scholarly journals Intrathecal nusinersen administration in adult spinal muscular atrophy patients with complex spinal anatomy

2020 ◽  
Vol 13 ◽  
pp. 175628641988761 ◽  
Author(s):  
Isabell Cordts ◽  
Paul Lingor ◽  
Benjamin Friedrich ◽  
Verena Pernpeintner ◽  
Claus Zimmer ◽  
...  
Keyword(s):  
Spinal Muscular Atrophy ◽  
Muscular Atrophy ◽  
Drug Efficacy ◽  
Intrathecal Administration ◽  
Common Adverse Event ◽  
Ct Guided ◽  
Severe Scoliosis ◽  
Delayed Treatment ◽  
Spine Deformities ◽  
Alternative Routes

Background: Intrathecal administration of nusinersen in adult spinal muscular atrophy (SMA) patients presents challenges owing to severe scoliosis and previous spinal surgery with metal implantation. In patients with a complex spinal situation, the potential risks of the intrathecal administration may lead to delayed treatment initiation. Methods: In this study, we analyzed 53 CT-guided lumbar punctures of 11 adult nonambulatory SMA type 2 and 3 patients. All patients had scoliosis and six patients had previously undergone metal implantation. Results: Drug administration was successful in 100% of the patients and none of the patients opted for treatment discontinuation. Complete osseous fusion precluded conventional posterior interlaminar access in eight lumbar punctures in four patients, which required alternative routes including transforaminal punctures and translaminar drilling. Median duration of all lumbar punctures was 9 min and median radiation exposure was 100 mGy* cm. The most common adverse event was post-lumbar puncture syndrome that occurred in five lumbar punctures (9.4%). Conclusions: Our data demonstrate that nusinersen can be successfully, safely, and rapidly administered in adult SMA patients with complex spinal conditions and suggest the translaminar drilling technique as an alternative delivery route. Therefore, intrathecal nusinersen treatment should not be withheld from patients because of severe spine deformities, however, drug efficacy in adult SMA patients needs to be investigated in further studies.

scholarly journals Experiences from treating seven adult 5q spinal muscular atrophy patients with Nusinersen

2020 ◽  
Vol 13 ◽  
pp. 175628642090780 ◽  
Author(s):  
Elisabeth Jochmann ◽  
Robert Steinbach ◽  
Thomas Jochmann ◽  
Ha-Yeun Chung ◽  
Annekathrin Rödiger ◽  
...  
Keyword(s):  
Spinal Muscular Atrophy ◽  
Muscular Atrophy ◽  
Drug Application ◽  
Case Series ◽  
Intrathecal Administration ◽  
Patient Reported Outcome ◽  
Ct Guided ◽  
Patient Reported ◽  
Severe Spinal Deformities ◽  
Short Time

Background: The antisense oligonucleotide Nusinersen recently became the first approved drug against spinal muscular atrophy (SMA). It was approved for all ages, albeit the clinical trials were conducted exclusively on children. Hence, clinical data on adults being treated with Nusinersen is scarce. In this case series, we report on drug application, organizational demands, and preliminary effects during the first 10 months of treatment with Nusinersen in seven adult patients. Methods: All patients received intrathecal injections with Nusinersen. In cases with severe spinal deformities, we performed computed tomography (CT)-guided applications. We conducted a total of 40 administrations of Nusinersen. We evaluated the patients with motor, pulmonary, and laboratory assessments, and tracked patient-reported outcome. Results: Intrathecal administration of Nusinersen was successful in most patients, even though access to the lumbar intrathecal space in adults with SMA is often challenging. No severe adverse events occurred. Six of the seven patients reported stabilization of motor function or reduction in symptom severity. The changes in the assessed scores did not reach a significant level within this short time period. Conclusions: Treating adult SMA patients with Nusinersen is feasible and most patients consider it beneficial. It demands a complex organizational and interdisciplinary effort. Due to the slowly decreasing motor functions in adult SMA patients, long observation phases for this recently approved treatment are needed to allow conclusions about effectiveness of Nusinersen in adults.

scholarly journals Ultra-low radiation dose protocol for CT-guided intrathecal nusinersen injections for patients with spinal muscular atrophy and severe scoliosis

2021 ◽  
Author(s):  
Grzegorz Rosiak ◽  
Anna Lusakowska ◽  
Krzysztof Milczarek ◽  
Dariusz Konecki ◽  
Anna Fraczek ◽  
...  
Keyword(s):  
Radiation Dose ◽  
Spinal Muscular Atrophy ◽  
Lumbar Puncture ◽  
Low Dose ◽  
Muscular Atrophy ◽  
Radiation Doses ◽  
Standard Protocol ◽  
Ct Guided ◽  
Low Radiation Dose ◽  
Severe Scoliosis

