Intrathecal administration of nusinersen for spinal muscular atrophy: report of three cases with severe spinal deformity

Nakao, S.; Yamada, Satoshi; Tsuda, Katsuya; Yokomizo, Taishi; Sato, Teruyuki; Tanoue, Shuichi; Hiraki, Teruyuki

doi:10.1186/s40981-020-00334-7

Cited by 3 publications

(3 citation statements)

References 12 publications

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“…Intrathecal (IT) access is commonly established using the landmark technique in normal subjects. However, patients with SMA are affected by severe scoliosis [ 3 – 5 ] and are often with vertebral instrumentation [ 3 , 4 ]. In those patients, landmarks for intrathecal access can be difficult to identify, and fluoloscopy and real-time computed tomography (CT)-guided puncture have been shown to help access an intrathecal space [ 6 , 7 ].…”

Section: Introductionmentioning

confidence: 99%

Ultrasound-assisted intrathecal injection of nusinersen in a patient with severe vertebral deformity: a case report

et al. 2020

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Background: Spinal muscular atrophy (SMA) is a mostly autosomal recessive genetic disease characterized by progressive muscle weakness from anterior horn degeneration. Nusinersen has recently been approved as a disease-modifying drug for SMA that needs to be administered intrathecally. Its injection is often associated with extreme difficulty since patients with SMA have severe vertebral deformity and may be with vertebral instrumentation. Case description: A 21-year-old female with type 2 SMA and spinal deformity underwent a series of intrathecal injections of nusinersen. The intrathecal injections have been safely and successfully done by using computed tomography imaging and ultrasonography-assisted technique. Conclusion: This the first report in which ultrasound-assisted technique has been used for the injection of nusinersen through a lumbar puncture in patients with severe spinal deformity. Use of preprocedural ultrasound imaging is highly recommended for treatments that repeatedly require intrathecal access.

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Section: Introductionmentioning

confidence: 99%

Ultrasound-assisted intrathecal injection of nusinersen in a patient with severe vertebral deformity: a case report

et al. 2020

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“…As reported, higher termination of dural sac in some SMA patients may limited the use of L5–S1 interspace for lumbar puncture [ 25 ]. In our cohort, attempt has been made to administrate nusinersen intrathecally via L5–S1 interval in 1 of the 2 patients with L5 as lowest instrumented vertebra but in vain.…”

Section: Discussionmentioning

confidence: 99%

Unilateral interlaminar fenestration on the convex side provides a reliable access for intrathecal administration of nusinersen in spinal muscular atrophy: a retrospective study

Wang,

Feng,

Jiao

et al. 2023

Orphanet J Rare Dis

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Background As the first gene therapy for spinal muscular atrophy (SMA), nusinersen is supposed to be administrated via intrathecal injection regularly for a lifetime. However, for SMA patients with severe spinal deformities, bony fusion following posterior spinal instrumentation sets great obstacles for the application of nusinersen. Therefore, efforts have been devoted to the exploration of appropriate approach for nusinersen administration. This study aims to evaluate the safety and reliability of unilateral interlaminar fenestration on the convex side during spinal fusion surgery for intrathecal nusinersen injection in SMA. Results SMA patients receiving posterior spinal fusion and interlaminar fenestration in Peking Union Medical College Hospital from January 2020 to October 2021 were retrospectively analyzed. 13 patients were included. Of the 13 patients, 10 were classified into SMA type II and 3 into SMA type III. Distal fusion to pelvis was undertaken in 11 patients; while L5 was selected as the lowest instrumented vertebra in the other 2 patients. All patients received interlaminar fenestration on the convex side only with an area of about 15 mm × 20 mm. Fenestration at L2–L3 level was performed in 6 patients; while L3–L4 level was selected for windowing in the remaining 7 patients. 9 of the 13 patients received lumbar puncture and intrathecal nusinersen administration during the 1-year follow-up, with an accumulative total of 50 times. All injections were performed successfully under ultrasound guidance, with no one transferred to radiographic assistance. No severe complications occurred after injection. Conclusions In SMA with severe scoliosis planning to receive posterior spinal fusion, unilateral lumbar interlaminar fenestration on the convex side provides a feasible and reliable access for intrathecal nusinersen administration after surgery.

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“…Cartwright MS et al also reported only 74% of patients with SMA and complicated spines were injected via lumbar puncture under fluoroscopic guidance, and others required alternative strategies for intrathecal injection of Nusinersen 10) . In some SMA patients with spinal deformity, the dural sac sometimes terminates at around the level of L 5, which is considered higher than normal 11) . Therefore, an intrathecal approach is considered difficult at L5/S1 level in patients with spinal deformity.…”

Section: Discussionmentioning

confidence: 99%

Posterior Spinal Correction and Fusion Surgery in Patients with Spinal Muscular Atrophy-Associated Scoliosis for Whom Treatment with Nusinersen Was Planned

Machida

Miyagi

Saito

et al. 2021

Spine Surg Relat Res

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Introduction Spinal muscular atrophy (SMA) is defined as a neuromuscular disorder induced by progressive weakness of the skeletal muscle and is usually accompanied by progressive spinal deformity including scoliosis. The newly developed Nusinersen, which is the first approved drug worldwide for SMA, requires accurate intrathecal injection, which is sometimes difficult in patients with severe spinal deformity. Technical Note For an accurate intrathecal approach in patients who have spinal fusion surgery to treat neuromuscular scoliosis, we have combined an L3 laminectomy with spinal correction and fusion surgery. Here, we review four cases of SMA in patients who underwent the additional L3 laminectomy during surgery to treat spinal scoliosis. A successful intrathecal approach was made using fluoroscopic guidance in all four patients, who were then administered with Nusinersen. Conclusions Our findings show that additional lumbar laminectomy during surgery for spinal scoliosis has effectively allowed for intrathecal injection of Nusinersen.

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Intrathecal administration of nusinersen for spinal muscular atrophy: report of three cases with severe spinal deformity

Cited by 3 publications

References 12 publications

Ultrasound-assisted intrathecal injection of nusinersen in a patient with severe vertebral deformity: a case report

Ultrasound-assisted intrathecal injection of nusinersen in a patient with severe vertebral deformity: a case report

Unilateral interlaminar fenestration on the convex side provides a reliable access for intrathecal administration of nusinersen in spinal muscular atrophy: a retrospective study

Posterior Spinal Correction and Fusion Surgery in Patients with Spinal Muscular Atrophy-Associated Scoliosis for Whom Treatment with Nusinersen Was Planned

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