 06:42
CRDT- 2022/07/05 17:14
PHST- 2024/02/05 06:42 [medline]
PHST- 2022/07/06 06:00 [pubmed]
PHST- 2022/07/05 17:14 [entrez]
AID - 10.1080/00207454.2022.2095270 [doi]
PST - ppublish
SO  - Int J Neurosci. 2024 Jun;134(2):153-162. doi: 10.1080/00207454.2022.2095270. Epub 
      2022 Jul 27.