Scoliosis and spinal muscular atrophy in the new world of medical therapy: providing lumbar access for intrathecal treatment in patients previously treated or undergoing spinal instrumentation and fusion

This study describes a new procedure for a safer and easier access for the intrathecal injection of the recently approved nusinersen therapy in spinal muscular atrophy. This therapy changed the natural history of the disease, but, to date, scoliosis surgery was an excluding criteria for nusinersen therapy. The bone mass, due to the posterior spinal fusion of the scoliosis surgery, prevents the needle for the nusinersen administration from intervertebral access. This is a single-center, single-surgeon case series descriptive study. A laminotomy at the L3–L4 level was performed to provide safer access for the intrathecal injection. The procedure was carried out during the scoliosis surgery in patients who underwent posterior spinal fusion (PSF) after the nusinersen therapy was introduced, whereas for those who underwent PSF earlier, a second procedure was necessary to perform a laminotomy. A fat grafting was used to prevent bone overgrowth in the laminotomy. Markers were applied as radiographic references for the intrathecal injection. Five patients were enrolled, four females and one male. The mean age of the patients was 11 years. Three patients underwent PSF before the introduction of the nusinersen therapy. Two patients underwent PSF after the nusinersen therapy was available. All of them underwent a laminotomy with a fat grafting at the L3–L4 laminotomy level and received nusinersen therapy without complications. The procedure described is simple and effective ...
Source: Journal of Pediatric Orthopaedics B - Category: Orthopaedics Tags: SPINE Source Type: research

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Conclusions: In SMA type 2 and 3 patients undergoing radiological imaging-assisted injections, the effective dose and DAP decreased during therapy with nusinersen. The mean effective dose in patients with spondylodesis was higher than that in patients without spondylodesis. Dosimetry should be monitored carefully in order to detect and prevent unnecessary radiation exposure.
Source: Frontiers in Neurology - Category: Neurology Source Type: research
In December 2016 and in June 2017, respectively, the FDA and EMA approved Nusinersen as the first treatment for SMA.Bambino Ges ù Hospital started to treat patients with the SMA type 1, from november 2016, as part of the expanded access programm (EAP), and from november 2017, after the commercial approval , the later onset forms. We recruited patients followed in the Neuromuscular Unit. We activated a multidisciplinary team composed of neurologists, pneumologists, anaesthesiologists, radiologists, physiotherapists and pharmacists, in order to prepare the drug, assess the motor functional abilities and monitor the ef...
Source: Neuromuscular Disorders - Category: Neurology Authors: Source Type: research
Nusinersen has to be repeatedly administered intrathecally in patients with spinal muscular atrophy (SMA). The development of scoliosis is a complication associated with these patients and may limit the administration of treatment. Describe our experience in the administration of intrathecal medication in patients with SMA. We propose a strategy that allows the intrathecal approach in patients with severe scoliosis. Prospective observational study. We collected demographic data related to the disease and the intrathecal administration procedure in patients with SMA treated with nusinersen from March 2018 to March 2019.
Source: Neuromuscular Disorders - Category: Neurology Authors: Source Type: research
Scoliosis is a highly prevalent feature of SMA and surgery is frequently required. Studies in small cohorts have reported a positive impact of spinal surgery on respiratory function, while limited data are available on motor function, weight gain, post-surgical skeletal pain and satisfaction. We retrospectively reviewed the notes of 33 patients (26 SMA II, 7 non-ambulant SMA III) who successfully underwent spinal fusion (25), magnetic (4) or traditional (4) growth rods followed by final growth spinal fusion (3) at Great Ormond Street Hospital-London.
Source: Neuromuscular Disorders - Category: Neurology Authors: Source Type: research
Meruna Bose, Shrutika Dilip Parab, Samidha M Patil, Nehal A Pandey, Gargi V Pednekar, Sarabjyot Singh SainiIndian Journal of Public Health 2019 63(3):254-257 The present study aimed to find out the effect of disease-related impairments on functional status in individuals with spinal muscular atrophy and identify perceived barriers to undergo physiotherapy. The cross-sectional observational study was conducted on 90 participants from January to March 2018 using validated patient-reported questionnaire via electronic mail, along with Fatigue Severity Scale and ACTIVLIM. Results revealed that difficulty in sitting was due to...
Source: Indian Journal of Public Health - Category: International Medicine & Public Health Authors: Source Type: research
Spinal Muscular Atrophy (SMA) is one of the most common neuromuscular disorders in childhood with an incidence of 1:10,000 [1] and the first genetic cause of infant mortality.
Source: Neuromuscular Disorders - Category: Neurology Authors: Source Type: research
CONCLUSIONS: Our initial experience has resulted in a protocol-driven approach based on simple or complex spinal anatomy. Patients with simple spines do not need preprocedural imaging or imaging-guided intrathecal nusinersen injections. In contrast, the complex spine subgroup requires preprocedural imaging for route planning and imaging guidance for therapy, with the primary approach being the transforaminal approach for intrathecal nusinersen injections.
Source: American Journal of Neuroradiology - Category: Radiology Authors: Tags: SPINE Source Type: research
Conclusion Ventilatory care for SMA type-1 is still based mainly on tracheostomy. This Chilean cohort of SMA patients had timely access to genetic diagnosis, ventilatory assistance, nutritional support, and scoliosis surgery. In this series, SMA type-1 is underrepresented, probably due to restrictions in access to early diagnosis and the high and early mortality rate.La Atrofia Muscular Espinal (AME) ha concitado mucha atenci ón en los últimos 2 años debido a la aprobación del primer tratamiento intratecal para esta enfermedad neurodegenerativa. América Latina necesita desarrollar la demo...
Source: Arquivos de Neuro-Psiquiatria - Category: Neurology Source Type: research
Abstract Nusinersen (Spinraza) is a US Food and Drug Administration-approved intrathecal medication for the treatment of spinal muscular atrophy (SMA). Adult patients with SMA often undergo thoracolumbar fusion to treat neurogenic scoliosis, preventing thecal access. The authors report a laminotomy technique and the ease of intrathecal access in three SMA patients with prior thoracolumbar fusions.Patients were positioned in the lateral decubitus position or prone. Lumbar laminotomy was performed below the conus, between the lateral longitudinal rods, to preserve mechanical stability. Fluoroscopy provided real-time...
Source: Journal of Neurosurgery.Spine - Category: Neurosurgery Authors: Tags: J Neurosurg Spine Source Type: research
Alcyone Lifesciences recently obtained Breakthrough Device Designation from the FDA for their novel implantable intrathecal bolus drug delivery catheter and port system, the ThecaFlex DRx System. The system is intended for use in conditions that requ...
Source: Medgadget - Category: Medical Devices Authors: Tags: Neurology Neurosurgery Pain Management Radiology Source Type: blogs
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