TY - JOUR
T1 - Intrathecal Injections in Children with Spinal Muscular Atrophy
T2 - Nusinersen Clinical Trial Experience
AU - Haché, Manon
AU - Swoboda, Kathryn J.
AU - Sethna, Navil
AU - Farrow-Gillespie, Alan
AU - Khandji, Alexander
AU - Xia, Shuting
AU - Bishop, Kathie M.
N1 - Publisher Copyright:
© SAGE Publications.
PY - 2016/6
Y1 - 2016/6
N2 - 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.
AB - 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.
KW - antisense oligonucleotide
KW - drug delivery
KW - lumbar puncture
KW - spinal muscular atrophy
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U2 - 10.1177/0883073815627882
DO - 10.1177/0883073815627882
M3 - Article
C2 - 26823478
AN - SCOPUS:84969287518
SN - 0883-0738
VL - 31
SP - 899
EP - 906
JO - Journal of child neurology
JF - Journal of child neurology
IS - 7
ER -