Abstract Purpose Intrathecal injection of nusinersen is an approved treatment of spinal muscular atrophy (SMA). CT-guided injection is a method of nusinersen administration in patients with severe scoliosis, in whom standard lumbar puncture is not feasible. The injections are repeated every 4 months for life, and accumulated radiation doses absorbed by the patient can increase the risk of cancer. In this study, we present the results of CT-guided intrathecal nusinersen injections with an ultra-low radiation dose protocol. Methods Eighteen patients (15 adults and three children) in whom standard lumbar puncture was not feasible due to severe scoliosis or spinal stabilization were included in this retrospective study. The first 23 injections were performed with a standard radiation dose protocol and the next 42 injections with an ultra-low-dose protocol. The radiation doses, measured as total dose length product (DLP), were acquired and compared between the protocols. Results Injections were successful in 100% of patients with both ultra-low-dose and standard protocols. The radiation dose, measured as DLP, was 111.2–1100.7 (Me = 248.1) mGy*cm for the standard protocol. For the ultra-low-dose protocol, the dose range was 5.0–54.4 (Me = 26.7) mGy*cm, which was significantly lower than with the standard protocol (p < 0.001, η2 = 0.67). Conclusion Radiation doses can be significantly decreased in the CT-guided injection of nusinersen. The proposed protocol allows for effective CT-guided intrathecal nusinersen administration in patients with SMA and severe scoliosis.

Computed tomography-guided transforaminal lumbar puncture using local anesthesia and a straight 22-gauge spinal needle for intrathecal nusinersen in adults: Findings in 77 procedures

2021 ◽  
pp. 159101992110328
Author(s):  
Mougnyan Cox ◽  
Kofi-Buaku Atsina ◽  
Preethi Ramchand ◽  
Jonathan Ji ◽  
Neda Sedora-Roman ◽  
...  
Keyword(s):  
Computed Tomography ◽  
Spinal Muscular Atrophy ◽  
Lumbar Puncture ◽  
Local Anesthesia ◽  
Muscular Atrophy ◽  
Intrathecal Administration ◽  
Spinal Needle ◽  
Technical Success ◽  
Computed Tomography Guidance ◽  
Blood Patch

Background Intrathecal nusinersen is the first Food and Drug Administration-approved treatment for spinal muscular atrophy. Reliable intrathecal access is critical for initial and maintenance therapy; however, this can be challenging in older patients with spinal muscular atrophy many of whom have had prior lumbar instrumentation and osseous fusion. Transforaminal lumbar punctures have emerged as a technique for intrathecal access that avoids the hazards of cervical punctures. We describe our technique for transforaminal lumbar punctures under computed tomography guidance using local anesthesia and a straight 22-gauge needle. Methods Following local institutional review board approval, medical records of all patients undergoing computed tomography-guided transforaminal lumbar puncture for intrathecal nusinersen injection were obtained and analyzed. The rate of technical success and immediate complications were recorded. Any delayed complications noted in a 3-day follow-up phone call and future office visit were also recorded. Data collation and analysis were performed using Excel. Results A total of 77 transforaminal lumbar punctures were performed with intrathecal administration of nusinersen, for a 100% technical success rate. Local anesthesia was used in 76 cases, with conscious sedation used in one case. General anesthesia was not used in any case. There were no major complications. One patient had a postdural puncture headache that resolved completely after a transforaminal epidural blood patch performed 4 days later. Conclusions Intrathecal administration of nusinersen is critical for treatment of patients with spinal muscular atrophy. Our described technique allows for reliable access to the intrathecal space using local anesthesia and a straight 22-gauge spinal needle under computed tomography guidance, and is easily reproducible.

Radiation exposure of image-guided intrathecal administration of nusinersen to adult patients with spinal muscular atrophy

2019 ◽  
Vol 61 (5) ◽  
pp. 565-574 ◽  
Author(s):  
D. Oldenburg ◽  
N. Guberina ◽  
B. Stolte ◽  
K. Kizina ◽  
E. Stenzel ◽  
...  
Keyword(s):  
Spinal Muscular Atrophy ◽  
Radiation Exposure ◽  
Muscular Atrophy ◽  
Intrathecal Administration ◽  
Adult Patients ◽  
Image Guided

Nusinersen in adult patients with spinal muscular atrophy

Neurology ◽  
2020 ◽  
Vol 95 (4) ◽  
pp. e413-e416 ◽  
Author(s):  
Orly Moshe-Lilie ◽  
Amy Visser ◽  
Nizar Chahin ◽  
Thomas Ragole ◽  
Diana Dimitrova ◽  
...  
Keyword(s):  
Side Effects ◽  
Spinal Muscular Atrophy ◽  
Muscular Atrophy ◽  
Treated Group ◽  
Adult Patients ◽  
Recurrent Pneumonia ◽  
Modest Improvement ◽  
Respiratory Impairment ◽  
Severe Scoliosis ◽  
Blood Patch

