PMID- 32935160
OWN - NLM
STAT- MEDLINE
DCOM- 20210618
LR  - 20210618
IS  - 1432-1459 (Electronic)
IS  - 0340-5354 (Linking)
VI  - 268
IP  - 3
DP  - 2021 Mar
TI  - Nusinersen treatment significantly improves hand grip strength, hand motor 
      function and MRC sum scores in adult patients with spinal muscular atrophy types 
      3 and 4.
PG  - 923-935
LID - 10.1007/s00415-020-10223-9 [doi]
AB  - BACKGROUND: Nusinersen recently became available as the first treatment for 
      Spinal Muscular Atrophy (SMA) and data on its effectiveness and safety in adult 
      SMA patients are still scarce. METHODS: We evaluated the effectiveness and safety 
      of nusinersen treatment during 14 months in 16 adult patients with SMA types 3 
      and 4 in a prospective study, and retrospectively detailed the natural history of 
      48 adult SMA patients types 2, 3 and 4. RESULTS: Hand grip strength (p = 0.03), 
      hand motor function (p = 0.04) as assessed by a sub-score of the Revised Upper 
      Limb Module (RULM) and the Medical Research Council (MRC) sum score (p = 0.04) 
      improved significantly at month 14. Importantly, the MRC sum score had declined 
      significantly (p < 0.01) prior to start of treatment in these patients. A minimal 
      clinically important difference (MCID) in the Hammersmith Functional Motor Scale 
      Expanded (HFMSE) and RULM scores was achieved in 31% and 50% of the patients, 
      respectively, but the mean changes from baseline failed to reach significance. 
      Forced Vital Capacity (FVC) transiently increased at month 6 (p = 0.01), whereas 
      the Peak Expiratory Flow (PEF) did not. The Activity Limitations scale declined 
      significantly prior to start of treatment (p < 0.01) and showed an improvement 
      with nusinersen which was not significant. The safety evaluation did not reveal 
      serious adverse events and no signs of nephrotoxicity or antisense 
      oligonucleotide (ASO)-mediated inflammation. CONCLUSIONS: We conclude that hand 
      grip strength and hand motor function, as well as MRC sum scores improved 
      significantly in nusinersen-treated adult patients with SMA types 3 and 4.
FAU - De Wel, Bram
AU  - De Wel B
AD  - Department of Neurology, University Hospitals Leuven, Campus Gasthuisberg, 
      Herestraat 49, 3000, Leuven, Belgium.
AD  - Laboratory for Muscle Diseases and Neuropathies, Department of Neurosciences, KU 
      Leuven, Leuven, Belgium.
FAU - Goosens, Veerle
AU  - Goosens V
AD  - Department of Radiology, University Hospitals Leuven, Leuven, Belgium.
FAU - Sobota, Atka
AU  - Sobota A
AD  - Department of Physical and Rehabilitation Medicine (Physiotherapy), University 
      Hospitals Leuven, Leuven, Belgium.
FAU - Van Camp, Elke
AU  - Van Camp E
AD  - Department of Physical and Rehabilitation Medicine (Physiotherapy), University 
      Hospitals Leuven, Leuven, Belgium.
FAU - Geukens, Ellen
AU  - Geukens E
AD  - Department of Physical and Rehabilitation Medicine (Occupational Therapy), 
      University Hospitals Leuven, Leuven, Belgium.
FAU - Van Kerschaver, Griet
AU  - Van Kerschaver G
AD  - Department of Physical and Rehabilitation Medicine (Occupational Therapy), 
      University Hospitals Leuven, Leuven, Belgium.
FAU - Jagut, Marlene
AU  - Jagut M
AD  - Belgian Neuromuscular Diseases Registry, Sciensano, Brussels, Belgium.
FAU - Claes, Kathleen
AU  - Claes K
AD  - Department of Nephrology, University Hospitals Leuven, Leuven, Belgium.
AD  - Department of Microbiology, Immunology and Transplantation, Nephrology and Renal 
      Transplantation Research Group, KU Leuven, Leuven, Belgium.
FAU - Claeys, Kristl G
AU  - Claeys KG
AUID- ORCID: 0000-0001-9937-443X
AD  - Department of Neurology, University Hospitals Leuven, Campus Gasthuisberg, 
      Herestraat 49, 3000, Leuven, Belgium. Kristl.Claeys@uzleuven.be.
AD  - Laboratory for Muscle Diseases and Neuropathies, Department of Neurosciences, KU 
      Leuven, Leuven, Belgium. Kristl.Claeys@uzleuven.be.
LA  - eng
PT  - Journal Article
DEP - 20200915
PL  - Germany
TA  - J Neurol
JT  - Journal of neurology
JID - 0423161
RN  - 0 (Oligonucleotides)
RN  - 5Z9SP3X666 (nusinersen)
SB  - IM
MH  - Adult
MH  - Hand Strength
MH  - Humans
MH  - *Muscular Atrophy, Spinal/drug therapy
MH  - Oligonucleotides
MH  - Prospective Studies
MH  - Retrospective Studies
MH  - *Spinal Muscular Atrophies of Childhood/drug therapy
OTO - NOTNLM
OT  - ActivLim
OT  - Muscle strength
OT  - Natural history
OT  - Outcome measures
OT  - SMA
OT  - Spinraza(R)
EDAT- 2020/09/17 06:00
MHDA- 2021/06/22 06:00
CRDT- 2020/09/16 05:40
PHST- 2020/07/15 00:00 [received]
PHST- 2020/09/08 00:00 [accepted]
PHST- 2020/08/30 00:00 [revised]
PHST- 2020/09/17 06:00 [pubmed]
PHST- 2021/06/22 06:00 [medline]
PHST- 2020/09/16 05:40 [entrez]
AID - 10.1007/s00415-020-10223-9 [pii]
AID - 10.1007/s00415-020-10223-9 [doi]
PST - ppublish
SO  - J Neurol. 2021 Mar;268(3):923-935. doi: 10.1007/s00415-020-10223-9. Epub 2020 Sep 
      15.