Intrathecal delivery of nusinersen in individuals with complicated spines

Cartwright, Michael S.; Ward, Zachary T.; White, E.P.; West, Thomas G.

doi:10.1002/mus.26899

Cited by 12 publications

(11 citation statements)

References 14 publications

(13 reference statements)

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“…For patients with type 1 SMA, life‐threatening respiratory symptoms, often requiring the presence of a multi‐disciplinary care team at the time of treatment administration, frequent sedation, and radiation exposure could cause concern. Spinal complications may limit the ability for a patient to receive nusinersen, although recent research has indicated that individuals with complicated spines can receive nusinersen injections using fluoroscopic guidance 20 In our study, however, the route of administration was noted as the primary reason for not being able to receive nusinersen treatment among both adult and pediatric patients.…”

Section: Discussioncontrasting

confidence: 55%

Early experiences of nusinersen for the treatment of spinal muscular atrophy: Results from a large survey of patients and caregivers

Er¹,

Dixon

Naik³

et al. 2020

Muscle and Nerve

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Background This study aimed to examine the early experience of nusinersen for spinal muscular atrophy (SMA) from the patient and caregiver perspective. Methods A 54‐item online survey was administered to adult patients and caregivers of pediatric patients diagnosed with SMA. Results Overall, respondents (56 patients and 45 caregivers) were satisfied with nusinersen. Satisfaction was highest on changes in energy, stamina, and motor function and lowest on treatment administration and overall time commitment. Differences were noted for treatment effect sustained over time as reported by adult patients vs caregivers reporting on behalf of pediatric patients. Respondents reported insurance approval as a key barrier to access, particularly among adult patients. Conclusions Despite therapeutic advances, there remain significant unmet needs for SMA. Challenges with administration and barriers to access potentially limit the number of patients treated or delay treatment. Continued efforts are needed to develop more treatment options and to improve access to treatments.

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Section: Discussioncontrasting

confidence: 55%

Early experiences of nusinersen for the treatment of spinal muscular atrophy: Results from a large survey of patients and caregivers

Er¹,

Dixon

Naik³

et al. 2020

Muscle and Nerve

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“…18 In patients with complicated spines, several centers have reported successful intrathecal access using radiologic guidance or reservoir placement. [19][20][21][22] We successfully performed conventional, palpation-guided lumbar puncture for the majority of injections in patients with complicated spines. In the remainder, all were successfully performed using fluoroscopy-guided interlaminar lumbar punctures.…”

Section: Discussionmentioning

confidence: 99%

Laboratory monitoring of nusinersen safety

et al. 2021

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Introduction: This retrospective study reports our tertiary care center's experience with intrathecal nusinersen administration in children and adults with spinal muscular atrophy (SMA). Methods: We reviewed safety monitoring laboratory results and need for procedural sedation and fluoroscopy-guidance in all SMA patients receiving nusinersen between February 2017 and March 2020. Results: Fifty-eight patients ages 1 mo-56 y received 494 nusinersen doses. There were 166 laboratory abnormalities in 45 patients. Most were either mild (145 [87.3%]) or were transient proteinuria (18 [10.8%]). None altered nusinersen treatment. Twenty-eight patients required either general anesthesia (75 doses) or anxiolysis with oral midazolam (133 doses, including 6 patients [23 doses] with SMA type I). Eight patients with complicated spines (45 doses) required fluoroscopic guidance. One treatment-related serious adverse event (emesis leading to intubation) occurred during general anesthesia. Two children had asymptomatic increased intracranial pressure. No patients discontinued treatment due to adverse events.Discussion: Intrathecal nusinersen is generally safe and well-tolerated, including in patients requiring oral anxiolysis, general sedation, and fluoroscopic guidance. Frequent serial laboratory monitoring did not identify any persistent significantly abnormal findings or alter treatment.

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“…Lakhotia et al [ 17 ] presented the successful, uncomplicated, off-label use of the Ommaya reservoir in a patient with SMA type 2 and concomitant scoliosis with spinal fusion. A recent retrospective cohort on intrathecal methods of nusinersen delivery in 1 institution reported that 22% of injections in patients with complicated spines were performed through Ommaya reservoirs placed in the lumbar region [ 18 ]. This cohort included both adults and children with SMA types 1–3.…”

Section: Discussionmentioning

confidence: 99%

Intrathecal Administration of Nusinersen Using the Ommaya Reservoir in an Adult with 5q-Related Spinal Muscular Atrophy Type 1 and Severe Spinal Deformity

et al. 2021

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Spinal muscular atrophy (SMA) is a hereditary neuromuscular disorder, typically caused by survival motor neuron 1 (SMN1) gene deletion in chromosome 5q resulting in loss of SMN protein. SMA type 1 progresses rapidly leading to increased mortality usually before the age of 2 years. Nusinersen, the first approved disease-modifying treatment for all 5q-SMA types and ages, is an antisense oligonucleotide administered intrathecally via repeated lumbar punctures. However, adult SMA patients typically present with severe scoliosis and spinal deformity. We present a 28-year-old patient with SMA type 1 and severe spinal deformity, who received nusinersen via a subcutaneously implanted Ommaya reservoir connected with an intrathecal catheter at the thoracic level. The repetitive administrations were completed uneventfully, obviating the need for repeated laborious lumbar punctures and eliminating radiation exposure. In adult SMA patients, performing recurrent lumbar punctures can be technically challenging raising the need for an alternative route of administration. The use of Ommaya reservoirs is a viable, practical for repeated infusions, and safe option for the intrathecal delivery of nusinersen for select cases such as an adult SMA type 1 survivor with severe spinal deformity.

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Intrathecal delivery of nusinersen in individuals with complicated spines

Cited by 12 publications

References 14 publications

Early experiences of nusinersen for the treatment of spinal muscular atrophy: Results from a large survey of patients and caregivers

Early experiences of nusinersen for the treatment of spinal muscular atrophy: Results from a large survey of patients and caregivers

Laboratory monitoring of nusinersen safety

Intrathecal Administration of Nusinersen Using the Ommaya Reservoir in an Adult with 5q-Related Spinal Muscular Atrophy Type 1 and Severe Spinal Deformity

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