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2016 ; 31
(7
): 899-906
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Intrathecal Injections in Children With Spinal Muscular Atrophy: Nusinersen
Clinical Trial Experience
#MMPMID26823478
Haché M
; Swoboda KJ
; Sethna N
; Farrow-Gillespie A
; Khandji A
; Xia S
; Bishop KM
J Child Neurol
2016[Jun]; 31
(7
): 899-906
PMID26823478
show ga
Nusinersen (ISIS-SMNRx or ISIS 396443) is an antisense oligonucleotide drug
administered intrathecally to treat spinal muscular atrophy. We summarize lumbar
puncture experience in children with spinal muscular atrophy during a phase 1
open-label study of nusinersen and its extension. During the studies, 73 lumbar
punctures were performed in 28 patients 2 to 14 years of age with type 2/3 spinal
muscular atrophy. No complications occurred in 50 (68%) lumbar punctures; in 23
(32%) procedures, adverse events were attributed to lumbar puncture. Most common
adverse events were headache (n = 9), back pain (n = 9), and post-lumbar puncture
syndrome (n = 8). In a subgroup analysis, adverse events were more frequent in
older children, children with type 3 spinal muscular atrophy, and with a 21- or
22-gauge needle compared to a 24-gauge needle or smaller. Lumbar punctures were
successfully performed in children with spinal muscular atrophy; lumbar
puncture-related adverse event frequency was similar to that previously reported
in children.