ObjectiveTo report our experience with adult patients with spinal muscular atrophy (SMA), some of whom were treated with nusinersen.MethodsWe reviewed charts of adult patients with SMA seen in our neuromuscular clinic between 2017 and 2019 and noted their demographics, clinical characteristics, treatment, and side effects.ResultsTwenty-two patients were included. Nine had type 2 and 13 type 3 SMA. Median age was 36 years (range 20–71). Most could not walk unassisted. Ten patients had significant respiratory impairment necessitating ventilation and 2 had tracheostomy. Seventeen had severe scoliosis. Ten patients were treated with nusinersen for 6–24 months (median 12 months), 3 of whom required bone laminectomy for intrathecal access. One developed bowel and bladder incontinence following the procedure. In the treated group, on average, % Medical Research Council change was 2.5% at 12 months and 3.9% at 24 months. Most untreated patients remained stable; 3 had slightly declined. Five treated patients reported subjective improvement. Treatment side effects included post lumbar puncture headache in 5 patients, 2 of whom needed blood patch, and 1 bacterial meningitis requiring inpatient treatment. Three patients stopped treatment after 12–24 months due to lack of improvement, recurrent pneumonia, or proteinuria.ConclusionSide effects of nusinersen can be serious. Whereas half of treated patients reported modest improvement in function, there were no significant objective changes, which may point largely to a placebo effect.Classification of evidenceThis study provides Class IV evidence that for some adult patients with SMA, nusinersen improves subjective function and causes serious adverse effects.

Intrathecal Administration of Nusinersen in Pediatric SMA Patients with and without Spine Deformities: Experiences and Challenges over 3 Years in a Single Center

2020 ◽  
Author(s):  
Jessika Johannsen ◽  
Deike Weiss ◽  
Friderike Schlenker ◽  
Michael Groth ◽  
Jonas Denecke
Keyword(s):  
Health Care ◽  
Antisense Oligonucleotide ◽  
Muscular Atrophy ◽  
Premature Death ◽  
Intrathecal Administration ◽  
Single Center ◽  
Application Form ◽  
Safety And Efficacy ◽  
Spine Deformities ◽  
Patient Will

AbstractSpinal muscular atrophy (SMA) is a rare neurodegenerative disease leading to progressive muscular atrophy, respiratory failure, and premature death. Secondary thorax and spine deformities are frequent. In July 2017, the antisense oligonucleotide nusinersen (Spinraza) was approved for the recurrent lifelong intrathecal treatment of SMA in Europe. Lumbar punctures are challenging especially in SMA patients with severe spine deformities and after spine surgery. In the light of alternative SMA therapies that are available or are expected to be available soon and which are administered orally or via one-time infusion, an appraisal of the established therapy is significant. Discussion about which therapy is the best for each individual patient will have to include not only the safety and efficacy of data but also the application form and its burden for the patient and the health care system. Therefore, we analyzed our 3-year experiences and challenges with 478 lumbar puncture procedures in 61 pediatric SMA patients with and without spine deformities or instrumentation.

scholarly journals P.154 Subcutaneous intrathecal catheter and port implants for administration of Nusinersen in patients with Spinal Muscular Atrophy

2021 ◽  
Vol 48 (s3) ◽  
pp. S64-S64
Author(s):  
A Bokeris ◽  
D Mcneely ◽  
c restrepo ◽  
J Sheriko
Keyword(s):  
Spinal Cord ◽  
Spinal Muscular Atrophy ◽  
Motor Neurons ◽  
Muscular Atrophy ◽  
Medication Administration ◽  
Intrathecal Baclofen ◽  
Outpatient Setting ◽  
Intrathecal Catheter ◽  
Spine Deformities ◽  
Health Canada

Background: Until recently, no effective treatment was available for spinal muscular atrophy (SMA). In 2017, Health Canada approved intrathecal Nusinersen a medication that prevents degeneration of the motor neurons in the spinal cord. The administration is intrathecally most commonly via lumbar puncture (LP) to have a direct effect on the motor neurons of the spinal cord. Many older patients with SMA and concomitant spinal deformities present technical challenges to access the thecal sac. Different routes have been described for delivery of the medication whoever these techniques may require sedation, are associated with radiation exposure, and demand experience personnel. Methods: A new surgical technique has been proposed to overcome these obstacles by combining two Health Canada approved devices: 1) an intrathecal catheter designed for intrathecal baclofen pumps and 2) an implantable subcutaneous port designed for intravascular medication administration Results: We describe the technical nuances and outline the clinical outcomes of six patients with complex spine deformities who have undergone such an implant for administration of Nusinersen. Conclusions: We discuss the benefits of the procedure which includes: 1) administration in the outpatient setting without sedation, 2) avoidance of costly imaging and experienced personnel, and 3) placement of the catheter in the cervicothoracic junction.

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