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190 Nusinersen treatment in adults with spinal-muscular-atrophy with complex-spinal anatomy at St. George’s Hospital, London
  1. Sathyajith Buddhika Ambawatte1,
  2. Sahil Chhabda2,
  3. Declan Johnson2,
  4. Derek Weidner1,
  5. Sherryl Chatfield1,
  6. Pamela Appleton1,
  7. Emma Matthews1,
  8. Niranjanan Nirmalananthan1,
  9. Clare Galtrey1
  1. 1Department of Neurology, Atkinson Morley Neurosciences Centre, St George’s Hospital
  2. 2Department of Neuroradiology, Atkinson Morley Neurosciences Centre, St George’s Hospital

Abstract

We describe nusinersen, the only licensed treatment for adults with SMA in the UK, administra- tion with Single-Plane-Fluoroscopic-Guided(SPFG), Bi-Plane-Fluoroscopic-Guided(BPFG), or Computerised-Tomography-guided(CTG).

We treated three patients with type-2-SMA (two with Severe-Scoliosis[SS], one with Posterior-Spinal- Fusion[PSF]) and four with type-3-SMA (two ambulant, one with Normal-Spine[NS] and one Mild-Lumbar- Spinal-Deformities[MLSD] and two non-ambulant; one each with PSF and MLSD) aged 17 to 57-years. All had homozygous exon-7 and 8 deletions. One had two copies, four had three copies, and the remainder had four copies of SMN-2-gene.

At baseline, forced-vital-capacity was between 38% to 153%. Ambulant patients were assessed with HFMSE(score 37-60), RHS(scores 31-67) and, 6MTM(range 43-492m), and non-ambulant patients were assessed with CHOP-ATTEND(score 17-38), RUL(2-33), 9-hole-peg-test and dynamometer as appropriate.

In type-2, patients with SS had a total of 6(20.7%) BPFG-injections. Only one had severe hypokalemia requiring ITU and treatment cessation. In one with PSF, BPFG-injection was unsuccessful.

In type-3, SPFG-injections were successful in all 19 attempts with MLSD or NS. One each was complicated by transient low-pressure symptoms and radicular pain. Three CTG-injections on a patient with PSF were uncomplicated. All with successful LPs(96.5%) did not drop out.

We found that nusinersen could be administered regardless of the spinal anatomy with interventional radiology.

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